6 monthly scan results

A couple of weeks ago I visited the Royal Marsden for my Summer 6 month scans – chest x-ray, ultrasound and boob squish.  Stupidly (and I should know better) I neglected to take some ibuprofen before my boob squish.  I was once again in extreme pain and so super envious of ladies who don’t have pain.  (How do some ladies go jogging without a secure bra?)  I suspected the mammographer had spotted something as she insisted on doing a couple of extra scans to ‘get in the sides’.

Next up was my ultrasound.  Whilst I was changing into my ‘everso sexy’ dressing gown, I heard the radiographer being called in to see my mammogram results.  Long story short she said she could see a small dot but suspected it is a cyst.

Today was my appointment with my consultant.  He has vast experience with phyllodes tumours as well as a wonderful disposition and honest, caring approach to me, the patient.  I’m so very grateful for such a fabulous consultant.

We talked about ‘the dot’.  We’re not sure it’s a cyst.  I’ve had no other cysts before now.  BUT rather than poke around and upset my breast tissue, we decided to leave it ‘just so’.  My consultant has asked that in six months we have a full set of scans and tests again (usually I have a mammogram once a year in the Summer).  He did, however say that if I had any concerns or worries at all, I was to contact him directly and an appointment would be found.  I’m happy with this.  I appreciate his experience and the open discussion.  I feel assured I’m in the best hands and care.

He also made sure we spoke about my auto-immune disease.  The drugs I had taken and the side affects I’m still left with (as well as the residual disease).

We spoke about my friend who’s currently undergoing surgeries following the discovery of new phyllodes tumours in her heart, liver and lung.  He wants me to forward details of the hospital and surgeon so he can learn more about the case and educate himself and his team.

We also spoke about a clinical trial that I’m trying to get set up in the UK for Phyllodes.  I’ll write more about it soon… hopefully with good news.  Once again, he wants me to keep him in the picture so that he can ensure he/the Royal Marsden are able to recruit for the trial.  Such positive news, we hope.

Finally we spoke about life.  His and mine.  He asked me how I was doing.  Was there anything else I was worried about.  Any other aches, pains or points of concern.

I feel totally looked after… and hopefully the dot will remain just that, a dot.

My Health Update

Well there’s good news and bad news…

The good news is that I had my 6 monthly ultrasound and chest x-ray and no sign of any lumps of mets from Phyllodes.  That’s the good news.

I also met my new consultant at the Royal Marsden.  Lovely chap who’s just returned from working in the US.  He understood the frustrations with a rare cancer diagnosis and I was delighted that he’d taken the time to read my notes and know about ME.

He kindly spent time with me chatting about how I’d been and asking if there was anything I was concerned about.  I told him that my boobs were still very painful (they always have been, but so much worse since Phyllodes, however probably nothing to be overly concerned about as I have such a good follow-up regimen).

I also showed him a rash I have had – I only showed him the rash on my wrists.  (The poor radiographer who had done my ultrasound a few weeks earlier had been utterly surprised by the rash on my boobs and armpits.  She looked genuinely shocked and horrified that I’d been waiting for several months to get a dermatology appointment…. more about that in the ‘bad news’ section.)  My consultant didn’t know what it could be but thought it may be auto-immune.  This then led me to ask him what he knew or suspected about a connection between Phyllodes and auto-immune illnesses.  His response **raised eyebrows** “that’s very interesting, why?”.  I told him that within our Facebook Phyllodes Support Group we had, and are still running, a poll asking members if they (or family members) had been ever been diagnosed with an auto-immune disease.  Although only 150odd people have responded there’s a big percentage that have a link.  Auto-immune disease is a spectrum of disease though and covers psoriasis to multiple sclerosis.

A good explanation of auto-immune disease is:

Autoimmune diseases are a large group of conditions. They include

Rheumatoid arthritis
Multiple sclerosis
Inflammatory bowel diseases
Skin conditions, such as psoriasis

If you have an autoimmune disease, your own immune system attacks your body tissues. Normally, our immune system protects our body against infections caused by bacteria, viruses and other parasites. It recognises when something foreign enters your body and can usually get rid of it before it causes you any harm. But if you have an autoimmune disease, your immune system can make mistakes. Your immune cells start to attack your own normal body cells.

I left the Royal Marsden feeling assured about my Phyllodes health but also that they are assisting me and others diagnosed with Phyllodes in researching and answering questions for our Phyllodes Support Group.  My consultant has subsequently emailed me to let me know he has chased the researcher who has a list of ‘basic’ Phyllodes questions to answer that I sent in a while ago.  The answers to these questions will allay many fears for newly diagnosed Phyllodes patients.  As it is so rare and there is little/no clinical research to rely on, I have suggested that they caveat any answers of concern with ‘in the experience of Royal Marsden’ or ‘to the best of our knowledge’ etc.  It would be good to have some answers on behalf of the UK sarcoma medical profession.  It is then aimed that the same questions will be put to medical professionals in the US, Australia and Asia.  All four sets of responses will then become available to new members of the Phyllodes Support Group.

My consultant also advised in his email that he’s asked the research team to look at any links between Phyllodes and auto-immune diseases.

The bad news…

…back to the rash.

At the end of October the soles of my feet started to really itch.  Initially there was nothing visible but soon there were small spots and dry patches.  I did wonder if I had fleas or bugs in the carpet.  I wish!  Next a sore angry rash appeared on my wrists, the palm of my left hand and both ankles.  I spent an age hoovering and rehoovering carpets, cleaning, dusting and generally convinced that it must be bugs.  Convinced that the spread to my hands and wrists must be because I’m scratching my feet.  I have been, as I’ve previously on my blog, very tired or fatigued for months/years but it had been getting worse. I know most of you see my facebook posts about my playing of tennis and going out… but you don’t get to see the posts that say I’m having an afternoon nap and didn’t wake up until midday and back in bed early or staying in most evenings.

Back to the rash.  By early November I could cope no more.  It sounded ridiculous but I was on the brink of tears trying to get a GP appointment asap for a ‘rash’.  I wasn’t sleeping well as the itching was so much worse at night.  My visual migraines returned and headaches for fun too.

My GP looked at the rash, said she didn’t know what it was and referred me to a dermatologist.  A few days later she called to say that the referral had been rejected and I needed to try a rash cream.  After a week of slathering myself in the cream, the rash had just spread and was as red and raw, if not more, than before the cream.  I booked another GP appointment.  This time she referred me again with a stronger note and saying the rash had now spread to my chest, armpits, groin, crook of my elbows and crook of my knee as well as my bellybutton… so basically anywhere that gets hot!

A few photos taken at the beginning of December:

LP - December

I waited… and waited… and chased up my appointment… and waited… and was told that my referral hadn’t worked in the system… had to chase my GP to do it again… and waited… and waited… and then called my GP again (in tears)… and waited… chased the referral service… who referred me back to my GP… who referred me back to the service… I cried… who then took pity on me and gave me a reference number, my secret NHS booking password and the number of the dermatology practice… I called them and was told the earliest appointment was over two months away… I booked it… put down the phone and cried again.

I know it was ‘just a rash’ but it F’ing hurt and believe it or not I was worried about the rate at which it was spreading.

I missed an ex-colleague’s funeral. Although the GP had said it wasn’t contagious, she didn’t know what it was and I couldn’t risk attending a funeral where other cancer patients may have compromised immune systems.   I’m sure Ally would have understood.

I was wanting friends, who’s daughter had been hit by a car in October and were ‘camping’ out in my local hospital at her bedside, to spend an evening with me so I could cook a homemade meal for them.  Again I was too scared that should this be contagious I’d make their situation worse just because I’d like to cook for them.  In the end I prepared a Friday night Indian takeaway meal of curry, dahl, naan, pilau rice, bhaji, poppadom, wine and beer.  The food prepped and cooked with me in latex gloves.  I also made some homemade soup for them for another night.  I did seem silly asking them to call round on the way home from the hospital to collect it without inviting them to stay.  I didn’t want to take the risk.  I’d never forgive my selfishness.

Apologies to anyone else I’ve not seen, rain-checked or not taken up some fun outing but I hope now you’ll understand why.

Just before Christmas I again called the GP and this time insisted on seeing my old GP at the practice, who’s been my GP since the 1980s.  He understood why I was so upset.  Looked at the spreading rash, he also said he didn’t believe it was contagious but didn’t know what it was.  He then wrote out a prescription that meant I had a drug that not only managed nighttime itching (generally eczema) but also had a sedative in it.  However he didn’t think it would cure the rash.  What a treat on 23rd December to have sleep!

Finally, 10 days ago, my appointment with a very kind dermatology specialist arrived.  I should have taken photos at this stage – definitely the worst time.  Diagnosis took just a few minutes.  Her opening line was “oooh I think I know what it is.  It’s very rare and you seem to have a severe case”.  (Oh joy another rare thing for me then!).  After consulting the computer and getting a second opinion from a colleague she told me that I’ve got an auto-immune disease, Lichen Planus.  So far my Lichen Planus (LP) is only external.

She did think that the LP may be caused by something else wrong in my body which has caused the LP to flare.  Blood tests ASAP.  Possible damaged liver or hepatitis.

I left with a prescription for some uber moisturiser and a really strong steroid cream.  She was concerned about giving me oral steroids as apparently what would be required for LP is super strong, has side affects and may be something that I have to continue to take.  So creams first.

I felt relieved to know what it was and had full confirmation that it’s not contagious.  But scared as to what it may be disguising.

As the itching was still horrendous (but being treated) my original GP also kindly did a new prescription for the sedative pills so I can sleep.

So it’s been a crazy time.  Lots of time slathering moisturiser and steroid creams on my body.  For the most part the rash is less angry, the blisters are rarer and for the most part I’m just scarred.   The worst areas are where it started and first spread ie the feet and wrists.  Some photos from earlier today:

Lichen PlanusI’m worried about some new itches and spots on my scalp and mouth ulcers and spots.  From what I’ve read these areas can only be treated orally and if it spreads on the scalp may lead to permanent alopecia.

I’ve got my next dermatology appointment at the end of this month.  Hopefully the creams will have sorted the external spots and the other itching is only in my mind!  I’ll then get the results of my blood tests too.

I’m also feeling much better than I did when first I heard the name Lichen Planus, as I’ve found a fabulous support group on Facebook.  It’s been reassuring to ‘chat’ with others who’ve been diagnosed.  For the most part they talk about it taking months/years to clear and then returning some years later.  I’ve also read about it being incurable but manageable.  It was also been reassuring to hear that there are others who have loss of balance, visual migraines and fatigue – seems this may be something to do with what I’ve been experiencing.

I have been truly fed up though.  I’m fed up of being ‘rare’.  I’m fed up of having stupid and many symptoms.  I’m fed up of worrying about going to see my GP.  I’m fed up with trying to pick my ‘worst’ symptoms to tell the GP about rather than bother them with them all in the limited appointment time.  Nothing seems to be specific or fit into the box.  I hate being a GP botherer however I’m wondering if maybe I should have been earlier ie before LP showed up as a rash.

Anyway enough for now… thought you should know.

BTW I did email my Phyllodes consultant to tell him I’ve been diagnosed with an auto-immune disease.  He’ll be adding LP to his research.

So there you go… the good, the bad and the ugly (well that’s evident from my photos!)

And also… it was results day!

This afternoon I met with my new consultant at the Royal Marsden to receive the results of my 6 monthly scans.

ALL CLEAR!  Bring it on… another 6 months under my belt.

I had a lovely chat with my new consultant.  What a wonderful Dr.  We were able to discuss a whole host of things that have been raised via the Phyllodes Support Group.  He’s assisting with putting some ‘advice sheets’ together for the Group and also investigating some links to other research I flagged today.  Interesting also as he has been working in the US for many years and understands the differences between the US and UK for Phyllodes.  Very useful insights.

He shook my hand and congratulated me when I told him I managed to get Phyllodes mentioned on TV today!

Mammogram time

You’ll remember I recently went for my six monthly scans which are all part of the follow up regimen following my diagnosis with malignant phyllodes and DCIS.  You may also remember that they had ‘forgotten’ to book my annual mammogram.  I’ve had some truly frustrating phone calls and several tears whilst trying to ‘remind’ the team what was agreed as a good regimen to monitor my health.

My appointment letter was received a few days ago and today I went into the RMH for my mammogram.  Unlike when I was there a few weeks ago, the clinic was virtually empty and I was seen immediately.  It’s crazy when I think about me having to chase and WANT a mammogram.  They’re usually painful and as my breasts get more lumpy with age and scar tissue they are becoming even more painful.  The mammographer was kind and tried to be as quick as possible but needed to take quite a few scans covering lots of tissue and angles.  Part of me wishes I didn’t need to do them at all as the pain is so bad (today was truly dreadful) but I also know that these are the best way to identify changes and see any Phyllodes appear.

Next stop is my appointment with the sarcoma team (and I presume a new consultant) for the results of the ultrasound, chest x-ray and now mammogram.  Only a few more sleepless nights…

Regular scans

You listen to me every 6 months talk about the follow up scans, the scanxiety and the results.

Well it’s that time again.  It explains why I’m more than a little antsy too.  No matter whether I’m thinking about it or not, somewhere in the depths of my brain is a little voice saying ‘maybe this time’.  With every twinge or pain that I get in my boobs (and believe me I get a lot) or my back, I wonder if it’s related.  I still get shooting pains which apparently is from the surgery and when I’ve previously discussed the pain with the consultants, I’m told that perhaps this is ‘normal’ for me.  But what IS normal?  How can I differentiate what would have been there anyway and what is because of the surgeries or phyllodes?

I digress but perhaps you might understand why I get anxious.  So last Friday I trotted along to the Royal Marsden.  Appointment card, request for a chest x-ray and letters making and changing my ultrasound appointment firmly in my hand.  I hadn’t received a mammogram appointment letter but presumed that this would be booked in when I got there and that the letter hadn’t yet arrived – previous experience of my receiving the appointment letter after the appointment had taken place fresh in mind!

The waiting room for ultrasound is full but we’re seen quickly.  I’m greeted by my usual lovely sonographer and introduced to a trainee sonographer who is due to qualify into the Marsden in a few months.  I mention that I’ve not received a mammogram appointment.  The trainee does the first exam.  It’s painful as she goes over lumps and bumps (I presume scar tissue) and on occasion stops to have a closer examination of areas.  It’s a little off-putting watching her face as she thinks she see something and hasn’t quite mastered the poker face yet – I guess that’s part of her training.  Then my usual sonographer takes over and they discuss their findings – “usual ‘oddness’, scar tissue and lobular neoplasia” but nothing unusual for me and apparently nothing to worry about.  I’m reminded that I should follow up for a mammogram appointment today.

Next stop chest x-ray.  This is such a quick procedure.  Stand in front of a black panel and breathe.  All done.  I was keen to get this x-ray today.  I’ve had a weird ‘catch’ in my breath and occasional wheezing of late… I’m hoping it relates to the virus I had a few months ago and nothing more sinister.

Now to head to the Outpatients Reception to see if I can find out about the missing mammogram.  I’m given a telephone number for a sarcoma CNS.  I call internally to discover the team are all in an MDT meeting.  Disappointed I head off and leave the Marsden.

I’ll get my results from my consultant at the next appointment which is in 5 weeks time.  It’s ridiculous that the results appointment is so long after the scans – more scanxiety!

Yesterday however I managed to speak to the sarcoma CNS.  She wasn’t sure why I hadn’t had a mammogram appointment and went away to check.  My consultant has now left the Marsden and apparently I’m now under a different consultant but my mammograms are under the breast cancer team.  WHY???

According to whomever she spoke with, I don’t need mammograms in my followup and that an ultrasound and chest x-ray is enough.  Aggggggh the agreed followup was for annual mammogram and six monthly ultrasounds and chest x-rays because Phyllodes doesn’t always show up on ultrasound until it’s grown bigger.  I explained this as calmly as I could… and then promptly burst into tears when I hung up the phone.

The Marsden are great.  They’re a centre for excellence for sarcoma.  BUT I’m waiting for an appointment that actually goes according to plan.  A time when I don’t have to run around the hospital chasing up something or booking in to a different location or calling up to find out what’s going on.  Truly I’m not sure but assume that within the hospital they have a multitude of non-communicating IT systems.  If only the realised the anxiety that these inefficiencies caused to their patients and carers.  I know I’m not the only this happens to as am often having my ear bashed in the waiting room listening to someone else’s anxiety.

So I guess I’ll have to wait for the sarcoma CNS to call me back.  And then wait for the mammogram appointment.  Or perhaps wait until my consultant appointment in July for the results of the ultrasound and chest x-ray.  Discuss with the consultant in July about my having a mammogram, get that booked in, turn up for another appointment and then have to have a further consultant appointment for the result.  Just as well I’m not in full-time employment with all these days off.

I wonder who my new consultant is?

Sarcoma SPAEN Conference – Day 3

PATIENT INVOLVEMENT IN CLINICAL RESEARCH
~~~~~~~~~~

Derek Stewart, OBE Associate Director for Involvement at National Institute for Health Research – Clinical Research Network, England
Public Involvement in clinical research
Improving Research – Involving patients, carers and the public

Whilst you have your voices, use them and use them to effect.”

I’m not a researcher, a doctor, etc but that shouldn’t stop us getting involved in research. We don’t need to know what they do but we need to sometimes tell them what we see. It is our job to bring the professionals back to earth. Never think you need knowledge, a university degree, particular skills… all of us can get involved in research.

From patient … to advocate, activist, ambassador and associate

www.throatcancerfoundation.org
Twitter
http://derek-online.blogspot.co.uk

  • Participation – taking part in a trial
  • Engagement – I get a newsletter, find out what’s going on, I’m asked to go to meetings.
  • In the middle – Patient Involvement. Where we actually hold hands with each other working together as clinicians, researchers and patients.

Why is it important for SPAEN?
Your Patient and Carer EXPERIENCE is of VALUE
Gold dust to researcher… they can work on it forever more. YOU have knowledge.

Did you know about cancer before you got it?

Your knowledge is fantastic.
You still have questions that are unanswered. How do I get those answers? That’s research.

Our bodies. When they ask us to get involved. It’s our bodies. We should have a say in that. It is our DNA what happens in it, what happens in genetics we should be able to speak about.

It’s our money £££$$$$. The charities are not the funders of research… it’s our money given to them directly or via taxes. Never forget it.

How can we make a difference?

Inform -> Form -> Influence

Inform – your experience can help. Tell them what happened but remember that sometimes researchers live in a little bubble and think that their idea is the best ever. They need the experience to understand… that’s us. We can work with them to learn and influence to improve the system and the policies.

Consultation -> Partnership -> Patient centred culture (bring fresh air to open the doors and windows)

Clinical Research Network in England. Supports the infrastructure in England Funding an integrated Clinical Research Network.

Supports Involvement of Patients, Carers and Public involvement
£3m put into it. 1,200 patients.

It is 70 days from accepting the trial to the first patient involvement.

A Movement for Change
Fight for Sarcoma, GIST and Desmoid. Bang that drum at all times.

  • But on the bigger picture. We’re all aiming to create better research. Based on our experience ad patient outcomes as a result.
  • We are all wanting to see simpler and more effective systems that get research on to the books.
  • We should all be making sure there is clearer access to research studies. (Hospitals claiming they’re a world leader in research but reception didn’t know… no leaflets etc… On the website ‘get involved in research’ it says how to donate body to research.
  • Improved recruitment. If we’ve not helped with the leaflets, what time, etc Think about what’s needed to improve the recruitment process.
  • Open access for results. We need ALL trial results to be published. They cannot hide the results.
  • Speedier application. Good research needs to be applied not sit on a shelf somewhere. The clinician (ALL) should be up to date and giving us the best and up to date information available based on the research. Always ask your consultant – ‘What’s the research on this?’ Is this the latest research?’
  • Better patient outcomes. Sometimes research is done for clever researchers but we need to ask what is the outcome and what is the difference that this is going to make.
  • Satisfactory experience. Do we ask people what their experience was like when they participated in trials?

Are we making a difference?

Action on Access booklet
Impact of Patient, Carer and Public Involvement in Cancer Research – NCIN leaflet.
Sarcoma UK’s Research Advisory Committee

  • Getting Involved
  • Understand the context. No point in going into a meeting with a researcher – are they looking at recruitment, funding or what?
  • Be clear about the goal and the purpose. Don’t expect an answer… Goal and Objective Settings. If you ask researchers you often get the answer – I’ve just been told to involve patients. Discuss.
  • What happened? What was the impact? If we don’t’ have a goal we won’t know if we’ve achieved it.
  • What was the benefit? (Benefit may be negative – feed it back to them..)

Context – purpose – impact and benefit.

What can you do? 20th May International Clinical Trials Day.
Let’s have you doing something. SPAEN members… promote it. Raise the profile of research.
Simon Denegri’s lay review

Involvement for Access
UK Clinical trials database 

When we first got involved the British Medical Journal wrote an article about patients getting involved in research about patients being aliens at the table.

View from the Top
Dame Sally Davies – “No matter how complicated the research, or how brilliant the researcher, patients and the public always offer unique, invaluable insights. Their advice when designing, implementing and evaluating research invariably makes studies more effective, more credible and often more cost efficient as well.

Signpost people to consent form, finding out information, exploring impact. Learn the language of trials. Do not give up…

When patients are present, we make a difference.

Q&A Session
Q – we are trying to involve Europe wide in clinical trials. One more problem is that European Clinical Trials Register is not suitable for giving information to patients as it’s not accurate or complete.
A – It’s why we need to be at the table and keep doing this. Danger in Europe from some drug companies fighting against open access. We mustn’t do solely fight for one cancer against another disease… we keep on.

Q – Inspiring presentation. Positive energy thank you. How do I respond to my Dr that once I have mentioned that I am participating in a patient group, they close all the doors and windows?
A – Only tip I can give you is try to find another Dr in the area, another clinician, another group…. Because when your Dr hears you are talking to them, they often think ‘I should be doing that too’. Often Derek asks ‘what are the problems you’re facing and how can I help?’. Ask them about why they get involved, what their passions are etc… build a rapport.

Q – We have the opportunity to create a patient group in bone sarcomas. Do you have the experience and wish to be involved?

Q – What would you say are the three main points to get involved?
A –
– Knock on the door.. if you can’t get through the door, go through the window.
– Never feel that you haven’t got a right to be there.
– Keep a simple diary, a few notes about what it felt like not knowing. We need to remember what it’s like for someone starting out. “I walked ½ way round the lake but was too tired, so walked all the way back”. As you know as you learn you improve… remember what it’s like at the start.

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Markus Wartenberg, SPAEN, Germany
Practical experiences and examples from patient organisations

Patients Involvement in Cancer Clinical Research.

When we look to our experience as patient groups.

  • Majority of patients. No or less knowledge about trials or experience. Perhaps about experiments not trials.
  • Very often we see we have no transparency about access. Where do we find Clinical trials in rare cancers?
  • Access and specifically in rare cancers. Access beyond borders in EU.
  • Access in the direction of losing patients who are not being referred to trials because doctors are losing the patients.
  • Practical issues – schedule? Distances? Costs for the patients.
  • Informed consent. Very often a medical legal wording that is too much to understand for patients.
  • Some patients are not aware they are on a clinical trial!
  • Is the right trial centre the real experts?
  • Sensitive topics like placebo trials in cancer.

Cancer patients 6-12% of cancer patients are participating in clinical trials.

  • Public image vs guinea pigs
  • Barriers to clinical trial accrual/recruitment

This leads to delay in knowledge/innovations, slower progress etc.

Trials and Endpoints?

  • Patients don’t care about ‘endpoints’
  • Their personal status/pathway/options -> important whether they ‘hope for/expect a
    • Cure
    • Stabilisation of the disease
    • Slow progressing disease
  • Important
    • Expert care in rare caners (centres of excellence)
    • Multi-model approach
    • Drugs – resection – radiation – clinical trials

Often it’s a ‘game’ against time…

Trials and Treatments?

  • Critical – how close are trials/trial results to daily practice?
  • Difficult:
    • Meaningful?
    • What drugs can deliver and what we need?
    • How to measure an innovation/a breakthrough?
    • What is value for money?
    • Target therapies – how to define progression?
    • Do we need more ‘me to drugs”?
  • Targeted therapies – Belittlement of the side effects
  • Quality of life is a very individual topic
  • Individual and ethical
  • Very critical – if treatments are/would be available but are not reimbursed.

Big issue at the moment is we need future options. We need better treatments for the future. But at the moment we also see that we are not able to get the best out the therapy. We need innovations but also need to be involved with the industry and experts to get the best out of the treatments at the moment.

Each patient is unique. There is no THE patient. The obvious differences, gender, age, ethnicity etc but so much more!

Patients who are going into clinical trials. They have an emotional overload as they enter the trials. Fear, shock, hope life death, new territory, confused, depression, anger, need support, options expectations.

Patients have physical aspects: Family commitments, work commitments, side effects, job career, fatigue etc.

Cognitive Aspects; regarding cancer but also for the trials. A new area to learn about.

What’s the role of patient organisations in clinical trials:

  • Information, knowledge, education, inclusion criteria.
  • Inform about background of clinical trials.
  • We can communicate as a patient organisation about available and upcoming trials. Work closely with the medical experts to find out, seek information and work ‘with’.
  • Making trial issues more understandable. Our job to interpret.
  • Supporting recruitment in specifically rare cancer sub-types.
  • Informed consent
  • Aspects of quality of life
  • Dissemination of the results… positive and negative results.
  • Involvement at the design stage. Work together on the issues for the future.

Dialogue with the Customers!

Patients are the customer… previously it was always the doctor… needs are changing

Partnership: Translate From/To The Patient/Customer
Physicians/Researchers <–> Patient Group <–> Researching Industry
<——————————————————————————->

Other players in the room – research organisations, regulators, HTA, payers etc.

Patients/Patient advocacy groups have a lot to offer

  • A common patient voice
  • Patient advocacy groups can argue in a way – experts/pharma can’t
  • Motivation to be involved
  • Needs overall
  • Expert patients
  • Etc
  • Etc

Clinical Trials’ offer some collaborate fields
Information access, design/quality and recruitment.

INVOLVE: Challenges

  • The general challenge is not specific enough for rarer cancers
  • Current trials system vs practice
  • Pharma/ experts often have no experience why and how.
  • EU trials directive – legal, confidentiality, regulatory aspects etc.
  • Involvement as early as possible. Not as troubleshooters but from the beginning.
  • Public media opinion. Collaboration is vital.
  • Patient organisations. How to identify patient organisations who are interested, prepared or willing to be involved in trials. Perhaps knowledge.
  • Patient organisations as a professional behaviours. Set rules with industry and behave!
  • We need to go back from talking more to action. Are there pilot projects to learn from.

INVOLVE: Solutions

  • How can we get the best out of our current therapies? Looking at them in practice and work out what to get out of them and improve.
  • New mindset: Patient centric research = Collaboration. Not lip service!
  • Awareness. Bring the topic to policy, healthcare, regulations and the public. Sometimes we need to seek it out other times people will come to us.
  • Working in initiative s for rarer cancers.
  • Working through a better approach for the informed consent EORTC.
  • Intense discussions with our industry colleagues. Getting feedback following completion of a clinical trial… this surely would improve the next one!
  • Training of patient organisations. Understanding cancer clinical research.
  • Medical advisory boards – we need to be involved in a collaborative way in each of these boards for the whole process and have the opportunity to talk to the regulators
  • Look at pilot projects.

~~~~~~~~~~

BASICS OF STRATEGIC PLANNING FOR PATIENT GROUPS

~~~~~~~~~~

Markus Wartenberg, SPAEN, German and Lindsey Bennister, Sarcoma UK
Lecture: The basics of strategic planning and practical experiences from a patient group

Strategic planning is an important part of business skills for patient groups. Idea of session is introduction to the topic…

Questions to consider:

  • Is your group thinking/planning strategically
  • Are you creating strategies from a share vision
  • DO you learn from the past and gather information from and about the external environment
  • Do you have immediate measurable goals in place
  • Are individuals or the boards (teams) accountable to plan
  • Is the organisation creative and flexible
  • Does it recognise, reward and institutionalise positive change.

What about shared vision?
Often people have a difference expectation and view of what is needed or required. You need to find a common shared vision.

A lot of areas in modern life are using ‘strategies’

  • marketing strategy
  • politics
  • elections
  • survival strategy
  • military
  • etc

What about patient organisations and non-profit organisations… makes sense to use strategies.

What is a strategy?

  • A method of plan chosen to bring about a desired future, such as achievement of a goal or solution to a problem.
  • The art and science of planning and marshalling resources for their most efficient and effective use. The team is derived from the Greek word of generalship or leading an army.

Your team! Your goal! Your proceeding? What Questions? How do you do it?

  • Setting part goal posts, markers to get there.
  • What materials do I need? What resources, tools, people?
  • Sharing responsibilities amongst the team members
  • Assuming responsibility as a team member.
  • What experience does the team have?
  • What conditions, environment etc influence your planning?

We have to think about the process and plan. Discuss and build up a strategy and planning process to get there.

What is Strategic Planning?
A systematic process of envisioning a desired future, and translating this vision into roadly defined goals or objectives and a sequence of steps to achieve them.
A systematic approach through which an organisation agrees on the priorities that are essential to its mission and responsive to its environment.
Future/Vision Mission
Goals
Analysis
Agreement
Strategy
Etc

What is strategic planning NOT…

  • A prediction for the future
  • A smooth, predictable, linear process
  • A substitute for judgement of leadership.

Hope is NOT a strategy.

Benefits of Strategic Planning.

  • Defines mission, vision and values
  • Establishes realistic coals, objectives and strategies
  • Ensures effective use of resources
  • Provides base to measure progress – need to assess value and feedback
  • Develops consensus on future direction
  • Builds stronger teams. A lot of people coming together motivated to do things but are they doing things in the right direction and utilising their skills and strengths.
  • Solves major problems.

Some thoughts for the process

  • Strategic planning involves choosing the highest priority achievements over the period of 3-5 years.
  • What is the strategy to achieve goals.
  • Strategic planning builds commitment to the vision.
  • Important for all levels of the organisation to be involved.
  • A strategic planning committee
  • Outside consultant/facilitator to facilitate conversations, capture external interviews, move the process along or to maybe draft the final plan.
  • In the end, Plan must be owned by the board/staff in order to move it successfully and strategically into the future….

First activity:

  • To assess the current situation and review the relevance of the mission and programs
  • Developing a ‘plan for the plan’ ie this means that if this is a big organisation how will you develop and implement the plan.
  • What is the outcome of the strategic process?
  • What is the time frame?
  • Strategic planning committees composition. Full board members, staff involvement and perhaps an external facilitator?
  • Thinking, collective experience, external and internal research.

Second activity:

  • Summarise the organisational history
  • What has been accomplished and what has NOT been accomplished between the plans.
  • Collecting data and information to make decision.

Book – strategic planning for non-profit orgs

The big 5 for strategic planning:

  • Mission
    • Why does the organisation exist?
    • What is the reason for being?
  • Vision
    • How will your community be changed and made better by what you have done?
    • What is your organisations vision of excellence
  • Value/Beliefs
    • What core principles should guide your organisation in the present and into the future?
  • Goals
    • SMART goals. These are outcome statements
    • Strategies
    • How are you going to meet the goals?

Remember if strategies get too detailed, you are moving away fro m strategic planning and into annual operational planning.

  • Swot – Analysis

What are strengths and weaknesses of organisation. Opportunities, threats etc facing us. This will help with key priorities and process.

Strengths and weaknesses

  • How is your organisation positioned?
  • What are the internal challenges?
  • What are the areas where the organisation shines?
  • Organisation reputation and history
  • Weaknesses such as capacity, funding, infrastructure etc

Questions:
Resources or strengths that help us to accomplish our mission or mandate or create value for our members (and their patients).
Internal weaknesses are deficiencies in resources or capabilities that hinder us to accomplish our mission or mandate or create value for our members (and their patients).

Opportunities and threats
These are the external factors to the organisation.
Are there new programme areas where you should be new, new funding, community collaborations, regulations, government, economy dependencies etc.

Questions:
External opportunities are primarily outside factors or situations that we can take advance of to better fulfil our mission or mandate or create.
External threats or challenges

SWOT and other tools
Identify the current environment and will inform your plan. You need to understand what is going on with your environment otherwise you will l have problems about reaching the goal and implementing the strategy.

Screen external relations. Who are the people the organisation are working with? Medical experts, Industry, payers, regulators etc.

What are the main stakeholders the organisation is dealing with. What are their roles, influences, interests, expectations? Are they target groups/audiences for future actions?

But also collection of available data? Do you know everything, have information on target groups, how do they behave etc. About environment and perhaps legal influencers in anyway. Do you know what the needs and expectation of your patients and carers really are?

To complete ‘your picture’ you can also initiate surveys, interviews focus groups etc.

All required to build your strategy for the future.

Putting Patients at the Centre of Healthcare – Strategic Plan 2010-2014 – iAPO

Measurable Activities…

~~~~~~~~~~

Lindsey Bennister, Sarcoma UK
Transforming the landscape for sarcoma
Sarcoma UK’s Goals and Strategy 2014-2020

If you don’t know what you’re going to do, you can’t know that you’ve made an impact

Lindsay summarised further the importance of planning and strategising that Markus had referred to in his presentation.

Sarcoma SPAEN Conference – Day 2

Dr Rachel Brindley (Clinical Psychologist) and Elaine Stewart (Cancer Support Specialist), London Maggie’s Cancer Centre
Morning Talk – What to do when treatment comes to an end? – Practical, emotional and psychological issues

What’s it like to be facing this diagnosis in your country?
What are the challenges?

  • Majority of people say they weren’t offered support.  But the search for information helped however it was their way of coping which might not be right for others.
  • Misunderstanding of what palliative and hospice care is.  Perhaps the communication isn’t good enough to understand what options are.  Discussions between Patient and Dr need to be better to understand the options but also to think about what is the job of an oncologist, palliative care, hospice etc.  Learning a new language.

What are the supports?
What are the main psychological issues that people with advanced disease face?

  • Living with uncertainty.  Realise the disease is progressing and recurrences but not knowing and living along side the disease whilst maintaining a quality of life.
  • Significant adjustment process that needs supporting.  Adjusting to the knowledge of advanced disease but finding your new place.
  • Fear of future of family if I die?

Ripple effect of cancer:
Medical – treatment effects, nutritional needs, physical
Quality of life – Sleep, leisure activity, shopping, socialising (eg meals)
Relationships – Changing roles, sexuality…
Difficult emotions – Low mood, anxiety, guilt, shame, anger (at disease, at family at God)
Self Image – Body image, self-esteem, confidence
Existential–Spiritual – Meaning of life, create a legacy, purpose…
Financial/Legal – Work status, holiday insurance…

Q – Psychological support to the physicians?
A – It’s also a burden on the clinicians and medical team.  Is this why they’re not best to share bad news.

Fears and questions about death and dying?
What are some of the fears?
What do people want to know?
What is it like talking about these?

Can be victims of complementary and alternative therapy?

Italian Dr shared a story – Shocked on his very first day of work in new role in LA, he was taken into patient’s room.  Colleague starts talking.. in next few hours/days explains respiratory and cardiac arrest is near.  We can resuscitate and will only delay a few days.  Shocked about how brutal they were with the patient.
In Italy the percentage of patients with this kind of information is only 1% or less that are given details about death and dying.  In Italy many patients don’t want to know they are going to die.  Physicians problems start much earlier than this point.

Very individual choice about knowing what is going on.. time to clear things, discuss with relatives, friends etc think about funeral and future of family.

Patient choice… has to be a sensitive and individual choice by patient.

When do you discuss?
Again very individual to find the time to discuss.  Discuss in part.   Listen to learn what the other person needs to hear.

Fears about death and dying
Fears about dying:
1.     Fear of the process of dying
2.     Fear of the consequences of death for loved ones
3.     Existential fear of death itself

Planning Ahead
What does this mean to you?
What would be helpful for you to think about?
What resources are available to help you to plan ahead?

  • Comment from member of the audience: for many in the UK we have never experienced death in the first 40 or so years of our life.   Death isn’t talked about.
  • Comment from another member of the audience: – Clinician discussions are often very different and difficult.  Training for clinicians and learning to signpost and be honest to refer.

Stages of Grief – Kubler-Ross
Denial -> Anger -> Bargaining -> (Anxiety) -> Depression -> Acceptance
Not a case of working through but cycling back.  Perhaps anxiety is missing on the diagram?
Perhaps should be ‘Not knowing’ at the beginning of diagram.
Some people may never get to the Acceptance phase but back and forth earlier on –ie they’re not able to accept death.
Perhaps include Guilt

Self-Care
Close involvement with people at end of life involves managing complex medical problems and working with high levels of emotional distress

Discussion
Consider the impact that working with people with advanced disease may have on you and other professionals.

1.     What signs would you want to be alerted to in
a.     Yourself?
b.     Others?
2.     What strategies do you feel would be helpful/important in preventing and managing these?

The heart must first pump blood to itself” – S.L. Shapiro
Support staff teams
Opportunities to debrief and reflect after difficult/distressing incidents
Opportunities to discuss what went well
Peer or individual supervision
Access to training, appropriate information
Time – to reflect on work, and for own needs
Feedback from senior management: positive, constructive
Balance between work and home life.

How might you be able to carry on some of these conversations/ideas when you get home?

~~~~~~~~~~

ADVOCACY MARKET PLACE SESSIONS
~~~~~~~~~~

Barbara Dore. Chair, Gist Support UK, SPAEN
How to Organise Patient Group Meetings

Think about and objectives for patient meeting.

Objectives

  • Information/Education
  • Exchange between patients
  • Support (you are not alone)
  • Patient empowerment
  • Relationships with
    • Experts who may speak at meetings
    • Industry experts.
    • If near clinic/training/university then perhaps invite team/students etc.
  • Reasons to donate (not just in terms of money.  Can also be their time).
  • Finding volunteers

Session formats

  • Lecture presentation
  • Workshops
  • Case studies
  • Panel discussions
  • New experiences (yoga…)
  • Background sessions psychological support etc)
  • Vary the pace of the meeting during the day/meeting
  • No more than 2 hours without a break.
  • Networking time!

Topics/Speakers

  • Consider the audience
    • Previous feedback
    • Newcomers/experienced
  • Variety of subjects – from the academic, technical and practical
  • Structure the day – after lunch spot needs interactive and care in planning.
  • Interaction keeps the audience engaged.
  • Brief the speaker as to content
    • Ask them if they want time cues
  • Reminder 1 week beforehand
  • Prepare some questions for Q&A
  • Write and thank speakers afterwards

Location

  • Budget
  • Site visit
  • Accessibility
    • Travel easy?
    • Disabled access
    • Car parking?
  • Layout – flow
  • Resources
    • A/v
    • Poster display
    • Flip charts
  • Registration desk.
    • Name labels,
    • programs
  • Signposting to meeting
  • Refreshments (every 2-2.5hrs)
  • Networking

Volunteers
Staff during the meeting

  • Registration Desk
  • Assistance to guests
  • Passing microphones around
  • Collecting evaluation form
  • Taking note from the meeting report
  • Distributing papers (Agenda etc)
  • Photographing
  • Getting Contact details
  • Circulating and meetings/ welcoming all participants
  • Getting photograph permission

Announcing the meeting

  • Inform and invite all attendees
  • Experts physicians nurses clinic
  • Invitation letters/leaflets posters
  • Invite by Email postal delivery, Facebook, twitter, website, email group
  • Press
  • Online calendar
  • Continuity slides.  Welcoming slide before presentation and also slide for lunch or welcome back to the afternoon session.

The Day

  • Meet and Greet
  • Network in Breaks
  • Feedback?
  • Think about next time.

Enjoy

~~~~~~~~~~

Roger Wilson and Claire Kelleher, Sarcoma UK
Patient Group Survey

Why do we do questionnaires?

  • need to get information
  • need to get opinions
  • experiences
  • often get new ideas

Need to be quite clear about objectives for survey.

Sarcoma UK is a patient led organisation.  Likes to involve patients in any initiative and involvement.   Part of Information Standard process is you must involve target participants ie patients.  Done by various means, feedback or through email, telephone, speak to people at support groups etc.

Clear objectives in mind when thinking about questionnaire.
Who do you want to contact?  Patients?  Carers

  • How will you contact them?
  • How are you going to find them?
  • How will they respond?
  • How will you record numbers in a meeting and is there any value?
  • Do you need documented or formal responses?

Once you’ve got data from responses what will you do with those replies?  Putting the data from the questionnaire into a spreadsheet for analysis is time-consuming.

  • How will you handle their replies and analyse the responses?
  • Once you have an analysis what are you going to do with it?

Got to have these things sorted in your mind before you start the survey process.  Otherwise it may be past its use by date by the time you use the analysis.

Claire – How we’ve used our surveys.  At British Sarcoma Group Conference in February we asked specifically about written information.
Asked the group in workshop (patients, clinicians and CNSs) what they thought of information (using Macmillan and Cancer Research UK’s written info). Most people said too complex and difficult to understand ie the lesson learnt use simplest terms that we can.
Negative feedback re glossaries – most people in the workshops found it difficult to flit back to glossary rather than explaining what a word means as they go along.

Survey about information and support – what info have you found? When did you receive it?  Etc… so Sarcoma UK can understand what patients are experiencing and better support patients as they should be.

Collate research data.  One reason why survey used?
Use methodologies and tools that have been validated academically.  So that the data can be taken seriously by professional bodies (wherever possible).

One of the tools that is most readily available is the EQ5D which looks at quality of life.

Is this form truly useful for cancer/sarcoma patients?

Currently working with Royal Marsden for sarcoma patients with advanced cancer.

Claire – Example of how patients’ information can be used within gynae-sarcomas.
There is a gap of information around this type of sarcoma.
People upon diagnosis not given information… there is very little.
Sarcoma UK are developing a booklet.
They have an information review panel (both patient review panel and a professional review panel).
All information goes through the process first with the professional panel looking for inaccuracies.
Next to the patient review panel – is it easy to understand? Is it the information they want to see?

One problem we had is that there isn’t that many people we have on the information review panel with this type of sarcoma.
We put a call out to the women we knew about to invite them to be involved in the review panel.
In January we’re holding a Webinar (with Maggies) to offer information and advice to ladies with gynae-sarcomas and also to gather information about what they would like to include in the information leaflet.

The information leaflet is then due to be published early next year and will be available on the Sarcoma UK website to download.

We do get it wrong as well…
IMG_4133
80 responses.
People had written all over the form and written on the back of the form.  Realised they were only scratching the surface with this survey.  Didn’t look at it well enough or in depth.
Hadn’t expected to also see hospital doctors lack of sarcoma information as well as GP (which had been expected).
If we’d been able to test the survey with 10 or 12 patients beforehand, Roger thinks a very different survey would have gone out.

Ended up writing a three page analysis of this survey which frankly was a load of waffle as the real conclusions had no data to support them as it was supplied anecdotally in the margins of the form rather than as answers to not very well worded questions.

Lesson – get objectives right before you start and test the questions to ensure you are asking the right questions in the right way to the right people.

Q – how would you deal with when a patient asks for privacy in answering a questionnaire.
A – Responses are anonymous.  Don’t ask for other personal information that might identify someone.
If someone doesn’t want to give information… they don’t have to.

~~~~~~~~~~

Ian Judson, The Royal Marsden Hospital UK
Marco Fiore, Istituto Nazionale Tumori Milan, Italy
Strategies for Metastasis in Sarcomas and Gist – Perspective of oncologist and surgeon

50% of people diagnosed with sarcoma will NEVER have another problem after primary treatment.

~~~~~~~~~~

Ian Judson – Sarcoma Unit, Royal Marsden
Management of metastatic disease – soft tissue sarcoma and GIST

What are the roles of chemotherapy for soft tissue sarcomas?

  • Palliation of advanced local or metastatic disease
  • Pre-operative treatment for large tumours?
  • Adjuvant chemo in large, high grade, extremity tumours?

Palliative treatment of advance disease.  Crudely if you lump all sarcoma together the median survival isn’t great. Probably about a year.

We did a trial in the EORTC combination v single agent – the 62012 trial

Slight improvement in progression free survival but no significant improvement in overall survival.

How can we use this data?

  • If objective is response – tumour shrinkage or remove specific symptom (pressure on nerve) then justified in using combination therapy.
  • If objective is palliation, then no real value in using combination therapy… single agent treatment (consequential if needed) is probably best.

What subtypes are particularly sensitive to chemotherapy?

  • Most sensitive subtypes appear to be
    • Synovial sarcoma
    • Myxoid/round cell liposarcoma
    • Uterine leiomysosarcoma
    • Other?
  • But N.B. – data are sparse on individual disease outcomes.

Other effective agents.  We use a combination of agents depending on the sarcoma type.  But we’re dealing with small numbers and difficult to assess.

Hormone sensitive sarcomas

  • Endometrial stromal sarcomas & some low grade ER/PR+ leiomyosarcomas, respond to oestrogen deprivation
  • In premonopausal women GnRH agonists or oophorectomy
  • In postmenopausal women – aromatase inhibitors
  • We usually use letrozole

When is chemo unhelpful?

What’s the future?

Molecular biological information that we can translate into new treatment.
In part:
Some tumours, types, mutations can be difficult to treat or apply a ‘rule’.

Some progress with translocations (chromosome swapping) ie synovial sarcoma translocations
Chromosomal amplifications – ie when it copies and becomes a driver for the sarcoma.

Median survival is improving as we discover more.   We have more drugs being used.  Differing regimens.

Conclusion
Range of treatments available for treating metastatic sarcoma.

PHYLLODES – COULD BE SARCOMA OR CARCINOMA (hence confusion about where it sits in medical teams)

~~~~~~~~~~

Marco Fiore, Instituto Nazionale Tumori Milan, Italy
Strategies for metastases in Sarcomas and GIST – A surgeon’s perspective

Looking at surgeries about how we run trials.
Conclusion should perhaps be that it is about patient choice and perhaps the studies should be addressed retrospectively in order to obtain patient randomisation.  Patient choice needs to be made with sufficient information to make an informed choice.

~~~~~~~~~~

AFTERNOON SESSION – SARCOMA TRACK

~~~~~~~~~~

Jean Yves Blay, Centre Leon Barard Lyon, France
Update on new and ongoing trials

There are many clinical trials….

  • You understood for clinical trials that we are dealing with a very well structured setting.  The majority of trials are done in multiple continents.
  • We are moving from a situation doing trials in all sarcoma sub-sets to a situation where we are targeting histologies.  This is changing the landscape an adds a level of complexity.  The end of the story will be not only focusing on sub-types but molecular sub-types within.  Results in small number of patients and work collectively globally.  It will not be possible to demonstrate the use of an agent

3 messages

  • Global approach
  • Histology Driven
  • Very important in know randomised trials, approval of the agent and prove that something is superior to another.

Bone sarcomas

  • Osteosarcoma
    • OS2006
      • Exploring bisphosphonates in a randomised setting
      • FSG, EORTC, others
    • Euramos 2?  5 years to do the first trial.  What next in Euramos 2 trial?
    • Country specific trials.
  • Ewing
    • Ewing 2012 (FP7)
      • Comparing VDC/IE to VIDE (comparing US regimen to the European regimen)
      • Exploring bisphosphonates
    • EuroEwing trial
      • Piloted by the German group.
  • PoC studies in Ewing/Osteo
    • Mifamurtide, linsitinib (EuroSARC WP6)
  • Chrondrosarcoma
    • PoC study of neoadjuvant mTOR inhibition
    • Hh inhibitors (vismodegib) negative
    • EuroSarc projects
  • Chordoma
  • Trials on mTOR, TKI (imatinib, sorafenib) completed

Soft tissue sarcomas

  • Neoadjuvant
    • Histology tailored treatment in EuroSARC (WP5)
      • ISG, GEIS, PSG, FR

Trying to address if we should give the same treatment.

  • Adjuvant chemotherapy
    • IRCI Uterine LMS 0 vs 4GT/4Doxo
      • US, EU (GOG, EORTC, UK)

Unsolved question in soft tissue sarcoma.  This is not the standard for all patients and we do not know who will or won’t benefit.
IRCI – this was the first trial in the US.. no treatment –v- combination treatment. It’s hoped it will answer the important question on this sub-type.

  • Surgery
    • Prospective trial of no treatment in desmoids (FR)

Phase 2 study of no treatment.  Often not mentioned.  This is where desmoids are not affecting other things.

  • (Neo)Adjuvant radiotherapy
    • Retroperitoneal sarcoma (EuroSARC WP4) exploring in selected localisation of disease.
    • Vortext tiral (UK)
    • SAR01 trial (FR) no radiotherapy with wide margin

Largest clinical trials are in the advanced phase:
Advanced phase – randomised trials
First line

  • Palifosfamide (closed Ziopharm sponsor)
  • TRS trial (Pharammar, completed) – Needs further investigation.
  • NCRI GT vs Doxo (UK) – Recruiting well – 220 out of 250 patients.  ASCO 2015
  • EorTC Trust Trial (closed for accrual).  Exploring agent in first line setting.
  • Dox +/-TH302 (ongoing, Threshold sponsor)
  • IRCI EORTC/UK in HGUS  High grade undifferentiated uterine sarcoma.
  • Taxol+/-Bevacizumab (FR, Angiosarcoma) – Close to completion of accrual.

Only a few trials are addressing in histological sub-types.

Subsequent lines

  • Trabectedine vs DTIC (JNJ, US, L-sarcomas)
  • Eribuline vs DTIC (Eisai, World, L-sarcomas)
  • Sunitinib vc cediranib (US NCI, ASPS)
  • Regorafenib vs – 0 (FR, all comers)
  • CDK4 inhibitor vs 0 in (US/World WD/DD LPS)
  • MV vsPazopanib (FR, Desmoids)
  • Cediranib vs BSC (UK ASPS)

Phase 1/11 trials

  • Subset specific (right question?)
  • Target specific (phase I/II)
  • Molecular immunotherapy.  Contrasting what was done in the 90s.  Watch in next 2 years to see new trials of this kind.
  • Passive immunotherapy.  Means antibody which is labelled with a nesotope and recognised.
  • Treated the driver target across histotypes

EORTC Network of Core Institutions
EORTC protocol 9010 (EudraCT number 2011-001988-52 NCT01524926)

Probably the future of what we are going to do in sarcoma.

Q&A Session
Q – Moving much more to molecular selection of patients in clinical trials.  Is the pathologist going to become more important in making the selection decisions.
A – The role of the pathologist is central.  This question is debated often.  How do we link pathologist to molecular biologist?  Research to routine is very challenging.

We need to bring up a budget wall with people talking to each other and exchanging information.  Challenging not sure.  If a pathologist is a real molecular biologist you’re fortunate!

~~~~~~~~~~
Short profiles of sarcoma subtypes
~~~~~~~~~~

Hans Keulen, Chordoma Foundation NL
Chordoma

Brief introduction into Chordoma and introduction to clinical trials
What is Chordoma?

  • Malignant tumour arising from the bone of the skull base and spine
  • It is a cancer and has a tenancy to be locally invasive and a tenancy to spread (metastasize)
  • It’s origin is traced to remnants of primitive embryonal cells called the “notochord”

Demographics

  • 5 % of the primary bone tumours are located in the spine
  • 8% of the spinal tumours are Chordomas
  • Incidence <0,7-1:1,000.000
  • Grows at skull base (Clivus, 35%), sacrum (50%) other spinal (15%)
  • Strikes people of all ages, most diagnosed in the 50s for sacral and 40s for other types
  • More frequent in men than women.

Phylum Chordata: Subphylum vertebrata

  • 550 million years ago Chordates emerged from the common ancestor
  • Presence of the notochord is the most prominent feature of the Phylum Chordata
  • Notochord is ectodermal and guides developed

Fate of the notochord

  • The notochord is essential for the creation of the embryo
  • Usually disappeared 10 weeks after gestation
  • The notochord should not be there at the latest after 10 years.
  • There are a lot of factors that can arrest the disappearance.

Recent discovery is that a certain gene called Brachyury (t-box gene).  Essential for development.  Absence is lethal.

Notochord and brachyury
Brachyury is over expressed in chordomas and many epithelial cancers.
Brachyury is expressed in chordomas but not in other bone and cartilage tumours.
Approximately 10% of chordoma patients it is familial.
Duplication of brachyrury gene has been observed in familial chrodomas.
When you inhibit brachyury in chordoma patients you will stop it growing.

Recent discovery from UCL – www.ncbi.nlm.nih.gov/pubmed/23064415

97% of chordoma patients harbor at least one allele of the common nonsynomymous SNP rs2305089 in the brachyury gene

Research is imperative.

Benign notochordal cell tumour (BNCT)
Should anything be done at this point?

Current treatment of chordoma
First line is still surgery.  Piece by piece or enbloc depending on location.
Sometimes stabilisation of the spine is required.
Radiation therapy is a high dose (usually 3x dose of breast cancer)  Specialised types, mostly proton beam or carbon ion.

Implications

  • Prognosis for chordoma patients Is not that good.
  • High rate of recurrence
  • A lot of people get a lot of mutilating surgeries.
  • Sacral chordoma usually in a wheelchair
  • Survival rate has gone up but sometimes at a cost of added mobility.

In the past we saw only 10% of mets.  However recently mets in sacral chordoma has recently been recorded that these are as high as 30%.  There is doubt if this might be due to surgery not being good enough – possibly caused by seeding.
No chemotherapy
20-20% cure rate.

Survival
Median was 6.29 years

Remarks – survival is increasing to 7-9 years, but with increased morbidity from surgery and radiation.

Chordoma vs Chondrosarcoma
Mistaken on routine histology
Epithelial v Mesenchymal origin
Immunohistochemistry
Cytokeratin
Brachyruy
Survival
Much better for chrondrosarcoma (gr1)
Misdiagnosis is less common now.

Recent developments
Using Carbon Ion instead of Proton radiation particularly in sacral chordomas night even replace surgery as first line treatment (ECCO 2013)

Radiation before or during surgery offers promising results with respect to recurrence and seeding.

Increasing number of target identified for trials with existing drugs.

Other trials….. not just drugs
e.g. Carbon Ion vs Proton Beam radiation – HIT Heidelberg

Carbon Ion vs Proton Beam radiation – HIT Heidelberg

About Chordoma Foundation
We are a very small patient group.  Worldwide we are less than Sarcoma UK!

Our mission is to improve, extend and ultimately save the lives of chordoma patients by:
Accelerating the development of more effective treatments
Helping patients across the world.

One in a million, on a mission – Chordoma Foundation

~~~~~~~~~~

Beatrice Seddon, UCL Hospital NHS Trust UK
Uterine Sarcoma

Gynaecological sarcomas – where are we in 2013?

Incidence of gynae sacomas
1985 – 2009 5950 gynae sarcomas diagnosed in the UK
9.2 case per million female population
285 cases diagnosed in 2009
Peak incidence 30-60 years

NCIN Gynae sarcoma report soon to be published.

Classification WHO 2003
Uterine Mesenchymal tumours *****
Smooth muscle tumours
Endometrial stromal tumours

Do not include malignant mixed Mullerian tumours

Location of tumours
85% of gynae sarcoma arise in the uterus
7% I the ovary
4% in the uterine malignancies

Sloan Kettering uterine leiomysosarcoma nomogram – overall survival probability prediction tools

Management of gynae sarcoma
Surgery – Most important component of treatment

~~~~~~~~~~

Marco Fiore
The interdisciplinary process of diagnosis in soft tissue sarcoma

MDT involved
Pathologist, medical Oncologist, thoracic surgeon
Web based National Rare tumours network, radiation oncologist
Surgical oncologist Radiologist

Sarcoma MDT
Many specialists have routine activity with MDT team.

Weekly routine meetings with the specialists, pathologic round, clinical round and MDT outpatients.

Biopsy
Any deep mass OR bigger than 5cm OR increasing in size

  • Hystologic exam (better than cytologic)
  • Think about possible surgical incision
  • Keep high-level of clinical suspicion
    • Be aware of usual misdiagnosis -> deep large ‘hematomas’ are virtually impossible if no trauma history and/or anticoagulant drugs.

Core-needle biopsy
Local clinic
Usually two samples.

Pathologist – new classifications WHO classification of soft tissue tumours

In common cancer they are white or black
Benign – no problem.
Malignant – diagnosis of cancer.

Within soft tissue transfer it is a scale of white, greys and black,
Benign, Intermediate aggressive tumour, (locally aggressive) or (rarely metastasizing)  Malignant.

Different decision for soft tissue sarcoma diagnosis.

Soft tissue sarcomas can be diagnosed anywhere in the body and surgery on sites can be very varied yet the tumour type could be the same histology.  Anatomic constraints.

First think the overall strategy:

  • Histology specific (and grade)
    • Combination of natural history
    • Different sensitivity to different drugs
    • Different radio-sensitivity
  • Site specific
    • Respectability
    • Reconstruction needs

What I can do + what the tumour can do = What I should do.

Please note, overall strategy should think in advance.

Sometimes it’s important for the surgeon to consider what to do rather than rush into it… difficult as most patients want surgery immediately but caution may in fact be best for the outcome.

Pushing tumour margins Or with infiltrative margins.

External lesions it may be better (angiosarcoma photo used) to have any chemo after surgery… that way the surgeon can see exactly where they need to perform surgery.

Functional outcome is an issue.  Needs planning to ensure that particularly with arms etc that reconstruction of nerves etc is also planned to provide patient with best outcome.

Cosmetic outcome can be problematic.  Such as on the head and neck.  Plastic surgeon should also be consulted as part of the planning.

INT – treatment criteria
Changing over time 1987-2007

  • Less amputations.  Decreased from the 1st to the 4th period (9% -> 3% -> 1% -> 1%)
  • Concurrent chemo—radiation therapy.  Preoperatively

Local recurrence was improved.  Survival did not change.  Functional outcome and quality of life did.

One-shot approach.  You need to think in advance for this strategy.

  • Re-excision: up to 50% of the cases referred to tertiary centres.
  • Impact on public health costs /reimbursement criteria??
  • Impact on extension of final surgery and functional outcome.
  • Psychological impact on patient and family (unexpected diagnosis: whoops! – delayed diagnosis – second opinion change diagnosis in 30%)
  • No prognostic impact.

Retroperitonal soft tissue sarcoma
Probability of finding via CT scan is low.

Biopsy remains the gold standard in diagnosing sarcoma!

~~~~~~~~~~

Julia Hill, Deputy National Programme Director, National Cancer Peer Review, UK
Peer review of quality of treatment, access to treatment and centres of excellence.

What is Peer Review?

  • Quality assurance programme based on National Guidance and National Standards.
  • For Sarcoma this wa the Improving Outcomes Guidance.
  • Guidance by nature is guidance and cannot specifically be measured.  Need to be benchmarked.
  • It’s not a statutory function but well supported in NHS.

Development of peer review

  • Developed initially around cancer services.  Broadening outside of cancer.
  • First reviews took place in 2001.  Been through many reviews and remodelling.
  • Number of independent reviews support the continuation with recommended support.

Aims of Peer Review

  • Providing safe services
  • Improving quality and effectiveness of care
  • Improving the patient and carer experience
  • Undertaking independent, fair reviews of services
  • Providing development and learning for all
  • Encouraging the dissemination of good practice.

Key principles of Peer Review

  • Clinically led
  • Consistent in delivery
  • Developmental
  • Focus on coordination within and across the organisation and pathway
  • Peer to peer.  Clinicians working in the area reviewing clinicians and teams.
  • Integration with other review systems.  Hopefully no duplicating information.
  • User/Carer involvement.

Benefits of the Peer Review Programme

  • Proven to be a catalyst for change.
  • Developmental programme
  • Provides director of services and information across the country
  • Identify any risks in the service by visiting and bringing to the attention we can get these involved quickly
  • We have a lot of clinicians involved in process (3-3,500 clinicians who review)
  • Rapid sharing
  • Provision of timely benchmarking data.

The Peer Review Methodology
Annual Self Assessment -> Internal Validation -> External verification -> Peer review visits.

Reviewing evidence

  • Quality measures.  Ask teams to provide evidence documents to keep workload minimal.  We ask for doctors that the team would be using in every day… work programme, operation policy, annual report.
  • Narrative report against key themes… for structure and function.  What membership of teams.  What’s the training.  Patient pathways and clinical guidelines.  Patient experience.  How do get patient feedback.
  • Clinical outcomes.  We are moving toward looking at clinical outcomes.

Development of the measures
National guidance
Expert Groups – nurses, allied health professionals, dieticians etc and also patients.
Consultation – get together to create a set of measures for the service.
Formal consultation – for approx 3 months.
Editing – meet again and edit etc.
Publication – measures reviewed on an annual basis to take into account changes in national guidelines.

Measures for Sarcoma published in August 2011NCAT Manual for Cancer Services – Sarcoma Measures
Cover aspects:

  • Sarcoma Advisory Group
  • Trust – inc Diagnostic Clinics
  • MDT – Bone and soft tissue.

What makes up a peer review visit:
Purpose:

  • Provide an opportunity to meet with members of a service to determine compliance with the quality measures.
  • Identify any broader issues relating to the delivery of a quality and safe service including a review of clinical indicators.
  • Provide a further external check on internal quality assurance processes.

Look at the wider picture of how the team functions against how they’re delivering against clinical indicators.

Who are Reviewers?
MDT – service users, clinicians, AHPs, Managers and commissioners…
Peers are people who have been trained and working in the same discipline as the people they are reviewing.

We don’t have reviewers reviewing the trust next door but are objective of the pathways.

Selection Criteria for a Peer Review Visit

  • We don’t visit all the centres but do a risk based target for the visit.  Are they meeting national guidance.
  • Were there any risks identified previously that have still not been involved.
  • We’re asked by organisations to visit.
  • Compliance against measures with lowest performance grouping… If teams are still not reaching 50% of measures then we need to go in and see what the problem is.
  • Concerns regarding the Internal Validation process.

2012/13 Peer Review Visits
12 Sarcoma Advisory Groups
145 Trusts, 19 Diagnostic teams
15 MDTs

Good Practice
Generally good provision for TYA Support for this patient group nationally
Good patient involvement overall and good examples of support
Good entry into clinical trials

Immediate risks
Inadequate referral population
Below 100 patients

Serious Concerns

  • Inadequate CNS provision
  • Lack of attendance at the SMDT by radiology and pathology
  • Lack of oncology capacity (non-surgical oncology)
  • Ambiguous/fragmented pathways (retroperitoneal and site specific)
  • Poor pathway/MDT governance / Data

Recurring Themes
Reiterate points before, particular issue was some Self Assessment Groups do not benefit from same support as more matter NSSGs

Clinical Outcomes
Clinical Indicators for sarcoma were introduced in April 2013:

  • % patients treated in Sarcoma centres
  • Caseload by Sarcoma centre
  • % patients receiving surgery
  • Readmission rates within 30 days of surgery
  • % patients with a recorded stage.

Working with NCIN to provide service profiles for each of our teams.  Ie Cancer Service Profiles for Breast cancer   Comprise of demographic data, specialist team, throughput, meeting times, practice, outcomes and recovery and Patient experience.

Outcomes of Peer Review

  • Confirm quality of services
  • Identify shortcomings and publish
  • Publish reports about quality of services
  • Timely information for commissioning
  • Validate information which is available to other stakeholders

My Cancer Treatment – is the website that patients can then see and compare local services and check what the results are for their local hospital etc.
Click on Find out more to see the narrative of the report.

We know that not only patients are looking at the website – commissioners are also doing so.

Sarcoma UK have prepared and posted on their website a summary of the Patient Experience Survey results

~~~~~~~

SPAEN Partnerships and Collaborations

Progress in Rare Cancer Care
Collaborations and Networks

EORTC and EURO/SARC
Winette van der Graaf, Chair EORTC STSG, Netherlands and Estelle Lecointe, SPAEN France

What drives us as a sarcoma specialist?
If you work on frequent cancers, do randomised trials.  If you work on rare cancers – find friends..

What is EORTC
Exists for 50 years.
Important institute in Europe.   Main aim is to collaborative academic research throughout Europe to improve the outcome for cancer patients.

There also groups that breaching these tumour groups such as elderly, quality of life, biomarkers etc

One family of sarcoma group.

Throughout Europe
Soft Tissue Bone Sarcoma Group

  • Throughout Europe
  • Randomised phase 2 an 3 studies
  • Interesting partner for Pharma.  Difficult in a rare disease to get pharma involved however with big databases they’re interested!
  • Big and relevant data bases
  • Gets support from EORTC,
  • Platform of discussing studies in such rare disease.  But also a platform of finding friends and collaborations.   We are all busy with a very rare disease for which there are not so many people available.
  • Collaborative spirit.

Organisation of STBSG

  • Board of group, period of 9 years
  • ExCo with charis of the subcommittees
  • 2 group’s meeting a year
  • Young investigators of the group.  Welcome new members interested in doing studies in sarcoma.

Group gets a scientific audit every 3 years.   So we are audited on the way we behave and want to see minutes and outcome and publications and all activities.   Proud to say the last audit took place this year and we ranked very high.  Always hope that you will have good advisors to make it even better.

Subcommittees

  • Local treatment – radiotherapy, surgery
  • Systemic treatment – chemo targeted agents
  • Pathology
  • Imaging.  Radiologist part of design of studies.
  • Quality of life since 2012
  • Public relations, starting from 2013
  • … pre-clinical group to look at what’s going on in other institutes (to be set up)

Collaboration with…

  • National groups in France, Germany, Spain, Italy, British, etc
  • Some members are members of World Sarcoma Network.  Number is increasing.
  • Pharmaceutical companies
  • EU grants and education (this needs to be extended)

Open studies…
Very few later stage open studies at the moment.    Working hard to get new studies active.

STRASS and CREATE studies.

New intiatives

  • Many database studies

Discussions about

  • Osteosarcoma French Study
  • Ewing sarcoma (FP7)
  • Liposarcoma CDK4 inhibitor
  • GIST: fist line, second line, third line
  • Elderly study: cyclophospharmide/prednisolone
  • Imaging studies on STRASS

World Sarcoma Network
This is a totally different institution created in 2009 by enthusiastic people in the sarcoma field.  Cooperative group gathering the main reference centres to stimulate rapid clinical drug development for sarcomas.
Enable clinical studies that could not be completed by the cooperative groups or at a national and continental level.  Where it needs a global level to discuss.

In Europe, in Australia Peter Mac, Ludwig Institute Australia

World Sarcoma Network the challenges

  • If you have no institute location and no funding how can you organise it.  Busy of thinking of a model of how to do this.  We have the international rare cancer initiative.
  • Very rare disease – unattractive for Pharma
  • ‘Sarcoma of the year’ 2013 gynae sarcomas.  If we are not more concrete what we are aiming at then for the outer world it will be a vague institute of nice people but no objective.
  • Biannual meetings during ASCO and CTOS

Examples of successful collaborations

  • Trials in GIST
  • EORTC – Italian and Australasian Sarcoma Group Centres in the US

With the input from all of you, there will be many more studies to come…

~~~~~~~~

EuroSARC

Jean Yves Blay, Centre Leon Barard Lyon, France
European Clinical Trials in Rare Sarcomas within an integrated translational trial network

How do we get funding for collaborative working?
Each network gets funding from different parts but how do we bring these together?

Where grants from EuroSARC came together.    Tools to enable this network of networks to move in the right direction.

Some times successful in some projects but other times on our own we may be unsuccessful.

We can build something on the basis of international grants.

GOAL – EuroSARC project – Academic clinical trials with a limited number of partners but inclusive for all networks and all groups.

Interaction between clinical research <-> Translational research <-> basic research

Objectives
To address major academic questions
Involve reference centres
Tod design structure and implement 9 innovative investigator driving clinical trials of different scales on a multinational level, evaluating novel

Conclusions

  • An important support for Ebased on previous work
  • Academic research only
  • Difficulties/problems
  • Adaption to the context/change of plans.

www.eurosarc.eu website

Website
1.     About sarcomas
2.     2 Eurosarc project
3.     Eurosarc clinical trials
4.     Patient and public info
5.     Utilities
6.     Members section

Follow up appointment

This morning I attended the breast cancer clinic for an appointment following my recent biopsy.  As in the previous posting the pathology indicated that the ‘dots’ weren’t anything to worry about.  However I felt that I needed to understand why they had ‘suddenly’ appeared (they weren’t on previous mammogram); what they really are; are they connected to the sporadic breast pain; and, of course, truly believe that they weren’t the start of something more sinister.

She explained that the dots were calcifications which are small spots of calcium salts.  Calcifications develop naturally as breast tissue ages however can also form if there’s been an infection in or injury to, the breast.  The concern though is that sometimes they  form because of other changes in the breast such as a cyst or fibroadenoma or as an early sign of breast cancer.   (Breast Cancer Care have a leaflet which explains in more detail – here)

Because of my history of malignant phyllodes (which initially biopsied as benign fibroadenoma) and of DCIS all of which were removed in 2009, there was a concern that these new dots may well be indicating something awry.  Fortunately the pathology and consultants believe that these are purely calcium salt spots ie nothing to worry about.  That said, they were keen to ensure that I continued with my regular check up regimen so that any changes will be picked up early.

Next screening scans are booked in for January 2014… Bubbles anyone?