Young patients denied second chance for life by NHS England

Blood Cancer – the issues around the highly complex topic of 2nd stem cell transplants.

Early in 2016 a MDS patient was denied a potentially life- saving 2nd bone marrow transplant despite showing a good chance of being cured by this 2nd treatment.

After 4 months of waiting, spent in and out of hospital, while NHS England turned down the treatment again, following an appeal, this patient contracted a severe infection and died.

He was only 39 years old – and a father of 3 young children.

End of 2016:

A mum with 2 young children is finding herself in a similar situation. She needs a 2nd transplant after a relapse of her blood cancer, Acute Myeloid Leukaemia.

The request for funding was denied just recently – meaning she has 2 choices: find the £100k herself to pay for the transplant or accept she may only have months left to live.

What is the background behind these appalling situations?

At the end of 2013, NHS England took the decision to stop funding allogeneic stem cell transplants (allo HSCT) for blood cancer patients who suffered a relapsed of their disease – after an initial successful transplant.
Expert clinicians requested a review of this decision, which did happen.

But in July 2016 NHS England came to a decision and stated they would no longer routinely fund 2nd allo HSCT’s – based on the evidence and success rates. These treatments were no longer deemed to be “cost-effective” for the current financial situation of the NHS.

It is true that overall, outcomes of 2nd transplants are not great – on average.
But transplants, types of blood cancer, sub-types, patients are very unique and these situations are rarely comparable.
Not only is the need for a 2nd HSCT rare, but not many patients would be so eager to undergo a 2nd transplant. Nor would haematologists be willing to perform one if there was not some good chance it would save someone’s life.
Clinicians and patients understand the issue of quantity versus quality of life.

Ever since 2013, haematologists deciding their patients would benefit from a 2nd HSCT, would have to make a compelling case for it – via an IFR (Individual Funding Request) which would then be submitted to an NHS England IFR Panel.
The IFR panel would then discuss whether the case was exceptional enough to agree the funding.

What does that mean in practice and in actual figures?¹

A bone marrow transplant costs about £100k per patient on average.
Between April 2013 and June 2016, only 19 requests for a 2 nd HSCT were made in all of England:

5 were declined by the IFR Screening team (the case was never seen by the IFR Panel)
5 were declined by the IFR panel
3 had “other outcome” (sic - as per NHS FOI answer)
6 were approved – this means just 2 cases per year. Total cost of approx £200k.

The 2 recent patients mentioned have been denied this chance – to save NHS England £200k.

It is crystal clear that the NHS cannot afford to treat everyone whatever the cost – so let’s set aside the
emotional and humanity argument.

It is the money side and management of NHS funds we take issue with.

NHS England refuses to consider, or key budget holders seem unable to take into account the cost of keeping these patients alive with supportive care – or palliative care.
The patient who died ended up costing the NHS closer to £250k – just to keep him alive for 5-6 months before his death – between frequent hospital stays, tons of antibiotics, expensive new medication, frequent complex blood and platelet transfusions.

A saving of £100k = an NHS bill of £250k + a death
This is not tax payer’s money well spent

Furthermore – we have uncovered that despite NHS England’s explanations, IFR cases are not assessed by transplant specialists, but by a mix of “trained” IFR staff, pharmacists and public health consultants or another random types of physicians.
There is no process forcing IFR staff to submit highly complex transplant IFR’s to relevant experts in that field.
There is an ongoing NHS England consultation on this very topic closing on 15/01/17.

Anthony Nolan, together with the Blood Cancer Alliance community and leading experts, published an open letter to the Secretary of State for Health (Times, July 2016) and led a significant campaign – but to no avail:

www.anthonynolan.org/news/2016/07/14/our-letter-following-nhs-england-announcement-funding-second-transplants

“We believe this is simply wrong. If a stem cell transplant is the best option for a patient, and a donor can be found, we believe the treatment should be available. We know that there are many people alive today, leading fulfilling and active lives with their families, because they had a second transplant.”
(Anthony Nolan campaign extract).

¹ Figures obtained through Freedom of Information Act enquiries – and from medical notes shared by the family

Below are the full stories of both families. 

This makes for very difficult reading.

We urge the government to review this nonsensical and inhumane situation.

There are cases where patients do stand a good chance to have a successful  2nd transplant – but these decisions must be made by expert haematologists – not public health physicians, let alone non-clinical staff – however well trained.

The savings are a false economy, as evidenced in this case.
Supportive care ends up costing twice as much, patients occupy badly needed hospital beds, families lose a bread-winner and government a tax-payer.

This makes no sense.

Sasha Jones – who urgently needs a transplant – but NHS England says NO

Dec 2016

I am a fun loving 34 year old nail artist from Greenwich, London. I am the wife of my childhood sweetheart Lloyd Williams and
Mother to our 13 year old daughter Katia and 8 year old Son Lj Williams. I am fighting for a second bone marrow transplant. I am
Sasha Jones (Williams) and I deserve to be alive just like you.

After a routine blood test on the morning of March 11 th 2015 due to me feeling unwell, I was sadly given the devastating news that
the tests suggested I was suffering from Acute Myeloid Leukaemia and was admitted within hours.

On Sunday 15 th March I started my chemotherapy treatment and a bone marrow aspirate revealed that the only chance I had of keeping the disease at bay was to have a bone marrow transplant. So the search begun to find a donor and my treatment commenced.

After my first round of chemo it was revealed the amazing news that I was in complete remission and had 3 donor matches!
3 rounds of chemo over a 5 month period later and I was admitted to Kings College Hospital for my graft. On 26th August 2016 and after 1 week of high intensity chemotherapy treatment I was given the bone marrow transplant I so desperately needed in the hope my leukaemia would stay in remission.

After 18 days in hospital, lots of blood treatment, pain management and rest I was relieved to seem well enough to go home to my husband and children. However, this was short lived when I was re-admitted 3 days later after Kings discovering the activation of a virus passed on through my new donor. I then spent another grueling 14 days in hospital trying to get rid of the reactivated virus only to be released home again to start my recovery.

But, yet again after another 2 days at home I was crucifying readmitted once more for yet another 14 days due to another virus called BK 2000 causing haemorrhagic cystitis. This left me with months and months of grumbling bladder pain (which I still have too this very day every time I urinate) and incontinence which made the recovery process a very uncomfortable, embarrassing and painful process.

By the beginning of October I returned home and managed to then stay home with medication and 2 visits a week to Kings College Hospital to monitor my progress. On top of all this my donor and I did not have the same blood type which also caused problems up until as recent as August 2016 when my blood changed to a type A+ from my original O+.

During 2016 I visited the hospital on a weekly basis and it started to look like everything was going right.
My recovery was long and hard but I never let it get me down, I had to do it for the children!
I could see the light at the end of the tunnel with my 1 year bone marrow milestone approaching on 26th August 2016!
Sadly my happiness was short lived...

On Tuesday 9th August 2016 after a routine blood test again I got the devastating news that my new bone marrow was now producing leukaemia cells and that the transplant had failed.
This was confirmed by a bone marrow aspirate result on August 23rd 2016, I had relapsed, the dreaded cancer had returned and I needed to start my battle all over again beginning with chemotherapy followed by an infusion of donor cells once in remission.
I was to be put forward for new clinical trials using donor cells and a vaccine to give me any hope of surviving without a second transplant as this option had been stripped from patients due to NHS cut backs. NHS England had announced that it would not routinely fund second stem cell transplants for patients with blood cancer or blood disorders who have relapsed.

I was then hit by the news that not only was NHS England denying patients these life-saving 2nd transplants, but that also my donor was no longer available to offer an infusion of cells or to assist with the completion of the trial.

I have therefore been left with no alternative than to have a 2nd bone marrow transplant and I’m currently waiting on a decision from NHS England as to whether they are going to provide me with the funding I so desperately need to save my life.
Given that they cannot offer me an alternative end to my treatment as my donor has been removed from the register due to circumstances unbeknownst to me, it is believed that under these exceptional circumstances (seeing as my life itself isn’t important enough) that I SHOULD be entitled to another bone marrow transplant to give me a chance of fighting the disease and beating it with a better matched donor that I have waiting for me right now.

I am young, in the prime of my life, have everything going for me and everything to live for, not to mention my 2 children who deserve the right to grow up with their mother. They have also the right to the love, support, care and stability that only a mother’s love can give.
We as a society and nation try only too hard every day to promote and push - only to take that chance away from 2 children for a mere £120k by not handing their mum a life line that they have such easy access to......

Another 2 children’s lives do not need to be destroyed, cancer already takes so many of us.
Why are we now giving up on us....
When did we stop trying to save lives over money but at the same time we put money into research to save lives that you then later let die...

#fingercrossedforsasha  #rideforsasha
https://www.gofundme.com/rvg4m54m

MDS UK update: Sasha has been refused the 2nd transplant by NHS England.

Her private fundraising has reached about £75k now – still not quite enough for the procedure. It is impossible to say how long she could wait – any infections could be fatal – as it was for the other patient. Do not let this happen to her and her family. Please share her story and sign the petitions below.

Family of Gavin – RIP

This is a summary of events – from diagnosis in 2012 – through to Gavin’s passing on the 6th May 2016.
Written by Lisa, his wife.

Nov 2012: Gavin gets his MDS diagnosis. Devastated and confused we are told he will need a bone marrow transplant in the new year.

June 2013: Gavin receives his first transplant and is home in little over 2 weeks. We are all elated and pray this is the new beginning for us all.

Oct 2013: Gavin is taken back into Kings for an infection that is destroying his red blood cells. Extremely ill his life is quite literally in the balance. But he fights it and is home and well within 3 weeks.
In between the above and September 2014 Gavin has top up transfusions of red blood cells and platelets (but rarely at this point).
The doctors are pleased and allow us all to travel to the USA for a month’s holiday.
Gavin was the healthiest he had been in a long time and we had a wonderful time.

Sept 2014: Gavin becomes unwell. A Bone Marrow Biopsy suggests that he may have relapsed.
A course of Chemotherapy is advised. We are all devastated but remain positive.
Gavin's condition is very up and down between September and December and he is taken into Kings before Christmas with another infection.
The Rollercoaster that is MDS is truly devastating to us all.

2015: Not great - highs and lows, lots of infections and stays at Kings and more Chemo, that had to be stopped.

June 2015: Gavin is needing regular red blood cell and platelets transfusions. A planned holiday to Menorca is cancelled due to Gavin's condition. We head to Devon for 3 weeks, but the holiday is full of interruptions as Gavin needs regular transfusions at Plymouth Hospital. He remains positive though, but we are dreading the thought that another transplant would be needed.

November 2015: We receive the devastating news that Gavin will require another transplant. His condition at this point is very up and down with infections putting him back into Kings at regular intervals, again, in the run up to Christmas. At this point he is also living his life going backwards and forwards for transfusions at our local hospital twice a week. This is becoming very draining on him and us as a family. But we remain positive.

Feb 2016: We receive the devastating news that Gavin's funding for transplant has been refused.
How? Why?
We arrange a meeting with our MP Nicholas Soames. He is very sympathetic and agrees to bring up Gavin's case with parliament.
Ironically that evening an ambulance is called as Gavin is taken ill with another infection.
We vow to raise the money, but are advised that another application for transplant will be put forward.
We feel like we are not doing anything to get this ball rolling - this is his life and we are desperate.Gavin's condition at this point is up and down, and he is struggling to remain positive.

April 2016:  After a wonderful day at the Isle of Wight, Gavin gets rushed back into hospital, East Surrey, our local hospital. It's the start of the 3 day doctor’s strike. We are all scared / devastated that this is happening again. On arrival there, we are told he will get the best care . How wrong were we. Gavin waits 8 hours for a simple bag of blood, he is climbing the walls in pain.
The doctors diagnose Pleurisy. He is put onto morphine for the pain. Gavin stays there for 3 days, and is not put onto the Kings Care plan until that 3rd day (when the doctors return back to work). At our total insistence as a family we get him transferred to Kings.
Dr Raj advises he needs this transplant but he needs to get well first. But he won't be leaving without it (hope again).
Gavin's condition deteriorates. He has a Lung Wash to clear the infection, the worst thing I've had to sit and listen too, I just wanted to run in and make them stop.
He improves a little and seems brighter. The morphine though has made him confused about things (very upsetting for myself and the children). It has also made him very constipated and blocked.
He can barely eat or drink.
It just feels like we are going 1 step forward 10 steps back.
The children just want their fun loving Daddy home and I my husband.

1st May 2016: Gavin gets told that there is nothing more they can do... we both can't believe it and won't believe it.
We put ourselves in a bubble and choose to ignore. Go to Costa and talk about anything but THAT.

2nd May 2016: Take the children to see him, very upsetting. He could barely be around them for no more than 15 mins.
Not like Gavin at all. He begs me not to leave that night.

Tuesday 3rd May 2016: I'm met by the palliative team at Kings. There is nothing more that can be done but to make him comfortable. I feel like my whole world has been blown up in front of me. My children, how do I tell them? We call all our family and friends. There is a constant flow of people for days.

Wednesday 4th May 2016: The children arrive and I tell them the news. They all react differently. Lots of tears, screaming the disbelief. I can safely say it was the hardest thing ever, but actually worst was to come.
We spend days and nights sitting and talking to him. The girls are amazing with him. Nursing him, holding his hand, cuddling him. Quite amazing for a 11 and 9 year old.

Friday 6th May 2016: My world is changed forever, our world. Gavin passes at 6.09am the exact time he was born on 17th Sept 1976. My mum brings the children up, I tell them there's another star in the sky.
They are destroyed - I am destroyed.

This should NEVER have happened. My gorgeous loving husband who had a zest for life and wanted to live was gone.
Myself and the children, 8 months on, are still devastated.
We receive regular counselling to get us through our grief, the pain and trying to understand WHY ?
The NHS failed Gavin when he needed it most...

To the UK Government

Fund Second Stem Cell Transplants. Only 7 days left!

Give Sasha a Chance!

Add your signature to Sacha's page on Change.org

A free, publicly funded NHS

The NHS must never be privatised

MDS UK Note:

We are regularly in touch with the family – but Lisa and the children are still understandably struggling heavily with the loss of Gavin – and therefore all enquiries should be directed to MDS UK. Thank you for your understanding.
We also sincerely thank the whole extended family, their friends, colleagues, employer for their ongoing support, participation, fundraising and awareness raising of MDS.
We have the deepest of admiration for Gavin’s two daughters especially, who have displayed an incredible courage, maturity and strength over the last 3 years.
They have spoken up about MDS, in school assembly, with friends, their athletics club, written to the press, participated in MDS UK activities, swam and baked for fundraisers, filmed an amazing awareness clip with their little brother – and even cut their hair off to make wigs for children with cancer.

There is hardly anyone they have not engaged around them- and still do so.

Please look up all of the above on our website www.mdspatientsupport.org.uk
We are immensely proud of them-and will continue to fight this issue in memory of Gavin – and for his family.
Their wish now is simply that no family should ever need to go through what they are experiencing – this is why they agreed to share these details now.

Gavin's Family Video to Raise Awareness on MDS


How to involve patients in their own care

Support patients to manage their condition better

Evidence tells us that when patients are actively involved in their own care, treatment and support can improve outcomes and experience for patients.

How to get involved

Everyone involved in care should advocate for the experience, voice and presence of patients, carers and the public to make a real difference to research in the NHS in order to improve health and well-being.

We all know importance of maintaining a healthy lifestyle through exercise and a healthy diet, but what else can be done?

Help and get actively involved in the work of the NIHR, the National Institute for Health and Research

In their website you'll find out about research that has taken place, you can take part in a free online course and contribute with questions and ideas for research.

The National Health Service (NHS) states:

The NHS is there to improve our health and wellbeing, supporting us to keep mentally and physically well, to get better when we are ill and, when we cannot fully recover, to stay as well as we can to the end of our lives. It works at the limits of science – bringing the highest levels of human knowledge and skill to save lives and improve health. It touches our lives at times of basic human need, when care and compassion are what matter most.

The NHS is founded on a common set of principles and values that bind together the communities and people it serves – patients and public – and the staff who work for it.

The Constitution Handbook adds:

Research is a core part of the NHS. Research enables the NHS to improve the current and future health of the people it serves. The NHS will do all it can to ensure that patients, from every part of England, are made aware of research that is of particular relevance to them.

Going the Extra Mile

The NIHR in England in their Going the Extra Mile report provides the strategic vision and direction for our work in engaging and actively involving patients, carers and the public in all aspects of research. It sets the following goals:

By 2025 we expect all people using health and social care, and increasing numbers of the public, to be aware of and choosing to contribute to research by:
- Identifying future research priorities and research questions
- Informing the design and development of innovations
- Participating in research studies
- Advocating for the adoption and implementation of research in the NHS

Both health workers and patients should ensure that they contribute to INVOLVE and ensure that information held on their website about where they work or receive care is accurate.

What's happening elsewhere?

We do need to consider what is happening elsewhere by exploring, for example: the European Patients‘ Academy on Therapeutic Innovation (EUPATI), the Irish Platform for Patient Organisations, Science and industry (IPPOSI). Further afield there is the Canadian Institutes for Health Research and their Strategy for Patient Orientated Research. You may also choose to look at the developments of Involving People in Research in Western Australia and the Patient Centred Outcomes Research Institute (PCORI) in the USA. These cover many of the leading economies and we risk deepening the health inequalities unless we also look at what is happening in there countries.

The great work of charities

There are many charities who focus on health research and others who carry out research themselves. Many are now actively involving patients and the public in the research that they fund. This ranges from priority setting, improving study design and sitting on funding decision making panels.

The Shared Learning Group on Involvement brings together a number of people working in charities doing patient, public involvement and engagement. They also have a ‘research sub-group’. Parkinson's recently produced an excellent guide for their researchers on patient and public involvement. The Association of Medical Research Charities (AMRC) is a further good source of information.

The role of Universities

There is a significant amount of public involvement taking place across all academic institutions. This is partly driven by the Research Excellence Framework which requires research work to have ‘reach’ and ‘significance’. It is worth finding out about the Academic Health Sciences Network (AHSN) and the local Collaborations for Leadership in Applied Health Research and Care (CLAHRC)

Take a look at current MDS clinical trials


What is CAR T cell therapy? Learn about how this immunotherapy works

CAR T cell therapy aims to boost the immune system to attack tumor cells

Cell therapies, sometimes called “living therapies,” are an especially promising and rapidly growing area of cancer research. One approach that’s been pioneered by Memorial Sloan Kettering researchers, led by investigator Michel Sadelain, is called CAR T cell immunotherapy. This type of targeted immunotherapy aims to boost the immune system by giving immune cells the information they need to better recognize tumor cells as foreign and attack them.

How does it work?

The technique involves filtering white blood cells called T cells from a patient’s blood and introducing a new gene into those cells. A disabled virus called a vector is used to carry the gene inside the T cells and insert it into the cells’ genomes.

The gene programs the T cells to make a chimeric antigen receptor (CAR), which enables them to recognize a specific protein that’s present in cancer cells. The CAR T cells are then grown in the laboratory and infused back into the patient, where they seek out and destroy the cancer.

CAR T cell therapy is currently being evaluated in the clinic at MSK for certain types of leukemia and lymphoma. In this approach, T cells are genetically engineered to recognize a protein called CD19, which is found on the surface of blood cells called B cells. In the largest study reported so far, for adult patients with B cell acute lymphoblastic leukemia — a rapidly progressing form of blood cancer — a report published by MSK researchers last year found that 88 percent of patients responded to the therapy. In late 2014, the US Food and Drug Administration granted MSK Breakthrough Therapy Designation for its CD19 CAR therapy.

The science behind it

  • A chimeric antigen receptor (CAR) helps T cells identify tumors.
  • These T cells then recognize tumors as foreign and attack them.
  • CAR T cell therapy is being used to treat leukemia and other cancers.

Take a look at current MDS clinical trials


The European MDS Registry: learning about the ‘real’ MDS patients

Research FOR Patients
For an informed and empowered opinion
and an improved consultation
Have you made your clinical paper accessible yet?

What is a clinical Registry?

In order to develop new drugs to treat MDS, clinical trials are mandatory, typically testing the new drug against the treatment that is considered to be the standard at that time. However patients in clinical trials are often different to those that we see every week in our clinics because the clinical trial will try to treat a group of patients who seem quite similar to each other and that means leaving out the patients with other diseases in addition to their MDS, or those too infirm to travel to and from the trial centre for example.

The best way to study a typical group of MDS patients without any restrictions is to set up a Registry. This involves obtaining consent from patients to collect information about their MDS and the treatments that they receive at their usual clinic or Day Case Unit visits. They may be asked to complete some questionnaires and sometimes to volunteer occasional extra blood samples but essentially the idea is simple; to systematically capture the usual care of all MDS patients over time into one large database.

European MDS Registry

The European MDS Registry (EUMDS) started on April 1st 2008, recording information from patients with ‘low-risk’ MDS in 10 European countries. Now the Registry has expanded to include 145 individual hospitals in 17 countries. The Registry has gathered information on more than 2000 low-risk MDS patients. 

Patients start in the registry within 3 months from their diagnosis and are followed for their lifetime with information recorded every 6 months. This information includes any treatment that they received, how they perceive their quality of life and giving a small ‘serum’ blood sample. Patients in the registry now have information from an average of four 6-monthly visits recorded. 

The UK has a prominent role in the project. We have registered 327 UK patients so far, the second highest number registered per country after France. The UK hospitals that are participating in order of number of patients recruited are:

Leeds, Aberdeen, Blackpool, Worcester, Airedale, Northampton, Mid Yorkshire, Bradford, Bournemouth, Harrogate, Truro, York, Oxford, Cambridge, Nottingham, Newcastle, Glasgow, Huddersfield, Manchester Christie, Birmingham Queen Elizabeth.

We have collected half of all of the blood ‘serum’ samples for the entire project, which are being used for interesting research studies (701 UK samples out of a total of 1211 samples analysed in EUMDS). Our patients have completed the quality of life questionnaires with a high completion rate (298/327 patients; better than most European countries). The UK is playing a prominent role in the organisational and strategic aspects of the EUMDS programme, which is led overall by Professor Theo de Witte from The Netherlands. Professor David Bowen leads the UK arm and is also Co-Chair of the Steering Committee for the EUMDS programme. The University of York Health Sciences Unit (Dr. Alex Smith and colleagues, https://www.york.ac.uk/healthsciences/research/ecsg/) hosts the database, provides the informational technology support and analyses the data.

We are building a picture of how low-risk MDS is cared for generally in Europe and also in different countries with some interesting patterns emerging. As well as the general information collected and analysed, there are more detailed research projects evolving within the registry, for example looking at the patients who have received blood transfusions, studying those that have received a blood stimulating drug like erythropoietin (EPO), or the possible importance (or not) of overloading with iron for patients receiving blood transfusions.

We are also studying the bone marrow samples using modern diagnostic tests like next-generation sequencing [NGS] which gives us more detail about the different biology of each patient’s type of MDS. We will then be able to look at how this new detailed information could help to predict how patients will fare generally (for example life expectancy/survival and the chances of MDS changing to more aggressive leukaemia) and maybe better predict who might respond to which treatments.

We are now moving onto the next phase of the programme, including higher-risk MDS patients in the registry and engaging new sponsors and new funding streams.

We are deeply grateful to those patients who are helping with this important research initiative.

MDS-RIGHT: a platform for research, for patients, and for other stakeholders

Although technically it maybe an ‘offshoot’ of EUMDS, the European Union funded 5-year MDS-RIGHT project is a wide ranging, ambitious programme with the potential to lead to an integrated European network for:

  1. MDS research
  2. patients
  3. influencing important stakeholders such as the regulatory agencies, the payers (Department of Health in UK) and policymakers.

Almost all key opinion leaders for MDS are centrally involved in MDS-RIGHT giving this prestigious programme high credibility and huge potential. Again the University of York is an important partner with Health Economics (cost effectiveness, led by Professor Manca, https://www.york.ac.uk/che/staff/research/andrea-manca) now added to the goals.

MDS-Europe

The European MDS Registry is supported by an unrestricted educational grant from Novartis Oncology Europe.
MDS-RIGHT is funded by the European Union (a programme called Horizon 2020 research and innovation under grant agreement No. 634789).
Key websites:
https://mds-europe.eu
http://www.eumds.org

MDS UK – Note to patients

MDS UK Note: Interested in taking part as a patient?
If you are newly diagnosed – please ask your haematologist about it.
Show them this article and state you’d like to volunteer your blood and biopsy samples – and contribute to this important research.

For any further information, please contact MDS UK:
Email: Mds-uk@mds-foundation.org or Tel: 020 733 7558

Please quote the following information if you wish to use our ResearchFORPatients article:

Source: www.mdspatientsupport.org.uk / ResearchFORpatients

Original reference paper:
Registry Nov 2016 Author: David Bowen, Honorary Professor of Myeloid Leukaemia Studies and Consultant Haematologist, St James's Institute of Oncology, Leeds


Clinical data on new medicines is now open to patients and researchers

Researchers and patients can access thousands of pages submitted by pharmaceutical companies

As of the 20th of October, the European Medicines Agency (EMA) gives open access to clinical reports for new medicines authorised in the European Union.
For every new medicine, citizens, including researchers and academics, will be able to directly access thousands of pages from clinical reports submitted by pharmaceutical companies. Clinical reports give information on the methods used and results of clinical trials conducted on medicines.
Vytenis Andriukaitis, European Commissioner for Health and Food Safety, said

"Transparency is an essential component in clinical research. Its outcome – whether positive or negative – should be made publicly available."

Learning from the experience of others

With EMA’s proactive approach to providing access to the data, patients and healthcare professionals will be able to find out more information about the data underpinning the approval of medicines they are taking or prescribing.

It will also facilitate the independent re-analysis of data by academics and researchers after a medicine has been approved. This will increase scientific knowledge, and potentially further inform regulatory decision making in the future.

Increased transparency will also benefit innovation. The shared knowledge about a medicine helps developers learn from the experience of others and can lead to more efficient medicine development programmes by reducing duplication of research and de-risking some new developments.

Protecting personal data

This will be a learning curve for the Agency and all its stakeholders, as they start to apply the policy for the first time.

While the policy gives an unprecedented proactive access to clinical data, it also demands the highest standard of protection of patients’ personal data. The process will evolve over time as more experience is gained and may lead to adaptations of EMA’s guidance.

Take a look at current MDS clinical trials


Help the NHS and Leukaemia CARE improve their services

Care and Treatment for Blood Cancer: Patient Survey

Leukaemia Care is asking patients to fill in a new questionnaire about their care and treatment for blood cancer. Its purpose is to provide information, which will help the NHS and Leukaemia CARE to monitor and improve the quality of health services for future patients with blood cancer.

Leukaemia CARE is interested in finding out more about your experience of treatment for blood cancer, in order to ensure that the services provided meet patient needs and to help focus campaigning efforts on areas where NHS care for blood cancer could be improved.

Taking part in this survey is voluntary. Published reports will not contain any personal details.

Leukaemia Care Survey

Who should complete the questionnaire?

The questions should be answered by you, as the person who has been treated for a blood cancer. If you need help to complete the questionnaire, the answers should be given from your point of view – not the point of view of the person who is helping.

If you have any queries about the questionnaire, please call the FREEPHONE helpline number on 0800 783 1775

How Can You Help?

Please complete the questionnaire and return it in the FREEPOST envelope. No stamp is needed. The questionnaire should take around 20 minutes to complete.

Or if you prefer you can complete the questionnaire online at: www.myonlinesurvey.co.uk/LE16ANON

Who is organising the survey?
The survey is being carried out by an experienced health research company, Quality Health, on behalf of Leukaemia CARE, who will produce public reports which will include the anonymous survey findings. These reports will be available to view on the Leukaemia CARE website in early 2017.

At no point will your name and address be linked to your responses for this survey. Your responses will only be used to provide information about the quality of services that blood cancer patients have experienced.

Need further information?

If you would like more information about the survey, protection of your data, or have questions on how to complete the questionnaire, you can call:
Quality Health’s FREEPHONE helpline on 0800 783 1775
The line is open between 9am and 5pm, Monday to Friday and there is an answerphone at all other times where you can leave a message.

Please take part in the MDS UK Survey as well


Stored Blood is as Good as Fresh Blood Reveals Study

Fresher is not better

It's been long thought that when blood transfusions are needed, it may be best to use the freshest blood, but McMaster University researchers have led a large international study proving that it is not so.

The study of almost 31,500 patients at six hospitals in four countries showed that having a transfusion with the freshest blood did not reduce the proportion of patients who died in hospital. The McMaster study was published in the New England Journal of Medicine.

"It's been a contentious issue, but our study finally puts an end to the question about whether stored blood could be harmful and fresher blood would be better," said Nancy Heddle, lead author and a professor emeritus of medicine for McMaster's Michael G. DeGroote School of Medicine. She is also the research director of the McMaster Centre for Transfusion Research.

"Our study provides strong evidence that transfusion of fresh blood does not improve patient outcomes, and this should reassure clinicians that fresher is not better."

She added that the results are also good news for blood suppliers worldwide as having a supply of stored blood helps to ensure that blood is available when a patient needs it.

The 31,497 adult patients studied were at hospitals in Australia, Canada, Israel and the U.S. The mortality rate was 9.1 per cent with people receiving the freshest blood, and 8.7 per cent among those receiving the oldest blood. There was no significant difference when looking at the patients' blood type, diagnosis, hospital or country.

Blood can now be stored up to 42 days before transfusion

John Eikelboom, a co-principal investigator of the study and professor of medicine of the Michael G. DeGroote School of Medicine, said more than 40 studies published earlier have failed to adequately answer the question about whether the freshest blood was best.

"Blood transfusions are a common medical intervention," he said. "Advances in blood storage now allow blood to be stored up to 42 days before transfusion and the usual practice is to use up the blood that has been in storage the longest. But, because there are biochemical, structural and functional changes in the blood during storage, there had been concerns about the use of 'older' blood.

"This study reassures us that aging is not bad - even for blood."

The study was funded by the Canadian Institutes of Health Research, Canadian Blood Services and Health Canada.

Article: Effect of Short-Term vs. Long-Term Blood Storage on Mortality after Transfusion, Nancy M. Heddle, M.Sc., et al., New England Journal of Medicine, doi: 10.1056/NEJMoa1609014, published 24 October 2016.


CAMELLIA

  1. Investigational drugs: Anti-CD47 monoclonal antibody, Hu5F9-G4 (target therapy that blocks the growth of cancer cells)
  2. For what level of MDS severity risk:
  3. What subtype of MDS: Patients with Acute Myeloid Leukemia that has returned after a period of improvement (relapsed) or has not responded to previous treatment (refractory).

Read More


Further cuts for stem cell transplants – for yet another rare blood cancer

Telegraph article from Saturday 13th of August– with further details about NHS England funding issues.

Patients with WM (Waldenstroem) are denied live-saving stem cell transplant treatment

You may have read or heard about the recent legal case involving a HIV patient advocacy group, NHS England and local commissioning groups. The outcome has been that NHS England is being legally forced to fund a new anti HIV treatment called Prep. NHS England is intending to appeal against the court decision – as it claims that NHS England does not have the funds.

As a further step, NHS England has announced that, as a consequence, they have no choice but to stop funding several other treatments for different groups of patients. Amongst the disease groups affected by this funding cut are patients with WM (Waldenstroem - a rare blood cancer) who require a stem cell transplant.

In support of our colleagues with WM, the Blood Cancer Alliance has issued the strongest objection regarding the way this issue is being handled by NHS England.

The letter, signed by Bloodwise, MDS UK and Leukaemia Care among others, reads:

Dear Sir

We are dismayed with the announcement last week that due to the High Court ruling on the commissioning of PrEP, NHS England has announced that access to nine treatments for other diseases that had previously been approved for NHS use are now on hold.

The example of one treatment, stem cell transplantation for patients with rare blood cancer Waldenström’s Macroglobulinaemia (WM), is particularly stark. Transplants for WM patients have been carried out successfully on the NHS for the past 10 years, and with this option now being withheld indefinitely pending the High Court appeal, patients will not have access to this last chance for survival. The withholding of these treatments has led to huge uncertainty and confusion for patients and clinicians.

There appears to be no valid or transparent process for why access to these treatments has been suspended by NHS England. For WM patients, this comes after the removal of the treatment bortezomib from the Cancer Drugs Fund last year, one of several blood cancer indications delisted for NHS use during 2015.

In suspending access to these treatments due to the legal ruling, NHS England is pitting one population of patients against another, which is hugely unfair to patients on all sides.

We demand that NHS England reinstates access to these immediately, and a long term, sustainable solution is found so all patients can continue to access the treatments they need at a cost the NHS can afford.

ACLT

Basil Skyers Myeloma Foundation

Bloodwise

CLL Support Association

Leukaemia Care

Lymphoma Association

MDS UK

Waldenström’s Macroglobulinaemia UK

And more background about this topic in the recent Daily Mail article here


Blood vs Bone marrow: New technologies can reduce the need for bone marrow biopsies

Research FOR Patients
For an informed and empowered opinion
and an improved consultation
Have you made your clinical paper accessible yet?

Bone Marrow Biopsies: a less painful alternative for routine check-ups

Until now, bone marrow sampling has been the primary technique for routine follow-up checks on MDS patients after initial diagnosis. The bone marrow is the heart of the disease and reveals important clues, for example, about whether a patient is responding to therapy or whether the disease is stable or worsening (progression).

During the procedure, which can be uncomfortable, an aspiration from the patient's marrow is taken, and specific blood cells derived from the bone marrow are analysed, allowing clinicians to monitor the ongoing disease status of a patient.

More specifically, clinicians may look for the presence of particular genetic mutations within the cells, what the DNA chromosomes physically looks like (a technique broadly called cytogenetics) and the shape of certain bone marrow blood cells (morphology).

However, although necessary, bone marrow biopsies have many downsides. Most notably the stress and physical discomfort to the patient, for which some patients require sedation. It is an invasive procedure which therefore always carries a risk of infection. This risk also increases in elderly patients, or those with a low or very low neutrophil (white blood cell) count. This makes frequent sampling problematic which means patients may not be followed as closely as clinicians would like. Overall, for many patients, regular biopsies are yet another 'painful' and inconvenient aspect of living with MDS.

Peripheral Blood Sample: An alternative to biopsy

 

An easier alternative to a biopsy would be a peripheral blood (PB) sample (i.e. the blood already circulating in the body, which is produced in the bone marrow).

Until recently, it had not been conclusively shown in a large scale study that PB could be used to obtain similar results as a bone marrow biopsy. Also, the commonly used testing technique, called metaphase cytogenetics, does not work very well for PB samples. Therefore until now, there has been little momentum in adopting a PB sampling as standard practice.

However recent research by a group at Kings College London and the Hospital may change that (A M Mohamedali et al). Their research has demonstrated that peripheral blood samples are an equally accurate and reliable source for monitoring the genetic mutations in bone marrow derived blood cells, and hence for monitoring the disease status of a patient (please see below for full publication details).

The research group looked for the presence of various genetic abnormalities known to be frequently associated with MDS in both bone marrow samples and PB samples, and compared the results against each other.

In order to do this, they used two specific testing methods which do work for PB samples. The first is a technique called SNP – Array karyotyping (a method used to identify changes to the number of DNA strands in a cell, a feature commonly observed in MDS). The second technique used was next-generation sequencing technology (NGS) to look at over 20 genes known to harbour mutations in up to 80% of MDS patients. They found that if a gene mutation or changes to the number of DNA strands could be detected in a bone marrow biopsy sample, it could also be detected in the PB sample of the same patient. Overall, they found that the same results could be obtained for both bone marrow biopsy and PB samples using these techniques (there was a 98% concordance in results, which is extremely high).

These are very promising results which demonstrated proof of concept that PB can be used as a substitute for bone marrow biopsies. The authors of the publication recommend the use of PB for follow-up checks and believe that PB sampling has many distinct benefits over bone marrow sampling.

The most obvious being the fact that the method is less invasive and virtually pain free, with little or no risk of infection. This allows for more frequent check-ups which in turn enables closer disease monitoring for better outcomes. The procedure is also quicker and easier to perform than a biopsy, and as no sedation is required, patients are also able to leave immediately with no recovery time required.

Aside from patient benefits there are also important advantages for hospitals too. The procedure is easier and quicker to carry out than a bone marrow biopsy, therefore does not require specialist staff and cuts down on procedure time. In some cases it may even free up hospital bed time and offer cost savings.

Additionally, once the PB sample is taken, it can be analysed relatively easily using the two testing techniques described by the research group. Both the SNP – Array karyotyping and 21st century sequencing techniques were semi-automated, reliable and provided robust results, making it attractive for hospital diagnostic labs to implement.

Although a bone marrow biopsy will always be essential for initial diagnosis, finding easier, less painful, yet still accurate and reliable ways to monitor MDS patients represents a major improvement. PB sampling could spare a large population of patients the need for repeat bone marrow biopsies, making the burden of their disease a little lighter, and allowing clinicians to follow patients more closely through more regular checks.

MDS UK – Note to patients

If you are not yet offered the choice of peripheral blood (PB) sampling during routine check-ups and would like more information about its use, please contact MDS UK. This is a fairly recent technology, therefore if your haematologist has not yet started using it please hand a copy of this article to him/her. We would be happy to provide more information directly to you and/or your haematology consultant.

MDS UK – Note to haematologists

Further details about this technology can be found via: Research paper in Leukemia.

Please quote the following information if you wish to use our ResearchFORPatients article:

Source: www.mdspatientsupport.org.uk / ResearchFORpatients
Author: MDSUK/Stephanie Brett email: mds-uk@mds-foundation.org twitter: @mds_uk

Original reference paper:
A M Mohamedali, J Gaken, M Ahmed, F Malik, A E Smith, SBest, S Mian, T Gaymes, R Ireland, A G Kulasekararaj, G J Mufti, High concordance of genomic and cytogenetic aberrations between peripheral blood and bone marrow in myelodysplastic syndrome (MDS)
Leukemia. 2015 Sep;29(9):1928-38

Clinician and Researchers Quotes

This research has provided us with the very important information that the genetic abnormalities found in the bone marrow of MDS patients are also detected in the blood. We already know that many patients acquire new genetic abnormalities during disease progression and it would therefore be possible to monitor for this on a blood sample. At present the main limiting factors for adopting this approach are the cost of these technologies as well as the complexity of analysing the data produced. The price will however fall over time and we will continue to simplify the data analysis process meaning that this has real potential for the future management of MDS patients. Unfortunately I don’t think this will replace the need for a bone marrow biopsy as this remains critical in confirming disease progression however it may allow us to detect changes early and determine when this procedure should be performed. Further research will be needed to find out if this can improve the overall management and outcome in MDS patients.

Dr Catherine Cargo, Consultant in Clinical Haematology, Haematological Malignancy Diagnostic Service (HMDS), Leeds Teaching Hospitals NHS Trust

From a clinical perspective, this study is the first of its kind to demonstrate the potential use of 21st century technologies in improving the management and treatment of human diseases, especially in a disease like MDS where the majority of the patients are of old age (> 70 years). This study has clearly shown that the genetic analysis that is usually performed on bone marrow biopsy can also be reliably done on peripheral blood, thus potentially eliminating the need for repeated painful and expensive bone marrow aspirations for disease monitoring. That being said, further larger studies involving multiple centres are needed to verify these results before being introduced into routine clinical practice. Although there are challenges that need to be addressed including the cost and the data management as well as interpretation of the results, however, this technological advancement has great potential for the clinical management of MDS patients and will also help in early intervention where disease progression is suspected.

Syed Mian, PhD, Research Associate (one of the authors of this research paper) Department of Haemato-Oncology, King’s College London

Currently only a handful of specialist laboratories are equipped to perform SNP-Array karyotyping or next generation sequencing mutation analysis in MDS. The number of centres tends to be small because these types of analysis are highly specialised, require the use of expensive, dedicated equipment and require highly skilled and experienced staff. These laboratories tend to be within specialised Haematological Malignancy Diagnostic Centers such as the service in Leeds Teaching Hospitals NHS Trust and my laboratory within King’s College Hospital London. The cost of these investigations is relatively high, however the amount of genetic information obtained using these methods is much greater and results in improved certainty of diagnosis. Some of these genetic findings are also useful for informing clinicians and patients about the likely course of the disease and can also influence treatment options in a way that the conventional methods may not. Here at King’s College Hospital we have been performing this next generation sequencing mutation analysis and SNP-array karyotyping in MDS for several years. We have performed analysis on hundreds of samples and these analyses are now available as diagnostic tests. Access to these analyses make replacement of some bone marrow biopsy samples with blood a reality for our patients.

Nicholas Lea, PhD, Clinical Scientist, Laboratory for Molecular Haemato-Oncology, Department of Haematology, King’s College Hospital London

Our study was designed primarily with the patient benefit in mind. Being a tertiary referral centre for MDS, there was a clear need to improve on existing methods in aiding patient diagnosis and enable frequent follow up of patients. The data is an extension of our earlier publication in the journal Blood (2013) and confirms the very high concordance of the genetic information obtained from the bone marrow and peripheral blood. I am delighted that MDS UK has taken the initiative to disseminate this information to the community so that patients may benefit from cutting edge research tools to help and with their MDS journey

Dr Azim M Mohamedali, PhD Senior Research Fellow, Department of Haemato-Oncology, King’s College London


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