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Prof Bowen talks about the results of MDS Right, a European study on MDS patients

Research FOR Patients
-For an informed and empowered opinion-
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MDS Right: a European observational study

If you are an MDS patient, it is important to find out about scientific research, and to know about ways you can contribute, as new treatments or improvements may come out of research.

Clinical trials are one way to develop new therapies, but observational studies are equally important.

Observational studies are those in which individuals are observed or certain outcomes are measured. No attempt is made to affect the outcome so no treatment changes needed, no additional decisions are made, no complex randomization and no placebo to take.

You don’t even need to be on treatment – so also Watch&Wait patients can take part.

All patients can participate, as opposed to the often complex or unrealistic eligibility criteria of drug trials, which only allow younger patients, or those without additional co-morbidities.

MDS Right aims to learn more about real MDS patients

MDS Right is an observational study, set up in May 2015, to gain access to real world data of MDS patients and elderly patients with anaemia symptoms. The hypothesis is that some of these patients might have low risk MDS. The study is running in 15 EU countries.

If you are not yet involved – please ask your haematologist about it and show them this article.

Watch this interview with Prof David Bowen, talking about the progress of the MDS Right Project and read the excerpts below.

Which improvements for patients are coming out of the Registry Programme?

Read the excerpts of David Bowen's Interview

We've got a large number of patients  (2,500) who've been followed for a few several years.

We're starting to generate a set of data that reflects what really happens to patients with MDS, how they're managed, and we can start to tease out how better to use the drugs that we have available.

We can also do research on a population of patients that reflects those that we see every day in the clinic not the super-fit patients and generally younger patients that we treat within clinical trials. So that the time has come and we've learnt quite a few things about this patient journey.

The findings around iron chelation therapy

There's been much debate about iron chelation therapy - removing iron in patients who have multiple transfusions. We're starting to generate evidence that supports the benefits of iron chelation therapy. There's been quite a bit of evidence generated in the past but not with the same degree of quality data and granularity of data.

We seem to be able to demonstrate that patients who receive iron chelation therapy are living longer than those who didn't so that's been quite valuable. It's a little bit preliminary at the moment but it supports a lot of other evidence and I think it's going to probably stack up quite well in the future.

How to use the drugs that we use for anemia, how to remove iron, these are very important aspects that we're generating, which affect the way that we manage patients.

Red cell transfusion and how to do that better

On the red cell transfusion side Simon Stanworth, my colleague, and I have worked on a trial that we've been running over the last two or three years with colleagues in Canada and in  Australia and we've basically been trying to work out whether we can allocate patients to two different types of transfusion schedule. We thought was going to be challenging and in fact we have managed to to do that: we have allocated patients to two different transfusions regimes and we see some differences in the outcome which is very exploratory at the moment but really quite exciting and our Canadian colleagues are just completing their trial. We'll see if they come up with the same findings and so we're moving on to the next phase of that shortly.

Watch Prof Simon Stanworth talking about REDDS, a study to look at transfusion patterns for low-risk MDS patients, and whether an alternative pattern of delivery might have a positive impact on quality of life

What are the drugs that we're evaluating at the moment to treat anemia in MDS?

Prof. Bowen: "The other session which is in the program on Wednesday is related to treatment of anemia. There's a new drug which is currently under consideration by the European Medicines Agency called Luspatercept and I'll be explaining exactly what the role of Luspatercept is in these patients that are the target for that, and also an idea of the various different drugs that are coming along, one or two of which could well also be useful in treating anaemia and be additional to what we've got at the moment. So we're covering several aspects of anaemia and MDS."

What is MDS RIGHT?

MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) is a research project that has been granted 6 million Euros from the Horizon2020 programme of the European Union.

In this project – which started in May 2015 and will end in 2020 - fifteen European partners have joined forces. By comparing existing health care interventions and by defining and implementing more effective and safer interventions for elderly European citizens with anaemia and/or lower-risk MDS, the project aims to lead to better treatment compliance and more (cost-)effective use of healthcare resources.

Myelodysplastic Syndromes (MDS ) are a group of chronic bone marrow malignancies, predominant in the elderly and complicated by severe anaemia (Anaemia of the Elderly, AoE). Symptoms and complications of MDS negatively impact the quality of life of patients, but in many cases MDS is not correctly diagnosed.

In elderly people anaemia is a medical condition that is often seen. It is hypothesized that 20% of all AoE cases - a total of 2 million European elderly citizens - might be explained by lower risk MDS. AoE is an increasing and complex challenge: the ageing population leads to an increasing financial burden to our healthcare systems, while insufficient awareness of the importance of a timely and right diagnosis increases the burden placed on patients and caregivers and decreases their health related quality of life (HRQoL).

The European project partners will work closely together to address this problem and to develop more (cost-)effective and safer evidence-based, tailored interventions for elderly patients with anaemia and/or lower risk MDS.

This project has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement No. 634789.

Visit MDS Europe website which covers MDS Right. It is also an amazing source of further information about MDS latest news, from a research and treatment perspective.

Whether you are an individual affected by MDS, or a patient organisation, do make a point of visiting it regularly.

Clinical Trials open to recruitment in the UK

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?

By Prof. David Bowen

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, 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.

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, now added to the goals.

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: -

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:
Tel: 020 733 7558

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

Source: / 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

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