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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.

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