this is archive.php


Research FOR Patients
-For an informed and empowered opinion-

All the trials listed on our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.


  1. SUB-TYPE OF MDS: Patients who have previously received a hypomethylating agent (eg. Azacitidine, Decitabine)
  2. SEVERITY OF MDS: Intermediate-2 or High risk
  3. NAME OF DRUG: Durvalumab, Tremelimumab, Azacitidine
  4. Aims and benefits: This is a Phase 1 study for patients who have previously received a hypomethylating agent. Durvalumab is a monoclonal antibody directed against programmed cell death ligand 1 (PD-L1) which activates the immune system to exert a response against tumour cells expressing PD-L1. Tremelimumab is a monoclonal antibody that stimulates the immune system to attack tumour cells. It does this by switching off the inhibitory mechanisms that stop tumour cells from being destroyed. This study aims to determine the safety and tolerability of MEDI4736 as Monotherapy or in Combination With Tremelimumab With or Without Azacitidine.

Read More

How is Quality of Life in patients with Myelodysplastic Syndromes (MDS) measured?

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

In this article David Steensma, MD FACP at the Dana-Farber Cancer Institute and Harvard Medical School, Boston, Massachusetts USA discusses how to incorporating the Patient’s Voice into MDS Care and Research.

Measuring quality of life: how to capture the voice of patients

Most studies of new treatments for myelodysplastic syndromes (MDS) track measurable changes in patients’ full blood counts and bone marrow cells, since improvements in these ‘objective’ parameters tend to correlate with better physical functioning, reduced symptoms, and sometimes even longer life. But the day-to-day experience of the patient is fundamentally important too, both in the context of clinical trials and in more routine haematology care.

Increasingly, investigators are capturing the voices of patients through the use of formal surveys that measure what are called ‘Patient Reported Outcomes’ (PROs), which can give insight into ‘health-related quality-of-life’ (HR-QOL).

While it has long been recognised that HR-QOL is critically important, the advent of PROs is a welcome development as certain symptoms can only be reported by patients and are not easily measured by an outside observer. For example, a doctor may notice when the patient in the clinic chair in front of her appears uncomfortable and fidgety, dozes off during conversation, or is breathing rapidly and audibly. But only that patient can tell the doctor just how much he is actually bothered by pain, anxiety, fatigue or breathlessness.

In addition, the influence of a diagnosis like MDS on patients’ daily lives, including the degree to which disease-associated symptoms get in the way of important activities such as exercise, cooking, bathing, working, or interacting meaningfully with family, is best reported by the patients themselves.

Patient Reported Outcomes (PRO)

There are many PRO tools with diverse designs. What all PRO tools have in common is that patients are asked a series of questions about the presence, absence, or severity of particular symptoms or concerns. Each ‘domain’ in a PRO survey instrument includes questions about a specific area of focus such as fatigue, sleep disturbance, or financial worries.

Some survey instruments explore a single domain like depression, or are designed for use by patients with a single disease such as dementia. Others are designed to assess health status more generally.

Some PROs are well-validated and have been employed in hundreds of studies, while others have been used only on one or two occasions.

The most useful PROs for clinical trial purposes are those that are most specific to the disease or problem being measured and that have been benchmarked in a large number of people with the condition of interest.

Widely used general PRO tools include:

  • The European Organization for the Research and Treatment of Cancer 30-question Quality of Life Questionnaire (EORTC QLQ-C30), which has been translated into over 100 languages and used in more than 3,000 studies to date.
  • The Psychological General Well-Being Index (PGWBI), which has been part of thousands of studies and is not limited to haematology-oncology.

More narrowly focused tools include:

  • The Brief Fatigue Inventory (BFI), which measures specifically the severity and impact of fatigue - the most common MDS-related symptom.
  • The series of Functional Assessment for Cancer Therapy (FACT) instruments developed by Chicago social scientist Dr. David Cella. The dozens of FACT tools are well validated in patients with cancer and blood disorders. One of the most commonly used is ‘FACT-An’, which specifically assesses the influence of anaemia-associated symptoms and transfusion burdens on patient’s quality of life. Given the frequency with which patients with MDS suffer from anaemia (over 90% are anaemic at diagnosis), the FACT-An tool is commonly used in MDS clinical trials, even though it was not developed specifically for MDS.

Dr. David Steensma on MDS research | Dana-Farber Cancer Institute

How can PRO tools help in clinical consultations?

PRO tools were first developed for use in health care services research, but are now integrated into almost every major clinical trial in haematology-oncology. We are also starting to see these tools used as part of general clinical care, where patients are being asked to complete surveys (either on paper or on an electronic tablet) while in the waiting area prior to an appointment.

Formally capturing PROs in practice allows the recording of trends in symptoms, and may save time in the clinic or allow triaging of which patients most need to be seen for consultation. For instance, in our practice in Boston, older patients are assessed using a PRO at the time of initial consultation and those with a high degree of symptoms specialist in addition to the haematologist.

In addition, some patients may be uncomfortable bringing up certain types of symptoms with their doctor (e.g. depression, anxiety, sexual concerns). But if the patient is ushered into the consultation room having completed a PRO instrument that discreetly highlights those symptoms, the doctor can then inquire specifically about such problems and offer help. This may be especially beneficial in some geographical areas where patients are more hesitant to discuss symptoms.

I was recently on a flight from Florida to Boston in which the person in the middle seat in the row in front of me began to discuss her bothersome rash, prompting a soliloquy from her neighbour about hip pain and sciatica and a later mention from the seatmate on the other side about how difficult it had been for her to get her infant to breastfeed properly. I was reminded that Americans tend to ‘over-share’, having few qualms about discussing intimate physical complaints with a casual acquaintance or someone they’ve just met. During the 2 years I spent in Oxford, I noted that patients in the UK tended to be a bit more reserved!

PRO scores and new drug approvals

Increasingly, PRO scores are reviewed by regulatory agencies considering new drug approvals, and are part of the deliberations of health technology assessment bodies and insurers when deciding which new drugs are worth paying for. For instance, ruxolitinib (Jakafi) was approved by the US Food and Drug Administration (FDA) in November 2011 for treatment of patients with the bone marrow disorder myelofibrosis, and the European Medicines Agency (EMA) approved ruxolitinib in April 2012.

Those regulatory approvals were primarily based on striking medication-induced improvements in patient scores on a disease-specific PRO tool called the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) designed by Drs. Ruben Mesa of Mayo Clinic and Claire Harrison in London and their colleagues. MPNSAF scores were supplemented by objective measures of radiographic improvement of spleen dimensions, since spleen enlargement is common in myelofibrosis, unlike MDS.

Once EMA approval had been received, the UK National Institute of Health and Clinical Excellence (NICE) and the Scottish Medicine Consortium (SMC) for NHS Scotland carefully considered MPN-SAF PRO scores and decided for which types of patients with myelofibrosis they would fund the drug.

How are patient-friendly PRO tools developed?

Of course, if PRO tools are very long and cumbersome, they are less likely be used, so when a new PRO tool is designed, considerable effort goes into developing the shortest tool that will reliably gather the desired information.

Developing a PRO tool is a multi-step process. For instance, when my Harvard colleague Gregory Abel and I developed the first MDS-specific PRO, Quality of Life in in Myelodysplasia Scale #1 (QUALMS-1) in 2014-2015, we began with a series of structured interviews with patients and caregivers, supplemented by findings from focus groups. We compared results to FACT-An and EORTC QLQ-C30 measurements from the same patients, and administered the tool on multiple occasions just in case patients were having a particularly bad day on the day they used the PRO.

We then used statistical tools with the help of experts in measuring HR-QOL to figure out which questions provided the most information and provided information that was not redundant and therefore should be incorporated into the final PRO, as well as which domains were independent of other domains. QUALMS-1 has been validated in international populations (Abel G et al Haematologica June 2016) and is now being used in several ongoing MDS prospective clinical trials.

Silver lining

One unexpected finding from our work on QUALMS-1 was that some patients noted a ‘silver lining’ from their MDS diagnosis.

A subset of patients expressed greater appreciation of the value of life in the context of regular reminders of its fragility, and they told us that their MDS diagnosis favorably influenced interactions with friends and family and made them appreciate each day more. We called this the ‘existential domain’, and it is an important part of the QUALMS-1 measurements.

It is a welcome development to see HR-QOL metrics and PROs incorporated into medication trials and haematology clinics. After all, at the end of the day it is the patient who has the disease and who is best able to judge whether a treatment makes a meaningful difference beyond merely altering measurable numbers.

MDS UK Nov 2018 Newsletter

This article was first published in the 9th Edition of the MDS UK Newsletter. If you haven't received it, please contact us.

MDS UK Survey: How do MDS Patients with anaemia report on their quality of life (QoL)?

Prof Fabio Efficace: Why is it important to measure Quality Of Life in MDS?

How does Myelodysplastic Syndrome and its treatment impact on the Quality of Life of patients?

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

Quality of Life on MDS Patients with Low Red Blood Cell Counts as measured on our MDS UK Survey

By Raqeebah Agberemi.

Myelodysplastic Syndromes (MDS) are a wide range of disorders which affect the functionality of the bone marrow. Thus, blood cells such as red blood cells, white blood cells and platelets may be affected. Low red blood cell (RBC) counts can lead to symptoms of anaemia such as fatigue and shortness of breath which can impact patients’ quality of life (QoL). The clinical management of MDS itself can impact on a patient's QoL.

This article will be concentrating on issues regarding red blood cells (RBC). Some categories of MDS patients are in need of supportive care in the form of RBC transfusions. However, these are associated with risks such as iron overload, infections and transfusion reactions. To assess the impact of MDS on QoL, we administered the QoL instrument QoL-e together with our survey. QoL-e(1) is a quality of life questionnaire used to assess the impact of MDS and its treatment on physical, functional, social, sexual and emotional wellbeing domains.

The aim of this article is to highlight the some of the specific issues relating to low RBC counts in MDS.

MDS Symptoms

The clinical manifestation of MDS varies amongst patients. It ranges from mild and stable cytopenia (low number of mature blood cells) to severe deficiency in all blood cells and increased risk of progression to acute myeloid leukaemia (AML). The most common symptom is fatigue.

Based on MDS UK Patient survey:

66% had symptoms attributed to MDS before diagnosis
Fatigue was reported to be the most common symptom of MDS (76%) followed by breathlessness (62%), bruising (32%), infections (18%), and bleeding (12%)

Depending on the type and risk category of MDS, MDS can seriously affect the QoL of patients. 29% of MDS patients who took part in the survey indicated that they carry out their usual activities with only minor symptoms of MDS and none reported that they required special care and assistance or hospitalization. However, patients indicated the impact MDS had on their lives – 22% reported it ‘’takes an effort to engage in normal activities’’; 18% report they can’t perform active work; and an additional 6% and 8% reported that they required occasional and a considerable amount of assistance respectively (See Graph below).

Question: Please tick one box that describes your abilities at the present time

Suffering from MDS causes a steady and persistent decrease in the functional ability of patients partially due to fatigue and this has a negative impact on QoL in a number of ways. Fatigue experienced in MDS patients is as a result of low haemoglobin levels due to anaemia and it could have an impact on the physical aspects of a patient’s life(2). The physical aspects of fatigue experienced by MDS patients is often associated with decreased strength, unusual tiredness and weakness, lack of energy and the persistent need to sleep or rest. This fatigue is usually not as a result of a recent strenuous activity and could affect the functional and cognitive ability of a patient. This can lead to early retirement or reduction in work hours for patients who are not of retirement age thus resulting in financial consequences for the patient and their family. Similarly, it can lead to restriction in daily activities, planning for the future and diminished independence for patients who are of retirement age.
Based on QoL-e scores(3), the majority of patients found it very difficult performing heavy activities and experienced added fatigue simply when carrying out daily activities.
When asked about the greatest challenge of having MDS, patients said:

'Chronic fatigue affects my day to day activities'
'I look too well to be ill and it is hard to explain the deep fatigue I feel'
'The fatigue and how it can impact on daily life some of the time. Finding it difficult to explain to people that the tiredness I feel is not the sort one might have in life in general'
'Sudden change in lifestyle from being fit and healthy to being restricted by fatigue and hospital visits'

Use of red blood cell (RBC) transfusions as a form of supportive care

Supportive care with red blood cell transfusions is usually the primary and standard management strategy particularly for patients with low-risk MDS based on the Revised International Prognostic Scoring System (IPSS-R)(4). MDS patients are usually anaemic at the time of their diagnosis and would rely on RBC transfusions to correct the symptoms at some point in their MDS journey.
Based on our survey:

62% received transfusions as part of their MDS treatment.
59% of those who received transfusions had red blood cell transfusion as part of their MDS treatment.

How often RBC transfusion is given varies between patients; some need them as often as every week while others need regular transfusions every couple of months. Likewise, pattern or frequency of transfusion can change over the course of the disease. In the UK, patients are usually transfused when their haemoglobin level drops to 80 to 85 grams per litre of blood(5). However once a patient is transfused, the level of haemoglobin does not remain constant and is not long-lasting. Haemoglobin levels start to drop after being transfused thus the symptoms of anaemia resurfaces before the next transfusion is due. This leads to fluctuation in the energy levels of the patient hence affecting QoL. Based on our survey, red blood cell transfusion dependence (RBC-TD) at the time of the survey correlates with decreased or compromised abilities compared to red blood cell transfusion free (RBC-TF) patients (See graph below).

Abilities - Segmented

There are no set haemoglobin concentrations at which blood is transfused at. It is based on different individuals; this level will differ between patients depending on the presence of co- morbidities such as heart conditions(6). Therefore, haematologists need to assess the symptoms of a patient before deciding what level they should be transfused at. Also, new research is going on to assess whether MDS patients should be transfused restrictively or in a more liberal manner. It also aims to investigate whether transfusions at more frequent intervals could potentially reduce fluctuations in energy levels therefore improving QoL(7). Visit our page to get more information on a recently concluded trial (REDDS trial) which investigates this:
The negative impact of MDS on a patient’s QoL can worsen as a result of the added efforts spent trying to cope with the disease such as repeated hospital visits for transfusions, waiting times as well as managing the side effects of transfusions.

RBC-TD patients will be on transfusions for a significant amount of time. At time of survey, 27% RBC-TD patients had received transfusions for 2 years or more.
According to our survey, the majority of RBC-TD patients are on a transfusion pattern of 2 - 4 weeks.

These efforts could potentially use up the limited energy stores of a patient thus affecting their QoL. Regular visits to the hospital for transfusion can have a financial impact on households.

31% and 21% RBC-TD patients respectively reported that cost for hospital travel and car parking had an impact on their household budget.

Also, reliance on family members or friends for support can affect a patient’s independence thus resulting in additional concern and stress for some patients and their families. This is worse for patients who don’t benefit from any support network. Based on our survey:

81% RBC-TD patients required support as a result of having MDS.
59% RBC-TD patients relied on their spouse for transport to medical appointments.

Apart from the physical health issues caused by MDS, emotional impact of the disease can be problematic as a result of uncertainties associated with the disease. RBC-TD patients worried about MDS to a significant extent compared to RBC-TF patients and suffered a higher emotional burden (See graphs below). QoL-E scores(8) were significantly lower (worse QoL) in RBC-TD patients in all domains (physical, functional, social, sexual and emotional wellbeing).

How frequently you worry about your disease?
How often do you feel that MDS is an emotional burden to you?

Iron Overload

Transfusion dependent MDS patients have been found to have a lower QoL compared to transfusion free patients(9). Our survey also supports this statement; QoL-E scores were found to be significantly lower (worse QoL) in RBC-TD patients in all domains. This could be due to the fluctuations in haemoglobin and energy levels, transfusion reactions, infections or accumulation of iron in the body.
Based on the MDS UK survey:

15% of RBC-TD patients had complications or side effects from transfusions.
44% of RBC-TD patients required hospitalisation due to the side effects.

Patients who are on long-term RBC transfusion are inevitably susceptible to developing iron overload. Increased amount of iron in the body results in the build-up of iron in various organs, in particular, the heart, liver, kidneys and endocrine organs thus leading to the continual deterioration of these organs. There are tests available to assess iron overload in a patient; the most common is a blood test called a Ferritin test. Iron chelation therapy is required to remove the excess iron in these patients and it is administered either via the Deferoxamine pump or oral tablet, Deferasirox (Exjade). Based on our survey:

24% of RBC-TD patients received iron chelation therapy.

Though iron chelators are administered to patients with iron overload, they can have side effects which can be challenging for patients. Common side effects are: body rash or hives, digestive problems, anaphylactic shock, swelling or lumps at the local infusion site to mention a few(10). Also, using the Deferoxamine pump can be inconvenient and uncomfortable for patients as they have to insert a needle into the stomach and wear the pump overnight. This can disrupt a patient’s sleep thus affecting their limited energy levels. Most side effects can be prevented or managed by working closely with the clinical staff. Discuss any symptoms you have developed after starting iron chelation therapy with your clinical team.

Below is a word cloud illustrating the most quoted words when patients were asked "What do you found to be the greatest challenge of your treatment for iron overload?"

What do you found to be the greatest challenge of your treatment for iron overload?

Other health issues

QoL-e results relating to physical well-being matched these findings from the general part of our survey. As a result, patients have to navigate the health system, speak to different consultants and manage multiple health visits sometimes in different hospitals. In some cases, patients are forced to ensure that there is communication between the consultants in the same and/or different hospital departments. As a result, there is a need for physicians treating the other health issues to liaise and collaborate with haematologists in charge of MDS care. Thus, an overall treatment plan adjusted for other health issues can be developed for the patient.


The consequences of MDS can be debilitating thus affecting the physical abilities of a patient. Additional factors associated with multiple disease management such as side effects from treatments, travel time and regular hospital visits by MDS patients can further impact QoL. Therefore, it is important to offer these patients some increased support and a more coordinated/collaborative care so as to improve QoL.

The impact of MDS on emotional well-being can be challenging. This is usually due to uncertainties which can arise as a result of inadequate understanding of the disease, fear of potential disease progression therefore leading to anxiety and worry. Unfortunately, care and attention is sometimes too focused on patients physical problems associated with MDS. This is where the role of an MDS nurse and counsellor lies. The former helps patients understand MDS better, identify and assess any physical issues associated with MDS as well as help navigate through the complex health care system. The latter helps patients understand, reflect on and work through concerns and worries they may have thus potentially improving their QoL. Patient support groups are also an excellent source of reassurance for MDS patients.


  • Emotional and psychological support should be offered at the earliest stage to patients who need it in order to help them navigate some of their challenges.
  • Coordinated, collaborative care and support for MDS patients to help with the development of a treatment plan adjusted for other health issues is important.
  • Clinicians should look into transfusing patients at more frequent intervals in order to potentially reduce fluctuations in energy levels thus improving their QoL.
  • Patient reported outcomes should be used to help provide information and elaborate on a patient’s experience. It is important to direct them to services according to their needs.
  • Remote monitoring needs are to be explored further for Watch & Wait patients to reduce the need for hospital visits.


  1. Esther N Oliva et al (2013) Development and Validation of QoL-E instrument for the assessment of health-related quality of life in myelodysplastic syndromes.
  2. Steensma et al., 2008 Common troublesome symptoms and their impact on Quality of Life in patients with myelodysplastic syndromes (MDS): Results of a large Internet-based survey
  3. Esther N Oliva et al (2013) Development and Validation of QoL-E instrument for the assessment of health-related quality of life in myelodysplastic syndromes.
  4. Lodovico Balducci, M.D. Transfusion independence in patients with Myelodysplastic Syndromes
  5. people-with-myelodysplastic-syndrome
  6. Sally Killick et al. Guidelines for the diagnosis of adult myelodysplastic syndromes
  8. Esther N Oliva et al (2013) Development and Validation of QoL-E instrument for the assessment of health-related quality of life in myelodysplastic syndromes.
  9. Thomas, ML et al. The importance of Quality of Life for patients living with Myelodysplastic Syndromes.
  10. Sandra Kurtin. Building blocks of hope; Strategies for patients and caregivers living with MDS.

MDS UK November 2018 Newsletter

This article was first published in the 9th Edition of the MDS UK Newsletter. If you haven't received it, please contact us.

Bone marrow donor drive at RAF Coningsby

The event was prompted by Adrian Cooper, following his wife Emma's diagnosis of MDS

On Thursday 22nd of November MDS UK was represented at a bone marrow donor drive at RAF Coningsby alongside the bone marrow donor charity, DKMS. The event was prompted by the husband of Emma Sheldon, Adrian Cooper, following his wife’s diagnosis of MDS.

Adrian, Emma and Jayne

As a great number of patients will know, the initial emotions that a great many experience on receiving this diagnosis can range from shock and disbelief to abject despair. Very often patients will ask “why me?”. This event was part of a concerted effort to support Emma and Adrian, to help them come to terms with the diagnosis, and see that there was a way forward. We hope that we have shown that the support is available and that we are ready to listen.

The support from the RAF was spectacular with over 75 RAF and support personnel swabbed

The support from the RAF was spectacular with over 75 RAF and support personnel swabbed

RAF Coningsby is an iconic location being the original home of ‘The Dambusters’. It’s currently home to a squadron of Typhoons amongst other aircraft.

RAF Coningsby was chosen because Adrian is a pilot with the RAF, and the RAF had been instrumental in supporting a bone marrow donor drive. We were lucky enough to have a great group of supporters on the day. These included Tom Lightfoot, himself an MDS patient who’s had a successful transplant, Jayne Snell, another MDS patient who’s also had a successful transplant, together with her husband, Martin, and Russell Cook, deputy chairman of MDS UK, there to represent the charity in an official capacity.

It was a long day - with some of our supporters driving 2 to 3 hours or more in order to get to the event - but well worth it. The support from the RAF was spectacular with over 75 RAF and support personnel swabbed ready to be put onto the bone marrow registry - and the general consensus on the day was that we could probably do this again with even greater effect.

RAF Coningsby Bone Marrow Drive

A suprised radio interview for Russell on BBC Radio Lincolnshire to raise awareness of MDS and the need for blood marrow donors

A surprise on the day was a radio interview for Russell during which he managed to ad lib his way through a 10 minute phone call, live on air, with Melvyn Prior of BBC Radio Lincolnshire! During the interview he emphasised how important it was for people to come forward as donors, that although the event was restricted to base personnel, that the public could join the bone marrow register by contacting the charities directly and asking for either a swab or spit kit. He also highlighted the problems that a great many MDS patients suffer with; namely, fatigue, nausea, bleeding and infections - and pointed out that a great many GPs will never have met a patient with MDS.

Although only 1 in 10 MDS patients will be eligible and fit enough to tolerate a bone marrow transplant it goes without saying that adding to the registry is absolutely vital. At any one moment in time there are circa 1600 people with blood cancer in the UK in desperate need of a bone marrow match but for whom nobody has as yet come forward.

Currently, only 69% of all blood cancer patients can find the best possible match from a stranger, and this drops dramatically to 20% if you're a patient from a black, Asian or ethnic minority background.

By building and diversifying the register we will be able to provide the best match to even more people with blood cancer. Somewhat shockingly only 2% of the population of the UK are registered as potential donors compared to 9% in Germany. This goes a long way to explain why somewhere in the region of 3 out of 5 donors to UK patients are from Germany. These events will hopefully add to the pool of resources that cancer patients in the UK are reliant on.

We hope that by highlighting MDS as a hidden and rare blood cancer that we will be able to garner further support either in person or financially so that we can help fund research into the disease.

MDS UK would like to thank Sqn Ldr Elizabeth Long, Leslie-Ann Kloed, and Jim Robinson (RAF communications) for organising the event and Sarah Gray from DKMS for the materials on the day.
We would like to make a special mention of Jayne Snell who volunteered to act as organiser for the event and was a central figure on the day.
Thank you to the RAF for their hospitality and their assistance on the day.
Of course not forgetting Fl Lt Adrian ‘Coops’ Cooper and Emma - we look forward to assisting in future events.

How to donate bone marrow

If you’re between 16 – 30 and in good health, sign up to Anthony Nolan's register and you could be a lifesaving match for someone with blood cancer

You Can Become a Donor!

Even if you’re older than 30 you can become a blood donor if you're in good health. Check your eligibility at the dkms site

MDS UK Patient Support Group Newsletter – November 2018

Our 9th edition of the MDS UK Newsletter is now out!

Read all about:

    Patient's Stories: Kes Grant ‘Coping with long stays in hospitals...’; Andrew Scott Married to MDSDS; Tilly Tilbrook ‘How I felt about losing my hair...’; Christina Fowler ‘Decisions, decisions...’
    Research: Prof D Wiseman Chronic Myelomonocytic Leukaemia (CMML), Prof D Steensma, Measuring quality of life; Prof T Chevassut The effects of vitamin C, Raqeebah Agberemi, Impact of myelodysplastic syndrome and its treatment on quality of life of patients
    Advocacy: Radio Day on MDS Awareness Day: Make Blood Cancer Visible Campaign
    Fundraising: Prudential RideLondon-Surrey 100, Cabbage Patch Race
    From MDS UK Board: Sophie Wintrich, Welcome Notes; Edward Peel LL.B (Hons) Chair MDS UK Growing and achieving

Read More

Vitamin C can help to correct a basic MDS problem: blood cells failing to differentiate properly

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

In this article Dr Timothy Chevassut, Consultant Haematologist at the Royal Sussex County Hospital, Brighton, looks at the benefits of Vitamin C in the treatment of MDS.

Linus Pauling greatest discovery

Not many people will have heard of Linus Pauling but he remains one of the greatest scientists of the 20th century. His early work was on the haemoglobin molecule, something that is of great importance to our understanding of anaemia and the symptoms of tiredness that many patients with myelodysplasia (MDS) will know all too well.

However, it was his deep held belief in vitamin C and its health benefits that Linus Pauling will be remembered for, particularly in preventing and treating cancer. At the time, not many people believed him, thinking that his claims for vitamin C were unfounded, but it turns out that Linus may have been right all along, at least in the case of the MDS.

The effects of vitamin C

Recent research has shown that vitamin C, or ascorbic acid, as it is known scientifically, can have profound effects on the blood. This is because vitamin C can affect the way that some proteins work in our blood cells. In particular, there is a protein called TET2 that carries out an important job in our bone marrow by controlling a process called DNA methylation. This is the mechanism that tells genes when to switch on and when to switch off. In turn, this determines what type of blood cells are produced, a process called differentiation.

In MDS, the fundamental problem is that cells fail to differentiate properly and very often this is due to a mutation in the TET2 gene that causes DNA methylation to go awry.

Remarkably, it turns out that vitamin C can help to correct this problem by binding to the TET2 protein and boosting its activity and thereby restoring normal DNA methylation! This is because vitamin C is a what’s called a ‘co-factor’ of the TET2 enzyme that is required for it to work properly: a discovery made only a few years ago by scientists working on DNA methylation.

In MDS, doctors use drugs such as azacytidine to help restore normal DNA methylation in the blood cells in order to treat anaemia and improve low cell counts. Azacytidine slows down progression of MDS to acute myeloid leukaemia (AML) and can even target the leukaemia cells themselves. In laboratory experiments, vitamin C can overcome the mutated TET2 protein and help to kill the leukaemic blasts in combination with azacytidine.

Current research

This exciting discovery has led to the testing of vitamin C in patients with MDS to see if it can improve current treatment with azacytidine. There is certainly evidence to suggest that vitamin C can enhance the effects of azacytidine but we don’t know yet whether this will work similarly in MDS patients. Nor do we know the dose of vitamin C that patients would need to take or even whether vitamin C is at all harmful at higher doses. That is why several clinical trials, both in Europe and the USA, are now looking at whether vitamin C can be a useful treatment in MDS.

First of all we have to establish that vitamin C is safe for patients and does not have unwanted side-effects.

Once we have found out the best dose, then there will be further clinical trials to explore whether it actually works to improve patients’ symptoms and prolong their lives. Hopefully, this is something we shall be looking at in the UK in the near future.

Oranges and lemons...Should patients diagnosed with MDS start taking vitamin C?

It is probably too early to say at this stage but it is certainly worth bearing in mind the many other health benefits of eating fruit, particularly citrus fruits such as oranges and lemons, as part of a well-balanced diet. Perhaps we should all be listening to the good advice of the bells of St Clement’s?

Vitamin C and how it can have an impact on the treatment of MDS

MDS UK Nov 2018 Newsletter

This article was first published in the 9th Edition of the MDS UK Newsletter. If you haven't received it, please contact us.

The Basics: Understanding Blood Formation: Blood Cells & Blood Structure

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

Radio Day: October 25, MDS World Awareness Day. Catch-up on the stories of 4 people affected by MDS

MDS is a rare form of blood cancer

Annually 2,240 patients in the UK are diagnosed with myelodysplastic syndromes (MDS)

On October 25 we marked MDS World Awareness Day to raise public awareness of myelodysplastic syndromes (MDS) and its symptoms. We joined forces with UK local and national radio stations

11 interviews

130 minutes of airtime on local and national radio stations

We reached 1.78m people!

Four people affected by MDS were interviewed on radio stations across the UK to spread the voice, for a greater understanding of MDS and for more research to be conducted to find a cure!

Follow us on Facebook and Twitter and share their stories!

MDS UK and the Cabbage Patch 10 Mile Race

This year, under the patronage of Caitlin Limmer, who also passes for race director of CB10, MDS UK had a team of 20 runners at the Cabbage Patch Run. This is the third year that MDS UK have had a big presence and it’s now our ‘signature’ event. Help us raise enough money to cover the cost of a patient forum meeting! We also hope to add to our vision of supporting research into MDS.

After the Cabbage Patch Run the lovely runners from the BearCat Running Club marked MDS Awareness Day with a wonderful a-cappella song!

MDS UK Patient Support Group: Supporting GPs and Healthcare Professionals

We produced a Factsheet for GPs and Healthcare Professionals to help them spot early signs of MDS and refer patients to a specialist.

GPs are advised to be aware of patients who have persistently low blood counts or an unexplained need for blood transfusions. It is also important to note that some people with MDS don’t present any signs or symptoms at the time of their initial presentation, and that the disease is often only picked up via routine blood tests or procedures, which sets MDS apart from other blood cancers.

MDS UK Patient Support Group: Supporting MDS Patients and their Families

We provide a range of services for people affected by Myelodysplastic Syndromes (MDS)

People access more than 900 pages on MDS UK website every single day. On Radio Day people visited almost 2,000 pages!

MDS Website PageViews

And we also organise local group meetings. Watch Claudia talking about the many benefits of attending a meeting

Join your Local MDS Group

When do they meet and how to contact them

We’d like to sincerely thank those who made the World Awareness Day Radio Day Experience possible:

Reynolds McKenzie Healthcare PR, specifically Sophie and Natacha, as well as 4 Media Relations, Ellie and her team – for working with us on all preparations, content and scheduling.

And Celgene UK as our main financial support for this radio event.

Catch up on the Cabbage Patch 10 Mile Race 2018: The Highlights

Cabbage Patch Race and a Surprise Choir for MDS Awareness Day 2018!

MDS UK and the Iconic Cabbage Patch 10 Mile Race

By Russell Cook, runner, ex-MDS Patient and Deputy Chairman Trustee & Director of MDS UK Patient Support Group

Sunday 14 October saw MDS UK represented at the iconic Cabbage Patch 10 mile race. You are probably wondering why it’s got such a funny name! Well, it’s named after the Cabbage Patch Pub in Twickenham, a well-known haunt of anybody who is a rugby fan going to the world-famous Stadium. The race itself was originally set up by the owners of the Cabbage Patch Pub and is almost as old as the world-famous London Marathon. It’s had some pretty significant winners too! Sir Mo Farah, no less, back in the day!

The race starts in the centre of Twickenham and heads out towards Richmond with some very pleasant sections along by the river. It’s quite flat and fast which is probably why it appeals to so many club runners. Apart from the first half mile or so, where the runners pretty much take over the roads, most of the race is held on open roads and paths! Although I can testify that traffic is not really a problem! One of the intriguing prizes at this event is the presentation of enormous cabbages to the winners! Often with some fairly amazed looks on the athletes faces!

This year, under the patronage of Caitlin Limmer, who also passes for race director of CB10, MDS UK had a team of 20 runners.

Against a background of torrential rain all managed the 10 mile course in a wide range of times. There is a great vibe to this race, it’s very community orientated, and it’s very popular with committed club runners as well as fun runners (and not forgetting old codgers like me….). It’s extremely well organised and this year there were over 140 marshals stood in pouring rain with enormous smiles on their faces urging on the athletes. I can only say what an enormously enthusiastic and committed bunch they were too! Many of them are members of the Bearcats Running Club and Stragglers Running Club. Bearcats have supported MDS UK through thick and thin over the last three years. We owe them an enormous debt of gratitude.

This is the third year that MDS UK have had a big presence and it’s now our ‘signature’ event. This year we hope to raise enough money that we can comfortably cover the cost of a patient forum meeting and ultimately we hope to add to our vision of supporting research into MDS.

In 2019, we will be there again and hope that we can encourage family members, if not the actual patients, to come along, support MDS UK and enjoy the day.

Catch up here on some of the testimonies from our amazing runners and the best pictures of the Team MDS runners in action

Most of the pics were taken by our dear Ian Trowbridge, who was able to get along to the Cabbage Patch 10 race and take photos and support the runners, near Ham House. Thanks so much Ian!

What do our runners say?

Eva Boyd and Ken Pearson:

A fantastically well organised event with many supportive people and encouraging marshalls right round the course. The weather was pretty grim but it didn't spoil the race and finishing definitely felt like an achievement! It was a real privilege to be involved with MDS UK and we would both be more than happy to fundraise again for this wonderful charity.

Joseph Ogbonna:

Great cause to support. Warm reception from MDS Patient Support team, in the form of Sophie who seemed to be everywhere with her smile. Fantastic race atmosphere with runner camaraderie enroute and the singing at the end topping it all.

Owain John:

When a loved one was diagnosed with the rare disease MDS, a condition about which my family and I (and countless others) had previously not heard, it was difficult to know where to turn for information. In addition to the first-class care and support provided by the haematology unit at the University Hospital of Wales, we have found the information provided by MDS UK, through its patient handbook, newsletter and website, to be invaluable. The charity's work, both in promoting knowledge of MDS and in supporting patients and their families, is truly inspirational. I'd like to think that the funds I raise as a result of running the Cabbage Patch 10 will represent a small but helpful contribution to its ongoing efforts.

Cabbage Patch Run 2018: The Best Pics!

What are the needs of patients suffering from MDS in the UK? Read the MDS UK Patient Survey Results

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

In 2012, MDS UK Patient Support Group started a research to assess the needs of patients suffering from MDS in the UK. To date, this is the largest UK-specific research ever conducted focusing exclusively on needs, experience and quality of life of MDS patients. The aim of the survey is to better understand the impact of MDS on patients’ quality of life and to ultimately improve the care and support offered to MDS patients.
Thus far, the progress of the survey has been positive; we have collected results from 171 participants across UK covering all age groups and including patients without access to the internet.

The following report has been compiled by Raqeebah Agberemi.

Based on our survey, 83% of the participants are over 65 years old. This correlates with data published worldwide which states that 85% of MDS patients are 65 years and above. The median age group for MDS diagnosis is 74 years old. Based on our survey, the calculated median age is 70.

MDS Treatment

Development of treatment for MDS has made extraordinary progress over the past 10 years and there are no signs of this progress slowing down. A pioneering drug, Azacitidine which was made available in February 2011 UK for patients living in the UK with intermediate and high risk MDS was developed to help slow the progression of the disease while Lenalidomide has been effective for patients with the del 5q syndrome.

What do UK MDS patients say about the treatment options provided at the time of diagnosis?

53% reported being placed on Watch & Wait
47% reported being placed on active treatment or supportive care

More than half of the participants were told they were on Watch & Wait status. Being placed on Watch & Wait can be extremely stressful due to the uncertainties about the disease progression therefore adding to the worry that comes naturally with being diagnosed with MDS.

Question: How frequently did you think about MDS in the last 4 weeks? (The traffic light colours of green, yellow/amber and red are used to encode positive, neither positive or negative and negative responses.)

Question: How frequently did you think about MDS in the last 4 weeks? The traffic light colours of green, yellow/amber and red are used to encode positive, neither positive or negative and negative responses

More than 50% Watch & Wait patients thought about MDS to a significant extent compared to those placed on active treatment or supportive care (see graph below)

Reaction to being placed on Watch & Wait differs amongst patients, some patients are able to cope well with it. However, some may suffer from anxiety or depression as a result of being placed on Watch & Wait. Anxiety or depression might affect some aspects of their lives; it might lead to difficulties relating to their work life thus resulting in temporary leave of work or reduction in working hours. This might have some financial consequences to the patient.

The feeling of being anxious or depressed is aggravated by the lack of information at the time of diagnosis. Based on our survey,
38% Watch & Wait patients did not receive any written information at the time of diagnosis
43% Watch & Wait patients received minimal verbal information at the time of diagnosis

These figures illustrate the importance of providing adequate information to patients. If patients are provided with information in a patient friendly and personalised manner at the earliest stage of diagnosis, this might reduce the worry and help them cope well with being placed on Watch & Wait. Likewise, it will reduce the time a patient spends in consultations with the clinical team, or even with a counsellor, thus freeing up time to see other patients.

When asked about the greatest challenge of having MDS, Watch & Wait patients said:

‘‘The Watch & Wait policy. Regular 6x weekly visits to the hospital for blood tests then lack of feedback on the results’’
‘‘The uncertainty over whether there will be progression of the disease’’
‘‘Watch & Wait is a bit like having a sharp sword hanging about your head. It seems to get lower every time you are heading for your next blood test’’

In comparison, patients on active treatment/supportive care said:

‘‘The side effects of Azacitidine’’
‘’The disruption to our lives. Unable to plan ahead as Chemo dates can change if my blood is not right. Having platelets every week is a real nuisance’’
‘’Waiting 8 hours for blood transfusion’’

Support for MDS patients and carers

There is a constant need to access information and advice for MDS patients and their carers. Access to these services provides them with knowledge as they face several challenges throughout their MDS journey. Access to a clinical nurse specialist, appropriate signposting to services and peer to peer support have been found to be useful for MDS patients.
We currently have local groups in several parts of the UK. If you would like to attend any of our meetings, please visit our page for further details or call us on 020 7733 7558.

Access to MDS Nurse or Haematology Clinical Nurse Specialist

Question: Do you have access to a specific MDS Nurse or Haematology CNS?

6% reported they had no access to a CNS but would like to see one 19% reported that they had never been offered this service
It is important that MDS patients have access to a Clinical Nurse Specialist (CNS) because they play a vital role in the management of patients concerns and support their health and wellbeing. We liaise with Clinical Nurse Specialists in several hospitals across the UK to provide assistance and to make it known that support and care is available for MDS patients.
According to the National Cancer Patient Experience Survey (NCPES), having a CNS is an important factor found to be positively associated with a better patience experience.1
To understand the importance of CNSs, we divided the patients who answered the question, ‘’Do you have access to a specific MDS Nurse or Haematology CNS?’’ into five categories based on their level of worry.

Question: How frequently did you worry about MDS in the last 4 weeks? (The traffic light colours of green, yellow/amber and red are used to encode positive, neither positive or negative and negative responses.)

40% of those who had no access to a CNS but wants to see one and 52% of those who had never been offered the service, worried about MDS to a significant extent. The lack of access to a named CNS aggravates the emotional impact of MDS and the findings of our survey illustrates this.

MDS patients are often confused at the time of diagnosis mainly due to the fact that they have never heard of disease; having a CNS as a main point of contact makes it easier for patients to ask questions pertaining their diagnosis and treatment thus reducing the emotional impact of MDS. CNSs can also recommend additional support services to patients should they need it. Likewise, having access means that CNSs can quickly recognise any developing issues that may require attention thus potentially avoiding any major problems.

As the ageing population continues to increase, so will the challenge of supporting patients with MDS. The availability of treatments and development of new ones means that patients will live longer with the condition thus illustrating the importance of CNS as patients will need continuous support over the years.

Information about MDS UK at the time of diagnosis

Question: At the time of diagnosis, did you receive information on MDS UK Patient Support If not, how did you hear about MDS UK Patient Support Group? 

More than half of the participants said they did not receive any information on MDS UK at the time of diagnosis.
Half of the participants who had not received information about MDS UK, learnt about MDS UK via the internet.

Due to lack of information on MDS at the time of diagnosis, patients and carers have to take active steps when trying to find out more information about MDS. This makes them vulnerable to coming across unreliable, inaccurate and out of date online information.


In the long term, providing information at the earliest stage of diagnosis to all patients can really save substantial time and concern to all involved (patients, families, clinical staff).
Practical Suggestion: Some haematologists take it a very helpful step further: after seeking express consent from the patients, they copy MDS UK Patient Support Group into the patient’s clinic letter requesting us to send information to the patients. This step takes only one minute during consultation and ensures no patient goes potentially unsupported.

Summary of findings

  • Adequate provision of information at the earliest stage of diagnosis should be provided to Watch & Wait patients to help them better understand diagnosis and cope better with it.
  • Emotional and psychological support should be offered at the earliest stage to Watch & Wait patients who need it in order to help them navigate some of the challenges they might be facing.
  • Provision of information at the time of diagnosis is important to prevent patients from becoming vulnerable to inaccurate and unreliable information on the internet.
  • All MDS patients should have access to a clinical nurse specialist who can provide support following their diagnosis.

MDS UK May 2018 Newsletter

This article was first published in the 8th Edition of the MDS UK Newsletter. If you haven't received it, please contact us.

Free donations by shopping