Azacitidine (Vidaza): Learn more about the latest developments around this important drug

What is Azacitidine (Vidaza) and how does it work?

Azacitidine is a type of drug called a hypomethylating agent. It works at the DNA level, "switching on" genes that stop the cancer cells growing and dividing. This reduces the number of abnormal blood cells and helps to control cell growth.

Hypomethylating agents are considered a non-intensive treatment. They are aimed at slowing the progression of the disease with as few side effects as possible, maintaining a good quality of life. They will not cure MDS but may ‘modify’ it and are usually given as an outpatient.

However, not all patients will respond to the treatment. Some will progress to leukaemia, others won't have an adequate response in terms of their blood count.

At the 14TH INTERNATIONAL SYMPOSIUM ON MYELODYSPLASTIC SYNDROMES, that took place in Valencia in May 2017, one of the topics discussed was the latest research around this important drug. Read more about how researchers are trying to establish which patients will have a good outcome with Azacitidine and whether the use of Azacitidine together with other drugs can make the treatment more effective.

Learn more about the latest MDS research: Download the full MDS Symposium Patient Summary

Predicting Good Outcomes with Azacitidine Treatment

Researchers are exploring which factors might be useful for predicting a good outcome in patients with MDS.

Certain treatment decisions for patients with MDS are based on cytogenetics, i.e. the study of the chromosomes. Azacitidine is appropriate for patients with higher risk MDS who often have mutations in chromosome 7 or three or more abnormalities in their chromosomes.

Dr. Raphael Itzykson, Université Paris Diderot, France, presented a study showing that if the platelet count after one cycle of the drug doubles, this is a good sign for an overall success of the treatment. However this happens in only a small proportion of patients.

Several ongoing efforts, including the HARMONY study, have a good chance of identifying more factors that predict azacitidine outcomes in MDS and other blood cancers and of predicting the effects of treatment on the patient's quality of life, healthcare costs, and care strategies.

The Treatment with Azacitidine Involves Several Courses

The Best Partner for AZA in Higher-Risk MDS

Dr. Mikkael Sekeres (Cleveland Clinic, Ohio) focused his presentation on combinations of Azacitidine and other drugs for higher-risk MDS.

The combination of azacitidine and vorinostat seemed promising. In a phase II clinical trial, about 70% of patients with untreated higher-risk MDS, CMML, or AML responded to the treatment, which was about double the expected rate for azacitidine alone. These responses lasted an average of 16 months.

Similarly, response rates and duration of response were promising in a phase I-II clinical trial of the combination of lenalidomide and azacitidine for higher-risk MDS.

Dr. Fenaux added that studies are also evaluating combinations of azacitidine with other treatments, such as valproic acid, venetoclax, immune checkpoint inhibitors, and idarubicin for higher-risk MDS or CMML.

Establishing The Correct Dose

Other research is assessing more intensive hypomethylating treatments or lower doses for longer use. Studies are testing different drugs, including venetoclax, cenersen, and a 10-day decitabine cycle (another hypomethylating agent) for MDS with TP53 mutations.

Dr. Sekeres concluded that azacitidine alone is still the standard treatment for higher-risk MDS. But some evidence
hints at better and more long-lasting responses for combination treatments if patients stay on them long enough. The hypomethylating drug “partners” under investigation might become options for higher-risk MDS in some patients.

Clinical Trials open to recruitment in the UK


Help improve the blood transfusion experience for MDS patients

How can blood transfusions be improved?

Depending on blood transfusion has a negative impact on the quality of life of MDS patients, but it is unavoidable for many patients.

That is the reason why a group of Canadian and United Kingdom haematologists are looking into running a series of studies to improve the red blood cell transfusion experience for MDS patients and improve their quality of life.

Before doing so, they are on a quest to understand better how red blood cell transfusions are being conducted today and ask patients what could be done, in their opinion, to make it better.

How can you help?

If you are a Myelodysplastic Syndromes (MDS) patient, you live in the US, the UK or Canada and you've been receiving at least one unit of red blood cells every eight weeks for the last 4 month period, the research team would like to hear from you.

MDS Red Blood Cell Transfusion Survey

You are invited to participate in this online survey.

The online survey will take approximately 5-10 minutes. Your responses will be kept strictly confidential. Be reassured that they will not collect any information that will personally identify you.

Your participation in the study is voluntary, i.e. you may choose not to participate at all and you may exit the survey at any time.

The Research Team

Rena Buckstein MD FRCPC Associate Professor, University of Toronto, Department of Medicine, Division of Hematology/Oncology
Yulia Lin MD FRCPC Associate Professor, University of Toronto, Department of Laboratory Medicine and Pathobiology
Jeannie Callum MD FRCPC Associate Professor, University of Toronto, Department of Laboratory Medicine and Pathobiology
Simon Stanworth MD Consultant Hematologist, NHS Blood and Transplant, Oxford University Hospitals NHS Trust

Take a look at current MDS clinical trials


When is the right time to give a blood transfusion?

When is a blood transfusion considered? How low should the cell count be?

When the blood lacks enough healthy red blood cells or haemoglobin, anemia symptoms develop, typically fatigue, shortness of breath, weight loss, paler than normal skin, etc and a blood transfusion is considered.

There is no set haemoglobin level at which a blood transfusion is given, but your doctor will assess your symptoms and you will decide together.

Prof. David Bowen says in a recent article:

I often delve back deep into the case notes to try to find a ‘normal’ haemoglobin level for that individual from many years before their diagnosis. I think that it helps to say to a patient for example ‘you are 30% down on your normal haemoglobin so it’s not surprising that you are feeling the effects’. This also helps us both to understand that the patient may have meaningful symptoms of anaemia despite their haemoglobin level being higher than that recommended for active treatment in the guidelines that we follow.

Why does anemia develop in MDS? Watch the video

How often can a patient have a blood transfusion?

How often you have transfusions will vary between patients; some need transfusions every few months whilst others need one every every couple of weeks. Very often, once a patient has started having regular blood transfusions, the length of time between transfusions will gradually get shorter.
We asked Prof. Bowen if there is a limit on how often a patient could have blood transfusions. Prof. Bowen replied:

The simple answer is that there is no limit.
If blood transfusions are becoming considerably more frequent then the doctor needs to consider if there could be an explanation in addition to the MDS such as bleeding (usually internal) or the red blood cells being destroyed more quickly (haemolysis). These may be able to be treated and the transfusions will then reduce. However by far the most common situation is that the red blood cell production from the bone marrow gets poorer and poorer with time in some patients. We will transfuse as often as is needed to maintain acceptable quality of life and we often transfuse patients every 2 weeks and sometimes weekly. There is a move towards thinking about more frequent transfusions with fewer bags each time as this mimics the normal situation better without the big peaks and troughs of energy gain then energy deterioration.

Blood transfusions lead to (temporary) improvements in a patient’s quality of life

The potential side effect of blood transfusions: Iron Overload

With every unit of blood you receive from a transfusion, you will receive an excess amount of iron. Over time this can accumulate in your body and could possibly cause damage to certain organs, like your heart or liver.

There is still considerable uncertainty whether too much iron in your body is always harmful. The level of iron in your body should be checked regularly, especially when you are on a regular transfusion program and you may need treatment for the build-up of excess iron. This is called iron chelation. There is currently uncertainty about the benefits of removing iron. Whether you are offered iron chelation treatment or not will depend on the likely benefits versus the likely disadvantages in your
individual case. This will be discussed with you before you make a decision to start iron chelation.

Some patients will develop complications that could be related to iron overload such as heart failure, liver abnormalities and diabetes, but these complications have other causes in older age, and it is always difficult to be certain how much iron overload is responsible, and we have drugs that can effectively remove iron from the body.

Says Prof Bowen.

Read Prof David Bowen full article in MDS EUROPE: "Which patients need more blood transfusions to improve quality of life and can we identify the patients who need iron chelation more precisely?"

Learn More About MDS Treatments


The RIGHT treatment for the right patient at the right time – a crucial EU initiative

More than 70 participants representing medical specialists and nurses caring for MDS patients, MDS patient advocates, medical researchers and data managers, healthcare authorities, regulators, HTA experts and industry representatives gathered for the first MDS-RIGHT multi-stakeholder meeting on the 3 May 2017 in Valencia, Spain - in conjunction with the biannual MDS 2017 International Symposium. 

This project is an essential and fantastic EU initiative – and we urge all dealing with Myelodysplastic Syndromes to keep abreast of news and developments.

Here are the main articles and reports:

You may also be interested in this recent interview with Prof David Bowen

More from MDS RIGHT


Would you like to run for us? Help patients by raising awareness & money for 3 patients meetings!

This year, our charity MDS UK was lucky enough to have 2 runners in the 2017 London Marathon Alec Holah, brother in law of Gavin Hepburn, who passed away a year ago, and Kirsty Crozier, an MDS Clinical Nurse Specialist in Oxford and Member of the MDS Committee.

We took this opportunity to conduct several interviews about the marathon, our charity and what it means to work closely with us.

We thank each one of our interviewees deeply for their kind, dedicated, thoughtful and generous input. They are wonderful friends, very close to our dear charity.

"It's not just about raising money, it's raising awareness" Dan & Alec Holah

Russel Cook, Deputy Chairman and Head of Fundraising. He ran several marathons after his transplant for MDS

"More people will start to understand the illness"... "The charity is amazing" Olivia & Tilly Hepburn

"We will always want to work with the charity. I feel we need to keep that awareness going" Lisa Hepburn


Can Azacitidine be administered at home? The Spanish experience

A new project was trialled for more than two years by nurses from home-care units in Spain: 'Azacitidine administration at home'.

Now it has been rolled out to include most of the hospitals in Spain administering Azacitidine. It was initially undertaken by nurses, not necessarily haematology nurses, who would visit patients at home usually by taxi from the hospital.

The economic savings were shown to be vast, saving around one-third compared to hospital administration. This equated to around 100.000 euros per year per patient.

See comparison below and download the full presentation here

Receiving Azacitidine at home increased patients' satisfaction

The patient videos and reports demonstrated clearly the increased level of patient satisfaction and improved quality of life for this patient group. Discussions around this included other units adopting the practice of caregivers trained to give azacitidine at home once they had collected from the hospital on daily basis. This again showed a significant positive impact on patients who, for example, were already fatigued.


Get the latest on MDS Treatment from the MDS Foundation Symposium

Janet Hayden, Myeloid Clinical Nurse Specialist, from Kings College NHS Foundation Trust, London (Adapted by MDS UK Patient Support Group) brings the latest in MDS Treatment from the Annual MDS Symposium in Valencia.

MDS Foundation Symposium May 2017

Some patients are suitable for home treatment after stem-cell transplant

Many interesting projects were presented during the nursing sessions, of which three were of particular interest and relevance to myself and my professional practice.

The first presentation - 'Home Care for MDS Patients Undergoing Allogeneic Bone Marrow Transplantation', by nurse Nuria Borras - covered a project by a nurses' group in Barcelona who were already working in an established ambulatory care unit that had undertaken many autologous transplants. Three MDS patients were chosen as a pilot to receive home care after their chemotherapy and stem cell infusion. After they were all successfully discharged to home care, they were visited twice a day at home and brought to the out-patient department twice weekly. All of these patients had achieved remission with Azacitidine prior to transplant and none of the patients received Campath or ATG as part of their conditioning. It was reported that none of the patients were readmitted. It was discussed that these patients had had a good or very good performance status prior to transplant and were already very familiar with the routine of self-management and receiving out-patient care. It was concluded that careful selection of patients may be appropriate to out-patient care delivery.

Azacitidine Treatment at Home

A further very interesting project was again presented by nurses in Spain: 'Azacitidine administration at home', by Laura Muñoz Tirado.

A project that has how been rolled out to include most of the hospitals in Spain administering Azacitidine, this project was undertaken across the country by nurses from home-care units. These nurses were not necessarily haematology nurses. They would visit patients at home usually by taxi from the hospital. The economic savings were shown to be vast, saving around one-third compared to hospital administration. This equated to around 100.000 euros per year per patient.

See comparison below and full presentation here.

The patient videos and reports demonstrated clearly the increased level of patient satisfaction and improved quality of life for this patient group. Discussions around this included other units adopting the practice of caregivers trained to give azacitidine at home once they had collected from the hospital on daily basis. This again showed a significant positive impact on patients who, for example, were already fatigued.

Photovoice: Patients' photos representing their MDS journey

Petra Lindroos-Kölqvist from Sweden presented Photovoice for MDS patients. This project allowed patients to take photos of things which represented their MDS journey. They were then interviewed and allowed to discuss their photos. A thematic analysis was made and themes such as hope, treatment, family and places were identified. Further work is proposed ....

See here the full presentation sent by our Swedish friend Petra and her nursing team

Managing your MDS with your Phone

As an international MDS nursing community, we rarely get the opportunity to get together and discuss our professional practice and patient care. The Nursing Leadership Board of which I am a member, facilitated some networking meetings during our stay in Valencia. In the past these meetings have produced collaborative projects and publications.

Of note and particular interest this year is that Sandy Kurtin has produced, as part of her ongoing PhD, an app for smartphones and tablets called MDS manager. The purposes of this app are to complement the already comprehensive publications of Building Blocks of Hope produced by the MDS Foundation. The app will have the capability to store MDS profile (IPSS-R scores, BM results), track bloods counts, transfusions and treatments, record symptoms, contacts of key professionals, medications, download reports, calendars for appointments and much more. Also, notifications can be sent to complete quality of life information and new clinical trials. Sandy is asking for interest to trial in centres internationally and we are looking forward to being involved in this initiative.

Other discussions and opinions revolved around looking at patient education in the light of the rapidly advancing science of MDS in identifying somatic mutations and the implications for the patients. Predominantly we discussed how we might best support patients in understanding this information and identifying the risks and benefits for them in having this information in understandable and translatable form.

As well as many excellent sessions in the scientific program, this meeting also offered a comprehensive nursing program which is described in this report.

CNS Janet Hayden was able to attend the MDS Symposium, thanks to a generous grant from the expert group in MDS – the UK MDS Forum – to whom she is very grateful for the opportunity.

The MDS Symposium takes place every two years. In 2015, our patient group, the MDS UK Patient Support Group, sponsored travel for 2 nurses (Janet Hayden and Geke Ong) to attend the Symposium in Washington.  www.mdspatientsupport.org.uk.

 

Our support opportunities are open to nurses nationwide, but unfortunately it is extremely difficult for these nurses to be granted time off or study leave – due to immense work pressure as well as to hospitals being short-staffed.

We urge hospitals and managers to allow nurses to attend such conferences – as it benefits everyone in the long term. Thank you.

If you are a nurse and are interested in our offers of support, please email us on mds-uk@mds-foundation.org


Realising the potential of haematology nurses and allied health professionals

Realising the potential of haematology nurses and allied health professionals

The Haematology Institute Nurses and Allied health Professionals Group is hosting a one day event on 10th July 2017 at the Robens Suite, 29th floor, Tower Wing, Guy’s Hospital London SE1 9RT. 

Read More


MDS UK tribute to Professor Rodney Taylor (1942-2017)

It is with immense sadness that we announce the death of our past Chairman, Professor Rodney Taylor on 3rd May 2017, after his MDS progressed to AML.

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MDS UK Patient Support Group Chairman

Rodney was diagnosed with MDS in 2006 and was invited to become Deputy Chairman when our little patient group was still in its infancy.

Following the death of the then Chairman David Hall, Rodney became Chairman in 2010 and thereafter dedicated an immense amount of his spare time working for and leading the group until late 2016, when it was clear his MDS had progressed and no further treatment was possible.

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Rodney's achievements for all MDS patients: NICE approval of Azacitidine

Rodney was an amazing, witty, gentle, very funny and extremely talented Chairman. MDS UK achieved an incredible amount of work under his guidance. The most significant achievement being the NICE approval of the drug Azacitidine in 2010. The MDS community as a whole owes him a huge debt of gratitude for this work which spanned 2 years.

You can read more about Rodney and his amazing career here: http://mdspatientsupport.org.uk/members/prof-rodney-taylor

During his time as Chairman, Rodney met with all stakeholders in the medical and pharmaceutical MDS sector and was immensely appreciated, liked and respected for his views, understanding, advice and personality across all professional and patient groups alike.

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Professor Rodney Taylor with an MDS Campaigner

Passionate about a good doctor-patient communication

Rodney had two topics he was particularly passionate about resulting from his being a medical doctor as well as a patient.

The first topic was the Doctor/Patient actual communication – as to him this was absolute key to a good consultation, relationship and care overall.
Rodney taught about this and one of his wishes was to re-instate regular courses for physicians about Advanced Communication Skills.
He knew this was a skill that can be improved – and could immensely assist clinicians and therefore also patients – especially in today’s stretched NHS.

Rodney's passion for Quality of Life for MDS patients

The second topic was equally close to his heart – Quality of Life (QOL) for patients. The fact that survival is not always the ultimate aim and that patients’ wishes, desires and situations can differ greatly and need to be respected at all times. Rodney believed that QOL must be measured and assessed on a regular basis at every point in a patient’s life – from diagnosis to death – and acted upon. Rodney believed totally in the QOL of MDS patients and followed his belief in his own treatment.

Rodney was treated with Azacitidine for almost 5 years before undergoing a stem cell transplant at age 71. Three years after the transplant his QOL of life had deteriorated and he did not wish to undergo any further experimental treatments that would have meant more intensive hospital care.

Despite a few serious side-effects over the course of those three years, Rodney carried on working with the CEO and the new Chair until the last few weeks of his life. He continued to give valuable advice, opinions and views on the way ahead for MDS UK, which was much appreciated and extremely helpful to the incoming Chair. His thoughts and views were always given in his caring, considerate style – and his lovely sense of humour.

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Euroblood Net
Euroblood Net

In Rodney Taylor's Memory

We at MDS UK have grown, learnt and improved thanks to Rodney’s leadership. His death is an immense loss to us. We will maintain our work and dedication to patient support and services – emphasising QOL and good communication at every opportunity - in his memory.

MDS UK was extremely important to him and the work we did was very close to his heart – and so were all the committee members and patients. He cared very deeply – and never missed an opportunity to express it. Equally – Rodney was very close to our heart too.

Rodney had not made his wishes known to MDS UK whilst alive, but he had arranged to have memorial donations directed to our organisation as an option.

We have therefore set up a page in his memory – where you can also leave a message which will be read by all family and friends:
http://uk.virginmoneygiving.com/SomeoneSpecial/RodneyTaylor

His death comes shortly after that of a previous MDS UK Committee Member, Sharon Berger, who passed away due to similar reasons, after a second transplant.

With the increased research being conducted into MDS we all hope, as Rodney did, that a real cure or at least improved treatments, are on the way for patients.

We thank all the friends, colleagues and patients who have sent in tributes and caring thoughts regarding Rodney. We are very grateful and will pass them on to his family.

We offer our most sincere and caring condolences to his wife Janet, to his children Alice, Beatrice and Romilly and to his many grand-children.

Rodney was a man of deep faith and quiet conviction. There is a Native American saying which says "when you were born you cried and the world rejoiced. Live your life so that when you die the world cries and you rejoice”. Rodney certainly lived his life well and I am sure has received a very warm welcome in heaven. May he rest in peace.

If you wish to leave a few words about Rodney, or a comment, tribute, thought – please email us on mds-uk@mds-foundation.org – with the Subject: In memory of Rodney.

We will then publish your comments on this page – with your name (if you wish), or anonymously if you prefer.

Very many thanks in advance.


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.

Learn More:
Science: Aug. 30, 2017
Modified T cells that attack leukemia become first gene therapy approved in the United States

Take a look at current MDS clinical trials


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