Haematology conference updates – Part 1

15 Jan. 2015

Further updates on clinical and research news from the two main haematology conferences in Europe and the USA.

Part 1 – Europe – from the EHA conference
Part 2 – International – from the ASH conference (report in preparation)

Report provided by
Dr Wendy Ingram
Consultant Haematologist, University Hospital of Wales, Cardiff

Report from the 19th Congress of the European Haematology Association (EHA), Milan, June 2014

The EHA Congress continues to attract international experts and delegates from across the globe providing the opportunity to exchange cutting edge scientific and clinical data.   With respect to MDS, significant advances have been made in the understanding of the biology of the disease and as such, remained the focus of this year’s meeting.

MDS Biology

An excellent educational session led by three key international MDS speakers provided an overview of the biology and genetic mutations implicated in MDS.  Technological advances have enabled the scientific community to identify many of the gene mutations involved in MDS, although the precise mechanism of how such genes cause disease remains unanswered.
Many of these genes are currently not tested for in routine practice.  The data presented supports the need to move towards integrating such developments, in order to help both with the diagnosis and with treatment decisions.
Some of the gene mutations have been shown to correlate with prognosis as well as response to therapy. One example of which is the TET2 mutation, which has been shown to predict a better response to azacitidine or decitabine therapy.
On the contrary, other mutations correlate with a poorer response to therapy in particular following allogeneic stem cell transplantation.
Current studies in larger groups of patients are being conducted to provide us with more knowledge of how and when such gene mutations should be used in daily practice.

Prognostic Scoring Systems

Many of you will be familiar with the newer prognostic scoring systems such as the IPSS-R (revised international prognostic scoring sytem) and WPSS (WHO prognostic scoring system).  Data was presented at both the educational and poster sessions supporting their continued use as they predict both survival and risk of transforming to leukaemia.  Furthermore, a correlation between prognostic score and outcome following transplantation was described.  As such, the use of prognostic scores remains an invaluable tool to guide clinicians and patients alike during the often difficult time of when to commence therapy.

The response to azacitidine remains difficult to predict.  An azacitidine prognostic score has been developed which correlates with the likelihood of responding to therapy.  Larger studies are required to support its use in routine practice.  Such scores will enable us to better predict if a therapy is likely to benefit a particular patient.

Advances in MDS Therapy

The older age of many MDS patients and frequent co-existing medical illnesses continues to be a challenge when treating MDS.  As such, supportive care with blood transfusions and treatment of infections remains the mainstay of therapy.  Enrolment into clinical trials often proves difficult as a result of the issues described above.  Nevertheless, despite current treatments being limited, new therapies are being explored.

With regards to lower risk MDS several clinical studies being conducted are focused on improving the blood count (anaemia) and reducing the need for blood transfusions.  One such drug called Rigosertib is an oral agent, which has shown promising results in early clinical studies.
Another exciting and promising agent called ACE-536 has been developed for the treatment of anaemia. Again a reduction in transfusions and improved blood counts has been observed.  Both of these agents require further studies in larger groups of patients.

An oral form of azacitidine is also being explored in lower risk MDS patients who are transfusion dependent and who have a low platelet count.  This study is currently recruiting is selected centres in the UK.

Drugs which increase the platelet count, (Romiplostim and Eltrombopag) also remain in clinical trial development and show promise in early studies.

Azacitidine remains the only licensed therapy in higher risk MDS.  Many of the studies in this group of patients are focused on the addition of agents to azacitidine.  An example of which is the ALLG MDS4 study, which is evaluating the outcome of dual therapy (lenalidomide and azacitidine).  The results of such studies are awaited.

I remain excited by the developments in the field of MDS and look forward to reporting any further developments over forthcoming years.
Dr Wendy Ingram

We thank Dr Ingram for her contribution, help and valuable time getting this information to us.

We are now awaiting reports from several other physicians on the more recent ASH congress – for Part 2 of this research update.

If you are a haematologist, researcher or nurse working in the field of MDS – and would like to report back to us on any conference you have attended, please contact us.  We are always keen to provide our patients with some clear and practical information regarding on-going research.
Please contact us on mds-uk@mds-foundation.org