MDS Current Trials UK

We aim to list an easily understandable description of any MDS clinical trials currently opened to recruitment in the UK.

Patients will be able to read up on the details of these trials and use this information to discuss with their consultants or nurses.

Not all patients can be enrolled in drug trials - as not every patient will fit the necessary inclusion criteria, which need to be followed very strictly. However, if a particular clinical trial drug is considered an option by the haematologist, it is sometimes possible to obtain the drug outside the trial, on the basis of compassionate use.

All the trials listed in this page have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust 

ACE-536-MDS-002 (Luspatercept)

  1. SUB-TYPE OF MDS:Lower Risk MDS patients who require red cell transfusions but have not received Erythropoietin
  2. SEVERITY OF MDS: IPSS-R Very Low, Low or Intermediate Risk MDS
  3. NAME OF DRUG: Luspatercept
  4. Aims and benefits: To compare the safety and efficacy of Luspatercept versus epoetin alfa for treating anaemia in patients who require red cell transfusions.
    Luspatercept promotes red blood cell formation by regulating the growth of red blood cells during the late-stage of their development. It works differently to erythropoietin.
    This is a Phase 3 Randomized Study between Luspatercept and Epoetin alfa.
  1. Primary outcome measures: 
    To determine the proportion of patients who become red cell transfusion free over the first 24 weeks from randomization.
  2. Secondary outcome measures:
    To establish the length of time it takes for patients to achieve improvements in haemoglobin and determine information about adverse events and how Luspatercept is distributed within the body.
  3. Basic inclusion criteria:
    • IPSS-R classification of very low, low, or intermediate risk disease, and < 5% blasts in the bone marrow
    • Serum erythropoietin level of < 500 U/L.
    • Require regular red cell transfusions
    • ECOG performance of 0 – 2 at screening
  4. Basic exclusion criteria:
    • Prior treatments with Erythropoiesis-stimulating agents (ESAs), Granulocyte colony-stimulating factor (G-CSF), Disease modifying agents (eg, immune-modulatory drug such as lenalidomide (with some exceptions), Hypomethylating agents (with some exceptions), Luspatercept (ACE-536) or sotatercept (ACE-011), Immunosuppressive therapy for MDS, stem cell transplant
    • Myelodysplastic/Myeloproliferative neoplasms (MDS/MPN) overlap syndromes
    • Diagnosis of secondary MDS
    • Significant myelofibrosis
    • MDS associated with 5q(del) abnormality
    • History of diagnosis of AML
    • Clinically significant anaemia due to other causes (unrelated to MDS)
  5. Trial sites/locations and name of physician in charge of trial (none are currently recruiting):
    • Aberdeen Royal Infirmary
    • Birmingham Heartlands Hospital
    • Royal Bournemouth Hospital
    • University of Oxford Headington
    • The Leeds Teaching Hospitals NHS Trust
    • St James's University Hospital
    • Lincoln County Hospital
    • Kings College Hospital
    • Christie Hospital NHS Trust

Please read information and always discuss trial information with your own physician.

Paper of the Month

Venetoclax and hypomethylating agents (HMAs) induce high response rates in MDS, including patients after HMA therapy failure

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IPSS-R MDS Risk Assessment Calculator

Revised International Prognostic Scoring System (IPSS-R) for Myelodysplastic Syndromes Risk Assessment Calculator

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MDS Fact Sheet

MDS Factsheet for GPs General Practicioners

MDS Fact Sheet

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Varicella Vaccine in MDS

Urgent Clarification on varicella vaccine policy

Current advice from the UK MDS Forum is not to give the Live Varicella Vaccine to patients with MDS.

Please read below correspondence between Professor Salisbury and Dr George Follows, the latter on behalf of the CLL Forum, on the varicella vaccine policy.
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NICE Guideline

Lenalidomide for treating myelodysplastic syndromes (MDS) associated with an isolated deletion 5q cytogenetic abnormality.

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BSH Guidelines

This is an external link to the home of the British Society for Haematology (BSH). 

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