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 

  1. SUB-TYPE OF MDS: Higher risk MDS, CMML, low blast count AML
  2. SEVERITY OF MDS: Intermediate, high risk or very high risk MDS as defined by the World Health Organization (WHO) criteria or Revised International Prognostic Scoring System (IPSS-R)
  3. NAME OF DRUG: Azacitidine, Pevonedistat
  4. Aims and benefits: Pevonedistat prevents the activity of a specific enzyme (Nedd8 activating enzyme) and thus may result in the inhibition of tumour cell growth and survival. This is a phase 3 study to determine if combining Pevonedistat with Azacitidine improves survival when compared with single agent Azacitidine.
  1. Primary outcome measures: 
    To determine event-free survival, an event being progression to AML or death.
  2. Secondary outcome measures:
    These include the assessment of complete remission rates, overall survival as well as survival rates at different time points.
  3. Basic inclusion criteria:
    • Higher risk MDS, CMML, low blast count AML.
    • Participants with AML (20%-30% blasts) must have a treatment-related mortality (TRM) score >=4 for intensive, induction chemotherapy as calculated using the simplified model described by Walter and coworkers.
    • - ECOG performance status 0-2.
  4. Basic exclusion criteria:
    • Previous treatment for higher risk MDS, CMML or low blast count AML. Treatment with hydroxycarbamide or Lenalidomide is permitted.
    • Eligible for intensive chemotherapy and/or allogeneic stem cell transplant.
    • CNS involvement by AML.
    • Active uncontrolled infection
    • Hepatic impairment
    • Cardiopulmonary disease
  5. Trial sites/locations and name of the physician in charge of trial:
    • Royal Bournemouth Hospital, Bournemouth
    • Maidstone Hospital, Maidstone, Kent
    • St. Bartholomew’s Hospital, London
    • Singleton Hospital, Swansea

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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