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. Primary outcome measures:
    Assessing maximum tolerated dose of the study drug and the rate of complete response.
  2. Secondary outcome measures:
    To determine more information about how the drug works in the body including the rate at which the drug is spread throughout and removed from the bloodstream.
  3. Main basic inclusion criteria:
    • Patients with Acute Myeloid Leukaemia or Myelodysplastic Syndrome which is relapsed or refractory to standard therapy and/or for which standard therapy is contraindicated or which has not adequately responded to standard therapy.
    • Patients with an IDH1 R132 mutation
    • Good performance status
    • Good kidney and liver function
  4. Main basic exclusion criteria:
    • Patients with symptomatic central nervous system (CNS) metastases or other tumour location (such as spinal cord compression, other compressive mass, uncontrolled painful lesion, bone fracture, etc.) necessitating an urgent therapeutic intervention, palliative care, surgery or radiation therapy
    • Heart disease including New York Heart Association Class III or IV heart failure, unstable angina pectoris, previous history of myocardial infarction within 1 year
    • Uncontrolled pulmonary disease
    • Active uncontrolled infection
  5. Trial sites/locations and name of the physician in charge of trial:

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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