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: MDS with 10% bone marrow blasts OR more; also acute myeloid leukaemia
    2. SEVERITY OF MDS: Intermediate – 2 or High Risk
    3. NAME OF DRUG: Mylotarg, CPX-351 (a novel drug which combines two anti-cancer drugs - cytarabine and daunorubicin in a new formulation)
  1. Aims and benefits: This trial is designed for adult patients aged 18 to 60 years but also for patients aged 60 years or over for whom intensive therapy is considered appropriate. It aims to answer several therapeutic questions including
    • To determine if a new dosing schedule of Mylotarg (two doses) is better than the current standard of a single dose.
    • Is survival improved by different chemotherapy regimens (eg. FLAG-Ida + Mylotarg vs DA + Mylotarg).
    • Is the novel drug CPX-351 better than FLAG-Ida when used in patients considered to be at high risk of relapse or in those found to have poor risk cytogenetics.
  2. Basic inclusion criteria:
    • Acute myeloid leukaemia or high risk Myelodysplastic Syndrome (>10% bone marrow blasts)
    • 18 years up to the age of 60, but patients over this age are eligible if intensive therapy is considered a suitable option.
    • Suitable for intensive chemotherapy
    • Adequate kidney function
  3. Basic exclusion criteria:
    • Patients who previously received demethylation treatment (eg. Azacitidine) for high risk MDS (>10% blasts)
    • Previous chemotherapy for acute myeloid leukaemia
    • Concurrent active malignancy excluding basal cell carcinoma
    • Pregnant or lactating
    • Known infection with human immunodeficiency virus (HIV)
  4. Trial sites/locations and name of physician in charge of trial: 
  5. More information:

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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