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:Patients newly diagnosed with CD123 positive adverse genetic risk acute myeloid leukaemia (AML), including patients with CD123 positive AML secondary to MDS
  2. SEVERITY OF MDS: AML
  3. NAME OF DRUG: UCART targeting CD123 positive leukaemia cells
  4. Aims and benefits: This is a phase 1 first in human study to determine the safety and effectiveness of multiple infusions of UCART123 (specially engineered T-cells that target leukaemia cells that express CD123).
  1. Basic inclusion criteria:
    • Aged 18 - 65 years
    • Weight ≥42 kg
    • Patients must have received 1 of the 2 trial-stipulated induction chemotherapy regimens
    • Availability of a suitable sibling or unrelated HLA matched donor
    • ECOG performance status of 0 or 1
  2. Basic exclusion criteria:
    • Patients with ≥ 20% blasts in bone marrow after 1 or 2 courses of standard intensive induction chemotherapy
    • Patients with AML transformed from previously diagnosed myeloproliferative disorder
    • Patients with APL
    • Evidence of CNS leukaemia on CSF examination
    • AML relapsing after first complete remission
    • Prior allogeneic stem cell transplant
    • Therapy-related AML
    • Favourable or intermediate risk AML
    • Prior gene or experimental cellular therapy
    • HIV/HBV/HCV Infection
  3. Trial sites/locations and name of physician in charge of trial (none are currently recruiting):
    • Kings College Hospital

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.
This is an external link to the British Society for Haematology  Click here

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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