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 

Subtype: Lower Risk MDS who are Red Blood Cell Transfusion dependent

Severity of MDS: IPSS-R Very Low, Low or Intermediate Risk

Name of drug: Luspatercept

Aims and benefits: To compare the effectiveness and safety of Luspatercept versus that of Erythropoiesis-stimulating agents (ESAs) in patients who are anaemic and require regular red cell transfusions and have never received ESAs in the past.

Luspatercept is a protein that has been found to promote 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 study is a Phase 3 Randomized Controlled Study in which there is a treatment period of a minimum of 24 weeks after which patients will be assessed for response to treatment. If a patient is receiving benefit they will continue on treatment and be reassessed every 24 weeks.

Primary outcome measures: To determine the proportion of patients who become red cell transfusion independent over the first 24 weeks of treatment.

Secondary outcome measures: To determine the proportion of patients who experience improvement in haemoglobin levels and red cell transfusion requirement and to assess improvements in quality of life.

Basic inclusion criteria:

- MDS with IPSS-R classification of very low, low or intermediate risk with <5% blasts

- Serum erythropoietin (sEPO) level of < 500 U/L

- Requires regular red cell transfusions

- ECOG performance of 0 – 2 at screening

Basic exclusion criteria:

- Prior treatment with:

-Erythropoiesis-stimulating agents (ESAs); Granulocyte colony-stimulating factor (GCSF), granulocyte-macrophage colony-stimulating factor (GM-CSF), unless given for treatment of febrile neutropenia; Disease modifying agents (eg, immune-modulatory drug [IMiDs such as lenalidomide]; Hypomethylating agents; Luspatercept (ACE-536) or sotatercept (ACE-011); Immunosuppressive therapy for MDS; Hematopoietic cell transplant

- MDS associated with del(5q) abnormality or MDS unclassifiable (MDS-U)

- Myelodysplastic/myeloproliferative neoplasms (MDS/MPN)

- Secondary MDS

- History of AML

- Specific treatment received in the preceding 8 weeks (including anticancer chemotherapy, steroid therapy, iron chelating therapy, Hydroxycarbamide). For other treatments included in this exclusion category please discuss with your Haematologist.

- Severe neutropenia or thrombocytopenia

- Significant kidney or liver abnormalities.

Trial sites:

- Aberdeen Royal Infirmary, Aberdeen

- Birmingham Heartlands Hospital, Birmingham

- Royal Bournemouth Hospital, Bournemouth

- University of Oxford, Headington

- Lincoln County Hospital, Lincoln

- Kings College Hospital, London

- Christie Hospital NHS Trust, Manchester

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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