The wider perspective: twenty years of clinical trials in myelodysplastic syndromes

Carolien Duetz*, David G.J. Cucchi, Tobias B. Polak, Jeroen J.W.M. Janssen, Gert J. Ossenkoppele, Elihu H. Estey, Arjan A. van de Loosdrecht

*Corresponding author for this work

Research output: Contribution to journalComment/Letter to the editorAcademicpeer-review

8 Citations (Scopus)
37 Downloads (Pure)


Most patients with myelodysplastic syndromes (MDS) require therapeutic intervention. However, there are few approved treatments for MDS. To explore reasons, we searched and for MDS trials from 2000 to 2020. We assessed which agents were under investigation and analysed clinical trial characteristics and continuation rates from phase I to II to III to approval. As such, we identified 384 unique agents in 426 phase I, 430 phase II and 48 phase III trials. Success rates for phase III trials and agents were low, and MDS trials took markedly longer to complete than the average clinical trial. Although success rates were higher when MDS-specific phase I trials were conducted, 52% of the agents had not been evaluated in a phase I trial for MDS. MDS trials often failed to include quality of life, an especially important outcome for older MDS patients. Our work identifies factors potentially contributing to the paucity of available agents for MDS. We suggest a framework to improve clinical research in MDS that might ultimately augment the number of available agents.

Original languageEnglish
Pages (from-to)329-335
Number of pages7
JournalBritish Journal of Haematology
Issue number2
Early online date10 Oct 2021
Publication statusPublished - Jan 2022

Bibliographical note

Publisher Copyright:
© 2021 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.


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