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Updated: Oct 17, 2025
Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
The wider perspective: twenty years of clinical trials in myelodysplastic syndromes
Carolien Duetz1, David G J Cucchi1, Tobias B Polak2
1Department of Hematology, Amsterdam UMC, location VUmc, Vrije Universiteit, Cancer Center Amsterdam, Amsterdam, The Netherlands.
Abstract:
Most patients with myelodysplastic syndromes (MDS) require therapeutic intervention. However, there are few approved treatments for MDS. To explore reasons, we searched clinicaltrials.gov and clinicaltrialsregister.eu 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.
Insights
Few treatments exist for myelodysplastic syndromes (MDS) despite most patients needing intervention. This study analyzed MDS clinical trials, finding low success rates and lengthy trial durations, suggesting improvements for drug development.
Area of Science:
- Hematology
- Clinical Research
- Drug Development
Background:
- Myelodysplastic syndromes (MDS) are a group of blood cancers requiring treatment for most patients.
- There is a significant unmet need for effective therapeutic interventions in MDS due to limited approved treatments.
Purpose of the Study:
- To investigate the reasons behind the limited number of approved treatments for MDS.
- To analyze the characteristics, progression, and success rates of MDS clinical trials conducted between 2000 and 2020.
Main Methods:
- A comprehensive search of clinicaltrials.gov and clinicaltrialsregister.eu was performed for MDS trials.
- Data on unique agents, trial phases (I, II, III), continuation rates, and trial completion times were collected and analyzed.
- The study assessed the impact of MDS-specific phase I trials and the inclusion of quality of life outcomes.
Main Results:
- 384 unique agents were identified across 426 phase I, 430 phase II, and 48 phase III trials.
- Phase III trials and agents demonstrated low success rates, with MDS trials taking longer than average.
- 52% of agents lacked prior MDS-specific phase I evaluation, and quality of life measures were often omitted.
Conclusions:
- Factors contributing to the scarcity of approved MDS agents include low trial success rates, long trial durations, and suboptimal trial design.
- Conducting MDS-specific phase I trials and incorporating quality of life assessments could improve research efficiency.
- A proposed framework aims to enhance MDS clinical research and increase the availability of therapeutic agents.
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