A Phase 2 interventional study of Hematopoietic stem-cell transplantation in Myelodysplastic Syndromes, sponsored by Assistance Publique - Hôpitaux de Paris. Not yet recruiting. Open to participants aged 50 Years to 70 Years. Per ClinicalTrials.gov, last updated 2024-01-31.
Sponsored by Assistance Publique - Hôpitaux de Paris · Phase 2, Interventional, and Treatment
Three recent prospective "transplant/no transplant" studies concluded to an advantage of OS with transplantation in patients with high or intermediate-2 IPSS risk (not significant in Kröger's study). No prospective randomized trial has assessed the pre-transplant therapy in MDS patients yet but some information can be extracted from these 3 recent studies. In the French study (n=162), 72% patients with a donor received HSCT, previously treated by hypomethylating agent (HMA) in 71% of them. There was a trend to a better survival in patients achieving a complete remission with pre-graft therapy (HR: 0.55, p=0.088) and higher risk of death in unresponsiveness patients transformed into AML (HR: 2.36, p=0.008). In Nakamura's study (n=384), 83% of patients with a donor were transplanted, previously treated by HMA in 68%2. The multivariable Cox model for Overall Survival (OS) and Leukemia-free survival showed an excess risk in patients treated by HMA. Moreover, responders still have a higher risk of mortality as compared to patients who did not receive any pre-graft therapy (HR: 2.417, p=0.0054). In the German study, the aim was to initiate azacytidine at inclusion and to transplant patients after 4 cycles if a donor was identified1. Among 170 registered patients, 162 initiated 5-aza but 36% of them were "lost during this pre-graft therapy" before allocation to "donor" or "no-donor" arm, for different reasons including death (n=12). After 4 cycles of 5-aza, 79/81 patients "donor arm" were transplanted. The multivariable analysis showed remission status did not influence OS. Those 3 previous clinical trials thus suggest that a substantial number of patients planned for transplantation are not transplanted nowadays while no evidence of HMA benefit before HSCT has been clearly identified. This phase 2 study aim to assess the feasibility of upfront HSCT in patients with high risk MDS in order to increase the probability to be transplanted and to achieve a subsequent remission and better survival.
2,124 studies on the registry are indexed under Myelodysplastic Syndromes; 320 are open to participants now.
This study's planned enrollment of 55 is above the median of 39 across 1,740 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,505 studies on the registry; 1,006 are open to participants now.
Counted across the registry records on this site, refreshed daily.
The disease fulfills at least one of the following criteria:
Usual criteria for Hematopoietic Stem Cell Transplantation (HSCT):
Exclusion Criteria:
Adults (Age ≥ 50 and ≤ 70 years) patients with MDS diagnosis for whom transplantation is indicated from a related donor identified.
Biological: Hematopoietic stem-cell transplantation
Upfront related donor transplantation
Disease-free survival
Time frame: 2 years after transplantation
Overall survival
Time frame: 2 years after transplantation
Non-relapse mortality
Time frame: 2 years after transplantation
Cumulative incidence of transformation into acute myeloid leukemia from inclusion
Time frame: 2 years after inclusion
Incidence of acute Graft versus Host Disease (GvHD) and grading
Time frame: 100 days after transplantation
Incidence of chronic GvHD and grading
Time frame: 2 years after transplantation
Percentage of engraftment
Engraftment is defined by hematological recovery and donor chimerism \> 95%
Time frame: 3 months after transplantation
Percentage of graft failure
Graft failure is defined by acute or late rejection and non-engraftment
Time frame: 2 years after transplantation
Incidence of severe infections
Severe infections are defined by Common Terminology of Adverse Events (CTAE) grade 3-4
Time frame: 3 months after transplantation
Incidence of severe infections
Severe infections are defined by Common Terminology of Adverse Events grade 3-4
Time frame: 6 months after transplantation
Incidence of severe infections
Severe infections are defined by Common Terminology of Adverse Events grade 3-4
Time frame: 12 months after transplantation
Incidence of severe infections
Severe infections are defined by Common Terminology of Adverse Events grade 3-4
Time frame: 24 months after transplantation
Incidence of cardiac events
CTAE grade 2-4
Time frame: 1 month after transplantation
Incidence of cardiac events
CTAE grade 2-4
Time frame: 3 months after transplantation
No study locations are listed for this record.
Plan to share: Undecided
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Aug 2023. You cannot join it, but the record below documents what was studied.
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Assistance Publique - Hôpitaux de Paris