Transplantation in patients with lower-risk MDS: a prospective phase 2 trial based on donor availability

Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only potential curative therapy for myelodysplastic syndrome (MDS), recommended in higher risk disease according to the International Prognostic Scoring System (IPSS). We conducted a phase 2 multicenter trial (MDS-ALLO-RISK) investigating whether allogeneic HSCT improves overall survival (OS) in patients with lower-risk MDS who exhibit additional high-risk features (intermediate or higher revised-IPSS risk, thrombocytopenia with <20 × 109/L, neutropenia <0.5 × 109/L, or failure to 2 lines of therapy). A total of 77 patients (median age, 62.5 years) with low or intermediate-1 IPSS scores were enrolled and stratified based on the presence of a matched HLA donor, 62 patients in the donor arm and 15 without a donor. Despite high remission rates in patients who had received a transplant (67.8% vs 21.4%), the 3-year OS did not significantly differ between arms (57.6% in the donor arm vs 64.3% in the no-donor arm; hazard ratio, 0.75; P = .53). The adjusted analysis using inverse probability of treatment weighting confirmed the lack of survival benefit with HSCT. Transplantation was associated with higher rates of chronic graft-versus-host disease, severe infections, and nonrelapse mortality (24.7%). Although quality of life improved slightly over time in patients who had received a transplant, the difference was not statistically significant. The trial was stopped early due to slow enrollment and futility. The findings highlight the need for improving posttransplant outcomes to justify HSCT in patients with lower-risk MDS with poor prognostic features. This trial was registered at www.clinicaltrials.gov as #NCT02757989.

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Journal
Blood Advances
Published
2025-10-08
DOI
https://doi.org/10.1182/bloodadvances.2025017035
Primary Topic
Acute Myeloid Leukemia Research
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article
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article

Transplantation in patients with lower-risk MDS: a prospective phase 2 trial based on donor availability

Lionel Adès, Marie Robin, Sylvain Thépot, Stéphanie Nguyen et al.
Blood Advances
Acute Myeloid Leukemia Research
article

Transplantation in patients with lower-risk MDS: a prospective phase 2 trial based on donor availability

Lionel Adès, Marie Robin, Sylvain Thépot, Stéphanie Nguyen, Michael L. Loschi, Odile Beyne Rauzy, Claude Preudhomme, Pierre Péterlin, Cendrine Chaffaut, Thibaud Lefèbvre, Fatiha Chermat, Sophie Park, Patrice Chevallier, Anne Huynh, Rosa Sapena, Patrice Céballos, Sylvie Chevret, A Charbonnier, Thomas Cluzeau, Régis Peffault de Latour, Gaëlle Fossard, Maud D’Aveni, Nicolas Duployez, Corentin Orvain, Marie‐Thérèse Rubio, Marie Sébert, Pierre Fenaux
article en

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only potential curative therapy for myelodysplastic syndrome (MDS), recommended in higher risk disease according to the International Prognostic Scoring System (IPSS). We conducted a phase 2 multicenter trial (MDS-ALLO-RISK) investigating whether allogeneic HSCT improves overall survival (OS) in patients with lower-risk MDS who exhibit additional high-risk features (intermediate or higher revised-IPSS risk, thrombocytopenia with <20 × 109/L, neutropenia <0.5 × 109/L, or failure to 2 lines of therapy). A total of 77 patients (median age, 62.5 years) with low or intermediate-1 IPSS scores were enrolled and stratified based on the presence of a matched HLA donor, 62 patients in the donor arm and 15 without a donor. Despite high remission rates in patients who had received a transplant (67.8% vs 21.4%), the 3-year OS did not significantly differ between arms (57.6% in the donor arm vs 64.3% in the no-donor arm; hazard ratio, 0.75; P = .53). The adjusted analysis using inverse probability of treatment weighting confirmed the lack of survival benefit with HSCT. Transplantation was associated with higher rates of chronic graft-versus-host disease, severe infections, and nonrelapse mortality (24.7%). Although quality of life improved slightly over time in patients who had received a transplant, the difference was not statistically significant. The trial was stopped early due to slow enrollment and futility. The findings highlight the need for improving posttransplant outcomes to justify HSCT in patients with lower-risk MDS with poor prognostic features. This trial was registered at www.clinicaltrials.gov as #NCT02757989.

Blood AdvancesVol. 10(2)
Inserm (FR), Université Fédérale de Toulouse Midi-Pyrénées (FR), Université Paris Cité (FR), Centre Hospitalier Universitaire de Grenoble (FR), Centre Hospitalier Universitaire de Lille (FR), Centre Hospitalier Universitaire de Nice (FR), Centre Hospitalier Universitaire de Nantes (FR), Centre Hospitalier Universitaire de Toulouse (FR), Sorbonne Université (FR), Hôpital Louis-Mourier (FR), Centre Hospitalier Universitaire de Montpellier (FR), Centre Hospitalier Universitaire Amiens-Picardie (FR), Assistance Publique – Hôpitaux de Paris (FR), Institut universitaire du cancer de Toulouse Oncopole (FR), Centre Hospitalier Régional et Universitaire de Nancy (FR), Centre Hospitalier Universitaire d'Angers (FR), Hôpital Lyon Sud (FR), Pitié-Salpêtrière Hospital (FR), Groupe Francophone des Myélodysplasies (FR), Hôpital Saint Eloi (FR), Hôpital Saint-Louis (FR), Université d'Angers (FR), Université Grenoble Alpes (FR), Université de Lorraine (FR), Nantes Université (FR)
Openalex Percentile: Top 64%
Acute Myeloid Leukemia Research
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