Conditioning regimens for HSCT for patients with high-risk myelodysplastic syndrome and secondary acute myeloid leukaemia
Mené en Chine sur 202 patients atteints d'une anémie réfractaire avec excès de blastes ou d'une leucémie myéloïde aiguë secondaire (syndrome myélodysplasique ayant évolué vers une leucémie) et recevant une greffe allogénique de cellules souches hématopoïétiques, cet essai de phase III évalue l'efficacité, du point de vue de l'incidence cumulée de récidive à 2 ans, et la toxicité de l'ajout de facteurs de stimulation des colonies de granulocytes (G-CSF) et de décitabine à un traitement de conditionnement par busulfan et cyclophosphamide
Myelodysplastic syndrome refers to a group of blood disorders characterised by clonal expansion of mutated blood cells, which can progress into secondary acute myeloid leukaemia. The median overall survival of patients with myelodysplastic syndrome was 1·7 years (95% CI 1·0–3·4) for patients with high-risk disease and 1·0 years (0·5–1·8) for patients with very-high-risk disease, in one prognostic study. 1 The only potentially curative therapy for high-risk myelodysplastic syndrome and secondary acute myeloid leukaemia is allogeneic haematopoietic stem-cell transplantation (HSCT), after which the 5-year overall survival was 39% for patients with high-risk myelodysplastic syndrome and was 23% for patients with very-high-risk myelodysplastic syndrome. 2 Relapse is the most common cause of treatment failure after allogeneic HSCT. Numerous factors influence relapse, including the International Prognostic Scoring System (IPSS)-based risk stratification, genomic profiling, conditioning regimen, stem-cell source, and optimal timing of HSCT. Of these, the choice of conditioning regimen is the primary therapeutic option to minimise the residual leukaemia burden to reduce disease recurrence. Several retrospective studies showed that overall survival was similar with different conditioning intensities and no ideal conditioning regimen has been identified, 3 a problem that needs to be resolved urgently.
The Lancet Haematology , commentaire, 2022