1. Alemtuzumab-based conditioning regimen before hematopoietic stem cell transplantation in patients with short telomere syndromes: a retrospective study of the SFGM-TC.
- Author
-
Hussein-Agha R, Kannengiesser C, Lainey E, Marcais A, Srour M, Sterin A, Buchbinder N, Borie R, Plessier A, Socié G, Peffault de Latour R, and Sicre de Fontbrune F
- Subjects
- Humans, Retrospective Studies, Adult, Male, Female, Middle Aged, Adolescent, Young Adult, Vidarabine analogs & derivatives, Vidarabine therapeutic use, Vidarabine administration & dosage, Telomere Shortening, Alemtuzumab therapeutic use, Alemtuzumab pharmacology, Alemtuzumab administration & dosage, Transplantation Conditioning methods, Hematopoietic Stem Cell Transplantation methods
- Abstract
While HSCT is the only curative option for patients with short telomere syndromes (STSs) and severe bone marrow failure (BMF) or myeloid malignancies (MM), their increase sensitivity to conditioning regimen strongly affect outcomes. To minimize HSCT related mortality, alemtuzumab-based conditioning regimens have been proposed, but the number of patients transplanted with those regimens reported in the literature remains very low. We retrospectively analyzed outcome of adults and adolescents with STSs transplanted after an alemtuzumab, fludarabine and cyclophosphamide based regimen registered by the SFGM-TC. Seven patients were transplanted for a BMF and 5 for a MM (median age 34 years, (IQR [22-45])). The 2-year GRFS for patients with MM was 20% (95% CI [3;100]), and 57% (95% CI [30;100]) in others. In univariate (hazard ratio, HR = 6, 95% CI [1;31]) and multivariate analysis (HR = 26, 95% CI [2;414]) stem cell source was a predictive factor for GRFS. Three of the 5 patients with pre-transplant MM relapsed and 2 of them died at last follow up. The 2-year OS was 66% (95% CI [43;99]) in the whole cohort with a median follow up of 32 months (IQR [13-56]). In conclusion, Alemtuzumab-based conditioning regimen with bone marrow is an option for patients with STSs and BMF, but others modalities have to be explored for patients with MM., (© 2024. The Author(s), under exclusive licence to Springer Nature Limited.)
- Published
- 2024
- Full Text
- View/download PDF