作者: Marie Robin , Raphael Porcher , Lionel Ades , Emmanuel Raffoux , Mauricette Michallet
DOI: 10.1182/BLOOD.V122.21.301.301
关键词: Prospective cohort study 、 Surgery 、 Regimen 、 Transplantation 、 Busulfan 、 Medicine 、 Comorbidity 、 Hematopoietic stem cell transplantation 、 Internal medicine 、 Thymoglobulin 、 Fludarabine
摘要: ![Graphic][1] Background Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for MDS patients (pts). Due to transplant-related mortality, pts with short life expectancy are referred HSCT, usually those IPSS int-2 or high risk. The aim of this prospective study was compare outcome in candidates HSCT according donor availability: no donor, HLA-matched sibling 10/10 unrelated and 9/10 HLA-mismatched donor. Method SFGM-TC GFM centers that had agreed on general recommendations concerning management participated (16 centers). Transplant indications were IPSS, int-1 refractory thrombocytopenia proliferative CMML. Pts registered if older than 50 years, when they acquired an indication absence comorbidity contraindicating HSCT. A research initiated at registration including (10/10) mismatched one allele. Other alternative donors (mismatched cord blood > 1 donor) not accepted. Transplantation scheduled upfront bone marrow blasts 10% ideally within 6 months a identified. Recommended reduced intensity conditioning regimen consisted fludarabine, busulfan anti-thymoglobulin peripheral cells (PB) as source cells. Characteristics disease compared 3 groups: (sibling 10/10), HLA-one (9/10). Overall free survivals (OS, DFS) using Kaplan Meier estimates. Cumulative incidences complete remission disease-related mortality Gray-test. Results From April 2007 January 2013, 163 included: 34 (21%) donor; 115 (71%) (34% 37% unrelated) 14 (9%) HLA donor. Groups well-balanced age, gender, time from diagnosis inclusion, WHO classification, cytogenetic (IPSS) classification. classification inclusion was: AML post 12, RAEB1 29, RAEB2 82, CMML1 2 20, RCMD other pts. Cytogenetics favorable 49 (30%), intermediate 37 (23%) poor 74 (45%) 8%, 69% 23% pts. Median follow-up 38 months. 117 treated by AZA 40 CT. Bone 3, 20% 20 pts), comorbidities after (9 early deaths (7 pts) causes Four have been transplanted yet. Probability 12 39% (95% CI: 22-55), 49% (95%CI: 40-58) 46% 17-71) without HLA-compatible Disease-related 48 significantly different % 29-68), 38% 27-49), 51% 17-77). DFS also 18% 7-44), 25% 16-37) 9% 1-57). In contrast, OS better donor: 35% (26-49), 17% (6-43) 8% (1-55), respectively, p=0.011 ([Figure 1][2]). survival observed may be due their small number requires further analysis larger cohort. Outcome receiving PB matched donor. ![Figure 1][3] Figure Conclusion study, we risk improved This confirmed should before acquisition HSCT. Disclosures: No relevant conflicts interest declare. [1]: /embed/inline-graphic-2.gif [2]: #F1 [3]: pending:yes