Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico, Bambino Gesù Children's Hospital, Piazzale S Onofrio, Rome, Italy.
Blood. 2012 Jul 12;120(2):473-6. doi: 10.1182/blood-2012-04-423822. Epub 2012 May 29.
Sixty thalassemia patients (median age, 7 years; range, 1-37) underwent allogeneic hematopoietic stem cell transplantation (HSCT) after a preparation combining thiotepa, treosulfan, and fludarabine. Before HSCT, 27 children were assigned to risk class 1 of the Pesaro classification, 17 to class 2, and 4 to class 3; 12 patients were adults. Twenty patients were transplanted from an HLA-identical sibling and 40 from an unrelated donor. The cumulative incidence of graft failure and transplantation-related mortality was 9% and 7%, respectively. Eight patients experienced grade II-IV acute GVHD, the cumulative incidence being 14%. Among 56 patients at risk, 1 developed limited chronic GVHD. With a median follow-up of 36 months (range, 4-72), the 5-year probability of survival and thalassemia-free survival are 93% and 84%, respectively. Neither the class of risk nor the donor used influenced outcome. This treosulfan-based preparation proved to be safe and effective for thalassemia patients given allogeneic HSCT.
60 例地中海贫血患者（中位年龄，7 岁；范围，1-37 岁）在接受硫替派、曲替拉滨和氟达拉滨联合预处理后接受了异基因造血干细胞移植（HSCT）。在 HSCT 前，27 例患儿被分配到佩萨罗分类的 1 类风险，17 例分到 2 类，4 例分到 3 类；12 例为成人。20 例患者接受 HLA 完全匹配的同胞供者移植，40 例接受无关供者移植。移植物失败和移植相关死亡率的累积发生率分别为 9%和 7%。8 例患者发生 II-IV 级急性移植物抗宿主病，累积发生率为 14%。在 56 例有风险的患者中，1 例发生局限性慢性移植物抗宿主病。中位随访 36 个月（范围，4-72），5 年生存率和无地中海贫血生存率分别为 93%和 84%。风险类别和供者使用均未影响结果。这种基于曲替拉滨的预处理方案对于接受异基因 HSCT 的地中海贫血患者是安全有效的。
