Department of Paediatric Hematology, Oncology and Blood and Marrow Transplantation, Apollo Cancer Institutes, Chennai, India.
Department of Biostatistics, Apollo Cancer Institutes, Chennai, India.
Pediatr Transplant. 2021 Mar;25(2):e13893. doi: 10.1111/petr.13893. Epub 2020 Oct 27.
Alternate donor HSCT for thalassemia major from a matched unrelated donor or haploidentical family donor is a feasible therapeutic option in children with no matched family donor. Aggressive pretransplant immunosuppression, reduced toxicity conditioning, and PTCY result in excellent thalassemia-free survival. We describe here our experience in this cohort. We performed a retrospective analysis of the data on children who underwent a haploidentical HSCT for thalassemia major with PTCY at our center from August 2017 to August 2019. All children received pretransplant immune suppression for 6 weeks with fludarabine and dexamethasone, hypertransfusion and chelation with intravenous desferrioxamine. Conditioning included thiotepa, fludarabine, rabbit ATG, and cyclophosphamide, and GvHD prophylaxis included PTCY with tacrolimus. Twenty children were included and nineteen children engrafted. Acute hypertension occurred in five children, bacterial infection in eight children and viral respiratory infection in three children. Three children suffered from graft rejection. Reactivation of viruses namely CMV, adenovirus, and BK virus was seen in 60% of children. Grades 1-2 acute GvHD of the skin in four children (20%) and limited chronic GvHD of the skin in four children (20%). Immune cytopenia was documented in three children (15%). Haploidentical HSCT offers a therapeutic option for children with thalassemia major with no suitably matched family or unrelated donors. Our reduced toxicity regimen with PTCY offers a DFS of 75% and OS of 95% with low transplant-related mortality of 5%.
来自匹配的无关供体或半相合家族供体的异体供者 HSCT 可作为无匹配家族供体的重型地中海贫血患儿的可行治疗选择。强烈的移植前免疫抑制、降低毒性的调理以及 PTCY 可导致极好的无地中海贫血生存。我们在此描述了我们在该队列中的经验。我们对 2017 年 8 月至 2019 年 8 月在我们中心接受 PTCY 半相合 HSCT 治疗重型地中海贫血的儿童进行了回顾性数据分析。所有儿童均接受了 6 周的移植前免疫抑制治疗，方案为氟达拉滨和地塞米松，静脉注射去铁胺进行高输血和螯合。调理方案包括噻替哌、氟达拉滨、兔抗胸腺细胞球蛋白和环磷酰胺，GVHD 预防方案包括 PTCY 联合他克莫司。共纳入 20 例患儿，19 例患儿植入成功。5 例患儿发生急性高血压，8 例患儿发生细菌感染，3 例患儿发生病毒性呼吸道感染。3 例患儿发生移植物排斥。60%的患儿出现病毒再激活，包括 CMV、腺病毒和 BK 病毒。4 例患儿（20%）出现 1-2 级皮肤急性 GVHD，4 例患儿（20%）出现局限性皮肤慢性 GVHD。3 例患儿（15%）出现免疫性血细胞减少症。半相合 HSCT 为无合适匹配家族或无关供体的重型地中海贫血患儿提供了一种治疗选择。我们使用 PTCY 的降低毒性方案可提供 75%的DFS 和 95%的 OS，移植相关死亡率仅为 5%。
