Division of Hematologic Malignancies and Cellular Therapy, Department of Medicine, Duke University School of Medicine, Durham, North Carolina, USA.
Department of Internal Medicine, Faculty of Medicine.
Curr Opin Hematol. 2019 Nov;26(6):399-405. doi: 10.1097/MOH.0000000000000545.
Discussing the currently available HSCT options for Hb SS patients highlighting advantages and disadvantages of each modality in the light of recently published data.
When MSD is available, myeloablative regimen is the preferred approach for otherwise healthy children whereas the nonmyeloablative (NMA) regimen is of choice for adults as well as children with SCD-associated morbidities. Mixed chimerism is common especially with NMA conditioning and is usually enough for cure. Alternative donor HSCT outcomes are progressively improving especially with posttransplant cyclophosphamide for GVHD prophylaxis.
Recent studies comparing HSCT and chronic transfusion in Hb SS patients increasingly come in favor of HSCT arm. Advances in HSCT field led to donor pool expansion and better tolerated regimens. It is easier now to tailor a personalized transplantation plan for almost every patient. A successful management plan should be sufficiently comprehensive addressing patients' and families' social and psychological concerns to ensure compliance and improve outcome.
讨论目前可供 Hb SS 患者选择的 HSCT 方案,根据最近发表的数据,强调每种方案的优缺点。
当 MSD 可用时,清髓性方案是健康儿童的首选,而非清髓性(NMA)方案是伴有 SCD 相关合并症的成人和儿童的首选。嵌合状态很常见,尤其是在 NMA 预处理时,通常足以治愈。异基因 HSCT 的结果在不断改善,尤其是在移植后用环磷酰胺预防移植物抗宿主病。
最近比较 HSCT 和慢性输血治疗 Hb SS 患者的研究越来越倾向于 HSCT 组。HSCT 领域的进展导致供者库扩大和更耐受的方案。现在更容易为几乎每个患者制定个性化的移植计划。成功的管理计划应充分全面地解决患者和家属的社会和心理问题,以确保依从性并改善结果。