Owattanapanich Weerapat, Karoopongse Ekapun, Kittivorapart Janejira, Meeudompong Utairat, Sathapanapitagkit Natchanon, Kungwankiattichai Smith, Vittayawacharin Pongthep, Jianthanakanon Jane, Donsakul Nawapotch, Bundhit Ratana, Kongsomchit Chiraporn, Poonmee Nootjaree, Luangtrakool Panpimon, Warindpong Thanatphak, Chamsai Sutthisak, Bintaprasit Wichitchai, Atakulreka Suparat, Kunacheewa Chutima
Division of Hematology, Department of Medicine, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Department of Transfusion Medicine, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
J Blood Med. 2025 Mar 30;16:151-161. doi: 10.2147/JBM.S511039. eCollection 2025.
Haploidentical (haplo-) hematopoietic stem cell transplantation (HSCT) has been a standard treatment for hematological malignancies for decades. However, it remains unreimbursable in Thailand due to resource constraints. Only one-fifth of the patients suitable for HSCT in our center had matched donors. Since October 2020, haplo-HSCT has been initiated for patients without matched donors using hospital funding, as it is not reimbursed by the national health policy. This cohort study aimed to demonstrate the clinical outcomes, identify problems, manage complications, adjust the protocol of haplo-HSCT in Thailand, and advocate for making haplo-HSCT accessible for treatment in developing countries.
Due to financial constraints, only eight patients with 6 acute myeloid leukemia, 1 acute lymphoblastic leukemia, and 1 lymphoma received haplo-HSCT in the first year. Unmanipulated peripheral blood stem cell haplo-HSCT was performed with post-transplant cyclophosphamide (PTCy)-based graft-versus-host disease (GvHD) prophylaxis.
All patients experienced cytokine release syndrome (CRS) grade 1-2 which improved after PTCy administration. One patient with active disease and HLA-DRB1 mismatch had worsening CRS after PTCy and required tocilizumab treatment. Two patients had grade 3 acute GvHD while a patient developed moderate chronic GvHD. Half of the patients had CMV viremia which was controlled with ganciclovir. At a median follow-up of 7.7 months, 7 patients were alive in remission.
Haplo-HSCT is a feasible treatment option for hematological malignancies, yielding satisfactory outcomes with controllable side effects. Enhanced monitoring and early intervention strategies can further improve patient outcomes. Advocating for haplo-HSCT to be accessible for treatment in developing countries could significantly improve patient survival outcomes.
单倍型相合造血干细胞移植（haplo-HSCT）几十年来一直是血液系统恶性肿瘤的标准治疗方法。然而，由于资源限制，在泰国它仍未被纳入医保报销范围。在我们中心，只有五分之一适合HSCT的患者有匹配的供者。自2020年10月起，由于国家卫生政策不报销，对于没有匹配供者的患者，我们开始使用医院资金开展单倍型HSCT。这项队列研究旨在展示临床结果，识别问题，处理并发症，调整泰国单倍型HSCT的方案，并倡导在发展中国家使单倍型HSCT可用于治疗。
由于资金限制，第一年只有8例患者接受了单倍型HSCT，其中6例为急性髓系白血病，1例为急性淋巴细胞白血病，1例为淋巴瘤。采用基于移植后环磷酰胺（PTCy）预防移植物抗宿主病（GvHD）的非处理外周血干细胞单倍型HSCT。
所有患者均经历了1-2级细胞因子释放综合征（CRS），在给予PTCy后有所改善。1例病情活动且HLA-DRB1不匹配的患者在使用PTCy后CRS恶化，需要使用托珠单抗治疗。2例患者发生3级急性GvHD，1例患者发生中度慢性GvHD。一半的患者发生巨细胞病毒血症，通过更昔洛韦得到控制。中位随访7.7个月时，7例患者存活且病情缓解。
单倍型HSCT是血液系统恶性肿瘤的一种可行治疗选择，能产生令人满意的结果且副作用可控。加强监测和早期干预策略可进一步改善患者预后。倡导在发展中国家使单倍型HSCT可用于治疗可显著改善患者生存结果。
