优化单倍体造血干细胞移植:提高资源有限环境下恶性血液病的预后。

IF 2.1 Q3 HEMATOLOGY
Journal of Blood Medicine Pub Date : 2025-03-30 eCollection Date: 2025-01-01 DOI:10.2147/JBM.S511039
Weerapat Owattanapanich, Ekapun Karoopongse, Janejira Kittivorapart, Utairat Meeudompong, Natchanon Sathapanapitagkit, Smith Kungwankiattichai, Pongthep Vittayawacharin, Jane Jianthanakanon, Nawapotch Donsakul, Ratana Bundhit, Chiraporn Kongsomchit, Nootjaree Poonmee, Panpimon Luangtrakool, Thanatphak Warindpong, Sutthisak Chamsai, Wichitchai Bintaprasit, Suparat Atakulreka, Chutima Kunacheewa
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引用次数: 0

摘要

目的:几十年来,单倍体(haploo -)造血干细胞移植(HSCT)一直是血液系统恶性肿瘤的标准治疗方法。但是,由于资源限制,这笔费用在泰国仍然无法偿还。在我们中心,只有五分之一适合移植的患者有匹配的供体。自2020年10月以来,由于国家卫生政策不报销,已开始使用医院资金对没有匹配供体的患者进行单倍造血干细胞移植。本队列研究旨在证明泰国单倍移植的临床结果,识别问题,管理并发症,调整单倍移植方案,并倡导在发展中国家使单倍移植可用于治疗。方法:由于资金限制,仅8例患者(6例急性髓性白血病,1例急性淋巴细胞白血病,1例淋巴瘤)在第一年接受了单倍造血干细胞移植。未经处理的外周血干细胞单倍造血干细胞移植采用移植后环磷酰胺(PTCy)为基础的移植物抗宿主病(GvHD)预防。结果:所有患者均经历1-2级细胞因子释放综合征(CRS),经PTCy治疗后症状有所改善。1例活动性疾病和HLA-DRB1不匹配的患者PTCy后CRS恶化,需要托珠单抗治疗。2例患者为3级急性GvHD, 1例患者为中度慢性GvHD。一半的患者有巨细胞病毒血症,用更昔洛韦控制。在中位随访时间为7.7个月时,有7名患者在缓解期存活。结论:单倍造血干细胞移植是一种可行的血液恶性肿瘤治疗方案,治疗效果满意,副作用可控。加强监测和早期干预策略可以进一步改善患者的预后。在发展中国家倡导单倍造血干细胞移植可以显著改善患者的生存结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Optimizing Haploidentical Hematopoietic Stem Cell Transplantation: Enhancing Outcomes in Hematologic Malignancies in Resource-Limited Settings.

Objective: 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.

Methods: 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.

Results: 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.

Conclusion: 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.

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来源期刊
CiteScore
3.50
自引率
0.00%
发文量
94
审稿时长
16 weeks
期刊介绍: The Journal of Blood Medicine is an international, peer-reviewed, open access, online journal publishing laboratory, experimental and clinical aspects of all topics pertaining to blood based medicine including but not limited to: Transfusion Medicine (blood components, stem cell transplantation, apheresis, gene based therapeutics), Blood collection, Donor issues, Transmittable diseases, and Blood banking logistics, Immunohematology, Artificial and alternative blood based therapeutics, Hematology including disorders/pathology related to leukocytes/immunology, red cells, platelets and hemostasis, Biotechnology/nanotechnology of blood related medicine, Legal aspects of blood medicine, Historical perspectives. Original research, short reports, reviews, case reports and commentaries are invited.
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