研究动态
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单倍体干细胞移植:一半匹配但更多希望!-来自印度西部的单中心经验。

Haploidentical Stem Cell Transplantation: Half Match but More Hope!-Single Centre Experience from Western India.

发表日期:2024 Jul
作者: Akanksha Garg, Maharshi Trivedi, Aishwarya Raj, Kamlesh Shah, Kinnari Patel, Ambika Vachhani, Hardik Solanki, Rajan Yadav, Sandip Shah
来源: Stem Cell Research & Therapy

摘要:

随着基于移植后环磷酰胺的平台的成功和临床护理的改善,半相合干细胞移植(HaploSCT)的数量在过去十年中激增。然而,来自印度的数据却很少。我们的目的是评估我们中心的 haploSCT 的结果。自政府计划启动以来,我们中心的许多患者都能够以补贴费用接受移植。我们对 2015 年 1 月至 2022 年 11 月期间进行的单倍体移植进行了回顾性分析。50 名患者符合这项研究的条件。患者详细信息是从病例档案中获得的。移植物抗宿主病(GVHD)预防是移植后环磷酰胺(PTCy)联合吗替麦考酚酯和环孢菌素/他克莫司/西罗莫司。所有患者均输注了捐赠者的外周血干细胞。移植后,患者继续按计划进行定期随访。根据单位方案给予支持性护理。使用 Kaplan-Meier 方法计算总生存期 (OS)。 50 名患者接受了 haploSCT。共有 50 名中位年龄为 20 岁(范围 3-53 岁)的患者接受了来自家庭捐赠者的半相合 HSCT。 23 名 (46%) 患者年龄 > 18 岁,其中 82% 为男性。移植的适应症包括良性和恶性血液疾病。最常用的预处理方案是氟达拉滨  白消安 环磷酰胺(n = 38,76%)。 35 名患者 (70%) 成功植入。在成功移植的患者中,中性粒细胞移植的中位时间为 16 天(范围 10-20 天),血小板移植的中位时间为 18 天(范围 10-32 天)。 14 名患者出现急性 GVHD(28%),3 名患者出现慢性 GVHD(6%)。中位随访时间为 30 个月,两年 OS 率为 43%,中位 OS 为 17 个月。 50 名患者 (42%) 中有 21 名(成人= 9,儿童= 12)还活着并且正在接受定期随访。对于没有合适的匹配供体且无法提供匹配的无关移植的患者,采用 PTCy 平台的 HaploSCT 是一种经济有效、有前途的治疗方式。在我们的中心,我们能够以可承受的成本使用非专利药物取得可接受的结果。移植相关死亡率 (TRM) 率与其他中心相当,但是,多重耐药细菌感染仍然是发展中国家进行半相合 HSCT 的一个挑战。© 作者,获得印度血液学和输血学会的独家许可2023. Springer Nature 或其许可方(例如协会或其他合作伙伴)根据与作者或其他权利持有人的出版协议拥有本文的专有权;作者对本文已接受的手稿版本的自行存档仅受此类出版协议和适用法律的条款的约束。
With the success of post-transplant cyclophosphamide based platform and improved clinical care, the number of haploidentical stem cell transplants (HaploSCT) have surged over the last decade. However, data from India is scarce. We aimed to evaluate the outcome of haploSCT at our centre. Since the inception of government schemes, many patients at our centre are able to undergo transplantation at subsidized cost. We conducted a retrospective analysis of the haploidentical transplants performed between January 2015 and November 2022. Fifty patients were eligible for this study. Patient details were obtained from case files. The graft versus host disease (GVHD) prophylaxis was post-transplant Cyclophosphamide (PTCy) with Mycophenolate-mofetil and Cyclosporine/tacrolimus/sirolimus. All patients were transfused peripheral blood stem cells from donors. Post-transplant, patients continued regular follow up as per schedule. Supportive care was given as per unit protocol. Overall survival (OS) was calculated using the Kaplan-Meier method. Fifty patients underwent haploSCT. A total of fifty patients with a median age of 20 years (range 3-53 years) underwent haploidentical HSCT from a family donor. Twenty three (46%) patients were > 18 years age and 82% were males. Indications for transplant included both benign and malignant hematological diseases. Most common conditioning regimen used was Fludarabine + Busulphan + Cyclophosphamide (n = 38, 76%). Thirty five patients (70%) engrafted successfully. In the patients who had successful engraftment, the median time to neutrophil engraftment was 16 days (range 10-20 days) and platelet engraftment was 18 days (range 10-32). Fourteen patients developed acute GVHD (28%), and three patients developed chronic GVHD (6%). The median follow-up was 30 months and the two-year OS was 43% with a median OS of 17 months. Twenty-one (adult = 9, pediatric = 12) out of 50 patients (42%) are alive and on regular follow-up. HaploSCT with a PTCy platform is a cost-effective, promising modality of treatment in patients who have no suitable matched donors and are not affording matched unrelated transplants. At our centre, we were able to achieve acceptable results with use of generic medications at affordable cost. Transplant Related Mortality (TRM) rates were comparable to other centres, however, multi-drug resistant bacterial infection remains a challenge in performing haploidentical HSCT in developing countries.© The Author(s), under exclusive licence to Indian Society of Hematology and Blood Transfusion 2023. Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.