Allogeneic stem cell transplant for myelofibrosis- A retrospective single-center study.

Dharma Choudhary, Divya Doval, Vipin Khandelwal, Rasika Setia, Anil Handoo
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Abstract

Introduction: Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment option for myelofibrosis (MF). Despite the benefits of long-term relapse-free survival, HSCT can be associated with substantial treatment-related morbidity and mortality.

Methods: This is an observational retrospective study of 15 consecutive patients with MF who underwent allogeneic HSCT at a tertiary care center in Northern India between June 2012 and January 2020. The pre-transplant Dynamic International Prognostic Scoring System (DIPSS) and hematopoietic cell transplantation-specific co-morbidity index (HCT-CI) scores were used. The primary endpoints were overall survival (OS) and disease-free survival (DFS), and the secondary endpoints were post-transplant complications (acute and chronic graft-versus-host-disease [GvHD], graft failure [GF], and cytomegalovirus reactivation [CMV]).

Results: The OS and DFS in our study were 60% with no relapse at a median follow-up of 364 days (range 7-2,815 days). Twenty-seven percent of patients developed acute GvHD and 27% of patients developed chronic (limited) GvHD. The non-relapse mortality (NRM) was 40%, with the main cause of death being sepsis, followed by acute GvHD.

Conclusion: MF remains a challenging condition to treat, with a poor prognosis. Our study showed that reduced toxicity conditioning provided good DFS and OS. Thus, it should be offered to patients with high DIPSS scores. Sepsis was the predominant cause of mortality in this cohort.

Abstract Image

同种异体干细胞移植治疗骨髓纤维化-一项回顾性单中心研究。
同种异体造血干细胞移植(HSCT)是目前治疗骨髓纤维化(MF)的唯一治疗选择。尽管有长期无复发生存的好处,但造血干细胞移植可能与大量治疗相关的发病率和死亡率相关。方法:这是一项观察性回顾性研究,研究对象为2012年6月至2020年1月在印度北部一家三级医疗中心连续接受同种异体造血干细胞移植的15名MF患者。采用移植前动态国际预后评分系统(DIPSS)和造血细胞移植特异性合并症指数(HCT-CI)评分。主要终点是总生存期(OS)和无病生存期(DFS),次要终点是移植后并发症(急性和慢性移植物抗宿主病[GvHD]、移植物衰竭[GF]和巨细胞病毒再激活[CMV])。结果:在我们的研究中,OS和DFS为60%,中位随访364天(7- 2815天)无复发。27%的患者发展为急性GvHD, 27%的患者发展为慢性(有限)GvHD。非复发死亡率(NRM)为40%,主要死亡原因为败血症,其次为急性GvHD。结论:MF仍然是一种具有挑战性的疾病,预后较差。我们的研究表明,减少毒性调节提供了良好的DFS和OS。因此,对于DIPSS评分较高的患者应给予治疗。脓毒症是该队列中主要的死亡原因。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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