Outcome of Haematopoietic Stem Cell Transplant in Beta-Thalassaemia Major: Single Centre Experience from a Low- and Middle-Income Country.

IF 0.7 4区医学 Q3 MEDICINE, GENERAL & INTERNAL

Jcpsp-Journal of the College of Physicians and Surgeons Pakistan Pub Date : 2025-04-01 DOI:10.29271/jcpsp.2025.04.513

Hashim Khan, Tariq Ghafoor, Nighat Shahbaz, Tariq Azam Khattak, Uzma Rahim, Munazza Nabi Awan

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引用次数: 0

Abstract

Objective: To determine the outcome of haematopoietic stem transplant (HSCT) in beta-thalassaemia major (BTM) patients.

Study design: Descriptive Study. Place and Duration of the Study: Department of Clinical Haematology, Armed Forces Bone Marrow Transplant Centre (AFBMTC), Rawalpindi, Pakistan, from April 2018 to December 2023.

Methodology: All cases of BTM undergoing HLA-matched allogeneic HSCT after myeloablative conditioning were included. Cases undergoing second HSCT and HSCT with treosulfan-based conditioning were excluded. Age, gender, complications, mortality, and associated factors were evaluated.

Results: This study analysed the data of 118 cases of BTM including 71 (60.2%) males undergoing HSCT after myeloablative conditioning during the study period. The mean age at the time of HSCT was 85.7 ± 33.6 months. Eighty-one (68.6%) cases were in Pesaro Class III. Neutropenic fever was the most common complication documented in 117 (99.2%) cases. Mortality at day-100 was 14 (11.9%). Overall treatment-related mortality (TRM) was 23 (19.4%). In univariate analysis, factors having a statistically significant association with TRM were graft failure (p = 0.001), Pesaro class (p = 0.03), severity of acute graft versus host disease (aGVHD) (p = 0.02), and veno-occlusive disease (VOD) (p = 0.02). The median follow-up time was 26.87 ± 16.60 months with overall survival (OS) and disease free survival (DFS) rates of 80.5% and 78.0%, respectively.

Conclusion: The OS of around 80% is promising which can be further improved with better transfusion services, regular iron chelation, and HSCT at a younger age.

Key words: Haematopoietic stem cell transplant, Beta-thalassaemia major, Pakistan.

查看原文本刊更多论文

造血干细胞移植治疗严重β -地中海贫血的结果：来自低收入和中等收入国家的单中心经验。

目的：探讨重度β -地中海贫血（BTM）患者造血干细胞移植（HSCT）的治疗效果。研究设计：描述性研究。研究地点和时间：2018年4月至2023年12月，巴基斯坦拉瓦尔品第武装部队骨髓移植中心（AFBMTC）临床血液科。方法：纳入所有在清髓后接受hla匹配异体造血干细胞移植的BTM病例。排除了第二次HSCT和以曲硫丹为基础的HSCT。评估年龄、性别、并发症、死亡率和相关因素。结果：本研究分析了118例BTM患者的资料，其中71例（60.2%）为男性，在研究期间进行了清髓调节后的HSCT。移植时的平均年龄为85.7±33.6个月。81例（68.6%）为Pesaro III类。117例（99.2%）病例中，中性粒细胞减少热是最常见的并发症。第100天死亡率为14（11.9%）。总治疗相关死亡率（TRM）为23（19.4%）。在单变量分析中，与TRM有统计学显著相关的因素是移植物衰竭（p = 0.001）、Pesaro分级（p = 0.03）、急性移植物抗宿主病（aGVHD）严重程度（p = 0.02）和静脉闭塞性疾病（VOD）（p = 0.02）。中位随访时间为26.87±16.60个月，总生存期（OS）为80.5%，无病生存期（DFS）为78.0%。结论：约80%的总生存率是有希望的，可以通过更好的输血服务，定期铁螯合和更年轻的HSCT进一步提高。关键词：造血干细胞移植；重度地中海贫血；巴基斯坦

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来源期刊

Jcpsp-Journal of the College of Physicians and Surgeons Pakistan MEDICINE, GENERAL & INTERNAL-

CiteScore

1.40

自引率

0.00%

发文量

453

审稿时长

3-6 weeks

期刊介绍： Journal of College of Physicians and Surgeons Pakistan (JCPSP), is the prestigious, peer reviewed monthly biomedical journal of the country published regularly since 1991. Established with the primary aim of promotion and dissemination of medical research and contributed by scholars of biomedical sciences from Pakistan and abroad, it carries original research papers, , case reports, review articles, articles on medical education, commentaries, short communication, new technology, editorials and letters to the editor. It covers the core biomedical health science subjects, basic medical sciences and emerging community problems, prepared in accordance with the “Uniform requirements for submission to bio-medical journals” laid down by International Committee of Medical Journals Editors (ICMJE). All publications of JCPSP are peer reviewed by subject specialists from Pakistan and locally and abroad.