Allogeneic Hematopoietic Stem Cell Transplantation in Severe Aplastic Anemia: A Single Centre Experience in Malaysia.

Gilbert Wilfred, Tee Chuan Ong, Syed Abd Kadir Sh Shahnaz, Ho Kim Wah, Edmund Syed Carlo, Sathar Jameela, Sen Mui Tan
{"title":"Allogeneic Hematopoietic Stem Cell Transplantation in Severe Aplastic Anemia: A Single Centre Experience in Malaysia.","authors":"Gilbert Wilfred,&nbsp;Tee Chuan Ong,&nbsp;Syed Abd Kadir Sh Shahnaz,&nbsp;Ho Kim Wah,&nbsp;Edmund Syed Carlo,&nbsp;Sathar Jameela,&nbsp;Sen Mui Tan","doi":"10.31547/bct-2021-018","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Hematopoietic stem cell transplantation (HSCT) provides curative therapy in almost 90% of patients with severe aplastic anemia (SAA). Older age, long duration of disease with consequent heavy exposure to transfusion, and active infection at the time of HSCT have a negative influence on the outcomes, causing graft failure (GF) and graft versus host disease (GVHD).</p><p><strong>Purpose: </strong>To describe the outcomes of all patients with SAA who received hematopoietic stem cell transplantation at a tertiary center in Malaysia.</p><p><strong>Materials and methods: </strong>We included a 20 y cohort of patients who underwent transplantation from January 1, 1999 to December 31, 2019. Data were obtained from electronic medical records. Demographics, clinical characteristics, and treatment outcomes were analyzed using descriptive statistics. Overall survival (OS) was analyzed using Kaplan-Meier curves. All analyses were conducted using the Statistical Package for the Social Sciences (SPSS) version 25.</p><p><strong>Results: </strong>Eighty patients were analyzed. The median age at diagnosis was 19 years, and 59% patients were male (n = 47). Malay ethnicity was the highest (52.5%), followed by Chinese (20.0%) and Native Sabah (15.0%). The median duration from diagnosis to transplantation was 13.5 weeks. A majority of patients received Cy-ATG conditioning (n = 51, 63.8%). Forty-one patients (51.2%) used peripheral blood as stem cell source, 36 patients (45.0%) used granulocyte colony stimulating factor (G-CSF) primed marrow graft and 3 patients (3.8%) used both. The mean nucleated mononuclear cell and CD34 cell doses were 4.7 ± 1.7 × 10<sup>8</sup>/kg and 4.6 ± 1.9 × 10<sup>6</sup>/kg, respectively. Median engraftment for WBCs and platelets was 14 and 15 days, respectively. There was no difference in WBC and platelet engraftment in patients who received peripheral blood stem cell transplantation or bone marrow transplant. At a median follow-up of 54 months, 49 patients (61.3%) achieved complete remission and 8 patients (10.0%) achieved partial remission. The estimated 5 y OS was 63% and higher among those who received HSCT within 3 months of diagnosis. Twenty-two patients (27.5%) died within 100 d of transplantation, and a majority of these died due to pre-engraftment death.</p><p><strong>Discussion and conclusions: </strong>Our study found that patients who received early allogeneic transplantation for SAA had better outcomes. Pre-engraftment failure was the major cause of transplant-related mortality within 100 d. Further studies are required to identify the factors responsible for delaying transplantation to improve treatment outcomes.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 2","pages":"45-53"},"PeriodicalIF":0.0000,"publicationDate":"2022-05-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a4/c6/2432-7026-5-2-0045.PMC9870683.pdf","citationCount":"1","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood cell therapy","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.31547/bct-2021-018","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 1

Abstract

Background: Hematopoietic stem cell transplantation (HSCT) provides curative therapy in almost 90% of patients with severe aplastic anemia (SAA). Older age, long duration of disease with consequent heavy exposure to transfusion, and active infection at the time of HSCT have a negative influence on the outcomes, causing graft failure (GF) and graft versus host disease (GVHD).

Purpose: To describe the outcomes of all patients with SAA who received hematopoietic stem cell transplantation at a tertiary center in Malaysia.

Materials and methods: We included a 20 y cohort of patients who underwent transplantation from January 1, 1999 to December 31, 2019. Data were obtained from electronic medical records. Demographics, clinical characteristics, and treatment outcomes were analyzed using descriptive statistics. Overall survival (OS) was analyzed using Kaplan-Meier curves. All analyses were conducted using the Statistical Package for the Social Sciences (SPSS) version 25.

Results: Eighty patients were analyzed. The median age at diagnosis was 19 years, and 59% patients were male (n = 47). Malay ethnicity was the highest (52.5%), followed by Chinese (20.0%) and Native Sabah (15.0%). The median duration from diagnosis to transplantation was 13.5 weeks. A majority of patients received Cy-ATG conditioning (n = 51, 63.8%). Forty-one patients (51.2%) used peripheral blood as stem cell source, 36 patients (45.0%) used granulocyte colony stimulating factor (G-CSF) primed marrow graft and 3 patients (3.8%) used both. The mean nucleated mononuclear cell and CD34 cell doses were 4.7 ± 1.7 × 108/kg and 4.6 ± 1.9 × 106/kg, respectively. Median engraftment for WBCs and platelets was 14 and 15 days, respectively. There was no difference in WBC and platelet engraftment in patients who received peripheral blood stem cell transplantation or bone marrow transplant. At a median follow-up of 54 months, 49 patients (61.3%) achieved complete remission and 8 patients (10.0%) achieved partial remission. The estimated 5 y OS was 63% and higher among those who received HSCT within 3 months of diagnosis. Twenty-two patients (27.5%) died within 100 d of transplantation, and a majority of these died due to pre-engraftment death.

Discussion and conclusions: Our study found that patients who received early allogeneic transplantation for SAA had better outcomes. Pre-engraftment failure was the major cause of transplant-related mortality within 100 d. Further studies are required to identify the factors responsible for delaying transplantation to improve treatment outcomes.

Abstract Image

Abstract Image

Abstract Image

异基因造血干细胞移植治疗严重再生障碍性贫血:马来西亚单一中心的经验。
背景:造血干细胞移植(HSCT)为近90%的严重再生障碍性贫血(SAA)患者提供了根治性治疗。年龄较大,疾病持续时间长,因此大量暴露于输血,以及移植时的活动性感染对结果有负面影响,导致移植物衰竭(GF)和移植物抗宿主病(GVHD)。目的:描述在马来西亚三级中心接受造血干细胞移植的所有SAA患者的结果。材料和方法:我们纳入了1999年1月1日至2019年12月31日期间接受移植的20例患者队列。数据来自电子病历。采用描述性统计分析人口统计学、临床特征和治疗结果。采用Kaplan-Meier曲线分析总生存期(OS)。所有分析均使用社会科学统计软件包(SPSS)第25版进行。结果:对80例患者进行分析。诊断时的中位年龄为19岁,59%的患者为男性(n = 47)。马来族最高(52.5%),其次是华人(20.0%)和沙巴本地人(15.0%)。从诊断到移植的中位持续时间为13.5周。大多数患者接受Cy-ATG调理(n = 51, 63.8%)。41例(51.2%)患者使用外周血作为干细胞来源,36例(45.0%)患者使用粒细胞集落刺激因子(G-CSF)诱导的骨髓移植,3例(3.8%)患者两者均使用。有核细胞和CD34细胞的平均剂量分别为4.7±1.7 × 108/kg和4.6±1.9 × 106/kg。白细胞和血小板的中位植入时间分别为14天和15天。外周血干细胞移植和骨髓移植患者的白细胞和血小板植入量无差异。在54个月的中位随访中,49例患者(61.3%)获得完全缓解,8例患者(10.0%)获得部分缓解。在诊断后3个月内接受造血干细胞移植的患者中,估计5年生存率为63%,甚至更高。22例(27.5%)患者在移植后100 d内死亡,其中大多数死于移植前死亡。讨论和结论:我们的研究发现,早期接受同种异体移植治疗SAA的患者预后较好。移植前失败是100天内移植相关死亡的主要原因。需要进一步的研究来确定延迟移植的因素,以改善治疗结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
自引率
0.00%
发文量
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术官方微信