Safety and efficacy of peripheral blood stem cells collection in healthy children and pediatric patients with thalassemia major weighing 20 kg or less

IF 1.4 4区医学 Q4 HEMATOLOGY

Journal of Clinical Apheresis Pub Date : 2024-01-08 DOI:10.1002/jca.22102

Libai Chen, Jianyun Wen, Xiaoxiao Xu, Jing Du, Yongsheng Ruan, Xiaoqin Feng, Juan Li, Yuelin He, Xuedong Wu

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引用次数: 0

Abstract

Background

Peripheral blood stem cell (PBSC) collection in children poses challenges due to their small size, low body weight (BW), and unique pediatric physiology, especially among children weighing 20 kg (kg) or less.

Methods

PBSC collection data of both healthy children and patients with thalassemia major (TM) weighing 20 kg or less between January 2013 and December 2020 were reviewed. Moreover, PBSCs characteristics along with various aspects of efficiency and safety between healthy donors and patients with TM were compared.

Results

A total of 262 PBSC procedures were performed on 255 children. Of these, 91 procedures were carried out on 85 allogeneic healthy donors, and 171 auto-backup collections were performed on 170 patients with TM to ensure PBSC availability and prevent transplantation failure. A minimum pre-apheresis hemoglobin (HGB) level of 60 g/L was discovered to be safe and feasible in patients with TM. The median CD34+ cell dose in the PBSC product during the initial apheresis procedure was higher in healthy donors compared to patients with TM (7.29 ± 5.28 × 10⁶ cells/kg vs5.88 ± 4.23 × 10⁶ cells/kg, P = .043). The total CD34+ cells/kg recipient weight exhibited a positive correlation with pre-apheresis monocyte counts, but a negative correlation with donor weight. Apheresis significantly reduced hematocrit and platelet counts in the allogeneic group compared to the autologous group. Patients with TM experienced a higher occurrence of bone pain related to granulocyte colony-stimulating factor treatment. Notably, no serious complications related to PBSCs mobilization, central venous catheter placement, or the apheresis procedure were observed in either group.

Conclusions

PBSCs collection was both safe and effective in healthy children and pediatric patients with TM weighing 20 kg or less.

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健康儿童和体重在 20 公斤或以下的重型地中海贫血症儿童患者采集外周血干细胞的安全性和有效性。

背景：儿童外周血干细胞（PBSC）采集工作因其体型小、体重低和独特的儿科生理结构而面临挑战，尤其是体重在20公斤或以下的儿童：方法：回顾了 2013 年 1 月至 2020 年 12 月期间体重在 20 公斤或以下的健康儿童和重型地中海贫血（TM）患者的 PBSC 采集数据。此外，还比较了健康捐献者和重型地中海贫血患者的 PBSCs 特征、效率和安全性等各个方面：共为 255 名儿童实施了 262 例 PBSC 手术。结果：共为 255 名儿童实施了 262 例 PBSC 手术，其中 91 例为 85 名异体健康供体，171 例为 170 名 TM 患者进行了自动备份采集，以确保 PBSC 的可用性并防止移植失败。研究发现，对 TM 患者来说，采血前血红蛋白 (HGB) 最低水平为 60 克/升是安全可行的。与 TM 患者相比，健康供体在初次血液净化过程中 PBSC 产物的 CD34+ 细胞剂量中位数更高（7.29 ± 5.28 × 106 cells/kg vs 5.88 ± 4.23 × 106 cells/kg，P = .043）。总 CD34+ 细胞/公斤受体体重与血液透析前单核细胞计数呈正相关，但与供体体重呈负相关。与自体移植组相比，异体移植组的血细胞比容和血小板计数明显降低。TM患者在接受粒细胞集落刺激因子治疗时，骨痛发生率较高。值得注意的是，两组患者均未出现与 PBSCs 动员、中心静脉导管置入或无细胞疗法相关的严重并发症：结论：对于健康儿童和体重不超过 20 千克的 TM 儿童患者来说，PBSCs 采集既安全又有效。

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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来源期刊

Journal of Clinical Apheresis 医学-血液学

CiteScore

2.80

自引率

13.30%

发文量

审稿时长

>12 weeks

期刊介绍： The Journal of Clinical Apheresis publishes articles dealing with all aspects of hemapheresis. Articles welcomed for review include those reporting basic research and clinical applications of therapeutic plasma exchange, therapeutic cytapheresis, therapeutic absorption, blood component collection and transfusion, donor recruitment and safety, administration of hemapheresis centers, and innovative applications of hemapheresis technology. Experimental studies, clinical trials, case reports, and concise reviews will be welcomed.