儿童造血干细胞移植86例临床分析

doi:10.12372/jcp.2023.22e0047

摘要/Abstract

摘要：

目的分析造血干细胞移植术（HSCT）治疗儿童血液肿瘤性疾病的临床疗效。方法回顾性分析2016年3月至2020年4月在徐州医科大学附属医院儿科行HSCT 86例患儿的临床资料。结果 86例HSCT患儿，男50例、女36例，中位年龄11（9~14）岁。急性淋巴细胞白血病（ALL）27例，急性髓系白血病（AML）28例，再生障碍性贫血（AA）16例，淋巴瘤5例，慢性粒细胞白血病（CML）4例，骨髓增生异常综合征（MDS）3例，神经母细胞瘤（NB）、系统新硬化症（SSc）及肾上腺脑白质营养不良（ALD）各1例。自体移植7例、脐血移植4例、异基因造血干细胞移植（allo-HSCT）75例。86例患儿中位随访时间29.5（17.6~44.0）月。植入成功84例，自体移植和allo-HSCT各失败1例；复发14例，自体移植复发4例，allo-HSCT复发10例；Ⅲ/Ⅳ移植物抗宿主病（GVHD）6例，均为allo-HSCT；死亡20例，其中自体移植4例，allo-HSCT16例。Fisher精确检验结果显示自体移植、脐血移植、同胞全相合、半相合4组间发生Ⅰ/Ⅱ级aGVHD差异有统计学意义（P<0.001）。结论 HSCT为治疗儿童多种恶性疾病的有效方法，但移植后复发、重度GVHD是影响预后重要因素，预防复发及控制排异在儿童HSCT中至关重要。

关键词: 造血干细胞移植, 临床分析, 预后因素, 儿童

Abstract:

Objective To analyze the clinical efficacy of hematopoietic stem cell transplantation (HSCT) in the treatment of hematological neoplastic diseases in children. Methods The clinical data of 86 children with hematological neoplastic diseases who underwent HSCT in the Affiliated Hospital of Xuzhou Medical University from March 2016 to August 2020 were retrospectively analyzed. Results Eighty-six children (50 boys and 36 girls) with HSCT were included, and the median age was 11 (9-14) years. There were 27 cases of acute lymphoblastic leukemia (ALL), 28 cases of acute myeloid leukemia (AML), 16 cases of aplastic anemia (AA), 5 cases of lymphoma, 4 cases of chronic myeloid leukemia (CML), 3 cases of myelodysplastic syndrome (MDS), 1 case of neuroblastoma (NB), 1 case of systemic neosclerosis (SSC) and 1 case of adrenoleukodystrophy (ALD). There were 7 cases of autologous transplantation, 4 cases of umbilical cord blood transplantation and 75 cases of allogeneic hematopoietic stem cell transplantation (allo-HSCT). The median follow-up time of 86 patients was 29.5 (17.6-44.0) months. A total of 84 patients were successfully implanted, and 1 patient failed autologous transplantation and 1 failed allo-HSCT. Fourteen patients relapsed, including 4 patients with autologous transplantation and 10 patients with allo-HSCT. Six patients developed grade Ⅲ/Ⅳ GVHD, all of whom were allo-HSCT. Twenty patients died, including 4 patients with autologous transplantation and 16 patients with allo-HSCT. Fisher's exact test showed that there was significant difference in the occurrence of grade Ⅰ/ⅡGVHD among the four groups of autologous transplantation, cord blood transplantation, SIB homozygous and half homozygous (P<0.001). Conclusions HSCT is an effective method to treat a variety of malignant diseases in children, but recurrence and severe GVHD after transplantation are important factors affecting the prognosis. Preventing recurrence and controlling rejection are very important in children's HSCT.

Key words: hematopoietic stem cell transplantation, clinical indicator, prognostic factor, child

王文鹏, 高吉照, 郭雷, 李艳, 卢立慧, 常颖. 儿童造血干细胞移植86例临床分析[J]. 临床儿科杂志, 2023, 41(3): 187-191.

WANG Wenpeng, GAO Jizhao, GUO Lei, LI Yan, LU Lihui, CHANG Ying. Clinical analysis of 86 csses of hematopoietic stem cell transplantation in children[J]. Journal of Clinical Pediatrics, 2023, 41(3): 187-191.

图/表 2

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项目	例数	植入成功	复发	aGVHD (Ⅲ/Ⅳ)	存活
自体移植	7	6(85.7)	4(57.1)	0(0.0)	3(42.9)
ALL	2	2(100.0)	2(100.0)	0(0.0)	1(50.0)
AML	1	1(100.0)	1(100.0)	0(0.0)	0(0.0)
淋巴瘤	3	2(66.7)	0(0.0)	0(0.0)	2(66.7)
NB	1	1(100.0)	1(100.0)	0(0.0)	0(0.0)
脐血移植	4	4(100.0)	0(0.0)	0(0.0)	4(100.0)
AML	2	2(100.0)	0(0.0)	0(0.0)	2(100.0)
AA	1	1(100.0)	0(0.0)	0(0.0)	1(100.0)
SSc	1	1(100.0)	0(0.0)	0(0.0)	1(100.0)
Allo-HSCT	75	74(98.7)	10(13.3)	6(8.0)	59(78.7)
ALL	25	24(96.0)	5(20.0)	1(4.0)	19(76.0)
AML	25	25(100.0)	3(12.0)	2(8.0)	21(84.0)
AA	15	15(100.0)	0(0.0)	2(13.3)	12(80.0)
MDS	3	3(100.0)	1(33.3)	0(0.0)	2(66.7)
CML	4	4(100.0)	1(25.0)	0(0.0)	3(75.0)
淋巴瘤	2	2(100.0)	0(0.0)	0(0.0)	2(100.0)
ALD	1	1(100.0)	0(0.0)	1(100.0)	0(0.0)
合计	86	84(97.7)	14(16.3)	6(7.0)	66(7.7)

组别	例数	存活	aGVHD
组别	例数	存活	Ⅰ/Ⅱ级	Ⅲ/Ⅳ级
自体移植	7	3(42.9)	0(0.0)	0(0.0)
脐血移植	4	4(100.0)	2(50.0)	0(0.0)
同胞全相合	14	12(85.7)	5(35.7)	0(0.0)
半相合	61	47(77.0)	55(90.1)	6(9.8)
P¹⁾		0.126	<0.001	0.823