基于 G-CSF/ATG 的小儿原发性嗜血细胞淋巴组织细胞增多症单倍体移植改良方案:单中心长期随访经验。

IF 2.4 3区 医学 Q2 HEMATOLOGY
Juan Xiao, Xingcheng Yang, Nanhai Wu, Shifen Fan, Zhouyang Liu, Fan Jiang, Jiao Chen, Jia Wei, Yuan Sun
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The conditioning regimen included 300-600 mg/m<sup>2</sup> etoposide (VP16), along with low doses of busulfan (Bu) (0.8-1.2 mg/kg every 6 hours on Days -8 to -6), cyclophosphamide (Cy) (10 mg/kg/day on Days -4 to -3), fludarabine (Flu) (30 mg/m<sup>2</sup>/day on Days -5 to -3), and ATG (8-9 mg/kg total dose on Days -5 to -2) to reduce complications.</p><p><strong>Results: </strong>All 42 patients achieved successful engraftment. Following a median follow-up period of 48.7 months, 32 of the 42 patients remained alive and disease free. The 2-year overall survival (OS) rate was 78.4%, and the 5-year OS rate was 73.7%. The 2-year failure-free survival (FFS) rate was 71.3%, and the 5-year FFS rate was 66.5%. Patients who achieved complete remission at the time of HSCT showed better OS (p < 0.05). The incidence of Grade III-IV acute graft-versus-host disease (GVHD) was 26.2%, and severe chronic GVHD was observed in 11.9% of patients. 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引用次数: 0

摘要

本文章由计算机程序翻译,如有差异,请以英文原文为准。
Modified G-CSF/ATG-Based Haploidentical Transplantation Protocol in Pediatric Primary Hemophagocytic Lymphohistiocytosis: A Long-Term Follow-Up Single-Center Experience.

Background: Primary hemophagocytic lymphohistiocytosis (HLH) is a hyperinflammatory syndrome caused by immune dysregulation. Hematopoietic stem cell transplantation (HSCT) represents the only option for long-term cure for primary HLH. However, only around 25% of patients have a fully HLA-matched donor.

Methods: In this retrospective study, we analyzed 42 pediatric patients with primary HLH who underwent haplo-SCT using the modified granulocyte colony-stimulating factor (G-CSF)/antithymocyte globulin (ATG)-based protocol. The conditioning regimen included 300-600 mg/m2 etoposide (VP16), along with low doses of busulfan (Bu) (0.8-1.2 mg/kg every 6 hours on Days -8 to -6), cyclophosphamide (Cy) (10 mg/kg/day on Days -4 to -3), fludarabine (Flu) (30 mg/m2/day on Days -5 to -3), and ATG (8-9 mg/kg total dose on Days -5 to -2) to reduce complications.

Results: All 42 patients achieved successful engraftment. Following a median follow-up period of 48.7 months, 32 of the 42 patients remained alive and disease free. The 2-year overall survival (OS) rate was 78.4%, and the 5-year OS rate was 73.7%. The 2-year failure-free survival (FFS) rate was 71.3%, and the 5-year FFS rate was 66.5%. Patients who achieved complete remission at the time of HSCT showed better OS (p < 0.05). The incidence of Grade III-IV acute graft-versus-host disease (GVHD) was 26.2%, and severe chronic GVHD was observed in 11.9% of patients. Thrombotic microangiopathy occurred in 13 patients, and veno-occlusive disease in two patients.

Conclusions: This modified G-CSF/ATG-based haploidentical protocol demonstrates significant potential for pediatric patients with primary HLH, exhibiting commendable effectiveness and safety.

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来源期刊
Pediatric Blood & Cancer
Pediatric Blood & Cancer 医学-小儿科
CiteScore
4.90
自引率
9.40%
发文量
546
审稿时长
1.5 months
期刊介绍: Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.
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