Treatment of pediatric primary hemophagocytic lymphohistiocytosis with the HLH-94/2004 regimens and hematopoietic stem c
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ORIGINAL ARTICLE
Treatment of pediatric primary hemophagocytic lymphohistiocytosis with the HLH-94/2004 regimens and hematopoietic stem cell transplantation in China Honghao Ma 1 & Rui Zhang 1 & Liping Zhang 1 & Ang Wei 1 & Xiaoxi Zhao 1 & Ying Yang 1 & Wei Liu 2 & Zhigang Li 3 & Maoquan Qin 1 & Tianyou Wang 1 Received: 7 June 2020 / Accepted: 27 July 2020 # Springer-Verlag GmbH Germany, part of Springer Nature 2020
Abstract We aimed to clarify the clinical characteristics, prognostic factors, and effectiveness of the HLH-94/2004 regimens and hematopoietic stem cell transplantation (HSCT) in pediatric patients with primary hemophagocytic lymphohistiocytosis (pHLH) in China. A retrospective analysis was performed on 38 patients with pHLH at Beijing Children’s Hospital. PRF1 (34.2%) and UNC13D (31.6%) were the most common mutations in the pHLH. Thirty-eight patients were treated with the HLH-94/2004 regimens after diagnosis. Twenty-six patients (72.2%) responded to first-line treatment (complete response: 55.5%, partial response: 16.7%). The median survival time was 23 months. The overall survival (OS) rate at 3 years was 74.7%. There was no significant difference in the response rate (72% vs. 63.6%, P = 0.703) or 3-year OS (83.6% vs. 66.7%, P = 0.443) between the patients treated with the HLH-94 regimen and those treated with the HLH-2004 regimen. The incidences of all side effects in patients treated with the HLH-94 or HLH-2004 regimen were 32.0% and 18.2%, respectively (P = 0.394). Among 15 patients treated with HSCT, neither the preconditioning regimen nor the donor type affected patient prognosis (P = 0.205 and P = 0.161, respectively). The disease status (remission or nonremission) before preconditioning did not affect prognosis or the incidence of GVHD. Furthermore, a higher bilirubin level (≥ 30 μmol/L) was correlated with a poorer prognosis in pHLH patients (P = 0.026). The effectiveness rates of the HLH-94 and HLH-2004 regimens, chemotherapy, and HSCT were similar in pHLH patients. A bilirubin level ≥ 30 μmol/L might be an adverse prognostic factor in pHLH. Keywords Primary hemophagocytic lymphohistiocytosis . HLH-94 . HLH-2004 . HSCT . Pediatric . China
Honghao Ma, Rui Zhang and Liping Zhang contributed equally to this work. * Zhigang Li [email protected] * Maoquan Qin [email protected] * Tianyou Wang [email protected] 1
Department of Hematology and Oncology, National Center for Children’s Health, Beijing Key Laboratory of Pediatric Hematology Oncology, Key Laboratory of Major Diseases in Children, Ministry of Education, National Key Discipline of Pediatrics, Beijing Children’s Hospital Affiliated to Capital Medical University, Nanlishi Road No. 56, Xicheng District, Beijing 100045, People’s Republic of China
2
Department of Hematology, Children’s Hospital of Zhengzhou City, Zhengzhou 450053, China
3
Hematology and Oncology Laboratory, National Center for Children’s Health, Beijing Key Laboratory of Pediatric Hematology Oncology, Key Laboratory of Major Diseases in Children, Ministry of Edu
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