羟基脲和甲氨蝶呤治疗复发性朗格汉斯细胞组织细胞增生症（LCH-HU- 初步研究）的安全性和有效性初步研究。

Pilot study to estimate the safety and effectiveness of hydroxyurea and methotrexate recurrent langerhans cell histiocytosis (LCH-HU-pilot).

机构信息

Children's Cancer Center, National Center for Child Health and Development, Tokyo, Japan.

Department of Pediatrics, Shiga University of Medical Science, Otsu, Japan.

出版信息

Medicine (Baltimore). 2022 Dec 16;101(50):e31475. doi: 10.1097/MD.0000000000031475.

DOI:10.1097/MD.0000000000031475

PMID:36550910

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9771280/

Abstract

This study was a non-blinded, multicenter, single-arm study. Recurrent (relapsed) LCH is defined as the appearance of new lesions or the enlargement of preexisting lesions due to LCH. In this study, all patients received hydroxyurea, and if the treatment response was unsatisfactory, methotrexate was added. The duration of treatment was 48 weeks. The primary endpoint was the rate of non-active disease achievement, which was 24 weeks after initiating hydroxyurea administration. No active disease is defined as the resolution of all the signs and symptoms related to LCH.

摘要

本研究为一项非盲、多中心、单臂研究。复发性（复发）朗格汉斯细胞组织细胞增生症（LCH）定义为新病灶的出现或由于 LCH 导致原有病灶增大。在本研究中，所有患者均接受羟基脲治疗，如果治疗反应不满意，则加用甲氨蝶呤。治疗持续时间为 48 周。主要终点是起始羟基脲治疗后 24 周时非活动疾病的发生率。无活动疾病定义为与 LCH 相关的所有体征和症状均已缓解。