Clinical efficacy and safety of high-dose methotrexate in the treatment of adult relapsed and (or) refractory Langerhans cell histiocytosis
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摘要: 目的 分析大剂量甲氨蝶呤(high-dose methotrexate，HD-MTX)治疗成人复发和(或)难治朗格汉斯细胞组织细胞增生症(relapsed and/or refractory Langerhans cell histiocytosis，rrLCH)的疗效和安全性。方法 回顾性分析2019—2022年于北京市隆福医院血液科住院并经HD-MTX治疗的成人rrLCH患者的一般资料、治疗方案、效果及随访情况。HD-MTX合计总量59 g/m2，在第1~2周5 g/m2，第3周7 g/m2，第4~5周休息，第6~7周9 g/m2，第8~9周休息，第10~11周12 g/m2。结果 共10例rrLCH患者采用HD-MTX治疗，男7例，女3例，中位年龄35(21~49)岁，BRAF-V600E阳性2例。2例患者为骨骼单系统多病灶，8例为多系统型。所有患者均使用过一线以上含阿糖胞苷的化疗。首次复发6例，多次复发4例。1例在完成前3次治疗(MTX累计30 g)后达完全缓解，自行中断治疗。其余9例完成全部治疗，MTX总剂量84.6~133.0 g，平均单次剂量为10 g/m2。8例获得完全缓解，1例为部分缓解，1例未缓解。8例患者治疗过程中出现肝功能升高，3例出现皮疹，3例出现发热。结论 HD-MTX对于成人rrLCH患者具有较好疗效，不良反应可控。
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关键词:
- 大剂量甲氨蝶呤 /
- 朗格汉斯细胞组织细胞增生症 /
- 复发/难治
Abstract: Objective To analyze the efficacy and safety of high-dose methotrexate(HD-MTX) in the treatment of adult relapsed and (or) refractory Langerhans cell histiocytosis(rrLCH).Methods The general data, treatment scheme, effect and follow-up of adult rrLCH patients hospitalized in the Hematology Department of Beijing Longfu Hospital and treated with HD-MTX from 2019 to 2022 were analyzed retrospectively. The total amount of HD-MTX was 59 g/m2.5 g/m2 was used in the first and second weeks, 7 g/m2 in the third week, rest in the fourth and fifth weeks, 9 g/m2 in the sixth and seventh weeks, rest in the eighth and ninth weeks, and 12 g/m2 in the tenth and eleventh weeks.Results A total of 10 patients with rrLCH were treated with HD-MTX, including 7 males and 3 females, with a median age of 35 years(21-49 years). Two patients were positive for BRAF-V600E. Two patients were single-system multifocal type and 8 patients were multisystem type. All patients received chemotherapy containing cytarabine above the first line. Six cases relapsed for the first time and 4 cases relapsed repeatedly. One patient achieved complete remission after completing the first three-week treatment(accumulated doses of MTX was 30 g), and voluntarily discontinue the treatment. The other 9 patients completed all the treatment, the total dose of MTX was 84.6-133.0 g, and the average single dose was 10 g/m2. Eight cases achieved complete remission, 1 case achieved partial remission and 1 case didn't achieve remission. During the treatment, 8 patients had a transient abnormal liver function, 3 cases had skin rash, and 3 cases had fever.Conclusion HD-MTX has a good therapeutic effect, especially for adult rrLCH patients with bone involvement, and the adverse events are controllable. -
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