Methotrexate and cytarabine for adult patients with newly diagnosed Langerhans cell histiocytosis: A single arm, single center, prospective phase 2 study

作者：Xinxin Cao, Li J, Ailin Zhao, Tianhua He, Xuemin Gao, Huacong Cai, Lu Zhang, Yan Zhang, Jun Feng, Tienan Zhu, Na Niu, Jian Sun, Zhi‐Yong Liang, Minghui Duan, Daobin Zhou · 发表于：American Journal of Hematology · 年份：2020 · DOI：10.1002/ajh.25864 · 被引用次数：36 · 研究领域：Histiocytic Disorders and Treatments、Genital Health and Disease、Mast cells and histamine

Langerhans cell histiocytosis (LCH) is a rare, heterogeneous histiocytic disorder occurring in patients of all ages. The current standard treatment protocol for children with de novo multisystem LCH is vinblastine plus prednisone.1 This regimen has never been proven effective for adults in a prospective study. Moreover, our previous report showed the reactivation frequency was 71%-78.6% after vindesine and prednisone-based therapy.2 These data indicate the concept of treatment for children probably cannot be translated to adults. A retrospective study showed an advantage for cytarabine monotherapy compared with vinblastine/prednisone in bone LCH patients.3 But no consensus on the best first-line treatment strategy has yet been reached. Oral methotrexate had been successfully used in skin LCH previously.4 Considering the relatively high frequency of pituitary involvement and late onset of neurodegenerative symptoms, patients may benefit from the combination of cytarabine and methotrexate, as both these drugs cross the blood-brain barrier. The successful experience of combination methotrexate/cytarabine used in non-Hodgkinlymphoma afforded us a reference point for choosing the dosage of methotrexate.5 This phase 2, prospective, single-center study was designed to evaluate the efficacy and safety of a methotrexate and cytarabine (MA) treatment regimen in adults. This is with newly diagnosed multisystem disease (MS) LCH or LCH with multifocal single system (SS-m) involvement. We ...