Clofarabine monotherapy in two patients with refractory Langerhans cell histiocytosis

Masahiro Irie, Tomohiro Nakano, Saori Katayama, Tasuku Suzuki, Kunihiko Moriya, Yuko Watanabe, Nobu Suzuki, Yuka Saitoh-Nanjyo, Masaei Onuma, Takeshi Rikiishi, Hidetaka Niizuma, Yoji Sasahara, Shigeo Kure

研究成果: Article査読


Background: Better therapeutic options other than conventional chemotherapy for pediatric patients with refractory Langerhans cell histiocytosis (LCH) remain undetermined. Case: We successfully treated two patients with refractory and risk organ negative LCH with clofarabine (CLO) monotherapy after recurrence. We administered total 23 courses of CLO monotherapy in patient 1 and 4 courses in patient 2. Both patients had distinct clinical manifestations but achieved a durable complete response with acceptable adverse effects of transient myelosuppression. CLO monotherapy was still effective when he had the second recurrent lesion after first completion of CLO in patient 1. We could discontinue prednisolone to control his refractory inflammation of LCH after completing CLO chemotherapy in patient 2. Conclusion: Although large-scale studies are warranted, CLO monotherapy could be a therapeutic option for high efficacy and feasibility besides other intensive combination chemotherapies or allogeneic hematopoietic stem cell transplantation for refractory LCH without risk organ involvement in children.

ジャーナルCancer Reports
出版ステータスPublished - 2022 8月

ASJC Scopus subject areas

  • 腫瘍学
  • 癌研究


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