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

doi:10.1002/cnr2.1579

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

Pediatrics

Research output: Contribution to journal › Article › peer-review

Abstract

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.

Original language	English
Article number	e1579
Journal	Cancer Reports
Volume	5
Issue number	8
DOIs	https://doi.org/10.1002/cnr2.1579
Publication status	Published - 2022 Aug

Keywords

allogeneic hematopoietic stem cell transplantation
cladribine
clofarabine
high dose-cytarabine
refractory Langerhans cell histiocytosis

ASJC Scopus subject areas

Oncology
Cancer Research

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1002/cnr2.1579

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@article{6664507ae81142b29e94521cfec4af28,

title = "Clofarabine monotherapy in two patients with refractory Langerhans cell histiocytosis",

abstract = "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.",

keywords = "allogeneic hematopoietic stem cell transplantation, cladribine, clofarabine, high dose-cytarabine, refractory Langerhans cell histiocytosis",

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

note = "Funding Information: We thank all colleagues in the Department of Pediatrics, Tohoku University Hospital who have been involved in patient care. We also thank Dr. Akira Morimoto in Jichi Medical University for his kind advice. This work was supported by grants‐in‐aid from the Ministry of Education, Culture, Sports, Science and Technology of Japan (19K08855 to Y.S.) and a grant from the Ministry of Health, Labour and Welfare of Japan (20EA1020 to Y.S.). Publisher Copyright: {\textcopyright} 2021 The Authors. Cancer Reports published by Wiley Periodicals LLC.",

year = "2022",

month = aug,

doi = "10.1002/cnr2.1579",

language = "English",

volume = "5",

journal = "Cancer Reports",

issn = "2573-8348",

publisher = "Wiley-Blackwell Publishing Ltd",

number = "8",

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TY - JOUR

T1 - Clofarabine monotherapy in two patients with refractory Langerhans cell histiocytosis

AU - Irie, Masahiro

AU - Nakano, Tomohiro

AU - Katayama, Saori

AU - Suzuki, Tasuku

AU - Moriya, Kunihiko

AU - Watanabe, Yuko

AU - Suzuki, Nobu

AU - Saitoh-Nanjyo, Yuka

AU - Onuma, Masaei

AU - Rikiishi, Takeshi

AU - Niizuma, Hidetaka

AU - Sasahara, Yoji

AU - Kure, Shigeo

N1 - Funding Information: We thank all colleagues in the Department of Pediatrics, Tohoku University Hospital who have been involved in patient care. We also thank Dr. Akira Morimoto in Jichi Medical University for his kind advice. This work was supported by grants‐in‐aid from the Ministry of Education, Culture, Sports, Science and Technology of Japan (19K08855 to Y.S.) and a grant from the Ministry of Health, Labour and Welfare of Japan (20EA1020 to Y.S.). Publisher Copyright: © 2021 The Authors. Cancer Reports published by Wiley Periodicals LLC.

PY - 2022/8

Y1 - 2022/8

N2 - 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.

AB - 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.

KW - allogeneic hematopoietic stem cell transplantation

KW - cladribine

KW - clofarabine

KW - high dose-cytarabine

KW - refractory Langerhans cell histiocytosis

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DO - 10.1002/cnr2.1579

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ER -

Clofarabine monotherapy in two patients with refractory Langerhans cell histiocytosis

Abstract

Keywords

ASJC Scopus subject areas

UN SDGs

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