Intensification of treatment with vinca alkaloid does not improve outcomes in pediatric patients with Langerhans cell histiocytosis: results from the JPLSG LCH-12 study.

Akira Morimoto, Yoko Shioda, Kazuko Kudo, Hirokazu Kanegane, Toshihiko Imamura, Katsuyoshi Koh, Yoshiyuki Kosaka, Yuki Yuza, Atsuko Nakazawa, Akiko M Saito, Tomoyuki Watanabe, Yozo Nakazawa

Journal: International journal of hematology 2023;118(1):107-118

PMID: 36871086

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Abstract

Chemotherapy with cytarabine, vincristine (VCR), and prednisolone has achieved low mortality rates in pediatric patients with Langerhans cell histiocytosis (LCH). However, relapse rates remain high, making event-free survival (EFS) rates unsatisfactory. A nationwide clinical trial, LCH-12, tested a modified protocol in which the early maintenance phase was intensified with increasing dosages of VCR. Patients newly diagnosed with multifocal bone (MFB) or multisystem (MS) LCH and aged < 20 years at diagnosis were enrolled between June 2012 and November 2017. Of the 150 eligible patients, 43 with MFB were treated for 30 weeks and 107 with MS LCH were treated for 54 weeks. One patient with MS LCH died of sepsis during the induction phase. The 3-year EFS rates among patients with MFB LCH, risk organ (RO)-negative MS LCH, and RO-positive MS LCH were 66.7% (95% confidential interval [CI], 56.5-77.0%), 66.1% (95% CI 52.9-76.4%), and 51.1% (95% CI 35.8-64.5%), respectively, similar to previously observed rates. EFS rates were significantly lower in patients with disease activity scores > 6 than in those with scores ≤ 6. The strategy that included more intense treatment with VCR was not effective. Other strategies are required to improve outcomes in patients with pediatric LCH.

Address: Department of Pediatrics, Jichi Medical University School of Medicine, Shimotsuke, Japan. [email protected].; Department of Pediatrics, Showa Inan General Hospital, 3230, Akaho, Komagane, Nagano, 399-4117, Japan. [email protected].; Children's Cancer Center, National Center for Child Health and Development, Tokyo, Japan.; Department of Pediatrics, Fujita Health University School of Medicine, Toyoake, Japan.; Department of Child Health and Development, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.; Department of Pediatrics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, Kyoto, Japan.; Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.; Department of Hematology/Oncology, Hyogo Prefectural Kobe Children's Hospital, Kobe, Japan.; Departments of Hematology/Oncology, Tokyo Metropolitan Children's Medical Center, Tokyo, Japan.; Department of Clinical Research, Saitama Children's Medical Center, Saitama, Japan.; Clinical Research Center, National Hospital Organization Nagoya Medical Center, Nagoya, Japan.; Department of Health and Nutrition, Faculty of Psychological and Physical Science, Aichi Gakuin University, Nisshin, Japan.; Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.

DOI: 10.1007/s12185-023-03568-0

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https://dx.doi.org/10.1007/s12185-023-03568-0

MeSH Terms: Child, Humans, Antineoplastic Agents, Cytarabine, Histiocytosis, Langerhans-Cell, Retrospective Studies, Treatment Outcome, Vincristine