Pediatric hematology and oncology
September 23, 2025 · via Europe PMC
Clofarabine monotherapy in refractory multisystem LCH with gastrointestinal involvement.
Aronson S, Tang V, Cios K, Pittman M, Çeliker MY.
AI summary — for doctors, generated from the abstract only
Two children with gastrointestinal Langerhans cell histiocytosis refractory to vinblastine, prednisone and cytarabine achieved remission with clofarabine monotherapy.
What was studied
Case report of two patients with multisystem LCH involving the gastrointestinal tract that was refractory to first- and second-line therapy.
Key findings
- Both cases refractory to vinblastine/prednisone and cytarabine
- Clofarabine monotherapy led to clinical remission in both
- Authors propose GI involvement be considered high risk in LCH stratification
Clinical relevance
This raises the question of whether clofarabine should be studied earlier for GI-LCH and whether GI involvement warrants high-risk classification, but evidence is limited to two cases.
Who should care
Limitations
Two-patient case report; no generalizable efficacy or safety data.
Clofarabine rescued two children with treatment-resistant gut involvement from LCH.
This is an AI-generated summary of the published abstract, not a clinical recommendation. It may not reflect the full text or subsequent updates — verify against the original source before acting on it.
Abstract
Langerhans Cell Histiocytosis (LCH) is a rare histiocytic disorder that involves various organ systems. Recognizing gastrointestinal (GI) tract involvement in LCH is crucial, as GI cases, even without high-risk organ involvement, may be refractory to initial as well as second line therapy. Here, we report two cases of LCH with GI involvement, that were refractory to vinblastine, prednisone and cytarabine, and ultimately required clofarabine to reach clinical remission. We propose that clofarabine should be further studied as a first choice for second line treatment for GI-LCH cases and that GI involvement should be considered as high risk in the LCH risk stratification algorithm.
DOI
10.1080/08880018.2025.2563535
PMID
40988414
Research metadata (titles, abstracts, authors, publication dates) is sourced from Europe PMC, which aggregates content from PubMed/MEDLINE, PubMed Central, and other providers under the U.S. National Library of Medicine (NLM/NCBI). NLM does not claim copyright on abstracts; the original journal publishers or authors may hold rights to the full articles. AI-generated summaries are derived only from the abstract, never the full text, and are always labeled as AI-generated.
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