Observational studyJournal of clinical oncology : official journal of the American Society of Clinical Oncology2019
Vemurafenib for Refractory Multisystem Langerhans Cell Histiocytosis in Children: An International Observational Study.
Observational study in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this map. Cited by 106 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Optimization of the Time and Dosage of Vemurafenib in BRAF Positive Juvenile Patients With Refractory Histiocytosis
A Real-world Investigation of the Combination of Darafenib or Trametinib and Clofarabine in the Treatment of High-risk, Recurrent, or Refractory Pediatric Patients With Langerhans Cell Histiocytosis.
A Multi-center, Open-label, Single-arm Study of Luvometinib Monotherapy in Pediatric Langerhans Cell Histiocytosis With Single-system Special-site Single and Multifocal Bone Involvement
Modified LCH-III Regimen Versus Modified LCH-III Regimen Combined With Luvometinib in the Treatment of Multisystem Pediatric Langerhans Cell Histiocytosis: A Multicenter, Open-Label, Randomized Controlled Clinical Trial
Who cites it
106 citing papers in PubMed, 1 synthesis or guideline pooled it, 214 citations in OpenAlex.
- Isolated clival Langerhans cell histiocytosis in a pediatric patient: systematic review and illustrative case report.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Pooled it
- Clofarabine for relapsed/refractory LCH and non-LC histiocytosis.Blood advances · 2026Trial
- Intensification of treatment with vinca alkaloid does not improve outcomes in pediatric patients with Langerhans cell histiocytosis: results from the JPLSG LCH-12 study.International journal of hematology · 2023Trial
- Phase 2 study of oral thalidomide-cyclophosphamide-dexamethasone for recurrent/refractory adult Langerhans cell histiocytosis.Leukemia · 2022Trial
- Nine Years of Vemurafenib Therapy in BRAF V600E-Positive Multisystem Langerhans Cell Histiocytosis with Asymptomatic Neurodegenerative Changes.Indian journal of pediatrics · 2026Article
- Langerhans cell histiocytosis: molecular mechanisms underlying pathogenesis and emerging targeted therapeutics.Cancer metastasis reviews · 2026Review
- Histiocytosis development and clinical variation through the lens of genomics.The Journal of pathology · 2026Review
- Advances and Challenges in the Multidisciplinary Treatment of Retroperitoneal Infantile Fibrosarcoma in Early Life.European journal of haematology · 2026Article
- Article
- When Skin-Limited Langerhans Cell Histiocytosis Becomes Life-Threatening: Severe Treatment-Related Morbidity in a Prematurely Born Infant-Case Report.Reports (MDPI) · 2026Article
- Langerhans cell histiocytosis in the adult lower gastrointestinal tract.BMJ case reports · 2026Article
- Cytarabine-based induction and oral maintenance therapy for a single system with single-site Langerhans cell histiocytosis.International journal of hematology · 2026Article
- [A rare case of Rosai-Dorfman disease involving the pituitary stalk and presenting initially with diabetes insipidus].Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences · 2026Article
- Origin, diagnosis and treatment of periocular Histiocytic disorders- state-of-the-art review.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2026Review
- Efficacy of MAPK inhibitors in children with neurodegenerative Langerhans cell histiocytosis: Results from the European Consortium for Histiocytosis.HemaSphere · 2026Article
- Long-term MAPK inhibition of childhood refractory-Langerhans cell histiocytosis: an observational study of 288 patients.Blood advances · 2026Observational
- Efficacy, safety, and relapse outcomes of MAPK inhibitors in pediatric Langerhans cell histiocytosis: A real-world study.International journal of cancer · 2026Article
- Cell-free BRAFHaematologica · 2026Article
- Article
- Sustained remission after discontinuation of ALK inhibition in ALK-positive histiocytosis.Blood cancer journal · 2026Article
46 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
46 authors at 20 institutions in 14 countries.
Funding
Abstract
purposeOff-label use of vemurafenib (VMF) to treat PATIENTS AND
methodsFifty-four patients from 12 countries took VMF 20 mg/kg/d. They were classified according to risk organ involvement: liver, spleen, and/or blood cytopenia. The main evaluation criteria were adverse events (Common Terminology Criteria for Adverse Events [version 4.3]) and therapeutic responses according to Disease Activity Score.
resultsLCH extent was distributed as follows: 44 with positive and 10 with negative risk organ involvement. Median age at diagnosis was 0.9 years (range, 0.1 to 6.5 years). Median age at VMF initiation was 1.8 years (range, 0.18 to 14 years), with a median follow-up of 22 months (range, 4.3 to 57 months), whereas median treatment duration was 13.9 months (for 855 patient-months). At 8 weeks, 38 complete responses and 16 partial responses had been achieved, with the median Disease Activity Score decreasing from 7 at diagnosis to 0 (
conclusionVMF seemed safe and effective in children with refractory
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.