Vemurafenib for refractory multisystem Langerhans cell histiocytosis in children: an international observational study
In children with life-threatening Langerhans cell histiocytosis that had not responded to chemotherapy, the BRAF inhibitor vemurafenib brought the disease under control in nearly all of them, though it usually returned when the drug was stopped.
Overview
International observational study of 54 children with BRAF V600E-mutant multisystem LCH refractory to chemotherapy, most with risk-organ involvement, treated with vemurafenib.
All children responded and 38 (70 percent) had a complete response at eight weeks; no deaths occurred on treatment. Among those who stopped vemurafenib, about 80 percent relapsed, so most resumed treatment. Skin toxicity was common.
- Complete response at eight weeks in 38 of 54 children (70 percent) with no on-treatment deaths.
- Relapse in about 80 percent of children after stopping vemurafenib.
Vemurafenib rescues children with refractory BRAF-mutant multisystem LCH, but it controls rather than cures, and how to stop it safely is an open question.
- Observational compassionate-use series without a comparator.
- Long-term effects of BRAF inhibition in children are unknown.
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