Oncology · Neurology · 1 h ago
BRAF–MEK inhibition shrank tumors in two papillary craniopharyngioma cases
A Swedish report describes two adults with BRAF V600E-mutated papillary craniopharyngioma treated with dabrafenib and trametinib. Both had tumor shrinkage; one also had improved visual acuity. The findings add to phase 2 evidence but do not establish how long treatment should continue or the risk of recurrence after stopping.
- Both cases had BRAF V600E-mutated tumors.
- Tumor shrinkage occurred within months of targeted therapy.
- Treatment duration and post-treatment recurrence risk remain uncertain.
Papillary craniopharyngiomas often carry a BRAF V600E mutation and can affect the hypothalamus, pituitary gland and visual pathways. The report describes two adults treated with the BRAF inhibitor dabrafenib and MEK inhibitor trametinib when surgery or radiotherapy posed concerns. The authors also summarize a phase 2 multicenter study of 16 patients treated with vemurafenib and cobimetinib after recurrence following surgery.
In the first case, tumor volume fell from 2,220 mm³ to 90 mm³ after 12 months of treatment. Therapy was stopped after 16 months, followed by radiotherapy; MRI showed no recurrence during two years of follow-up. In the second case, tumor volume decreased from 633 mm³ to 483 mm³ during 31 months of therapy, and visual acuity in the left eye improved from 0.05 to 0.3. The patient remained recurrence-free 43 months after treatment ended. In the phase 2 study, 15 of 16 patients had tumor shrinkage, with a median reduction of 91%.
Reported adverse effects in the two cases included uveitis, mild gastrointestinal symptoms and transient NT-proBNP elevation. These cases support considering molecularly targeted treatment in selected patients, particularly when local treatment risks damage to critical structures. Treatment duration and recurrence risk after discontinuation remain uncertain; a Swedish study (NCT05525273) is investigating the approach.
In practice
For patients with BRAF V600E-mutated papillary craniopharyngioma, BRAF–MEK inhibition may be a treatment option to discuss when surgery or radiotherapy risks injury to structures such as the hypothalamus or optic pathways. This report describes tumor shrinkage in two adults, and cites a 16-patient phase 2 study in which 15 had tumor reduction. The evidence does not establish optimal treatment duration or recurrence risk after stopping; the approach is being studied prospectively and does not by itself define a standard of care.
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Läkartidningen: BRAF- och MEK- hämmare vid papillära kraniofaryngiom ↗This is an automated AI-condensed summary that has not yet been reviewed by an editor. Always consult the full item at the original source.