A single-centre experience of patients with metastatic melanoma enrolled in a dabrafenib named patient programme

作者：Lau David K; Andrews Miles C; Turner Natalie; Azad Arun A; Davis Ian D; Cebon Jonathan S^*

来源：Melanoma Research, 2014, 24(2): 144-149.

DOI：10.1097/CMR.0000000000000036

• dabrafenib
• named patient programme
• BRAF inhibitor
• cerebral metastasis
• metastatic melanoma

We studied the efficacy, tolerability and clinical courses of dabrafenib in patients with metastatic melanoma who were ineligible for enrolment into a clinical trial. Between July 2011 and May 2013, patients with unresectable stage III or stage IV, V600-mutated metastatic melanoma who were not eligible for inclusion into clinical trials were offered treatment with dabrafenib through a named patient programme. Routine efficacy and toxicity data were collected throughout treatment and studied retrospectively. The endpoints were progression-free survival (PFS), overall survival and best overall response. Thirty-one patients commenced dabrafenib therapy including six individuals who had progressed on a prior BRAF-inhibitor treatment. The majority of patients had cerebral metastases (n=17) and/or a poor performance status [Eastern Cooperative Oncology Group (ECOG)>= 2, n=11]. Median overall survival was 5.6 months (range 0.1-22 months). Median PFS was 3.3 months (range 0.1-21) and was similar despite performance status. One patient had a complete response and eight showed partial responses to treatment. Patients with cerebral metastases (n=17) had a median PFS of 4.6 months. Five patients (16%) had dose-limiting toxicities. Despite several poor prognostic features, dabrafenib is a safe and effective treatment in the community setting, with occasional impressive outcomes.

• 出版日期2014-4