Association of afamelanotide with improved outcomes in patients with erythropoietic protoporphyria in clinical practice
JAMA Mar 22, 2020
Wensink D, Wagenmakers MAEM, Barman-Aksözen J, et al. - Researchers conducted this single-center, prospective postauthorization safety and efficacy cohort study to explore the correlation of afamelanotide treatment with outcomes in individuals with erythropoietic protoporphyria (EPP) in regular practice during longer-term follow-up. This investigation was directed and approved by the European Medicines Agency. In total, 117 patients with EPP (mean [SD] age, 43.0 [15.5] years) were treated with afamelanotide. For patients with EPP, afamelanotide treatment was related to improved clinical results and a good safety profile. The treatment has clinically important, sustained positive connections with quality of life, is associated with increased sun exposure duration, and is related to lower phototoxic reactions. Minor and self-limiting adverse events including nausea, fatigue, and headache have been identified.
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