Into the Light: Afamelanotide and the Treatment of Erythropoietic Protoporphyria in the United States.
| Autor: | Resnik SR, Targett D, Resnik BI |
|---|---|
| Jazyk: | angličtina |
| Zdroj: | Journal of drugs in dermatology : JDD [J Drugs Dermatol] 2023 Sep 01; Vol. 22 (9), pp. 941-949. |
| DOI: | 10.36849/JDD.7126 |
| Abstrakt: | Background: Erythropoietic protoporphyria (EPP) is a rare disease that causes disabling cutaneous photosensitivity with pain and burning sensations. In 2019, afamelanotide, an α-melanocyte-stimulating hormone analogue, was approved in the United States for treatment of EPP. In this study, patients receiving afamelanotide filled out questionnaires assessing the benefit of treatment. Outcomes measured included: return to normal activities, experience of phototoxic reactions, effect on patient confidence, and more. Patients ranked their experience on a descriptive scale ranging from "very much" to "never". Results: Prior to treatment, 75% of patients indicated that EPP affected their lives "very much" or "a lot". This number fell to 11% after the 1st implant and to 0% after each subsequent implant. The number of patients that willingly ventured outside increased with each subsequent implant. Conclusion: The results of this study clearly show that afamelanotide treatment can dramatically and positively impact the lives of EPP patients. Citation: Resnik SR, Targett D, Resnik BI. Into the light: afamelanotide and the treatment of erythropoietic protoporphyria in the United States. J Drugs Dermatol. 2023;22(9):941-949. doi:10.36849/JDD.7126R1. |
| Databáze: | MEDLINE |
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