J Drugs Dermatol. 2023 Sep 1;22(9):941-949. doi: 10.36849/JDD.7126.
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".
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.
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.
红细胞生成性原卟啉症（EPP）是一种罕见的疾病，可导致严重的皮肤光敏性，伴有疼痛和烧灼感。2019 年，美国批准了一种α-促黑素细胞激素类似物阿法美拉诺肽用于 EPP 的治疗。在这项研究中，接受阿法美拉诺肽治疗的患者填写了评估治疗益处的问卷。测量的结果包括：恢复正常活动、光毒性反应的体验、对患者信心的影响等。患者根据描述性量表对他们的体验进行评分，范围从“非常”到“从不”。
在治疗前，75%的患者表示 EPP“非常”或“很大”程度上影响了他们的生活。这一数字在第一次植入后降至 11%，随后每次植入后降至 0%。愿意冒险外出的患者人数随着每次后续植入而增加。
这项研究的结果清楚地表明，阿法美拉诺肽治疗可以显著地、积极地影响 EPP 患者的生活。
