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阿法美拉肽:一种具有广泛皮肤科应用潜力的孤儿药。

Afamelanotide: An Orphan Drug with Potential for Broad Dermatologic Applications.

出版信息

J Drugs Dermatol. 2021 Mar 1;20(3):290-294. doi: 10.36849/JDD.5526.

DOI:10.36849/JDD.5526
PMID:33683075
Abstract

Afamelanotide (SCENESSE®) is a synthetic analogue of α-melanocyte-stimulating hormone that is FDA-approved to increase pain-free sunlight exposure in adult patients with erythropoietic protoporphyria. Its dual photoprotective and anti-inflammatory effects also make it a promising therapy for other photosensitive dermatologic diseases that are resistant to treatment. The PubMed/MEDLINE and ClinicalTrials.gov databases were searched for literature and ongoing trials describing the use of afamelanotide in treating cutaneous diseases. There is randomized controlled trial (RCT) evidence for the successful use of afamelanotide in several conditions beyond erythropoietic protoporphyria, including polymorphic light eruption and vitiligo. Smaller studies have also demonstrated its efficacy in treating acne vulgaris, Hailey-Hailey disease, and solar urticaria. No serious adverse effects with afamelanotide use have been reported, though diffuse hyperpigmentation is experienced by almost all patients. Larger scale studies are needed to confirm the efficacy of afamelanotide in treating dermatologic conditions beyond erythropoietic protoporphyria, and further research should focus on determining the safety, efficacy, and optimal dosing of afamelanotide for pediatric patients.J Drugs Dermatol. 2021;20(3):290-294. doi:10.36849/JDD.5526.

摘要

阿法美拉汀(SCENESSE®)是一种合成的 α-促黑素细胞激素类似物,已获得美国食品药品监督管理局(FDA)批准,可增加红细胞生成性原卟啉症成年患者无痛日晒时间。其双重光保护和抗炎作用也使其成为一种有前途的治疗方法,可用于治疗其他对治疗有抗药性的光敏性皮肤病。检索了 PubMed/MEDLINE 和 ClinicalTrials.gov 数据库,以获取描述阿法美拉汀治疗皮肤疾病的文献和正在进行的试验。有随机对照试验(RCT)证据表明,阿法美拉汀在除红细胞生成性原卟啉症以外的几种疾病中的应用取得了成功,包括多形性日光疹和白癜风。较小的研究也表明其在治疗寻常痤疮、Hailey-Hailey 病和光线性荨麻疹方面的疗效。尽管几乎所有患者都会出现弥漫性色素沉着,但尚未报告使用阿法美拉汀的严重不良反应。需要更大规模的研究来证实阿法美拉汀在治疗除红细胞生成性原卟啉症以外的皮肤科疾病方面的疗效,进一步的研究应侧重于确定阿法美拉汀治疗儿科患者的安全性、疗效和最佳剂量。J 皮肤病药物杂志。2021;20(3):290-294.doi:10.36849/JDD.5526.

相似文献

1
Afamelanotide: An Orphan Drug with Potential for Broad Dermatologic Applications.阿法美拉肽:一种具有广泛皮肤科应用潜力的孤儿药。
J Drugs Dermatol. 2021 Mar 1;20(3):290-294. doi: 10.36849/JDD.5526.
2
Pharmacokinetics and Pharmacodynamics of Afamelanotide and its Clinical Use in Treating Dermatologic Disorders.阿法美拉肽的药代动力学和药效学及其在治疗皮肤病中的临床应用。
Clin Pharmacokinet. 2017 Aug;56(8):815-823. doi: 10.1007/s40262-016-0501-5.
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[Medicaments and oral healthcare. Hyperpigmentation of oral soft tissues due to afamelanotide].[药物与口腔保健。阿法美拉酮所致口腔软组织色素沉着]
Ned Tijdschr Tandheelkd. 2020 Apr;127(4):237-243. doi: 10.5177/ntvt.2020.04.19115.
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Expert Rev Clin Pharmacol. 2021 Feb;14(2):151-160. doi: 10.1080/17512433.2021.1879638.
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A review and update on melanocyte stimulating hormone therapy: afamelanotide.促黑素细胞激素疗法综述与更新:阿法美拉肽
J Drugs Dermatol. 2013 Jul 1;12(7):775-9.
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Afamelanotide: A Review in Erythropoietic Protoporphyria.阿法美拉诺肽:红细胞生成性原卟啉症治疗药物评价。
Am J Clin Dermatol. 2016 Apr;17(2):179-85. doi: 10.1007/s40257-016-0184-6.
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Pseudoleucoderma after injections of afamelanotide in a patient with atopic dermatitis.一名特应性皮炎患者注射阿法美拉汀后出现假白斑。
Acta Derm Venereol. 2011 Sep;91(5):578-9. doi: 10.2340/00015555-1062.
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Skin Therapy Lett. 2018 Nov;23(6):6-10.
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Long-term observational study of afamelanotide in 115 patients with erythropoietic protoporphyria.长程观察研究阿法美拉诺肽治疗 115 例红细胞生成性原卟啉症患者。
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The effects of cholecalciferol and afamelanotide on vitamin D levels in erythropoietic protoporphyria: a multicentre cohort study.胆钙化醇和阿法美拉诺肽对红细胞生成性原卟啉症患者维生素 D 水平的影响:一项多中心队列研究。
Br J Dermatol. 2024 Aug 14;191(3):357-364. doi: 10.1093/bjd/ljae148.

引用本文的文献

1
[Solar urticaria and polymorphous light eruption].[日光性荨麻疹和多形性日光疹]
Dermatologie (Heidelb). 2024 Jul;75(7):518-527. doi: 10.1007/s00105-024-05368-x. Epub 2024 Jun 12.
2
Afamelanotide in protoporphyria and other skin diseases: a review.阿法美拉酮在原卟啉症及其他皮肤病中的应用:综述
Postepy Dermatol Alergol. 2024 Apr;41(2):149-154. doi: 10.5114/ada.2024.138818. Epub 2024 Apr 12.
3
Protoporphyrin IX-induced phototoxicity: Mechanisms and therapeutics.原卟啉 IX 诱导的光毒性:机制与治疗。
Pharmacol Ther. 2023 Aug;248:108487. doi: 10.1016/j.pharmthera.2023.108487. Epub 2023 Jun 29.