Association of Afamelanotide With Improved Outcomes in Patients With Erythropoietic Protoporphyria in Clinical Practice
| العنوان: | Association of Afamelanotide With Improved Outcomes in Patients With Erythropoietic Protoporphyria in Clinical Practice |
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| المؤلفون: | Joost van Rosmalen, Jasmin Barman-Aksözen, Debby Wensink, J. H. Paul Wilson, Janneke G. Langendonk, Margreet A E M Wagenmakers, Edith C. H. Friesema |
| المساهمون: | Internal Medicine, Epidemiology |
| المصدر: | JAMA Dermatology, 156(5), 570-575. American Medical Association JAMA Dermatol |
| سنة النشر: | 2020 |
| مصطلحات موضوعية: | Adult, Male, medicine.medical_specialty, Adolescent, Protoporphyria, Erythropoietic, Dermatology, Cohort Studies, Young Adult, 030207 dermatology & venereal diseases, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Quality of life, Interquartile range, Internal medicine, Humans, Medicine, Prospective Studies, Young adult, Prospective cohort study, Adverse effect, Aged, business.industry, Brief Report, Middle Aged, medicine.disease, Treatment Outcome, chemistry, alpha-MSH, 030220 oncology & carcinogenesis, Quality of Life, Sunlight, Female, Afamelanotide, Dermatologic Agents, Erythropoietic protoporphyria, business, Cohort study |
| الوصف: | IMPORTANCE: The effectiveness of afamelanotide treatment in patients with erythropoietic protoporphyria (EPP) in clinical practice who experience pain after light exposure that substantially impairs quality of life is unknown. OBJECTIVE: To evaluate the association of afamelanotide treatment with outcomes in patients with EPP in regular practice during longer-term follow-up. DESIGN, SETTING, AND PARTICIPANTS: This single-center, prospective postauthorization safety and efficacy cohort study was directed and approved by the European Medicines Agency. Data were collected from patients with EPP treated with afamelanotide at Erasmus MC between June 2016 and September 2018. Analysis began October 2018. MAIN OUTCOMES AND MEASURES: Time spent outside during treatment, number of phototoxic reactions, disease-specific quality of life, usage of protective clothing, and adverse events. RESULTS: A total of 117 patients with EPP (59 women [50.4%]; mean [SD] age, 43.0 [15.5] years) were treated with afamelanotide. Nearly all patients continued treatment (115 [98%]) with a median (interquartile range) follow-up of 2.0 (1.3-2.1) years. Compared with baseline, mean time spent outside during treatment increased significantly by an added 6.1 hours per week (95% CI, 3.62-8.67; P |
| اللغة: | English |
| تدمد: | 2168-6068 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::35e925800d9ca5e7c25f99b2ccd0d087Test https://doi.org/10.1001/jamadermatol.2020.0352Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....35e925800d9ca5e7c25f99b2ccd0d087 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 21686068 |
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