Abstract | BACKGROUND: In erythropoietic protoporphyria (EPP), an inherited disease of porphyrin-biosynthesis, the accumulation of protoporphyrin in the skin causes severely painful phototoxic reactions. Symptom prevention was impossible until recently when afamelanotide became available. Afamelanotide-induced skin pigmentation has statistically significantly improved light-tolerance, although the clinical significance of the statistical effect was unknown. OBJECTIVES: To assess clinical effectiveness by recording compliance and safety during prolonged use. METHODS: We report longitudinal observations of 115 ambulatory patients with EPP, who were treated with a total of 1023 afamelanotide implants over a period of up to 8 years at two porphyria centres; one in Rome, Italy, and the other in Zurich, Switzerland. RESULTS: Since the treatment first became available in 2006, the number of patients treated with 16 mg afamelanotide implants rose continuously until June 2014, when 66% of all patients with EPP known to the porphyria centres were treated. Only three patients considered afamelanotide did not meet their expectations for symptom improvement; 23% discontinued the treatment for other, mostly compelling, reasons such as pregnancy or financial restrictions. The quality of life (QoL) scores, measured by an EPP-specific questionnaire, were 31 ± 24% of maximum prior to afamelanotide treatment, rose to 74% after starting afamelanotide and remained at this level during the entire observation period. Only minor adverse events attributable to afamelanotide, predominantly nausea, were recorded. CONCLUSION: Based on the improved QoL scores, high compliance and low discontinuation rates, we conclude that afamelanotide exhibits good clinical effectiveness and good safety in EPP under long-term routine conditions.
|
Authors | G Biolcati, E Marchesini, F Sorge, L Barbieri, X Schneider-Yin, E I Minder |
Journal | The British journal of dermatology
(Br J Dermatol)
Vol. 172
Issue 6
Pg. 1601-1612
(Jun 2015)
ISSN: 1365-2133 [Electronic] England |
PMID | 25494545
(Publication Type: Clinical Trial, Phase II, Clinical Trial, Phase III, Journal Article, Multicenter Study, Observational Study)
|
Copyright | © 2014 The Authors. British Journal of Dermatology published by John Wiley & Sons Ltd on behalf of British Association of Dermatologists. |
Chemical References |
- Delayed-Action Preparations
- Dermatologic Agents
- Melanins
- alpha-MSH
- afamelanotide
|
Topics |
- Administration, Cutaneous
- Adult
- Delayed-Action Preparations
- Dermatologic Agents
(administration & dosage, adverse effects)
- Female
- Humans
- Long-Term Care
- Male
- Medication Adherence
- Melanins
(metabolism)
- Protoporphyria, Erythropoietic
(drug therapy)
- Quality of Life
- Retrospective Studies
- Treatment Outcome
- alpha-MSH
(administration & dosage, adverse effects, analogs & derivatives)
|