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Br J Dermatol. 2015 Jun;172(6):1601-12. doi: 10.1111/bjd.13598. Epub 2015 Apr 30.

Long-term observational study of afamelanotide in 115 patients with erythropoietic protoporphyria.

Author information

1
Porphyria Centre, San Gallicano Dermatological Institute, IRCCS - IFO, Rome, Italy.
2
FarmED Service, Pharmacovigilance - Pharmacy - IFO, Rome, Italy.
3
Institute of Laboratory Medicine, Municipal Hospital Triemli, Zurich, Switzerland.

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.

Comment in

PMID:
25494545
DOI:
10.1111/bjd.13598
[Indexed for MEDLINE]
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