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

Biolcati G, Marchesini E, Sorge F, Barbieri L, Schneider-Yin X, Minder EI

The British journal of dermatology30 Apr 2015Cited by 71 (Europe PMC)

PMID25494545DOI10.1111/bjd.13598PepBankPUB-25494545

Publisher page PubMedAsk about this study

Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: CC BY-NC-ND. Results still quote short attributed sentences.

Results from this study6

6 results from studies that gave the peptide. 6 results from people.

01HumanObservational studyEfficacy outcome
In patients with erythropoietic protoporphyria, quality of life scores rose from 31% of maximum before afamelanotide to 74% after starting it and stayed at that level.
In plain wordsIn people with erythropoietic protoporphyria, quality of life scores rose after starting afamelanotide and stayed higher during the whole observation period.
Peptide
Melanotan I (afamelanotide)
Outcome
IncreaseQuality of life score (EPP-specific questionnaire)
Study
n = 115
Outcome group
Quality of life · Skin and wound
Studied dose
16 mg · subcutaneous implant · up to 8 yearsAs given in this study (human).
Measurements
QoL score before treatment
31 % of maximum
before afamelanotide
QoL score after treatment
74 % of maximum
after starting afamelanotide
Source sentences
Summary
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.
1 context sentence(design, population, supporting numbers)
Summary · context
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.
Report a problemFND-2549454501
02HumanObservational studySafety outcome
In patients with erythropoietic protoporphyria, only minor adverse events attributable to afamelanotide were recorded.
In plain wordsIn people with erythropoietic protoporphyria, afamelanotide caused only minor side effects.
Peptide
Melanotan I (afamelanotide)
Outcome
Not applicableAdverse events attributable to afamelanotide
Study
n = 115
Outcome group
Adverse events · Skin and wound
Studied dose
16 mg · subcutaneous implant · up to 8 yearsAs given in this study (human).
Source sentences
Summary
Only minor adverse events attributable to afamelanotide, predominantly nausea, were recorded.
1 context sentence(design, population, supporting numbers)
Summary · context
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.
Report a problemFND-2549454502
03HumanObservational studySafety outcome
In patients with erythropoietic protoporphyria, nausea was the predominant minor adverse event attributable to afamelanotide.
In plain wordsIn people with erythropoietic protoporphyria, nausea was the most common minor side effect of afamelanotide.
Peptide
Melanotan I (afamelanotide)
Outcome
Not applicableNausea
Study
n = 115
Outcome group
Adverse events · Gastrointestinal
Studied dose
16 mg · subcutaneous implant · up to 8 yearsAs given in this study (human).
Source sentences
Summary
Only minor adverse events attributable to afamelanotide, predominantly nausea, were recorded.
1 context sentence(design, population, supporting numbers)
Summary · context
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.
Report a problemFND-2549454503
04HumanObservational studyOther
In patients with erythropoietic protoporphyria, 23% discontinued afamelanotide for other, mostly compelling, reasons such as pregnancy or financial restrictions.
In plain wordsIn people with erythropoietic protoporphyria, 23% stopped afamelanotide, mostly for reasons such as pregnancy or cost.
Peptide
Melanotan I (afamelanotide)
Outcome
Not applicableTreatment discontinuation for other reasons
Study
n = 115
Outcome group
Treatment adherence and retention · Whole body
Studied dose
16 mg · subcutaneous implant · up to 8 yearsAs given in this study (human).
Measurements
Discontinuation
23%
other reasons such as pregnancy or financial restrictions
Source sentences
Summary
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.
1 context sentence(design, population, supporting numbers)
Summary · context
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.
Report a problemFND-2549454504
05HumanObservational studyEfficacy outcome
In patients with erythropoietic protoporphyria, only three considered that afamelanotide did not meet their expectations for symptom improvement.
In plain wordsIn people with erythropoietic protoporphyria, only three patients felt afamelanotide did not meet their expectations.
Peptide
Melanotan I (afamelanotide)
Outcome
Not applicablePatients whose symptom improvement did not meet expectations
Study
n = 115
Outcome group
Quality of life · Skin and wound
Studied dose
16 mg · subcutaneous implant · up to 8 yearsAs given in this study (human).
Source sentences
Summary
Only three patients considered afamelanotide did not meet their expectations for symptom improvement
1 context sentence(design, population, supporting numbers)
Summary · context
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.
Report a problemFND-2549454505
06HumanObservational studyOther
At two porphyria centres, 66% of all patients with erythropoietic protoporphyria known to the centres were treated with 16 mg afamelanotide implants by June 2014.
In plain wordsBy June 2014, about two thirds of known patients with erythropoietic protoporphyria at two centres were treated with afamelanotide implants.
Peptide
Melanotan I (afamelanotide)
Outcome
Not applicableShare of known EPP patients treated by June 2014
Study
n = 115
Outcome group
Patterns of use · Skin and wound
Studied dose
16 mg · subcutaneous implant · up to 8 yearsAs given in this study (human).
Measurements
Patients treated
66%
June 2014, two porphyria centres
Source sentences
Summary
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.
1 context sentence(design, population, supporting numbers)
Summary · context
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.
Report a problemFND-2549454506

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Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
13 Oct 2026, 21:44 UTC
Study results updated
Version 1
13 Oct 2026, 16:31 UTC
First added · source update from Europe PMC

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