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Study · Observational study · n = 38 · 2021

7 results

Beyond pigmentation: signs of liver protection during afamelanotide treatment in Swiss patients with erythropoietic protoporphyria, an observational study.

Minder AE, Barman-Aksoezen J, Schmid M, Minder EI, Zulewski H, Minder CE, Schneider-Yin X

Therapeutic advances in rare disease1 Jan 2021Cited by 6 (Europe PMC)

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. Results still quote short attributed sentences.

Results from this study7

7 results from studies that gave the peptide. 7 results from people.

  1. 01HumanObservational studyEfficacy outcome

    In 38 patients with erythropoietic protoporphyria, protoporphyrin concentrations were lower during afamelanotide treatment than before treatment.

    In plain wordsIn people with erythropoietic protoporphyria, protoporphyrin levels were lower during afamelanotide treatment than before it.

    Outcome
    DecreaseProtoporphyrin concentration
    Study

    n = 38

    Comparator
    Pretreatment values
    Outcome group
    Porphyria markers and attacks · Whole body
    Studied dose
    Dose not stated

    Measurements

    Protoporphyrin before treatment
    21.39 µmol/L
    before afamelanotide
    Protoporphyrin during treatment
    16.83 µmol/L
    during afamelanotide

    Source sentences

    Summary
    Protoporphyrin concentrations dropped from 21.39 ± 11.12 (mean ± SD) before afamelanotide to 16.83 ± 8.24 µmol/L (p < .0001) during treatment.
    1 context sentence
    Summary · context
    From the 47 Swiss EPP-patients treated at our center since 2006, we included those 38 patients in the current analysis who received at least one afamelanotide dose between 2016 and 2018 and underwent regular laboratory testing before and during the treatment.
  2. 02HumanObservational studyEfficacy outcome

    In 38 patients with erythropoietic protoporphyria, aspartate aminotransferase was lower during afamelanotide treatment than before treatment.

    In plain wordsIn people with erythropoietic protoporphyria, a liver blood test (aspartate aminotransferase) was lower during afamelanotide treatment than before it.

    Outcome
    DecreaseAspartate aminotransferase
    Study

    n = 38

    Comparator
    Pretreatment values
    Outcome group
    Liver fat and liver function · Liver
    Studied dose
    Dose not stated

    Measurements

    Aspartate aminotransferase before treatment
    26.67 IU/L
    before afamelanotide
    Aspartate aminotransferase during treatment
    22.9 IU/L
    during afamelanotide

    Source sentences

    Summary
    Aspartate aminotransferase decreased from 26.67 ± 13.16 to 22.9 ± 7.76 IU/L (p = .0146).
    1 context sentence
    Summary · context
    From the 47 Swiss EPP-patients treated at our center since 2006, we included those 38 patients in the current analysis who received at least one afamelanotide dose between 2016 and 2018 and underwent regular laboratory testing before and during the treatment.
  3. 03HumanObservational studyEfficacy outcome

    In 38 patients with erythropoietic protoporphyria, patients with higher baseline protoporphyrin and aspartate aminotransferase values showed a more progressive decrease during afamelanotide treatment.

    In plain wordsIn people with erythropoietic protoporphyria, those with higher starting values showed a larger drop in protoporphyrin and a liver test during afamelanotide treatment.

    Outcome
    DecreaseDecrease in protoporphyrin and aspartate aminotransferase by baseline value
    Study

    n = 38

    Comparator
    Pretreatment values
    Outcome group
    Porphyria markers and attacks · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    For both entities, patients with higher values showed a more progressive decrease, indicating a risk reduction of EPP-related liver disease.
    1 context sentence
    Summary · context
    From the 47 Swiss EPP-patients treated at our center since 2006, we included those 38 patients in the current analysis who received at least one afamelanotide dose between 2016 and 2018 and underwent regular laboratory testing before and during the treatment.
  4. 04HumanObservational studyEfficacy outcome

    In 38 patients with erythropoietic protoporphyria, the pre-existing hypochromia and broad red-cell distribution width were further augmented during afamelanotide treatment.

    In plain wordsIn people with erythropoietic protoporphyria, pale red cells and uneven red-cell size, already present, increased further during afamelanotide treatment.

    Outcome
    IncreaseHypochromia and red-cell distribution width
    Study

    n = 38

    Comparator
    Pretreatment values
    Outcome group
    Porphyria markers and attacks · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    The pre-existing hypochromia and broad mean red-cell distribution width were further augmented under afamelanotide.
    1 context sentence
    Summary · context
    From the 47 Swiss EPP-patients treated at our center since 2006, we included those 38 patients in the current analysis who received at least one afamelanotide dose between 2016 and 2018 and underwent regular laboratory testing before and during the treatment.
  5. 05HumanObservational studyEfficacy outcome

    In 38 patients with erythropoietic protoporphyria, mean zinc-protoporphyrin decreased significantly during afamelanotide treatment.

    In plain wordsIn people with erythropoietic protoporphyria, zinc-protoporphyrin fell during afamelanotide treatment.

    Outcome
    DecreaseZinc-protoporphyrin
    Study

    n = 38

    Comparator
    Pretreatment values
    Outcome group
    Porphyria markers and attacks · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    as mean zinc-protoporphyrin decreased significantly and ferritin remained unchanged.
    1 context sentence
    Summary · context
    From the 47 Swiss EPP-patients treated at our center since 2006, we included those 38 patients in the current analysis who received at least one afamelanotide dose between 2016 and 2018 and underwent regular laboratory testing before and during the treatment.
  6. 06HumanObservational studyEfficacy outcome

    In 38 patients with erythropoietic protoporphyria, ferritin remained unchanged during afamelanotide treatment.

    In plain wordsIn people with erythropoietic protoporphyria, ferritin stayed the same during afamelanotide treatment.

    Outcome
    No significant changeFerritin
    Study

    n = 38

    Comparator
    Pretreatment values
    Outcome group
    Porphyria markers and attacks · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    and ferritin remained unchanged.
    1 context sentence
    Summary · context
    From the 47 Swiss EPP-patients treated at our center since 2006, we included those 38 patients in the current analysis who received at least one afamelanotide dose between 2016 and 2018 and underwent regular laboratory testing before and during the treatment.
  7. 07HumanObservational studySafety outcome

    In 38 patients with erythropoietic protoporphyria, no serious afamelanotide-related adverse events were observed over 240 treatment years.

    In plain wordsIn people with erythropoietic protoporphyria, no serious side effects linked to afamelanotide were seen over 240 treatment years.

    Outcome
    No significant changeSerious adverse events
    Study

    n = 38

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    No serious afamelanotide-related adverse events were observed for a total of 240 treatment years.
    1 context sentence
    Summary · context
    From the 47 Swiss EPP-patients treated at our center since 2006, we included those 38 patients in the current analysis who received at least one afamelanotide dose between 2016 and 2018 and underwent regular laboratory testing before and during the treatment.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

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.

  1. Version 2Current

    13 Oct 2026, 21:44 UTC

    Study results updated

  2. Version 1

    13 Oct 2026, 16:31 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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