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Study · Case report or series · n = 1 · 2026

6 results

Trofinetide-Induced Enterocolitis Syndrome in a Child with Rett Syndrome.

Jones SG, Alberty J, Bell M

Journal of child neurology22 Dec 2025Cited by 1 (Europe PMC)

PMID41428727 (opens in a new tab)DOI10.1177/08830738251404099 (opens in a new tab)PepBankPUB-41428727
Publisher pagePubMedAsk about this study
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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: Publisher copyright. Results still quote short attributed sentences.

Results from this study6

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

  1. 01HumanCase report or seriesSafety outcome

    In a girl with Rett syndrome, emesis, pallor, and lethargy developed after 2 months of trofinetide treatment.

    In plain wordsIn a girl with Rett syndrome, vomiting, pallor, and lethargy developed after 2 months of trofinetide treatment.

    Peptide
    Trofinetide
    Outcome
    Not applicableEmesis, pallor and lethargy after 2 months of treatment
    Study

    n = 1

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated · 2 monthsAs given in this study (human).

    Source sentences

    Summary
    We report a case of a young girl with genetically confirmed Rett syndrome who developed emesis, pallor, and lethargy after 2 months of treatment with trofinetide.
  2. 02HumanCase report or seriesSafety outcome

    In a girl with Rett syndrome, pallor developed after 2 months of trofinetide treatment.

    In plain wordsIn a girl with Rett syndrome, pale skin developed after 2 months of trofinetide treatment.

    Peptide
    Trofinetide
    Outcome
    Not applicablePallor
    Study

    n = 1

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · 2 monthsAs given in this study (human).

    Source sentences

    Summary
    We report a case of a young girl with genetically confirmed Rett syndrome who developed emesis, pallor, and lethargy after 2 months of treatment with trofinetide.
  3. 03HumanCase report or seriesSafety outcome

    In a girl with Rett syndrome, lethargy developed after 2 months of trofinetide treatment.

    In plain wordsIn a girl with Rett syndrome, lethargy developed after 2 months of trofinetide treatment.

    Peptide
    Trofinetide
    Outcome
    Not applicableLethargy
    Study

    n = 1

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · 2 monthsAs given in this study (human).

    Source sentences

    Summary
    We report a case of a young girl with genetically confirmed Rett syndrome who developed emesis, pallor, and lethargy after 2 months of treatment with trofinetide.
  4. 04HumanCase report or seriesSafety outcome

    In a girl with Rett syndrome, adverse symptoms recurred when trofinetide restarted after a week without treatment.

    In plain wordsIn a girl with Rett syndrome, symptoms recurred after trofinetide restarted following a week without treatment.

    Peptide
    Trofinetide
    Outcome
    Not applicableAdverse symptoms after restart
    Study

    n = 1

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

    Source sentences

    Summary
    An attempt was made to restart the medication after being held for a week, but the patient again experienced adverse symptoms and the drug was held.
    1 context sentence
    Summary · context
    We report a case of a young girl with genetically confirmed Rett syndrome who developed emesis, pallor, and lethargy after 2 months of treatment with trofinetide.
  5. 05HumanCase report or seriesSafety outcome

    In a girl with Rett syndrome, symptoms recurred within hours of trofinetide rechallenge after 3 months without treatment, consistent with FPIES.

    In plain wordsIn a girl with Rett syndrome, symptoms consistent with enterocolitis recurred within hours of trofinetide rechallenge after 3 months without treatment.

    Peptide
    Trofinetide
    Outcome
    Not applicableEnterocolitis syndrome on rechallenge
    Study

    n = 1

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    After 3 months without the medication, she experienced recurrence of initial symptoms within hours of rechallenge, consistent with food protein-induced enterocolitis syndrome (FPIES).
    1 context sentence
    Summary · context
    We report a case of a young girl with genetically confirmed Rett syndrome who developed emesis, pallor, and lethargy after 2 months of treatment with trofinetide.
  6. 06HumanCase report or seriesSafety outcome

    In a girl with Rett syndrome, trofinetide was discontinued after rechallenge.

    In plain wordsIn a girl with Rett syndrome, trofinetide was discontinued after rechallenge.

    Peptide
    Trofinetide
    Outcome
    Not applicableTreatment discontinuation
    Study

    n = 1

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

    Source sentences

    Summary
    The medication was not continued after the re-challenge.
    1 context sentence
    Summary · context
    We report a case of a young girl with genetically confirmed Rett syndrome who developed emesis, pallor, and lethargy after 2 months of treatment with trofinetide.

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

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:37 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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