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Study · Observational study · n = 51 · 2026

4 results

Real-World Demographics, Clinical Characteristics, and Predictors of Persistence Among Males with Rett Syndrome Treated with Trofinetide.

Rajagopalan K, Yakkala V, Rashid N

Advances in therapy30 Jun 2026

PMID42377739 (opens in a new tab)PMCIDPMC13619775 (opens in a new tab)DOI10.1007/s12325-026-03686-1 (opens in a new tab)PepBankPUB-42377739
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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: CC BY-NC. Results still quote short attributed sentences.

Results from this study4

4 results from studies that gave the peptide. 4 results from people.

  1. 01HumanObservational studyOther

    Among treated males with RTT, 52.9% were persistent on trofinetide and 47.1% were non-persistent.

    In plain wordsAmong males with RTT receiving trofinetide, 52.9% were persistent and 47.1% were non-persistent.

    Peptide
    Trofinetide
    Outcome
    Not applicablePersistence
    Study

    n = 51

    Outcome group
    Treatment adherence and retention · Pharmacology
    Studied dose
    Dose not stated

    Measurements

    Persistent
    52.9%
    reported result
    Non-persistent
    47.1%
    reported result

    Source sentences

    Summary
    Among treated males, 52.9% were persistent and 47.1% were non-persistent.
    2 context sentences
    Summary · context
    This retrospective cohort study using IQVIA database linked to TROF specialty pharmacy claims from 01/01/2021 to 09/30/2024 identified males with ≥ 1 medical claim for RTT (ICD-10 F84.2).
    Summary · context
    Among treated males, Kaplan-Meier analysis assessed time to non-persistence, and logistic regression evaluated predictors of non-persistence.
  2. 02HumanObservational studyOther

    Approximately 70% of treated males with RTT remained on trofinetide for at least 6 months.

    In plain wordsApproximately 70% of treated males with RTT continued trofinetide for at least 6 months.

    Peptide
    Trofinetide
    Outcome
    Not applicableContinuation at six months
    Study

    n = 51

    Outcome group
    Treatment adherence and retention · Pharmacology
    Studied dose
    Dose not stated

    Measurements

    Continuation
    ≈ 70%
    at least 6 months

    Source sentences

    Summary
    Approximately 70% remained on TROF for ≥ 6 months and > 50% for ≥ 17 months.
    2 context sentences
    Summary · context
    This retrospective cohort study using IQVIA database linked to TROF specialty pharmacy claims from 01/01/2021 to 09/30/2024 identified males with ≥ 1 medical claim for RTT (ICD-10 F84.2).
    Summary · context
    Among treated males, Kaplan-Meier analysis assessed time to non-persistence, and logistic regression evaluated predictors of non-persistence.
  3. 03HumanObservational studyOther

    More than 50% of treated males with RTT remained on trofinetide for at least 17 months.

    In plain wordsMore than 50% of treated males with RTT continued trofinetide for at least 17 months.

    Peptide
    Trofinetide
    Outcome
    Not applicableContinuation at seventeen months
    Study

    n = 51

    Outcome group
    Treatment adherence and retention · Pharmacology
    Studied dose
    Dose not stated

    Measurements

    Continuation
    > 50%
    at least 17 months

    Source sentences

    Summary
    Approximately 70% remained on TROF for ≥ 6 months and > 50% for ≥ 17 months.
    2 context sentences
    Summary · context
    This retrospective cohort study using IQVIA database linked to TROF specialty pharmacy claims from 01/01/2021 to 09/30/2024 identified males with ≥ 1 medical claim for RTT (ICD-10 F84.2).
    Summary · context
    Among treated males, Kaplan-Meier analysis assessed time to non-persistence, and logistic regression evaluated predictors of non-persistence.
  4. 04HumanObservational studyOther

    Among trofinetide-treated males with RTT, pre-index neurologic disorders were significantly associated with non-persistence.

    In plain wordsPre-index neurologic disorders were linked to non-persistence among males with RTT receiving trofinetide.

    Peptide
    Trofinetide
    Outcome
    Direction not reportedNeurologic disorder association with non-persistence
    Study

    n = 51

    Outcome group
    Treatment adherence and retention · Pharmacology
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Pre-index neurologic disorders were significantly associated with non-persistence.
    2 context sentences
    Summary · context
    This retrospective cohort study using IQVIA database linked to TROF specialty pharmacy claims from 01/01/2021 to 09/30/2024 identified males with ≥ 1 medical claim for RTT (ICD-10 F84.2).
    Summary · context
    Among treated males, Kaplan-Meier analysis assessed time to non-persistence, and logistic regression evaluated predictors of non-persistence.

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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