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Study · Observational study · n = 1,175 · 2026

11 results

Rett syndrome and real-world treatment patterns of trofinetide in the United States.

Rashid N, Yakkala VK, Syed SS, Rajagopalan K

Journal of medical economics13 Jun 2026Cited by 1 (Europe PMC)

PMID42287113 (opens in a new tab)DOI10.1080/13696998.2026.2682668 (opens in a new tab)PepBankPUB-42287113
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: CC BY-NC-ND. Results still quote short attributed sentences.

Results from this study11

11 results from studies that gave the peptide. 11 results from people.

  1. 01HumanObservational studyOther

    Among RTT individuals initiating trofinetide, 54.9% were persistent and 45.1% were non-persistent.

    In plain wordsAmong RTT individuals starting trofinetide, 54.9% were persistent and 45.1% were non-persistent.

    Peptide
    Trofinetide
    Outcome
    Not applicablePersistence
    Study

    n = 1,175

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

    Measurements

    Persistent
    54.9%
    reported result
    Non-persistent
    45.1%
    reported result

    Source sentences

    Summary
    Among 1,175 TROF initiators, 54.9% were persistent and 45.1% were non-persistent; 7.6% of non-persistent individuals restarted.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  2. 02HumanObservational studyOther

    Persistent RTT individuals had median trofinetide treatment time of 14.3 months versus 3.0 months in non-persistent individuals.

    In plain wordsPersistent RTT individuals used trofinetide for a median 14.3 months versus 3.0 months in non-persistent individuals.

    Peptide
    Trofinetide
    Outcome
    IncreaseTime on treatment
    Study

    n = 1,175

    Comparator
    Non-persistent individuals
    Outcome group
    Treatment adherence and retention · Pharmacology
    Studied dose
    Dose not stated

    Measurements

    Median time
    14.3 months
    persistent; IQR 4.6
    Median time
    3 months
    non-persistent; IQR 3.8

    Source sentences

    Summary
    Mean ± SD age was 14.7 ± 10.8 vs. 16.5 ± 11.6 years and median ± IQR time on TROF was 14.3 ± 4.6 vs. 3.0 ± 3.8 months, respectively.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  3. 03HumanObservational studyOther

    Over 75% of RTT trofinetide initiators remained on treatment beyond 3 months.

    In plain wordsOver 75% of RTT individuals starting trofinetide continued beyond 3 months.

    Peptide
    Trofinetide
    Outcome
    Not applicableTreatment continuation beyond three months
    Study

    n = 1,175

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

    Measurements

    Continuation
    > 75%
    beyond 3 months

    Source sentences

    Summary
    Over 75% remained on treatment beyond 3 months, and more than half continued through study follow-up.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  4. 04HumanObservational studyOther

    More than half of RTT trofinetide initiators continued through study follow-up.

    In plain wordsMore than half of RTT individuals starting trofinetide continued through study follow-up.

    Peptide
    Trofinetide
    Outcome
    Not applicableTreatment continuation through follow-up
    Study

    n = 1,175

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

    Source sentences

    Summary
    Over 75% remained on treatment beyond 3 months, and more than half continued through study follow-up.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  5. 05HumanObservational studyOther

    Persistent RTT individuals had lower mean twice-daily trofinetide doses at RX1, 7422.0 mg versus 7850.8 mg in non-persistent individuals.

    In plain wordsAt RX1, persistent RTT individuals received lower mean trofinetide doses than non-persistent individuals.

    Peptide
    Trofinetide
    Outcome
    DecreaseDose at first prescription
    Study

    n = 1,175

    Comparator
    Non-persistent individuals
    Outcome group
    Treatment adherence and retention · Pharmacology
    Studied dose
    Dose not stated

    Measurements

    Mean BID dose
    7422 mg
    persistent RX1
    Mean BID dose
    7850.8 mg
    non-persistent RX1

    Source sentences

    Summary
    Mean twice daily (BID) TROF dose was lower among persistent (7422.0 mg) vs. non-persistent (7850.8 mg) at RX1 and increased to 7953.7 mg vs 8423.4 mg, respectively at RX2.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  6. 06HumanObservational studyOther

    Mean twice-daily trofinetide doses increased to 7953.7 mg in persistent RTT individuals and 8423.4 mg in non-persistent individuals at RX2.

    In plain wordsAt RX2, mean trofinetide doses increased in both persistent and non-persistent RTT individuals.

    Peptide
    Trofinetide
    Outcome
    IncreaseDose at second prescription
    Study

    n = 1,175

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

    Measurements

    Mean BID dose
    7953.7 mg
    persistent RX2
    Mean BID dose
    8423.4 mg
    non-persistent RX2

    Source sentences

    Summary
    Mean twice daily (BID) TROF dose was lower among persistent (7422.0 mg) vs. non-persistent (7850.8 mg) at RX1 and increased to 7953.7 mg vs 8423.4 mg, respectively at RX2.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  7. 07HumanObservational studyOther

    Persistent RTT individuals maintained stable trofinetide doses after RX2, while non-persistent individuals had greater fluctuations.

    In plain wordsPersistent RTT individuals had more stable trofinetide doses than non-persistent individuals after RX2.

    Peptide
    Trofinetide
    Outcome
    DecreaseDose variability
    Study

    n = 1,175

    Comparator
    Non-persistent individuals
    Outcome group
    Treatment adherence and retention · Pharmacology
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Thereafter, the persistent individuals maintained stable dosing, while the non-persistent showed greater fluctuation over time.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  8. 08HumanObservational studyOther

    In RTT trofinetide users, percentage target daily dose followed similar persistent versus non-persistent patterns to absolute dose.

    In plain wordsIn RTT trofinetide users, target-dose percentages followed similar group patterns to absolute doses.

    Peptide
    Trofinetide
    Outcome
    Direction not reportedPercentage target daily dose patterns
    Study

    n = 1,175

    Comparator
    Non-persistent individuals
    Outcome group
    Treatment adherence and retention · Pharmacology
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Similar patterns were observed for %TDD.
    3 context sentences
    Summary · context
    Mean twice daily (BID) TROF dose was lower among persistent (7422.0 mg) vs. non-persistent (7850.8 mg) at RX1 and increased to 7953.7 mg vs 8423.4 mg, respectively at RX2. Thereafter, the persistent individuals maintained stable dosing, while the non-persistent showed greater fluctuation over time.
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  9. 09HumanObservational studyOther

    Among RTT trofinetide initiators, older age was significantly associated with non-persistence.

    In plain wordsOlder age was linked to non-persistence among RTT individuals starting trofinetide.

    Peptide
    Trofinetide
    Outcome
    Direction not reportedAge association with non-persistence
    Study

    n = 1,175

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

    Source sentences

    Summary
    Older age and history of infectious disorders were significantly associated with non-persistence.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  10. 10HumanObservational studyOther

    Among RTT trofinetide initiators, a history of infectious disorders was significantly associated with non-persistence.

    In plain wordsA history of infectious disorders was linked to non-persistence among RTT individuals starting trofinetide.

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

    n = 1,175

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

    Source sentences

    Summary
    Older age and history of infectious disorders were significantly associated with non-persistence.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.
  11. 11HumanObservational studyOther

    Among non-persistent RTT individuals, 7.6% restarted trofinetide.

    In plain wordsAmong non-persistent RTT individuals, 7.6% restarted trofinetide.

    Peptide
    Trofinetide
    Outcome
    Not applicableTreatment restart
    Study

    n: not reported in source

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

    Measurements

    Restart
    7.6%
    non-persistent individuals

    Source sentences

    Summary
    Among 1,175 TROF initiators, 54.9% were persistent and 45.1% were non-persistent; 7.6% of non-persistent individuals restarted.
    2 context sentences
    Summary · context
    A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed.
    Summary · context
    RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent.

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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.

  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

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