Trofinetide Use and Treatment Patterns Among Children Aged 2-4 Years with Rett Syndrome in the United States: A Retrospective Specialty Pharmacy Linked Claims Database Analysis.

Rashid N, Yakkala VK, Doshi D, Rajagopalan K

International journal of general medicine29 Jun 2026

PMID42404293PMCIDPMC13332313DOI10.2147/ijgm.s627959PepBankPUB-42404293

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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 study10

10 results from studies that gave the peptide. 10 results from people.

01HumanObservational studyOther
Among children aged 2-4 years with RTT starting trofinetide, 65.4% were persistent and 34.6% were non-persistent.
In plain wordsAmong children aged 2-4 years with RTT starting trofinetide, 65.4% were persistent and 34.6% were non-persistent.
Peptide
Trofinetide
Outcome
Not applicablePersistence
Study
n = 159
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Measurements
Persistent
65.4%
reported result
Non-persistent
34.6%
reported result
Source sentences
Summary
Among 159 individuals, 65.4% were persistent and 34.6% were non-persistent; 14.5% of non-persistent individuals restarted TROF.
2 context sentences(design, population, supporting numbers)
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429301
02HumanObservational studyOther
Non-persistent young RTT trofinetide users had higher baseline dysphagia rates than persistent users, 34.5% versus 16.3%.
In plain wordsNon-persistent young RTT trofinetide users had more baseline dysphagia than persistent users.
Peptide
Trofinetide
Outcome
IncreaseDysphagia association with non-persistence
Study
n = 159
Comparator
Persistent children
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Measurements
Dysphagia
34.5%
non-persistent
Dysphagia
16.3%
persistent
Source sentences
Summary
Non-persistent individuals had higher rates of dysphagia (34.5% vs 16.3%), gastrostomy (20.0% vs 3.8%), and breathing irregularities (16.4% vs 1.9%).
3 context sentences(design, population, supporting numbers)
Summary · context
Non-persistent children had significantly higher baseline rates of dysphagia, gastrostomy, and breathing irregularities, suggesting that early disease-related multisystem complications may be associated with reduced treatment continuity.
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429303
03HumanObservational studyOther
Non-persistent young RTT trofinetide users had higher baseline gastrostomy rates than persistent users, 20.0% versus 3.8%.
In plain wordsNon-persistent young RTT trofinetide users had more baseline gastrostomy than persistent users.
Peptide
Trofinetide
Outcome
IncreaseGastrostomy association with non-persistence
Study
n = 159
Comparator
Persistent children
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Measurements
Gastrostomy
20%
non-persistent
Gastrostomy
3.8%
persistent
Source sentences
Summary
Non-persistent individuals had higher rates of dysphagia (34.5% vs 16.3%), gastrostomy (20.0% vs 3.8%), and breathing irregularities (16.4% vs 1.9%).
3 context sentences(design, population, supporting numbers)
Summary · context
Non-persistent children had significantly higher baseline rates of dysphagia, gastrostomy, and breathing irregularities, suggesting that early disease-related multisystem complications may be associated with reduced treatment continuity.
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429304
04HumanObservational studyOther
Non-persistent young RTT trofinetide users had higher baseline breathing irregularity rates than persistent users, 16.4% versus 1.9%.
In plain wordsNon-persistent young RTT trofinetide users had more baseline breathing irregularities than persistent users.
Peptide
Trofinetide
Outcome
IncreaseBreathing irregularity association with non-persistence
Study
n = 159
Comparator
Persistent children
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Measurements
Breathing irregularities
16.4%
non-persistent
Breathing irregularities
1.9%
persistent
Source sentences
Summary
Non-persistent individuals had higher rates of dysphagia (34.5% vs 16.3%), gastrostomy (20.0% vs 3.8%), and breathing irregularities (16.4% vs 1.9%).
3 context sentences(design, population, supporting numbers)
Summary · context
Non-persistent children had significantly higher baseline rates of dysphagia, gastrostomy, and breathing irregularities, suggesting that early disease-related multisystem complications may be associated with reduced treatment continuity.
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429305
05HumanObservational studyOther
Persistent and non-persistent children aged 2-4 years with RTT had similar mean twice-daily trofinetide doses across shipments.
In plain wordsPersistent and non-persistent young children with RTT received similar mean twice-daily trofinetide doses across shipments.
Peptide
Trofinetide
Outcome
Direction not reportedMean twice-daily dose
Study
n = 159
Comparator
Non-persistent children
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Source sentences
Summary
Mean BID dose and %TDD were similar between groups across shipments.
2 context sentences(design, population, supporting numbers)
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429306
06HumanObservational studyOther
Persistent and non-persistent children aged 2-4 years with RTT had similar trofinetide target daily dose percentages across shipments.
In plain wordsPersistent and non-persistent young children with RTT received similar trofinetide target-dose percentages across shipments.
Peptide
Trofinetide
Outcome
Direction not reportedPercentage target daily dose
Study
n = 159
Comparator
Non-persistent children
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Source sentences
Summary
Mean BID dose and %TDD were similar between groups across shipments.
2 context sentences(design, population, supporting numbers)
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429307
07HumanObservational studyOther
Persistent young RTT trofinetide users had median treatment time of 13.3 months versus 4.4 months in non-persistent users.
In plain wordsPersistent young RTT trofinetide users had longer median treatment time than non-persistent users.
Peptide
Trofinetide
Outcome
IncreaseTime on treatment
Study
n = 159
Comparator
Non-persistent children
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Measurements
Median time
13.3 months
persistent; IQR 6.1-17.2
Median time
4.4 months
non-persistent; IQR 0.8-12.6
Source sentences
Summary
Median (IQR) time on treatment was 13.3 (6.1-17.2) months in the persistent group vs 4.4 (0.8-12.6) months in the non-persistent group.
2 context sentences(design, population, supporting numbers)
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429308
08HumanObservational studyOther
More than 87.5% of young children with RTT remained on trofinetide beyond 3 months.
In plain wordsMore than 87.5% of young children with RTT continued trofinetide beyond 3 months.
Peptide
Trofinetide
Outcome
Not applicableContinuation beyond three months
Study
n = 159
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Measurements
Continuation
> 87.5%
beyond 3 months
Source sentences
Summary
Kaplan-Meier analysis showed >87.5% remained on TROF beyond 3 months and >65.0% remained on TROF through end of available follow-up.
2 context sentences(design, population, supporting numbers)
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429309
09HumanObservational studyOther
More than 65.0% of young children with RTT remained on trofinetide through available follow-up.
In plain wordsMore than 65.0% of young children with RTT continued trofinetide through available follow-up.
Peptide
Trofinetide
Outcome
Not applicableContinuation through follow-up
Study
n = 159
Outcome group
Treatment adherence and retention · Pharmacology
Studied dose
Dose not stated
Measurements
Continuation
> 65%
end of available follow-up
Source sentences
Summary
Kaplan-Meier analysis showed >87.5% remained on TROF beyond 3 months and >65.0% remained on TROF through end of available follow-up.
2 context sentences(design, population, supporting numbers)
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429310
10HumanObservational studyOther
Among non-persistent children aged 2-4 years with RTT, 14.5% restarted trofinetide.
In plain wordsAmong non-persistent children aged 2-4 years with RTT, 14.5% 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
14.5%
non-persistent children
Source sentences
Summary
Among 159 individuals, 65.4% were persistent and 34.6% were non-persistent; 14.5% of non-persistent individuals restarted TROF.
2 context sentences(design, population, supporting numbers)
Summary · context
A retrospective cohort study was conducted using linked medical and pharmacy claims from 01/01/2021-09/30/2024 (study period).
Summary · context
Individuals aged 2-4 years with RTT who initiated TROF during 04/01/2023-03/31/2024 (identification period) were included (index date=first TROF RX).
Report a problemFND-4240429302

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.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
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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Keywords

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