Real-world benefits and tolerability of trofinetide for the treatment of Rett syndrome: The LOTUS study.

Cosand L, Mayman H, Downs J, Abler V

Developmental medicine and child neurology11 Sept 2025Cited by 12 (Europe PMC)

PMID40936177PMCIDPMC12875184DOI10.1111/dmcn.16482PepBankPUB-40936177

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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 study10

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

01HumanObservational studyOther
In routine Rett syndrome care, median trofinetide dose increased from 36.0% of target at week 1 to over 80% from week 10.
In plain wordsIn Rett syndrome care, median trofinetide dose increased from 36.0% of target at week 1 to over 80% from week 10.
Peptide
Trofinetide
Outcome
IncreaseDose escalation
Study
n: not reported in source
Outcome group
Treatment Exposure · Pharmacology
Studied dose
Dose not stated
Measurements
Median dose
36 % of target
week 1
Median dose
> 80 % of target
week 10 onwards
Source sentences
Summary
The median trofinetide dose at week 1 was 36.0% of the target dose and increased to over 80% by week 10 onwards; 55.2% of patients took the full trofinetide recommended daily dose.
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617701
02HumanObservational studyOther
In routine Rett syndrome care, 55.2% of patients took the full recommended daily trofinetide dose.
In plain wordsIn Rett syndrome care, 55.2% of patients took the full recommended daily trofinetide dose.
Peptide
Trofinetide
Outcome
Not applicableFull recommended dose use
Study
n: not reported in source
Outcome group
Treatment Exposure · Pharmacology
Studied dose
Dose not stated
Measurements
Full recommended dose use
55.2%
patients
Source sentences
Summary
The median trofinetide dose at week 1 was 36.0% of the target dose and increased to over 80% by week 10 onwards; 55.2% of patients took the full trofinetide recommended daily dose.
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617702
03HumanObservational studyEfficacy outcome
In Rett syndrome care, caregivers reported behavioral improvement during trofinetide treatment in 71%-90% across months 1 to 12.
In plain wordsIn Rett syndrome care, 71%-90% of caregivers reported behavioral improvement during trofinetide treatment across months 1 to 12.
Peptide
Trofinetide
Outcome
IncreaseBehavioral improvement
Study
n: not reported in source
Outcome group
Locomotor and behavioral activity · Nervous system and cognition
Studied dose
Dose not stated
Measurements
Behavioral improvement
71 to 90%
months 1 to 12
Source sentences
Summary
Caregivers (71%-90%) reported behavioral improvements across months 1 to 12 with the BIQ, including non-verbal communication (48%-71%), alertness (44%-70%), and social interaction and connectedness (33%-58%).
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617703
04HumanObservational studyEfficacy outcome
In Rett syndrome care, caregivers reported non-verbal communication improvement during trofinetide treatment in 48%-71% across months 1 to 12.
In plain wordsIn Rett syndrome care, caregivers reported better non-verbal communication during trofinetide treatment in 48%-71% across months 1 to 12.
Peptide
Trofinetide
Outcome
IncreaseNon-verbal communication
Study
n: not reported in source
Outcome group
Locomotor and behavioral activity · Nervous system and cognition
Studied dose
Dose not stated
Measurements
Non-verbal communication improvement
48 to 71%
months 1 to 12
Source sentences
Summary
Caregivers (71%-90%) reported behavioral improvements across months 1 to 12 with the BIQ, including non-verbal communication (48%-71%), alertness (44%-70%), and social interaction and connectedness (33%-58%).
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617704
05HumanObservational studyEfficacy outcome
In Rett syndrome care, caregivers reported alertness improvement during trofinetide treatment in 44%-70% across months 1 to 12.
In plain wordsIn Rett syndrome care, caregivers reported better alertness during trofinetide treatment in 44%-70% across months 1 to 12.
Peptide
Trofinetide
Outcome
IncreaseAlertness
Study
n: not reported in source
Outcome group
Locomotor and behavioral activity · Nervous system and cognition
Studied dose
Dose not stated
Measurements
Alertness improvement
44 to 70%
months 1 to 12
Source sentences
Summary
Caregivers (71%-90%) reported behavioral improvements across months 1 to 12 with the BIQ, including non-verbal communication (48%-71%), alertness (44%-70%), and social interaction and connectedness (33%-58%).
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617705
06HumanObservational studyEfficacy outcome
In Rett syndrome care, caregivers reported social interaction and connectedness improvement during trofinetide treatment in 33%-58% across months 1 to 12.
In plain wordsIn Rett syndrome care, caregivers reported better social interaction during trofinetide treatment in 33%-58% across months 1 to 12.
Peptide
Trofinetide
Outcome
IncreaseSocial interaction and connectedness
Study
n: not reported in source
Outcome group
Locomotor and behavioral activity · Nervous system and cognition
Studied dose
Dose not stated
Measurements
Social improvement
33 to 58%
months 1 to 12
Source sentences
Summary
Caregivers (71%-90%) reported behavioral improvements across months 1 to 12 with the BIQ, including non-verbal communication (48%-71%), alertness (44%-70%), and social interaction and connectedness (33%-58%).
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617706
07HumanObservational studyEfficacy outcome
In Rett syndrome care, median QI-Disability score change during trofinetide treatment ranged from 4.7 to 4.6 across months 1 to 12.
In plain wordsIn Rett syndrome care, median quality-of-life score change during trofinetide treatment ranged from 4.7 to 4.6 across months 1 to 12.
Peptide
Trofinetide
Outcome
IncreaseQI-Disability total score
Study
n: not reported in source
Comparator
baseline
Outcome group
Quality of life · Nervous system and cognition
Studied dose
Dose not stated
Measurements
QI-Disability score change
4.7 to 4.6 points
months 1 to 12
Source sentences
Summary
The median QI-Disability total score change from baseline ranged from 4.7 to 4.6 in months 1 to 12.
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617707
08HumanObservational studySafety outcome
In Rett syndrome care, diarrhea incidence during trofinetide treatment was 23%-50% across weeks 1 to 12.
In plain wordsIn Rett syndrome care, diarrhea incidence during trofinetide treatment was 23%-50% across weeks 1 to 12.
Peptide
Trofinetide
Outcome
Not applicableDiarrhea during weeks 1 to 12
Study
n: not reported in source
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated
Measurements
Diarrhea incidence
23 to 50%
weeks 1 to 12
Source sentences
Summary
The incidence of diarrhea varied from weeks 1 to 12 (23%-50%) and months 4 to 12 (26%-38%); most reports of diarrhea were contained inside the patient's diaper.
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617708
09HumanObservational studySafety outcome
In Rett syndrome care, diarrhea incidence during trofinetide treatment was 26%-38% across months 4 to 12.
In plain wordsIn Rett syndrome care, diarrhea incidence during trofinetide treatment was 26%-38% across months 4 to 12.
Peptide
Trofinetide
Outcome
Not applicableDiarrhea during months 4 to 12
Study
n: not reported in source
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated
Measurements
Diarrhea incidence
26 to 38%
months 4 to 12
Source sentences
Summary
The incidence of diarrhea varied from weeks 1 to 12 (23%-50%) and months 4 to 12 (26%-38%); most reports of diarrhea were contained inside the patient's diaper.
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617709
10HumanObservational studySafety outcome
In Rett syndrome care, most reported diarrhea during trofinetide treatment remained inside the patient's diaper.
In plain wordsIn Rett syndrome care, most reported diarrhea during trofinetide treatment remained inside the patient's diaper.
Peptide
Trofinetide
Outcome
Not applicableDiarrhea containment
Study
n: not reported in source
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated
Source sentences
Summary
The incidence of diarrhea varied from weeks 1 to 12 (23%-50%) and months 4 to 12 (26%-38%); most reports of diarrhea were contained inside the patient's diaper.
2 context sentences(design, population, supporting numbers)
Summary · context
Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate.
Summary · context
Data are reported to 12 months since the initiation of trofinetide.
Report a problemFND-4093617710

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

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