Evidence map›Paper›PMID 42694035›Full record

ArticlePatient related outcome measures2026

Assessing the effectiveness of the TURALIO (pexidartinib) risk evaluation and mitigation strategy program: patient knowledge, attitudes, and behavior survey (2020-2024).

Charles Dharmani, Oluwatosin Fofah, Margaret Wooddell, Janet L Ramsey, Michele Riggen

Abstract read
In one paragraph

Article in Patient related outcome measures, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Charles DharmaniEpidemiology, Clinical Safety and Pharmacovigilance, Daiichi Sankyo Inc, Basking Ridge, NJ, USA.ORCID https://orcid.org/0000-0003-3641-9738
Oluwatosin FofahEpidemiology, Clinical Safety and Pharmacovigilance, Daiichi Sankyo Inc, Basking Ridge, NJ, USA.ORCID https://orcid.org/0000-0003-2470-8276
Margaret WooddellGlobal Oncology Medical Affairs, Daiichi Sankyo Inc, Basking Ridge, NJ, USA.ORCID https://orcid.org/0009-0008-3657-6614
Janet L RamseyBiometrics, United BioSource LLC, King of Prussia, PA, USA.ORCID https://orcid.org/0009-0000-4848-0689
Michele RiggenProject Management Organization and KAB Programs, United BioSource LLC, King of Prussia, PA, USA.ORCID https://orcid.org/0009-0008-8159-4109

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Treatment of tenosynovial giant cell tumor with TURALIO (pexidartinib) is associated with serious hepatotoxicity risk. To ensure its safe use, a Risk Evaluation and Mitigation Strategy (REMS) program was implemented, incorporating structured education, mandatory enrollment, and liver function monitoring. The assessment of patient and caregiver knowledge, attitudes, and behaviors (KAB) is a central component of evaluating REMS effectiveness. Methods: Four waves of TURALIO Patient KAB Survey were conducted between 2020 and 2024 among REMS-enrollees. Surveys assessed understanding of two Key Risk Messages (KRMs): (KRM 1) hepatotoxicity risk and symptom recognition, and (KRM 2) liver monitoring requirements before and during treatment. Response rates and demonstrated understanding of KRMs were summarized descriptively. Results: Across four waves of this voluntary survey, 239 participants completed KAB surveys. Survey response rates ranged from approximately one-third to one-half of invited participants across waves. Correct response rates for individual knowledge items were consistently high. Demonstrated understanding of hepatotoxicity risk remained at ≥80% (the predefined threshold) across waves (87.5% in Wave 1; 80.0% in Wave 4). Understanding liver monitoring requirements improved over time, reaching 100% in Wave 3. Combined demonstrated understanding of KRM1 and KRM2 was <80% in three survey waves, particularly in Wave 2 (73.4%). Demonstrated understanding of KRMs remained generally stable despite evolving patient demographics and treatment duration over time. Conclusion: Across KAB Survey waves, patients receiving TURALIO demonstrated sustained knowledge of hepatotoxicity risk and monitoring requirements supporting the effectiveness of the tREMS program in ensuring the safe real-world use of pexidartinib.

Indexed as

hepatotoxicitykey risk message (KRM)knowledge attitudes and behavior (KAB)liver injurypexidartinibrisk evaluation and mitigation strategy (REMS)Tenosynovial giant cell tumor (TGCT)

Identifiers

PMID42694035
PMCPMC13536961

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.