Evidence map›Paper›PMID 40053897›Full record

ArticleBlood2025

Long-term outcomes with single-agent BRAF inhibitor therapy in Erdheim-Chester disease.

Gaurav Goyal, Anne S Reiner, Dana Bossert, Allison M Sigler, Mario E Lacouture, Veronica Rotemberg, Jasmine H Francis, Ronald S Go, Raajit K Rampal, Eli L Diamond

Abstract read
In one paragraph

Article in Blood, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Review
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

10 authors.

Gaurav GoyalDivision of Hematology and Oncology, Department of Medicine, The University of Alabama at Birmingham, Birmingham, AL.ORCID 0000-0001-6148-5177
Anne S ReinerDepartment of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY.ORCID 0000-0002-1258-6112
Dana BossertDepartment of Neurology, Memorial Sloan Kettering Cancer Center, New York, NY.
Allison M SiglerDepartment of Neurology, Memorial Sloan Kettering Cancer Center, New York, NY.
Mario E LacoutureDermatology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.
Veronica RotembergDermatology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.
Jasmine H FrancisOphthalmic Oncology Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY.ORCID 0000-0002-7637-3108
Ronald S GoDivision of Hematology, Mayo Clinic, Rochester, MN.
Raajit K RampalLeukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.
Eli L DiamondDepartment of Neurology, Memorial Sloan Kettering Cancer Center, New York, NY.ORCID 0000-0001-5456-5961

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4M
Molecular Pathogenesis and Therapy of Systemic Histiocytic NeoplasmsR37CA259260 · NCI · SLOAN-KETTERING INST CAN RESEARCH · PI Eli Lous Diamond · 2021 to 2026
$3.3M
NCI NIH HHS P30 CA008748NCI NIH HHS R37 CA259260
6 · The paper itself

Abstract

abstractAmong 64 patients with Erdheim-Chester disease treated with a BRAF inhibitor (median follow-up 4 years), we found high response rates (85%) but frequent discontinuations (61%), primarily because of adverse events. Additionally, patients experienced poor health-related quality of life and high symptom burden.

Indexed as

Erdheim-Chester DiseaseProtein Kinase InhibitorsProto-Oncogene Proteins B-rafAdultAgedFemaleFollow-Up StudiesHumansMaleMiddle AgedQuality of LifeTreatment OutcomeVemurafenibBRAF protein, humanProtein Kinase InhibitorsProto-Oncogene Proteins B-rafVemurafenib

Identifiers

PMID40053897
PMCPMC12105718

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.