Evidence map›Paper›PMID 42801439›Full record

ArticleInternational journal of hematology2026

Dabrafenib plus trametinib in BRAF V600E-mutant Erdheim-Chester disease: a case series including CNS involvement.

Yuya Kurihara, Akira Honda, Shuhei Matsumoto, Teruaki Yamaguchi, Hirofumi Takano, Mitsuhiro Yuasa, Takayuki Tsujimoto, Munetoshi Hinata, Mariko Tanaka, Mineo Kurokawa

Abstract read
PubMed Publisher
In one paragraph

Article in International journal of hematology, 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

10 authors.

Yuya KuriharaDepartment of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Akira HondaDepartment of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Shuhei MatsumotoDepartment of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Teruaki YamaguchiDepartment of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Hirofumi TakanoDepartment of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Mitsuhiro YuasaDepartment of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Takayuki TsujimotoDepartment of Pathology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Munetoshi HinataDepartment of Pathology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Mariko TanakaDepartment of Pathology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan.
Mineo KurokawaDepartment of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-Ku, Tokyo, 113-8655, Japan. kurokawa@m.u-tokyo.ac.jp.ORCID http://orcid.org/0000-0002-4034-2422

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundErdheim-Chester disease (ECD) is a rare clonal non-Langerhans cell histiocytosis characterized by the infiltration of foamy, CD68-positive/CD1a-negative histiocytes into multiple organ systems. Activating MAPK/ERK pathway mutations, most commonly BRAF V600E, represent common targets for ECD therapies. Although vemurafenib has been approved for BRAF V600E-mutant ECD, data regarding dual BRAF and MEK inhibition remain scarce.

methodsWe recently treated three patients with BRAF V600E-mutant ECD using dabrafenib combined with trametinib. The clinical presentations included skeletal, cardiovascular, retroperitoneal, and central nervous system (CNS) involvement.

resultsTwo patients achieved clinical and radiologic improvement, including resolution of CNS symptoms. Notably, BRAF V600E immunohistochemical staining was faint, equivocal, or negative in all patients, whereas molecular testing confirmed the mutation. One patient developed grade 3 rhabdomyolysis after one year, requiring dose reduction. The third patient maintained stable disease, with positron emission tomography demonstrating decreased fluorodeoxyglucose uptake that indicated early metabolic improvement.

conclusionThis case series highlights the effectiveness and tolerability of dabrafenib plus trametinib for BRAF V600E-mutant ECD, including cases with CNS and cardiovascular involvement. Molecular diagnosis and targeted therapy appear to be critical for optimizing outcomes, although further prospective studies are warranted to clarify the long-term effectiveness, resistance mechanisms, and optimal duration of this treatment approach.

Indexed as

BRAF V600E mutationCardiovascular diseasesCentral nervous system diseasesDabrafenibErdheim–Chester diseaseTrametinib

Identifiers

PMID42801439

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.