Evidence map›Paper›PMID 41959727›Full record

ArticleHemaSphere2026

Erdheim-Chester disease associated with myeloid neoplasm: Clinical features, molecular landscape, and treatment outcomes.

Ambroise Le Pogam, Matthias Papo, Pierre Hirsch, Fleur Cohen-Aubart, Jean-François Emile, François Delhommeau, Pierre Duffau, Jérôme Razanamahery, Florence Nguyen-Khac, Christopher Nunes Gomes and 13 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in HemaSphere, 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

23 authors.

Ambroise Le PogamInternal Medicine Department, Saint-Antoine Hospital Assistance Publique-Hôpitaux de Paris Sorbonne University Paris France.ORCID https://orcid.org/0000-0002-7452-4304
Matthias PapoInternal Medicine Department 2, French National Referral Center for Histiocytoses, Sorbonne University, Assistance Publique-Hôpitaux de Paris Pitié-Salpêtrière Hospital Paris France.
Pierre HirschINSERM, Saint-Antoine Research center, CRSA, AP-HP, SIRIC CURAMUS, Saint-Antoine Hospital, Department of biological hematology Sorbonne University Paris France.
Fleur Cohen-AubartInternal Medicine Department 2, French National Referral Center for Histiocytoses, Sorbonne University, Assistance Publique-Hôpitaux de Paris Pitié-Salpêtrière Hospital Paris France.
Jean-François EmileAmbroise-paré Hospital APHP Boulogne France.
François DelhommeauINSERM, Saint-Antoine Research center, CRSA, AP-HP, SIRIC CURAMUS, Saint-Antoine Hospital, Department of biological hematology Sorbonne University Paris France.ORCID https://orcid.org/0000-0002-4915-0734
Pierre DuffauBordeaux University Hospital Bordeaux France.
Jérôme RazanamaheryDepartment of Internal Medicine and Clinical Immunology Dijon University Hospital Dijon France.
Florence Nguyen-KhacCentre de Recherche des Cordeliers, Sorbonne University, Paris Cité University, Inserm UMRS 1138 Drug Resistance in Hematological Malignancies Team Paris F-75006 France.
Christopher Nunes GomesAngers University Hospital Angers France.
Felicien TribouletInternal Medicine Department 2, French National Referral Center for Histiocytoses, Sorbonne University, Assistance Publique-Hôpitaux de Paris Pitié-Salpêtrière Hospital Paris France.
Jeanne de La RochefoucauldInternal Medicine Department 2, French National Referral Center for Histiocytoses, Sorbonne University, Assistance Publique-Hôpitaux de Paris Pitié-Salpêtrière Hospital Paris France.
Chloé McAvoyInternal Medicine Department, Saint-Antoine Hospital Assistance Publique-Hôpitaux de Paris Sorbonne University Paris France.
Estibaliz LazaroBordeaux University Hospital Bordeaux France.
Mehdi Hage-SleimanINSERM, Saint-Antoine Research center, CRSA, AP-HP, SIRIC CURAMUS, Saint-Antoine Hospital, Department of biological hematology Sorbonne University Paris France.
Damien Roos-WeilClinical Hematology Department, Pitié-Salpêtrière Hospital Sorbonne University Paris France.
François ChassetDermatology et Allergology department, Sorbonne University, Tenon Hospital Assistance Publique-Hôpitaux de Paris Paris CIMI U1135 France.
Vincent JachietInternal Medicine Department, Saint-Antoine Hospital Assistance Publique-Hôpitaux de Paris Sorbonne University Paris France.
Arsène MekinianInternal Medicine Department, Saint-Antoine Hospital Assistance Publique-Hôpitaux de Paris Sorbonne University Paris France.
Olivier FainInternal Medicine Department, Saint-Antoine Hospital Assistance Publique-Hôpitaux de Paris Sorbonne University Paris France.
Zahir AmouraInternal Medicine Department 2, French National Referral Center for Histiocytoses, Sorbonne University, Assistance Publique-Hôpitaux de Paris Pitié-Salpêtrière Hospital Paris France.
Jérôme HadjadjInternal Medicine Department, Saint-Antoine Hospital Assistance Publique-Hôpitaux de Paris Sorbonne University Paris France.
Julien HarocheInternal Medicine Department 2, French National Referral Center for Histiocytoses, Sorbonne University, Assistance Publique-Hôpitaux de Paris Pitié-Salpêtrière Hospital Paris France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Erdheim-Chester disease (ECD) is frequently associated with clonal hematopoiesis and myeloid neoplasms (MN), but clinical phenotype and response to kinase inhibitors (KI) in this setting remain unclear. We analyzed 67 patients with ECD associated with MN (ECD-MN) from a French national cohort and assessed ECD treatment response, MN progression, and leukemic transformation. Outcomes were compared with those of 348 patients with ECD without MN. ECD-MN were characterized by low blast counts and favorable MN prognostic scores. Compared with ECD patients without MN, those with MN were older (median 65 vs. 59 years, P < 0.0001) and had more frequent cardiac (54% vs. 37%, P < 0.01), pulmonary (48% vs. 31%, P < 0.01), gastrointestinal (34% vs. 11%, P < 0.0001), and lymph node involvement (31% vs. 8%, P < 0.0001). KI therapy led to higher ECD response rates at 6 and 12 months compared with pegylated-interferon α (Peg-IFN) (96% vs. 58%, P = 0.02). MN progression and leukemic transformation at 6 months occurred, respectively, in 25% and 3% of KI-treated patients versus 25% and 18% of those treated with Peg-IFN. After a median follow-up of 25 (12-38) months on KI, the mutational landscape remained unchanged. Median overall survival was shorter in patients with MN (76 vs. 163 months, P < 0.001). In conclusion, ECD associated with MN represents a distinct clinical entity, marked by broader organ involvement and poorer prognosis. KI therapy provides a superior response without increasing the risk of MN progression or leukemic transformation, supporting its use as a frontline treatment in this population.

Identifiers

PMID41959727
PMCPMC13060758

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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.