Long-term outcome and prognosis of mixed histiocytosis (Erdheim-Chester disease and Langerhans Cell Histiocytosis).

BRAF Erdheim-Chester disease Histiocytosis Langerhans Mixed histiocytosis Pediatric histiocytosis

Journal

EClinicalMedicine
ISSN: 2589-5370
Titre abrégé: EClinicalMedicine
Pays: England
ID NLM: 101733727

Informations de publication

Date de publication:
Jul 2024
Historique:
received: 27 02 2024
revised: 03 05 2024
accepted: 07 05 2024
medline: 6 6 2024
pubmed: 6 6 2024
entrez: 6 6 2024
Statut: epublish

Résumé

Erdheim-Chester disease (ECD) is a rare histiocytosis that may overlap with Langerhans Cell Histiocytosis (LCH). This "mixed" entity is poorly characterized. We here investigated the clinical phenotype, outcome, and prognostic factors of a large cohort of patients with mixed ECD-LCH. This retrospective study was performed at two referral centers in France and Italy (Pitié-Salpêtrière Hospital, Paris; Meyer Children's Hospital, Florence). We included children and adults with ECD diagnosed in 2000-2022 who had biopsy-proven LCH, available data on clinical presentation, treatment and outcome, and a minimum follow-up of one year. Outcomes included differences in clinical presentation and survival between mixed ECD-LCH and isolated ECD; we also investigated response to treatments and predictors of survival in the mixed cohort. Survival was analyzed using the Kaplan-Maier method and differences in survival with the long-rank test. Cox regression models were used to evaluate the potential impact of age and gender on survival and to identify predictors of non-response and survival. Out of a cohort of 502 ECD patients, 69 (14%) had mixed ECD-LCH. Compared to isolated ECD, mixed ECD-LCH occurred more frequently in females (51 Mixed ECD-LCH is a multisystem disease driven by the None.

Sections du résumé

Background UNASSIGNED
Erdheim-Chester disease (ECD) is a rare histiocytosis that may overlap with Langerhans Cell Histiocytosis (LCH). This "mixed" entity is poorly characterized. We here investigated the clinical phenotype, outcome, and prognostic factors of a large cohort of patients with mixed ECD-LCH.
Methods UNASSIGNED
This retrospective study was performed at two referral centers in France and Italy (Pitié-Salpêtrière Hospital, Paris; Meyer Children's Hospital, Florence). We included children and adults with ECD diagnosed in 2000-2022 who had biopsy-proven LCH, available data on clinical presentation, treatment and outcome, and a minimum follow-up of one year. Outcomes included differences in clinical presentation and survival between mixed ECD-LCH and isolated ECD; we also investigated response to treatments and predictors of survival in the mixed cohort. Survival was analyzed using the Kaplan-Maier method and differences in survival with the long-rank test. Cox regression models were used to evaluate the potential impact of age and gender on survival and to identify predictors of non-response and survival.
Findings UNASSIGNED
Out of a cohort of 502 ECD patients, 69 (14%) had mixed ECD-LCH. Compared to isolated ECD, mixed ECD-LCH occurred more frequently in females (51
Interpretation UNASSIGNED
Mixed ECD-LCH is a multisystem disease driven by the
Funding UNASSIGNED
None.

Identifiants

DOI: 10.1016/j.eclinm.2024.102658 PMID: 38841707 PMC: PMC11152896
pubmed: 38841707
doi: 10.1016/j.eclinm.2024.102658
pii: S2589-5370(24)00237-2
pmc: PMC11152896
doi:

Types de publication

Journal Article

Langues

eng

Pagination

102658

Informations de copyright

© 2024 The Author(s).

Déclaration de conflit d'intérêts

AI reports research grants from Transgene, Sanofi, and Nutritheragene; consulting fees from Novocure, LeoPharma, Polytone Laser, and Novartis; honoraria from Novocure and Neurologies; travel funding from LeoPharma, Novocure, and Carthera. BT report consulting fees and honoraria from GSK, AstraZeneca, CSL Vifor, Boehringer Ingelheim, and Novartis; advisory board activity for Amgen.

Auteurs

Francesco Pegoraro (F)

Sorbonne University, Internal Medicine Department 2, Institut E3M, French Reference Centre for Histiocytosis, Pitié-Salpȇtrière Hospital, CIMI INSERM-UMRS 1135, Assistance Publique-Hôpitaux de Paris, Paris, France.
Hematology and Oncology Department, Meyer Children's Hospital IRCCS, Florence, Italy.
Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.

Matthias Papo (M)

Sorbonne University, Internal Medicine Department 2, Institut E3M, French Reference Centre for Histiocytosis, Pitié-Salpȇtrière Hospital, CIMI INSERM-UMRS 1135, Assistance Publique-Hôpitaux de Paris, Paris, France.

Fleur Cohen-Aubart (F)

Sorbonne University, Internal Medicine Department 2, Institut E3M, French Reference Centre for Histiocytosis, Pitié-Salpȇtrière Hospital, CIMI INSERM-UMRS 1135, Assistance Publique-Hôpitaux de Paris, Paris, France.

Francesco Peyronel (F)

Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.
Nephrology and Dialysis Unit, Meyer Children's Hospital IRCCS, Florence, Italy.

Gianmarco Lugli (G)

Rare Disease Center, Meyer Children's Hospital IRCCS, Florence, Italy.

Irene Trambusti (I)

Hematology and Oncology Department, Meyer Children's Hospital IRCCS, Florence, Italy.
Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.

Gildas Baulier (G)

Internal Medicine and Clinical Immunology Department, Périgueux University Hospital, Périgueux, France.

Mathilde de Menthon (M)

Paris-Saclay University, Internal Medicine and Clinical Immunology Department, Bicetre Hospital, Assistance Publique - Hôpitaux de Paris, Le Kremlin Bicêtre, France.

Tanguy Le Scornet (T)

Internal Medicine Department, Hotel Dieu, Nantes University Hospital, Nantes, France.

Eric Oziol (E)

Internal Medicine Department, Béziers Hospital, Béziers, France.

Nicole Ferreira-Maldent (N)

Internal Medicine Department, University Hospital of Tours, Tours, France.

Olivier Hermine (O)

Hematology Department, Hôpital Necker - Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.

Benoit Faucher (B)

Internal Medicine Department, La Timone Hospital, Assistance Publique-Hôpitaux de Marseille, Marseille, France.

Dirk Koschel (D)

Internal Medicine and Pneumology Department, Fachkrankenhaus Coswig, Lung Center, Coswig and Medical Department I, Division of Pneumology, University Hospital Carl Gustav Carus, TU Dresden, Dresden, Germany.

Nicole Straetmans (N)

Hematology Department, University Hospital Saint-Luc, Brussels, Belgium.

Noémie Abisror (N)

Internal Medicine Department, Saint Antoine Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.

Benjamin Terrier (B)

Internal Medicine Department, Referral Center for Rare Autoimmune and Systemic Diseases, Cochin Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.

François Lifermann (F)

Internal Medicine Department, Dax University Hospital, Dax, France.

Jerome Razanamahery (J)

Internal Medicine and Clinical Immunology Department, Dijon University Hospital, Dijon, France.

Yves Allenbach (Y)

Internal Medicine Department, Sorbonne University, INSERM UMRS 974, Pitié-Salpêtrière Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.

Jeremy Keraen (J)

Internal Medicine and Immunology Department, Cornouaille Hospital Center, Quimper, France.

Sophie Bulifon (S)

Paris-Saclay University, Faculty of Medicine, Le Kremlin-Bicêtre, France.
INSERM UMR_S 999, Le Kremlin-Bicêtre, France.
Respiratory and Intensive Care Medicine Department, Pulmonary Hypertension National Referral Center, Bicêtre Hospital, Assistance Publique-Hôpitaux de Paris, Le Kremlin-Bicêtre, France.

Baptiste Hervier (B)

Internal Medicine Department, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.

Annamaria Buccoliero (A)

Pathology Department, Meyer Children's Hospital IRCCS, Florence, Italy.

Frederic Charlotte (F)

Pathology Department, Pitié-Salpêtrière Hospital, Sorbonne University, Assistance Publique-Hôpitaux de Paris, Paris, France.

Quentin Monzani (Q)

Polyvalent and Oncologic Radiology Department-Musculoskeletal Unit, Pitié-Salpêtrière Hospital, Paris, France.

Samia Boussouar (S)

Cardiovascular and Thoracic Imaging Unit, Pitié-Salpêtrière Hospital, Assistance Publique-Hôpitaux de Paris, Institute of Cardiometabolism and Nutrition (ICAN), Paris, France.

Natalia Shor (N)

Neuro-Radiology Department, Pitié-Salpêtrière Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.

Annalisa Tondo (A)

Hematology and Oncology Department, Meyer Children's Hospital IRCCS, Florence, Italy.

Stephane Barete (S)

Sorbonne University, Dermatology Unit, Pitié-Salpêtrière Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.

Ahmed Idbaih (A)

Sorbonne University, Neuro-Oncology Department, Paris Brain Institute - ICM, Inserm, CNRS, Pitié-Salpêtrière Hospital, DMU Neurosciences, Assistance Publique-Hôpitaux de Paris, Paris, France.

Abdellatif Tazi (A)

UFR de Médecine, Paris Cité University, Paris, France.
INSERM UMR 976 Saint Louis Research Institute, Paris, France.
Pneumology Department, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.

Elena Sieni (E)

Hematology and Oncology Department, Meyer Children's Hospital IRCCS, Florence, Italy.

Zahir Amoura (Z)

Sorbonne University, Internal Medicine Department 2, Institut E3M, French Reference Centre for Histiocytosis, Pitié-Salpȇtrière Hospital, CIMI INSERM-UMRS 1135, Assistance Publique-Hôpitaux de Paris, Paris, France.

Jean-François Emile (JF)

EA4340 BECCOH, Versailles SQY University, Pathology Department, Ambroise Paré Hospital, Assistance Publique-Hôpitaux de Paris, Boulogne, France.

Augusto Vaglio (A)

Nephrology and Dialysis Unit, Meyer Children's Hospital IRCCS, Florence, Italy.
Department of Biomedical, Experimental and Clinical Sciences "Mario Serio", University of Florence, Florence, Italy.

Julien Haroche (J)

Sorbonne University, Internal Medicine Department 2, Institut E3M, French Reference Centre for Histiocytosis, Pitié-Salpȇtrière Hospital, CIMI INSERM-UMRS 1135, Assistance Publique-Hôpitaux de Paris, Paris, France.

Classifications MeSH