Article Dans Une Revue Blood Année : 2024

Childhood Langerhans cell histiocytosis hematological involvement: severity associated with BRAFV600E loads

1 Centre Léon Bérard [Lyon]
2 CHU Montpellier = Montpellier University Hospital
3 IHOPe - Institut d'hématologie et d'oncologie pédiatrique [CHU - HCL]
4 CHU Trousseau [APHP]
5 CHUGA - Centre Hospitalier Universitaire [CHU Grenoble]
6 CHU Nice - Centre Hospitalier Universitaire de Nice
7 CHU - BREST - Hôpital de la Cavale Blanche - CHRU Brest
8 CHU de Bordeaux Pellegrin [Bordeaux]
9 IGR - Institut Gustave Roussy
10 Institut Curie [Paris]
11 Hôpital Robert Debré
12 AP-HP - Assistance publique - Hôpitaux de Paris (AP-HP)
13 CHU Limoges
14 Centre Hospitalier Universitaire de Rennes [CHU Rennes] = Rennes University Hospital [Pontchaillou]
15 IGDR - Institut de Génétique et Développement de Rennes
16 CHU Nancy - Centre Hospitalier Universitaire de Nancy
17 CHU Toulouse - Centre Hospitalier Universitaire de Toulouse
18 TIMONE - Hôpital de la Timone [CHU - APHM]
19 APHM - Assistance Publique - Hôpitaux de Marseille
20 HCL - Hospices Civils de Lyon
21 CHU Amiens-Picardie
22 UNISTRA - Université de Strasbourg
23 CHU de Poitiers [La Milétrie] - Centre hospitalier universitaire de Poitiers = Poitiers University Hospital
24 Hôpital Jeanne de Flandre [Lille]
25 CHRU Lille - Centre Hospitalier Régional Universitaire [CHU Lille]
26 CHU ST-E - Centre Hospitalier Universitaire de Saint-Etienne [CHU Saint-Etienne]
27 MSKCC - Memorial Sloan Kettering Cancer Center
28 UT - Université de Tours
29 CHU Angers - Centre Hospitalier Universitaire d'Angers
30 CRCI2NA - Centre de Recherche en Cancérologie et Immunologie Intégrée Nantes-Angers
31 CHU La Réunion - Centre Hospitalier Universitaire de La Réunion
32 Nantes Univ - Nantes Université
33 Hôpital Necker - Enfants Malades [AP-HP]
34 CRSA - Centre de Recherche Saint-Antoine
35 Hôpital Ambroise Paré [AP-HP]
Julian Thalhammer
Nathalie Aladjidi
Charlotte Rigaud
Amaury Leruste
Thomas Lauvray
Solenne Le Louet
Julien Lejeune
Audrey Grain
Caroline Thomas
Zofia Helias-Rodzewicz
D. Moshous
Odile Fenneteau
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  • PersonId : 1093079
Aurore Coulomb-l'Hermine
Sébastien Héritier
Jean Donadieu
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Résumé

Hematological involvement (HI) is one of the life-threatening risk organs (ROs) in Langerhans cell histiocytosis (LCH). Lahey criteria have defined HI since 1975 as hemoglobin <10 g/dL and/or platelets <100 G/L and/or leukopenia (white blood cell count <4 G/L) and/or neutrophils <1.5 G/. Among the 2313 patients <18 years old enrolled in the French National Histiocytosis Registry (1983-2023), 331 developed HI (median age at diagnosis: 1 year); median follow-up lasted 8.1 years. Bone-marrow aspirate smears and biopsies may show reactive histiocytes, hemophagocytosis or myelofibrosis but never confirm the diagnosis. Fifty-eight (17%) patients developed macrophage-activation syndrome, sometimes related to acute Epstein-Barr virus or cytomegalovirus infection, sometimes months before typical LCH manifestations appeared. Hemoglobin and platelet thresholds for initiating transfusion(s) appear to accurately distinguish 2 groups: mild HI (MHI; >7 g/dL and >20 G/L, respectively) and severe HI (SHI; ≤7 g/dL and ≤20 G/L). Each entity has different organ involvements, laboratory parameters, mutational status, blood BRAFV600E loads, drug sensitivities and outcomes (respective MHI and SHI 10-year survival rates: 98% and 73%). Since 1998, mortality first declined with combination Cladribine-cytarabine therapy, and then with mitogen-activated protein-kinase inhibitors since 2014. Forty-one (12%) patients developed neurodegenerative complications that have emerged as a risk for long-term survivors. These results suggest limiting the HI-RO definition to SHI, as it encompasses almost all medical complications of LCH. Future clinical trials might demonstrate that targeted-therapy approaches would be better adapted for these patients, while MHI can be managed with classic therapies.
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mercredi 2 avril 2025
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mercredi 2 avril 2025
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Dates et versions

hal-04893579 , version 1 (20-02-2025)

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Julian Thalhammer, Eric Jeziorski, Perrine Marec-Bérard, Mohamed-Aziz Barkaoui, Anne Pagnier, et al.. Childhood Langerhans cell histiocytosis hematological involvement: severity associated with BRAFV600E loads. Blood, 2024, ⟨10.1182/blood.2024025625⟩. ⟨hal-04893579⟩
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