HSCT may lower leukemia risk in ELANE neutropenia: a before–after study from the French Severe Congenital Neutropenia Registry
| Autor: | Yves Bertrand, Blandine Beaupain, Despina Moshous, Ouahiba Nachit, Nathalie Aladjdi, Bruno Filhon, Flore Sicre de Fontbrune, Christine Bellanné-Chantelot, Jean-Hugues Dalle, Benedicte Neven, Gioacchino Andrea Rotulo, Fanny Rialland, J. Donadieu, Marie Ouachee, E. Dore, Catherine Paillard, Claire Galambrun, Virginie Gandemer, Regis Peffault de la Tour |
|---|---|
| Přispěvatelé: | Centre de Recherche Saint-Antoine (CR Saint-Antoine), Sorbonne Université (SU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), CHU Trousseau [APHP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Universita degli studi di Genova, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Centre hospitalier universitaire de Nantes (CHU Nantes), Université de Strasbourg (UNISTRA), Hôpital de la Timone [CHU - APHM] (TIMONE), CHU Pontchaillou [Rennes], Institut d'hématologie et d'oncologie pédiatrique [CHU - HCL] (IHOPe), Hospices Civils de Lyon (HCL), CHU Necker - Enfants Malades [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), CHU Clermont-Ferrand, CHU Bordeaux [Bordeaux], CHU Rouen, Normandie Université (NU), Hopital Saint-Louis [AP-HP] (AP-HP), Service de Génétique Cytogénétique et Embryologie [CHU Pitié-Salpêtrière], CHU Pitié-Salpêtrière [AP-HP], Sorbonne Université - Faculté de Médecine (SU FM), Sorbonne Université (SU), Service d'Hématologie Biologique [Hôpital Robert Debré, Paris], AP-HP Hôpital universitaire Robert-Debré [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP) |
| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
Pediatrics
medicine.medical_specialty Neutropenia [SDV]Life Sciences [q-bio] 03 medical and health sciences 0302 clinical medicine hemic and lymphatic diseases Congenital Bone Marrow Failure Syndromes Humans Medicine Registries Congenital Neutropenia Before after study Transplantation Acute leukemia business.industry Hematopoietic Stem Cell Transplantation Hematology medicine.disease 3. Good health Leukemia Controlled Before-After Studies 030220 oncology & carcinogenesis Cohort France business 030215 immunology |
| Zdroj: | Bone Marrow Transplantation Bone Marrow Transplantation, Nature Publishing Group, 2020, 55 (8), pp.1614-1622. ⟨10.1038/s41409-020-0800-1⟩ |
| ISSN: | 0268-3369 |
| DOI: | 10.1038/s41409-020-0800-1⟩ |
| Popis: | International audience; ELANE neutropenia is associated with myelodysplasia and acute leukemia (MDS–AL), and severe infections. Because the MDS–AL risk has also been shown to be associated with exposure to GCSF, since 2005, in France, patients receiving high daily GCSF doses (>15 μg/kg/day) are eligible for HSCT, in addition to classic indications (MDS–AL or GCSF refractoriness). We analyzed the effect of this policy. Among 144 prospectively followed ELANE-neutropenia patients enrolled in the French Severe Congenital Neutropenia Registry, we defined two groups according to period: “before 2005” for those born before 2005 and followed until 31/12/2004 (1588 person-years); and “after 2005” comprised of those born after 2005 or born before 2005 but followed after 2005 until 31/03/2019 (1327 person-years). Sixteen of our cohort patients underwent HSCT (14 long-term survivors) and six developed MDS–ALs. Six leukemic transformations occurred in the before-2005 group and none after 2005 (respective frequencies 3.8 × 10–3 vs. 0; P |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|
Full text from SpringerLink