Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis

Primary hemophagocytic lymphohistiocytosis, a rare genetic immune disorder characterized by hyperinflammation, manifests in infancy and is associated with high mortality. In a study involving 34 children, an antibody to interferon-γ (emapalumab) produced responses in 65%; it served as a bridge to ma...

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Veröffentlicht in:	The New England journal of medicine 2020-05, Vol.382 (19), p.1811-1822
Hauptverfasser:	Locatelli, Franco, Jordan, Michael B, Allen, Carl, Cesaro, Simone, Rizzari, Carmelo, Rao, Anupama, Degar, Barbara, Garrington, Timothy P, Sevilla, Julian, Putti, Maria-Caterina, Fagioli, Franca, Ahlmann, Martina, Dapena Diaz, Jose-Luis, Henry, Michael, De Benedetti, Fabrizio, Grom, Alexei, Lapeyre, Genevieve, Jacqmin, Philippe, Ballabio, Maria, de Min, Cristina
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Age of Onset Anti-Inflammatory Agents - administration & dosage Antibodies, Monoclonal - administration & dosage Antibodies, Monoclonal - adverse effects Antibodies, Neutralizing - administration & dosage Antibodies, Neutralizing - adverse effects Chemokine CXCL9 - blood Chemokines Child Child, Preschool Confidence intervals Dexamethasone Dexamethasone - administration & dosage Drug Therapy, Combination Female Hematopoietic Stem Cell Transplantation Histiocytosis Histoplasmosis Humans Hypotheses Immunotherapy Infant Infections - etiology Interferon-gamma - antagonists & inhibitors Kaplan-Meier Estimate Laboratories Lymphatic diseases Lymphocytosis Lymphohistiocytosis, Hemophagocytic - complications Lymphohistiocytosis, Hemophagocytic - drug therapy Lymphohistiocytosis, Hemophagocytic - mortality Lymphohistiocytosis, Hemophagocytic - therapy Male Monoclonal antibodies Mortality Patients Statistical analysis Stem cell transplantation Toxicity Treatment Outcome γ-Interferon
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Beschreibung
Zusammenfassung:	Primary hemophagocytic lymphohistiocytosis, a rare genetic immune disorder characterized by hyperinflammation, manifests in infancy and is associated with high mortality. In a study involving 34 children, an antibody to interferon-γ (emapalumab) produced responses in 65%; it served as a bridge to marrow transplantation in 70% of those who had received previous treatment.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1911326