Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis.

Adolescent Age of Onset Anti-Inflammatory Agents / administration & dosage Antibodies, Monoclonal / administration & dosage Antibodies, Neutralizing / administration & dosage Chemokine CXCL9 / blood Child Child, Preschool Dexamethasone / administration & dosage Drug Therapy, Combination Female Hematopoietic Stem Cell Transplantation Humans Infant Infections / etiology Interferon-gamma / antagonists & inhibitors Kaplan-Meier Estimate Lymphohistiocytosis, Hemophagocytic / complications Male Treatment Outcome

Journal

The New England journal of medicine
ISSN: 1533-4406
Titre abrégé: N Engl J Med
Pays: United States
ID NLM: 0255562

Informations de publication

Date de publication:
07 05 2020
Historique:
entrez: 7 5 2020
pubmed: 7 5 2020
medline: 20 5 2020
Statut: ppublish

Résumé

Primary hemophagocytic lymphohistiocytosis is a rare syndrome characterized by immune dysregulation and hyperinflammation. It typically manifests in infancy and is associated with high mortality. We investigated the efficacy and safety of emapalumab (a human anti-interferon-γ antibody), administered with dexamethasone, in an open-label, single-group, phase 2-3 study involving patients who had received conventional therapy before enrollment (previously treated patients) and previously untreated patients who were 18 years of age or younger and had primary hemophagocytic lymphohistiocytosis. The patients could enter a long-term follow-up study until 1 year after allogeneic hematopoietic stem-cell transplantation or until 1 year after the last dose of emapalumab, if transplantation was not performed. The planned 8-week treatment period could be shortened or extended if needed according to the timing of transplantation. The primary efficacy end point was the overall response, which was assessed in the previously treated patients according to objective clinical and laboratory criteria. At the cutoff date of July 20, 2017, a total of 34 patients (27 previously treated patients and 7 previously untreated patients) had received emapalumab; 26 patients completed the study. A total of 63% of the previously treated patients and 65% of the patients who received an emapalumab infusion had a response; these percentages were significantly higher than the prespecified null hypothesis of 40% (P = 0.02 and P = 0.005, respectively). In the previously treated group, 70% of the patients were able to proceed to transplantation, as were 65% of the patients who received emapalumab. At the last observation, 74% of the previously treated patients and 71% of the patients who received emapalumab were alive. Emapalumab was not associated with any organ toxicity. Severe infections developed in 10 patients during emapalumab treatment. Emapalumab was discontinued in 1 patient because of disseminated histoplasmosis. Emapalumab was an efficacious targeted therapy for patients with primary hemophagocytic lymphohistiocytosis. (Funded by NovImmune and the European Commission; NI-0501-04 and NI-0501-05 ClinicalTrials.gov numbers, NCT01818492 and NCT02069899.).

Sections du résumé

BACKGROUND
Primary hemophagocytic lymphohistiocytosis is a rare syndrome characterized by immune dysregulation and hyperinflammation. It typically manifests in infancy and is associated with high mortality.
METHODS
We investigated the efficacy and safety of emapalumab (a human anti-interferon-γ antibody), administered with dexamethasone, in an open-label, single-group, phase 2-3 study involving patients who had received conventional therapy before enrollment (previously treated patients) and previously untreated patients who were 18 years of age or younger and had primary hemophagocytic lymphohistiocytosis. The patients could enter a long-term follow-up study until 1 year after allogeneic hematopoietic stem-cell transplantation or until 1 year after the last dose of emapalumab, if transplantation was not performed. The planned 8-week treatment period could be shortened or extended if needed according to the timing of transplantation. The primary efficacy end point was the overall response, which was assessed in the previously treated patients according to objective clinical and laboratory criteria.
RESULTS
At the cutoff date of July 20, 2017, a total of 34 patients (27 previously treated patients and 7 previously untreated patients) had received emapalumab; 26 patients completed the study. A total of 63% of the previously treated patients and 65% of the patients who received an emapalumab infusion had a response; these percentages were significantly higher than the prespecified null hypothesis of 40% (P = 0.02 and P = 0.005, respectively). In the previously treated group, 70% of the patients were able to proceed to transplantation, as were 65% of the patients who received emapalumab. At the last observation, 74% of the previously treated patients and 71% of the patients who received emapalumab were alive. Emapalumab was not associated with any organ toxicity. Severe infections developed in 10 patients during emapalumab treatment. Emapalumab was discontinued in 1 patient because of disseminated histoplasmosis.
CONCLUSIONS
Emapalumab was an efficacious targeted therapy for patients with primary hemophagocytic lymphohistiocytosis. (Funded by NovImmune and the European Commission; NI-0501-04 and NI-0501-05 ClinicalTrials.gov numbers, NCT01818492 and NCT02069899.).

Identifiants

DOI: 10.1056/NEJMoa1911326 PMID: 32374962
pubmed: 32374962
doi: 10.1056/NEJMoa1911326
doi:

Substances chimiques

Anti-Inflammatory Agents 0
Antibodies, Monoclonal 0
Antibodies, Neutralizing 0
CXCL9 protein, human 0
Chemokine CXCL9 0
Emapalumab 0
Dexamethasone 7S5I7G3JQL
Interferon-gamma 82115-62-6

Banques de données

ClinicalTrials.gov
['NCT01818492', 'NCT02069899']

Types de publication

Clinical Trial, Phase II Clinical Trial, Phase III Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1811-1822

Commentaires et corrections

Type : CommentIn
Type : CommentIn

Informations de copyright

Copyright © 2020 Massachusetts Medical Society.

Auteurs

Franco Locatelli (F)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Michael B Jordan (MB)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Carl Allen (C)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Simone Cesaro (S)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Carmelo Rizzari (C)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Anupama Rao (A)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Barbara Degar (B)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Timothy P Garrington (TP)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Julian Sevilla (J)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).
ORCID: 0000-0002-6852-1860

Maria-Caterina Putti (MC)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Franca Fagioli (F)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Martina Ahlmann (M)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Jose-Luis Dapena Diaz (JL)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Michael Henry (M)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Fabrizio De Benedetti (F)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Alexei Grom (A)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Genevieve Lapeyre (G)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Philippe Jacqmin (P)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Maria Ballabio (M)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

Cristina de Min (C)

From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology-Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children's Hospital, Rome, Pediatric Hematology-Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology-Oncology Unit, Department of Pediatrics, University of Milano-Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic of Pediatric Hematology-Oncology, University Hospital of Padova, Padua (M.-C.P.), and the Division of Pediatric Onco-Hematology, Regina Margherita Hospital, Turin (F.F.) - all in Italy; the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics (M.B.J.), and the Division of Rheumatology (A.G.), Cincinnati Children's Hospital Medical Center, and the University of Cincinnati College of Medicine (M.B.J.) - all in Cincinnati; the Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston (C.A.); the Department of Hematology, Great Ormond Street Hospital for Children, London (A.R.); the Department of Pediatric Hematology-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (B.D.); the Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora (T.P.G.); the Departments of Pediatric Hematology-Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid (J.S.), and the Department of Pediatric Hematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona (J.-L.D.D.); the Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany (M.A.); the Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ (M.H.); NovImmune, Plan-les-Ouates, Switzerland (G.L., M.B., C.M.); and MnS Modelling and Simulation, Dinant, Belgium (P.J.).

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adolescent › Emapalumab in Children with Primary...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Facteurs épidémiologiques › Facteurs âges › Âge de début › Emapalumab in Children with Primary...
questionsmedicales.fr › Actions chimiques et utilisations › Actions pharmacologiques › Utilisations thérapeutiques › Anti-inflammatoires › Emapalumab in Children with Primary...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Globulines › Sérum-globulines › Immunoglobulines › Anticorps › Anticorps monoclonaux › Emapalumab in Children with Primary...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Globulines › Sérum-globulines › Immunoglobulines › Anticorps › Anticorps neutralisants › Emapalumab in Children with Primary...
questionsmedicales.fr › Facteurs biologiques › Protéines et peptides de signalisation intercellulaire › Cytokines › Chimiokines › Chimiokines CXC › Chimiokine CXCL9 › Emapalumab in Children with Primary...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Emapalumab in Children with Primary...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Enfant d'âge préscolaire › Emapalumab in Children with Primary...
questionsmedicales.fr › Composés polycycliques › Composés à cycles fusionnés › Stéroïdes › Stéroïdes fluorés › Dexaméthasone › Emapalumab in Children with Primary...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Association de médicaments › Emapalumab in Children with Primary...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches hématopoïétiques › Emapalumab in Children with Primary...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Emapalumab in Children with Primary...
questionsmedicales.fr › Personnes › Tranches d'âge › Nourrisson › Emapalumab in Children with Primary...
questionsmedicales.fr › Infection › Emapalumab in Children with Primary...
questionsmedicales.fr › Facteurs biologiques › Protéines et peptides de signalisation intercellulaire › Cytokines › Lymphokines › Facteurs d'activation des macrophages › Interféron gamma › Emapalumab in Children with Primary...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Statistiques comme sujet › Analyse de survie › Estimation de Kaplan-Meier › Emapalumab in Children with Primary...
questionsmedicales.fr › Hémopathies et maladies lymphatiques › Maladies lymphatiques › Histiocytose › Histiocytose non langerhansienne › Lymphohistiocytose hémophagocytaire › Emapalumab in Children with Primary...
questionsmedicales.fr › Qualité, accès, évaluation des soins de santé › Qualité des soins de santé › Mécanismes d'évaluation des soins de santé › Évaluation des résultats et des processus en soins de santé › Évaluation de résultat (soins) › Résultat thérapeutique › Emapalumab in Children with Primary...