Avapritinib versus Placebo in Indolent Systemic Mastocytosis.

Journal

NEJM evidence
ISSN: 2766-5526
Titre abrégé: NEJM Evid
Pays: United States
ID NLM: 9918317485806676

Informations de publication

Date de publication:
Jun 2023
Historique:
medline: 6 2 2024
pubmed: 6 2 2024
entrez: 6 2 2024
Statut: ppublish

Résumé

Avapritinib in Indolent Systemic MastocytosisIn a randomized trial, patients with indolent systemic mastocytosis were treated with avapritinib or placebo along with supportive care. The trial primary end point was the change in mean total symptom scores at 24 weeks. Avapritinib-treated patients had a decrease in mean total symptom score of 15.6 points compared with 9.2 points in the placebo group.

Identifiants

DOI: 10.1056/EVIDoa2200339 PMID: 38320129
pubmed: 38320129
doi: 10.1056/EVIDoa2200339
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

EVIDoa2200339

Auteurs

Jason Gotlib (J)

Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA.

Mariana Castells (M)

Department of Medicine, Division of Allergy and Clinical Immunology, Brigham and Women's Hospital, Harvard Medical School, Boston.

Hanneke Oude Elberink (HO)

Department of Allergology, University Medical Center, Groningen Research Institute Asthma and COPD, University of Groningen, Groningen, the Netherlands.

Frank Siebenhaar (F)

Institute of Allergology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin.
Fraunhofer Institute for Translational Medicine and Pharmacology, Allergology and Immunology, Berlin.

Karin Hartmann (K)

Division of Allergy, Department of Dermatology, University Hospital Basel and University of Basel, Basel, Switzerland.
Department of Biomedicine, University Hospital Basel and University of Basel, Basel, Switzerland.

Sigurd Broesby-Olsen (S)

Department of Dermatology and Allergy Centre, Odense University Hospital, Odense, Denmark.

Tracy I George (TI)

Associated Regional and University Pathologists, Inc. Laboratories, Department of Pathology, University of Utah School of Medicine, Salt Lake City.

Jens Panse (J)

Department of Oncology, Hematology, Hemostaseology, and Stem Cell Transplantation, University Hospital Aachen, Medical Faculty, Rheinisch-Westfälische Technische Hochschule Aachen University, Aachen, Germany.
Center for Integrated Oncology, Aachen, Bonn, Cologne, Düsseldorf (ABCD), Aachen, Germany.

Iván Alvarez-Twose (I)

Institute of Mastocytosis Studies of Castilla-La Mancha, Virgen del Valle Hospital, Toledo, Spain.

Deepti H Radia (DH)

Guy's & St. Thomas' National Health Service Foundation Trust, London.

Tsewang Tashi (T)

Huntsman Cancer Institute, University of Utah, Salt Lake City.

Cristina Bulai Livideanu (C)

Department of Dermatology, Centre of Reference for Mastocytosis, Toulouse University Hospital, Toulouse, France.

Vito Sabato (V)

Department of Immunology, Allergology and Rheumatology, University of Antwerp and Antwerp University Hospital, Antwerp, Belgium.

Mark Heaney (M)

Department of Medicine, Columbia University Medical Center, New York.

Paul Van Daele (P)

Department of Internal Medicine and Immunology, Erasmus Medical Center, Rotterdam, the Netherlands.

Sonia Cerquozzi (S)

Department of Medicine, University of Calgary, Calgary, AB, Canada.

Ingunn Dybedal (I)

Department of Hematology, Oslo University Hospital, Oslo.

Andreas Reiter (A)

Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Germany.

Thanai Pongdee (T)

Division of Allergic Diseases, Mayo Clinic, Rochester, MN.

Stéphane Barete (S)

Unit of Dermatology, Centre of Reference for Mastocytosis, Pitié-Salpêtrière Hospital, Assistance Publique-Hôpitaux de Paris, Sorbonne Université, Paris.

Celalettin Ustun (C)

Department of Internal Medicine, Division of Hematology, Oncology and Cell Therapy, Section of Bone Marrow Transplantation and Cellular Therapy, Rush Medical College, Chicago.

Lawrence Schwartz (L)

Virginia Commonwealth University, Richmond, VA.

Brant R Ward (BR)

Virginia Commonwealth University, Richmond, VA.

Philippe Schafhausen (P)

Department of Oncology, Hematology, and Bone Marrow Transplantation with Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

Peter Vadas (P)

Department of Medicine, Division of Clinical Immunology and Allergy, St. Michael's Hospital, University of Toronto, Toronto.

Prithviraj Bose (P)

Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston.

Daniel J DeAngelo (DJ)

Dana-Farber Cancer Institute, Boston.

Lindsay Rein (L)

Department of Medicine, Duke University School of Medicine, Durham, NC.

Pankit Vachhani (P)

Department of Medicine, University of Alabama at Birmingham, Birmingham, AL.

Massimo Triggiani (M)

Division of Allergy and Clinical Immunology, University of Salerno, Salerno, Italy.

Patrizia Bonadonna (P)

Allergy Unit and Asthma Center, Azienda Ospedaliera Universitaria Integrata di Verona, Verona, Italy.

Mark Rafferty (M)

The Beatson West of Scotland Cancer Centre, Glasgow, Scotland.

Nauman M Butt (NM)

The Clatterbridge Cancer Centre, Bebington, Wirral, United Kingdom.

Stephen T Oh (ST)

Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine, Washington University, St. Louis.

Friederike Wortmann (F)

Oberärztin Hämatologie/Onkologie bei Uksh Campus Lübeck, Universitätsklinikum Schleswig-Holstein, Universität zu Lübeck, Lübeck, Schleswig-Holstein, Germany.

Johanna Ungerstedt (J)

Department of Medicine, Huddinge (H7), Karolinska University Hospitale, Stockholm.

Mar Guilarte (M)

Hospital UniversitariVall d'Hebron, Institut de Recerca Vall d'Hebron (VHIR), Barcelona.

Minakshi Taparia (M)

University of Alberta, Edmonton, AB, Canada.

Andrew T Kuykendall (AT)

Department of Malignant Hematology, H. Lee Moffitt Cancer Center, Tampa, FL.

Cecilia Arana Yi (C)

Division of Hematology and Medical Oncology, Mayo Clinic, Phoenix, AZ.

Princess Ogbogu (P)

Division of Pediatric Allergy, Immunology and Rheumatology, Department of Pediatrics, University Hospitals Rainbow Babies and Children's Hospital, Case Western Reserve University School of Medicine, Cleveland.

Caroline Gaudy-Marqueste (C)

Service de Dermatologie et de cancérologie cutanée, Assistance Publique-Hopitaux de Marseille, Aix-Marseille Université, Marseille, France.

Mattias Mattsson (M)

Department of Hematology, Uppsala University Hospital and Department of Immunology, Genetics and Pathology, Uppsala, Sweden.

William Shomali (W)

Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA.

Matthew P Giannetti (MP)

Division of Allergy and Clinical Immunology, Brigham and Women's Hospital, Boston.

Ilda Bidollari (I)

Blueprint Medicines Corporation, Cambridge, MA.

Hui-Min Lin (HM)

Blueprint Medicines Corporation, Cambridge, MA.

Erin Sulllivan (E)

Blueprint Medicines Corporation, Cambridge, MA.

Brenton Mar (B)

Blueprint Medicines Corporation, Cambridge, MA.

Robyn Scherber (R)

Blueprint Medicines Corporation, Cambridge, MA.

Maria Roche (M)

Blueprint Medicines Corporation, Cambridge, MA.

Cem Akin (C)

University of Michigan, Ann Arbor, MI.

Marcus Maurer (M)

Institute of Allergology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin.
Fraunhofer Institute for Translational Medicine and Pharmacology, Allergology and Immunology, Berlin.

Classifications MeSH