Managing Severe Hemophilia A in Children: Pharmacotherapeutic Options

Autor: Regling K, Callaghan MU, Sidonio R Jr

Publikováno v: Pediatric Health, Medicine and Therapeutics, Vol Volume 13, Pp 27-35 (2022)

Katherine Regling,1,2 Michael U Callaghan,1– 3 Robert Sidonio Jr4 1Pediatric Hematology Oncology, Children’s Hospital of Michigan, Detroit, MI, USA; 2Central Michigan University School of Medicine, Mount Pleasant, MI, USA; 3Agios Pharmaceuticals,

Externí odkaz: https://doaj.org/article/6e6564ea969c4075a0fdabc928e43846

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Consensus definition of essential, optimal, and suggested components of a pediatric sickle cell disease center.

Autor: Hulbert ML; Division of Pediatric Hematology/Oncology, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA., Manwani D; Division of Pediatric Hematology/Oncology, Children's Hospital at Montefiore/Albert Einstein College of Medicine, New York, New York, USA., Meier ER; Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA., Alvarez OA; Division of Pediatric Hematology/Oncology, University of Miami School of Medicine, Miami, Florida, USA., Brown RC; Division of Pediatric Hematology/Oncology, Children's Healthcare of Atlanta/Emory University School of Medicine, Atlanta, Georgia, USA., Callaghan MU; Division of Pediatric Hematology/Oncology, Children's Hospital of Michigan, Detroit, Michigan, USA., Campbell AD; Division of Pediatric Hematology, Children's National Medical Center, Washington, District of Columbia, USA., Coates TD; Cancer and Blood Disease Institute, Department of Pediatrics, Children's Hospital Los Angeles/University of Southern California Keck School of Medicine, Los Angeles, California, USA., Frei-Jones MJ; Division of Pediatric Hematology/Oncology, University of Texas School of Medicine-San Antonio, San Antonio, Texas, USA., Hankins JS; Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA., Heeney MM; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Department of Pediatrics, Harvard Medical School, Boston, Massachusetts, USA., Hsu LL; Division of Pediatric Hematology/Oncology, University of Illinois at Chicago, Chicago, Illinois, USA., Lebensburger JD; Division of Pediatric Hematology/Oncology, University of Alabama at Birmingham, Birmingham, Alabama, USA., Quinn CT; Division of Pediatric Hematology/Oncology, Cincinnati Children's Hospital Medical Center and, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA., Shah N; Divisions of Pediatric Hematology Oncology and Hematology, Duke University School of Medicine, Durham, North Carolina, USA., Smith-Whitley K; Department of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Global Blood Therapeutics, San Francisco, California, USA., Thornburg C; Division of Pediatric Hematology/Oncology, Rady Children's Hospital/Department of Pediatrics, University of California San Diego School of Medicine, La Jolla, California, USA., Kanter J; Division of Hematology, University of Alabama at Birmingham, Birmingham, Alabama, USA.

Publikováno v: Pediatric blood & cancer [Pediatr Blood Cancer] 2023 Jan; Vol. 70 (1), pp. e29961. Date of Electronic Publication: 2022 Sep 12.

Untreated bleeds in people with hemophilia A in a noninterventional study and intrapatient comparison after initiating emicizumab in HAVEN 1-3.

Autor: Callaghan MU; Department of Pediatrics Central Michigan University Detroit Michigan USA.; Agios Pharmaceuticals Cambridge Massachusetts USA., Asikanius E; Product Development, F. Hoffmann-La Roche Ltd Basel Switzerland.; The Finnish Medicines Agency Helsinki Finland., Lehle M; Product Development, F. Hoffmann-La Roche Ltd Basel Switzerland., Oldenburg J; Institute of Experimental Haematology and Transfusion Medicine University of Bonn Bonn Germany., Mahlangu J; Haemophilia Comprehensive Care Centre, Faculty of Health Sciences University of the Witwatersrand and NHLS Johannesburg South Africa., Uguen M; PDB Biostatistics, F. Hoffmann-La Roche Ltd Basel Switzerland., Chebon S; PDB Biostatistics, F. Hoffmann-La Roche Ltd Basel Switzerland., Kruse-Jarres R; University of Washington and Washington Center For Bleeding Disorders Seattle Washington USA., Jiménez-Yuste V; Department of Hematology La Paz Hospital, Autónoma University Madrid Spain., Shima M; Department of Pediatrics Nara Medical University Kashihara Japan., Trask P; Patient Centered Outcomes Research, Genentech, Inc. South San Francisco California USA., Kempton CL; Department of Hematology and Medical Oncology and Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory Emory University School of Medicine Atlanta Georgia USA., Kessler CM; The Division of Coagulation Georgetown University Hospital Washington District of Columbia USA., Levy GG; Product Development, Genentech, Inc. South San Francisco California USA.; Spark Therapeutics, Inc. Philadelphia Pennsylvania USA., Peyvandi F; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center Milan Italy.; Department of Pathophysiology and Transplantation Università degli Studi di Milano Milan Italy.

Publikováno v: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2022 Sep 13; Vol. 6 (6), pp. e12782. Date of Electronic Publication: 2022 Sep 13 (Print Publication: 2022).

Prophylactic Hydroxyurea Treatment Is Associated with Improved Cerebral Hemodynamics as a Surrogate Marker of Stroke Risk in Sickle Cell Disease: A Retrospective Comparative Analysis.

Autor: Peine BR; Clinical Research Institute, College of Medicine, Central Michigan University, 1280 S. East Campus Dr., Mount Pleasant, MI 48859, USA., Callaghan MU; Clinical Research Institute, College of Medicine, Central Michigan University, 1280 S. East Campus Dr., Mount Pleasant, MI 48859, USA.; Children's Hospital of Michigan, 3901 Beaubien Blvd, Detroit, MI 48201, USA., Callaghan JH; Accounting Program, School of Business Administration, Oakland University, 201 Meadow Brook Road, Rochester, MI 48309, USA., Glaros AK; Clinical Research Institute, College of Medicine, Central Michigan University, 1280 S. East Campus Dr., Mount Pleasant, MI 48859, USA.; Children's Hospital of Michigan, 3901 Beaubien Blvd, Detroit, MI 48201, USA.

Publikováno v: Journal of clinical medicine [J Clin Med] 2022 Jun 17; Vol. 11 (12). Date of Electronic Publication: 2022 Jun 17.