HINODE study: haemophilia A in infancy and newborns – protocol for a prospective, multicentre, observational study evaluating the coagulation potential and safety of emicizumab prophylaxis

IntroductionEmicizumab prophylaxis is approved for people of all ages with haemophilia A (HA) including infants and children. Although previous studies have demonstrated the efficacy and tolerability of emicizumab in infants with HA, real-world data on emicizumab use in infants are limited. The Haem...

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Veröffentlicht in:	BMJ open 2024-12, Vol.14 (12), p.e087556
Hauptverfasser:	Ohga, Shouichi, Takeyama, Masahiro, Ishimura, Masataka, Inoue, Hirosuke, Nosaka, Daisuke, Iwasaki, Keisuke, Mitsui, Chika, Nogami, Keiji
Format:	Artikel
Sprache:	eng
Schlagworte:	Age Antibodies, Bispecific - administration & dosage Antibodies, Bispecific - therapeutic use Antibodies, Monoclonal, Humanized - therapeutic use Bleeding disorders & coagulopathies Blood Coagulation - drug effects Blood Coagulation Tests Disease prevention Female HAEMATOLOGY Haematology (incl blood transfusion) Hemophilia Hemophilia A - drug therapy Humans Infant Infant, Newborn Japan Male Monoclonal antibodies Multicenter Studies as Topic NEONATOLOGY Newborn babies Observational studies Observational Studies as Topic PAEDIATRICS Pediatrics Prospective Studies Protocol
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Zusammenfassung:	IntroductionEmicizumab prophylaxis is approved for people of all ages with haemophilia A (HA) including infants and children. Although previous studies have demonstrated the efficacy and tolerability of emicizumab in infants with HA, real-world data on emicizumab use in infants are limited. The Haemophilia A in Infancy and NewbOrns: multi-instituional prospective observational study to assess the efficacy anD safety of Emicizumab (HINODE) study aims to evaluate the coagulation potential and safety of emicizumab prophylaxis in infants with congenital HA from birth to
ISSN:	2044-6055 2044-6055
DOI:	10.1136/bmjopen-2024-087556