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© 2024 Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. http://creativecommons.org/licenses/by-nc/4.0/ This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See:  http://creativecommons.org/licenses/by-nc/4.0/ . Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.

Abstract

Introduction

Emicizumab 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 <12 months of age.

Methods and analysis

This is a multicentre, observational study conducted in Japan in infants with congenital HA aged <12 months who are receiving or are scheduled to receive prophylactic emicizumab at an approved dosing regimen: 1.5 mg/kg weekly, 3 mg/kg every 2 weeks or 6 mg/kg every 4 weeks. The target inclusion is 50 infants. The primary endpoint is to evaluate the relationship between global coagulation test parameters (using clot waveform analysis and thrombin generation assay) and plasma emicizumab concentrations in infants aged from 6 to <12 months. Secondary endpoints include evaluating coagulation profiles in infants aged <6 months and changes between the age of <6 and 6 to <12 months. Additionally, coagulation parameters will be evaluated with the in vitro addition of anti-idiotype antibodies against emicizumab or the addition of a factor VIII product in infants aged from 6 to <12 months. The study will also evaluate adverse events and bleeds.

Ethics and dissemination

The study was approved by the MINS Clinical Trial Review Committee (no. 230214) and will be conducted in compliance with the Declaration of Helsinki, the Act on the Protection of Personal Information and the Guidance of Ethical Guidelines for Medical and Biological Research Involving Human Subjects. Written informed consent for participation in the study will be obtained from a legally acceptable representative. Results will be published in scientific/medical journals and presented at international congresses.

Trial registration number

Japan Registry of Clinical Trials; jRCT1031230264.

Details

Title
HINODE study: haemophilia A in infancy and newborns – protocol for a prospective, multicentre, observational study evaluating the coagulation potential and safety of emicizumab prophylaxis
Author
Ohga, Shouichi 1   VIAFID ORCID Logo  ; Takeyama, Masahiro 2 ; Ishimura, Masataka 1 ; Inoue, Hirosuke 1 ; Nosaka, Daisuke 3 ; Iwasaki, Keisuke 4 ; Mitsui, Chika 3 ; Nogami, Keiji 5   VIAFID ORCID Logo 

 Department of Paediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan 
 Department of Paediatrics, Nara Medical University, Kashihara, Japan; Division of Haemophilia, National Hospital Organization, Osaka National Hospital, Osaka, Japan 
 Specialty Medical Affairs Division, Chugai Pharmaceutical Co., Ltd, Tokyo, Japan 
 Biometrics Department, Chugai Pharmaceutical Co., Ltd, Tokyo, Japan 
 Department of Paediatrics, Nara Medical University, Kashihara, Japan 
First page
e087556
Section
Haematology (incl blood transfusion)
Publication year
2024
Publication date
2024
Publisher
BMJ Publishing Group LTD
e-ISSN
20446055
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
3149199796
Copyright
© 2024 Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. http://creativecommons.org/licenses/by-nc/4.0/ This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See:  http://creativecommons.org/licenses/by-nc/4.0/ . Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.