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Hemophilia A in infancy and newborns; multi-institutional prospective observational study to assess the efficacy and safety of emicizumab

HINODE study

Nogami Keiji

Nara Medical University Hospital

840,shijocho,kashihara-shi,Nara,Japan

pediatrics@naramed-u.ac.jp

Furukawa Shoko

Nara Medical University Hospital

840,shijocho,kashihara-shi,Nara,Japan

+81-744-22-3051

pediatrics@naramed-u.ac.jp

Recruiting

July. 29, 2023

Observational

Inclusion criteria;
1)Patientss for whom consent to participate in the study has been obtained from proxy consenters by means of written informed consent
2) Confirmed diagnosis with Congenital haemophilia A at enrollment.
3)Patients selected for the treatment with emicizumab as the most appropriate medical treatment (those who planned to receive emicizumab treatment in the selection of the treatment between physicians and patients) and who are receiving or scheduled to receive emicizumab according to the current electronic package insert dosage and administration.
4)Patients who have reached or are likely to reach the maintenance phase* of emicizumab at less than 12 months of age and are able to obtain blood samples in the maintenance phase* of less than 12 months of age.
*The maintenance phase in this study refers to the fifth and subsequent doses of emicizumab.

Exclusion criteria;
1)Patients with inherited or acquired bleeding disorders other than haemophilia A.
2)Patients to be inappropriate to enter this study for some other reason judged by the investigators.

Both

Congenital haemophilia A

Relationship between various parameters of comprehensive coagulation tests and plasma emicizumab levels in congenital haemophilia A aged 6 months or older and younger than 12 months.

1)Generalized coagulation tests* such as FVIII activity in congenital haemophilia A patients aged 6 months or older and younger than 12 months, and various parameters of comprehensive coagulation tests when anti-emicizumab idiotype antibody or FVIII is added.
2)Relationship between various coagulability (comprehensive coagulation test, generalized coagulation tests such as FVIII activity)* and plasma emicizumab levels in patients with congenital hemophilia A younger than 6 months of age.
3)Various changes in coagulation ability* between younger than 6 months and older than 6 months and younger than 12 months.
4)Adverse events (particularly anti-FVIII inhibitor development and abnormal laboratory findings)
5)Hemorrhage requiring treatment with blood coagulation factor products (especially if intracranial hemorrhage occurs)
*If there is a residue in the blood sample volume, various coagulability under emicizumab neutralization after adding anti-emicizumab idiotype antibody and various coagulability after adding FVIII preparation are also evaluated.

+81-3-6416-1868

May. 18, 2023

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