Health Condition 1: D66- Hereditary factor VIII deficiency
Conditions
Interventions
Intervention1: Low dose emicizumab prophylaxis: Subcutaneously 3mg per kg emicizumab once a month directly with no loading dose
Control Intervention1: standard dose emicizumab prophylaxis: A loading d
Sponsors
Department of Health Research, India
Eligibility
Inclusion criteria
Inclusion criteria: All persons with haemophilia A(PWH)with FVIII levels less than 2 percentage as seen in case notes with age ranging from 2 to 17 years PWH with and without the presence of FVIII inhibitors PWH with inhibitors not planning for immune tolerance induction or where immune tolerance induction has failed PWH with hepatitis and HIV Parents or patients willing to signed and give written informed consent or assent as appropriate Children and caregivers willing to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the questionnaires. Written or verbal assent as appropriate from children participating in the study
Exclusion criteria
Exclusion criteria: Patients who will find it difficult to follow the protocol based on PI judgement Patients posted for elective surgery Inherited or acquired bleeding disorder other than hemophilia A Ongoing Immune tolerance Induction therapy (prophylaxis regimens with FVIII and or bypassing agents must be discontinued prior to enrollment)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annual Bleed Rate, Annual Joint Bleed Rate, target joint and laboratory dataTimepoint: 9 months prior starting low or standard dose emicizumab, baseline and during emicizumab prophylaxis: 4 weeks, 8 weeks, 12 weeks, 16 weeks, 20 weeks, 24 weeks. Post emicizumab prophylaxis: 4 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Difference in the number of infusions & cost comparison which will depend on the total units of Emicizumab used between low dose group and standard dose group. Timepoint: 24 weeks after start of emicizumab prophylaxis;Haemophilia Joint Health Score [HJHS joint score] Timepoint: 9 months prior, During emicizumab prophylaxis: 12 weeks & 24 weeks Post: 4 weeks | — |
Countries
India
Contacts
Public ContactDr Rucha Patil
ICMR National Institute of Immunohaematology
Outcome results
None listed