Low-dose ITI Strategy for Children in Hemophilia A With High-titer Inhibitor and Poor ITI Risk in China (HA-LD-ITI)

Capital Medical University

Status and phase: Unknown; Phase 4

Conditions: Hemophilia A With Inhibitor

Treatments: Drug: Coagulation Factor VIII; Drug: Prednisone; Drug: Rituximab

Study type: Interventional

Funder types: Other

Identifiers

NCT03598725

BCH-ITI-20180123

Details and patient eligibility

About

The study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%～5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled. The low-dose ITI was alone or combined with immunosuppression.

Full description

Poor risk(s) includes:①peak historical inhibitor titer≥200BU ②inhibitor titer≥10BU before ITI initiation ③peak inhibitor titer during ITI≥200BU ④time to titer decline to<10BU before ITI≥24 months ⑤age≥8 years at start of ITI ⑥ITI initiated ≥5 years after inhibitor diagnosis ⑦interruptions in ITI≥2 weeks in duration. The low-dose ITI strategy consist of FⅧ（25-50IU/kg）alone or combining with immunosuppression: prednisone and Rituximab when the inhibitor titer ≥40BU ml/ml before or during ITI.

Enrollment: 55 estimated patients

Sex: Male

Ages: 1 to 14 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Males
• from 1 to 14 years old
• severe or moderate hemophilia A;
• inhibitors positive before ITI started.

Exclusion criteria

• Females
• <1 or >14 years old
• hemophilia B or mild haemophilia A;
• inhibitor negative before ITI started.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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55 participants in 1 patient group

ITI strategy

Experimental group

Description:

The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically.

Treatment:

Drug: Rituximab

Drug: Coagulation Factor VIII

Drug: Prednisone