The outcome comparison of low- and medium-dose immune tolerance induction therapy using domestic recombinant coagulation factor VIII in severe hemophilia A children with high titer inhibitor: a randomized controlled clinical trial

Conditions

hemophilia A

Li Z, Li Z, Cheng X, Zhang H, Yang C, Xu Q, Chen Z, Zhen Y, Li G, Liu G, Yao W, Zhou M, Jin J, Huang J, Fang Y, Xie L, Poon MC, Wu R.

2024-11-28

10.1016/j.rpth.2024.102639

Chinese Severe Hemophilia a Children with High-Titer Inhibitors Obtain More Benefit from Intermediate-Dose Immune Tolerance Induction: The Interim Result of Lome Part a

Zhengping Li, Runhui Wu

Blood2023-11-02

10.1182/blood-2023-185290

P1619: LOME PILOT STUDY: A MULTICENTER CONTROLLED RANDOMIZED CLINICAL TRIAL OF LOW- VERSUS INTERMEDIATE-DOSE IMMUNE TOLERANCE INDUCTION WITH RECOMBINANT FACTOR VIII (OMFILOCTOCOG ALFA, SCT800) IN CHINA

Zengping LI, Runhui Wu

HemaSphere2023-08-01

10.1097/01.hs9.0000973352.39161.d6

Protocol and Primary Analysis of Lome: A Randomized Trial of Low- Versus Intermediate-Dose Immune Tolerance Induction with Recombinant Factor VIII (Omfiloctocog alfa, SCT800) in China

Zhengping Li, Guoqing Liu, Yingzi Zhen, Xiaoling Cheng, Zhenping Chen et al. (10 authors)

Blood2022-11-15

10.1182/blood-2022-164951

Interventions

low-dose ITI group:low-dose ITI therapy (FVIII 50IU/kg QoD)

medium-dose ITI group:medium-dose ITI therapy (FVIII 100IU/kg QD)

Eligibility

Sex/Gender

Male

Age

No minimum to 12 Years

Inclusion criteria

Inclusion criteria: 1. Severe hemohilia A 2. 0-=5BU/mL-<200BU/mL at two consecutive visits

Exclusion criteria

Exclusion criteria: 1. Suffering from other immune diseases 2. Using immunosuppressant IS to treat other disease(s) 3. The requests of major surgery during study 4. Poor patients' compliance

Design outcomes

Primary

Measure	Time frame
success rate of ITI;	—

Secondary

Measure	Time frame
partical success rate of ITI;median time to ITI success;median time to ITI partical success;Annualized Bleeding Rates and Annualized Joint Bleeding Rates;Ultrasonic Measurement of Joint ;CHO-KLAT 2.0(The Canadian Hemophilia Outcomes-Kids Life Assessment Tool 2.0);BCH activity of daily life;MRI Measurement of Joint;HJHS 2.0 (Hemophilia Joint Health Score 2.0);patients' compliance;therapy cost;absence from school;safety;	—

Measure

Time frame

partical success rate of ITI;median time to ITI success;median time to ITI partical success;Annualized Bleeding Rates and Annualized Joint Bleeding Rates;Ultrasonic Measurement of Joint ;CHO-KLAT 2.0(The Canadian Hemophilia Outcomes-Kids Life Assessment Tool 2.0);BCH activity of daily life;MRI Measurement of Joint;HJHS 2.0 (Hemophilia Joint Health Score 2.0);patients' compliance;therapy cost;absence from school;safety;

—

Countries

China

Contacts

Public ContactRunhui Wu

Beijing Children's Hospital Affiliated to Capital Medical University

runhuiwu@hotmail.com+86 13370115037

Outcome results

None listed