PB1256 - Escalating low-dose individualized haemophilia prophylaxis: A focus in China at Dawn of a new era of cost effective Individualized prophylaxis

Tuesday, June 25, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

Wanru Yao, MA

Doctor
Haemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education; Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, China, 100045
Beijing, Beijing, China (People's Republic)

Presenting Author(s)

Runhui Wu, PhD, MD

Professor
Department of Hematology, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
Beijing, Beijing, China (People's Republic)

Co-Author(s)

DA

Di Ai

PhD student
Beijing Children’s Hospital
Beijing, Beijing, China (People's Republic)
KH

Kun Huang

Doctor
Hematology Department II, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education; Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, China
Beijing, Beijing, China (People's Republic)
Zhenping Chen

Associate professor
Hematologic Diseases Laboratory, Hematology Center, Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, ChinaChildren’s Health, Beijing, 100045, China
Beijing, Beijing, China (People's Republic)

Co-Author(s)

KL

Koon Hung Luke

Consultant Hematology, Adjunct Professor, University of Ottawa,
Dept of Pediatrics Division of Hematology and Oncology Children Hospital of Eastern Ontario, U of Ottawa, Ottawa Ontario Canada
Ottawa, Ontario, Canada

Background: Low dose prophylaxis developed in China proved effective to reduce joint bleeding but not protect long term joint health. In 2016 research focus shifted to develop coseffective individualized prophylaxis.

Aims: This abstract reported the long term cost effective outcomes of the first three clinical trials.

Methods: The first Chinese individualized prophylaxis study launched in 2016. Priori dose escalations criteria were based on Index joint bleedings and joint assessment by Haemophilia Joint Health scores (HJHS) and ultrasound gray scale and Color Doppler. Second study was based on CHIPS but the dose escalations were adjusted by individual FVIII trough levels measured by the WAPPS- HAEM. The third study was a three follow up the Severe haemophilia A (SHA) boys from CHIPS.

Results: ( see table 1)
Bleeding Events: Significant reduction in AJBR from 5.2 to 1.2 in first CHIPS. By optimizing the escalated dose adjusted to individual FVIII tough level, AJBR reduced to 0 .02(p < 0.0001).
Dose escalation: 34 escalations in CHIPS and 16 during follow up. 5/18(29%) by index joint bleedings. 47 escalations in second CHIPS a study and 8/47(60%) by index joint bleeds.
Heterogeneous patient outcome: At exit of CHIP 5/16 on step 4 treatment and in second CHIPS 17/47(28%) consistent with 33% of high Erik patients in the CHAP a study Canada.
FVIII consumption: Mean FVIII u/kg/ year between 2900 to 3500, good saving from current yard stick protocols.
Ultrasound: minimal changes.
QoL of life of boys (CHO-KLAT sores): Great improvement from 57to 77 in CHIPS and around 80 at exit in second chips.

Conclusion(s): The escalation low dose individualize prophylaxis study is long term cost effective in significant reduce haemarthrosis and now ready for implement.