A Review of Long-term Efficacy by Different Therapeutic Approaches on Chinese Severe Haemophiliacs

This abstract has open access
Abstract Summary
Abstract ID :
Submission Type
Presentation Upload :
If the file does not load, click here to open/download the file.
Authors (including presenting author) :
Fong NFT(1)(2), Kho BCS(1), Lao WC(1), Chan, GCF(2)
Affiliation :
(1) Department of Medicine, Pamela Youde Nethersole Eastern Hospital, (2) Department of Paediatrics & Adolescent Medicine, LKS Faculty of Medicine, The University of Hong Kong
Introduction :
There is a lack of study reporting the comparisons of intermittent-dose or high-dose (primary and secondary) prophylaxis versus on-demand therapy of our local haemophilic population with moderate-to-severe diseases under the public health care system. The long-term efficacies of primary and secondary radiosynovectomy on them also remain elusive.
Objectives :
(1) Compare the efficacies of prophylaxis and on-demand therapy on reducing bleeding and preventing other complications of our local haemophiliacs, (2) Study the non-bleeding duration of recurrent haemarthrosis achieved by primary and secondary radiosynovectomy
Methodology :
Methodology: A retrospective, case-controlled, cross-sectional study was conducted to investigate the above issues. The study was divided into two parts - Part A and B. Part A compared the annualised bleeding rate (ABR) of patients on prophylaxis (n=21) versus on-demand treatment (n=5). Secondary outcomes were prevention of complications, e.g. haemophilia arthropathy and major bleeds. Part B investigated the mean interval of recurrent haemarthrosis after primary (1st) and secondary (2nd) radiosynovectomy (n=13 and n=5, respectively). Recurrent haemarthrosis was defined as more than 3 breakthrough joint bleeds within 2 months. It was an important indicator in reflecting the status of haemophilic arthropathy. It also suggests an annulment of previous radiosynovectomy necessitating another radiosynovectomy. Time-to-event analysis was presented by Kaplan-Meier curve.
Result & Outcome :
Results: Part A: A significant difference was found in ABR between prophylaxis and on-demand groups (4.7±4.8 vs 51.6±59.6 bleeds/year, 95% CI: 2.24-23.2, p=0.0002). The number needed to treat (NNT) of prophylaxis for haemophilic arthropathy was 4 with absolute risk reduction (ARR) of 26.7 % (95% CI: - 20.7%-74.1%). The NNT of prophylaxis for major bleeds was 5 with ARR of 23.8 % (95% CI: 5.6%-42%). Part B: The mean interval of a recurrent haemarthrosis after 1st radiosynovectomy was 23.1 ± 26.8 months, which was in line with our pre-set null hypothesis. However, recurrent benefit of 2nd radiosynovectomy was very transient and the interval of rebleeding was only 4.6 ± 3.04 months, which was remarkably shorter than expected. Conclusion: Our results were consistent with the literature. As expected, prophylaxis significantly reduced bleeding and prevented complications in severe haemophiliacs. Prophylaxis should become a standard practice in our local setting. The effect of 1st radiosynovectomy lasted for approximately 24 months but 2nd radiosynovectomy only lasted for 5 months which was much shorter than anticipated duration. Earlier intervention may yield a better result. Our results provided valuable reference for the haemophilia care in Hong Kong.
Registered nurse

Similar Abstracts by Type

Abstract ID
Abstract Title
Abstract Topic
Submission Type
Primary Author
Clinical Safety and Quality Service I
HA Staff
Maria SINN Dr
Enhancing Partnership with Patients and Community
HA Staff
Donna TSE
Enhancing Partnership with Patients and Community
HA Staff
Clinical Safety and Quality Service II
HA Staff