Abstract Number: PB0872
Meeting: ISTH 2020 Congress
Background: Haemophilia is a group of inherited bleeding disorders, affecting the coagulation factors responsible for haemostasis. With the prevalence of 5.1 per 100,000 population, clinical data among PwH in Malaysia are scarce.
Aims: To evaluate the socio-demographic, clinical, and treatment characteristics among PwH in our haemophilia treatment centre (HTC).
Methods: A retrospective review was conducted in Ampang Hospital, Malaysia to include patients with the diagnosis of haemophilia A (HA) and haemophilia B (HB) from 2008 to 2018. Demographic data, clinical characteristics, prophylaxis state, and treatment outcomes were evaluated.
Results: 260 patients were recruited with median age of 31 (IQR;2-84) years. The median age at diagnosis was 2 (IQR;1-73) years and all patients were male. 52.7% were Malays followed by Chinese (34.6%), and Indian (10%). 211 patients were diagnosed with HA with 72.5% of them had severe disease, while 49 patients had HB (severe disease, 65.3%). About two-third had significant family history. Bleeding events among PwH are illustrated in table 1. Chronic arthropathy and synovitis collectively accounted for more than half of the musculoskeletal complications with only 39% of them underwent intervention such as radioisotope synovectomy and arthroplasty. 30.1% of the patients had contracted hepatitis C followed by hepatitis B (1.8%), and HIV (0.8%). 12.7% of the patients developed inhibitor with 12 patients successfully underwent immune tolerance induction. Treatment with clotting factor concentrate (CFC) is illustrated in table 2. 15 patients with inhibitor were treated with on-demand/prophylaxis bypassing agents. 5 patients were treated with emicizumab and 2 patients were recruited in futisiran clinical trial.
|Annual bleeding event (mean±SD)||4.91 (±6.07)|
|Types of bleeding||N (%)|
|Musculoskeletal (At least one episode)||226 (86.9)|
|Presence of target joints||209 (80.4)|
|Psoas bleeding||41 (15.8)|
|Intracranial bleeding||38 (14.6)|
|Surgical bleeding||25 (9.6)|
|Gastrointestinal bleeding||20 (7.7)|
[Table 1: Bleeding events among PwH]
|Characteristics||Mild; n (%)||Moderate; n (%)||Severe; n (%)|
|On-demand CFC||27 (96.4)||18 (64.3)||29 (19.6)|
|Prophylaxis CFC||1 (3.6)||10 (35.7)||123 (80.4)|
|On-demand CFC||7 (100.0)||6 (60.0)||7 (21.9)|
|Prophylaxis CFC||0 (0)||4 (40.0)||25 (78.1)|
|Mean CFC||Dose (IU/kg/week)|
|PwH A||41.3 (± 19.1)|
|PwH B||48.6 (± 21.5)|
[Table 2: Treatment characteristics for PwH]
Conclusions: Despite the availability of treatment in our centre, significant proportion of patients with severe haemophilia still refused prophylaxis factor replacement. It is worthwhile to further evaluate their quality of life especially among patients with recurrent bleeding events.
To cite this abstract in AMA style:Boo YL, Liam CCK, Yong KY, Fann RJ, Lee GWC, Wilfred G, Sathar J. Clinical Review of Patients with Haemophilia (PwH) in a Malaysian Tertiary Center [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/clinical-review-of-patients-with-haemophilia-pwh-in-a-malaysian-tertiary-center/. Accessed October 2, 2023.
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