MATERIALS AND METHODS: A total of 260 patients were recruited in this retrospective cross-sectional analysis. Clinical data, including treatment regimens and outcome, were collected and analysed.
RESULTS: A total of 211 patients were diagnosed with haemophilia A (HA) (severe disease, 72.5%) and 49 patients had haemophilia B (HB) (severe disease, 65.3%). The median age was 31 (IQR;2-84) years. Majority of the patients had at least one episode of musculoskeletal bleeding since diagnosis. The mean annual bleeding event (ABE) was 4.91 (SD±6.07) in 2018. Target joints were identified in 80.4% of the patients. Chronic arthropathy and synovitis collectively accounted for more than half of the musculoskeletal complications. 30.1% of the patients had contracted hepatitis C with less than half received treatment. Thirty-one patients (16.8%) with severe haemophilia developed inhibitor and 12 patients successfully underwent immune tolerance induction. More than three-quarters of the severe haemophilia patients were treated with factor concentrate prophylaxis. The mean prophylaxis dose for HA and HB were 41.3 (SD±19.1) and 48.6 (SD±21.5) IU/kg/week, respectively. In patients with severe disease, prophylaxis significantly reduced the ABE (5.45,9.03;p=0.005).
CONCLUSION: The importance of utilising a low to moderate dose regimen as prophylaxis in haemophilic patients is highlighted in our study. Future studies should include QOL assessment will further improve the management in haemophilia.
MATERIALS AND METHODS: A total of 119 AA patients had been identified from hospital records of the involved sites, namely Queen Elizabeth Hospital in Sabah, Sarawak General Hospital, Sibu Hospital, Miri Hospital and Bintulu Hospital in Sarawak from Jan 2006 to Dec 2017.
RESULTS: The median age at diagnosis was 46 years, and native ethnic group from Sabah, Kadazan-Dusun, recorded the highest percentage of 41.2%, which could be explained by higher frequency of HLA-DRB1*15:01, an alelle linked to increased risk of AA, among this ethnic group. The majority of patients (59.7%) received cyclosporine (CsA) as monotherapy or in combination with other non-IST agents such as danazol, which was instituted in 48.7% of the patients, while a third of them (33.7%) received antithymocyte globulin (ATG) therapy with or without CsA, and 12.4% underwent allogenic SCT. The five-year overall survival (OS) for all AA patients was 76.1%. Elderly patients >60 years old and those with severe disease had more inferior 5-year survival.
CONCLUSION: A prospective study is warranted to determine the true incidence rate, epidemiological distributions, treatment outcome and overall survival of AA patients in Malaysia. Establishment of allogenic SCT in East Malaysia is imperative to make this curative therapy more accessible to patients with severe disease and improve the outcome.