Background: Glanzmann thrombasthenia is associated with abnormalities in the glycoprotein IIb/IIIa receptor. This study, conducted at Ampang Hospital, Malaysia, aimed to assess outcomes of blood management strategies for Glanzmann thrombasthenia. Methods: Ten patients with Glanzmann thrombasthenia aged 9 years (2009‒2018) were examined.Data on clinical characteristics, transfusion practices, and patient blood management were obtained from medical records. Patient blood management methods included parenteral iron, erythropoietin, hormonal pills, intrauterine progesterone contraceptive devices, tranexamic acid, and recombinant factor VIIa. Primary outcomes were hemoglobin levels and the proportion of patients who received blood transfusion. Secondary outcomes were morbidity and mortality. Results: The median age at diagnosis was 8.2 years (range, 1‒15 yr). The female-to-male ratio was 9:1. Eight patients had type 2 disease (5‒20% of normal GPIIb/IIIa), and two patients had type 1 disease (normal GPIIb/IIIa ＜5%). All patients had iron deficiency. All female patients presented with significant menorrhagia. Other bleeding symptoms included epistaxis, spontaneous skin bruising, hemoptysis, gingival bleeding, knee hemarthrosis, and pelvic hematoma. No patient experienced life-threatening bleeding. Our patients had a mean hemoglobin level of 5.6 g/dL at diagnosis. All patients were optimized using non-transfusion methods as described above. Our patient had a current mean hemoglobin level of 11 g/dL. Approximately 70% (7/10) of patients did not experience receiving blood transfusions in the last 5 years. No patient experienced non-transfusion-related morbidities such as sepsis, thromboembolism, or cardiorespiratory events. Conclusion High cost, transfusion-related adverse events, and immunomodulation could be effectively prevented by avoiding unnecessary blood transfusions.

Anaemia is a common condition in Malaysia, and is mostly due to iron deficiency. In many cases, allogeneic blood transfusion (ABT) is administered unnecessarily to treat anaemia. Patient blood management (PBM) is a concept whereby a patient becomes his or her “own blood bank”, instead of receiving ABT. The concept encompasses three pillars namely optimising erythropoiesis, minimising blood loss and harnessing human physiological reserve. We present a safe and fruitful outcome of managing severe anaemia without utilising any ABT, made possible with the PBM approach including administration of intravenous iron.

Adolescent ; Adult ; Aged ; Anxiety ; psychology ; Blood Transfusion ; Cross-Sectional Studies ; Depression ; psychology ; Female ; Health Status ; Humans ; Linear Models ; Male ; Middle Aged ; Multivariate Analysis ; Quality of Life ; Severity of Illness Index ; Surveys and Questionnaires ; Young Adult ; beta-Thalassemia ; psychology ; therapy

BACKGROUND: Thrombotic microangiopathy (TMA) with non-deficient ADAMTS-13 (a disintegrin-like and metalloprotease with thrombospondin type 1 motif 13) outcome is unknown hence the survival analysis correlating with ADAMTS-13 activity is conducted in Malaysia. METHODS: This was a retrospective epidemiological study involving all cases of TMA from 2012–2016. RESULTS: We evaluated 243 patients with a median age of 34.2 years; 57.6% were female. Majority of the patients were Malay (62.5%), followed by Chinese (23.5%) and Indian (8.6%). The proportion of patients with thrombotic thrombocytopenic purpura (TTP) was 20.9%, 72.2% of which were acquired while 27.8% were congenital. Patients with ADAMTS-13 activity ≥5% had a four-fold higher odds of mortality compared to those with ADAMTS-13 activity <5% (odds ratio: 4.133, P=0.0425). The mortality rate was 22.6% (N=55). Most cases had secondary etiologies (42.5%), followed by acquired TTP (16.6%), atypical hemolytic uremic syndrome (HUS) or HUS (12.8%) and congenital TTP (6.4%). Patients with secondary TMA had inferior overall survival (P=0.0387). The secondary causes comprised systemic lupus erythematosus (30%), infection (29%), pregnancy (10%), transplant (8%), malignancy (6%), and drugs (3%). Transplant-associated TMA had the worst OS (P=0.0016) among the secondary causes. Plasma exchange, methylprednisolone and intravenous immunoglobulin were recorded as first-line treatments in 162 patients, while rituximab, bortezomib, vincristine, azathioprine, cyclophosphamide, cyclosporine, and tacrolimus were described in 78 patients as second-line treatment. CONCLUSION: This study showed that TMA without ADAMTS-13 deficiency yielded inferior outcomes compared to TMA with severeADAMTS-13 deficiency, although this difference was not statistically significant.
Asian Continental Ancestry Group ; Atypical Hemolytic Uremic Syndrome ; Azathioprine ; Bortezomib ; Cyclophosphamide ; Cyclosporine ; Epidemiologic Studies ; Female ; Humans ; Immunoglobulins ; Lupus Erythematosus, Systemic ; Malaysia* ; Methylprednisolone ; Mortality ; Plasma Exchange ; Pregnancy ; Purpura, Thrombotic Thrombocytopenic ; Retrospective Studies ; Rituximab ; Tacrolimus ; Thrombospondins ; Thrombotic Microangiopathies* ; Vincristine

Antiphospholipid antibodies (aPL) are autoantibodies that attack phospholipid through anti-beta 2-glycoprotein 1. The actions of aPL are associated with events leading to thrombosis and morbidity in pregnancy. Antiphospholipid syndrome (APS) is diagnosed when a patient is persistently positive for aPL and also has recognised clinical manifestations such as recurrent pregnancy losses, arterial or venous thrombosis and in a catastrophic case, can result in death. Unfortunately, the pathogenesis of APS is still not well established. Recently, microRNA expressed in many types of diseased tissues were claimed to be involved in the pathological progression of diseases and has become a useful biomarker to indicate diseases, including APS. Objective: This systematic review aims to search for research papers that are focussing on microRNA expression profiles in APS. Method: Three search engines (Ebcohost, ProQuest and Ovid) were used to identify papers related to expression of specific microRNA in antiphospholipid syndrome. Results and Discussion: A total of 357 papers were found and screened, out of which only one study fulfilled the requirement. In this particular study blood samples from APS patients were tested. The microRNAs found to be related to APS were miR-19b and miR-20a. No data was found on specific microRNA being expressed in obstetric antiphospholipid syndrome. Analysis on the microRNA target genes revealed that most genes targeted by miR-19b and miR-20a involve in TGF-Beta Signalling and VEGF, hypoxia and angiogenesis pathways. Conclusion: In view of the limited data on the expressions of microRNA in APS we recommend further research into this field. Characterization of microRNA profile in blood as well as in placenta tissue of patients with APS could be useful in identifying microRNAs involved in obstetric APS.

Background: The PLASMIC score is a convenient tool for predicting ADAMTS13 activity of ＜10%.Lactate dehydrogenase (LDH) is widely used as a marker of haemolysis in thrombotic thrombocytopenic purpura (TTP) monitoring, and could be used as a replacement marker for lysis. We aimed to validate the PLASMIC score in a multi-centre Asia Pacific region, and to explore whether LDH could be used as a replacement marker for lysis. Methods: Records of patients with thrombotic microangiopathy (TMA) were reviewed. Patients’ ADAMTS13 activity levels were obtained, along with clinical/laboratory findings relevant to the PLASMIC score. Both PLASMIC scores and PLASMIC-LDH scores, in which LDH replaced traditional lysis markers, were calculated. We generated a receiver operator characteristics (ROC) curve and compared the area under the curve values (AUC) to determine the predictive ability of each score. Results: 46 patients fulfilled the inclusion criteria, of which 34 had ADAMTS13 activity levels of ＜10%. When the patients were divided into intermediate-to-high risk (scores 5‒7) and low risk (scores 0‒4), the PLASMIC score showed a sensitivity of 97.1% and specificity of 58.3%, with a positive predictive value (PPV) of 86.8% and negative predictive value (NPV) of 87.5%. The PLASMIC-LDH score had a sensitivity of 97.1% and specificity of 33.3%, with a PPV of 80.5% and NPV of 80.0%. Conclusion Our study validated the utility of the PLASMIC score, and demonstrated PLASMIC-LDH as a reasonable alternative in the absence of traditional lysis markers, to help identify high-risk patients for treatment via plasma exchange.

Introduction/Objective: The management of potential treatment-related complications and bleeding events in haemophilia is challenging in developing countries. Providing optimal care among these patients improve their quality of life (QOL) and life expectancy. This study explores the demographic characteristics and treatment outcome in a major haemophilia treatment centre in Malaysia. 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.

Haematological cellular structures may be elucidated using automated full blood count (FBC) analysers such as Unicel DxH 800 via cell population data (CPD) analysis. The CPD values are generated by calculating volume, conductivity, and five types of scatter angles of individual cells which would form clusters or populations. This study considered 126 CPD parameter values of 1077 healthy Malaysian adults to develop reference intervals for each CPD parameter. The utility of the CPD reference interval established may range from understanding the normal haematological cellular structures to analysis of distinct cellular features related to the development of haematological disorders and malignancies.