As future doctors, we know that this duty requires us to learn for the rest of our lives. We must keep updated on every single piece of information—the newest management, diagnosis, and many more. That is why AMSA International held a webinar about the Current View on Haemophilia Work-Up and Management to commemorate World Haemophilia Day 2021.
The event was held on 17 April 2021 via Zoom, with two lecture sessions and the WHD Competition results announcement on three major categories—which was divided into three presentations for each category’s 1st winner. Before the webinar session began, all participants were required to fill in the pre-test questions via Google Form.
It started with the first lecture by Dr Lorraine Marie Item, a Haemato-Oncology Paediatric Consultant at Ospital ng Maynila Medical Center, Philippines, with the topic “Diagnosis of Haemophilia: Early Recognition for a Better Future”. In the opening of the lecture, Dr Lorraine gave us pictures of Queen Victoria’s family tree—therefore, haemophilia is also called the royal blood disease. Before she discussed further, she recalled again about our body physiology of haemostasis. She also provided examples of cases and interacted with the audiences via polls. She stated four steps to approach a patient; do the complete history (such as the type and sites of the bleeding, the duration, the childhood history, etc.), physical examination (inspecting the bleeding signs), screening tests for bleeding disorders (such as blood count, platelet count, prothrombin and thrombin time, etc.), and special tests to detect the precise nature of the defect (the mixing studies, the coagulation factor assays, and the platelet aggregation studies). She said that haemophilia could be diagnosed with these events; consistent bleeding, history and physical examination for a coagulation defect, prolonged aPTT and normal PT. The factor activity level can also confirm the diagnosis, establish the type of haemophilia and assess its severity. She also gave some basic information about haemophilia—that haemophilia is the most common serious congenital coagulation deficiency and the most common coagulation deficiency with high treatment cost, and it is an X-linked recessive disorder. In addition, she also showed the genetic inheritance of haemophilia, and patients with haemophilia usually show some clinical presentations from the beginning of their lives until they are in mature age. Last but not least, Dr Lorraine also discussed the principles of management—that we need to remember to prevent future bleeding (such as lifestyle modification and avoiding dangerous activities) and also treat early acute bleeds within two hours.
The second lecture was explained by Prof. Hamidah Alias, a Haemato-Oncology Paediatric Consultant at Department of Paediatric, UKM Medical Centre, Faculty of Medicine, The National University of Malaysia; with the topic “Perspective of Living with Haemophilia: Current Treatment and Patient/Caregiver Education.” Prof. Hamidah first explained about the overview of haemophilia itself, where haemophilia is a rare X-linked congenital bleeding disorder characterised by a deficiency of coagulation factor VIII called haemophilia A, and coagulation factor IX called haemophilia B. Haemophilia A is more common than haemophilia B, and any presence of joint bleeding in very young age children is a key indicator for severe haemophilia. The most common problem is recurrent joint hemarthrosis, which can cause synovial inflammation and can lead to progressive joint deformity and permanent disability.
Next, Prof. Hamidah explained the treatment goals and options of haemophilia. The treatment goals of haemophilia are to protect the patients from bleeding, reduce pain and musculoskeletal damage, and improve the overall health and quality of the patients’ life. Management of haemophilia itself consists of five important points. First, the management should encompass family-centred care provided by multidisciplinary experts and an experienced team involved in the diagnosis, treatment, and complication management. Second, procurement of the missing clotting factors, which can be Factor VIII and Factor IX, where all countries should maintain a sustained availability of clotting factor concentrates (CFCs) by establishing a national tender system for the procurement and distribution of haemophilia therapies. Third, deliver standard care for all severe haemophilia patients by giving a CFCs prophylaxis or other haemostasis products to prevent bleeding in young children (before 3 years old). When selecting any haemostasis products, evaluation of product safety, quality, purity, viral inactivation, and efficacy are required. There are many types of haemostasis products, including Clotting Factor Concentrates (CFCs), bypassing agents, cryoprecipitate and FFP, desmopressin, tranexamic acid, factor VIII mimetic (emicizumab), anti TFPI antibodies (concizumab), and anti-thrombin inhibitor (fitusiran). So far, CFCs are the treatment of choice in patients with haemophilia. CFCs can be administered as a prophylaxis to prevent joint and muscle damage, can be administered in demand when bleeding occurs, and can be used for surgery or treatment of severe bleeding which require prolonged replacement therapy. Fourth, home therapy and self-management monitored by the HCC team are important to improve the quality of life of the patients. Home therapy includes recognition of bleeds and complications, first aid measures, dosage calculations, CFCs storage and preparation, aseptic techniques, injection and infusion skills, proper storage and disposal of needles or any sharp objects, and blood spills handling. Last but not least, systematic surveillance for inhibitors which encompass inhibitor screening is required. The earlier the management, the better the outcome will be. Assessing outcomes of patients can be done in three ways, including assessment of annual bleeding rate (ABR), annual haemophilia joint health scores (HJHS), and ultrasound of knees, ankles, and elbows when feasible.
After the lecture session, Prof. Hamidah provided case presentations to be solved by all participants. All webinar participants answered the quiz actively. Next, the participants were required to fill in the post-test and their feedback in a Google Form link. Following the lecture session was the announcement of the winners of the World Haemophilia Day Competition by the Director of Academics. The winners are as follows:
Scientific Paper
First Place
Kohar K, et al
AMSA Indonesia
Recombinant versus Plasma-derived Factor VIII Concentrates on Inhibitor Development in Previously Untreated Patients with Haemophilia: A 2021 Update of Systematic Review and Meta-analysis
Second Place
Penantian RM, et al
AMSA Indonesia
Exploring the Efficacy and Safety of Factor VIII-Mimetic Function of Humanized Bispecific Antibody (Emicizumab/ACE910) in Classic Haemophilia Patients with or without Inhibitors: A Systematic Review and Meta-Analysis
Third Place
El Milla NF, et al
AMSA Indonesia
Efficacy and Safety of Emicizumab for Treatment in Mild Haemophilia: A Systematic Review
Scientific Poster
First Place
Milla CC, et al
AMSA Indonesia
Oral Bioencapsulated Coagulation Factor IX Fused with Cholera Toxin Beta-Subunit Protein as a Promising Immune Tolerance Induction Therapy for Haemophilia B: A Systematic Review
Second Place
Asyura MM, et al
AMSA Indonesia
Effectiveness of Low-dose FVIII Prophylaxis as an Alternative Management in Resource-limited Settings for Haemophilia A in Paediatric Population: A Systematic Review and Meta-analysis
Third Place
Vighneshwara DV, et al
AMSA Indonesia
Effectiveness of Low-dose FVIII Prophylaxis as an Alternative Management in Resource-limited Settings for Haemophilia A in Paediatric Population: A Systematic Review and Meta-analysis
Public Poster
First Place
Dyson NG, et al
AMSA Indonesia
”TREAT THEM SPECIAL”: A Practical Guide for Parents To Educate Their Children with Haemophilia
Second Place
Firdaus IZ, et al
AMSA Indonesia
Safer Hemophilia with #HFA (Haemophilia for All)
Third Place
Kurnia K, et al
AMSA Indonesia
Haemophilia: Reject The MYTHS, Remember The FACTS
Congratulations to all the winners of the World Haemophilia Day Competition! We hope that this event could motivate us all to do more research and action towards our society in the future.
Written by:
Adeela Sandria Fitri Aini
Chief Editor of eNewsletter AMSA International
Stefanie Belinda
eNewsletter Editorial Board AMSA Internatiomal