Effectiveness of emicizumab in preventing bleeding events in severe and moderate hemophilia A: A single-center experience in Bangladesh.

Emicizumab is a monoclonal antibody that bridges activated factor IX (FIX) and factor X (FX) to replace the function of missing activated factor VIII (FVIII) in hemophilia A patients irrespective of FVIII inhibitor status. This study assessed the effectiveness of emicizumab in preventing bleeding episodes in patients with hemophilia A. This observational study included patients with moderate to severe hemophilia A who were undergoing episodic FVIII replacement therapy. The primary endpoint was the difference in annualized bleeding rates (ABR) and the secondary endpoint was the difference in Hemophilia Joint Health Score (HJHS) before and after emicizumab prophylaxis. A total of 30 male hemophilia patients were included, the mean age was 16.7 (SD: ±8.1) years, and most of them had moderate hemophilia A [63.3%]. Before prophylaxis, the median ABR was 48 (interquartile range [IQR]: 35-60), and 93.3% of patients had ABR greater than eight, whereas after prophylaxis the median ABR decreased significantly (median [IQR]: 0 [0.0-0.4], p < 0.001), and 56.7% had zero bleeds. ABR was not significantly different in patient with and without FVIII inhibitors. The HJHS scores significantly improved after prophylaxis (10 vs. 2.5, p < 0.001). The bleeding events were reduced significantly (23 vs. 0.0, p < 0.001), and zero new target joints were reported after prophylaxis. Most of the patients [93.3%] did not face any serious adverse events after prophylaxis. Emicizumab prophylaxis was associated with a significantly lower rate of bleeding events among participants with hemophilia A, regardless of inhibitor status.

Clinical profile and demographic characteristics of moderate and severe hemophilia patients in a tertiary care hospital of Bangladesh.

BACKGROUND: Hemophilia is one of the commonest inherited bleeding disorders which may lead to chronic bleeding tendencies and life-long disabilities if not properly managed. Knowing the pattern of the disease aids in the prevention of disability and improvement of quality of life in hemophilia. However, there is a dearth of literature on the issue in Bangladesh. So, this study was designed to explore the frequency and site of spontaneous bleeding in moderate and severe hemophilia patients visiting in a tertiary level hospital. METHODS: This descriptive cross-sectional study was conducted at the department of Hematology and Bone Marrow Transplantation (BMT) Center in Dhaka Medical College Hospital, Dhaka between February 2020 and August 2020. A total of 44 diagnosed cases of moderate to severe hemophilia were included in the study according to inclusion criteria. A detailed inquiry of history, thorough physical examination and relevant investigations were done and were recorded in case-record form. Informed written consent was taken from patients or their guardians where appropriate. All procedures were done according to Declaration of Helsinki. After entry and checking, data was analysed using SPSS version 26. RESULTS: Out of 44 participants, 25 (56.8%) and 19 (43.2%) had moderate and severe hemophilia. Mean age of the study population was 21.31 (± 9.78) years with the majority aged between 11 and 20 years (45.5%). All sociodemographic features were similar across severity. Hemophilia A and B was found in 90.9% and 9.1%, respectively. However, all type B patients severe hemophilia making it statistically significantly different from type A (p = 0.029). The median age of first bleeding was 3.5 years and median age of first diagnosis was 5 years. Nevertheless, approximately 67.4% patients were diagnosed as a case of hemophilia at the time of their first diagnosis. The median spontaneous bleedings episodes among all patients was 32 (range: 0-97) which did not different significantly between severe and moderate patients. The most common affected (target) joint was knee joint (88.6%) followed by elbow joint (64%) among all patients. The knee joint was more commonly involved in severe than moderate disease. CONCLUSION: This study observed the variations in pattern and frequency of spontaneous bleeding in patients with hemophilia. Severe disease was more frequent in hemophilia B than A and knee joint was the most frequent site of bleeding. However, further extensive studies are recommended.

Hematopoietic Stem-Cell Transplantation in the Resource-Limited Setting: Establishing the First Bone Marrow Transplantation Unit in Bangladesh.

PURPOSE: Treatment of malignant and nonmalignant hematologic diseases with hematopoietic stem-cell transplantation (HSCT) was first described almost 60 years ago, and its use has expanded significantly over the last 20 years. Whereas HSCT has become the standard of care for many patients in developed countries, the significant economic investment, infrastructure, and health care provider training that are required to provide such a service have prohibited it from being widely adopted, particularly in developing countries. METHODS: Over the past two decades, however, efforts to bring HSCT to the developing world have increased, and several institutions have described their efforts to establish such a program. We aim to provide an overview of the current challenges and applications of HSCT in developing countries as well as to describe our experience in developing an HSCT program at Dhaka Medical College and Hospital in Bangladesh via a partnership with health care providers at Massachusetts General Hospital. RESULTS AND CONCLUSION: We discuss key steps of the program, including the formation of a collaborative partnership, infrastructure development, human resource capacity building, and financial considerations.