Inherited Bleeding Disorders—Experience of a Not-for-Profit Organization in Pakistan

Abstract

Patient registry is a powerful tool for planning health care and setting groundwork for research. This survey reports a detailed registry of inherited bleeding disorders (IBD) and their management at a not-for-profit organization in a developing country to form the basis for planning development and research. We reviewed medical records of patients with IBD from 8 hemophilia treatment centers of Fatimid Foundation located in various cities. Information collected included sociodemographic data, diagnostic tests, severity of hemophilia A and B, number of bleeding episodes per year, site and frequency of hemarthrosis, and seropositivity for viral diseases. We analyzed 1497 patients from November 1, 2015, to April 30, 2016. There were 1296 (87%) males and 201 (13%) females with a mean age of 24.5 (11) years (range, 6 months to 65 years). Hemophilia A constituted the bulk of IBD (848, 57%) followed by von Willebrand disease (172, 11%), hemophilia B (144, 10%), platelet function defect (106, 7%), and rare bleeding disorders (70, 5%). Mucocutaneous bleeding (1144, 76%) and hemarthrosis (1035 patients, 69%) were the main complications. There were 1026 (69%) patients who received only blood components for treatment of any bleeding episode while the remaining 464 (31%) were on combination therapy (blood components and factor concentrate). Seroreactivity for hepatitis C was frequent (28%), while hepatitis B (1%) and human immunodeficiency virus (0.01%) were less commonly seen. This study was an important step toward a patient registry in a hemophilia treatment center in Pakistan. Hemophilia A is the most common bleeding disorder and hepatitis C is the most frequent treatment-related complication.

Bleeding Disorders among women Presenting with Menorrhagia at Tertiary Care Hospital

“…Our study also indicated that menorrhagia (in females of childbearing age) is a common presenting symptom observed to be present in Glanzmann thrombasthenia, which is consistent with previous findings. 20,21 Known as Bernard Soulier Syndrome, it is a hereditary platelet function abnormality that manifests as mucocutaneous haemorrhage, thrombocytopenia, and large platelets in the absence of platelet aggregation in response to Ristocetine treatment. Bernard and Soulier were the first to define BSS as having an autosomalrecessive inheritance pattern in 1948.…”

Fibrin glue in managing intractable gingival bleeding in patients with inherited bleeding disorders—a quasi-experimental pilot study

“…Pakistan, a South Asian country, faces challenges in providing haemophilia treatment due to limited haemophilia treatment centres (HTCs) and resources. Mucocutaneous bleeding, including gingival bleeding, is a common issue in IBD patients in Pakistan, 2 often requiring blood transfusions. As prophylaxis therapy is not widely available, on-demand therapy, cost-effective local haemostatic agents like Tranexamic acid, and blood transfusions are the primary approaches for managing acute bleeding episodes.…”

Management of Severe Hemophilia A: Low-Dose Prophylaxis vs. On-Demand Treatment

“…Recent studies have also investigated low-dose factor prophylaxis as an alternative economically feasible approach [8,9]. The provision of clotting factor concentrates is a frequently faced obstacle in managing Hemophilia A patients in Pakistan [10].…”