Project Description

Author: Morsed et al.

Summary:

Hemophilia, one of the most common inherited bleeding disorders characterized by deficiencies of clotting factors VIII and IX results in frequent and spontaneous bleeding.1 The classic form termed as hemophilia A, is caused by deficiency of factor VIII, while hemophilia B or Christmas disease is caused by deficiency of factor IX.2 Both of these diseases shared a classic X-linked pattern of inheritance, which results from the deficiency of a coagulation factor. As a result, both of the type of hemophilia affect predominantly males and females becoming carriers of the conditions.1 Acquired hemophilia is a separate non inherited condition and much rarer when compared with congenital hemophilia usually results from autoimmune related etiology.

Hemophilia is equally distributed among all ethnic groups worldwide. Global data suggest that overall prevalence of hemophilia A is 17.1 cases per 100,000 males, among which 6 cases develop severe form of hemophilia A.3 Although it is a rare condition, the number of cases has been tripled since last 20 years and most notably the higher number of cases were observed (80% hemophilic patients) in developing countries.4 In Bangladesh, there is a lack of comprehensive data regarding the prevalence of hemophilia. Acknowledging underreports, the most recent estimates suggest that Bangladesh is burdened with 2,233 patients with hemophilia.3

The most common manifestation of both types of hemophilia is bleeding due to spontaneous or traumatic hemorrhages that range from easy bruising and hemarthroses of the joint to more severe forms of intracranial or intra-abdominal bleeding.5 Among survivors, long-term disability is apparent. This may lead to poor quality of life and can affect emotional, social and physical components of patients’ well‐being.6 One of the most common complications faced by these patients is chronic pain due to frequent bleeding in joints resulting in hemarthrosis which limits their movement and daily activities which may further impact their physical and emotional health. Several studies evidenced that patients with hemophilia have a deteriorated quality of life especially patients of older age, with severe disease and frequent acute bleeding, presence of inhibitors to clotting factors, moderate-to-severe joint manifestations, etc.6–9

Being a rare disease, there is scarcity of evidence on quality of life of patients with hemophilia especially in developing countries including Bangladesh. There are hemophilia-specific scales available for measuring the health-related quality of life of these patients.10 However, there are few studies reporting real-world scenario of patients with hemophilia as, until recently, majority of the studies assessing health related quality of life of these patients had been conducted as a part of interventional clinical trials.11,12

Despite limitations, several government and nongovernment organizations in Bangladesh are running functions in to aid patients with hemophilia. The hemophilia treatment center (HTC) of Rajshahi Medical College Hospital is one of the largest HTC in the country with 287 registered patients. However, detailed evidence on characteristics of these patients as well as treatment outcome in terms of patients’ health related quality of life are still not ready at hand. To fill up the scientific gap, the study is designed to evaluate the health-related quality of life of registered hemophilia cases attending in Rajshahi Medical College Hospital with the aim to develop baseline information in this topic to aid regulatory decision-making, improve quality of care and ultimately optimize patients’ outcomes.

Status: Ongoing

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