Comparison of Joint Bleeding and Costs: On-Demand vs. Prophylaxis
Journal name: The Malaysian Journal of Medical Sciences
Original article title: A Comparison of the Incidence Rate for Joint Bleeding and FVIII Consumption between On-Demand versus Prophylaxis Factor VIII Replacement Therapy and the Direct Cost of Prophylaxis Treatment in Severe Haemophilia A Patients
The Malaysian Journal of Medical Sciences (MJMS) is a peer-reviewed, open-access journal published online at least six times a year. It covers all aspects of medical sciences and prioritizes high-quality research.
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Munirah Jamal, Jameela Sathar, Jamaliah Jamari, Shamin Mohd Saffian
The Malaysian Journal of Medical Sciences:
(A peer-reviewed, open-access journal)
Full text available for: A Comparison of the Incidence Rate for Joint Bleeding and FVIII Consumption between On-Demand versus Prophylaxis Factor VIII Replacement Therapy and the Direct Cost of Prophylaxis Treatment in Severe Haemophilia A Patients
Year: 2023 | Doi: 10.21315/mjms2023.30.1.11
Copyright (license): CC BY 4.0
Download the PDF file of the original publication
Summary of article contents:
Introduction
Haemophilia A, an inherited bleeding disorder stemming from insufficient levels of clotting factor VIII (FVIII), presents significant treatment challenges, particularly in its severe form. Patients may receive either on-demand therapy, which addresses bleeding episodes as they occur, or prophylaxis therapy, involving regular FVIII infusions to prevent such episodes. This study aimed to compare the bleeding incidence and FVIII consumption rates between adult patients undergoing on-demand therapy versus those on prophylaxis at Ampang Hospital in Malaysia.
Prophylaxis vs. On-Demand Therapy
The findings revealed that patients in the prophylaxis group experienced a significantly lower annual average of joint bleeding events, averaging 2.79 compared to 21.36 in the on-demand group (P < 0.001). Although the prophylaxis approach led to happier patients with fewer bleeding occurrences, it also resulted in a higher annual total FVIII usage of 1,506 IU/kg/year compared to 365.26 IU/kg/year for on-demand therapy (P = 0.001). This elevated FVIII consumption translates to a considerable increase in treatment costs, approximately RM786.54 annually for prophylaxis compared to RM166.68 for on-demand therapy, emphasizing the economic implications of these treatment regimens.
Conclusion
While prophylaxis was shown to significantly reduce bleeding incidents and improve patients’ quality of life, it comes with high financial burdens due to increased FVIII consumption. This study underlines the necessity for healthcare providers to weigh clinical benefits against economic factors when making treatment decisions for patients with severe haemophilia A. Future research is encouraged to focus on optimizing treatment strategies that balance efficacy and cost-effectiveness, contributing to better management of this challenging condition in resource-limited settings.
FAQ section (important questions/answers):
What is Haemophilia A and its main treatment approaches?
Haemophilia A is a bleeding disorder caused by insufficient factor VIII levels. The primary treatment methods for severe cases include on-demand therapy, which treats bleeding episodes as they occur, and prophylaxis therapy, which involves regular infusions of factor VIII to prevent bleeding.
What were the key findings of the study at Ampang Hospital?
The study compared bleeding incidence between on-demand and prophylaxis therapy in severe haemophilia A patients. Results showed significantly fewer joint bleeds in the prophylaxis group (2.79 bleeds) compared to the on-demand group (21.36 bleeds), highlighting prophylaxis as an effective treatment.
How does the cost of prophylaxis therapy compare to on-demand treatment?
Prophylaxis therapy in severe haemophilia A patients is associated with higher costs. The study found an annual cost of RM786 for prophylaxis treatment compared to RM167 for on-demand therapy, reflecting the greater usage of factor VIII in prophylaxis.
What was the adherence rate to prophylaxis therapy observed in the study?
The study reported that 87.5% of patients in the prophylaxis group adhered to their treatment regimen, defined as administering more than 80% of the prescribed factor VIII infusions, indicating a high level of commitment to this preventive approach.
Glossary definitions and references:
Scientific and Ayurvedic Glossary list for “Comparison of Joint Bleeding and Costs: On-Demand vs. Prophylaxis”. This list explains important keywords that occur in this article and links it to the glossary for a better understanding of that concept in the context of Ayurveda and other topics.
1) Bleeding:
Bleeding is a critical concern in the context of haemophilia A, a genetic disorder characterized by insufficient clotting factors. The management and treatment of bleeding episodes are central to improving patient outcomes, making bleeding a key focus in clinical studies and palliative care strategies for affected individuals.
2) Study (Studying):
The study mentioned in the text is a retrospective analysis comparing bleeding incidence rates between two treatment modalities for severe haemophilia A. Research and studies in medical fields are vital as they inform treatment protocols, evaluate efficacy and safety, and ultimately aim to enhance patient care and outcomes.
3) Table:
Tables in medical literature serve to present complex data clearly and concisely, enabling easy comparison of variables such as demographics, bleeding incidents, treatment outcomes, and other relevant statistics. They are an essential part of research publications, facilitating efficient data interpretation for clinicians and researchers.
4) Blood:
Blood is crucial in the diagnosis and management of haemophilia A, as it contains clotting factors essential for hemostasis. Disorders related to blood, like haemophilia, emphasize the need for effective treatment regimens to reduce the risk of bleeding and improve overall health outcomes for patients.
5) Disease:
The term disease refers to pathological conditions affecting the body, such as haemophilia A, which hinders normal bodily functions like blood coagulation. Understanding disease mechanisms is fundamental in medicine to develop appropriate therapies, improve quality of life, and guide public health interventions to manage healthcare burdens.
6) Life:
Life quality is significantly impacted by chronic conditions like haemophilia A. The availability and effectiveness of treatment regimens, such as prophylaxis compared to on-demand therapies, directly influence the life experience of patients, informing their daily activities, emotional well-being, and long-term health prospects.
7) Activity:
Physical activity plays a vital role in the lives of individuals with haemophilia A. Depending on their treatment and health status, patients may engage in diverse activities, which requires careful management to prevent bleeding episodes while promoting a healthy lifestyle that integrates exercise with safe practices.
8) Quality:
Quality of life is a critical outcome measure for patients with haemophilia A, as chronic illness may affect physical, emotional, and social dimensions of well-being. Effective treatments that minimize bleeding events can enhance patients' quality of life, allowing them better participation in daily and social activities.
9) Potter:
In the context of this study, 'Potter' refers to a specific research investigation, the POTTER study, comparing treatment methods for severe haemophilia A. Such studies contribute valuable insights into the effectiveness of prophylactic therapies versus on-demand treatments, driving advancements in clinical practice.
10) India:
India is mentioned in discussions of global health and management of haemophilia A, particularly in the context of developing countries. Studies from India provide insights into the challenges and opportunities in treating chronic disorders within resource-limited settings, informing public health policies and treatment strategies.
11) Developing:
Developing countries often face unique challenges in healthcare, including access to treatments for conditions like haemophilia A. Understanding these challenges is crucial to address global health disparities, improve access to therapies, and inform the design of effective health interventions tailored to the needs of such populations.
12) Srivastava (Sri-vastava, Shrivastava, Shri-vastava):
Dr. Srivastava led the guidelines for the management of hemophilia that are referenced in the study. The contributions of prominent researchers like Srivastava are vital for establishing evidence-based practices, providing frameworks for treatment approaches, and enhancing clinical care for patients with hemophilia globally.
13) Narayana (Nara-ayana, Nara-yana):
Narayana is referenced here likely in connection with research or studies conducted in India, focusing on treatment adherence and outcomes for patients with haemophilia A. Contributions from various researchers are essential for establishing effective treatment guidelines and improving healthcare delivery in this area.
14) Indian:
The term 'Indian' signifies the geographical and cultural context of the study, referencing patients, researchers, and healthcare systems involved in haemophilia treatment in India. The considerations of local practices, cultural beliefs, and resource availability are crucial for successful implementation of treatment protocols.
15) Pillai:
Pillai could refer to a co-author or a researcher involved in the study, contributing to knowledge on haemophilia management. The inclusion of various authors with diverse backgrounds fosters collaborative approaches to research, facilitating comprehensive understanding of disease management from multiple perspectives.
16) Rules:
In the context of haemophilia, 'rules' might refer to guidelines established by health organizations (e.g., WFH guidelines) regarding treatment protocols. Adhering to these rules ensures standardized care delivery, improves patient safety, and enhances the efficacy of treatment strategies across different healthcare settings.
17) Visit:
A visit to a haemophilia treatment center is vital for patients to receive ongoing medical care, monitoring, and education about their condition. Regular visits facilitate timely interventions, proper management of bleeding episodes, and improved patient engagement in their treatment plans.
18) Tai:
The inclusion of 'Thai' likely relates to studies conducted in Thailand that provide comparative insights into the management of haemophilia. Research from diverse geographical areas helps identify effective strategies tailored to specific populations and enhances understanding of global practices in haemophilia care.
19) Calculation:
Calculation in the context of the study typically refers to the quantitative analysis performed on data collected from patients. This includes statistical calculations related to bleeding incidence rates or FVIII consumption, which provide critical insights for evaluating treatment efficacy and guiding clinical decisions.
20) Discussion:
The discussion section of a research study interprets the results, contextualizes findings with existing literature, and offers insights into implications for practice. It is essential for critically analyzing the impact of research outcomes on current treatment approaches for conditions like haemophilia A.
21) Practising (Practicing):
Practising refers to the implementation of treatment protocols in real-world clinical settings. It addresses how recommendations from research are applied in daily medical practice, emphasizing the importance of ongoing education and adaptation to improve patient management outcomes for chronic conditions like haemophilia.
22) Science (Scientific):
Science underpins all medical research exploring the pathophysiology, treatment, and management of conditions like haemophilia A. Scientific inquiry drives the discovery of innovative therapies, enhances our understanding of disease mechanisms, and leads to improved clinical practices and patient outcomes in healthcare.
23) Cina:
China's mention highlights the global nature of haemophilia research, including studies that inform treatment practices. Insights from diverse countries, including China, contribute to a richer understanding of the disease's impact, management strategies, and cultural considerations in the delivery of care.
24) Hand:
In the context of haemophilia, the term 'hand' might reference physical health or dexterity. Joint health and the risk of bleeding into joints, including those in hands, are significant health concerns for affected patients, impacting daily activities and quality of life.
25) Post:
The term post may refer to a publication or communication related to the findings of the research study. Sharing results through academic or clinical posts helps disseminate knowledge among healthcare professionals, facilitating broader application of successful treatment methodologies in managing haemophilia.