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Published on: September 30, 2021
Unmet Needs in Hemophilia: A Narrative Review
Rabindra K Jena1, Tuphan K Dolai2, Jina Bhattacharyya3
1Professor and Bone Marrow Transplant Physician, Department of Clinical Hematology, Srirama Chandra Bhanja Medical College and Hospital, Cuttack, Odisha, India, Corresponding Author, Orcid: https://orcid.org/0000-0001-9525-3016.
This review highlights unmet needs in hemophilia management, including inadequate bleed protection and the burden of hemophilic arthropathy. Addressing these is key to improving patient quality of life and outcomes.
Area of Science:
- Hematology
- Genetics
- Public Health
Background:
- Hemophilia is a rare genetic bleeding disorder requiring lifelong, comprehensive management.
- Current treatments aim to improve life expectancy and quality of life (QoL).
- Despite progress, significant challenges remain in hemophilia care.
Purpose of the Study:
- To review the current state of hemophilia management.
- To identify and analyze unmet clinical needs in hemophilia.
- To emphasize the impact of hemophilia on patients' lives.
Main Methods:
- Literature review of hemophilia management strategies.
- Analysis of current treatment options and their limitations.
- Assessment of patient-reported outcomes and quality of life.
Main Results:
- Suboptimal bleed protection with existing prophylactic therapies.
- Development of inhibitory alloantibodies complicates treatment.
- Hemophilic arthropathy (HA) leads to joint damage and disability.
- Significant physical and psychological burden impacts education, employment, and QoL.
Conclusions:
- Innovative therapies are needed to improve bleed control.
- Addressing alloantibody development is critical.
- Individualized treatment strategies and improved access to care are essential.
- Reducing the burden of hemophilia, especially in high-prevalence regions like India, requires focused interventions.
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