Aggarwal Sunita, Tomar Anshul, Kumar Kapil, Kumar Ankitesh, Kumar Suresh, Garg Sandeep, Kumar Naresh, Nandi Sudipta
Department of Medicine, Lok Nayak Hospital, New Delhi, India.
Indian J Pharmacol. 2024 Sep 1;56(5):312-316. doi: 10.4103/ijp.ijp_333_24. Epub 2024 Dec 16.
Very low-dose prophylaxis of antihemophilic factor (AHF) in hemophilia involves administration of minimal amounts of clotting factor concentrates, typically below standard doses, to maintain baseline levels and reduce bleeding episodes. This method uses smaller, more frequent doses to balance bleeding prevention and cost-effectiveness. Close monitoring ensures adequate protection while avoiding overtreatment, offering a tailored, cost-effective approach to improve the quality of life (QoL) of the hemophiliacs.
The study aims to study the bleeding risks in patients with severe hemophilia who are on very low-dose AHF prophylaxis. It also seeks to assess the annual consumption of AHF in the hemophiliacs. In addition, the research will determine the QoL in both groups using the social functioning (SF)-36 questionnaire,evaluate patient compliance with the prophylactic regimen, and check functional disability using the Functional Independence Score in Hemophilia (FISH).
The patients selected for the study were diagnosed with cases of hemophilia and were planned for any surgical procedure. The study was a cross-sectional study lasting 12 months. Patients over 13 years were included and patients with any other bleeding tendencies were excluded from the study.
The differences found in the study, were significant in terms of annual bleed rate (ABR), amount of AHF consumed, SF-36, and FISH scores.
Our study found that very low-dose prophylaxis significantly reduced the ABR in hemophilia patients. Functional independence worsened in the control group but remained stable in the test group, which showed significant improvements in SF-36 domains. The test group had no inhibitor development and showed a decline in severe bleeds, proving the efficacy and superiority of low-dose prophylaxis over on-demand therapy.
血友病患者使用极低剂量的抗血友病因子（AHF）进行预防，是指给予极少量的凝血因子浓缩物，通常低于标准剂量，以维持基线水平并减少出血发作。这种方法使用较小且更频繁的剂量来平衡出血预防和成本效益。密切监测可确保提供足够的保护，同时避免过度治疗，提供一种量身定制的、具有成本效益的方法来提高血友病患者的生活质量（QoL）。
本研究旨在研究接受极低剂量AHF预防治疗的重度血友病患者的出血风险。它还旨在评估血友病患者AHF的年消耗量。此外，该研究将使用社会功能（SF）-36问卷确定两组患者的生活质量，评估患者对预防方案的依从性，并使用血友病功能独立性评分（FISH）检查功能残疾情况。
入选本研究的患者被诊断为血友病，并计划进行任何外科手术。该研究为一项为期12个月的横断面研究。纳入13岁以上的患者，排除有任何其他出血倾向的患者。
研究中发现的差异在年出血率（ABR）、AHF消耗量、SF-36和FISH评分方面具有统计学意义。
我们的研究发现，极低剂量预防显著降低了血友病患者的ABR。对照组的功能独立性恶化，但试验组保持稳定，试验组在SF-36领域有显著改善。试验组未出现抑制剂产生，且严重出血有所减少，证明了低剂量预防相对于按需治疗的有效性和优越性。
