Romadhon Pradana Zaky, Auliya Kamila, Heryana Made Oka, Erawati Ajeng Ayu, Mahdi Bagus Aulia, Suryantoro Satriyo Dwi, Putri Aditea Etnawati, Yusoff Narazah Mohd
Doctoral Program of Medical Science, Faculty of Medicine, Universitas Airlangga, Surabaya, Indonesia.
Department of Internal Medicine, Airlangga University Hospital, Surabaya, Indonesia.
J Blood Med. 2025 May 2;16:205-220. doi: 10.2147/JBM.S511906. eCollection 2025.
Studies on high-dose prophylaxis therapy using Factor VIII show promising decrease in the Annual Bleeding Rates (ABR) in hemophilia patients. However, the greater dose and frequency raise concerns on cost considerations and adherence of the patients, especially in several countries where resources are limited. Other data has proven that the low dose prophylaxis is also promising regarding the decrease of ABR. The purpose of this systematic review is to conduct a comprehensive analysis of the lower dosage formulation used for prophylaxis in hemophilia. A PubMed and Embase database search was performed based on articles that met the following criteria: written in English language and published within the last 10 years. Consequently, the following key terms were used in combination: 'high dose', 'low dose', 'recombinant', 'prophylaxis', and 'hemophilia' in different combinations. 19 articles were included for this review. 10 of the investigated papers demonstrated decrease in ABR, functional improvement of affected joints, alleviation of pain, and a better quality of life in hemophilia patients. Low dose prophylaxis has proven to significantly improve symptoms, lower ABR and preserve joint and bone health compared to episodic or on-demand treatment. Furthermore, low dose prophylaxis (LDP) was also observed to be cost-effective and more convenient in certain countries, especially in south-east Asia where resources are limited. Overall, low dose prophylaxis appears to be non-inferior in improving the overall Quality of Life in people with hemophilia, and therefore could be a beneficial alternative in countries of the south east Asian region.
关于使用凝血因子 VIII 进行高剂量预防治疗的研究表明，血友病患者的年出血率（ABR）有望降低。然而，更大的剂量和频率引发了对成本考量以及患者依从性的担忧，尤其是在一些资源有限的国家。其他数据已证明，低剂量预防在降低 ABR 方面也很有前景。本系统评价的目的是对用于血友病预防的低剂量制剂进行全面分析。基于符合以下标准的文章在 PubMed 和 Embase 数据库中进行了检索：用英语撰写且在过去 10 年内发表。因此，以下关键词以不同组合方式使用：“高剂量”、“低剂量”、“重组”、“预防”和“血友病”。本评价纳入了 19 篇文章。其中 10 篇被调查论文表明血友病患者的 ABR 降低、受累关节功能改善、疼痛减轻且生活质量提高。与按需治疗或间歇性治疗相比，低剂量预防已被证明能显著改善症状、降低 ABR 并保护关节和骨骼健康。此外，在某些国家，尤其是资源有限的东南亚地区，低剂量预防（LDP）也被观察到具有成本效益且更方便。总体而言，低剂量预防在改善血友病患者的整体生活质量方面似乎并不逊色，因此可能是东南亚地区国家的一种有益替代方案。
