Real-world analysis of extended half-life product posology in hemophilia A: results from a retrospective analysis of medical chart and claims data.

IF 2.3 4区医学 Q2 HEMATOLOGY

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-06-24 DOI:10.1080/17474086.2025.2520312

Cedric Hermans, John Waller, Steven R Lentz

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引用次数: 0

Abstract

Background: Extended half-life (EHL) products have changed the treatment landscape of patients with hemophilia as patients maintain protective FVIII levels with minimal occurrence of spontaneous bleeding.

Research design and methods: Two independent datasets from the multi-country patient record form (PRF)-based study and PicnicHealth/Komodo Health study were analyzed to understand the real-world use of EHL products for patients with hemophilia A (HA).

Results: Most patients receiving EHL prophylaxis were on per-label dosing. Those receiving individualized dosing had moderate to severe disease. EHL dose adjustment occurred in 20% of patients of which 14.3% experienced a second dose adjustment. Treatment efficacy was mainly monitored via annualized bleeding rate (ABR) with quality of life, tolerability, and treatment efficacy being the primary considerations for Healthcare Personnel when selecting treatment. Switching to a different EHL or standard half-life (SHL) product did not significantly reduce median ABR while switching to non-factor therapy significantly reduced median ABR from 5.2 to 0.94, p < 0.0001. Patients on individualized dosing had higher ABRs than those on per-label dosing both before index EHL treatment and while on EHL treatment, whereas individualization via dose adjustment was associated with significant median ABR reduction (p < 0.0001).

Conclusions: Individualization may support improved outcomes in patients unable to achieve satisfactory outcomes on a per-label dosing regimen.

查看原文本刊更多论文

血友病a中延长半衰期产品病理学的实际分析：来自医疗图表和索赔数据的回顾性分析结果。

背景：延长半衰期（EHL）产品改变了血友病患者的治疗前景，因为患者维持保护性FVIII水平，自发性出血发生率最低。研究设计和方法：分析了来自多国患者病历表（PRF）研究和PicnicHealth/Komodo Health研究的两个独立数据集，以了解EHL产品在血友病A （HA）患者中的实际使用情况。结果：大多数接受EHL预防的患者都是按标签给药。接受个体化给药的患者患有中度至重度疾病。20%的患者发生了EHL剂量调整，其中14.3%的患者经历了第二次剂量调整。治疗效果主要通过年化出血率（ABR）监测，生活质量、耐受性和治疗效果是医护人员选择治疗时的主要考虑因素。切换到不同的EHL或标准半衰期（SHL）产品并没有显著降低中位ABR，而切换到非因素治疗将中位ABR从5.2显著降低到0.94,p。结论：个体化可能支持改善在按标签给药方案中无法获得满意结果的患者的预后。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Expert Review of Hematology HEMATOLOGY-

CiteScore

4.70

自引率

3.60%

发文量

审稿时长

6-12 weeks

期刊介绍： Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.