Estimated prophylactic dose required to achieve 3% trough as a function of age and concentrate class in multi-country severe WAPPS-Hemo haemophilia patients

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2024-05-13 DOI:10.1111/hae.15032

Dagmar M. Hajducek, Pierre Chelle, Alfonso Iorio, Emma Iserman, Andrea N. Edginton

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引用次数: 0

Abstract

Introduction

Web-Accessible Population–Pharmacokinetic Service–Haemophilia (WAPPS-Hemo) data are available to study factor-concentrate usage, defined as the required weekly dose to achieve a 3% trough (WD3T), across standard and extended half-life (SHL/EHL) products.

Aim

To provide baseline usage data including (i) differences across plasma-derived (pdSHL) versus recombinant (rSHL) products, (ii) SHL versus EHL, and (iii) effect of age and positive inhibitor history.

Methods

PK profiles (n = 14,416 patients, 0.3–85.2 years) and linear mixed effects models were used to estimate usage versus age, controlling for significant factors, using 95% confidence intervals to perform comparisons across all ages and posthoc tests to assess the differences.

Results

Average usage was significantly higher for pdSHL versus rSHL in patients with a positive inhibitor history (PIH; 1.9–2.5 times higher), for SHL versus EHL (4–10 times), and was significantly associated with age.

Conclusion

Baseline usage patterns from 2017 to early 2023 provide a benchmark for assessing the impact of emerging technologies in haemophilia.

Abstract Image

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多国重症 WAPPS-Hemo 血友病患者达到 3% 谷值所需的估计预防剂量与年龄和浓缩物等级的关系。

简介：网络可及人群药代动力学服务-血友病（WAPPS-Hemo）数据可用于研究标准半衰期（SHL/EHL）产品的浓缩因子用量，即达到 3% 谷值（WD3T）所需的每周剂量。目的：提供基线用量数据，包括(i)血浆来源（pdSHL）与重组（rSHL）产品的差异，(ii)SHL与EHL的差异，以及(iii)年龄和抑制剂阳性史的影响：采用 PK 资料（n = 14,416 名患者，0.3-85.2 岁）和线性混合效应模型估算用量与年龄的关系，控制重要因素，使用 95% 置信区间对所有年龄进行比较，并进行事后检验以评估差异：在有抑制剂阳性病史（PIH；高1.9-2.5倍）的患者中，pdSHL的平均用量明显高于rSHL，SHL的平均用量明显高于EHL（4-10倍），且与年龄显著相关：2017年至2023年初的基线使用模式为评估血友病新兴技术的影响提供了基准。

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

Haemophilia 医学-血液学

CiteScore

6.50

自引率

28.20%

发文量

226

审稿时长

3-6 weeks

期刊介绍： Haemophilia is an international journal dedicated to the exchange of information regarding the comprehensive care of haemophilia. The Journal contains review articles, original scientific papers and case reports related to haemophilia care, with frequent supplements. Subjects covered include: clotting factor deficiencies, both inherited and acquired: haemophilia A, B, von Willebrand''s disease, deficiencies of factor V, VII, X and XI replacement therapy for clotting factor deficiencies component therapy in the developing world transfusion transmitted disease haemophilia care and paediatrics, orthopaedics, gynaecology and obstetrics nursing laboratory diagnosis carrier detection psycho-social concerns economic issues audit inherited platelet disorders.