Bayer AG, Wuppertal, Germany.
Bayer, Whippany, New Jersey, USA.
Haemophilia. 2023 Jul;29(4):1142-1149. doi: 10.1111/hae.14813. Epub 2023 Jun 19.
Inhibition of tissue factor pathway inhibitor (TFPI) is a potential new mode of action to achieve haemostasis in haemophilia A and B patients.
Knowledge about potential developmental changes of TFPI levels during childhood are a prerequisite to translate adult doses of TFPI inhibitors to doses in paediatric patients.
In this study we present longitudinal data for total TFPI concentrations (TFPI-T) and TFPI activity (TFPI-A) from 48 paediatric Haemophilia A patients in the age range from 3 to 18 years (2-12 observations per patient).
TFPI-T and TFPI-A tend to decrease over age during childhood. Lowest values were observed between 12 and <18 years. On average, TFPI-T and TFPI-A were lower in adolescent haemophilia patients than in adult haemophilia patients.
In summary, the presented information on TFPI levels in children adds to the current knowledge of developmental haemostasis and it can be helpful in evaluating how children respond to haemophilia treatment including the new class of anti-TFPI compounds.
抑制组织因子途径抑制剂（TFPI）是实现血友病 A 和 B 患者止血的一种新的潜在作用模式。
了解儿童期 TFPI 水平的潜在发育变化是将成人剂量的 TFPI 抑制剂转化为儿科患者剂量的前提。
本研究提供了 48 例年龄在 3 至 18 岁（每个患者 2-12 个观察值）的血友病 A 患儿的总 TFPI 浓度（TFPI-T）和 TFPI 活性（TFPI-A）的纵向数据。
TFPI-T 和 TFPI-A 在儿童期随年龄增长而降低。在 12 至<18 岁之间观察到最低值。平均而言，青少年血友病患者的 TFPI-T 和 TFPI-A 低于成年血友病患者。
总之，本研究提供的儿童 TFPI 水平信息增加了对发育性止血的现有认识，有助于评估儿童对包括新型抗 TFPI 化合物在内的血友病治疗的反应。
