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Longitudinal observations of TFPI levels in paediatric Haemophilia A patients
Stephan Schwers1, Anita Shah2, Camila Linardi2
1Bayer AG, Wuppertal, Germany.
Insights
Tissue factor pathway inhibitor (TFPI) levels decrease with age in children with haemophilia A. Lower TFPI concentrations and activity in adolescents inform paediatric treatment strategies for haemophilia.
Area of Science:
- Paediatric Haematology
- Coagulation and Haemostasis
- Pharmacodynamics
Background:
- Inhibiting tissue factor pathway inhibitor (TFPI) offers a novel therapeutic strategy for haemostasis in haemophilia A and B.
- Understanding TFPI developmental changes in children is crucial for adapting adult TFPI inhibitor dosages for paediatric use.
Purpose of the Study:
- To investigate the longitudinal changes in total TFPI concentrations (TFPI-T) and TFPI activity (TFPI-A) in children with haemophilia A.
- To establish normative TFPI levels during childhood for effective paediatric haemophilia treatment.
Main Methods:
- Longitudinal study of 48 paediatric Haemophilia A patients aged 3 to 18 years.
- Collected 2-12 observations per patient for TFPI-T and TFPI-A.
- Analyzed age-related trends in TFPI levels.
Main Results:
- TFPI-T and TFPI-A showed a decreasing trend with age throughout childhood.
- The lowest TFPI levels were observed in the 12 to <18 years age group.
- Adolescent haemophilia patients exhibited lower TFPI-T and TFPI-A compared to adult haemophilia patients.
Conclusions:
- The study provides essential data on TFPI levels in children, contributing to the understanding of developmental haemostasis.
- Findings can aid in evaluating paediatric responses to haemophilia treatments, including novel anti-TFPI therapies.
Introduction:
Inhibition of tissue factor pathway inhibitor (TFPI) is a potential new mode of action to achieve haemostasis in haemophilia A and B patients.
Aim:
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.
Methods:
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).
Results:
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.
Conclusion:
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.
