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Updated: May 31, 2026

Implementation of Non-invasive Point of Care Transient Elastography for Evaluation of Liver Disease in Pediatric Populations with Cystic Fibrosis
05:56

Implementation of Non-invasive Point of Care Transient Elastography for Evaluation of Liver Disease in Pediatric Populations with Cystic Fibrosis

Published on: August 29, 2025

Comprehensive Assessment of the Transition to Efanesoctocog Alfa Prophylaxis in a Paediatric Haemophilia A Cohort

Rubén Berrueco1,2,3, Nuria Caballero1,2,3, Cristina Benedicto1,2,3

  • 1Pediatric Hematology Department, Hospital Sant Joan De Déu, Esplugues de Llobregat, Spain.

Haemophilia : the Official Journal of the World Federation of Hemophilia
|May 30, 2026
PubMed

Insights

Efanesoctocog alfa offers safe and effective prophylaxis for children with haemophilia A, improving joint health and quality of life. This novel factor VIII (FVIII) treatment demonstrates significant clinical and psychosocial benefits.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Efanesoctocog alfa is a novel recombinant factor VIII (FVIII) with an ultra-extended half-life.
  • It is approved for prophylaxis in children with haemophilia A (HA).

Purpose of the Study:

  • To describe the transition experience of pediatric patients with HA to efanesoctocog alfa.
  • To assess clinical and psychosocial outcomes using comprehensive evaluations.

Main Methods:

  • Prospective observational study in a pediatric haemophilia unit.
  • Transitioned pediatric patients (<18 years, no inhibitors) from prior prophylaxis to efanesoctocog alfa.
  • Compared treatment regimen, adherence, ABR, pharmacokinetics, joint health, QoL, and treatment burden pre- and post-transition.

Main Results:

  • Eighteen pediatric patients were included; adherence improved, and no inhibitors developed.
  • Significant increase in FVIII half-life (13.57 to 57.13 h), leading to a reduced ABR (0.39 to 0.05).
  • Improved joint health, reduced synovitis, enhanced quality of life (EQ-VAS), and decreased treatment burden were observed.

Conclusions:

  • Transitioning to efanesoctocog alfa was safe and effective in this pediatric cohort.
  • Efanesoctocog alfa offers meaningful clinical benefits, including improved joint health and reduced bleeding.
  • The therapy also provides significant psychosocial benefits, with strong patient/caregiver preference.
Abstract

Keywords:
Haemophilia Afactor VIIImonitoringpaediatricspatient preferencequality of life

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