Changes in Physical Activity of Pediatric Patients Treated With Damoctocog Alfa Pegol During the Alfa-PROTECT Clinical Trial
Author(s)
Izabella Lunk, Msc, Daphne Arzoumanidou, Msc.
Bayer Consumer Care AG, Basel, Switzerland.
Bayer Consumer Care AG, Basel, Switzerland.
OBJECTIVES: Regular exercise that strengthens muscles and improves joint-stability, can be especially beneficial for the health of pediatric hemophilia patients, by helping to reduce the risk of bleeds and improving quality of life. However, fear of injury and bleeds can prevent patients from performing higher intensity activities. The Alfa-PROTECT trial demonstrated the safety and efficacy of damoctocog alfa pegol in 7-<12 -year-old patients with hemophilia A. The aim of this research is to characterize the changes in physical health and activity levels of patients treated with damoctocog alfa pegol during the Alfa-PROTECT trial.
METHODS: Haemo-QoL short-form questionnaire was used in the Alfa-PROTECT phase 3, multinational, open-label study, measuring changes in quality of life (QoL) of patients treated with damoctocog alfa pegol, including relevant dimensions of Physical health and Sports. Furthermore, a qualitative questionnaire-based study (VOICE) was conducted with caregivers of patients treated in Alfa-PROTECT. The questionnaire focused on burden of disease for patient and caregiver, including activity levels of the patient.
RESULTS: QoL data was available for 33 patients. The mean reduction in impairment was 13.51 points on the Physical health dimension, 10.28 points reduction on the Sports dimension and 7.46 points in the Feelings dimension. The VOICE study interviewed 7 parents of 8 children. During the trial, patients increased their weekly high-intensity activities by +3.2 activities and +110 minutes on average, their medium-intensity activities by +0.6 activities and +35.4 minutes and their low-intensity activities by +0.8 activities and +45 minutes on average. 100% of caregivers reported improvement in the patient’s physical status and 88% reported improved or similar emotional state of the patient as before initiating the trial.
CONCLUSIONS: Patients reported improvements in their overall physical and emotional health after initiating treatment with damoctocog alfa pegol. Intensity and frequency of sport activities increased as well, suggesting confidence in the bleed-prevention of the treatment.
METHODS: Haemo-QoL short-form questionnaire was used in the Alfa-PROTECT phase 3, multinational, open-label study, measuring changes in quality of life (QoL) of patients treated with damoctocog alfa pegol, including relevant dimensions of Physical health and Sports. Furthermore, a qualitative questionnaire-based study (VOICE) was conducted with caregivers of patients treated in Alfa-PROTECT. The questionnaire focused on burden of disease for patient and caregiver, including activity levels of the patient.
RESULTS: QoL data was available for 33 patients. The mean reduction in impairment was 13.51 points on the Physical health dimension, 10.28 points reduction on the Sports dimension and 7.46 points in the Feelings dimension. The VOICE study interviewed 7 parents of 8 children. During the trial, patients increased their weekly high-intensity activities by +3.2 activities and +110 minutes on average, their medium-intensity activities by +0.6 activities and +35.4 minutes and their low-intensity activities by +0.8 activities and +45 minutes on average. 100% of caregivers reported improvement in the patient’s physical status and 88% reported improved or similar emotional state of the patient as before initiating the trial.
CONCLUSIONS: Patients reported improvements in their overall physical and emotional health after initiating treatment with damoctocog alfa pegol. Intensity and frequency of sport activities increased as well, suggesting confidence in the bleed-prevention of the treatment.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR41
Topic
Clinical Outcomes, Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Pediatrics, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)