D66
Conditions
Interventions
Group 1: In this observational study, clinical and functional assessments of patients with moderate and severe haemophilia A, who have been switched to efanesoctocog alfa prophylaxis will be prospecti
Sponsors
Bergische Universität Wuppertal, Lehrstuhl für Sportmedizin
Eligibility
Sex/Gender
Male
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1) Patients suffering from moderate to severe haemophilia A 2) Age = 18 years 3) Treatment being switched to efanesoctocog alfa prophylaxis from other short half life, enhanced half life and non factor therapy prophylaxis 4) Inclusion in the study (T0 examinations) within a time frame of 6 weeks prior or post switch to efanesoctocog alfa 5) Submitted written informed consent
Exclusion criteria
Exclusion criteria: 1) Patients suffering from other bleeding disorders 2) Patients suffering from different hematological diseases like M. Bechterew, Psoriasis or other local or generalized joint infections (Borreliosis, septic arthritis) 3) Patients with inhibitors 4) Patients being treated with on-demand therapies 5) Any surgeries up to 6 months before the first examination date 6) Any planned surgeries at T0 7) Patients without written informed consent 8) Age < 18 years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1) Annual bleeding rates (ABR): To determine the frequency of all documented bleeding episodes experienced by participants after switching to efanesoctocog alfa, providing a quantitative measure of the treatment's efficacy in bleeding control. 2) Joint health assessed via ultrasound: To conduct a detailed evaluation of joint health using ultrasound and the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) protocol with the focus on inflammatory synovial changes. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Annual traumatic, spontaneous and joint bleeding rates 2) Number of Annual bleeding rates of zero 3) Factor substitution amount and frequency: To determine dosage and frequency of efanesoctocog alfa administration 4) Clinical Joint health: To evaluate the patients’ joint health using the Haemophilia Joint Health Score (HJHS) and the total HEAD-US score 5) Pain status using the numeric rating scale (NRS) 6) Semi-objective pain status assesed trough pain pressure thresholds at the most affected haemophilic joints. 7) Measurement of grip strength 8) Muscle stiffness of the M. rectus femoris assesd with myotonometry 9) Muscle thickness of the M. rectus femoris assesed with ultraound 10) Functional mobility and balance are evaluated with the Timed Up and Go (TUG) Test and the single leg stand test in single and dual tasking conditions 11) Questionnaires and surveys are used to evaluate the patients’ subjective functional, pain, and disease specific states: PROMIS Questionnaire, German Pain Questionnaire, Haemo-QoL, HEP-Test-Questionnaire, Haemophilia Activities List, Veritas-Pro Questionnaire, and semi-structured questions | — |
Countries
Germany
Contacts
Public ContactThomas Hilberg
Bergische Universität Wuppertal
Outcome results
None listed