Patient-reported Outcomes in Subjects Treated With ReFacto AF Routine Prophylaxis

Withdrawn

• Conditions: Hemophilia A
• Interventions: Drug: Refacto AF

• Registration Number: NCT02718677
• Lead Sponsor: Pfizer

Brief Summary

This NIS aims to assess the patient-reported outcomes (PROs) in enrolled subjects

Detailed Description

The design of the study is observational: both prospective and retrospective, national, multicenter, non- interventional. During this observational study, subjects diagnosed with severe or moderately severe (with severe phenotype) hemophilia A receiving routine prophylaxis treatment with ReFacto AF as per local standard clinical care, and in line with local labeling, are to be observed for 12 months.

The primary objective is to assess the health-related quality of life in subjects treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months.

The secondary objectives are:

* To assess the subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months;

* To assess the treatment satisfaction of caregivers of children and subjects above 18 years treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months;

* To determine in routine clinical practice the mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis;

* To describe the status of joint health measured by HJHS before and after starting routine prophylaxis for subjects above 18 years.

Study Timeline

• Study First Submitted: 2016-02-22
• Study First Submitted QC: 2016-03-18
• Study First Posted: 2016-03-24
• Study Start: 2019-09-01
• Status Verified: 2020-11
• Last Update Submitted: 2020-11-12
• Last Update Posted: 2020-11-16
• Primary Completion: 2021-05-01
• Study Completion: 2022-01-01

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: Male
• Target Recruitment: Not specified

Inclusion Criteria

1. Severe (FVIII: C <1%) or moderately severe (FVIII: C ≥1 - ≤2% with severe phenotype [at least 4 spontaneous bleeds clinically documented during a 6 months period] and without inhibitors) male subjects with hemophilia A receiving routine prophylaxis treatment.

2. Age:

   • From 2 to 18 years;
   • Subjects above 18 years who began routine prophylaxis treatment in childhood (and were part of the national prophylaxis program when turned 18 years).

3. Previously treated patients (PTPs) with hemophilia A. Previously treated patient (PTP) is considered in this study as a subject who has at least 50 exposure days to any FVIII product. An exposure day (ED) is a 24-hour period during which a dose of FVIII concentrate has been administered, irrespective of size and frequency.

4. No history and no current FVIII inhibitor defined as a titer ≥0.6 BU/mL or any measured Bethesda inhibitor titer greater than the upper limit of normal for the laboratory performing the assay.

5. Subjects who are scheduled by their treating physician to initiate prophylaxis with ReFacto AF or to continue previously initiated prophylaxis with ReFacto AF, and subjects who are switching from prophylaxis with another FVIII product to ReFacto AF.

6. Evidence of a personally signed and dated informed consent and assent (for children 6-17 years of age) document indicating that the subject (or a legally acceptable representative) has been informed of all pertinent aspects of the study.

Main

Exclusion Criteria

1. Subject has known hypersensitivity to the active substance or any of the excipients.
2. Subject has known allergic reaction to hamster proteins.
3. Presence of any bleeding disorder in addition to hemophilia A.
4. Treatment with any investigational agent or device within the past 30 days.
5. Any other contraindications according to Summary of Product Characteristics (SPC).
6. Unsuitable to participate in study for any other reason as assessed by the investigator.
7. Subjects (or a legally acceptable representative) not able to understand study documents and study procedure.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
1. Refacto AF (NIS)	Refacto AF	Non-Interventional Study

Primary Outcome Measures

Name	Time	Method
The health-related quality of life in subjects treated with ReFacto AF routine prophylaxis	Baseline and after 6 and 12 months.	HRQoL will be assessed by applying the self-reported hemophilia-specific quality of life Haemo-QoL questionnaire.; The endpoint will include the domains and total scores of the questionnaire. The results of this study will be presented using descriptive statistics and there will be no hypothesis testing. The domain scores and total scores will be summarized by: N, mean (sd), median, min-max and the 95% confidence interval at baseline, end of study, and the change from baseline.
The health-related quality of life	Baseline and after 6 and 12 months.	HRQoL will be be evaluated also by recording the number of days of absence from kindergarten, school or high-school. There are no a priori hypotheses specified and results will be presented as descriptive statistics.

Secondary Outcome Measures

Name	Time	Method
The subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis	Baseline and after 6 and 12 months;	Subjects will be asked to report their subjective physical functioning using the HEP-Test-Q.; The endpoint will include the domains and total scores of the questionnaire. The results of this study will be presented using descriptive statistics and there will be no hypothesis testing. The domain scores and total scores will be summarized by: N, mean (sd), median, min-max and the 95% confidence interval at baseline, end of study, and the change from baseline.
The treatment satisfaction of caregivers of children and subjects above 18 years treated with ReFacto AF routine prophylaxis	Baseline and after 6 and 12 months;	Caregivers will evaluate their satisfaction with the treatment of their child and the above 18 years subjects will evaluate their own satisfaction with the treatment using the Hemo-Satp questionnaire.; The endpoint will include the domains and total scores of the questionnaire. The results of this study will be presented using descriptive statistics and there will be no hypothesis testing. The domain scores and total scores will be summarized by: N, mean (sd), median, min-max and the 95% confidence interval at baseline, end of study, and the change from baseline.
The mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis;	After 12 months	ABR will be calculated at the end of the study based on the number of bleeds recorded during the 12-months observation period. The ABR will be presented as N, mean (sd), median, min-max and the 95% confidence interval.
Joint health for subjects above 18 years	After 12 months	Status of joint health will be described for subjects above 18 years using the HJHS (Hemophilia Joint Health Score) before and after starting routine prophylaxis. HJHS for one particular joint (elbow, knee or ankle) represents the sum between the joint totals and the global gait score.The minimum score is 0 and the maximum is 24.; The joint totals is the sum between the scores for swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain and strength; the maximum score is 20.; The global gait score evaluates walking, stairs, running and hopping in one leg; the maximum score is 4.