Clinical trial: Patient-reported Outcomes in Subjects Treated With ReFacto AF Routine Prophylaxis (NCT02718677)

Eligibility criteria:
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.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 2 Years old.
Subject must be at most 30 Years

Patient description (medical language): A 20-year-old man comes to the emergency due to bleeding after a tooth extraction.  The bleeding has persisted for approximately 30 minutes despite constant direct pressure.  He is a known case of Hemophilia type A treated with FVIII. Blood pressure is 95/60 mm Hg and pulse is 105/min. His weight is 70 Kg. Family history is positive for Hemophilia type A in his maternal uncle. He also has a lipoma on his left arm which he plans to remove surgically. His FVIII activity is 40%.

Patient description (patient's own words): I got my tooth extracted and I could not stop bleeding for 30 min. I'm a 20 yo dude but I have Hemophilia type A treated with FVIII. My blood pressure was 95/60 and my pulse 105/min. I'm 70 kg. My uncle on my mom's side also has type A hemophilia. I also have a fatty swelling on my left arm and I was supposed to remove it. My FVIII activity was 40%.

Does the patient match the trial criteria? 0