Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice. Besides aspects such as general product safety and efficacy, there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Study Type
OBSERVATIONAL
Enrollment
80
Number of Patients With FVIII Inhibitors
FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Time frame: Screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Number of Patients With Adverse Drug Reactions
Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded by patients in treatment diaries which will be reviewed at each Follow-up Visit.
Time frame: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment
Total number of bleeding episodes under prophylaxis treatment divided by the duration of prophylactic phase (in years)
Time frame: Monitored throughout the study from screening through to study completion (minimum 3.7 months; maximum 21.2 months)
Assessment of the Efficacy of On-demand Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
At the end of a BE, treatment efficacy was to be assessed either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4-point scale including the four items 'excellent,' 'good,' moderate,' and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
Time frame: Monitored throughout the study from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
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University of Florida
Gainesville, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Tulane University
New Orleans, Louisiana, United States
Hemophilia Treatment Center of Nevada
Las Vegas, Nevada, United States
Gulf States Hemophilia and Thrombophilia
Houston, Texas, United States
Centro de Tratamiento de la Hemofilia Cordoba
Córdoba, Argentina
CTH Centro de Tratamiento de Hematologia y Hemoterapia Córdoba S.A.
Córdoba, Argentina
Fundación de Hemofilia de Salta
Salta, Argentina
Centro Mayo
Santiago del Estero, Argentina
Belarusian Research Center for Pediatric Oncology, Hematology and Immunology
Borovlyany, Belarus
...and 29 more locations
Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians
At the end of the postoperative period, an overall assessment of the efficacy of treatment in the pre-, peri-, and postoperative periods using the 'excellent,' 'good,' moderate,' and 'none' scale will be done jointly by the surgeon and the hematologist. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
Time frame: From start of surgery until end of post-operative period
Assessment of the Efficacy of Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
At the end of a BE, treatment efficacy was to be assessed either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4-point scale including the four items 'excellent', 'good', 'moderate', and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
Time frame: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)