Haemophilia B
Conditions
Keywords
FIX, Coagulation FIX
Brief summary
OrPHEe is a non-interventional, prospective and national study which aim is to record real life data in haemophilia B French patients treated with Idelvion® to confirm the efficacy and safety of this product established in clinical development studies.
Detailed description
Haemophilia B is a congenital coagulation disorder caused by a deficiency or anomaly of coagulation factor IX (FIX). The severity of the haemophilia depends on the extent of the FIX deficiency with clinical manifestations differing depending on the location of the bleed. Treatment of this disease involves the administration of the deficient factor, i.e. FIX, to patients. Depending on the severity of the disease and context, a range of treatment regimens are available (long-term prophylactic treatment for the prevention of non-surgical bleeds, short-term prophylactic treatment for high-risk periods, treatment for the prevention of surgical bleeds or on-demand curative treatment). CSL Behring has developed a long-acting recombinant FIX, i.e. rIX-FP (Idelvion®), to extend the intervals between the administrations of treatment while also providing a therapeutic benefit. Data from clinical studies clearly confirm the therapeutic benefit of Idelvion® in adult and paediatric patients with haemophilia B previously receiving an on-demand treatment for bleeding episodes, for long-term prophylaxis, as well as in patients undergoing surgery.
Interventions
None listed
Sponsors
CSL Behring
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Have agreed to participate in the observatory after receiving written information on the purpose of the study and the personal data to be collected (agreement of parents for under-age patients); * Present with haemophilia B and are currently being treated or have previously been treated with Idelvion® as a long-term prophylactic treatment, on-demand treatment or short-term treatment for surgical procedures or to cover periods with a high-risk of bleeding. * Do not have FIX targeted antibodies at the time of the inclusion visit
Exclusion criteria
* Refusal by the patient or his/her legal representative to participate in the study; * Existence of a contraindication to the use of Idelvion® treatment (known hypersensitivity to FIX or hamster proteins); * Simultaneous participation in an interventional clinical study on a drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annual bleeding rates (overall, spontaneous and traumatic) under the scope of long-term prophylaxis or on-demand treatment of non-surgical bleeding events | Up to 36 months |
| Number of spontaneous bleeding events per patient | Up to 36 months |
| Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes | Up to 36 months |
| Incidence of non-surgical bleeding episodes | Up to 36 months |
Secondary
| Measure | Time frame |
|---|---|
| The number of infusions and total dose of Idelvion® | up to 36 months |
| The type and incidence of adverse events (AE) in particular severe AE,and AE related to Idelvion® | up to 36 months |
| The number of infusions and total dose of Idelvion® (in IU/kg) required to prevent or treat surgical bleeding episodes | up to 36 months |
| Completion of the following questionnaire: EQ-5D-Y for children from 8 to 12 years | up to 36 months |
| Completion of the following questionnaire: EQ-5D-3L for adults | up to 36 months |
| Incidence of surgical bleeding episodes | up to 36 months |
| The number of infusions and total dose of Idelvion® (in IU/kg) required to cover a high risk of bleeding | up to 36 months |
| The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion® | up to 36 months |
Countries
France
Outcome results
None listed