Observatory of Patients With Haemophilia B Treated by IdElvion®

Active, not recruiting

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.

Recruitment & Eligibility

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

Primary Outcome Measures

Name	Time	Method
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
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
Incidence of non-surgical bleeding episodes	Up to 36 months

Secondary Outcome Measures

Name	Time	Method
The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion®	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
Incidence of surgical bleeding episodes	up to 36 months
The number of infusions and total dose of 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-3L for adults	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
Completion of the following questionnaire: EQ-5D-Y for children from 8 to 12 years	up to 36 months

Locations (28): CHU Amiens - Picardie
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Amiens, France
CH Annecy Genevois
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Annecy, France
CHRU Besançon
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Besançon, France
CHU Bordeaux - Hôpital Pellegrin
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Bordeaux, France
CHRU Brest
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Brest, France
CHU Caen
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Caen, France
CHU Clermont-Ferrand
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Clermont-Ferrand, France
CHU Dijon
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Dijon, France
Hôpital Simone Veil
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Eaubonne, France
CHU de Grenoble
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Grenoble, France
CRC-MHC Hôpital Kremlin Bicêtre
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Le Kremlin-Bicêtre, France
CH du Mans
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Le Mans, France
CHRU Lille
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Lille, France
CHU de Limoges
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Limoges, France
Hôpital Edouard Herriot
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Lyon, France
CHU Montpellier
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Montpellier, France
CHRU Nancy
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Nancy, France
CHU Nantes
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Nantes, France
CHU Nice
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Nice, France
Hôpital Necker Enfants Malades
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Paris, France
CHU de Reims
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Reims, France
CHU Rennes
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Rennes, France
CHU Rouen
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Rouen, France
CHU Saint-Etienne
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Saint-Étienne, France
CHRU Strasbourg
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Strasbourg, France
CHU Toulouse
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Toulouse, France
CHU Tours
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Tours, France
Hôpital André Mignot - CH Versailles
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Versailles, France