title: Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children. Results of a phase 3 trial.
abstract: A global phase 3 study evaluated the pharmacokinetics, efficacy and safety of a recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in 27 previously treated male children (1-11 years) with severe and moderately severe haemophilia B (factor IX [FIX] activity ≤2 IU/dl). All patients received routine prophylaxis once every seven days for up to 77 weeks, and treated any bleeding episodes on-demand. The mean terminal half-life of rIX-FP was 91.4 hours (h), 4.3-fold longer than previous FIX treatment and clearance was 1.11 ml/h/kg, 6.4-fold slower than previous FIX treatment. The median (Q1, Q3) annualised spontaneous bleeding rate was 0.00 (0.00, 0.91) and was similar between the <6 years and ≥6 years age groups, with a weekly median prophylactic dose of 46 IU/kg. In addition, patients maintained a median trough level of 13.4 IU/dl FIX activity on weekly prophylaxis. Overall, 97.2 % of bleeding episodes were successfully treated with one or two injections of rIX-FP (95 % CI: 92 % to 99 %), 88.7 % with one injection, and 96 % of the treatments were rated effective (excellent or good) by the Investigator. No patient developed FIX inhibitors and no safety concerns were identified. These results indicate that rIX-FP is safe and effective for preventing and treating bleeding episodes in children with haemophilia B with weekly prophylaxis. Routine prophylaxis with rIX-FP at treatment intervals of up to 14 days are currently being investigated in children with severe and moderately severe haemophilia B. Clinicaltrials.gov (NCT01662531).
authors: Gili Kenet, The Israeli National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Sackler Medical School, Tel Aviv University, Israel, Tel.: +972 3 5302950, Fax: +972 3 5351806, E-mail: gili.kenet@sheba.health.gov.il.
authors: Gili
authors: G
authors: Kenet
authors: Hervé
authors: H
authors: Chambost
authors: Christoph
authors: C
authors: Male
authors: Thierry
authors: T
authors: Lambert
authors: Susan
authors: S
authors: Halimeh
authors: Tatiana
authors: T
authors: Chernova
authors: Maria Elisa
authors: ME
authors: Mancuso
authors: Julie
authors: J
authors: Curtin
authors: Christine
authors: C
authors: Voigt
authors: Yanyan
authors: Y
authors: Li
authors: Iris
authors: I
authors: Jacobs
authors: Elena
authors: E
authors: Santagostino
mesh_terms: D002648:Child; D002675:Child, Preschool; D005164:Factor IX / Q000627:therapeutic use*; D006207:Half-Life; D002836:Hemophilia B / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D007223:Infant; D008297:Male; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use
chemical_list: D011993:Recombinant Fusion Proteins; D005164:Factor IX
keywords: Haemophilia B; factor IX; on-demand treatment; prophylactic regimen; rIX-FP