Evaluation of Prophylactic Replacement Therapy in Haemophilia B

Morfini, Massimo; Pm, Mannucci; Mariani, G.; Panicucci, F; Petrucci, Francesco; Baicchi, U; Capitanio, A; Pl, Ferrini; Mandelli, Franco

doi:10.1111/j.1600-0609.1976.tb01115.x

Cited by 37 publications

(31 citation statements)

References 19 publications

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“…In 1976, Morfini et al published the first randomized trial comparing once-weekly and twiceweekly prophylaxis with on-demand treatment in 10 subjects with haemophilia B over 1 year [10]. In 1976, Morfini et al published the first randomized trial comparing once-weekly and twiceweekly prophylaxis with on-demand treatment in 10 subjects with haemophilia B over 1 year [10].…”

Section: Discussionmentioning

confidence: 99%

Multicentre, randomized, open‐label study of on‐demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects

Valentino

Rusen²,

Elezović

et al. 2014

Haemophilia

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Few randomized studies have reported on the use of factor IX (FIX) for secondary prophylaxis in haemophilia B patients. This study aimed to evaluate the efficacy and safety of two secondary prophylaxis regimens of recombinant coagulation FIX, nonacog alfa, compared with on-demand therapy. Male subjects aged 6-65 years with severe or moderately severe haemophilia B (FIX:C ≤ 2, n = 50) and ≥12 bleeding episodes (including ≥6 haemarthroses episodes) within 12 months of study participation were enrolled in this multicentre, randomized, open-label, four-period crossover trial. The primary measure was the annualized bleeding rate (ABR) of two prophylactic regimens vs. on-demand therapy. In the intent-to-treat group, mean ABR values were 35.1, 2.6 and 4.6 for the first on-demand period, the 50 IU kg(-1) twice-weekly period, and the 100 IU kg(-1) once-weekly period respectively. Differences in ABR between the first on-demand period and both prophylaxis regimens were significant (P < 0.0001); no significant differences were observed between prophylaxis regimens (P = 0.22). Seven serious adverse events occurred in five subjects, none related to study drug. Results demonstrated that secondary prophylaxis therapy with nonacog alfa 50 IU kg(-1) twice weekly or 100 IU kg(-1) once weekly reduced ABR by 89.4% relative to on-demand treatment. Both prophylaxis regimens demonstrated favourable safety profiles in subjects with haemophilia B.

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Section: Discussionmentioning

confidence: 99%

Multicentre, randomized, open‐label study of on‐demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects

Valentino

Rusen²,

Elezović

et al. 2014

Haemophilia

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“…There was improved range of motion in the joints of 23/26 joints and the benefit was related to the number of days in which a measurable level of FIX was obtained in the plasma [62].…”

Section: Richey Neuman Addressed Prophylaxis In Haemophilia B: Lookinmentioning

confidence: 90%

Coagulation deficiencies: a look to the future¹

Lee

Aledort

2007

Haemophilia

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“…One of these studies evaluated patients with hemophilia B and two were conducted over 30 years ago. The study designs precluded a meaningful meta-analysis due to divergent intervention and patient age 60–63…”

Section: Randomized Trials Of Hemophilia Prophylaxismentioning

confidence: 99%

Review of antihemophilic factor injection for the routine prophylaxis of bleeding episodes and risk of joint damage in severe hemophilia A

Rossbach¹

2010

VHRM

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Individuals with severe factor VIII deficiency experience recurrent hemorrhages and develop progressive joint damage. Large retrospective, nonrandomized studies of patient cohorts followed over decades show that factor prophylaxis initiated at an early age before the onset of recurrent bleeding reduces the incidence of hemophilic arthropathy. Two recent prospective, multicenter, randomized trials conducted in Europe (the ESPRIT study) and the USA (the Joint Outcome Study) confirm the efficacy of prophylaxis in the prevention of hemarthroses and arthropathy. Regular prophylaxis initiated in early childhood enhances the quality of life for patients with severe hemophilia and reduces the risk of inhibitor development. The substantial costs of such preventative therapy may be offset by the reduced expenditures that the care of degenerative joint disease in adult hemophilia patients would otherwise require.

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Evaluation of Prophylactic Replacement Therapy in Haemophilia B

Cited by 37 publications

References 19 publications

Multicentre, randomized, open‐label study of on‐demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects

Multicentre, randomized, open‐label study of on‐demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects

Coagulation deficiencies: a look to the future¹

Review of antihemophilic factor injection for the routine prophylaxis of bleeding episodes and risk of joint damage in severe hemophilia A

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Evaluation of Prophylactic Replacement Therapy in Haemophilia B

Cited by 37 publications

References 19 publications

Multicentre, randomized, open‐label study of on‐demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects

Multicentre, randomized, open‐label study of on‐demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects

Coagulation deficiencies: a look to the future1

Review of antihemophilic factor injection for the routine prophylaxis of bleeding episodes and risk of joint damage in severe hemophilia A

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Coagulation deficiencies: a look to the future¹