scholarly journals Long-term safety and efficacy of turoctocog alfa in prophylaxis and treatment of bleeding episodes in severe haemophilia A: Final results from the guardian 2 extension trial

Haemophilia ◽  
2018 ◽  
Vol 24 (6) ◽  
pp. e391-e394 ◽  
Author(s):  
Steven R. Lentz ◽  
Dragana Janic ◽  
Kaan Kavakli ◽  
Predrag Miljic ◽  
Johannes Oldenburg ◽  
...  
Keyword(s):  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy ◽  
Bleeding Episodes ◽  
The Guardian ◽  
Extension Trial

scholarly journals PROTECT VIII kids extension study: Long‐term safety and efficacy of BAY 94‐9027 (damoctocog alfa pegol) in children with severe haemophilia A

Haemophilia ◽  
2021 ◽  
Author(s):  
Maria Elisa Mancuso ◽  
Tina Biss ◽  
Kathelijn Fischer ◽  
Monika Maas Enriquez ◽  
MacGregor Steele ◽  
...  
Keyword(s):  
Extension Study ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy

Long-acting recombinant factor VIII Fc fusion protein (rFVIIIFc) for perioperative haemostatic management in severe haemophilia A

2016 ◽  
Vol 116 (07) ◽  
pp. 1-8 ◽  
Author(s):  
Johnny Mahlangu ◽  
Margaret Ragni ◽  
Naresh Gupta ◽  
Savita Rangarajan ◽  
Robert Klamroth ◽  
...  
Keyword(s):  
Blood Loss ◽  
Frequency Factor ◽  
Pharmacokinetic Data ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy ◽  
Dental Procedures ◽  
Dosing Regimens ◽  
Bleeding Episodes ◽  
Long Acting

SummaryThe Phase 3 A-LONG and Kids A-LONG studies demonstrated the prolonged half-life of rFVIIIFc compared with rFVIII, and the safety and efficacy of rFVIIIFc in subjects with severe haemophilia A. Eligible subjects from A-LONG and Kids A-LONG continued rFVIIIFc treatment by enrolling in ASPIRE, an ongoing extension study. Based on combined data from the primary studies and ASPIRE interim data, the safety and efficacy of rFVIIIFc in subjects requiring surgery were evaluated. Perioperative dosing regimens were determined by investigators with guidance based on pharmacokinetic data and recommendations from a clinical dosing committee. In addition to dosing frequency, factor consumption, blood loss, transfusions, bleeding episodes, and haemostatic response were assessed. Across studies, 21 subjects underwent 23 evaluable major surgeries, including 19 orthopaedic surgeries; 41 subjects underwent 52 minor surgeries, including 30 dental procedures. No major and 10 minor surgeries were performed in paediatric subjects. Of the major (n = 22) and minor (n = 32) surgeries assessed for haemostatic response, all were rated as excellent or good by the investigator/surgeon. During most major surgeries (95.7 %), haemostasis was maintained with one rFVIIIFc infusion. Blood loss in major surgeries was consistent with similar surgeries in subjects without haemophilia. Across studies, rFVIIIFc was well tolerated; no subject developed an inhibitor.

Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study

2015 ◽  
Vol 114 (07) ◽  
pp. 35-45 ◽  
Author(s):  
Giulio Feola ◽  
Angelo Claudio Molinari ◽  
Cristina Santoro ◽  
Gianna Franca Rivolta ◽  
Dorina Bianca Cultrera ◽  
...  
Keyword(s):  
Controlled Trial ◽  
Secondary Prophylaxis ◽  
Annual Number ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Open Label ◽  
On Demand ◽  
Adolescents And Adults ◽  
Bleeding Episodes

SummaryRigorous evidence is lacking on long-term outcomes of factor VIII (FVIII) prophylaxis initiated in adolescent or adult patients with severe haemophilia A. The prospective, open-label Prophylaxis versus On-demand Therapy Through Economic Report (POTTER) study (Clinical-Trials.gov NCT01159587) compared long-term late secondary prophylaxis (recombinant FVIII-FS 20–30 IU/kg thrice weekly) with on-demand treatment in patients aged 12 to 55 years with severe haemophilia A. The annual number of joint bleeding episodes (primary endpoint), total bleeding episodes, orthopaedic and radiologic (Pettersson) scores, health-related quality of life (HRQoL), pharmacoeconomic impact, and safety were evaluated over a > 5-year period (2004–2010). Fifty-eight patients were enrolled at 11 centres in Italy; 53 (27 prophylaxis, 26 on demand) were evaluated and stratified into 2 age subgroups (12–25 and 26–55 years). Patients receiving prophylaxis experienced a significantly lower number of joint bleeding episodes vs the on–demand group (annualised bleeding rate, 1.97 vs 16.80 and 2.46 vs 16.71 in younger and older patients, respectively; p=0.0043). Results were similar for total bleeding episodes. Prophylaxis was associated with significantly fewer target joints (p< 0.001), better orthopaedic (p=0.0019) and Pettersson (p=0.0177) scores, better HRQoL, and fewer days of everyday activities lost (p< 0.0001) but required significantly higher FVIII product consumption. The POTTER study is the first prospective, controlled trial documenting long-term benefits of late secondary prophylaxis in adolescents and adults with severe haemophilia A. The benefits of reduced bleeding frequency, improved joint status, and HRQoL may offset the higher FVIII consumption and costs.

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