Original Primary Outcome: To evaluate the pharmacokinetics and incremental recovery of ReFacto AF in pediatric subjects less than 12 years of age after a single exposure to ReFacto AF [ Time Frame: 2 years ]

Current Secondary Outcome:

• Mean Annualized Bleeding Rates (ABRs): All Participants [ Time Frame: Baseline up to Month 24 ]
  ABR for each participant was calculated as the number of bleeds requiring administration of FVIII replacement product (taken from the Infusion Log Diary case report form), divided by the total therapy duration (in days), then multiplied by 365.25. ABR for the participants who reported following a primary or secondary prophylaxis, on-demand regimen or preventive regimen at baseline were reported.
• Response to First On-Demand Treatment for New Bleeds: All Participants [ Time Frame: Baseline up to Month 24 ]
  A 4-point scale of assessment of 'on-demand' treatment (administration of an unscheduled bolus infusion of Refacto-AF to stop bleeding) is defined as: 1. Excellent: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered. 2. Good: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least one additional infusion administered for complete resolution of the bleeding episode;or, Definite pain relief and/or improvement in signs of bleeding starting after 8 hours following infusion, with no additional infusion administered. 3. Moderate: Probable or slight improvement starting after 8 hours following the infusion, with at least one additional infusion administered for complete resolution of the bleeding episode. 4. No Response: No improvement at all between infusions or during the 24-hour interval following an infusion, or condition worsens.
• Number of On-Demand ReFacto AF Infusions to Treat a New Bleed: All Participants [ Time Frame: Baseline up to Month 24 ]
  The infusion log diary case report form (CRF) was used to determine the number of on-demand (administration of an unscheduled bolus infusion of Refacto-AF to stop bleeding) ReFacto AF infusions administered to treat a new bleed. This was calculated by adding the initial for a new bleed (on-demand) infusion to any subsequent (on-demand) infusions for the same "previously treated bleed" (same bleed with same start date/time).
• Number of Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of ReFacto AF: All Participants [ Time Frame: Baseline up to Month 24 ]
  The number of breakthrough bleeds within 48 hours following a prophylaxis dose of ReFacto AF was summarized. The infusion log diary CRF was used to determine the number of infusions administered to treat a new bleed counting only those infusions which were administered <=48 hours after an infusion marked as "prophylaxis" (which had no associated bleed).
• Average Infusion Dose of ReFacto AF: All Participants [ Time Frame: Baseline up to Month 24 ]
  The average infusion dose (by weight) for each participant was calculated as his total factor FVIII consumption (in IU) divided by weight (in kg) divided by the number of infusions administered in total study duration. Data was reported separately for participants classified at baseline as following non-prophylaxis regimen (for example: on-demand regimen, preventive, or not specified), and participants classified at baseline following a primary or secondary prophylaxis regimen.
• Total Factor VIII Consumption: All Participants [ Time Frame: Baseline up to Month 24 ]
  Total factor VIII consumption for each participant was calculated by sum of the total amount of ReFacto AF (in IU) infused for each ReFacto AF infusion (recorded in the infusion log diary CRF). Data was reported separately for participants classified at baseline as following non-prophylaxis regimen (for example: on-demand regimen, preventive, or not specified), and participants classified at baseline following a primary or secondary prophylaxis regimen.
• Number of Less-Than-Expected-Therapeutic Effect (LETE) Bleeds in the On-Demand Setting: All Participants [ Time Frame: Baseline up to Month 24 ]
  LETE in on-demand setting was based on response to treatment of a bleeding episode. LETE in the on-demand setting occurred if participant recorded 2 successive "no response" ratings after 2 successive ReFacto AF infusions. Both infusions were to be administered at an interval of 24 hours for treatment of same bleeding event in absence of confounding factor which included: known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than 4 hours between onset of bleed to infusion, delay of greater than 24 hours before administration of a follow-up infusion, known compromised ReFacto AF, faulty administration of ReFacto AF, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator (e.g., kidney stones or use of medications known to impair platelet function, such as aspirin or NSAIDs),or ongoing trauma responsible for continued bleeding.
• Number of Less-Than-Expected-Therapeutic Effect (LETE) Bleeds in the Prophylaxis Setting: All Participants [ Time Frame: Baseline up to Month 24 ]
  LETE in the prophylaxis setting
  
  

  Original Secondary Outcome: Evaluate the safety and the efficacy of ReFacto AF in pediatric subjects less than 12 years of age including the frequency of less than expected therapeutic effect. [ Time Frame: 2 years ]
  
  Information By: Pfizer
  

  Dates:
  Date Received: June 4, 2009
  Date Started: December 2009
  Date Completion:
  Last Updated: December 21, 2016
  Last Verified: December 2016
  

  

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