Clinical Trial: Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Post Marketing Surveillance To Observe Safety And Efficacy Of BeneFIX In Patients With Hemophilia B

Brief Summary:

To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including:

1) Unknown adverse reactions, especially serious adverse reactions; 2) Changes in the incidences of adverse reactions under the routine drug uses.

3) Factors that may affect the safety of the drug 4) Factors that may affect the effectiveness of the drug


Detailed Summary: The patients who meet the inclusion criteria will be enrolled consecutively.
Sponsor: Pfizer

Current Primary Outcome:

  • Number of Participants With Adverse Events (AEs) According to Baseline Characteristics [ Time Frame: Baseline up to 6 months ]
    AE: any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. AE assessed by baseline characteristics (chr) included age, gender, pediatric/geriatric status, liver disorder, BeneFIX treatment (previously/newly), factor nine (FIX) gene mutation, prior exposure to plasma-derived FIX products, prior FIX regimen(s) utilized, personal history of FIX inhibitor, family history of hemophilia B, severity of bleeding, medical history, concomitant medication and therapy.
  • Number of Participants With Adverse Events (AEs) According to Severity [ Time Frame: Baseline up to 6 months ]
    AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AE was assessed according to severity; mild (not causing any significant problem, dose adjustment not required), moderate (caused problem that does not interfere significantly with usual activities or the clinical status, dose adjustment needed due to adverse event) and severe (caused problem that interferes significantly with usual activities or the clinical status, study drug stopped due to adverse event).
  • Number of Participants With Action Taken in Response to Adverse Events (AEs) [ Time Frame: Baseline up to 6 months ]
    AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. After an onset of an AE, relevant actions were undertaken on the study drug or the part

    Original Primary Outcome:

    Current Secondary Outcome:

    • Mean Annualized Bleeding Rate (ABR) [ Time Frame: Baseline up to 6 months ]
      An annualized bleeding rate (ABR) was calculated as the number of bleeds requiring administration of BeneFIX (for on-demand therapy and surgery), divided by total period of bleeding multiplied by 365.25. Total period of bleeding is the number of days on treatment for prophylaxis purpose and on-demand therapy and surgery.
    • Number of Responses to On-demand Treatment With Study Medication [ Time Frame: Baseline up to 6 months ]
      Responses to on-demand treatment were rated by participant/caregiver or physician each time the drug was administered, on 4-point scale. Score 1=excellent (definite pain relief [PR] and improvement [imp] within 8 hours [h] of infusion [inf], no additional inf); score 2=good (definite PR and imp within 8h of inf, at least 1 additional inf for complete resolution [CR] of bleeding or starting after 8h of inf, no additional inf); score 3=moderate (probable or slight imp starting after 8h of inf, at least 1 additional inf for CR of bleeding); score 4=no imp at all, or condition worsens).
    • Mean Number of Infusion of Study Medication [ Time Frame: Baseline up to 6 months ]
      Mean frequency of BeneFIX administration of each participant was calculated from number of BeneFIX infusions which each participant received for treatment of each new bleed. Mean frequency of BeneFIX administration for total participants was summarized.
    • Mean Number of Breakthrough Bleeds Within 48 Hours of Study Medication [ Time Frame: Baseline up to 6 months ]
      Mean frequency of breakthrough (spontaneous/non-traumatic) bleeds of each participant within 48 hours of a preventive/prophylaxis dose of BeneFIX was calculated from number of irregular bleeding which occurred in each participant. Mean frequency breakthrough bleeds for total participants within 48 hours of a preventive/prophylaxis dose of BeneFIX was summarized.
    • Average Infusion Dose of Study Medication [ Time Frame: Baseline up to 6 months ]
      Average of dose per infusion per kilogram (kg) body weight was reported for prophylaxis purpose or on-demand therapy and surgery.
    • Total Infusion of Study Medication [ Time Frame: Baseline up to 6 months ]
      Total dose of study drug infused was calculated over the study duration.
    • Percentage of Participants With Efficacy Evaluation [ Time Frame: Baseline up to 6 months ]
      The efficacy of study drug was rated as 'very effective', 'effective', 'slightly ineffective' and 'ineffective'.


    Original Secondary Outcome:

    Information By: Pfizer

    Dates:
    Date Received: June 7, 2007
    Date Started: August 2007
    Date Completion:
    Last Updated: May 15, 2013
    Last Verified: May 2013