The purpose of this post-approval study is to evaluate the safety and efficacy of Benefix in subjects with hemophilia B in usual care settings in China.
The purpose of this post-approval study is to provide supplementary information relating to the use of BeneFIX in Chinese subjects with hemophilia B, especially on the safety and efficacy in different populations of Chinese hemophilia B patients, in particular in pediatric patients \<6 years of age, pediatric patients ≥6 to ≤12 years of age, Previously Untreated Patients (PUPs) , subjects receiving prophylaxis treatment after enrollment in the study, and severe patients (FIX activity \<1%).
Study Type
INTERVENTIONAL
Purpose
TREATMENT
Masking
NONE
Enrollment
70
Subjects will be treated by the investigator according to usual care in China and in accord with the China BeneFIX Package Insert. The treatment duration is approximately 6 months (±7 days) or approximately 50 Exposure Days (EDs) (±5 EDs) (see protocol definition on EDs) whichever occurs first.
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, China
The Affiliated Hospital of Guizhou Medical University
Percentage of Participants Who Developed Factor IX Inhibitor
Factor IX (FIX) inhibitor development was defined as any Bethesda inhibitor titer greater than the laboratory's normal range or Bethesda inhibitor titer \>=0.6 Bethesda Unit (BU)/mL.
Time frame: Up to 6 months
Number of Participants With Allergic Reactions
FIX product allergy was defined as a hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgments.
Time frame: Up to 6 months
Number of Participants With Thrombotic Events
Thrombotic event was defined as any event associated with formation of a blood clot including catheter-associated thrombi and thrombotic complications.
Time frame: Up to 6 months
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product or medical device, regardless of the causality with the treatment or usage. A serious adverse event (SAE) was any untoward occurrence at any dose that resulted in death; was life threatening (immediate risk of death); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both serious and non-serious AEs. Treatment-emergent AEs were those with initial onset or increasing in severity after the first dose of study drug.
Time frame: Up to 7 months (28 calendar days after end of 6-month or 50-exposure day study treatment)
Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period
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Guiyang, Guizhou, China
Henan Provincial People's Hospital
Zhengzhou, Henan, China
Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei, China
Xiangya Hospital of Centre-South University
Changsha, Hunan, China
Department of Hematology,The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
Department of Hematology,The Affiliated Hospital of Xuzhou Medical University
Xuzhou, Jiangsu, China
Department of Hematology,Jiangxi Provincial People's Hospital
Nanchang, Jiangxi, China
Blood Center of Shandong Province
Jinan, Shandong, China
Ruijin Hospital Affiliated to Shanghai JiaoTong University School of Medicine/Hematology Department
Shanghai, Shanghai Municipality, China
...and 6 more locations
For prophylaxis period, annualized bleeding rate (ABR) was derived for each participant by the following formula: ABR = number of bleeds in prophylaxis period / (number of days in prophylaxis period/365.25). A prophylaxis period was defined as time from first prophylaxis infusion through 6 calendar days after the day of last prophylaxis infusion, or the day of study conclusion visit, whichever was earlier. A break in the prophylaxis period was any period of 28 days or longer in which no prophylaxis infusions were given. For a prophylaxis regimen to have been qualified to have ABR calculated, the sum of its periods needed to be \>=14 days.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX
The prophylaxis infusion time, bleed start time and bleed type (etiology) were used to determine the number of spontaneous, non-traumatic breakthrough bleeds that occurred \<=48 hours after a prophylaxis infusion. If there was more than 1 bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period
For on-demand period, the annualized bleeding rate (ABR) was derived for each participant by the following formula: ABR = number of bleeds in on-demand period / (number of days in on-demand period/365.25). The on-demand period was defined as the entire time of enrollment in the study (ie, from the date of the enrollment visit through the day before the Final/Early Termination visit) except time in any prophylaxis period. The breaks in the prophylaxis period were considered on-demand periods. For an on-demand regimen to have been qualified to have ABR calculated, the sum of its periods needed to be \>= 14 days.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response
Response was assessed using 4-point On-Demand Hemostasis Efficacy Rating Scale. Excellent means definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered; good means 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 the infusion, with no additional infusion administered; moderate means 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; no response means no improvement between infusions or during the 24 hour interval following an infusion, or condition worsens. Both first infusions and follow-up infusions were included.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Number of BeneFIX Infusions to Treat Each New Bleed
The number of BeneFIX infusions administered to treat each new bleed was calculated by adding the on-demand initial treatment and any on-demand follow-up treatments for the same bleed. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Average Infusion Dose and Total Factor IX Consumption in On-demand Setting
The total amount (international units \[IU\]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Average Infusion Dose and Total Factor IX Consumption in Prophylaxis Setting
The total amount (international units \[IU\]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Average Infusion Dose and Total Factor IX Consumption in Recovery Setting
The total amount (international units \[IU\]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in On-demand Setting
Less than expected therapeutic effect (LETE) in the on-demand setting was defined as 2 successive "no response" ratings recorded after 2 successive BeneFIX drug infusions, respectively.
Time frame: Up to 6 months or 50 exposure days whichever occurred first
Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in Prophylaxis Setting
Less than expected therapeutic effect (LETE) in the prophylaxis setting was defined as occurrence of any spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of BeneFIX (which was not used to treat a bleed).
Time frame: Up to 6 months or 50 exposure days whichever occurred first