Hemophilia B
Conditions
Interventions
None listed
Sponsors
CSL Behring K.K.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Pediatric patients with a diagnosis of hemophilia B who were <12 years of age at initiation of rIX-FP. 2) Patients who initiated rIX-FP between 1 May 2017 and 30 September 2023 as prophylaxis, on-demand, and/or perioperative treatment(s) according to the label and continued to use rIX-FP for >6 months or had any activity record in medical chart for >6 months. 3) Provision of the patient's or the legally acceptable representative's informed consent or assent in pediatric research according to the pediatric patient's capacity to consent. In cases of difficulty in obtaining written consent due to transfer to a different hospital, death, or other reasons, only if the central ethics review committee permits opt-out, in which the patients or the legally acceptable representatives can access the opt-out information and are given the opportunity to refuse the provision of his/her existing information, enrollment of the patient will be permitted.
Exclusion criteria
Exclusion criteria: 1) Participating in any interventional clinical trials during the period between 1 May 2017 and 30 June 2024. If patients discontinue rIX-FP during the period above, patients who participated in any interventional clinical trials during the period between 1 May 2017 and the date of discontinuation will be excluded from the study. * Treatment discontinuation is defined as: (1) a switch to other FIX products (excluding a temporary switch defined as <3 doses of other FIX products/<1 month duration of other FIX products) and (2) loss to follow up due to transfer to a different hospital, death, or other reasons. The date of discontinuation is defined as (1) the date of a switch to other FIX products, (2)the date of transfer to a different hospital, the date of death, or the date of loss to follow-up confirmed by the investigator. 2) Patients who have previously treated with other FIX product(s) before initiation of rIX-FP 3) Patients enrolled in rIX-FP post-marketing surveillance (PMS)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1) To describe the real-world treatment patterns of rIX-FP in pediatric PUPs with hemophilia B a. Treatment duration (days) b. Total number of doses [i.e., Expousre days (EDs) to rIX-FP] c. Total dosage (IU) d. Mean number of doses per month e. Dose per body weight (IU/kg) f. Dosage interval (days) g. Treatment regimen (prophylaxis, on-demand, perioperative) h. Use of CV port (days) i. Plasma FIX activity (%) during the treatment period 2) To describe the patient background and baseline clinical characteristics of pediatric PUPs with hemophilia B who initiated treatment with rIX-FP. a. Sex (male, female) b. Body weight (kg) c. Age at diagnosis (months, years) d. Age at initiation of rIX-FP (<2, 2<=to<6, 6<=to<12 years) e. Severity (severe, plasma FIX activity<1%; moderate, 1%<=to<=5%; mild, 5%< to <40%; unknown) f. Family history of hemophilia g. Family history of inhibitor h. IVR ([IU/dL]/[IU/kg]) i. History of bleeding events j. Reason for visiting the facility (bleeding event, family history, others) k. Comorbidities and medical history at baseline (before and at initiation of rIX-FP) | — |
Secondary
| Measure | Time frame |
|---|---|
| To describe the real-world effectiveness on bleeding (all bleeding, treatment required) events and safety on the incidence of FIX inhibitor development after initiation of treatment with rIX-FP. a. Number of patients with bleeding events at follow-up b. Number of bleeding events at follow-up - all bleeding, treatment required, bleeding type (spontaneous, traumatic, surgical, others) c. Number of bleeding events per patient and annualized bleeding rate (ABR) at follow-up d. Number of patients with FIX inhibitor development at follow-up e. Allergic drug reactions | — |
Countries
Japan
Contacts
Public ContactYuki Murabayashi
Mebix, Inc. Research Promotion Headquarters
Outcome results
None listed