NCT02796222已完成不适用
Factor Product Utilization and Health Outcomes in Patients With Hemophilia A and B in Canada: An Observational Study of Real-world Outcomes
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 61
- 试验地点
- 1
- 主要终点
- Change in the total annualized factor consumption (in units/kilogram/year)
研究概览
简要总结
Recombinant factor VIII Fc (rFVIIIFc) and recombinant factor IX Fc (rFIXFc) are extended half-life coagulation factors approved by Health Canada in 2014 for the treatment of severe hemophilia A and B, respectively. The objectives of this observational study is to describe the change in annual factor consumption, clinical and patient-reported outcomes for patients who switch from recombinant factor VIII (rFVIII) and recombinant factor IX (rFIX) to rFVIIIFc/ rFIXFc in Canada, and to explore clinicians' and patients' reasons for switching or not switching.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males ≥12 years of age with severe and moderate congenital hemophilia A or B (baseline factor activity<5%)
- •Ability to understand the purpose and risks of the study and provide signed and dated informed consent or assent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations.
排除标准
- •Unable or unwilling to provide informed consent
- •Patients with an existing bleeding disorder other than hemophilia A or B
- •History of hypersensitivity or severe allergic reactions to factor products
- •Patients currently participating in a phase 1-3 study with another factor replacement product
- •Unable to adhere to the study requirements based on the judgment of the Prescribing Physician (e.g. unable to enter accurate and timely infusion and bleeding records)
结局指标
主要结局
Change in the total annualized factor consumption (in units/kilogram/year)
时间窗: From baseline to 24-month period on rFVIIIFc or rFIXFc
次要结局
- Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score(From baseline to 3 months, 12 months and 24 months)
- Change in health-related quality of life (HRQoL) SF-36(From baseline to 3 months, 12 months and 24 months)
- Change in health-related quality of life (HRQoL) Haem-A-Qol(From baseline to 3 months, 12 months and 24 months)
- Change in health-related quality of life (HRQoL) CHO-KLAT(From baseline to 3 months, 12 months and 24 months)
- Change in chronic pain Numeric Rating Scale (0-10)(From baseline to 3 months, 12 months and 24 months)
- Change in chronic pain "Bodily Pain" subscale of SF-36(From baseline to 3 months, 12 months and 24 months)
- Change in physical activity "Physical Functioning" subscale of SF-36.(From baseline to 3 months, 12 months and 24 months)
- Product used for treatment of breakthrough bleeding and surgical procedures(Baseline through study completion, an average of 2 years)
- Change in physical activity (IPAQ)(From baseline to 3 months, 12 months and 24 months)
- Change in treatment satisfaction "Treatment" domain of Haem-A-QoL(From baseline to 3 months, 12 months and 24 months)
- Annualized bleeding rate(From baseline to 24-month period after product switch (or 24-month period on study for non-switchers))
- Number of infusions required to treat a breakthrough bleed(From baseline to 24-month period after product switch (or 24-month period on study for non-switchers))
- Change in treatment satisfaction abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.(From baseline to 3 months, 12 months and 24 months)
- Change in mood "Mental Health" subscale of SF-36(From baseline to 3 months, 12 months and 24 months)
- Clinicians' and patients' reason for switching to rFVIIIFc(Baseline through study completion, an average of 2 years)
- Clinicians' and patients' reason for switching to rFIXFc(Baseline through study completion, an average of 2 years)
- Ratio of annual factor consumption-to-annual factor prescription(From baseline to 24-month period after product switch (or 24-month period on study for non-switchers))
- Change in mood partner/caregiver subjective assessment numeric rating scale (0-10)(From baseline to 3 months only)
- Total annualized number of factor infusions(From baseline to 24-month period after product switch (or 24-month period on study for non-switchers))
- Incremental factor utilization per joint bleed avoided(From baseline to 24-month period after product switch (or 24-month period on study for non-switchers))
- Adverse events leading to permanent discontinuation of rFVIIIFc or rFIXFc(From baseline to 24-month period after product switch (or 24-month period on study for non-switchers))
- Serious adverse events(From baseline to 24-month period after product switch (or 24-month period on study for non-switchers))
研究者
Shannon Jackson
Clinical Associate Professor
University of British Columbia
研究点 (1)
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