A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Hemophilia
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 395
- 试验地点
- 28
- 主要终点
- Safety of treatment products used for hemophilia care will be assessed based on the number of reportable European Haemophilia Safety Surveillance (EUHASS) events documented on the ATHN Adverse Event Module Form.
研究概览
简要总结
This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment. The hemophilia treatment center (HTC) physician and participant will decide on the FDA-approved treatment to be used which may include non-factor products, bypassing agents, or clotting factor replacement products. The goal of this research is to study the use of hemophilia treatment products and their outcomes.
详细描述
This non-interventional, minimal risk cohort study will enroll and follow patients with hemophilia A or B as they receive hemophilia treatment for 4 years. This is a pragmatic study of real-world practices across a wide range of patients which will be ongoing as new treatment products receive FDA approval and will be advantageous to the entire hemophilia community. The total study duration is planned for 6 years.
The patients are seen at baseline, annually, and at study exit. Patients will also receive routine quarterly follow-up phone calls from HTC staff to review medical history, bleed events, and product treatment history. Other visits for unplanned events or for the change of treatment product will be scheduled as necessary. All required study visits will be planned to coincide with routine clinical care whenever possible. Co-enrollment in the ATHNdataset by patients is required to participate in the study.
Please note - the treatment regimen will be at the discretion of the patients' hemophilia caregivers. No treatment products are being provided by the study nor will the participants be paid. However, inhibitor titer testing will be provided at no cost to patients by the Center for Disease Control and Prevention (CDC).
The primary objective is to determine the safety of non-factor products, bypassing agents or clotting factor replacement products when used for people with hemophilia with or without inhibitors. Safety will be measured by those events listed in the European Haemophilia Safety Surveillance (EUHASS).
Data collected will include eligibility, demographics, medical history, hemophilia history (genotype and family history), inhibitor history and immune tolerance induction (ITI) treatment regimen (if applicable), co-morbidities at baseline (i.e., HIV, Hepatitis C), detailed treatment product(s) usage, bleeding events, surgical procedures, and EUHASS adverse events and other adverse events of special interest. Data collection will also include patient-reported outcome questionnaires regarding health-related quality of life, treatment use and patient perceptions of treatment products.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Congenital hemophilia A or B of any severity with or without inhibitors receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility;
- •Able to give informed consent (by patient or parent/authorized guardian); and
- •Co-enrollment in the ATHNdataset.
排除标准
- •Presence of any known bleeding disorder other than congenital hemophilia A or B;
- •Presence of concurrent hemophilia and a second hemostatic defect (low Von Willebrand Factor (VWF) without Von Willebrand disease (VWD) diagnosis is not excluded); and
- •Unable or unwilling to comply with the study protocol.
结局指标
主要结局
Safety of treatment products used for hemophilia care will be assessed based on the number of reportable European Haemophilia Safety Surveillance (EUHASS) events documented on the ATHN Adverse Event Module Form.
时间窗: 6 years
All treatment-related reportable adverse events will be documented on the ATHN Adverse Event Module Form based on EUHASS reportable events which include: death, factor inhibitor development, venous thrombosis, allergic reactions, treatment-emergent side effects, new malignancies, cardiovascular events, and blood-borne infections. Other treatment-related events to be documented on the ATHN Adverse Event Module Form including thrombotic microangiopathies, injection site reactions, drug-induced liver injury and anti-drug antibodies.
次要结局
- Real world effectiveness of treatment products assessed by the healthcare providers as measure by the number and types of medical visits and/or hospitalizations per year.(6 years)
- Effectiveness of non-factor products, bypassing agents and clotting factor replacement products will be evaluated based on the participant's number of bleeding events reported as the annualized bleeding rate (ABR).(6 years)
- Dosing regimens for hemophilia treatment products and total amount utilized by the study participant for prophylaxis and treatment of bleeds will be assessed.(6 years)
- Target joint monitoring(6 years)
- Efficacy of treatment is rated by health-related outcomes tools: EQ-5D-5L, Patient-Reported Outcomes Measurement Information System (PROMIS), Global Adherence Rating (GAR), and Treatment Satisfaction with Medicines Questionnaire (SATMED-Q).(6 years)