Burden of Beta-Thalassemia - Quality of Life and Health Care Resource Utilization- A Prospective Observational Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Celgene
- 入组人数
- 100
- 试验地点
- 5
- 主要终点
- Change in the Physical component score (PSC) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
研究概览
简要总结
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand). Approximately one to two study sites will be identified per country and approximately 20 β-thalassemia subjects will be enrolled per country (10 transfusion dependent (TD) and 10 Non-transfusion dependent (NTD) with a total of approximately 100 subjects. This study will not interfere with or influence the routine clinical management of β-thalassemia patients. Outcomes of interest will be collected prospectively for up to 6 months.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For the transfusion dependent (TD) subjects:
- •Male or female, >18 years of age at the time of signing the informed consent document (ICF);
- •Documented diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia;
- •TD - defined as: ≥6 Red Blood Cells (RBC) units in the 24 weeks prior to study participation and no transfusion free period for ≥35 days during that period; and
- •Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 to
- •For the non-transfusion dependent (NTD) subpopulation
- •Male or female, >18 years of age at the time of signing the informed consent document (ICF).
- •Documented diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
- •NTD - defined as: 0 to 5 RBC units received during the 24-week period prior to study participation (not including RBC transfusions units administered for elective surgery);
- •Most recent hemoglobin ≤10 g/dL (hemoglobin values ≤ 21 days post-transfusion will be excluded)
- •Performance status: ECOG score of 0 to 1.
排除标准
- •For TD Subpopulation:
- •A diagnosis of hemoglobin S/β-thalassemia;
- •Any significant psychiatric or medical conditions not related to thalassemia that would prevent the subject from participating in the study;
- •Inability to read or understand the local official languages; or
- •Participated in another clinical trial (interventional) <30 days prior to study participation
- •For NTD Subpopulation:
- •Received RBC transfusion ≤ 8 weeks prior to study enrollment;
- •A diagnosis of hemoglobin S/β-thalassemia;
- •Any significant psychiatric or medical conditions not related to thalassemia that would prevent the subject from participating in the study;
- •Inability to read or understand the local official languages; or
- •Participated in another clinical trial (interventional) <30 days prior to study participation.
结局指标
主要结局
Change in the Physical component score (PSC) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
时间窗: Up to 6 months
Short Form 36-Item Health Survey, Version 2 (SF-36) is a self-administered instrument that measures the impact of disease on overall quality of life and consists of 36 questions in eight domains (physical function, pain, general and mental health, vitality, social function, physical and emotional health). Norm-based scores were used in analyses, calibrated so that 50 is the average score and the standard deviation equals 10. Higher scores indicate a higher level of functioning. The physical functioning domain assesses limitations in physical activities because of health problems. A positive change from Baseline score indicates an improvement.
Change in the Mental component score (MCS) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
时间窗: Up to 6 months
Short Form 36-Item Health Survey, Version 2 (SF-36) is a self-administered instrument that measures the impact of disease on overall quality of life and consists of 36 questions in eight domains (physical function, pain, general and mental health, vitality, social function, physical and emotional health). Norm-based scores were used in analyses, calibrated so that 50 is the average score and the standard deviation equals 10. Higher scores indicate a higher level of functioning. The physical functioning domain assesses limitations in physical activities because of health problems. A positive change from Baseline score indicates an improvement.
次要结局
- Changes in the annual Healthcare Resource Care (HRC) involving the number of emergency room visits(Up to 6 months)
- Change in the total scores over the study period using the The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire(Up to 6 months)
- Change in the total scores over the study period using the Patient Reported Outcome (PRO) questionnaire in non-transfusion dependent (NTD) subjects(Up to 6 months)
- Changes in the annual Healthcare Resource Care (HRC) involving the number of days that a patient is hospitalized(Up to 6 months)
- Change in the total scores over the study period using the Tran-QOL questionnaire(Up to 6 months)
- Changes in the annual Healthcare Resource Care (HRC) involving the number of office visits(Up to 6 months)
- Number of minutes spent in sedentary, lightly active, fairly active, or very active lifestyles(Up to 6 months)
- Changes in the annual Healthcare Resource Care (HRC) involving the number of lab assessments completed(Up to 6 months)
- Percentage of time spent in sedentary, lightly active, fairly active, or very active lifestyles(Up to 6 months)
- Changes in the annual Healthcare Resource Care (HRC) involving the number of prescriptions dispensed(Up to 6 months)
- Changes in the annual Healthcare Resource Care (HRC) involving the number of procedures undergone(Up to 6 months)