To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care

Study Overview
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Brief Summary
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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.

Official Title
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Burden of Beta-Thalassemia - Quality of Life and Health Care Resource Utilization- A Prospective Observational Study

Conditions
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Beta-Thalassemia

Intervention / Treatment
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* Other: Quality of Life (QOL) questionnaires
* Other: Healthcare Resource Utilization


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 1. 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. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| β-thalassemia transfusion dependent subjects<br>Participants will complete 3 quality of life instruments (i.e. FACT-AN, the SF-36v2, and the TranQol) once every 3 weeks, in addition to a TranQol instrument on the day of a RBC transfusion. Continuous monitoring of Healthcare Resource Utilization will be conducted throughout the course of the study at each clinical visit. | Other: Quality of Life (QOL) questionnaires<br>* Monitoring of FACT-AN, SF-36V2, TransQol, and NTD PRO assessments,<br>Other: Healthcare Resource Utilization<br>* Monitoring and reviewing HealthCare Resource Utilization information.<br>|
| β-thalassemia Non Transfusion Dependent (NTD) subjects<br>Participants will complete 2 quality of life instruments (i.e. FACT-An and the the SF-36v2l) once every 3 weeks, in addition to completing the non-transfusion dependent Patient Recorded Outcome (PRO) tool on a daily basis. Continuous monitoring of Healthcare Resource Utilization will be conducted throughout the course of the study at each clinical visit. | Other: Quality of Life (QOL) questionnaires<br>* Monitoring of FACT-AN, SF-36V2, TransQol, and NTD PRO assessments,<br>Other: Healthcare Resource Utilization<br>* Monitoring and reviewing HealthCare Resource Utilization information.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 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. | Up to 6 months | 
| 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 | 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. | Up to 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the total scores over the study period using the The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire | The FACT-An questionaire is used to assess leath-related quality of life (HRQol). It measures the impact of fatigue and other anemia-related symptoms on patient functioning. The overall score range for the FACT-An is 0-188. Higher scores indicate better HRQoL. In addition to general HRQoL, the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning. Patients with higher hemoglobin levels and better performance status reported significantly higher scores on these instruments (including the newly created subscales) than did those with lower hemoglobin levels and poorer performance status | Up to 6 months | 
| Change in the total scores over the study period using the Tran-QOL questionnaire | The Tran Qol is a new disease specific QOL measure for adults with B-Thalassemia with a score ranging from 0-100. It measures the quality of life issues of patients with B-Thalassemia by assessing physical health, emotional health, family functioning and school/career functioning. Higher scores indicate better health related quality of life (HRQOL). A positive change from baseline indicates an improvement in overall HRQol. | 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 | For NTD subjects, they will complete a Patient Reported Outcome (PRO) PRO via e-diary each evening before bedtime. For all subjects, physical activities will be recorded continuously via Fitbit for the entire study period. | Up to 6 months | 
| Changes in the annual Healthcare Resource Care (HRC) involving the number of office visits | Healthcare resource utilization will be measured by the number of office visits a B-thal patient has over the course of 6 months while involved in the study. | Up to 6 months | 
| Number of minutes spent in sedentary, lightly active, fairly active, or very active lifestyles | Number of minutes a patient will spend in sedentary, lightly active, fairly active, and very active levels. | Up to 6 months | 
| Changes in the annual Healthcare Resource Care (HRC) involving the number of prescriptions dispensed | Healthcare resource utilization will be measured by the number of prescriptions dispensed for a B-thal patient over the course of 6 months while involved in the study | Up to 6 months | 
| Changes in the annual Healthcare Resource Care (HRC) involving the number of procedures undergone | Healthcare resource utilization will be measured by the number of procedures undergone for a B-thal patient over the course of 6 months while involved in the study | Up to 6 months | 
| Changes in the annual Healthcare Resource Care (HRC) involving the number of lab assessments completed | Healthcare resource utilization will be measured by the number of lab assessments completed for a B-thal patient over the course of 6 months while involved in the study, relating to the following lab assessments: hematology, clinical chemistry, renal, hepatic, iron parameters, endocrine parameters, and urinalysis. | Up to 6 months | 
| Changes in the annual Healthcare Resource Care (HRC) involving the number of days that a patient is hospitalized | Healthcare resource utilization will be measured by the number of days the B-thal patient was hospitalized over the course of 6 months while involved with the study, including the number of days that the patient was in the Intensive Care Unit (ICU) | Up to 6 months | 
| Changes in the annual Healthcare Resource Care (HRC) involving the number of emergency room visits | Healthcare resource utilization will be measured by the number of emergency room visits the B-thal patient had over the course of the 6 months while involved with the study | Up to 6 months | 
| Percentage of time spent in sedentary, lightly active, fairly active, or very active lifestyles | Percentage of time a patient will spend in sedentary, lightly active, fairly active, or very active lifestyles | Up to 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Beta-Thalassemia, Quality of Life, Healthcare Utilization, Transfusion dependent (TD) β-thalassemia, Non-transfusion dependent β-thalassemia, Standard of care, TransQoL, SF36v2, Fact-An, NTD beta-thalassemia PRO