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

NCT02626689 | Completed

• 1 other identifier: ACE-536-B-THAL-003
• Study Type: observational
• Target: 100
• Locations: 5 countries
• Sites: 5

Brief Summary

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.

Conditions

Beta-Thalassemia

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (11)

• 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 Tran-QOL 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 office visits

  Up to 6 months

• Number of minutes spent in sedentary, lightly active, fairly active, or very active lifestyles

  Up to 6 months

Study Arms (2)

β-thalassemia transfusion dependent subjects

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; Other: Healthcare Resource Utilization

β-thalassemia Non Transfusion Dependent (NTD) subjects

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; Other: Healthcare Resource Utilization

Interventions

Quality of Life (QOL) questionnaires OTHER

Monitoring of FACT-AN, SF-36V2, TransQol, and NTD PRO assessments,

β-thalassemia Non Transfusion Dependent (NTD) subjects; β-thalassemia transfusion dependent subjects

Healthcare Resource Utilization OTHER

Monitoring and reviewing HealthCare Resource Utilization information.

β-thalassemia Non Transfusion Dependent (NTD) subjects; β-thalassemia transfusion dependent subjects

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

β-thalassemia subjects who are transfusion dependent (TD) and non-transfusion dependent (NTD)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Celgene: lead

Study Sites (5)

Aghia Sofia Children's Hosptial

Athens, Goudi, 11527, Greece

Location

Fondazione IRCCS Ca Granda Ospedale Maggiore

Milan, 20122, Italy

Location

American University of Beirut Medical Center

Beirut, 4407 2020, Lebanon

Location

Faculty of Medicine Siriraj Hospital, Mahidol University

Bangkok, Bangkoknoi, 10700, Thailand

Location

Cukurova University Medical Faculty Balcali Hospital

Çukurova, 1330, Turkey (Türkiye)

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Quality of Life; Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Health Status; Demography; Epidemiologic Measurements; Public Health; Environment and Public Health; Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation

Study Officials

• Xiaohan (Henry) HU, MD, MPH, PhD

  Celgene

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 5, 2015
• First Posted: December 10, 2015
• Study Start: March 1, 2016
• Primary Completion: January 3, 2017
• Study Completion: January 3, 2017
• Last Updated: April 7, 2017

Record last verified: 2017-04

Locations

LE (1); GR (1); TH (1); TU (1); IT (1)