Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions

• ClinicalTrials.gov Identifier: NCT00872833
• Recruitment Status: Completed
• First Posted: March 31, 2009
• Results First Posted: August 27, 2014
• Last Update Posted: October 7, 2015
• Sponsor: HealthCore-NERI
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI)
• Information provided by (Responsible Party): HealthCore-NERI

Tracking Information

• First Submitted Date: March 30, 2009
• First Posted Date: March 31, 2009
• Results First Submitted Date: April 9, 2014
• Results First Posted Date: August 27, 2014
• Last Update Posted Date: October 7, 2015
• Study Start Date: July 2009
• Actual Primary Completion Date: October 2010 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 12, 2014)

Report of Pain by Age Group [ Time Frame: measured daily over the 3 transfusion cycles ]

Subjects reported whether or not they had pain (yes/no) during each transfusion cycle (data on up to 3 transfusion cycles were collected for each subject). The percent of subject-cycles with pain was calculated for the quartiles of the transfusion cycle.

• Original Primary Outcome Measures (submitted: March 30, 2009): Assess whether reports of pain vary over the transfusion cycle [ Time Frame: Measured at Month 12 ]

Current Secondary Outcome Measures (submitted: August 12, 2014)

Report of Pain by Length of the Transfusion Cycle [ Time Frame: measured daily over the 3 transfusion cycles ]

Subjects reported whether or not they had pain (yes/no) during each transfusion cycle (data on up to 3 transfusion cycles were collected for each subject). The percent of subject-cycles with pain was calculated for the quartiles of the transfusion cycle.

Original Secondary Outcome Measures (submitted: March 30, 2009)

• Assess whether the length of the transfusion cycle affects pain level [ Time Frame: Measured at Month 12 ]
• Assess whether pain varies by pre-transfusion hemoglobin and reticulocyte levels [ Time Frame: Measured at Month 12 ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions
• Official Title: Assessment of Pain in Transfusion Dependent Patients With Thalassemia During Transfusion Cycles
• Brief Summary: Thalassemia is an inherited blood disorder that can result in mild to severe anemia. Regular blood transfusions, which refresh the healthy red blood cell supply, are one treatment for thalassemia. People with thalassemia often experience pain, but the exact source of pain remains unknown. This study will examine how pain varies during the blood transfusion cycle in people with thalassemia who are treated with regular blood transfusions.

Detailed Description

Thalassemia is an inherited blood disorder in which the body makes an abnormal form of hemoglobin-the protein in red blood cells that carries oxygen. People with thalassemia often experience fatigue, shortness of breath, and pain. There have been no previous research studies that have fully examined pain levels in people with thalassemia, and as a result, the sources of pain remain unknown. This study is a substudy of the Assessment of Pain study, which is a Thalassemia Clinical Research Network (TCRN) study that is examining the prevalence and severity of pain in people with transfusion-dependent thalassemia and non-transfusion-dependant thalassemia. This study will enroll a subset of participants from the Assessment of Pain study who have transfusion-dependant thalassemia. The purpose of this study is to examine whether pain varies during the blood transfusion cycle and whether the length of the transfusion cycle affects pain levels in people with transfusion-dependent thalassemia.

Participants will complete daily questionnaires through an automated telephone system to assess pain levels during three blood transfusion cycles. Each transfusion cycle will last between 2 to 4 weeks, depending on the individual needs of the participant, and the cycles will be separated by at least 3 months but no more than 4 months. Prior to each transfusion cycle, study researchers will review participants' medical records for certain blood level measurements.

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: People with transfusion-dependent thalassemia who receive care at sites funded by the Thalassemia Clinical Research Network and their satellites.
• Condition: Thalassemia
• Intervention: Not Provided

Study Groups/Cohorts

• age 18-29
  People age groups 18-29 with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study.
• age 30+
  People age groups 30+ years with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study.

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 12, 2014): 32
• Original Estimated Enrollment (submitted: March 30, 2009): 25
• Actual Study Completion Date: October 2010
• Actual Primary Completion Date: October 2010 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Participating in the Thalassemia Clinical Research Network Assessment of Pain study
• Has a transfusion dependence of at least eight transfusions per year
• Diagnosis of beta thalassemia or E-beta-thalassemia
• Experiences at least "mild" pain in the 1 month before study entry, as measured by the response to the Brief Pain Inventory (BPI) question #6 from the Assessment of Pain study

Exclusion Criteria:

• Unwillingness or inability to complete the Brief Pain Inventory Short Form (BPI-SF) on a daily basis

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, United States

Administrative Information

• NCT Number: NCT00872833
• Other Study ID Numbers: 640; U01HL065238 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: HealthCore-NERI
• Original Responsible Party: Sonja McKinlay, PhD, New England Research Institutes
• Current Study Sponsor: HealthCore-NERI
• Original Study Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI)

Investigators

• Principal Investigator: Dru Foote, NP; Children's Hospital and Research Institute Oakland
• Study Chair: Jeanne Boudreaux, MD; Children's Healthcare of Atlanta
• Study Chair: Thomas Coates, MD; Children's Hospital Los Angeles
• Study Chair: Elliott Vichinsky, MD; UCSF Benioff Children's Hospital Oakland
• Study Chair: Michael Jeng, MD; Stanford University
• Study Chair: Janet Kwiatkowski, MD; Children's Hospital of Philadelphia
• Study Chair: Nancy Olivieri, MD; University Health Network - Toronto General Hospital
• Study Chair: Patricia J. Giardina, MD; Weill Medical College of Cornell
• Study Chair: Brigitta Mueller, MD; Baylor College of Medicine
• Study Chair: Alexis A. Thompson, MD, MPH; Ann & Robert H Lurie Children's Hospital of Chicago

• PRS Account: HealthCore-NERI
• Verification Date: April 2012