Assessment of Pain in People With Thalassemia (Pain)

• ClinicalTrials.gov Identifier: NCT00872339
• Recruitment Status: Completed
• First Posted: March 31, 2009
• Results First Posted: June 5, 2014
• Last Update Posted: June 5, 2014
• 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: January 2, 2014
• Results First Posted Date: June 5, 2014
• Last Update Posted Date: June 5, 2014
• Study Start Date: March 2009
• Actual Primary Completion Date: June 2010 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: May 27, 2014): Prevalence of Pain [ Time Frame: Measured at Month 9 ]
• Original Primary Outcome Measures (submitted: March 30, 2009): Prevalence of pain in participants with transfusion and non-transfusion dependent thalassemia [ Time Frame: Measured at Month 9 ]

Current Secondary Outcome Measures (submitted: May 27, 2014)

• Common Sites of Pain [ Time Frame: Measured at Month 9 ]
• Pain Occurrence by Age [ Time Frame: Measured at Month 9 ]
• Impact of Pain on Functioning and Well-being [ Time Frame: Measured at Month 9 ]

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

• Common sites of pain by age, gender, and diagnosis [ Time Frame: Measured at Month 9 ]
• Pain severity and influence of age, gender, or diagnosis [ Time Frame: Measured at Month 9 ]
• Impact of pain on functioning and well-being [ Time Frame: Measured at Month 9 ]
• Effectiveness of treatment and medication over time [ Time Frame: Measured at Month 9 ]
• Correlate the Brief Pain Inventory (BPI) and Adolescent Pediatric Pain Tool (APPT) in the adolescent population [ Time Frame: Measured at Month 9 ]

• 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
• Official Title: Assessment of Pain in People With Thalassemia
• Brief Summary: Thalassemia is an inherited blood disorder that can result in mild to severe anemia. People with thalassemia often experience pain, but the exact sources and prevalence of pain remain unknown. This study will examine the prevalence and severity of pain in people with thalassemia who are treated with regular blood transfusions and people with thalassemia who are not 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. Recent medical advances in treating people with thalassemia who receive regular blood transfusions-a standard procedure that refreshes the healthy red blood supply-have resulted in increased life spans for these people. However, with the extended life spans have come additional issues, including pain. There have been no previous research studies that have examined pain levels in people with thalassemia, and as a result, the sources and prevalence of pain remain unknown. The purpose of this study is to assess the prevalence and severity of pain, common pain sites, and the impact of pain on functioning and well-being in people with thalassemia who receive regular blood transfusions and people with thalassemia who do not receive regular blood transfusions.

This study will enroll people with transfusion-dependant thalassemia and people with non-transfusion-dependant thalassemia. At a baseline study visit, participants will complete a demographic questionnaire and a pain assessment questionnaire. At Months 3, 6, and 9, study researchers will telephone participants to go over the same pain assessment questionnaire again.

• 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: Thalassemia patients who receive regular blood transfusions and those who do not receive regular blood transfusions.
• Condition: Thalassemia
• Intervention: Not Provided

Study Groups/Cohorts

• transfusion-dependant
  People with transfusion-dependant thalassemia who received at least 8 transfusions in the past year.
• non-transfusion-dependant
  People with non-transfusion-dependant thalassemia who received no transfusions in the past year.
• intermittently transfused
  Intermittently transfused patients- individuals who received at least one but fewer than eight transfusions in the last year

• Publications *: Haines D, Martin M, Carson S, Oliveros O, Green S, Coates T, Eile J, Schilling L, Dinu B, Mendoza T, Gerstenberger E, Trachtenberg F, Vichinsky E; Thalassemia Clinical Research Network. Pain in thalassaemia: the effects of age on pain frequency and severity. Br J Haematol. 2013 Mar;160(5):680-7. doi: 10.1111/bjh.12177. Epub 2012 Dec 30.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: June 29, 2011): 252
• Original Estimated Enrollment (submitted: March 30, 2009): 300
• Actual Study Completion Date: December 2010
• Actual Primary Completion Date: June 2010 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Thalassemia, as documented by clinical diagnosis, including the following:

  1. B-thalassemia (intermedia or major)
  2. Hgb H disease
  3. Hgb H with non-deletional mutations (e.g., Hgb H Constant Spring)
  4. E-B-thalassemia
  5. Homozygous alpha thalassemia
  6. Other thalassemic conditions not explicitly excluded
  7. Thalassemia intermedia due to heterozygous B mutation with an alpha excess
• Participants can be of any race, ethnicity, and either gender.

Exclusion Criteria:

• Thalassemia trait (i.e., single recessive B gene mutation, 2 gene alpha mutation) and thalassemia Hgb S, C, or D compound heterozygotes
• Unwillingness or inability to complete the Brief Pain Inventory (BPI) on a quarterly basis
• Has had a successful bone marrow transplant

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years to 90 Years (Child, 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: NCT00872339
• Other Study ID Numbers: 639; 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, PI, 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: Jeanne Boudreeaux, MD; Children's Healthcare of Atlanta
• Principal Investigator: Ellis Neufeld, MD; Boston Children's Hospital
• Principal Investigator: Alexis Thompson, MD; Children's Memorial Hospital of Chicago
• Principal Investigator: Brigitta Mueller, MD; Baylor College of Medicine at Houston
• Study Chair: Dru Foote, RN, NP; Children's Hospital and Research Institute of Oakland
• Principal Investigator: Patricia Giardina, MD; Weill Medical College of Cornell
• Principal Investigator: Janet Kwiatkowski, MD; Children's Hospital of Philadelphia
• Principal Investigator: Nancy Olivieri, MD; Toronto General Hospital

• PRS Account: HealthCore-NERI
• Verification Date: January 2014