Assessment of Pain in People With Thalassemia (Pain)

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.

Study Overview

• Status: Completed
• Conditions: Thalassemia

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
• Enrollment (Actual): 252

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
      • Toronto General Hospital
• United States
  • California
    • Oakland, California, United States, 94609
      • Children's Hospital and Research Institute at Oakland
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Children's Healthcare of Atlanta
  • Illinois
    • Chicago, Illinois, United States, 60614
      • Children's Memorial Hospital of Chicago
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Children's Hospital of Boston
  • New York
    • New York, New York, United States, 10065
      • Weill Medical College
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Texas
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 90 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Thalassemia patients who receive regular blood transfusions and those who do not receive regular blood transfusions.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Prevalence of Pain	Measured at Month 9

Secondary Outcome Measures

Outcome Measure	Time Frame
Common Sites of Pain	Measured at Month 9
Pain Occurrence by Age	Measured at Month 9
Impact of Pain on Functioning and Well-being	Measured at Month 9

Collaborators and Investigators

• Sponsor: HealthCore-NERI
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI)
• Investigators: Principal Investigator: Jeanne Boudreeaux, MD, Children's Healthcare of Atlanta; 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

Publications and helpful links

General 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.

Study record dates

Study Major Dates

• Study Start: March 1, 2009
• Primary Completion (Actual): June 1, 2010
• Study Completion (Actual): December 1, 2010

Study Registration Dates

• First Submitted: March 30, 2009
• First Submitted That Met QC Criteria: March 30, 2009
• First Posted (Estimate): March 31, 2009

Study Record Updates

• Last Update Posted (Estimate): June 5, 2014
• Last Update Submitted That Met QC Criteria: May 27, 2014
• Last Verified: January 1, 2014

More Information

Terms related to this study

• Keywords: Transfusion-Dependent Thalassemia; Non-Transfusion-Dependent Thalassemia
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia
• Other Study ID Numbers: 639; U01HL065238 (U.S. NIH Grant/Contract)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No