Clinical trial NCT00872339: Assessment of Pain in People With Thalassemia
Official title: Assessment of Pain in People With Thalassemia
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Thalassemia
Keywords: Transfusion-Dependent Thalassemia
Non-Transfusion-Dependent Thalassemia
Lead sponsor: Carelon Research
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2009-03-31
Enrollment: 252
Sex: ALL
Minimum age (years): 12
Maximum age (years): 90
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Thalassemia patients who receive regular blood transfusions and those who do not receive regular blood transfusions.

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.

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

Primary outcomes:
measure: Prevalence of Pain
timeFrame: Measured at Month 9
Secondary outcomes:
measure: Common Sites of Pain
timeFrame: Measured at Month 9
measure: Pain Occurrence by Age
timeFrame: Measured at Month 9
measure: Impact of Pain on Functioning and Well-being
timeFrame: Measured at Month 9
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Children's Hospital and Research Institute at Oakland
state: California
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Children's Memorial Hospital of Chicago
state: Illinois
city: Boston
country: United States
facility: Children's Hospital of Boston
state: Massachusetts
city: New York
country: United States
facility: Weill Medical College
state: New York
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
Study officials:
affiliation: Children's Healthcare of Atlanta
name: Jeanne Boudreeaux, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Boston Children's Hospital
name: Ellis Neufeld, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Memorial Hospital of Chicago
name: Alexis Thompson, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Baylor College of Medicine at Houston
name: Brigitta Mueller, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital and Research Institute of Oakland
name: Dru Foote, RN, NP
role: STUDY_CHAIR
affiliation: Weill Medical College of Cornell
name: Patricia Giardina, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital of Philadelphia
name: Janet Kwiatkowski, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Toronto General Hospital
name: Nancy Olivieri, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.