Thalassemia
Conditions
Keywords
Transfusion-Dependent Thalassemia, Non-Transfusion-Dependent 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.
Interventions
None listed
Sponsors
National Heart, Lung, and Blood Institute (NHLBI)
Carelon Research
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to 90 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Prevalence of Pain | Measured at Month 9 |
Secondary
| 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 |
Countries
Canada, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Transfusion-dependant People with transfusion-dependant thalassemia. | 201 |
| Intermittently Transfused Intermittently transfused patients- individuals who received fewer than eight transfusions in the last year | 14 |
| Non-transfusion-dependant People with non-transfusion-dependant thalassemia. | 37 |
| Total | 252 |
Baseline characteristics
| Characteristic | Intermittently Transfused | Transfusion-dependant | Non-transfusion-dependant | Total |
|---|---|---|---|---|
| Age, Continuous | 35.8 years STANDARD_DEVIATION 14.4 | 28.2 years STANDARD_DEVIATION 10.4 | 29.5 years STANDARD_DEVIATION 16.2 | 28.8 years STANDARD_DEVIATION 11.8 |
| Sex: Female, Male Female | 6 Participants | 117 Participants | 14 Participants | 137 Participants |
| Sex: Female, Male Male | 8 Participants | 84 Participants | 23 Participants | 115 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Prevalence of Pain
Time frame: Measured at Month 9
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Transfusion-dependant | Prevalence of Pain | 75 participants |
| Intermittently Transfused | Prevalence of Pain | 7 participants |
| Non-transfusion-dependant | Prevalence of Pain | 11 participants |
p-value: 0.45Chi-squared
Secondary
Common Sites of Pain
Time frame: Measured at Month 9
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Transfusion-dependant | Common Sites of Pain | Lower back | 76 participants |
| Transfusion-dependant | Common Sites of Pain | Either leg | 52 participants |
| Transfusion-dependant | Common Sites of Pain | Head | 45 participants |
| Transfusion-dependant | Common Sites of Pain | Midback | 44 participants |
| Transfusion-dependant | Common Sites of Pain | Either hip | 36 participants |
| Transfusion-dependant | Common Sites of Pain | Upper back | 36 participants |
| Transfusion-dependant | Common Sites of Pain | Either arm | 29 participants |
| Transfusion-dependant | Common Sites of Pain | Abdomen | 26 participants |
| Transfusion-dependant | Common Sites of Pain | Chest | 25 participants |
Secondary
Impact of Pain on Functioning and Well-being
Time frame: Measured at Month 9
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Transfusion-dependant | Impact of Pain on Functioning and Well-being | Mood | 83 participants |
| Transfusion-dependant | Impact of Pain on Functioning and Well-being | Normal work | 76 participants |
| Transfusion-dependant | Impact of Pain on Functioning and Well-being | General activities | 74 participants |
| Transfusion-dependant | Impact of Pain on Functioning and Well-being | Sleep | 74 participants |
| Transfusion-dependant | Impact of Pain on Functioning and Well-being | Walking | 73 participants |
| Transfusion-dependant | Impact of Pain on Functioning and Well-being | Enjoyment of life | 69 participants |
| Transfusion-dependant | Impact of Pain on Functioning and Well-being | Interpersonal relations | 57 participants |
Secondary
Pain Occurrence by Age
Time frame: Measured at Month 9
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Transfusion-dependant | Pain Occurrence by Age | 25-34 (n=61) | 22 participants |
| Transfusion-dependant | Pain Occurrence by Age | 35-44 (n=61) | 34 participants |
| Transfusion-dependant | Pain Occurrence by Age | 45-71 (n=24) | 14 participants |
| Transfusion-dependant | Pain Occurrence by Age | 12-17 yo (n=50) | 4 participants |
| Transfusion-dependant | Pain Occurrence by Age | 18-24 (n=56) | 19 participants |
p-value: <0.001Chi-squared