Thalassemia
Conditions
Keywords
Transfusion-Dependant Thalassemia, Pain, Mild Pain
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.
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
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Report of Pain by Age Group | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Report of Pain by Length of the Transfusion Cycle | 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. |
Countries
Canada, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Overall Both cohorts combined | 32 |
| Total | 32 |
Baseline characteristics
| Characteristic | Overall |
|---|---|
| Age, Continuous | 31.8 years STANDARD_DEVIATION 10.5 |
| Sex: Female, Male Female | 18 Participants |
| Sex: Female, Male Male | 14 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
Report of Pain by Age Group
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.
Time frame: measured daily over the 3 transfusion cycles
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Younger Cohort | Report of Pain by Age Group | 25% through cycle | 18 percentage of participant-cycles |
| Younger Cohort | Report of Pain by Age Group | 75% through cycle | 20 percentage of participant-cycles |
| Younger Cohort | Report of Pain by Age Group | 50% through cycle | 15 percentage of participant-cycles |
| Younger Cohort | Report of Pain by Age Group | End of cycle | 18 percentage of participant-cycles |
| Younger Cohort | Report of Pain by Age Group | Start of cycle | 23 percentage of participant-cycles |
| Older Cohort | Report of Pain by Age Group | End of cycle | 56 percentage of participant-cycles |
| Older Cohort | Report of Pain by Age Group | Start of cycle | 61 percentage of participant-cycles |
| Older Cohort | Report of Pain by Age Group | 25% through cycle | 51 percentage of participant-cycles |
| Older Cohort | Report of Pain by Age Group | 50% through cycle | 47 percentage of participant-cycles |
| Older Cohort | Report of Pain by Age Group | 75% through cycle | 54 percentage of participant-cycles |
p-value: <0.001Chi-squared
Secondary
Report of Pain by Length of the Transfusion Cycle
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.
Time frame: measured daily over the 3 transfusion cycles
Population: Unit of analysis was participant-cycle. Data were collected over (at most) three transfusion cycles for each subject. Transfusion cycle lengths varied, and subjects may be represented in more than one arm/group.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Younger Cohort | Report of Pain by Length of the Transfusion Cycle | 75% through cycle | 60 percentage of participant-cycles |
| Younger Cohort | Report of Pain by Length of the Transfusion Cycle | 25% through cycle | 50 percentage of participant-cycles |
| Younger Cohort | Report of Pain by Length of the Transfusion Cycle | End of cycle | 50 percentage of participant-cycles |
| Younger Cohort | Report of Pain by Length of the Transfusion Cycle | 50% through cycle | 50 percentage of participant-cycles |
| Younger Cohort | Report of Pain by Length of the Transfusion Cycle | Start of cycle | 50 percentage of participant-cycles |
| Older Cohort | Report of Pain by Length of the Transfusion Cycle | 50% through cycle | 48 percentage of participant-cycles |
| Older Cohort | Report of Pain by Length of the Transfusion Cycle | 75% through cycle | 52 percentage of participant-cycles |
| Older Cohort | Report of Pain by Length of the Transfusion Cycle | End of cycle | 44 percentage of participant-cycles |
| Older Cohort | Report of Pain by Length of the Transfusion Cycle | 25% through cycle | 48 percentage of participant-cycles |
| Older Cohort | Report of Pain by Length of the Transfusion Cycle | Start of cycle | 40 percentage of participant-cycles |
| 22 - 28 Days | Report of Pain by Length of the Transfusion Cycle | 50% through cycle | 23 percentage of participant-cycles |
| 22 - 28 Days | Report of Pain by Length of the Transfusion Cycle | Start of cycle | 42 percentage of participant-cycles |
| 22 - 28 Days | Report of Pain by Length of the Transfusion Cycle | 25% through cycle | 35 percentage of participant-cycles |
| 22 - 28 Days | Report of Pain by Length of the Transfusion Cycle | 75% through cycle | 31 percentage of participant-cycles |
| 22 - 28 Days | Report of Pain by Length of the Transfusion Cycle | End of cycle | 31 percentage of participant-cycles |
| > 28 Days | Report of Pain by Length of the Transfusion Cycle | 75% through cycle | 18 percentage of participant-cycles |
| > 28 Days | Report of Pain by Length of the Transfusion Cycle | 25% through cycle | 14 percentage of participant-cycles |
| > 28 Days | Report of Pain by Length of the Transfusion Cycle | Start of cycle | 41 percentage of participant-cycles |
| > 28 Days | Report of Pain by Length of the Transfusion Cycle | 50% through cycle | 14 percentage of participant-cycles |
| > 28 Days | Report of Pain by Length of the Transfusion Cycle | End of cycle | 32 percentage of participant-cycles |
p-value: 0.028Chi-squared