Assessment of Pain in People With Thalassemia

• Sponsor: HealthCore-NERI (Other)
• Overall Status: Completed
• CT.gov ID: NCT00872339
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI) (NIH)
• Enrollment: 252
• Locations: 8
• Duration (Months): 21
• Patients Per Site: 31.5
• Patients Per Site Per Month: 1.5

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
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 Design

• Study Type: Observational
• Actual Enrollment: 252 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Assessment of Pain in People With Thalassemia
• Study Start Date: Mar 1, 2009
• Actual Primary Completion Date: Jun 1, 2010
• Actual Study Completion Date: Dec 1, 2010

Arms and Interventions

Arm	Intervention/Treatment
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

Outcome Measures

Primary Outcome Measures

1. Prevalence of Pain [Measured at Month 9]

Secondary Outcome Measures

1. Common Sites of Pain [Measured at Month 9]

2. Pain Occurrence by Age [Measured at Month 9]

3. Impact of Pain on Functioning and Well-being [Measured at Month 9]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 12 Years to 90 Years
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital and Research Institute at Oakland	Oakland	California	United States	94609
2	Children's Healthcare of Atlanta	Atlanta	Georgia	United States	30342
3	Children's Memorial Hospital of Chicago	Chicago	Illinois	United States	60614
4	Children's Hospital of Boston	Boston	Massachusetts	United States	02115
5	Weill Medical College	New York	New York	United States	10065
6	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104
7	Baylor College of Medicine	Houston	Texas	United States	77030
8	Toronto General Hospital	Toronto	Ontario	Canada	M5G 2C4

Sponsors and Collaborators

• HealthCore-NERI
• 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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: HealthCore-NERI
• ClinicalTrials.gov Identifier: NCT00872339
• Other Study ID Numbers: 639; U01HL065238
• First Posted: Mar 31, 2009
• Last Update Posted: Jun 5, 2014
• Last Verified: Jan 1, 2014

• Keywords provided by HealthCore-NERI: Transfusion-Dependent Thalassemia; Non-Transfusion-Dependent Thalassemia
• Additional relevant MeSH terms: Thalassemia

Study Results

Participant Flow

Arm/Group Title	Transfusion-dependant	Intermittently Transfused	Non-transfusion-dependant
Arm/Group Description	People with transfusion-dependant thalassemia.	Intermittently transfused patients- individuals who received fewer than eight transfusions in the last year	People with non-transfusion-dependant thalassemia.
Period Title: Overall Study
STARTED	201	14	37
COMPLETED	201	14	37
NOT COMPLETED	0	0	0

Baseline Characteristics

Arm/Group Title	Transfusion-dependant	Intermittently Transfused	Non-transfusion-dependant	Total
Arm/Group Description	People with transfusion-dependant thalassemia.	Intermittently transfused patients- individuals who received fewer than eight transfusions in the last year	People with non-transfusion-dependant thalassemia.	Total of all reporting groups
Overall Participants	201	14	37	252
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	28.2 (10.4)	35.8 (14.4)	29.5 (16.2)	28.8 (11.8)
Sex: Female, Male (Count of Participants)
Female	117 58.2%	6 42.9%	14 37.8%	137 54.4%
Male	84 41.8%	8 57.1%	23 62.2%	115 45.6%

Outcome Measures

1. Primary Outcome

Title	Prevalence of Pain
Time Frame	Measured at Month 9

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Transfusion-dependant	Intermittently Transfused	Non-transfusion-dependant
Arm/Group Description	People with transfusion-dependant thalassemia.	Intermittently transfused patients- individuals who received fewer than eight transfusions in the last year	People with non-transfusion-dependant thalassemia.
Measure Participants	201	14	37
Number [participants]	75 37.3%	7 50%	11 29.7%

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Transfusion-dependant, Intermittently Transfused, Non-transfusion-dependant
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.45
	Comments
	Method	Chi-squared
	Comments

2. Secondary Outcome

Title	Common Sites of Pain
Time Frame	Measured at Month 9

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	All Subjects Combined
Arm/Group Description	Subjects with pain in the last 7 days were combined into one group.
Measure Participants	93
Lower back	76 37.8%
Either leg	52 25.9%
Head	45 22.4%
Midback	44 21.9%
Either hip	36 17.9%
Upper back	36 17.9%
Either arm	29 14.4%
Abdomen	26 12.9%
Chest	25 12.4%

3. Secondary Outcome

Title	Pain Occurrence by Age
Time Frame	Measured at Month 9

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	All Subjects Combined
Arm/Group Description	Subjects were combined into one group.
Measure Participants	252
12-17 yo (n=50)	4 2%
18-24 (n=56)	19 9.5%
25-34 (n=61)	22 10.9%
35-44 (n=61)	34 16.9%
45-71 (n=24)	14 7%

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Transfusion-dependant
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	<.001
	Comments
	Method	Chi-squared
	Comments

4. Secondary Outcome

Title	Impact of Pain on Functioning and Well-being
Time Frame	Measured at Month 9

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	All Subjects Combined
Arm/Group Description	Subjects with pain in the last 7 days were combined into one group.
Measure Participants	93
Mood	83 41.3%
Normal work	76 37.8%
General activities	74 36.8%
Sleep	74 36.8%
Walking	73 36.3%
Enjoyment of life	69 34.3%
Interpersonal relations	57 28.4%

Adverse Events

Time Frame	Adverse events were not collected for this study.
Arm/Group Title	Adverse Events Were Not Collected
Arm/Group Description	Adverse events were not collected for this study.
All Cause Mortality
	Adverse Events Were Not Collected
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Adverse Events Were Not Collected
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)
Other (Not Including Serious) Adverse Events
	Adverse Events Were Not Collected
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.