Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions

• Sponsor: HealthCore-NERI (Other)
• Overall Status: Completed
• CT.gov ID: NCT00872833
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI) (NIH)
• Enrollment: 32
• Locations: 9
• Duration (Months): 15
• Patients Per Site: 3.6
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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.

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. 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.

Study Design

• Study Type: Observational
• Actual Enrollment: 32 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Assessment of Pain in Transfusion Dependent Patients With Thalassemia During Transfusion Cycles
• Study Start Date: Jul 1, 2009
• Actual Primary Completion Date: Oct 1, 2010
• Actual Study Completion Date: Oct 1, 2010

Arms and Interventions

Arm	Intervention/Treatment
age 18-29 People age groups 18-29 with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study.
age 30+ People age groups 30+ years with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study.

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital of Los Angeles	Los Angeles	California	United States	90027
2	Children's Hospital Research Center Oakland	Oakland	California	United States	94609
3	Stanford University - School of Medicine	Stanford	California	United States	94305
4	Children's Healthcare of Atlanta	Atlanta	Georgia	United States	30342
5	Children's Memorial Hospital - Chicago	Chicago	Illinois	United States	60614
6	Weill Medical College of Cornell	New York	New York	United States	10065
7	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104
8	Baylor College of Medicine	Houston	Texas	United States	77030
9	University Health Network - Toronto General Hospital	Toronto	Ontario	Canada	M5G 2C4

Sponsors and Collaborators

• HealthCore-NERI
• National Heart, Lung, and Blood Institute (NHLBI)

Investigators

• Principal Investigator: Dru Foote, NP, Children's Hospital and Research Institute Oakland
• Study Chair: Jeanne Boudreaux, MD, Children's Healthcare of Atlanta
• Study Chair: Thomas Coates, MD, Children's Hospital Los Angeles
• Study Chair: Elliott Vichinsky, MD, UCSF Benioff Children's Hospital Oakland
• Study Chair: Michael Jeng, MD, Stanford University
• Study Chair: Janet Kwiatkowski, MD, Children's Hospital of Philadelphia
• Study Chair: Nancy Olivieri, MD, University Health Network - Toronto General Hospital
• Study Chair: Patricia J. Giardina, MD, Weill Medical College of Cornell
• Study Chair: Brigitta Mueller, MD, Baylor College of Medicine
• Study Chair: Alexis A. Thompson, MD, MPH, Ann & Robert H Lurie Children's Hospital of Chicago

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: HealthCore-NERI
• ClinicalTrials.gov Identifier: NCT00872833
• Other Study ID Numbers: 640; U01HL065238
• First Posted: Mar 31, 2009
• Last Update Posted: Oct 7, 2015
• Last Verified: Apr 1, 2012

• Keywords provided by HealthCore-NERI: Transfusion-Dependant Thalassemia; Pain; Mild Pain
• Additional relevant MeSH terms: Thalassemia

Study Results

Participant Flow

Arm/Group Title	Younger Cohort	Older Cohort
Arm/Group Description	People age groups 18-29 with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study.	People age groups 30+ years with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study.
Period Title: Overall Study
STARTED	15	17
COMPLETED	15	17
NOT COMPLETED	0	0

Baseline Characteristics

Arm/Group Title	Overall
Arm/Group Description	Both cohorts combined
Overall Participants	32
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	31.8 (10.5)
Sex: Female, Male (Count of Participants)
Female	18 56.3%
Male	14 43.8%

Outcome Measures

1. Primary Outcome

Title	Report of Pain by Age Group
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Younger Cohort	Older Cohort
Arm/Group Description	People age groups 18-29 with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study.	People age groups 30+ years with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study.
Measure Participants	15	17
Measure Participant-cycles	40	43
Start of cycle	23	61
25% through cycle	18	51
50% through cycle	15	47
75% through cycle	20	54
End of cycle	18	56

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Younger Cohort, Older Cohort
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	<.001
	Comments
	Method	Chi-squared
	Comments

2. Secondary Outcome

Title	Report of Pain by Length of the Transfusion Cycle
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	<= 14 Days	15 - 21 Days	22 - 28 Days	> 28 Days
Arm/Group Description	Subjects with transfusion cycles of 14 days or less	Subjects with transfusion cycles between 15 and 21 days	Subjects with transfusion cycles between 22 and 28 days	Subjects with transfusion cycles greater than 28 days
Measure Participants	5	12	17	13
Measure Participant-cycles	10	25	26	22
Start of cycle	50	40	42	41
25% through cycle	50	48	35	14
50% through cycle	50	48	23	14
75% through cycle	60	52	31	18
End of cycle	50	44	31	32

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Younger Cohort, Older Cohort, 22 - 28 Days, > 28 Days
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.028
	Comments
	Method	Chi-squared
	Comments

Adverse Events

Adverse Event Reporting Description	Adverse event data were not collected for this observational study
Arm/Group Title	Adverse Events Were Not Collected
Arm/Group Description	Adverse events were not collected as part of this observational 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.