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

• ClinicalTrials.gov Identifier: NCT00872833
• Recruitment Status: Completed
• First Posted: March 31, 2009
• Results First Posted: August 27, 2014
• Last Update Posted: October 7, 2015
• Sponsor: HealthCore-NERI
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI)
• Information provided by (Responsible Party): HealthCore-NERI

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Condition: Thalassemia
• Enrollment: 32

Participant Flow

Recruitment Details
Pre-assignment Details

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 Number of Baseline Participants		32
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	32 participants
		31.8 (10.5)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	32 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.
Overall Number of Participants Analyzed	15	17
Overall Number of Units Analyzed; Type of Units Analyzed: Participant-cycles	40	43
Measure Type: Number; Unit of Measure: percentage of participant-cycles
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	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	<.001
	Comments	[Not Specified]
	Method	Chi-squared
	Comments	[Not Specified]

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
Overall Number of Participants Analyzed	5	12	17	13
Overall Number of Units Analyzed; Type of Units Analyzed: Participant-cycles	10	25	26	22
Measure Type: Number; Unit of Measure: percentage of participant-cycles
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	<= 14 Days, 15 - 21 Days, 22 - 28 Days, > 28 Days
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.028
	Comments	[Not Specified]
	Method	Chi-squared
	Comments	[Not Specified]

Adverse Events

Time Frame	[Not Specified]
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 (%)
Total	--/--
Serious Adverse Events
	Adverse Events Were Not Collected
	Affected / at Risk (%)
Total	0/0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Adverse Events Were Not Collected
	Affected / at Risk (%)
Total	0/0

Limitations and 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.

Results Point of Contact

• Name/Title: Eric Gerstenberger
• Organization: New England Research Institutes, Inc.
• Phone: 6179237747
• EMail: egerstenberger@neriscience.com

• Responsible Party: HealthCore-NERI
• ClinicalTrials.gov Identifier: NCT00872833
• Other Study ID Numbers: 640; U01HL065238 ( U.S. NIH Grant/Contract )
• First Submitted: March 30, 2009
• First Posted: March 31, 2009
• Results First Submitted: April 9, 2014
• Results First Posted: August 27, 2014
• Last Update Posted: October 7, 2015