Trial Outcomes & Findings for Continuous Positive Airway Pressure (CPAP) After Adenotonsillectomy in Children (NCT NCT01554527)

Last Updated: 2019-01-14

Results Overview

The sum of the T-scores of The Conners' Parent Rating Scales (CPRS-R:L, ADHD index) and the Child Behavior Checklist (CBCL, Attention Deficit/Hyperactivity Problems) are used to construct the primary study outcome measure Behavioral Index. Behavioral index is a T score (adjusted for age and gender) with a range of "\<10" to "\>90" - where higher scores mean worse behavior and lower scores mean better behavior, so a negative change score represents an improvement in behavior. T-scores with a mean of 50 and SD of 10 are computed.

Recruitment status

COMPLETED

Study phase

Target enrollment

120 participants

Primary outcome timeframe

assessed as change from baseline to 6 months of CPAP therapy or no-CPAP

Results posted on

2019-01-14

Participant Flow

Of 120 consented, 105 were randomized to CPAP or control. (Of the 15 who weren't randomized, 8 were lost to follow up; the other withdrew for various reasons)

Participant milestones

Participant milestones
Measure	Control Group (Surgery Only - no CPAP) The participants who were not assigned to CPAP	CPAP After Surgery (Adherent) Those participants assigned to CPAP who had at least 4 hours of electronically recorded CPAP use per night	CPAP After Surgery (Non-adherent) Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night.
Overall Study STARTED	34	38	33
Overall Study Crossed Over to Control	0	0	2
Overall Study Had CPAP Titration	0	38	30
Overall Study Initiated CPAP Treatment at Home	0	38	28
Overall Study Completed as a Control	33	0	1
Overall Study COMPLETED	33	38	25
Overall Study NOT COMPLETED	1	0	8

Reasons for withdrawal

Reasons for withdrawal
Measure	Control Group (Surgery Only - no CPAP) The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night.
Overall Study Withdrawal by Subject	1	3
Overall Study Lost to Follow-up	0	4
Overall Study Physician Decision	0	1

Baseline Characteristics

Behavioral Index was not available for one control

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Control Group (Surgery Only - no CPAP) n=34 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Adherent) n=38 Participants Those participants assigned to CPAP who had at least 4 hours of electronically recorded CPAP use per night	CPAP After Surgery (Non-adherent) n=33 Participants Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night.	Total n=105 Participants Total of all reporting groups
Age, Continuous	7.9 years STANDARD_DEVIATION 2.0 • n=34 Participants	8.3 years STANDARD_DEVIATION 2.2 • n=38 Participants	8.0 years STANDARD_DEVIATION 2.0 • n=33 Participants	8.1 years STANDARD_DEVIATION 2.1 • n=105 Participants
Sex: Female, Male Female	13 Participants n=34 Participants	21 Participants n=38 Participants	16 Participants n=33 Participants	50 Participants n=105 Participants
Sex: Female, Male Male	21 Participants n=34 Participants	17 Participants n=38 Participants	17 Participants n=33 Participants	55 Participants n=105 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	5 Participants n=34 Participants	2 Participants n=38 Participants	0 Participants n=33 Participants	7 Participants n=105 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	29 Participants n=34 Participants	36 Participants n=38 Participants	33 Participants n=33 Participants	98 Participants n=105 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=34 Participants	0 Participants n=38 Participants	0 Participants n=33 Participants	0 Participants n=105 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=34 Participants	0 Participants n=38 Participants	0 Participants n=33 Participants	0 Participants n=105 Participants
Race (NIH/OMB) Asian	1 Participants n=34 Participants	1 Participants n=38 Participants	1 Participants n=33 Participants	3 Participants n=105 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=34 Participants	0 Participants n=38 Participants	0 Participants n=33 Participants	0 Participants n=105 Participants
Race (NIH/OMB) Black or African American	1 Participants n=34 Participants	5 Participants n=38 Participants	8 Participants n=33 Participants	14 Participants n=105 Participants
Race (NIH/OMB) White	28 Participants n=34 Participants	29 Participants n=38 Participants	19 Participants n=33 Participants	76 Participants n=105 Participants
Race (NIH/OMB) More than one race	4 Participants n=34 Participants	3 Participants n=38 Participants	5 Participants n=33 Participants	12 Participants n=105 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=34 Participants	0 Participants n=38 Participants	0 Participants n=33 Participants	0 Participants n=105 Participants
Region of Enrollment United States	34 Participants n=34 Participants	38 Participants n=38 Participants	33 Participants n=33 Participants	105 Participants n=105 Participants
Behavioral Index	61.6 units on a scale (T-Score) STANDARD_DEVIATION 13.4 • n=33 Participants • Behavioral Index was not available for one control	56.5 units on a scale (T-Score) STANDARD_DEVIATION 11.3 • n=38 Participants • Behavioral Index was not available for one control	57.8 units on a scale (T-Score) STANDARD_DEVIATION 11.6 • n=33 Participants • Behavioral Index was not available for one control	58.5 units on a scale (T-Score) STANDARD_DEVIATION 12.1 • n=104 Participants • Behavioral Index was not available for one control
NIH Toolbox Age adjusted composite score	95.7 units on a scale STANDARD_DEVIATION 11.9 • n=34 Participants • Subject ages too young for this particular test so their data are not included	96.2 units on a scale STANDARD_DEVIATION 12.3 • n=38 Participants • Subject ages too young for this particular test so their data are not included	93.9 units on a scale STANDARD_DEVIATION 11.6 • n=29 Participants • Subject ages too young for this particular test so their data are not included	95.1 units on a scale STANDARD_DEVIATION 11.6 • n=101 Participants • Subject ages too young for this particular test so their data are not included
Epworth Sleepiness Scale	7.2 units on a scale STANDARD_DEVIATION 4.6 • n=34 Participants • Epworth sleepiness scale score missing for one participant	6.9 units on a scale STANDARD_DEVIATION 3.9 • n=37 Participants • Epworth sleepiness scale score missing for one participant	6.4 units on a scale STANDARD_DEVIATION 3.0 • n=33 Participants • Epworth sleepiness scale score missing for one participant	6.8 units on a scale STANDARD_DEVIATION 3.9 • n=104 Participants • Epworth sleepiness scale score missing for one participant
Multiple Sleep Latency Test (MSLT)	24.7 minutes STANDARD_DEVIATION 6.0 • n=34 Participants	25.9 minutes STANDARD_DEVIATION 4.6 • n=38 Participants	24.4 minutes STANDARD_DEVIATION 5.4 • n=33 Participants	25.0 minutes STANDARD_DEVIATION 5.3 • n=105 Participants
Quality of Life	78.2 units on a scale STANDARD_DEVIATION 13.7 • n=34 Participants	78.7 units on a scale STANDARD_DEVIATION 16 • n=38 Participants	77.3 units on a scale STANDARD_DEVIATION 17.2 • n=33 Participants	78.1 units on a scale STANDARD_DEVIATION 14.9 • n=105 Participants
Academic Achievement Score	104.1 units on a scale STANDARD_DEVIATION 13.4 • n=34 Participants	107.2 units on a scale STANDARD_DEVIATION 14.6 • n=38 Participants	102.1 units on a scale STANDARD_DEVIATION 10.7 • n=33 Participants	104.6 units on a scale STANDARD_DEVIATION 13.1 • n=105 Participants
Fluid Cognition Composite Age Adjusted score	97.3 units on a scale STANDARD_DEVIATION 13.2 • n=34 Participants • Subject age was too young for this particular test, so their data is not included.	96 units on a scale STANDARD_DEVIATION 14.2 • n=38 Participants • Subject age was too young for this particular test, so their data is not included.	93.9 units on a scale STANDARD_DEVIATION 11.6 • n=30 Participants • Subject age was too young for this particular test, so their data is not included.	95.8 units on a scale STANDARD_DEVIATION 13.1 • n=102 Participants • Subject age was too young for this particular test, so their data is not included.
(Apnea-Hypopnea Index (AHI)	2.4 events/hour STANDARD_DEVIATION 2.0 • n=34 Participants	2.2 events/hour STANDARD_DEVIATION 1.7 • n=38 Participants	2.1 events/hour STANDARD_DEVIATION 2.0 • n=33 Participants	2.2 events/hour STANDARD_DEVIATION 1.9 • n=105 Participants
Sleep Related Breathing Disturbances (SBRD) sub-scale of Pediatric Sleep Questionnaire	0.36 Ratio STANDARD_DEVIATION 0.24 • n=34 Participants	0.32 Ratio STANDARD_DEVIATION 0.21 • n=38 Participants	0.37 Ratio STANDARD_DEVIATION 0.17 • n=33 Participants	0.35 Ratio STANDARD_DEVIATION 0.21 • n=105 Participants

PRIMARY outcome

Timeframe: assessed as change from baseline to 6 months of CPAP therapy or no-CPAP

Population: Data for two participants in the control group are not available.

Outcome measures

Outcome measures
Measure	Control Group (Surgery Only - no CPAP) n=32 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) n=25 Participants Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period	CPAP After Surgery (Adherent) n=38 Participants Those participants assigned to CPAP who had an average of at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period
Change in Behavioral Index After 6 Months of CPAP or No-CPAP	-2.7 T score Standard Deviation 5.8	.9 T score Standard Deviation 4.8	-1.2 T score Standard Deviation 6.3

SECONDARY outcome

Timeframe: assessed as change from baseline to 6 months of CPAP therapy or no-CPAP

Population: Lower numbers of participants analyzed are shown here because the tool is not appropriate for younger children

Scores are reported as standardized scores with a mean of 100 and SD of 15. Minimum value 40 and Maximum value 160. Higher scores are better

Outcome measures

Outcome measures
Measure	Control Group (Surgery Only - no CPAP) n=27 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) n=23 Participants Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period	CPAP After Surgery (Adherent) n=38 Participants Those participants assigned to CPAP who had an average of at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period
Change in Cognition as Shown by NIH Toolbox Composite Score	5.5 score on a scale Standard Deviation 9.5	2.8 score on a scale Standard Deviation 9.8	3.9 score on a scale Standard Deviation 7.8

SECONDARY outcome

Timeframe: assessed as change from baseline to 6 months of CPAP therapy or no-CPAP

Population: Data for two participants are not available

Epworth Sleepiness Scale scores range from 0 to 24 where higher scores mean greater sleepiness.

Outcome measures

Outcome measures
Measure	Control Group (Surgery Only - no CPAP) n=33 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) n=25 Participants Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period	CPAP After Surgery (Adherent) n=37 Participants Those participants assigned to CPAP who had an average of at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period
Change in Sleepiness as Measured by Epworth Sleepiness Scale	-1.6 score on a scale Standard Deviation 3.7	-1.0 score on a scale Standard Deviation 2.2	-1.9 score on a scale Standard Deviation 3.8

SECONDARY outcome

Timeframe: assessed as change from baseline to 6 months of CPAP therapy or no-CPAP

Population: Data for one participant are not available

Multiple Sleep Latency Test (MSLT) is an objective measure of sleepiness determined by measure of brain waves and other physiological signals over a 30 minute period. This is measured for five 30 minute periods across the day and average latency to sleep for each participant across those times were used to calculate the mean sleep latency. The score is measured in how quickly one would fall asleep, measured in minutes, so a negative number of minutes in the change score means that participants fell asleep more quickly than previously.

Outcome measures

Outcome measures
Measure	Control Group (Surgery Only - no CPAP) n=33 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) n=25 Participants Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period	CPAP After Surgery (Adherent) n=38 Participants Those participants assigned to CPAP who had an average of at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period
Change in Sleepiness After AT as Measured by Multiple Sleep Latency Test (MSLT)	-1.8 minutes Standard Deviation 4	-1.3 minutes Standard Deviation 5.1	-1.1 minutes Standard Deviation 4.3

SECONDARY outcome

Timeframe: assessed as change from baseline to 6 months of CPAP therapy or no-CPAP

Population: Data for one participant is not available

Peds-QL is a quality of life symptom measurement with a score range of 0 to 100. Higher scores are better quality of life

Outcome measures

Outcome measures
Measure	Control Group (Surgery Only - no CPAP) n=33 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) n=25 Participants Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period	CPAP After Surgery (Adherent) n=38 Participants Those participants assigned to CPAP who had an average of at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period
Change in Quality of Life as Measured by Peds QL	4.1 score on a scale Standard Deviation 9.0	0.5 score on a scale Standard Deviation 10.2	2.4 score on a scale Standard Deviation 12.1

SECONDARY outcome

Timeframe: Starting at 4 months after AT and continuing through 10 months after AT

CPAP adherence data will be downloaded from CPAP machines. It is defined for this study as using the CPAP machine for at least an average of 4 hours per night during the last 60 days of the assignment.

Outcome measures

Outcome measures
Measure	Control Group (Surgery Only - no CPAP) n=71 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period	CPAP After Surgery (Adherent) Those participants assigned to CPAP who had an average of at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period
CPAP Adherence as Measured by Number of Participants Who Used the CPAP Consistently.	38 Participants	—	—

OTHER_PRE_SPECIFIED outcome

Timeframe: assessed as change from baseline to 6 months of CPAP therapy or no-CPAP

Population: Lower numbers are shown here as the tool is not appropriate for younger children

Scores are reported as standardized scores with a mean of 100 and SD of 15. Minimum value 40, Maximum value 160. Higher scores are better

Outcome measures

Outcome measures
Measure	Control Group (Surgery Only - no CPAP) n=29 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) n=23 Participants Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period	CPAP After Surgery (Adherent) n=38 Participants Those participants assigned to CPAP who had an average of at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period
Change in Cognition as Measured by Fluid Cognition Scores	6.7 score on a scale Standard Deviation 12.4	1.7 score on a scale Standard Deviation 11.5	3.4 score on a scale Standard Deviation 10.1

OTHER_PRE_SPECIFIED outcome

Timeframe: assessed as change from baseline to 6 months of CPAP therapy or no-CPAP

Population: Data from two participants are not available

Academic achievement mean score is a standardized score with a mean of 100 and SD 15. Minimum value 40 and Maximum value 160. Higher scores are better

Outcome measures

Outcome measures
Measure	Control Group (Surgery Only - no CPAP) n=32 Participants The participants who were not assigned to CPAP	CPAP After Surgery (Non-adherent) n=25 Participants Those participants assigned CPAP who did not have at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period	CPAP After Surgery (Adherent) n=37 Participants Those participants assigned to CPAP who had an average of at least 4 hours of electronically recorded CPAP use per night over the last 60 days of the 6 month usage period
Change in Cognition After AT as Shown by Academic Achievement	-1.0 units on a scale Standard Deviation 5.6	1.1 units on a scale Standard Deviation 4.7	-2.5 units on a scale Standard Deviation 8.6

Adverse Events

CPAP Treatment Group on CPAP

Serious events: 6 serious events

Other events: 59 other events

Deaths: 0 deaths

No CPAP Treatment Group

Serious events: 7 serious events

Other events: 26 other events

Deaths: 0 deaths

Non-randomized Participants

Serious events: 4 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	CPAP Treatment Group on CPAP n=69 participants at risk Children randomized to this arm were assigned and began up to 6 months of CPAP treatment, beginning at approximately 4 months after AT, in addition to standard of care. This arm shows AEs that happened to participants randomized to CPAP but who didn't cross over to the No CPAP group prior to sleep lab. (n=69; original n=71 minus n=2 who crossed over to no-CPAP group). Since both adherent and non-adherent CPAP participants received CPAP and had at minimum one night of CPAP exposure, these two groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.	No CPAP Treatment Group n=36 participants at risk Children shown in this comparison arm were randomized to NO CPAP or were crossed over to No CPAP prior to any CPAP use or CPAP trial in sleep lab.) (N = 36, original 34 plus two crossed over from those randomized to CPAP)	Non-randomized Participants n=15 participants at risk Participants who were consented but withdrew prior to randomization (n = 15)
Respiratory, thoracic and mediastinal disorders Hospitalization for ashthma	1.4% 1/69 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.	0.00% 0/36 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.	0.00% 0/15 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.
Surgical and medical procedures Post-operative hospitalization	5.8% 4/69 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.	19.4% 7/36 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.	26.7% 4/15 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.
Psychiatric disorders ER admission due to panic attack	1.4% 1/69 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.	0.00% 0/36 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.	0.00% 0/15 • Adverse events were followed from consent (which could be before surgery or after surgery) until 10 months after surgery equating to six months after assignment. Based on the adverse event collection structure, it bears noting that some reported AEs may have happened prior to assignment to CPAP or no CPAP use, to participants who ended up reaching baseline visit (4 months after surgery). Since both adherent and non-adherent CPAP participants received CPAP with minimum 1 night of CPAP, these 2 groups were combined for the purposes of AE reporting in order to determine whether any CPAP exposure vs control group (no CPAP) had a different proportion of AE's.

Other adverse events

Additional Information

Dr. Ronald Chervin

University of Michigan

Phone: 734-647-9064

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place