Trial Outcomes & Findings for Hemodynamics Response to Upper Airway Obstruction in Marfan Syndrome (NCT NCT03985657)

Results Overview

Pleural pressure (Pes) in mmHg monitored using an esophageal catheter.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

31 participants

Primary outcome timeframe

Overnight on both CPAP and No CPAP nights

Results posted on

2020-12-28

Participant Flow

193 persons with Marfan syndrome were screened. Persons were recruited from the Johns Hopkins Marfan Clinic and the external Marfan Community via the Marfan foundation. Inclusion was based on a clinical diagnosis of Marfan syndrome (Ghent Criteria) or genetic testing. Persons were excluded if they had any chronic lung disease, other sleep disorders aside from sleep apnea, were on blood thinners, or had uncontrolled blood pressure. Recruitment occurred between 09/2018 and 11/2019.

Participant milestones

Participant milestones
Measure	Baseline Sleep Study Baseline sleep polysomnography involved the monitoring of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP. Participants in this arm would switch to CPAP within one week of the Baseline Sleep Study.	CPAP Sleep Study In a randomized cross-over fashion, participants were treated with continuous positive airway pressure (CPAP) on a separate night. Room air at pressures between 6-9 centimeters of water (cmH2O) were delivered via heated humidified tubing and a nasal mask. Participants in this arm would switch to Baseline Sleep Study within one week of the CPAP Sleep Study.
Intervention 1 (One Night) STARTED	20	11
Intervention 1 (One Night) COMPLETED	20	10
Intervention 1 (One Night) NOT COMPLETED	0	1
Washout (Up to 1 Week) STARTED	20	10
Washout (Up to 1 Week) COMPLETED	20	10
Washout (Up to 1 Week) NOT COMPLETED	0	0
Intervention 2 (One Night) STARTED	20	10
Intervention 2 (One Night) COMPLETED	20	10
Intervention 2 (One Night) NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Participants n=31 Participants All participants enrolled in this study.
Age, Continuous	37.5 years STANDARD_DEVIATION 15.9 • n=31 Participants
Sex: Female, Male Female	17 Participants n=31 Participants
Sex: Female, Male Male	14 Participants n=31 Participants
Region of Enrollment United States	31 Participants n=31 Participants

PRIMARY outcome

Timeframe: Overnight on both CPAP and No CPAP nights

Population: Participants with upper airway obstruction (apneas, hypopneas, and flow limitation) during the Baseline sleep study, who had pleural pressure and blood pressure data collected during both Baseline and CPAP sleep studies.

Pleural pressure (Pes) in mmHg monitored using an esophageal catheter.

Outcome measures

Outcome measures
Measure	Baseline Sleep Study n=10 Participants Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.	CPAP Sleep Study n=10 Participants Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. CPAP: Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.
Pleural Pressure (Pes)	9.3 mmHg Standard Deviation 3.4	4.4 mmHg Standard Deviation 1.6

PRIMARY outcome

Timeframe: Overnight on both Baseline and CPAP studies

Population: Participants with upper airway obstruction (apneas, hypopneas, and flow limitation) during the Baseline sleep study, who had pleural pressure and blood pressure data collected during both Baseline and CPAP sleep studies.

Continuous blood pressure monitored using a non-invasive finger cuff. Data captured in mmHg.

Outcome measures

Outcome measures
Measure	Baseline Sleep Study n=10 Participants Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.	CPAP Sleep Study n=10 Participants Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. CPAP: Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.
Mean Arterial Blood Pressure (MAP)	93.8 mmHg Standard Deviation 14.0	88.3 mmHg Standard Deviation 12.4

SECONDARY outcome

Timeframe: 15 minutes in the morning post Baseline and CPAP studies

Population: Participants with adequate sleep time (\>2hrs) on both Baseline and CPAP studies and AI measurements after both Baseline and CPAP studies.

The AI (measured as "percentage of pulse pressure") was assessed in the morning after both Baseline and CPAP studies. It is a measure of arterial stiffness that represents the degree of and can range from -10% to +10% in healthy individuals, with values generally higher in females and increases with age.

Outcome measures

Outcome measures
Measure	Baseline Sleep Study n=28 Participants Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.	CPAP Sleep Study n=28 Participants Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. CPAP: Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.
Augmentation Index (AI)	11.0 percentage of pulse pressure Standard Error 3.8	2.4 percentage of pulse pressure Standard Error 3.7

SECONDARY outcome

Timeframe: 15 minutes

Population: Participants with adequate sleep time (\>2hrs) on both Baseline and CPAP studies and morning RHI measurements after both Baseline and CPAP studies.

The RHI is a measure of endothelial function that was measured in the morning after both Baseline and CPAP studies. It is unitless and scored on a range of 1 to 3 in healthy individuals with lower values indicating poor endothelial function.

Outcome measures

Outcome measures
Measure	Baseline Sleep Study n=26 Participants Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.	CPAP Sleep Study n=26 Participants Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. CPAP: Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.
Reactive Hyperemia Index (RHI)	2.2 score on a scale Standard Error 0.1	2.2 score on a scale Standard Error 0.2

Adverse Events

Baseline Sleep Study

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

CPAP Sleep Study

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Mudiaga Sowho

Johns Hopkins University

Phone: 4105506264

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place