Trial Outcomes & Findings for Diagnosis and Monitoring of Eosinophilic Esophagitis Using the Cytosponge (NCT NCT02114606)

Results Overview

The primary outcome variables are sensitivity (percent agreement between positive results) and specificity (percent agreement between negative results) of the Cytosponge ability to detect the presence of EoE as compared to upper endoscopy with biopsy (the gold standard for diagnosis and monitoring of EoE). Overall agreement is defined as percentage of Cytosponge procedures yielding results consistent with endoscopic biopsy results. Presence of EoE is measured by the count of eosinophils present per high power field (eos/HPF) with active EoE defined as \>=15 eos/HPF. Sensitivity was calculated via percentage of positive (active EoE) results obtained via Cytosponge as compared to results indicating active EoE via endoscopy with biopsy. Specificity was calculated via percentage of negative (inactive EoE) results obtained via Cytosponge as compared to results indicating inactive EoE via endoscopy with biopsy.

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

86 participants

Primary outcome timeframe

At study enrollment and initial procedure and each additional procedure, up to 1 year after enrollment

Results posted on

2021-02-17

Participant Flow

Participant milestones

Participant milestones
Measure	EoE Patients Patients who have been diagnosed with EoE as per recent guidelines will be enrolled. Samples will be obtained using the Cytosponge™ Cell Collection Device (Cytosponge) prior to participants' routine endoscopy with biopsy.
Overall Study STARTED	86
Overall Study COMPLETED	80
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	EoE Patients Patients who have been diagnosed with EoE as per recent guidelines will be enrolled. Samples will be obtained using the Cytosponge™ Cell Collection Device (Cytosponge) prior to participants' routine endoscopy with biopsy.
Overall Study Unable to swallow Cytosponge	6

Baseline Characteristics

Diagnosis and Monitoring of Eosinophilic Esophagitis Using the Cytosponge

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	EoE Patients n=80 Participants Patients who have been diagnosed with EoE as per recent guidelines will be enrolled. Samples will be obtained using the Cytosponge™ Cell Collection Device (Cytosponge) prior to participants' routine endoscopy with biopsy.
Age, Continuous	42 years n=5 Participants
Sex: Female, Male Female	27 Participants n=5 Participants
Sex: Female, Male Male	53 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	80 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	80 Participants n=5 Participants

PRIMARY outcome

Timeframe: At study enrollment and initial procedure and each additional procedure, up to 1 year after enrollment

Population: Of the 105 procedures performed on 80 participants, 101 yielded results from both Cytosponge and endoscopy with biopsy for comparison.

The primary outcome variables are sensitivity (percent agreement between positive results) and specificity (percent agreement between negative results) of the Cytosponge ability to detect the presence of EoE as compared to upper endoscopy with biopsy (the gold standard for diagnosis and monitoring of EoE). Overall agreement is defined as percentage of Cytosponge procedures yielding results consistent with endoscopic biopsy results. Presence of EoE is measured by the count of eosinophils present per high power field (eos/HPF) with active EoE defined as \>=15 eos/HPF. Sensitivity was calculated via percentage of positive (active EoE) results obtained via Cytosponge as compared to results indicating active EoE via endoscopy with biopsy. Specificity was calculated via percentage of negative (inactive EoE) results obtained via Cytosponge as compared to results indicating inactive EoE via endoscopy with biopsy.

Outcome measures

Outcome measures
Measure	EoE Patients n=101 Evaluable Specimens Patients who have been diagnosed with EoE as per recent guidelines will be enrolled. Samples will be obtained using the Cytosponge™ Cell Collection Device (Cytosponge) prior to participants' routine endoscopy with biopsy.
Percent Agreement Between Cytosponge and Endoscopic Biopsy Results Sensitivity	75 Percent
Percent Agreement Between Cytosponge and Endoscopic Biopsy Results Specificity	86 Percent
Percent Agreement Between Cytosponge and Endoscopic Biopsy Results Overall Agreement	80 Percent

SECONDARY outcome

Timeframe: At study enrollment and initial procedure and each additional procedure, up to 1 year after enrollment

Population: Of the 105 procedures performed on 80 participants, 101 yielded evaluable specimens from both Cytosponge and endoscopy with biopsy for comparison.

Overall agreement (Cytosponge procedures yielding results consistent with endoscopic biopsy results) as measured by Cohen's Kappa. Overall Cohen's Kappa is a statistical measure for assessing the reliability of agreement between the two results by taking into account the element of chance. Cohen's kappa can range from 0 to 1 with 1 indicating perfect agreement and 0 indicating an agreement equivalent to chance.

Outcome measures

Outcome measures
Measure	EoE Patients n=101 Results of Evaluable Specimens Patients who have been diagnosed with EoE as per recent guidelines will be enrolled. Samples will be obtained using the Cytosponge™ Cell Collection Device (Cytosponge) prior to participants' routine endoscopy with biopsy.
Overall Agreement Between Cytosponge and Endoscopic Biopsy Results as Measured by Kappa	0.61 Kappa coefficient

SECONDARY outcome

Timeframe: 7 days after each procedure

Acceptability of Cytosponge compared to endoscopic biopsy as measured by visual analogue scale. Participants were asked to rate their experience of the procedures on a scale of 0-10, where 0 indicates "unacceptable, very difficult even for a medical test," and 10 indicates "not an issue, would take test." A higher score indicates a more acceptable test. Acceptability was measured after each procedure and scores from each assessment were summed to obtain the mean and standard deviation.

Outcome measures

Outcome measures
Measure	EoE Patients n=80 Participants Patients who have been diagnosed with EoE as per recent guidelines will be enrolled. Samples will be obtained using the Cytosponge™ Cell Collection Device (Cytosponge) prior to participants' routine endoscopy with biopsy.
Acceptability of Cytosponge Compared to Endoscopic Biopsy, as Measured by Visual Analog Scale Endoscopy with Biopsy Acceptability	5.83 Units on a scale Standard Deviation 2.90
Acceptability of Cytosponge Compared to Endoscopic Biopsy, as Measured by Visual Analog Scale Cytosponge Acceptability	7.24 Units on a scale Standard Deviation 2.21

SECONDARY outcome

Timeframe: 7 days after each procedure

Population: Not all participants competed the IES scale. Mean and Standard Deviation are reported for the 18 participants who completed the IES scale seven days after the procedure.

Acceptability of Cytosponge as measured by the Impact of Events (IES) scale. The IES measures subjective distress (such as intrusive thoughts or emotions and avoidant or anxious behavior) following a stressful event. Respondents are asked to answer questions to indicate the amount of stress from the event. Scores are calculated using the following scale: Not at all =0, Rarely =1, Sometimes =3, Often =4. The total score is calculated by adding each response, with a total final score ranging from (0-60). Scores ranging 0-8 indicate no meaningful impact, scores ranging 9-25 indicate impact, and scores of 26 and above are considered very important (26-43 = powerful impact, 44-75 = severe impact). Acceptability was measured after each procedure and scores from each assessment were summed to obtain the mean and standard deviation.

Outcome measures

Outcome measures
Measure	EoE Patients n=18 Participants Patients who have been diagnosed with EoE as per recent guidelines will be enrolled. Samples will be obtained using the Cytosponge™ Cell Collection Device (Cytosponge) prior to participants' routine endoscopy with biopsy.
Acceptability of Cytosponge as Measured by the Impact of Events Scale	1.4 Units on a scale Standard Deviation 2.7

OTHER_PRE_SPECIFIED outcome

Timeframe: 7 days after each procedure

Population: Data reported for participants who indicated a preference at follow up.

The number of responses indicating preference for Cytosponge to endoscopic biopsy. Preference was measured by asking participants after each procedure, "which procedure would you prefer to undergo again if your physician indicated it was medically necessary?" with the options "Traditional Upper Endoscopy" and "Cytosponge." The total number of responses recorded as "Cytosponge" and the total number of responses recorded as "Traditional Upper Endoscopy" were summed.

Outcome measures

Outcome measures
Measure	EoE Patients n=48 Participants Patients who have been diagnosed with EoE as per recent guidelines will be enrolled. Samples will be obtained using the Cytosponge™ Cell Collection Device (Cytosponge) prior to participants' routine endoscopy with biopsy.
Number of Responses Indicating Preference for Cytosponge Over Endoscopic Biopsy Cytosponge Preferred	42 responses
Number of Responses Indicating Preference for Cytosponge Over Endoscopic Biopsy Endscopic Biopsy Preferred	6 responses

Adverse Events

EoE Patients

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

Evan Dellon, MD

University of North Carolina at Chapel Hill

Phone: 919-966-2511

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place