ESCAPED: Comparison of Patient Centered Outcomes for People With Sickle Cell Disease in the Acute Care Setting

• Sponsor: Johns Hopkins University (Other)
• Overall Status: Completed
• CT.gov ID: NCT02411396
• Collaborator: (none)
• Enrollment: 483
• Locations: 3
• Actual Duration (Months): 38
• Patients Per Site: 161
• Patients Per Site Per Month: 4.2

Study Details

Study Description

Brief Summary

The Emergency Department has been the standard location where patients with Sickle Cell Disease (SCD) go to seek care for the treatment of acute painful events. Vaso- Occlusive Crisis (VOC) is the most common complication of SCD,

The purpose of this study is to compare patient centered outcomes for patients being treated for an uncomplicated VOC in Infusion Centers (IC) and Emergency Departments (ED) in four locations around the United States.

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease

Detailed Description

Emergency Department care is marked by long delays, lack of efficacy, and conflict. A sub-specialty Infusion Center staffed by expert clinicians and delivering individualized care can improve care quality while reducing costs. The study will examine whether care provided in an Infusion Center (IC) is more patient centered and efficient than care provided in an Emergency Department (ED) for adults with Sickle Cell Disease (SCD) and uncomplicated Vaso-Occlusive Crisis (VOC).

Sites will prospectively enroll patients in VOC seen in participating centers from either the EDs or the ICs. Specific data from the acute visits (e.g. Times of arrival, time to first dose of analgesic, etc) will be captured. This study will compare: pain management, disposition of subjects (home or admission) and patient experiences of care delivery in both settings. Subjects will complete surveys/questionnaires to asses subjects' experiences in the setting where care was provided.

The four sites to participate in the study are Baltimore, Maryland (Johns Hopkins Hospital), Cleveland, Ohio (Cleveland Medical Center), Milwaukee, Wisconsin (Medical College of Wisconsin), and Baton Rouge, Louisiana (Our Lady of the Lake Hospital). A maximum of 500 subjects will participate in the study. Participants will be enrolled for 18 months.

Study Design

• Study Type: Observational
• Actual Enrollment: 483 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Patient Centered Outcomes Research Institute (PCORI) ESCAPED Study: Comparison of Patient Centered Outcomes for People With SCD in the Acute Care Setting
• Actual Study Start Date: Apr 1, 2015
• Actual Primary Completion Date: Jun 1, 2018
• Actual Study Completion Date: Jun 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Patients With SCD Patients treated for uncomplicated VOC in ICs and EDs.

Outcome Measures

Primary Outcome Measures

1. Time (Minutes) From Arrival to Center to Time First Dose of Parenteral Pain Medication Administered [Within 6 hours after arrival]

   Time is recorded from the time the patient arrives for pain treatment at either the ED or IC until the time the patient is dosed with pain medication administered parenterally. Guideline recommendations are that patients receive non-oral pain medication within 60 minutes of arrival.

Secondary Outcome Measures

1. Disposition From Acute Care Visit [Day 1 of admission]

   Odds for admission to the hospital versus discharge to home (ED vs IC)

2. Pain Reassessment Within 30 Minutes of First Dose of Parenteral Pain Medication Administered [30 minutes after administration]

   Odds of being re-assessed for pain within 30 minutes of receiving first dose of pain medication in ED vs IC. NHLBI guidelines recommend that patients are re-assessed for adequacy of pain management 30 minutes after receiving pain medication.

3. Patient Reported Satisfaction With Care Received [within 72 hours of acute visit]

   Survey to capture patient satisfaction with the quality of care in either the ED or IC. Validated a new tool to assess satisfaction with care in the acute care setting. The new tool was developed based on existing tools that assessed several domains: adequacy of pain management, communication with providers, interpersonal aspects of care, provider competence, involvement of family/friends, and access to care. The final 15 item validated Patient Satisfaction with Pain Management in Sickle Cell Disease (SCD) (PSPS) scale was used to compare satisfaction of care comparing ED to IC acute visits. Overall mean satisfaction scores ranged from 0-7 with higher scores signifying greater satisfaction

4. Patient Reported Perception of Risk From Visit [within 72 hours of acute visit]

   One question on the survey asked patients to rate the overall level of medical safety they felt during their visit to the ED or IC. Choices for responses: Excellent, Very Good, Good, Fair or Poor. Excellent and Very Good were determined as having greater feelings of overall safety while patients who chose Good, Fair or Poor were determined having lesser feelings of overall safety.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Confirmed Sickle Cell Disease patients who live within 60 miles of the study center or who already receive regular care at the participating centers.

Exclusion Criteria:

• Stable patients who have been on chronic transfusion therapy and have not had a painful episode within two years of enrollment.

• Patients who are pregnant.

• Patients who are unwilling or unable to sign consent.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Our Lady of the Lake Hospital	Baton Rouge	Louisiana	United States	70809
2	Cleveland Medical Center at University Hospitals	Cleveland	Ohio	United States	44106
3	Medical College of Wisconsin, Blood Center	Milwaukee	Wisconsin	United States	53201

Sponsors and Collaborators

• Johns Hopkins University

Investigators

• Principal Investigator: Sophie Lanzkron, MD, MHS, Johns Hopkins University, Division of Hematology

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Feb 17, 2015

More Information

Publications

• Bediako SM. Predictors of employment status among African Americans with sickle cell disease. J Health Care Poor Underserved. 2010 Nov;21(4):1124-37. doi: 10.1353/hpu.2010.0945.
• Hassell KL. Population estimates of sickle cell disease in the U.S. Am J Prev Med. 2010 Apr;38(4 Suppl):S512-21. doi: 10.1016/j.amepre.2009.12.022.
• Haywood C Jr, Tanabe P, Naik R, Beach MC, Lanzkron S. The impact of race and disease on sickle cell patient wait times in the emergency department. Am J Emerg Med. 2013 Apr;31(4):651-6. doi: 10.1016/j.ajem.2012.11.005. Epub 2013 Feb 4.
• Lanzkron S, Carroll CP, Haywood C Jr. The burden of emergency department use for sickle-cell disease: an analysis of the national emergency department sample database. Am J Hematol. 2010 Oct;85(10):797-9. doi: 10.1002/ajh.21807.
• Lanzkron S, Carroll CP, Hill P, David M, Paul N, Haywood C Jr. Impact of a dedicated infusion clinic for acute management of adults with sickle cell pain crisis. Am J Hematol. 2015 May;90(5):376-80. doi: 10.1002/ajh.23961. Epub 2015 Feb 25.
• McClish DK, Penberthy LT, Bovbjerg VE, Roberts JD, Aisiku IP, Levenson JL, Roseff SD, Smith WR. Health related quality of life in sickle cell patients: the PiSCES project. Health Qual Life Outcomes. 2005 Aug 29;3:50.
• Platt OS, Brambilla DJ, Rosse WF, Milner PF, Castro O, Steinberg MH, Klug PP. Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med. 1994 Jun 9;330(23):1639-44.

• Responsible Party: Johns Hopkins University
• ClinicalTrials.gov Identifier: NCT02411396
• Other Study ID Numbers: IRB00054029; PCORI-1403-11888
• First Posted: Apr 8, 2015
• Last Update Posted: Jun 27, 2019
• Last Verified: Jun 1, 2019

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Additional relevant MeSH terms: Anemia, Sickle Cell

Study Results

Participant Flow

Recruitment Details	There are a total of 483 adult patients with sickle cell disease (SCD) enrolled from the end of April 2015 to December, 2016. 145 from Johns Hopkins (JH), 101 from Case Western (CW), 110 from Our Lady of the Lake Hospital (OLL) and 127 from Blood Center of Wisconsin (BCW). Four hundred forty two patients have completed 18 months follow up

Arm/Group Title	Vaso-Occlusive Crisis (VOC) in Patients With SCD
Arm/Group Description	Patients treated for uncomplicated Vaso-Occlusive Crisis (VOC) in ICs and EDs.
Period Title: Overall Study
STARTED	483
COMPLETED	442
NOT COMPLETED	41

Baseline Characteristics

Arm/Group Title	VOC in Patients With SCD
Arm/Group Description	Patients treated for uncomplicated VOC in ICs and EDs.
Overall Participants	483
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	476 98.6%
>=65 years	7 1.4%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	34.4 (11.1)
Sex: Female, Male (Count of Participants)
Female	293 60.7%
Male	190 39.3%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	6 1.2%
Not Hispanic or Latino	477 98.8%
Unknown or Not Reported	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	2 0.4%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	471 97.5%
White	0 0%
More than one race	10 2.1%
Unknown or Not Reported	0 0%
Region of Enrollment (participants) [Number]
United States	483 100%

Outcome Measures

1. Primary Outcome

Title	Time (Minutes) From Arrival to Center to Time First Dose of Parenteral Pain Medication Administered
Description	Time is recorded from the time the patient arrives for pain treatment at either the ED or IC until the time the patient is dosed with pain medication administered parenterally. Guideline recommendations are that patients receive non-oral pain medication within 60 minutes of arrival.
Time Frame	Within 6 hours after arrival

Outcome Measure Data

Analysis Population Description

Not all participants enrolled in the study visited the ED or the IC for uncomplicated vaso-occlusive crisis. The total number of subjects analyzed exceeds the total number of subjects because the same subject could visit the ED or IC one or more times.

Arm/Group Title	VOC in Patients With SCD Who Went to EDs	VOC in Patients With SCD Who Went to ICs
Arm/Group Description	Patients treated for uncomplicated VOC in EDs.	Patients treated for uncomplicated VOC in ICs
Measure Participants	317	265
Measure number of visits	1558	1469
Mean (95% Confidence Interval) [minutes]	125.3	61.8

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	VOC in Patients With SCD Who Went to EDs, VOC in Patients With SCD Who Went to ICs
	Comments	This analysis is to compare outcome measure between ED or IC visits by using time varying propensity score method developed by our group to adjust imbalance of covariates between the two arms. Each patient can have multiple visits to the facility of choice.
	Type of Statistical Test	Other
	Comments	In order to eliminate bias from the naïve estimation on the treatment effects due to the confounders, time varying propensity score model was developed. This balanced the covariates between the two arms at each single acute visit of each patient. A sub classification on the propensity scores was used to estimate the potential outcome for each arm and the average treatment effects (ATE). Bootstrapping was employed to estimate the standard error of ATE.
Statistical Test of Hypothesis	p-Value
	Comments
	Method
	Comments
Method of Estimation	Estimation Parameter	Mean Difference (Final Values)
	Estimated Value	124.6
	Confidence Interval	(2-Sided) 95%; 118.3 to 130.9
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 3.2
	Estimation Comments

2. Secondary Outcome

Title	Disposition From Acute Care Visit
Description	Odds for admission to the hospital versus discharge to home (ED vs IC)
Time Frame	Day 1 of admission

Outcome Measure Data

Analysis Population Description

Not all participants enrolled in the study visited the ED or the IC for uncomplicated vaso-occlusive crisis. The total number of subjects analyzed exceeds the total number of subjects because the same subject could visit the ED or IC one or more times.

Arm/Group Title	VOC in Patients With SCD Who Went to EDs	VOC in Patients With SCD Who Went to ICs
Arm/Group Description	Patients treated for uncomplicated VOC in EDs.	Patients treated for uncomplicated VOC in ICs
Measure Participants	317	265
Measure number of visits	1556	1468
Number of visits being hospitalized	517	128
Number of visits being sent home	1039	1340

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	VOC in Patients With SCD Who Went to EDs, VOC in Patients With SCD Who Went to ICs
	Comments
	Type of Statistical Test	Other
	Comments	In order to eliminate bias from the naïve estimation on the treatment effects due to the confounders, time varying propensity score model was developed. This balanced the covariates between the two arms at each single acute visit of each patient. A sub classification on the propensity scores was used to estimate the potential outcome for each arm and the average treatment effects (ATE). Bootstrapping was employed to estimate the standard error of ATE.
Statistical Test of Hypothesis	p-Value
	Comments
	Method
	Comments
Method of Estimation	Estimation Parameter	Odds Ratio (OR)
	Estimated Value	5.14
	Confidence Interval	(2-Sided) 95%; 4.13 to 6.41
	Parameter Dispersion	Type: Value:
	Estimation Comments	VOC in patients with SCD whom went to EDs represents the numerator, VOC in patients with SCD whom went to ICs represents the denominator for Odds Ratio.

3. Secondary Outcome

Title	Pain Reassessment Within 30 Minutes of First Dose of Parenteral Pain Medication Administered
Description	Odds of being re-assessed for pain within 30 minutes of receiving first dose of pain medication in ED vs IC. NHLBI guidelines recommend that patients are re-assessed for adequacy of pain management 30 minutes after receiving pain medication.
Time Frame	30 minutes after administration

Outcome Measure Data

Analysis Population Description

Not all participants enrolled in the study visited the ED or the IC for uncomplicated vaso-occlusive crisis. The total number of subjects analyzed exceeds the total number of subjects because the same subject could visit the ED or IC one or more times.

Arm/Group Title	VOC in Patients With SCD Who Went to EDs	VOC in Patients With SCD Who Went to ICs
Arm/Group Description	Patients treated for uncomplicated VOC in EDs.	Patients treated for uncomplicated VOC in ICs
Measure Participants	317	265
Measure number of visits	1394	1455
Number of visits being reassessed in 30 mins	287	544
Number of visits not being reassessed in 30 mins	1107	911

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	VOC in Patients With SCD Who Went to EDs, VOC in Patients With SCD Who Went to ICs
	Comments
	Type of Statistical Test	Other
	Comments	In order to eliminate bias from the naïve estimation on the treatment effects due to the confounders, time varying propensity score model was developed. This balanced the covariates between the two arms at each single acute visit of each patient. A sub classification on the propensity scores was used to estimate the potential outcome for each arm and the average treatment effects (ATE). Bootstrapping was employed to estimate the standard error of ATE.
Statistical Test of Hypothesis	p-Value
	Comments
	Method
	Comments
Method of Estimation	Estimation Parameter	Odds Ratio (OR)
	Estimated Value	2.24
	Confidence Interval	(2-Sided) 95%; 1.84 to 2.72
	Parameter Dispersion	Type: Value:
	Estimation Comments	VOC in patients with SCD whom went to ICs represents the numerator, VOC in patients with SCD whom went to EDs represents the denominator for Odds Ratio.

4. Secondary Outcome

Title	Patient Reported Satisfaction With Care Received
Description	Survey to capture patient satisfaction with the quality of care in either the ED or IC. Validated a new tool to assess satisfaction with care in the acute care setting. The new tool was developed based on existing tools that assessed several domains: adequacy of pain management, communication with providers, interpersonal aspects of care, provider competence, involvement of family/friends, and access to care. The final 15 item validated Patient Satisfaction with Pain Management in Sickle Cell Disease (SCD) (PSPS) scale was used to compare satisfaction of care comparing ED to IC acute visits. Overall mean satisfaction scores ranged from 0-7 with higher scores signifying greater satisfaction
Time Frame	within 72 hours of acute visit

Outcome Measure Data

Analysis Population Description

207 participants completed the satisfaction survey after their first visit during the study time period.

Arm/Group Title	VOC in Patients With SCD Who Went to the ED	VOC in Patients With SCD Who Went to the IC
Arm/Group Description	VOC in Patients with SCD that went to the ED	VOC in patients with SCD that went to the IC
Measure Participants	85	122
Mean (Standard Deviation) [score on a scale]	4.8 (1.1)	5.8 (1.0)

5. Secondary Outcome

Title	Patient Reported Perception of Risk From Visit
Description	One question on the survey asked patients to rate the overall level of medical safety they felt during their visit to the ED or IC. Choices for responses: Excellent, Very Good, Good, Fair or Poor. Excellent and Very Good were determined as having greater feelings of overall safety while patients who chose Good, Fair or Poor were determined having lesser feelings of overall safety.
Time Frame	within 72 hours of acute visit

Outcome Measure Data

Analysis Population Description

Only 205 patients completed the perception of risk question at the first visit to either the ED or the IC.

Arm/Group Title	VOC in Patients With SCD Who Went to ED	VOC in Patients With SCD Who Went to IC
Arm/Group Description	Patients treated for uncomplicated VOC in EDs	Patients treated for uncomplicated VOC in ICs
Measure Participants	87	118
Greater feelings of overall medical safety	43 8.9%	98 NaN
Lesser feelings of overall safety	44 9.1%	20 NaN

Adverse Events

Time Frame	Participants were followed for 18 months
Adverse Event Reporting Description	No adverse events were collected since observational study.
Arm/Group Title	VOC in Patients With SCD
Arm/Group Description	Patients treated for uncomplicated VOC in ICs and EDs
All Cause Mortality
	VOC in Patients With SCD
	Affected / at Risk (%)	# Events
Total	10/483 (2.1%)
Serious Adverse Events
	VOC in Patients With SCD
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)
Other (Not Including Serious) Adverse Events
	VOC in Patients With SCD
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE 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.