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

• ClinicalTrials.gov Identifier: NCT02411396
• Recruitment Status: Completed
• First Posted: April 8, 2015
• Results First Posted: June 17, 2019
• Last Update Posted: June 27, 2019
• Sponsor: Johns Hopkins University
• Information provided by (Responsible Party): Johns Hopkins University

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Condition: Sickle Cell Disease
• Enrollment: 483

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
Pre-assignment Details

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 [1]	483
Completed	442
Not Completed	41
Reason Not Completed
Lost to Follow-up	31
Death	10
[1] First participant enrolled on 4/30/2015 at Johns Hopkins

Baseline Characteristics

Arm/Group Title		VOC in Patients With SCD
Arm/Group Description		Patients treated for uncomplicated VOC in ICs and EDs.
Overall Number of Baseline Participants		483
Baseline Analysis Population Description		[Not Specified]
Age, Categorical [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	483 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	476 98.6%
	>=65 years	7 1.4%
		[1] Measure Description: age of participant in years
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	483 participants
		34.4 (11.1)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	483 participants
	Female	293 60.7%
	Male	190 39.3%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	483 participants
	Hispanic or Latino	6 1.2%
	Not Hispanic or Latino	477 98.8%
	Unknown or Not Reported	0 0.0%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	483 participants
	American Indian or Alaska Native	2 0.4%
	Asian	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	471 97.5%
	White	0 0.0%
	More than one race	10 2.1%
	Unknown or Not Reported	0 0.0%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	483 participants
		483

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
Overall Number of Participants Analyzed	317	265
Overall Number of Units Analyzed; Type of Units Analyzed: Number of visits	1558	1469
Mean (95% Confidence Interval); Unit of Measure: minutes
	125.3; (118.3 to 131.8)	61.8; (59.3 to 64.3)

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.
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	[Not Specified]

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
Overall Number of Participants Analyzed	317	265
Overall Number of Units Analyzed; Type of Units Analyzed: Number of visits	1556	1468
Measure Type: Number; Unit of Measure: number of visits
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	[Not Specified]
	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.
Method of Estimation	Estimation Parameter	Odds Ratio (OR)
	Estimated Value	5.14
	Confidence Interval	(2-Sided) 95%; 4.13 to 6.41
	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
Overall Number of Participants Analyzed	317	265
Overall Number of Units Analyzed; Type of Units Analyzed: Number of visits	1394	1455
Measure Type: Number; Unit of Measure: number of visits
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	[Not Specified]
	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.
Method of Estimation	Estimation Parameter	Odds Ratio (OR)
	Estimated Value	2.24
	Confidence Interval	(2-Sided) 95%; 1.84 to 2.72
	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
Overall Number of Participants Analyzed	85	122
Mean (Standard Deviation); Unit of Measure: 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
Overall Number of Participants Analyzed	87	118
Measure Type: Count of Participants; Unit of Measure: Participants
Greater feelings of overall medical safety	43 49.4%	98 83.1%
Lesser feelings of overall safety	44 50.6%	20 16.9%

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 (%)
Total	10/483 (2.07%)
Serious Adverse Events
	VOC in Patients With SCD
	Affected / at Risk (%)
Total	0/0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	VOC in Patients With SCD
	Affected / at Risk (%)
Total	0/0

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Sophie Lanzkron, MD, MHS, Associate Professor of Medicine and Oncology
• Organization: Johns Hopkins University School of Medicine
• Phone: 410-502-8642
• EMail: slanzkr@jhmi.edu

Publications:

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.

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. doi: 10.1056/NEJM199406093302303.

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. doi: 10.1186/1477-7525-3-50.

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.

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.

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.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Lanzkron S, Little J, Wang H, Field JJ, Shows JR, Haywood C Jr, Saheed M, Proudford M, Robertson D, Kincaid A, Burgess L, Green C, Seufert R, Brooks J, Piehet A, Griffin B, Arnold N, Frymark S, Wallace M, Abu Al Hamayel N, Huang CY, Segal JB, Varadhan R. Treatment of Acute Pain in Adults With Sickle Cell Disease in an Infusion Center Versus the Emergency Department : A Multicenter Prospective Cohort Study. Ann Intern Med. 2021 Sep;174(9):1207-1213. doi: 10.7326/M20-7171. Epub 2021 Jul 6. Erratum In: Ann Intern Med. 2021 Aug 3;:

• Responsible Party: Johns Hopkins University
• ClinicalTrials.gov Identifier: NCT02411396
• Other Study ID Numbers: IRB00054029; PCORI-1403-11888 ( Other Grant/Funding Number: PCORI )
• First Submitted: March 30, 2015
• First Posted: April 8, 2015
• Results First Submitted: January 22, 2019
• Results First Posted: June 17, 2019
• Last Update Posted: June 27, 2019