Predictors and Outcomes in Patients With Sickle Cell Disease

• ClinicalTrials.gov Identifier: NCT03431935
• Recruitment Status: Recruiting
• First Posted: February 13, 2018
• Last Update Posted: July 11, 2022
• Sponsor: St. Jude Children's Research Hospital
• Collaborators: Methodist Comprehensive Sickle Cell Center; University of Memphis; National Heart, Lung, and Blood Institute (NHLBI)
• Information provided by (Responsible Party): St. Jude Children's Research Hospital

Tracking Information

• First Submitted Date: February 7, 2018
• First Posted Date: February 13, 2018
• Last Update Posted Date: July 11, 2022
• Actual Study Start Date: July 31, 2018
• Estimated Primary Completion Date: January 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 14, 2018)

• Number of emergency room/hospital admissions in the previous 12 months [ Time Frame: Once, at age 19 ]
  The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
• Percent of clinic visits completed in the previous 12 months [ Time Frame: Once, at age 19 ]
  The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
• Percent hydroxyurea prescription refill rate [ Time Frame: Once, at age 19 ]
  The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
• Health-related quality of life as assessed by the PedsQL [ Time Frame: Once, at age 19 ]
  The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.

Original Primary Outcome Measures (submitted: February 7, 2018)

• Number of emergency room/hospital admissions in the previous 12 months [ Time Frame: Once, at age 19 ]
  The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
• Percent of clinic visits completed in the previous 12 months [ Time Frame: Once, at age 19 ]
  The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
• Percent hydroxyurea prescription refill rate [ Time Frame: Once, at age 19 ]
  The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.
• Health-related quality of life [ Time Frame: Once, at age 19 ]
  The primary objective will utilize data collected at age 19 years, 0 months (±2 months), the age participants will have transferred to adult care. This data will come from participants enrolled between the ages of 17 years, 0 months and 19 years, 0 months.

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Predictors and Outcomes in Patients With Sickle Cell Disease
• Official Title: Longitudinal Examination of Predictors and Outcomes of Sickle Cell Disease Healthcare Transition

Brief Summary

Children with sickle cell disease (SCD) are living longer with the advent of medical advances such as prophylactic penicillin, chronic transfusion, and hydroxyurea. Despite greater longevity in SCD, the period following the transition from pediatric to adult care is critical; youth aged 18-30 years are at high risk for mortality and have high rates of healthcare utilization, leading to high healthcare costs. As such, health care transition (HCT) programs have been created to prepare patients for adult-centered care and subsequently, improve health outcomes. However, very few programs have been evaluated for effectiveness in achieving optimal health outcomes in SCD. This paucity of program evaluation is attributed to a lack of identifiable predictors and outcomes.

Researchers at St. Jude Children's Research Hospital want to identify factors and patterns of successful HCT. This information will be used to develop approaches to best evaluate HCT interventions and identify areas of improvement of HCT programming.

PRIMARY OBJECTIVE: Describe hospital utilization, treatment adherence, and health-related quality of life in a cohort of patients with sickle cell disease (SCD) who will transfer to adult care during the study period.

SECONDARY OBJECTIVE: Examine the associations between various factors and health care transition (HCT) outcomes.

Detailed Description

Participants will be asked to complete a set of questions during an outpatient clinic visit at St. Jude or Methodist Adult Comprehensive Sickle Cell Center. The questions ask about sickle cell disease knowledge and self-management skills, access to care, general adjustment, quality of life, and stress. These questions will take about 60 minutes to complete.

Participants will answer questionnaires on a password protected laptop or tablet using a computer program called Audio Computer-Assisted Self-Interviews (ACASI). The laptop or tablet will be kept by the study team. A study team member will be available during this time to address any technical issues or answer any questions. Participants will answer one of the questionnaires verbally, and the study team member will record responses from the questionnaire with paper and pen. Questionnaires given verbally will be audio-recorded and reviewed by the lead researcher or other study team member. The recordings will be destroyed immediately after review. No transcript or written record of the recordings will be made. Study members will meet five times with each participant during regularly-scheduled clinical visits over a two year period.

Two institutions will collaborate in the proposed project. St. Jude Children's Research Hospital (St. Jude) and the Methodist Comprehensive Sickle Cell Center will be the primary source of participants. Faculty from the University of Memphis, Department of Psychology, will be involved in methodological considerations and analyzing the data.

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Participants who meet eligibility criteria and have a pre-existing relationship as a patient at St. Jude Children's Research Hospital.
• Condition: Sickle Cell Disease
• Intervention: Not Provided
• Study Groups/Cohorts: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: February 7, 2018): 146
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: January 2026
• Estimated Primary Completion Date: January 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Diagnosis of sickle cell disease (all genotypes).
• Age 16.0 - 20.99 years at initial assessment.
• Primary language is English.

Exclusion Criteria:

• Participant unable to complete the questionnaires due to refusal, current acute illness (e.g., pain crisis), or significant cognitive impairment as judged by health care providers at the SCD clinic.
• Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 16 Years to 20 Years (Child, Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Jerlym Porter, PhD, MPH; 866-278-5833; referralinfo@stjude.org

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03431935
• Other Study ID Numbers: LEAPS; K01HL125495-01A1 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: St. Jude Children's Research Hospital
• Original Responsible Party: Same as current
• Current Study Sponsor: St. Jude Children's Research Hospital
• Original Study Sponsor: Same as current

Collaborators

• Methodist Comprehensive Sickle Cell Center
• University of Memphis
• National Heart, Lung, and Blood Institute (NHLBI)

Investigators

• Principal Investigator: Jerlym Porter, PhD, MPH; St. Jude Children's Research Hospital

• PRS Account: St. Jude Children's Research Hospital
• Verification Date: July 2022