Development and Validation of the Patient/Caregiver Reported Hydroxyurea Evaluation of Adherence for Life (HEAL) Scale.

Introduction: Hydroxyurea reduces the incidence of vaso-occlusive episodes, stroke, and respiratory, cardiac, and renal damage in sickle cell disease by increasing fetal hemoglobin. However, because suboptimal adherence to hydroxyurea limits its effectiveness, understanding patient-specific barriers to hydroxyurea adherence could help improve adherence and health outcomes in patients with sickle cell disease. The aim of this single-site, prospective, IRB-approved study was to validate a 24-item patient- and caregiver-reported hydroxyurea treatment adherence questionnaire, the Hydroxyurea Evaluation of Adherence for Life (HEAL) scale. Methods: A sample of 24 adults with sickle cell disease and 16 caregivers of children with sickle cell disease completed the HEAL scale, and a subset of the original sample provided a second HEAL scale for test-retest reliability. HEAL scale results were validated against global adherence ratings from participants and health-care providers, records of access to pill bottles, and laboratory values for fetal hemoglobin and absolute neutrophil count. Results and Discussion: Results demonstrated excellent internal consistency for the HEAL Total score and eight (3-item) subscale scores (Dose, Remember, Plan, Cost, Understand, Effectiveness, Laboratory, and Pharmacy), as well as strong test-retest reliability for all HEAL scores except the Cost subscale. HEAL Total scores correlated significantly with validity measures, including global adherence ratings and lab values. The HEAL scale offers significant clinical potential for understanding adherence in individual sickle cell disease patients and significant research potential for characterizing adherence in persons with sickle cell disease who are treated with hydroxyurea.

No child left behind: Building a comprehensive sickle cell disease care oasis in the Lake County, Indiana care desert.

BACKGROUND: Sickle cell disease (SCD) leads to end-organ damage and shortened life expectancy. The second highest incidence of SCD in Indiana is in Lake County, but until 2017, there was no SCD expert within 65 miles. The Indiana Hemophilia and Thrombosis Center (IHTC) developed the Sickle Care coordination OutReach and Education (SCORE) program in 2017 to bring high-quality, guideline-based care to children with SCD. PROCEDURE: The St. Vincent IRB deemed this retrospective analysis of SCORE clinic care from 2017 to 2020 exempt. Data on the number of transcranial Dopplers (TCD) performed, HU dosing escalation, and vaccine rates were collected along with the number of school and home visits completed. RESULTS: Fifty-three children, adolescents, and young adults completed 288 SCORE clinic visits during the study period; over 75% completed at least three clinic visits. Mean HU dose increased significantly with SCORE clinic care. TCD screening rates increased every year starting in 2018 through 2020 when we added local care coordination. One hundred seventy-three vaccines were administered in SCORE outreach clinics. The PPSV23 vaccines had a 100% acceptance rate, and seasonal influenza had a 75.8% acceptance rate. CONCLUSION: Access to care coordination services and local hematology specialty care alleviates barriers to care and enables comprehensive SCD care delivery to children in need. Prior to the establishment of the SCORE clinic, 75% of children in Lake County were not receiving recommended stroke screening. The SCORE clinic model demonstrates feasibility and impact when delivering on the promise of high-quality care for children with SCD.

Ring the Bell for Sickle Cell: Encouraging Advocacy in an Underserved Community.

Sickle cell disease (SCD) was once a disease of childhood because of a limited life expectancy. Due to medical advances, it is now common for people with SCD to live into adulthood. Funding and resources for adults with SCD, however, remain limited. Adult patients would benefit from increased access to medical care, mental health care services, and workforce development. The Indiana Sickle Cell Consortium, a group of medical providers and community-based organizations, worked closely with people living with SCD and their family members to create a campaign advocating for state funding for programs for adults with SCD. This campaign culminated with the passage of a bill that provides $250,000 in funding for program development for adults with SCD. The bill also directs the Indiana Department of Health to carry out a needs assessment for people with SCD in Indiana. However, continued efforts are needed to reduce health disparities for people with SCD. The Indiana Sickle Cell Consortium will continue advocacy efforts in future legislative cycles and bring attention to the health inequities that affect people with SCD.

Adherence to Quality of Care Indicators and Location of Sickle Cell Care Within Indiana.

Newborn screening (NBS) follow-up programs for infants with sickle cell disease (SCD) are highly variable among states. Initiated in 2009, Sickle SAFE, the NBS follow-up program for infants with SCD in Indiana, follows infants through home visits and phone contact. The current study assessed the attainment rates for recently published quality indicators of pediatric SCD care for Sickle SAFE participants. Using retrospective data, we determined the proportion of children who received transcranial Doppler (TCD) screening, influenza, and pneumococcal vaccination and were prescribed hydroxyurea. We calculated the mean age at confirmatory testing, time to receipt of penicillin prophylaxis, and mean age when genetic counseling was offered. One hundred ninety-eight children born with SCD in Indiana between July 1, 2009 and June 30, 2017 were followed for at least 1 year. While 97.5% received at least one dose of conjugated pneumococcal vaccine, vaccination with the 23 valent pneumococcal vaccine varied by location (county) of care (Allen: 14.3%, Lake: 26.7%, St. Joseph: 40.0%, Marion: 73.3%). Overall TCD screening rate for eligible children was 53%; TCD screening rate varied widely by location of care (Lake: 25% vs. Marion: 63.8%). Similarly, hydroxyurea prescribing practices varied significantly by location of care (p < 0.001). Identified gaps in adherence to quality indicators in SCD care will serve as the basis for future quality improvement initiatives.

Community Health Workers as Support for Sickle Cell Care.

Community health workers are increasingly recognized as useful for improving health care and health outcomes for a variety of chronic conditions. Community health workers can provide social support, navigation of health systems and resources, and lay counseling. Social and cultural alignment of community health workers with the population they serve is an important aspect of community health worker intervention. Although community health worker interventions have been shown to improve patient-centered outcomes in underserved communities, these interventions have not been evaluated with sickle cell disease. Evidence from other disease areas suggests that community health worker intervention also would be effective for these patients. Sickle cell disease is complex, with a range of barriers to multifaceted care needs at the individual, family/friend, clinical organization, and community levels. Care delivery is complicated by disparities in health care: access, delivery, services, and cultural mismatches between providers and families. Current practices inadequately address or provide incomplete control of symptoms, especially pain, resulting in decreased quality of life and high medical expense. The authors propose that care and care outcomes for people with sickle cell disease could be improved through community health worker case management, social support, and health system navigation. This paper outlines implementation strategies in current use to test community health workers for sickle cell disease management in a variety of settings. National medical and advocacy efforts to develop the community health workforce for sickle cell disease management may enhance the progress and development of "best practices" for this area of community-based care.

A validated measure of adherence to antibiotic prophylaxis in children with sickle cell disease.

BACKGROUND: Antibiotic prophylaxis is a mainstay in sickle cell disease management. However, adherence is estimated at only 66%. This study aimed to develop and validate a Sickle Cell Antibiotic Adherence Level Evaluation (SCAALE) to promote systematic and detailed adherence evaluation. METHODS: A 28-item questionnaire was created, covering seven adherence areas. General Adherence Ratings from the parent and one health care provider and medication possession ratios were obtained as validation measures. RESULTS: Internal consistency was very good to excellent for the total SCAALE (α=0.89) and four of the seven subscales. Correlations between SCAALE scores and validation measures were strong for the total SCAALE and five of the seven subscales. CONCLUSION: The SCAALE provides a detailed, quantitative, multidimensional, and global measurement of adherence and can promote clinical care and research.

Noninvasive buccal swab antigen sample and molecular testing provides extended antigen typing for patients with hemoglobinopathies.

OBJECTIVE: To demonstrate the feasibility of performing a noninvasive, molecular-based red blood cell (RBC) antigen test on infants and very young children with sickle cell disease as part of a statewide newborn screening follow-up program. STUDY DESIGN: A prospective pilot project was conducted using a noninvasive buccal swab and test kit to perform DNA-based, extended RBC phenotyping in 92 children participating in a newborn hemoglobinopathy screening follow-up program. Reported data include the extended panel of antigens detected by molecular analysis compared with unaffected population estimates. RESULTS: Molecular-based RBC antigen testing was successful, with extended RBC typing generated for all subjects. Molecular testing detected several rare negative or rare positive phenotypes, demonstrating the utility of obtaining an extended antigen panel. CONCLUSION: This study demonstrates the feasibility of performing antigen testing on buccal swab specimens from children with sickle cell disease as part of a newborn screening follow-up program with the aim of allowing specific unit matching to prevent alloimmunization with RBC transfusions. The general applicability of testing may be limited by a lack of uniform insurance coverage for buccal swab testing, however.