Publications by authors named "Jerlym Porter"

32 Publications

Genetic therapies for the first molecular disease.

J Clin Invest 2021 Apr;131(8)

Department of Hematology.

Sickle cell disease (SCD) is a monogenic disorder characterized by recurrent episodes of severe bone pain, multi-organ failure, and early mortality. Although medical progress over the past several decades has improved clinical outcomes and offered cures for many affected individuals living in high-income countries, most SCD patients still experience substantial morbidity and premature death. Emerging technologies to manipulate somatic cell genomes and insights into the mechanisms of developmental globin gene regulation are generating potentially transformative approaches to cure SCD by autologous hematopoietic stem cell (HSC) transplantation. Key components of current approaches include ethical informed consent, isolation of patient HSCs, in vitro genetic modification of HSCs to correct the SCD mutation or circumvent its damaging effects, and reinfusion of the modified HSCs following myelotoxic bone marrow conditioning. Successful integration of these components into effective therapies requires interdisciplinary collaborations between laboratory researchers, clinical caregivers, and patients. Here we summarize current knowledge and research challenges for each key component, emphasizing that the best approaches have yet to be developed.
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http://dx.doi.org/10.1172/JCI146394DOI Listing
April 2021

Feasibility of Medical Student Mentors to Improve Transition in Sickle Cell Disease.

J Pediatr Psychol 2021 Mar 29. Epub 2021 Mar 29.

Rutgers Cancer Institute of New Jersey.

Objective: Advances in medical care have resulted in nearly 95% of all children with sickle cell disease (SCD) living to adulthood. There is a lack of effective transition programming, contributing to high rates of mortality and morbidity among adolescents and young adults (AYAs) during the transition from pediatric to adult healthcare. This nonrandomized study evaluated the feasibility, acceptability, and preliminary outcomes of a novel medical student mentor intervention to improve transition outcomes.

Methods: Eligible participants were ages 18-25 years, either preparing for transition or had transferred to adult care within the past year. Twenty-four AYA with SCD (Mage = 20.3, SD = 2.6) enrolled in the program and were matched with a medical student mentor. Feasibility and acceptability of the intervention was assessed through enrollment rates, reasons for refusal, retention rates, engagement with the intervention, satisfaction, and reasons for drop-out. Dependent t-tests were used to evaluate the preliminary effects of the intervention on patient transition readiness, health-related quality of life, self-efficacy, SCD knowledge, medication adherence, and health literacy.

Results: Participants (N = 24) demonstrated adequate retention (75.0%), adherence to the intervention (M = 5.3 of 6 sessions), and satisfaction with the intervention. Participants demonstrated significant improvements in transition readiness (p = .001), self-efficacy (p = .002), medication adherence (p = .02), and health literacy (p = .05).

Conclusions: A medical student mentor intervention to facilitate transition from pediatric to adult care for AYA with SCD is both feasible and acceptable to patients and medical students. Preliminary results suggest benefits for patients, warranting a larger efficacy study.
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http://dx.doi.org/10.1093/jpepsy/jsab031DOI Listing
March 2021

Cognitive performance as a predictor of healthcare transition in sickle cell disease.

Br J Haematol 2021 Mar 11;192(6):1082-1091. Epub 2021 Feb 11.

Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.

Neurocognitive deficits in sickle cell disease (SCD) may impair adult care engagement. We investigated the relationship between neurocognitive functioning and socio-environmental factors with healthcare transition outcomes. Adolescents aged 15-18 years who had neurocognitive testing and completed a visit with an adult provider were included. Transition outcomes included transfer interval from paediatric to adult care and retention in adult care at 12 and 24 months. Eighty adolescents (59% male, 64% HbSS/HbSβ -thalassaemia) were included. Mean age at adult care transfer was 18·0 (±0·3) years and transfer interval was 2·0 (±2·3) months. Higher IQ (P = 0·02; P  = 0·05) and higher verbal comprehension (P = 0·008; P  = 0·024) were associated with <2 and <6 month transfer intervals respectively. Better performance on measures of attention was associated with higher adult care retention at 12 and 24 months (P = 0·009; P  = 0·05 and P = 0·04; P  = 0·12 respectively). Transfer intervals <6 months were associated with smaller households (P = 0·02; P  = 0·06) and households with fewer children (P = 0·02; P  = 0·06). Having a working parent was associated with less retention in adult care at 12 and 24 months (P = 0·01; P = 0·02 respectively). Lower IQ, verbal comprehension, attention difficulties and environmental factors may negatively impact transition outcomes. Neurocognitive function should be considered in transition planning for youth with SCD.
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http://dx.doi.org/10.1111/bjh.17351DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8092972PMC
March 2021

Empirically Derived Profiles of Health-Related Quality of Life in Youth and Young Adults with Sickle Cell Disease.

J Pediatr Psychol 2021 03;46(3):293-303

Department of Psychology, St. Jude Children's Research Hospital, Memphis, TN.

Objective: Determining how the health-related quality of life (HRQOL) is impacted by living with Sickle Cell Disease (SCD) can inform psychosocial interventions. The purpose of the present study is to determine if demographic and treatment variables predict membership into empirically derived subgroups of HRQOL among youth and young adults with SCD.

Methods: Three hundred and seven youth and young adults with SCD (mean 17.63 years ± 3.74 years, 50.5% female) completed the Pediatric Quality of Life InventoryTM Sickle Cell Disease Module. Latent profile analysis examined subgroups/classes of HRQOL and relationships with demographic and treatment variables.

Results: Three distinct classes emerged: High HRQOL (34% of the sample), Moderate HRQOL (44% of the sample), and Low HRQOL (22% of the sample). Being female was associated with increased odds of being in the moderate or low groups. Living with more severe SCD (genotypes HbSS and HbSβ0 thalassemia) was associated with increased odds of being in the Low HRQOL group. Treatment with chronic red blood cell transfusion therapy was associated with increased odds of being in the High HRQOL group. Older age predicted a small increase in the odds of being in the Low versus High HRQOL group.

Conclusions: The present study adds to the literature on HRQOL in SCD by exploring person-centered, empirically derived groups of HRQOL. Identification of demographic and treatment factors that predict membership into those groups within a large sample assists in tailoring needed psychosocial interventions for youth with SCD.
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http://dx.doi.org/10.1093/jpepsy/jsaa104DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7977438PMC
March 2021

Use of Wise Device Technology to Measure Adherence to Hydroxyurea Therapy in Youth With Sickle Cell Disease.

J Pediatr Hematol Oncol 2021 01;43(1):e19-e25

Hematology, St. Jude Children's Research Hospital, Memphis, TN.

Despite broad support for hydroxyurea (HU) therapy, suboptimal adherence is reported for youth with sickle cell disease. Valid adherence measurement is crucial to understanding the relationship between medication behavior, disease response, and patient-centered health outcomes. The current pilot study examined the feasibility of the Wise electronic device for longitudinal HU adherence measurement in a sample of 36 youths prescribed HU. The study also explored the association between HU adherence, as measured by the Wise device, with other adherence measures (ie, family report, lab values, pill count, and medication possession ratio). A measure of family-reported acceptability was also completed. Overall, results supported the feasibility of the Wise device (rate of consent=82%, device use=75%, device failure=3%) for HU adherence measurement and most families rated their experience using their device positively (favorable responses ranged from 67% to 100%). Associations between HU adherence, as measured by the Wise device, and other adherence measures were not significant. Overall, the feasibility was supported. The Wise device allows longitudinal measurement of adherence with HU from initiation as a young child (ie, with liquid formulations) through adolescence and provides a novel means of adherence measurement for both clinical and research use.
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http://dx.doi.org/10.1097/MPH.0000000000001997DOI Listing
January 2021

Intentional and unintentional nonadherence to hydroxyurea among people with sickle cell disease: a qualitative study.

Blood Adv 2020 09;4(18):4463-4473

Department of Psychology, St. Jude Children's Research Hospital, Memphis, TN.

Hydroxyurea is an efficacious treatment for sickle cell disease (SCD), but adoption is low among individuals with SCD. The objective of this study was to examine barriers to patients' adherence to hydroxyurea use regimens by using the intentional and unintentional medication nonadherence framework. We interviewed individuals with SCD age 15 to 49.9 years who were participants in the Sickle Cell Disease Implementation Consortium (SCDIC) Needs Assessment. The intentional and unintentional medication nonadherence framework explains barriers to using hydroxyurea and adds granularity to the understanding of medication adherence barriers unique to the SCD population. In total, 90 semi-structured interviews were completed across 5 of the 8 SCDIC sites. Among interviewed participants, 57.8% (n = 52) were currently taking hydroxyurea, 28.9% (n = 26) were former hydroxyurea users at the time of the interview, and 13.3% (n = 12) had never used hydroxyurea but were familiar with the medication. Using a constructivist grounded theory approach, we discovered important themes that contributed to nonadherence to hydroxyurea, which were categorized under unintentional (eg, Forgetfulness, External Influencers) and intentional (Negative Perceptions of Hydroxyurea, Aversion to Taking Any Medications) nonadherence types. Participants more frequently endorsed adherence barriers that fell into the unintentional nonadherence type (70%) vs intentional nonadherence type (30%). Results from this study will help SCD health care providers understand patient choices and decisions as being either unintentional or intentional, guide tailored clinical discussions regarding hydroxyurea therapy, and develop specific, more nuanced interventions to address nonadherence factors.
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http://dx.doi.org/10.1182/bloodadvances.2020001701DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7509876PMC
September 2020

Development of the InCharge Health Mobile App to Improve Adherence to Hydroxyurea in Patients With Sickle Cell Disease: User-Centered Design Approach.

JMIR Mhealth Uhealth 2020 05 8;8(5):e14884. Epub 2020 May 8.

Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, United States.

Background: Sickle cell disease (SCD) is an inherited blood disorder causing acute complications and chronic progressive end organ damage. SCD is associated with significant morbidity, early mortality, impaired health-related quality of life, and increased acute health care utilization. Hydroxyurea is a US Food and Drug Administration-approved medication that reduces disease complications, acute health care utilization, and costs. However, adherence to hydroxyurea is suboptimal. Mobile health (mHealth) interventions have the potential to improve hydroxyurea adherence, but few examples exist that are specific to the SCD population.

Objective: This study aimed to design a mHealth intervention for individuals with SCD to improve adherence to hydroxyurea, using a user-centered design that was informed by specific barriers to hydroxyurea adherence and utilization in this population.

Methods: This study consisted of 4 phases. In phase 1, individuals with SCD and health care providers participated in an optimization digital workshop. In phase 2, patients completed surveys pertaining to their interest in mHealth use, barriers and facilitators to hydroxyurea use, and health literacy. Phases 3 and 4 involved semistructured interviews and focus groups, respectively, and used the Health Belief Model (HBM) as the framework to investigate drivers of poor hydroxyurea adherence and to inform the development of an app prototype. In addition, in phase 4, we have incorporated the patients' feedback on the preliminary app prototype and its features.

Results: Barriers to hydroxyurea adherence were consistent with the literature and included forgetfulness and several specific thoughts and emotions associated with hydroxyurea use (eg, fear of side effects, depression, stigma, and hopelessness). In addition, more than half of the participants reported potentially low health literacy. Preferred patient app features included 7 key components, namely (1) medication reminders and tracker, (2) disease education, (3) communication, (4) personalization, (5) motivation, (6) support during pain episodes, and (7) social support. Utilizing a user-centered design approach, data obtained from patients and providers were translated into features within the app, mapping to components of the HBM and the specific drivers of hydroxyurea adherence and matching the literacy level of the population, resulting in the development of a novel mobile app called InCharge Health.

Conclusions: The InCharge Health app is an mHealth intervention developed with substantial input from users and by mapping the HBM as the framework that guided the choice for its components. InCharge Health is a customized product for the SCD population aimed at optimizing medication adherence, with the end goal of improving quality of life and health outcomes among patients with SCD. The efficacy and implementation of the InCharge Health app as an mHealth intervention to promote hydroxyurea adherence will be tested in a future stepped-wedge multicenter trial for adolescents and adults with SCD.
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http://dx.doi.org/10.2196/14884DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7245000PMC
May 2020

Paediatric to adult transition care for patients with sickle cell disease: a global perspective.

Lancet Haematol 2020 Apr;7(4):e329-e341

University of California San Francisco Benioff Children's Hospital Oakland, Oakland, California, USA.

Sickle cell disease is a life-threatening inherited condition designated as a public health priority by WHO. Increased longevity of patients with sickle cell disease in high-income, middle-income, and low-income countries present unprecedented challenges for all settings; however, a globally standardised solution for patient transition from paediatric to adult sickle cell disease health care is unlikely to address the challenges. We established a task force of experts from a multicountry (the USA, Europe, Middle East, and Africa) consortium. We combined themes from the literature with viewpoints from members of the task force and invited experts to provide a global overview of transition care practice, highlighting barriers to effective transition care and provide baseline recommendations that can be adapted to local needs. We highlighted priorities to consider for any young person with sickle cell disease transitioning from paediatric to adult health care: skills transfer, increasing self-efficacy, coordination, knowledge transfer, linking to adult services, and evaluating readiness (the SICKLE recommendations). These recommendations aim to ensure appropriate benchmarking of transition programming, but multisite prospective studies are needed to address this growing public health need.
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http://dx.doi.org/10.1016/S2352-3026(20)30036-3DOI Listing
April 2020

Association between hydroxycarbamide exposure and neurocognitive function in adolescents with sickle cell disease.

Br J Haematol 2020 06 26;189(6):1192-1203. Epub 2020 Feb 26.

Departments of, Department of, Psychology, St. Jude Children's Research Hospital, Memphis, TN, USA.

Patients with sickle cell disease (SCD) are at increased risk for neurocognitive impairments. While disease-modifying treatment, such as hydroxycarbamide (hydroxyurea), may decrease this risk, it has not been systematically investigated in children with SCD. We screened neurocognitive functioning in 103 adolescents with SCD (16-17 years, 50% female) and compared outcomes between patients with a history of exposure to hydroxycarbamide (n = 12 HbSC/HbSβ thalassaemia; n = 52 HbSS/HbSβ thalassaemia) and those never treated with hydroxycarbamide (n = 31 HbSC/HbSβ thalassaemia; n = 8 HbSS/HbSβ thalassaemia). Demographic distributions were similar between the groups. After adjusting for socioeconomic status, the hydroxycarbamide group had significantly higher scores on nonverbal IQ (HbSC/HbSβ thalassaemia: P = 0·036, effect size [d] = 0·65), reaction speed (HbSS/HbSβ thalassaemia: P = 0·002, d = 1·70), sustained attention (HbSS/HbSβ thalassaemia: P = 0·014, d = 1·30), working memory (HbSC/HbSβ thalassaemia: P = 0·034, d = 0·71) and verbal memory (HbSC/HbSβ thalassaemia: P = 0·038, d = 0·84) when compared to those who did not receive hydroxycarbamide. In patients with HbSS/HbSβ thalassaemia, longer treatment duration with hydroxycarbamide was associated with better verbal memory (P = 0·009) and reading (P = 0·002). Markers of hydroxycarbamide effect, including higher fetal haemoglobin (HbF), higher mean corpuscular volume (MCV) and lower white blood cell count (WBC), were associated with better verbal fluency (HbF: P = 0·014, MCV: P = 0·006, WBC: P = 0·047) and reading (MCV: P = 0·021, WBC: P = 0·037). Cognitive impairment may be mitigated by exposure to hydroxycarbamide in adolescents with SCD.
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http://dx.doi.org/10.1111/bjh.16519DOI Listing
June 2020

Attention difficulties are associated with lower engagement in adult care amongst youth with sickle cell disease.

Br J Haematol 2020 04 26;189(1):e27-e30. Epub 2020 Feb 26.

Department of Child and Adolescent Psychiatry and Behavioral Sciences, Children's Hospital of Philadelphia, Philadelphia, PA, USA.

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http://dx.doi.org/10.1111/bjh.16421DOI Listing
April 2020

Web-Based Technology to Improve Disease Knowledge Among Adolescents With Sickle Cell Disease: Pilot Study.

JMIR Pediatr Parent 2020 Jan 7;3(1):e15093. Epub 2020 Jan 7.

Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, United States.

Background: Advancements in treatment have contributed to increased survivorship among children with sickle cell disease (SCD). Increased transition readiness, encompassing disease knowledge and self-management skills before transfer to adult care, is necessary to ensure optimal health outcomes. The Sickle Cell Transition E-Learning Program (STEP) is a public, Web-based, 6-module tool designed to increase transition readiness for youth with SCD.

Objective: The objective of our study was to investigate the participation rate of youth with SCD in STEP and its association with transition readiness.

Methods: This was a single-center, Institution Review Board-approved, retrospective cohort review. A total of 183 youths with SCD, aged between 12 and 15 years, were offered STEP as an adjunct to in-clinic disease education sessions. Participation rate (number of patients who used at least one STEP module divided by those approached) was calculated. The association among the number of STEP modules completed, disease knowledge, and self-management was explored.

Results: Overall, 53 of the 183 approached adolescents completed at least one STEP module, yielding a participation rate in STEP of 29.0%. Of the 53 participants, 37 and 39 adolescents had disease knowledge and self-management confidence rating available, respectively. A positive correlation (r=0.47) was found between the number of STEP modules completed and disease knowledge scores (P=.003). No association was found between the number of modules completed and self-management confidence ratings. Disease knowledge scores were significantly higher among participants who completed ≥3 STEP modules compared with those who completed <3 STEP modules (U=149.00; P=.007).

Conclusions: Improvement in disease knowledge in adolescence is critical to ensure the youth's ability to self-care during the period of transition to adult care. Despite low participation, the cumulative exposure to the STEP program suggested greater promotion of disease knowledge among adolescents with SCD before transfer to adult care.
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http://dx.doi.org/10.2196/15093DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6996770PMC
January 2020

A program of transition to adult care for sickle cell disease.

Hematology Am Soc Hematol Educ Program 2019 12;2019(1):496-504

Departments of Hematology and.

Most children with sickle cell disease (SCD) today survive into adulthood. Among emerging adults, there is a marked increase in acute care utilization and a rise in mortality, which can be exacerbated by not establishing or remaining in adult care. Health care transition programs are therefore essential to prepare, transfer, and integrate emerging adults in the adult care setting. The Six Core Elements of Health Care Transition, created by the Center for Health Care Transition Improvement, define the basic components of health care transition support as follows: (1) transition policy, (2) tracking and monitoring progress, (3) assessing transition readiness, (4) planning for adult care, (5) transferring to adult care, and (6) integrating into adult care. Programs that implement the Six Core Elements have experienced significant declines in care abandonment during adolescence and young adulthood and higher early adult care engagement. Most of the core transition activities are not currently reimbursable, however, posing a challenge to sustain transition programs. Ongoing studies are investigating interventions in comparative effectiveness trials to improve health-related quality of life and reduce acute care utilization among emerging adults with SCD. Although these studies will identify best practices for health care transition, it is also important to define how the transition outcomes will be measured, as no consensus definition exists for successful health care transition in SCD. Future research is needed to define best practices for health care transition, systematically assess transition outcomes, and revise payment models to promote sustainability of health care transition programs.
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http://dx.doi.org/10.1182/hematology.2019000054DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6913425PMC
December 2019

The Adolescent and Caregiver Sickle Cell Disease Self-management Skills Checklist: Preliminary Reliability and Validity.

J Pediatr Hematol Oncol 2020 01;42(1):12-19

Department of Psychology.

Adolescents with sickle cell disease (SCD) need assistance in developing the knowledge and skills that contribute to increased disease self-management and successful transition to adult-based health care. This study evaluated the preliminary psychometric properties of the Self-Management Skills Checklist (SMSC and SMSC-C; Adolescent and Caregiver versions), a measure of perceived SCD-specific knowledge and skills. A retrospective cohort study included 114 adolescents (mean=15.6 y) and their caregivers. We examined internal structure and reliability, score changes over time, and group differences. Cronbach coefficient alphas were 0.79 and 0.74 for caregiver-reported Skills and caregiver-reported knowledge, respectively, and 0.77 and 0.44 for adolescent-reported skills and adolescent-reported knowledge, respectively, indicating good internal consistency for 3 of the subscales. Poor reliability in the adolescent-reported knowledge summary score and factor analysis suggest an interpretation item-by-item, independent of one another. Participant group differences in age and chronic transfusion treatment existed in both summary and subscale scores of the SMSC and SMSC-C. Follow-up administrations of the scales indicated an increase in caregiver-reported skills for their adolescents from time 1 scores (M=3.72±0.83) to time 2 scores (M=3.99±0.63) (t16=2.178, P=0.045). Findings provide preliminary support for the usage of the SMSC and continued development to improve its psychometrics.
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http://dx.doi.org/10.1097/MPH.0000000000001618DOI Listing
January 2020

Racial and ethnic disparities in neurocognitive, emotional, and quality-of-life outcomes in survivors of childhood cancer: A report from the Childhood Cancer Survivor Study.

Cancer 2019 10 10;125(20):3666-3677. Epub 2019 Jul 10.

Department of Epidemiology and Cancer Control, St. Jude Children's Research Hospital, Memphis, Tennessee.

Background: Survivors of childhood cancer are at risk of neurocognitive impairment, emotional distress, and poor health-related quality of life (HRQOL); however, the effect of race/ethnicity is understudied. The objective of this study was to identify race/ethnicity-based disparities in neurocognitive, emotional, and HRQOL outcomes among survivors of childhood cancer.

Methods: Self-reported measures of neurocognitive function, emotional distress (the Brief Symptom Inventory-18), and HRQOL (the Medical Outcomes Study Short Form-36 health survey) were compared between minority (Hispanic, n = 821; non-Hispanic black [NHB], n = 600) and non-Hispanic white (NHW) (n = 12,287) survivors from the Childhood Cancer Survivor Study (median age, 30.9 years; range, 16.0-54.1 years). By using a sample of 3055 siblings, the magnitude of same-race/same-ethnicity survivor-sibling differences was compared between racial/ethnic groups, adjusting for demographic and treatment characteristics and current socioeconomic status (SES).

Results: No clear pattern of disparity in neurocognitive outcomes by race/ethnicity was observed. The magnitude of the survivor-sibling difference in the mean score for depression was greater in Hispanics than in NHWs (3.59 vs 1.09; P = .004). NHBs and Hispanics had greater survivor-sibling differences in HRQOL than NHWs for mental health (NHBs: -5.78 vs -0.69; P = .001; Hispanics: -3.87 vs -0.69; P = .03), and social function (NHBs: -7.11 vs -1.47; P < .001; Hispanics: -5.33 vs -1.47; P = .001). NHBs had greater survivor-sibling differences in physical subscale scores for HRQOL than NHWs. In general, the findings were not attenuated by current SES.

Conclusions: Although no pattern of disparity in neurocognitive outcomes was observed, differences across many HRQOL outcomes among minorities compared with NHWs, not attenuated by current SES, were identified. This suggests that further research into environmental and sociocultural factors during and immediately after treatment is needed.
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http://dx.doi.org/10.1002/cncr.32370DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6763349PMC
October 2019

Sickle Cell Clinical Research and Intervention Program (SCCRIP): A lifespan cohort study for sickle cell disease progression from the pediatric stage into adulthood.

Pediatr Blood Cancer 2018 09 24;65(9):e27228. Epub 2018 May 24.

School of Public Health, University of Memphis, Memphis, Tennessee.

Background: Previous natural history studies have advanced the understanding of sickle cell disease (SCD), but generally have not included sufficient lifespan data or investigation of the role of genetics in clinical outcomes, and have often occurred before the widespread use of disease-modifying therapies, such as hydroxyurea and chronic erythrocyte transfusions. To further advance knowledge of SCD, St. Jude Children's Research Hospital established the Sickle Cell Clinical Research and Intervention Program (SCCRIP), to conduct research in a clinically evaluated cohort of individuals with SCD across their lifetime.

Procedures: Initiated in 2014, the SCCRIP study prospectively recruits patients diagnosed with SCD and includes retrospective and longitudinal collection of clinical, neurocognitive, geospatial, psychosocial, and health outcomes data. Biological samples are banked for future genomics and proteomics studies. The organizational structure of SCCRIP is based upon organ/system-specific working groups and is opened to the research community for partnerships.

Results: As of August 2017, 1,044 (92.3% of eligible) patients with SCD have enrolled in the study (860 children and 184 adults), with 11,915 person-years of observation. Population demographics included mean age at last visit of 11.3 years (range 0.7-30.1), 49.8% females, 57.7% treated with hydroxyurea, 8.5% treated with monthly transfusions, and 62.9% hemoglobin (Hb) SS or HbSB -thalassemia, 25.7% HbSC, 8.4% HbsB -Thalassemia, 1.7% HbS/HPFH, and 1.2% other.

Conclusions: The SCCRIP cohort will provide a rich resource for the conduct of high impact multidisciplinary research in SCD.
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http://dx.doi.org/10.1002/pbc.27228DOI Listing
September 2018

Using Qualitative Perspectives of Adolescents with Sickle Cell Disease and Caregivers to Develop Healthcare Transition Programming.

Clin Pract Pediatr Psychol 2017 Dec;5(4):319-329

Department of Hematology, St. Jude Children's Research Hospital.

Youth with sickle cell disease (SCD) are living longer, requiring transition from pediatric to adult health care. Transition programs have been created to improve transition readiness and help patients take responsibility for their health. The aim of this study was to explore the usefulness of current transition materials and identify unmet transition needs from the perspective of adolescents with sickle cell disease (SCD) and caregivers to refine transition programming and interventions. Focus groups were conducted with 14 adolescents with SCD (Mean age = 14.6 years, = 1.9) and 20 caregivers (Mean age = 43.2 years, = 9.3) to gather perspectives about transition to adult care, current transition program materials and recommendations for future programming. Four themes emerged: (a) transition skills and knowledge needs, (b) change in health care responsibility, (c) concerns with adult SCD care, and (d) useful transition readiness strategies and resources. The findings of this study were used to develop Web based educational modules, experiential transition skills learning, and an adolescent and caregiver hematology support group. Findings highlight the need to conduct periodic readiness assessments, provide opportunities and scaffolding to learn skills based on readiness level, and help build social support networks to encourage and facilitate learning.
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http://dx.doi.org/10.1037/cpp0000212DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6532654PMC
December 2017

Pediatric to adult care co-location transitional model for youth with sickle cell disease.

Am J Hematol 2018 01 10;93(1):E30-E32. Epub 2017 Nov 10.

Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee.

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http://dx.doi.org/10.1002/ajh.24953DOI Listing
January 2018

Pediatric to Adult Care Transition: Perspectives of Young Adults With Sickle Cell Disease.

J Pediatr Psychol 2017 10;42(9):1016-1027

Department of Psychology, St. Jude Children's Research Hospital, Memphis, TN.

Objectives: The aim of this study was to explore perspectives of transition and transition readiness of young adult patients (YAs) with sickle cell disease (SCD) who have transitioned to adult health care.

Methods: In all, 19 YAs with SCD (ages 18-30 years) participated in one of three focus groups and completed a brief questionnaire about transition topics. Transcripts were coded and emergent themes were examined using the social-ecological model of adolescent and young adult readiness for transition (SMART).

Results: Themes were consistent with most SMART components. Adult provider relationships and negative medical experiences emerged as salient factors. YAs ranked choosing an adult provider, seeking emergency care, understanding medications/medication adherence, knowing SCD complications, and being aware of the impact of health behaviors as the most important topics to include in transition programming.

Conclusions: The unique perspectives of YAs can inform the development and evaluation of SCD transition programming by incorporating the identified themes.
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http://dx.doi.org/10.1093/jpepsy/jsx088DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6251560PMC
October 2017

Clinic Attendance of Youth With Sickle Cell Disease on Hydroxyurea Treatment.

J Pediatr Hematol Oncol 2017 07;39(5):345-349

Departments of *Psychology †Hematology, St. Jude Children's Research Hospital, Memphis, TN.

Objective: The objective of this study is to describe rates of clinic attendance of youth with sickle cell disease prescribed hydroxyurea and examine potential demographic and medical factors related to consistent clinic attendance.

Methods: Participants included 148 youth diagnosed with sickle cell disease and prescribed hydroxyurea during a single calendar year. Clinic attendance and potential demographic and medical factors related to attendance were extracted via systematic retrospective medical chart review.

Results: Youth attended 90.3% of scheduled appointments and 85.1% of youth attended at least 80% of scheduled clinic appointments during the study window. Adjusting for other factors, multivariate analysis revealed families with fewer children in the household, families with private insurance, youth experiencing fever, and youth not experiencing pain during the calendar year were more likely to consistently attend clinic visits.

Conclusions: Adherence to clinic appointments is critical to optimizing health outcomes for youth with sickle cell disease and integral for adequate monitoring of youth prescribed hydroxyurea, in particular. Findings may aid providers in appropriately identifying possible barriers to clinic attendance to develop attendance promotion interventions.
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http://dx.doi.org/10.1097/MPH.0000000000000859DOI Listing
July 2017

Body Image and Risk Behaviors in Youth with HIV.

AIDS Patient Care STDS 2017 Apr 23;31(4):176-181. Epub 2017 Mar 23.

1 Department of Infectious Diseases, St. Jude Children's Research Hospital , Memphis, Tennessee.

Body image concerns are common among people living with HIV. Among adults with HIV, body image concerns have been shown to be related to risky sexual behaviors; little research has been conducted among youth living with HIV (YLWH). The current study examined the predictors, including body image, of sexual risk behaviors among YLWH. Adolescents from a single clinic (n = 143; age range, 16-24 years; 69% male; 95% African American) completed a computerized self-report survey to assess demographic, behavioral, and body image domains. Demographic and clinical data were abstracted from the medical record. Logistic regression analyses assessed associations between risk factors and risky sexual behaviors. Results indicated that YLWH who reported less favorable body image perceptions (p = 0.04) and more sexual partners (p = 0.05) were less likely to use condoms during their last sexual encounter. YLWH with six or more sexual partners were more likely to use drugs or alcohol during their last sexual encounter (p = 0.03). A belief that their HIV medications changed their body physically (p = 0.05), history of HIV-related complications (p = 0.03), an undetectable viral load at their most recent clinical laboratory draw (p = 0.01), and having a high school diploma or equivalent (p = 0.001) were independently associated with disclosure of participant's HIV status to a romantic/sexual partner. Findings suggest that body image perceptions may influence risky sexual behavior in YLWH. Further study is warranted to understand and intervene upon this relationship to improve individual and public health outcomes.
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http://dx.doi.org/10.1089/apc.2016.0259DOI Listing
April 2017

Characterizing Body Image in Youth with HIV.

AIDS Behav 2016 08;20(8):1585-90

Department of Infectious Diseases, St. Jude Children's Research Hospital, 262 Danny Thomas Place, Mailstop 600, Memphis, TN, 38105, USA.

Emerging research in adults with HIV suggests negative body image may be found at a higher rate in this group. To date, few studies have examined body image in adolescents living with HIV. This exploratory study aimed to characterize body image perceptions among youth living with HIV. Adolescents (n = 143; age range 16-24 years; 69 % male) completed an Audio Computer Assisted Self-Interview Questionnaire that assessed body image, psychosocial, medical and sociodemographic information. Medical history and physical functioning information were abstracted from medical records. Results showed normative global body image on the Multidimensional Body Self-Relations Questionnaire-Appearance Scales. Some subscale elevations were observed; including decreased interest in self-care and appearance, as well as concerns with individual body areas. Overall, youth reported preference for own body shape on the Figure Rating Scale; however, 41 % of youth classified as "overweight" per CDC body mass index reported contentment with current body size. Further, 47 % of youth classified as "normal" weight desired to have larger body size. Youth identified as men who have sex with men most often reported desiring larger body size. Implications for clinical care are discussed.
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http://dx.doi.org/10.1007/s10461-015-1271-zDOI Listing
August 2016

International and Interdisciplinary Identification of Health Care Transition Outcomes.

JAMA Pediatr 2016 Mar;170(3):205-11

End-Stage Kidney Disease Program, University of North Carolina-Chapel Hill.

Importance: There is a lack of agreement on what constitutes successful outcomes for the process of health care transition (HCT) among adolescent and young adults with special health care needs.

Objective: To present HCT outcomes identified by a Delphi process with an interdisciplinary group of participants.

Design, Setting, And Participants: A Delphi method involving 3 stages was deployed to refine a list of HCT outcomes. This 18-month study (from January 5, 2013, of stage 1 to July 3, 2014, of stage 3) included an initial literature search, expert interviews, and then 2 waves of a web-based survey. On this survey, 93 participants from outpatient, community-based, and primary care clinics rated the importance of the top HCT outcomes identified by the Delphi process. Analyses were performed from July 5, 2014, to December 5, 2014.

Exposures: Health care transition outcomes of adolescents and young adults with special health care needs.

Main Outcomes And Measures: Importance ratings of identified HCT outcomes rated on a Likert scale from 1 (not important) to 9 (very important).

Results: The 2 waves of surveys included 117 and 93 participants as the list of outcomes was refined. Transition outcomes were refined by the 3 waves of the Delphi process, with quality of life being the highest-rated outcome with broad agreement. The 10 final outcomes identified included individual outcomes (quality of life, understanding the characteristics of conditions and complications, knowledge of medication, self-management, adherence to medication, and understanding health insurance), health services outcomes (attending medical appointments, having a medical home, and avoidance of unnecessary hospitalization), and a social outcome (having a social network). Participants indicated that different outcomes were likely needed for individuals with cognitive disabilities.

Conclusions And Relevance: Quality of life is an important construct relevant to HCT. Future research should identify valid measures associated with each outcome and further explore the role that quality of life plays in the HCT process. Achieving consensus is a critical step toward the development of reliable and objective comparisons of HCT outcomes across clinical conditions and care delivery locations.
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http://dx.doi.org/10.1001/jamapediatrics.2015.3168DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6345570PMC
March 2016

Caregiver Perspectives of Stigma Associated With Sickle Cell Disease in Adolescents.

J Pediatr Nurs 2016 Jan-Feb;31(1):55-63. Epub 2015 Nov 1.

St. Jude Children's Research Hospital, Memphis TN.

Unlabelled: Patients and families affected by various medical conditions report experiencing health-related stigma, which contributes to detrimental physical, psychological, and social outcomes. Sickle cell disease (SCD) is a genetic disorder that affects 89,000 individuals in the United States and is often associated with negative stereotypes and incorrect assumptions. The present study explored the perception of stigma as reported by caregivers of adolescents with SCD.

Design And Methods: Focus groups were conducted with 20 caregivers of patients with SCD. Focus groups were audio recorded and transcribed. The data were coded independently by two authors, and then reviewed conjointly until consensus was reached.

Results: Caregivers reported the perception of stigma in academic, medical, community, and family settings. They also reported internalized stigma including negative feelings toward having a child with SCD, feeling upset with others, and seeing negative emotions in their child due to SCD. Caregivers reported a general lack of knowledge about SCD across settings.

Conclusion: These results demonstrated that stigma may affect individuals with SCD across multiple settings. These results also highlighted areas for intervention, with a focus on increasing communication and education toward medical providers, schools, and communities.

Practical Implications: Interventions can utilize technology, social media, and advertisement campaigns. Additionally, support groups for patients with SCD may help decrease stigma and validate patients' experiences.
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http://dx.doi.org/10.1016/j.pedn.2015.09.011DOI Listing
February 2017

APHON/ASPHO Policy Statement for the Transition of Patients With Sickle Cell Disease From Pediatric to Adult Health Care.

J Pediatr Oncol Nurs 2015 Nov-Dec;32(6):355-9

Boston University School of Medicine/Boston Medical Center, Boston, MA, USA.

Unlabelled: With advances in medical care, the majority of children with sickle cell disease are surviving to adulthood. Patients, families, and providers now face the need for this growing population to move from pediatric- to adult-focused care. In order to facilitate a successful transfer to adult health care, and prepare young adults for greater independence, it is recommended that all pediatric patients with sickle cell disease receive transition preparation.

Association Position: As the professional voice of pediatric hematology/oncology healthcare practice, the Association of Pediatric Hematology/Oncology Nurses (APHON) and the American Society of Pediatric Hematology Oncology (ASPHO) recommends that the discussion of transition begin early and is presented as part of the natural process of becoming an adult; that patients, providers, and families are all involved in creating a transition plan and assessing transition preparedness annually; and that transfer of care involve direct communication between the pediatric team and the accepting adult provider.
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http://dx.doi.org/10.1177/1043454215591954DOI Listing
September 2016

Genetic education and sickle cell disease: feasibility and efficacy of a program tailored to adolescents.

J Pediatr Hematol Oncol 2014 Oct;36(7):572-7

Departments of *Psychology †Hematology ‡Biostatistics, St Jude Children's Research Hospital, Memphis, TN.

Sickle cell disease (SCD) genetic knowledge is important when individuals make reproductive decisions. This study assessed feasibility and efficacy of delivering basic genetic information to 101 adolescents with SCD. Participants completed a questionnaire to test SCD genetic knowledge at 3 timepoints: before genetic education session (pretest), after the session (posttest), and 6 months later (follow-up). Scores at 3 timepoints were compared by Wilcoxon signed-rank tests, and group differences were compared by Wilcoxon-Mann-Whitney and Kruskal-Wallis tests. Participants' median scores significantly increased from pretest to posttest and from pretest to follow-up. Males had a greater change in scores than females. Scores decreased slightly from posttest to follow-up. Participants with HbSS/HbSβ⁰-thal genotype and participants with more prior pain episodes exhibited a smaller increase in median scores than those with HbSC/HbSβ⁺-thal genotype and no prior pain history; however, all groups had substantial gains from pretest to posttest and follow-up tests demonstrating that adolescents with SCD can learn basic genetics. This study established that genetic education can successfully be incorporated in transition to adult care programs for adolescents with SCD. Genetic education should be included in the standard plan of care for adolescents with SCD to assist them in making informed reproductive choices.
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http://dx.doi.org/10.1097/MPH.0000000000000226DOI Listing
October 2014

Transition from pediatric to adult care in sickle cell disease: perspectives on the family role.

J Pediatr Nurs 2014 Mar-Apr;29(2):158-67. Epub 2013 Oct 16.

St. Jude Children's Research Hospital, Memphis, TN.

Transition from pediatric to adult care poses challenges for adolescents with sickle cell disease (SCD). This study explored the transition perspectives of adolescents with SCD, their siblings, and caregivers. Focus groups were conducted with 12 African American families. Adolescents, siblings, and caregivers demonstrated awareness of transition and need for disease management responsibility. Siblings' and caregivers' concerns included adolescent medication adherence. Family concerns included leaving the pediatric environment and adult providers' lack of knowledge. Families recommended more transition preparation opportunities. Family members' perspectives are valuable in informing transition planning. Family-focused interventions designed to prepare and support families during transition are necessary.
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http://dx.doi.org/10.1016/j.pedn.2013.10.002DOI Listing
April 2015

Sickle cell disease patients' perceptions of emergency department pain management.

J Natl Med Assoc 2012 Sep-Oct;104(9-10):449-54

St. Jude Children's Research Hospital, Department of Psychology, Memphis, TN 38105, USA.

Patients with sickle cell disease (SCD) experience painful crises that often require admission to the emergency department (ED) for pain management. Factors such as ED overcrowding and negative perception and stigmatization of SCD may impact patients' perceptions of the quality of pain management in the ED. Data from a multisite prospective cohort study was assessed to determine whether demographic (age and sex), clinical (time to administration of initial analgesia, number of analgesic doses, discharge disposition, and clinical site), or interpersonal factors (separately measured perceptions of being treated with trust and respect by ED triage nurses, nurses, and physicians) were associated with patient ratings of their pain management in the ED. Patients were adults with SCD seen at 3 EDs (2 urban and 1 rural). Demographic and clinical information was derived from medical record review; interpersonal and ED pain management ratings were derived from interviews conducted 1 week post ED visit. A total of 209 interviews by 98 patients were analyzed. Results indicated significant differences among the ED sites on the demographic, clinical, and interpersonal factors. Overall, patients reported being treated with trust and respect by ED clinicians. Adjusted logistic regression analyses indicated that ED clinical site 1 (odds ratio [OR], 10.42; 95% confidence interval [Cl], 1.44-7.36) and being treated with trust and respect by the ED physician (OR, 25.53; 95% CI, 2.07-314.96) predicted good ED pain management ratings. Interpersonal health care experiences may be an important indicator of patient satisfaction and quality of care received by patients with SCD in the ED.
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http://dx.doi.org/10.1016/s0027-9684(15)30199-1DOI Listing
February 2013

Exploring family communication about sickle cell disease in adolescence.

J Pediatr Oncol Nurs 2012 Nov-Dec;29(6):323-36. Epub 2012 Sep 19.

College of Nursing, University of Tennessee Health Science Center, Memphis, TN 38105, USA.

Sickle cell disease (SCD) is a lifelong disorder that involves progressive organ damage and requires ongoing medical attention to prevent and treat episodic acute complications. Children with SCD need ongoing monitoring and extra attention that may be stressful to family members. Communication within families can help resolve family stress and may be associated with medical follow-up and management of SCD. Focus groups were conducted with 12 African American families to explore the communication that occurred within and outside of the family from the perspectives of adolescents with SCD, siblings, and parents. Factors that influence family communication were explored. The extended family was an important social network and resource to adolescents, siblings, and parents. Family member knowledge of SCD was an important factor that influenced communication about SCD; adolescents and parents communicated more easily than siblings and also reported having more knowledge of SCD than siblings. Future research focusing on the knowledge of immediate and extended family members and their recognition of their contribution to the child with SCD is recommended.
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http://dx.doi.org/10.1177/1043454212456086DOI Listing
March 2013

A qualitative analysis of best self-management practices: sickle cell disease.

J Natl Med Assoc 2010 Nov;102(11):1033-41

Department of Emergency Medicine, Institute for Healthcare Studies, Feinberg School of Medicine, Northwestern University, Chicago, Illinois 60611, USA.

Background: Sickle cell disease (SCD) is associated with serious comorbidities resulting in a shortened lifespan, and many clients suffer from frequent pain episodes. However, others successfully manage their disease in the outpatient setting without the need for frequent health care utilization. The purpose of this project was to describe specific strategies used by adult clients with sickle cell disease to achieve optimal physical health.

Method: A Best Self-management Practices workshop was held in conjunction with the Sickle Cell Disease Association of America meeting. A panel discussion was organized; adult clients were recruited for participation. The workshop was divided into 3 topics: complementary and alternative medicine, psychosocial issues, and work/education/training. Panel discussions were audiotaped, transcribed, and analyzed using the constant comparative method.

Results: Seven adult patients with sickle cell disease and 1 social worker participated. The following themes emerged: self-awareness, emotional support, career selection and success factors, nutrition, advocacy, knowledge, physical, and complementary and alternative medicine. Self-awareness was the most reported strategy with emphasis on journaling and body awareness. Emotional support included spiritual support, friends, family, professional counseling, and spiritual support. A variety of suggestions were discussed related to the other themes. All participants used many strategies daily to maintain optimal health.
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http://dx.doi.org/10.1016/s0027-9684(15)30730-6DOI Listing
November 2010

Psychosocial factors and perspectives on weight gain and barriers to weight loss among adolescents enrolled in obesity treatment.

J Clin Psychol Med Settings 2010 Jun;17(2):98-102

Institute for Healthcare Studies, Feinberg School of Medicine, Northwestern University, 750 N. Lake Shore Drive, 10th floor, Chicago, IL 60611-3152, USA.

The aim of the current descriptive study was to explore factors related to psychosocial wellbeing, weight gain and perceived barriers to exercise and nutrition in 135 obese adolescents enrolled in a multidisciplinary weight management program. Participants completed initial intake interviews, which included information about psychosocial well-being, factors associated with weight gain, and barriers to exercise and nutrition. We examined the associations among psychosocial factors and participants' attendance compliance for the nutrition, exercise, and behavioral support and modification components of the program. Results indicated that familial factors were associated with weight gain, and family, peer, and individual factors were associated with barriers to healthy eating and exercise. Among the psychosocial factors, history of trauma was negatively associated with compliance. Findings emphasize the importance of addressing psychosocial well-being and using a systems approach to weight management for obese adolescents.
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http://dx.doi.org/10.1007/s10880-010-9186-3DOI Listing
June 2010