An Updated Equitable Model of Readiness for Transition to Adult Care: Content Validation in Young People With Sickle Cell Disease.

Importance: Despite elevated health risks during young adulthood, many adolescents and young adults with serious health care needs face barriers during the transfer to an adult specialty practitioner, and health disparities may occur during the transition. Objective: To validate the content of an updated Social-Ecological Model of Adolescent and Young Adult Readiness for Transition to Promote Health Equity (SMART-E) in a group of adolescents and young adults with sickle cell disease (SCD) and their supports. Design, Setting, and Participants: Health equity framework components were reviewed. Systems of power (eg, institutional and practitioner bias) and environments or networks (eg, peer or school support) were added as SMART-E preexisting factors, and health literacy was included within readiness factors. Adolescents and young adults aged 16 to 29 years with SCD, caregivers, and practitioners participated in this convergent, mixed-methods study within Children's Hospital of Philadelphia between January and August 2022. Main Outcomes and Measures: Content validity was assessed through nominations of top 3 most important transition barriers prior to interviews and focus groups, ratings on importance of SMART-E factors (0-4 scale; ratings >2 support validity) after interviews and focus groups, nominations of 3 most important factors for transition and for health equity, and qualitative content analysis of interview transcripts. Results: The study enrolled 10 pediatric adolescents and young adults (mean [SD] age, 18.6 [2.9] years; 4 female and 6 male), 10 transferred adolescents and young adults (mean [SD] age, 22.9 [2.1] years; 8 female and 2 male), 9 caregivers (mean [SD] age, 49.8 [8.7] years; 5 female and 4 male), and 9 practitioners (mean [SD] age, 45.6 [10.5] years; 8 female and 1 male). Quantitative ratings supported the content validity of SMART-E and met established criteria for validity. Systems of power was the most endorsed transition barrier (14 of 38 participants) reported prior to interviews and focus groups. After the interview, participants endorsed all SMART-E factors as important for transition, with new factors systems of power and environments and networks rated at a mean (SD) 2.8 (1.23) and 3.1 (0.90), respectively, on a 0 to 4 scale of importance. The most important factors for transition and equity varied by participant group, with all factors being endorsed, supporting the comprehensiveness of SMART-E. Qualitative data corroborated quantitative findings, further supporting validity, and minor modifications were made to definitions. Conclusions and Relevance: SMART-E obtained initial content validation with inclusion of health equity factors for adolescents and young adults with SCD, caregivers, and practitioners. The model should be evaluated in other populations of adolescents and young adults with chronic disease.

Acceptability, Feasibility, and Quality of Telehealth for Adolescent Health Care Delivery During the COVID-19 Pandemic: Cross-sectional Study of Patient and Family Experiences.

BACKGROUND: Data regarding the acceptability, feasibility, and quality of telehealth among adolescents and young adults (AYA) and their parents and caregivers (caregivers) are lacking. OBJECTIVE: The aim of this study was to assess the noninferiority of telehealth versus in-person visits by comparing acceptability with respect to efficiency, effectiveness, equity, patient-centeredness, and confidentiality. METHODS: Cross-sectional web-based surveys were sent to caregivers and AYA following video visits within an Adolescent Medicine subspecialty clinic in May-July 2020. Proportions of AYA and caregivers who rated telehealth as noninferior were compared using chi-squared tests. Feasibility was assessed via items measuring technical difficulties. Deductive thematic analysis using the Institute of Medicine dimensions of health care quality was used to code open-ended question responses. RESULTS: Survey response rates were 20.5% (55/268) for AYA and 21.8% (123/563) for caregivers. The majority of the respondents were White cisgender females. Most AYA and caregivers rated telehealth as noninferior to in-person visits with respect to confidentiality, communication, medication management, and mental health care. A higher proportion of AYA compared to caregivers found telehealth inferior with respect to confidentiality (11/51, 22% vs 3/118, 2.5%, P<.001). One-quarter (14/55) of the AYA patients and 31.7% (39/123) of the caregivers reported technical difficulties. The dominant themes in the qualitative data included advantages of telehealth for efficiency and equity of health care delivery. However, respondents' concerns included reduced safety and effectiveness of care, particularly for patients with eating disorders, owing to lack of hands-on examinations, collection of vital signs, and laboratory testing. CONCLUSIONS: Telehealth was highly acceptable among AYA and caregivers. Future optimization should include improving privacy, ameliorating technical difficulties, and standardizing at-home methods of obtaining patient data to assure patient safety.

Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium.

Despite a remarkable increase in the genomic profiling of cancer, integration of genomic discoveries into clinical care has lagged behind. We report the feasibility of rapid identification of targetable mutations in 153 pediatric patients with relapsed/refractory or high-risk leukemias enrolled on a prospective clinical trial conducted by the LEAP Consortium. Eighteen percent of patients had a high confidence Tier 1 or 2 recommendation. We describe clinical responses in the 14% of patients with relapsed/refractory leukemia who received the matched targeted therapy. Further, in order to inform future targeted therapy for patients, we validated variants of uncertain significance, performed ex vivo drug-sensitivity testing in patient leukemia samples, and identified new combinations of targeted therapies in cell lines and patient-derived xenograft models. These data and our collaborative approach should inform the design of future precision medicine trials. SIGNIFICANCE: Patients with relapsed/refractory leukemias face limited treatment options. Systematic integration of precision medicine efforts can inform therapy. We report the feasibility of identifying targetable mutations in children with leukemia and describe correlative biology studies validating therapeutic hypotheses and novel mutations.See related commentary by Bornhauser and Bourquin, p. 1322.This article is highlighted in the In This Issue feature, p. 1307.