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.

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.

BACKGROUND:Adolescence and young adulthood are vulnerable developmental periods for individuals with sickle cell disease (SCD), particularly given the impact of social inequities, challenges with transitioning to adult healthcare services, and increased risk for morbidity and mortality. Systems of power, such as institutionalized and interpersonal manifestations of bias, could impact SCD transfer and engagement in adult care through their influence on healthcare transition readiness; yet research in this area is limited. OBJECTIVE:To characterize how systems of power impact transition readiness factors described in the Social-ecological Model of AYA Readiness for Transition to Promote Health Equity (SMART-E) framework at the patient, caregiver, and practitioner levels. METHODS:Pediatric adolescents and young adults (AYA), transferred AYA, caregivers, and practitioners participated in semi-structured focus groups and individual interviews examining health equity and systems of power during healthcare transition. Focus groups/interviews were transcribed and coded using a deductive approach via the updated SMART-E framework. RESULTS:Ten pediatric AYA with SCD, nine transferred AYA with SCD, eight caregivers, and nine practitioners participated in a focus group or interview. Qualitative findings across reporters emphasize the impact of systems of power (e.g., racial bias and disease stigma) on knowledge, skills and self-efficacy, beliefs and expectations, goals and motivation, and emotions and psychosocial functioning at the patient, caregiver, and practitioner levels. CONCLUSION:Systems of power are prevalent with respect to transition barriers for AYA with SCD and their supports. Structural, institutional, and individual factors with potential to reduce the influence of systems of power should be further identified and targeted for intervention.

combination of MEK inhibitor with venetoclax is synergistic.

BackgroundCaregivers and adolescents and young adult (AYA) cancer survivors may be at greater psychosocial risk from the COVID-19 pandemic than healthy peers due to complex and traumatic medical histories. This study describes COVID-19-related event exposures, impact, and distress among a large sample of caregivers and AYA cancer survivors and the relationship of these variables to demographic and cancer characteristics. ProcedureFrom May 2020 to December 2021, 422 caregivers and 531 AYA survivors completed the COVID-19 Exposures and Family Impact Survey (CEFIS) and CEFIS-AYA, respectively. Total COVID-19-related exposures, average COVID-19-related impact, and COVID-19-related distress were calculated. Conventional content analysis was used to analyze free-text responses about the negative and positive effects of COVID-19. ResultsCaregivers and AYA reported an average of 7.4-7.8 COVID-19 exposures to pandemic-related events and a slightly negative impact of COVID-19 across psychosocial domains, with some positive impacts reported. COVID-19-related distress was moderate and clinically meaningful (4.9-5.2/10) for AYA and caregivers. Racial and ethnically minoritized AYA and caregivers reported higher COVID-19-related distress than non-Hispanic white caregivers. For AYA, distress was also higher among female, college-age (18-22 years), and long-term survivors compared with males, younger AYA, White and those recently off treatment. CEFIS outcomes remained relatively stable over time. ConclusionsCOVID-19 had a significant and consistent negative impact on caregivers and AYA survivors. Racial and ethnically minoritized families and female, college-age, and long-term AYA survivors may require additional psychosocial support. Assessing for COVID-19 impact and distress is important in pediatric oncology to evaluate adjustment and plan targeted interventions.

Compound sensitivity data for primary patient samples.

combination of MEK inhibitor with venetoclax in primary patient samples and KOPN-8 cells.

Young adult caregivers (YACs) are faced with the atypical challenge of providing informal (i.e. not paid) care to a family member, while simultaneously at a critical developmental stage in which many major life decisions and milestones are taking place. Adding the challenge of caring for a family member during this already complex time period may have a detrimental impact on young adults' (YAs) own overall health and well-being. The aim of this study was to examine differences in overall health, psychological distress, and financial strain among a propensity matched sample of YACs compared to young adult non-caregivers (YANCs) from a nationally representative database and to examine differences in these outcomes by caregiving role (caring for a child vs. other family member). YAs (aged 18-39, N = 178) identifying as a caregiver (n = 74) were matched with YANCs (n = 74) on age, gender, and race. Results revealed that YACs exhibited higher psychological distress, lower overall health and more sleep disturbance and higher financial strain compared to YANCs. YAs caring for family members other than children also reported higher anxiety and fewer hours spent caregiving compared to YAs caring for a child. YACs appear to be at risk for impairments in health and well-being compared to their matched peers. Longitudinal research is needed to understand how caregiving during young adulthood impacts health and well-being across time.

Inhibitor Recommendation Usage Summary.

Supplementary Data file including supplementary methods and clinical case vignettes.

Gender-affirming care (GAC) is critical to the well-being of transgender and gender diverse youth and was limited by COVID-19 stay-at-home orders. Telehealth created opportunities for youth to continue receiving lifesaving care. We examined the attitudes of patients (n=21) and caregivers (n=38) receiving telehealth-delivered GAC (TGAC) from May to July 2020. Participants completed surveys after telehealth visits. Descriptive statistics compared telehealth with in-person visits across key domains. Overall, 86.5% of patients and 95.4% of caregivers were satisfied with medical TGAC and 94.3% and 93.3% were satisfied with behavioral health TGAC. Future research should determine the effectiveness of TGAC and identify areas for improvement.

Introduction: Although over 95% of children with sickle cell disease (SCD) live into adulthood, the transition period between pediatric and adult medical care holds the highest rate of morbidity and mortality across the lifespan. Transition is also characterized by systemic inequities including limited access to care, implicit bias, and disease stigma. Many adolescents and young adults (AYA) are not successful in transferring to an adult hematology provider, further compounding barriers to care. The purpose of this study was to characterize barriers and facilitators of the transition from pediatric to adult care with an emphasis on how systems of inequity impact AYA with SCD transition readiness factors. Method: Pediatric and transferred AYA completed questionnaires and then participated in a semi-structured individual interview or focus group. Questionnaires assessed transition barriers by asking participants to rate the top 3 challenges to transition from a list of common transition-related stressors. In addition, AYA responded to two questions inquiring about the impact of discrimination on healthcare and transition with the option of elaborating in open-ended text responses, and they completed the Perceptions of Racism in Children and Youth (PRaCY) instrument which describes AYA experiences with discrimination outside of the medical system. Individual and focus group interviews were used to obtain detailed descriptions and examples of barriers to transition and how systems of inequity (e.g., racism, socioeconomic disadvantage, etc.) impact AYA expectations or experiences with adult care. Quantitative data were assessed using means and standard deviations. Qualitative data from open-ended questionnaire responses and interviews were analyzed using a deductive-inductive approach via a revised version of the Social-ecological Model of AYA Readiness for Transition (SMART) informed by the Health Equity Framework. Overlaps among systems of inequity and other transition barriers are reported here. Results: Ten AYA with SCD receiving pediatric hematology care (Mage = 18.6; 5 boys, 4 girls, 1 nonbinary; 9 HbSS/Sβ0, 1 HbSC) and 10 AYA with SCD who had transferred to adult care (Mage = 23, 2 men, 8 women; 7 HbSS, 3 HbSC/SD; M years since last pediatric hematology visit/transfer = 1.17) completed questionnaires. All AYA identified as Black or African American, and one AYA also identified as mixed-race. AYA ranked a comprehensive range of top 3 challenges to transition (see Figure 1). Systems of inequity was among the most endorsed barriers alongside skills in being independent in their healthcare, adult provider relationships, beliefs/expectations, and insurance. Overall, AYA reported an average of 2.0 (SD = 1.63) experiences with discrimination on the PRaCY (pediatric: M = 1.78, SD = 1.30, range = 0 to 4; transferred: M = 2.20, SD = 1.93, range = 0 to 6). Further, 10% of AYA reported that their personal identity has impacted their healthcare, and 20% endorsed expecting or experiencing their personal identity (i.e., race, gender, income, sexual orientation) negatively impacting their transition to adult care. Of the participants in the questionnaire portion of the study, 95% (10 pediatric and 9 transferred) were interviewed. All AYA reported that systems of inequity are relevant to transition, though transferred AYA provided more detailed examples of how systemic inequities amplify transition barriers than pediatric patients. Qualitative themes of how systems of inequity impact the quantitatively most endorsed barriers are described in Table 1. Themes emphasize the impact of inequities related to racism, sexism, and socioeconomic disadvantage during engagement in adult-oriented care. Conclusions: AYA across pediatric conditions experience barriers to transition; however, these barriers are amplified for AYA with SCD due to the pervasive impact of systems of inequity. Findings suggest that patients might under-report experiences with systemic inequities on quantitative measures relative to qualitative discussions. Systems of inequity should be incorporated into the SMART framework as a pre-existing factor to promote health equity during transition. Institutional and clinician-level interventions should address interpersonal barriers, implicit bias, and stigma, yet a focus on systemic change is necessary to reduce the compounded burden for this population. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal