Decision Support Needs for Transgender and Gender-Diverse Youth and Families: A Patient-Centered Needs Assessment.

PURPOSE: Due to the intertwining of medical and social decision-making, new approaches to shared decision-making are likely needed for supporting decisions related to the care of transgender and gender-diverse (TGD) adolescents. Prior to developing decision support interventions for TGD youth, a decision support needs assessment must be completed. METHODS: Self-identified TGD youth, family members of TGD youth, clinicians caring for this population, and community advocates participated in one of six group level assessments (GLAs). GLA is a structured, participatory qualitative method that engages diverse groups of stakeholders in generating and evaluating ideas on the topic of interest. Upon completion of all GLAs, a survey was developed and distributed to GLA participants inviting them to rank ideas generated during the GLAs. RESULTS: Six major themes emerged from the GLAs regarding decision support needs, including: improving healthcare provider skills and education, increasing access to support outside the healthcare system, strengthening community and societal support, developing special information resources, supporting youth in leading decision-making about transition, and modifying the healthcare system. In the follow-up survey, improving healthcare provider skills and education was the most commonly chosen top priority. DISCUSSION: Participants identified decision support needs for TGD youth and their families that were mostly distinct from traditional decision support approaches. Participants' focus on the need to improve healthcare provider skills and education provides an opportunity to couple gender-focused education with shared decision-making skills, an approach that may be more sustainable than tools for specific decisions.

Intergenerational Decision Making: The Role of Family Relationships in Medical Decision Making.

A symposium held at the 42nd annual Society for Medical Decision Making conference on October 26, 2020, focused on intergenerational decision making. The symposium covered existing research and clinical experiences using formal presentations and moderated discussion and was attended by 43 people. Presentations focused on the roles of pediatric patients in decision making, caregiver decision making for a child with complex medical needs, caregiver involvement in advanced care planning, and the inclusion of spillover effects in economic evaluations. The moderated discussion, summarized in this article, highlighted existing resources and gaps in intergenerational decision making in four areas: decision aids, economic evaluation, participant perspectives, and measures. Intergenerational decision making is an understudied and poorly understood aspect of medical decision making that requires particular attention as our society ages and technological advances provide new innovations for life-sustaining measures across all stages of the lifespan.

Design and implementation of a decision aid for juvenile idiopathic arthritis medication choices.

BACKGROUND: Randomized trials have demonstrated the efficacy of patient decision aids to facilitate shared decision making in clinical situations with multiple medically reasonable options for treatment. However, little is known about how best to implement these tools into routine clinical practice. In addition, reliable implementation of decision aids has been elusive and spread within pediatrics has been slow. We sought to develop and reliably implement a decision aid for treatment of children with juvenile idiopathic arthritis. METHODS: To design our decision aid, we partnered with patient, parent, and clinician stakeholders from the Pediatric Rheumatology Care and Outcomes Improvement Network. Six sites volunteered to use quality improvement methods to implement the decision aid. Four of these sites collected parent surveys following visits to assess outcomes. Parents reported on clinician use of the decision aid and the amount of shared decision making and uncertainty they experienced. We used chi-square tests to compare eligible visits with and without use of the decision aid on the experience of shared decision making and uncertainty. RESULTS: After 18 rounds of testing and revision, stakeholders approved the decision aid design for regular use. Qualitative feedback from end-users was positive. During the implementation project, the decision aid was used in 35% of visits where starting or switching medication was discussed. Clinicians used the decision aid as intended in 68% of these visits. The vast majority of parents reported high levels of shared decision making following visits with (64/76 = 84%) and without (80/95 = 84%) use of the decision aid (p = 1). Similarly, the vast majority of parents reported no uncertainty following visits with (74/76 = 97%) and without (91/95 = 96%) use of the decision aid (p = 0.58). CONCLUSIONS: Although user acceptability of the decision aid was high, reliable implementation in routine clinical care proved challenging. Our parsimonious approach to outcome assessment failed to detect a difference between visits with and without use of our aid. Innovative approaches are needed to facilitate use of decision aids and the assessment of outcomes.

Parents' information needs and influential factors when making decisions about TNF-α inhibitors.

BACKGROUND: Parents struggle when making treatment decisions for children with arthritis or other chronic conditions. Understanding their decision-making process is an essential step towards improving the decision-making experience. The objective of this study was to describe parents' information needs and the influences on their decision making about treatment with TNF-α inhibitors. METHODS: Survey domains were developed based on qualitative data and cognitive interviewing. We mailed the survey to parents of children with juvenile idiopathic arthritis or inflammatory bowel disease who had initiated treatment with TNF-α inhibitors in the prior 2 years. Data were analyzed using descriptive and non-parametric statistics. RESULTS: Survey response rate was 54.9 %. Each item had <2 % missing responses. Parents used an array of information sources when deciding about treatment with TNF-α inhibitors. Resources other than their child's specialist were most often used to increase confidence in parents' decisions or because they wanted to know more about other people's experiences being treated with TNF-α inhibitors, rather than due to a lack of understanding. All but two (cost and route of administration) of the influential decision factors were very or extremely important to the majority of participants with factors related to long-term side effects, treatment efficacy, and disease impact being most important. CONCLUSIONS: This study describes parents' information needs and influential factors in treatment decision making. Results suggest that future work should be aimed at helping families weigh risks and benefits, such as through decision support interventions, as well as developing opportunities to include people beyond the family and physician in the decision-making process.

Physicians' Perceptions of Shared Decision Making in Chronic Disease and Its Barriers and Facilitators.

This study assessed pediatric physicians' use of shared decision making (SDM) in 2 chronic conditions. Most physicians indicated that parent and adolescent trust and emotional readiness facilitated SDM, physicians' preferred approach to decision making. At the same time, they perceived few barriers, other than insurance limitations, to using SDM.

Understanding treatment decision making in juvenile idiopathic arthritis: a qualitative assessment.

BACKGROUND: The increase in therapeutic options for juvenile idiopathic arthritis (JIA) has added complexity to treatment decisions. Shared decision making has the potential to help providers and families work together to choose the best possible option for each patient from the array of choices. As part of a needs assessment, prior to design and implementation of shared decision making interventions, we conducted a qualitative assessment of clinicians' current approaches to treatment decision making in JIA. METHODS: Pediatric rheumatology clinicians were recruited from 2 academic children's hospitals affiliated with a quality improvement learning network, using purposive and snowball sampling. Semi-structured interviews elicited how clinicians with prescribing authority (n = 10) interact with families to make treatment decisions. Interviews were audio-recorded and transcribed verbatim. A multi-disciplinary research team used content analysis to analyze the interview data.To validate data from individual interviews and enrich our understanding, we presented the interview results to pediatric rheumatology clinicians attending a learning network meeting (n = 24 from 12 children's hospitals). We then asked the clinicians questions to further identify and discuss areas of variation in the decision-making processes. RESULTS: Clinicians described a decision-making process in which they, rather than the family or other care team members, consistently initiated treatment decisions. Initial treatment options presented to families generally reflected the clinician's preferred treatment approaches, which differed across clinicians. Clinicians used various methods to inform families about treatment options and tailor information according to perceptions of a family's information needs, level of comprehension or mood (e.g. anxiety). The attributes of medication presented to families fell into 4 categories: benefits, risks, logistics and family preferences. Clinicians typically included family members in the decision to initiate JIA treatment after limiting the options to fit the clinical situation and the clinician's own preferences. Family members' preferences were seen as more integral in the decision to stop treatment after symptom remission. CONCLUSIONS: Decision making about initial JIA treatment appears to be largely driven by clinician preferences. Family preferences are more likely to be considered for treatment discontinuation. Opportunities exist to develop, test, and implement tools to facilitate shared decision making in pediatric rheumatology.

Systematic evidence review of newborn screening and treatment of severe combined immunodeficiency.

CONTEXT: Severe combined immunodeficiency (SCID) is a group of disorders that leads to early childhood death as a result of severe infections. Recent research has addressed potential newborn screening for SCID. OBJECTIVE: To conduct a systematic review of the evidence for newborn screening for SCID, including test characteristics, treatment efficacy, and cost-effectiveness. METHODS: We searched Medline and the OVID In-Process & Other Non-Indexed Citations databases. We excluded articles if they were reviews, editorials or other opinion pieces, or case series of fewer than 4 patients or if they contained only adult subjects or nonhuman data. The remaining articles were systematically evaluated, and data were abstracted by 2 independent reviewers using standardized tools. For topics that lacked published evidence, we interviewed experts in the field. RESULTS: The initial search resulted in 719 articles. Twenty-six met inclusion criteria. The results of several small studies suggested that screening for SCID is possible. Interviews revealed that 2 states have begun pilot screening programs. Evidence from large case series indicates that children receiving early stem-cell transplant for SCID have improved outcomes compared with children who were treated later. There is some inconclusive evidence regarding the need for donor-recipient matching and use of pretransplant chemotherapy. Few data on the cost-effectiveness of a SCID-screening program. CONCLUSIONS: Evidence indicates the benefits of early treatment of SCID and the possibility of population-based newborn screening. Better information on optimal treatment and the costs of treatment and screening would benefit policy makers deciding among competing health care priorities.

Impact of false-positive newborn metabolic screening results on early health care utilization.

PURPOSE: To analyze the association between false-positive newborn screening results and health care utilization. METHODS: We surveyed parents regarding their children's health care utilization. Parents of children who received false-positive newborn screening results were primarily enrolled by a screening laboratory in Pennsylvania. Parents of children with normal results were recruited through the Massachusetts birth registry. We used bivariate tests and multivariate regression to assess the association between newborn screening results and primary care utilization, emergency room use, and hospitalization by the age of 6 months. RESULTS: Our sample included 200 children with false-positive results and 137 with normal results. Variation in recruitment strategies led to sample children with false-positive results being more likely to be non-white, have unmarried parents, and be of lower socioeconomic status. After adjusting for significant covariates, such as age, race, and socioeconomic status, there were no significant associations between newborn screening results and child health care utilization. CONCLUSIONS: Despite the reported negative psychosocial effects of false-positive results, our study found no impact on early health care utilization. These results may assist in economic analyses of newborn screening as they suggest that medical costs associated with false-positive results are limited to the cost of diagnostic testing and follow-up.