Disease-modifying therapies for sickle cell disease: Decisional needs and supports among adolescents and young adults.

BACKGROUND: Shared decision-making is one promising solution to addressing barriers in use of disease-modifying therapies for adolescents and young adults (AYAs) with sickle cell disease (SCD). A thorough understanding of decisional needs can guide the development of decisional supports and promote shared decision-making. PROCEDURE: Informed by the Ottawa Decision Support Framework (ODSF), we conducted a qualitative analysis to assess decisional needs and supports reported by AYAs with SCD, their caregivers, and healthcare providers. Semi-structured qualitative interviews were conducted with AYAs and their caregivers, and online crowdsourcing was used with SCD providers. Thematic and descriptive content analyses were used to summarize perspectives on decisional needs and supports regarding disease-modifying therapies. RESULTS: Fourteen AYAs (Mage  = 21 years, 57% male, 93% non-Hispanic Black, 79% HbSS), 11 caregivers (80% female, 100% non-Hispanic Black), and 40 healthcare providers (65% female, 65% non-Hispanic White, Myears in practice  = 14.8 years, 75% physicians) participated. Thematic analysis revealed needs related to: decisional conflict, inadequate knowledge, unclear expectations, and inadequate supports and resources. Six forms of support emerged as important for decision-making: establishing an open and trusting patient/family-provider relationship, providing information, accepting ambivalence and unreadiness, supporting implementation of a decision, addressing inadequate health and social services, and promoting adequate social, emotional, and instrumental help. CONCLUSIONS: This is the first study to assess decisional needs and supports for AYAs with SCD considering disease-modifying therapies. Additional research is needed to examine which decision supports are the most impactful to promote effective shared decision-making in this population.

Exploring Referral and Service Utilization Patterns Within an Outpatient Interdisciplinary Pediatric Chronic Pain Program.

Purpose: We examine referral sources and clinical characteristics for youth presenting to an outpatient interdisciplinary pediatric chronic pain program. Patients and Methods: Referral data were extracted from the electronic health record. PROMIS Pediatric Anxiety and Pain Interference Scales were administered at an initial evaluation visit. Results: The program received 1488 referrals between 2016 and 2019, representing 1338 patients, with increasing volume of referrals over time. Referrals were primarily from orthopedics (19.6%), physical medicine and rehabilitation (18.8%), neurology (14.4%), and rheumatology (12.6%). Patients referred were primarily female (75.4%), White (80.1%), English-speaking (98.4%) adolescents (median=15.0 years). Of those referred, 732 (54.7%) attended an interdisciplinary evaluation (ie, with ≥2 disciplines). Adolescent anxiety was within the expected range by self-report (N=327, MT-score=55.67) and parent proxy-report (N=354, MT-score=57.70). Pain interference was moderately elevated by self-report (N=323, MT-score=61.52) and parent proxy-report (N=356, MT-score=64.02). There were no differences between patients referred who attended versus did not attend an interdisciplinary evaluation based on age, sex, ethnicity, or language. A smaller than expected proportion of referred Black patients (44%, P=0.02) and patients referred from orthopedics (40%) or pulmonology (11%) attended an evaluation, whereas a larger than expected proportion of those referred from physical medicine and rehabilitation (78%) were evaluated (P<0.001). Conclusion: Results highlight the demand for outpatient interdisciplinary pediatric chronic pain treatment. Findings can inform decisions related to staffing and service design for pediatric hospitals that aim to establish or grow outpatient pediatric chronic pain programs.

Psychological Symptoms and Service Utilization in Prepubertal and Pubertal Transgender and Gender-Diverse Patients.

STUDY OBJECTIVE: To describe the demographic characteristics and psychological symptoms of gender-diverse patients and to compare symptoms between prepubertal and pubertal subgroups METHODS: This was a retrospective chart review of all gender-diverse patients seen by at least 1 provider at the Nemours Gender Wellness Program from March 2015 to December 2020. The extracted data included demographic and psychosocial characteristics at baseline and 1-year follow-up visits. Pubertal status was determined by Tanner staging by a pediatric endocrinologist or gynecologist. Descriptive statistics were used to compare these variables between prepubertal and pubertal subgroups. RESULTS: Our sample included 177 individuals at baseline and 96 subjects at the 1-year follow-up visit. Most patients were White (83.0%), non-Hispanic (92.0%), transgender male (72.9%), and pubertal (90.4%). Compared with prepubertal patients, at the baseline visit, pubertal patients had significantly higher rates of current (68.1% vs 17.6%, P < .001) and lifetime (80.0% vs 23.5%, P < .001) depressive symptoms, current anxiety symptoms (70.0% vs 41.2%, P = .01), lifetime suicide attempts (12.5% vs 0%, P < .001), and a formal diagnosis of an eating disorder (5.0% vs 0%, P < .001). Symptoms did not change significantly over time from baseline to the 1-year follow-up visit. CONCLUSION: We found elevated rates of psychological symptoms and diagnoses in gender-diverse youth, with higher rates in pubertal compared with prepubertal patients. By elucidating how the psychosocial characteristics of gender-diverse children and adolescents differ based on pubertal status, these data can be used to improve current outreach and treatment strategies for transgender pediatric patients.

Outcomes of Menstrual Management Use in Transgender and Gender-Diverse Adolescents.

OBJECTIVE: To describe and compare the outcomes of various menstrual-management methods, including method choice, continuation, bleeding patterns, amenorrhea rates, effect on moods and dysphoria, and side effects, in transgender and gender-diverse adolescents. METHODS: This was a retrospective chart review of all patients seen in a multidisciplinary pediatric gender program from March 2015 to December 2020 who were assigned female at birth, had achieved menarche, and used a menstrual-management method during the study period. Data were abstracted on patient demographics and menstrual-management method continuation, bleeding patterns, side effects, and satisfaction at 3 months (T1) and 1 year (T2). Outcomes were compared between method subgroups. RESULTS: Among the 101 included patients, 90% chose either oral norethindrone acetate or a 52-mg levonorgestrel (LNG) intrauterine device (IUD). There were no differences in continuation rates for these methods at either follow-up time. Almost all patients had improved bleeding at T2 (96% for norethindrone acetate and 100% for IUD users), with no difference between subgroups. Amenorrhea rates were 84% for norethindrone acetate and 67% for IUD at T1 and 97% and 89%, respectively, at T2, with no differences at either point. The majority of patients had improved pain, menstrually related moods, and menstrually related dysphoria at both follow-up points. There were no differences in side effects between subgroups. There were no differences in method satisfaction between the groups at T2. CONCLUSION: Most patients chose norethindrone acetate or an LNG IUD for menstrual management. Continuation, amenorrhea, and improved bleeding, pain, and menstrually related moods and dysphoria were high for all patients, indicating that menstrual management is a viable intervention for gender-diverse patients who experience increased dysphoria related to menses.

Menstrual Management Choices in Transgender and Gender Diverse Adolescents.

PURPOSE: Transgender and gender diverse patients who are assigned female at birth may request menstrual management to alleviate an increased dysphoria due to menses. The objective of this study is to describe the initiation and use over time of menstrual management methods (MMMs) in transgender and gender diverse adolescents. METHODS: A retrospective chart review was conducted of patients in a multidisciplinary pediatric gender program from March 2015 to December 2020 who were assigned female at birth, identified as transgender or gender diverse, and had achieved menarche. A descriptive statistical analysis was performed. RESULTS: Of 133 patients, 119 (90%) identified as transgender male, 11 (8%) as gender nonbinary, and 3 (2%) as another gender identity. Mean age was 15 (standard deviation 1.6) years. Only 12 (9%) patients had ever been sexually active. During the study period, 48 (36%) used gender-affirming testosterone. At the initial visit, 114 (86%) patients were not using an MMM. Of 80 patients who initiated a new MMM, 3 (4%) chose continuous oral contraceptive pills, 65 (83%) used norethindrone acetate (NETA), and 9 (11%) planned levonorgestrel intrauterine device (IUD) insertion. At 1 year, 56 patients were using NETA and 20 had an IUD in place. DISCUSSION: This study provides data on MMM choice in transgender and gender diverse adolescents using these methods almost exclusively for menstrual management and not contraception. Although few patients were using an MMM at baseline, most opted to start a method when given the opportunity. The most common methods were NETA or an levonorgestrel IUD.

Family-centered communication in pediatric sickle cell disease.

BACKGROUND: Individuals with sickle cell disease (SCD) experience systemic barriers in accessing high-quality care. Research suggests that patient/family-provider relationships are an important indicator of healthcare quality and can influence disease self-management and outcomes. The Patient Centered Communication (PCC) framework holds that patient/family-centered communication (e.g., eliciting, understanding, and validating patients' perspectives within their unique psychosocial contexts) contributes to improved family-provider relationships, as well as self-efficacy for disease management, adherence, and health outcomes. While the PCC framework has been useful in guiding the evaluation of patient/family-provider communication in other pediatric populations, it has not yet been applied in the context of pediatric SCD. This study aimed to use this framework to examine patient and family perceptions of communication with pediatric SCD healthcare providers. PROCEDURE: Total 17 caregivers (82% mothers, 94% Black/African American) and eight patients (62% female, aged 13-19 years, M = 16.50) completed semi-structured interviews. The PCC framework informed the development of a preliminary codebook. Thematic content analysis summarized family perspectives regarding communication with providers. RESULTS: For youth with SCD and their caregivers, specific themes related to family-centered communication included: reducing patient/family distress, supporting disease self-management efforts, facilitating information exchange and decision-making, and fostering positive and trusting relationships with providers. CONCLUSIONS: This study helps to address gaps in the literature related to patient/family-provider communication within pediatric SCD. Results underscore the importance of patient- and family-centered communication across pediatric SCD care. These findings can inform future research and clinical care initiatives to improve patient/family-provider interactions and health outcomes for this underserved population.

Experiences with Menses in Transgender and Gender Nonbinary Adolescents.

STUDY OBJECTIVE: To describe menstrual history, associated dysphoria, and desire for menstrual management in transgender male and gender diverse adolescents who were assigned female at birth DESIGN: Retrospective chart review SETTING: Tertiary care children's hospital PARTICIPANTS: All patients seen in a multidisciplinary pediatric gender program from March 2015 through December 2020 who were assigned female at birth, identified as transgender male or gender nonbinary, and had achieved menarche INTERVENTION: None MAIN OUTCOME MEASURES: Patient demographics, menstrual history, interest in and prior experiences with menstrual management, parental support, and concerns about menstrual management RESULTS: Of the 129 included patients, 116 (90%) identified as transgender male and 13 (10%) as gender nonbinary, with an average age of 15 (SD 1.6) years. Almost all (93%) patients reported menstrual-related dysphoria. Most (88%) were interested in menstrual suppression. The most common reasons for desiring suppression were achievement of amenorrhea (97%) and improvement of menstrual-related dysphoria (63%). CONCLUSIONS: Most gender diverse patients assigned female at birth reported dysphoria associated with menses and desired menstrual suppression. This information can encourage physicians to raise this topic and offer menstrual management for gender diverse patients who experience distress related to menses, especially for those who are not ready for or do not desire gender-affirming hormonal treatment. Future research is needed to better understand patients' experiences with menses and to determine the optimal menstrual management methods. This could be an important intervention to improve outcomes for this vulnerable population.

Feasibility of a trauma-informed parent-teacher cooperative training program for Syrian refugee children with autism.

Although the number of Syrians affected by the civil war rises, little work has been done to address the needs of Syrian refugee children with autism spectrum disorder. This research aimed to test the feasibility and acceptability of a culturally sensitive intervention developed specifically for children with autism spectrum disorder affected by trauma. Local partners advised the program team on cultural norms. Nine parents and 11 teachers were recruited to participate in 12-week parent-teacher cooperatives in a large Turkish city. We used qualitative methods to analyze interviews with each participant after intervention completion. A total of 14 participants completed the program (70%). All interviewees were women and Syrian refugees. Local political events, transportation costs and safety, and illnesses impacted attrition and attendance. All participants were satisfied with program content, including gains in autism knowledge, behavior management skills, and social support. Program-related challenges included applying skills to nonverbal children. The majority of participants made recommendations for program improvement, including a need for services outside urban areas. Flexible program delivery methods, including online options, might better accommodate participants unable to travel due to distance, political unrest, or safety. More research is needed to rigorously test program outcomes and to evaluate efforts to train local program leaders.