Clinical Practice Patterns for Discussing Hydroxyurea Initiation With Families of Children With Sickle Cell Disease

Pediatric Blood & Cancer · 2026-04-02

BACKGROUND: Despite robust evidence of safety and efficacy, hydroxyurea (HU) uptake remains low for children with sickle cell disease (SCD). Guidelines recommend use of shared decision-making for HU initiation, but limited resources exist to inform these conversations with families. This study examined practices among hematology providers when introducing HU to families of children with SCD. PROCEDURE: In Study 1, 11 pediatric SCD clinics within the United States completed process maps delineating their practice for discussing HU initiation with families. Process maps were compared to summarize processes. In Study 2, individual semi-structured interviews were conducted with nine healthcare providers from one clinic from Study 1. Thematic and descriptive content analysis were used to summarize provider perspectives on existing processes. RESULTS: Process maps highlighted the following common steps: (1) check in for appointment and obtain laboratory studies (n = 8); (2) medical provider discusses HU with family (n = 11); (3) family given HU materials (n = 8); and, (4) follow-up HU discussion at next visit (n = 6). Process variations were also identified. In Study 2, providers universally reported introducing HU shortly after diagnosis and strongly encouraging it for patients with disease complications. HU education was provided primarily via in-person conversation, written materials, and websites, focusing on its expected benefits, historical context, and side effects. CONCLUSIONS: We identified both similarities and variations in clinical practice for HU initiation in children with SCD. Results highlight potential opportunities to improve the ways in which HU initiation is discussed with families of children with SCD.

PTSD Symptom Trajectories in the Wake of Traumatic Events: a Systematic Review and Meta-Analysis of 20 Years of Research

2026-04-07

Posttraumatic Stress Disorder (PTSD) symptom trajectories considerably vary between individuals upon potentially traumatic events. In this systematic review, meta-analysis and meta-regression, we evaluated twenty years of literature on common variations in PTSD symptom trajectories. We specifically focused on longitudinal studies applying data-driven identification of latent trajectory subgroups based on symptom severity patterns over time Eligible studies applied statistical Growth Mixture Modeling to longitudinally assessed PTSD symptoms using validated continuous measures at minimally three time points across 12 weeks. Traditional systematic search methods, machine learning- and large language model–assisted approaches were combined to identify eligible articles published in any language until June 2025. To ensure coherence and consistency within subsequent data aggregation, we developed a standardized consensus-derived PTSD symptom trajectory classification framework. Pooled relative trajectory prevalence estimates were calculated using generalized linear mixed-effects models, and moderator analyses were conducted using meta-regression models. In total, we included 99 studies representing 113 independent samples and 215,136 participants from 21 countries. Five core PTSD symptom trajectories were identified. The pooled relative prevalences were 74.0% for the low symptoms trajectory, 31% for the moderate symptoms trajectory, 15.9% for the decreasing symptoms trajectory, 9.2% for the high symptoms trajectory, and 8.5% for the increasing symptoms trajectory. Several socio-demographic, trauma-related and methodological sample characteristics significantly moderated relative trajectory prevalences across samples. Our findings provide empirical support for and further refinements of leading theoretical conceptualizations of PTSD. Furthermore, the obtained relative prevalence estimates and their identified moderators may guide population-tailored mental health care following trauma exposure.

PTSD Symptom Trajectories in the Wake of Traumatic Events: a Systematic Review and Meta-Analysis of 20 Years of Research

OSF Preprints (OSF Preprints) · 2026-04-07

Posttraumatic Stress Disorder (PTSD) symptom trajectories considerably vary between individuals upon potentially traumatic events. In this systematic review, meta-analysis and meta-regression, we evaluated twenty years of literature on common variations in PTSD symptom trajectories. We specifically focused on longitudinal studies applying data-driven identification of latent trajectory subgroups based on symptom severity patterns over time Eligible studies applied statistical Growth Mixture Modeling to longitudinally assessed PTSD symptoms using validated continuous measures at minimally three time points across 12 weeks. Traditional systematic search methods, machine learning- and large language model–assisted approaches were combined to identify eligible articles published in any language until June 2025. To ensure coherence and consistency within subsequent data aggregation, we developed a standardized consensus-derived PTSD symptom trajectory classification framework. Pooled relative trajectory prevalence estimates were calculated using generalized linear mixed-effects models, and moderator analyses were conducted using meta-regression models. In total, we included 99 studies representing 113 independent samples and 215,136 participants from 21 countries. Five core PTSD symptom trajectories were identified. The pooled relative prevalences were 74.0% for the low symptoms trajectory, 31% for the moderate symptoms trajectory, 15.9% for the decreasing symptoms trajectory, 9.2% for the high symptoms trajectory, and 8.5% for the increasing symptoms trajectory. Several socio-demographic, trauma-related and methodological sample characteristics significantly moderated relative trajectory prevalences across samples. Our findings provide empirical support for and further refinements of leading theoretical conceptualizations of PTSD. Furthermore, the obtained relative prevalence estimates and their identified moderators may guide population-tailored mental health care following trauma exposure.

Natural History of Morquio A Syndrome

Journal of Inherited Metabolic Disease · 2026-05-01

Mucopolysaccharidosis IVA (Morquio A) is a progressive lysosomal disorder caused by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase, resulting in the accumulation of glycosaminoglycans, primarily in cartilage and bone. The condition leads to skeletal dysplasia, impaired ossification, and growth imbalance, causing cervical compression, tracheal obstruction, hip dysplasia, and cardiopulmonary complications. Although existing natural history studies have clarified several aspects of disease progression, objective methods to quantify skeletal pathology and its functional impact remain limited. Standard endurance-based assessments, including the 6-min walk test, 3-min stair climb, and spirometry, are variable and infeasible for young children, wheelchair-dependent individuals, or patients with respiratory or postoperative limitations. Their dependence on motivation, training, and site-specific differences restricts their value as reliable endpoints. Significant gaps persist in the objective evaluation of skeletal dysplasia progression and its functional impact. There remains an unmet need for non-invasive, quantitative tools that capture multisystem involvement across the full phenotypic spectrum. Objective tools exist as we reported but have not been widely implemented or standardized, and data supporting their use as regulatory endpoints remain limited. Emerging longitudinal, multidisciplinary evaluations and surrogate biochemical biomarkers show potential to define disease severity, support prognosis, and serve as validated endpoints for therapeutic monitoring and surgical risk stratification. In conclusion, to address the limitations of prior studies, we propose a novel natural history program of innovative, non-invasive assessments based on our compiled data. Such approaches may contribute to more consistent longitudinal data and help inform the development of clinical endpoints for emerging therapies.

Challenges, Preparations, and Recommendations: Family Perspectives on Pediatric Cardiac Surgery Across Childhood and Adolescence

Journal of Family Nursing · 2026-04-29

Congenital heart disease (CHD), the most common birth defect globally, often requires hospitalization for cardiac surgery. While the stress of infant surgery is well-documented, less is known about the experiences of families with older children. We explored the experiences of families of children aged 3-17 hospitalized for cardiac surgery. Semi-structured interviews were conducted. Data were analyzed using qualitative content and thematic analysis. Thirty-two parents (30 mothers and 2 fathers) and 14 patients (10 males and 4 females) participated. Seven themes emerged across three domains: (a) Challenges: disruptions in family and social support, unexpected events and inadequate communication, and uncertainty and strong emotions; (b) Preparations: emotionally preparing yourself, your children and your family, and planning for hospitalization and surgical recovery; and (c) Recommendations: enhance communication and connection for support, and provide resources and education for empowerment. Findings highlight opportunities for targeted interventions to support families across child development.

Small but mighty: how can pediatric psychologists harness the power of digital single-session interventions?

Journal of Pediatric Psychology · 2026-04-09

OBJECTIVE: Single-session interventions (SSIs) are an innovative, scalable approach for addressing the unmet behavioral and mental health needs of pediatric patients and their families. Specifically, digital SSIs, which are self-guided and delivered online, offer a low-cost and accessible approach but are currently underutilized in pediatric psychology. METHODS: This topical review aims to describe SSIs broadly and the specific need for digital SSIs, explore potential applications within pediatric psychology, outline future directions and considerations, and present two examples of community-engaged digital SSI design. RESULTS: SSIs evidence small to medium effects across a range of mental health concerns, including distress, anxiety, and depression, supporting their broad applicability. Incorporating community voices during the development process is essential to ensure SSIs are contextually relevant in pediatric populations. Two illustrative SSIs currently in development within pediatric psychology are described: (1) an intervention supporting adolescent siblings of children with cancer and (2) a program targeting depression in adolescents with type 1 diabetes. Both examples incorporate input from key community members, underscoring the value of participatory design in enhancing intervention relevance and impact. CONCLUSIONS: SSIs offer a brief and scalable strategy to help address gaps in psychological care, particularly for youth and families with limited access to services. Pediatric psychologists are well positioned to develop, evaluate, and implement SSIs across diverse populations and intervention targets. Ensuring the effectiveness, equity, and sustainability of SSIs will require ongoing collaboration with community partners and integration across pediatric care settings.

Clinical Practice Patterns for Discussing Hydroxyurea Initiation with Families of  Children with Sickle Cell Disease

Research Explorer (The University of Manchester) · 2026-03-04

<b>Background: </b>Despite robust evidence of safety and efficacy, hydroxyurea (HU) uptake remains low for children with sickle cell disease (SCD). Guidelines recommend use of shared decision-making for HU initiation, but limited resources exist to inform these conversations with families. This study examined practices among hematology providers when introducing HU to families of children with SCD. <br/><br/><b>Procedure:</b> In Study 1, 11 pediatric SCD clinics within the United States completed process maps delineating their practice for discussing HU initiation with families. Process maps were compared to summarize processes. In Study 2, individual semi-structured interviews were conducted with nine healthcare providers from one clinic from Study 1. Thematic and descriptive content analysis were used to summarize provider perspectives on existing processes. <br/><br/><b>Results:</b> Process maps highlighted the following common steps: 1) check in for appointment and labs (n=8); 2) medical provider discusses HU with family (n=11); 3) family given HU materials (n=8); and, 4) follow-up HU discussion at next visit (n=6). Process variations were also identified. In Study 2, providers universally reported introducing HU shortly after diagnosis and strongly encouraging it for patients with disease complications. HU education was provided primarily via in-person conversation, written materials, and websites, focusing on its expected benefits, historical context, and side effects. <br/><br/><div/><div><b>Conclusions:</b> We identified both similarities and variations in clinical practice for HU initiation 58 in children with SCD. Results highlight potential opportunities to improve the ways in which HU initiation is discussed with families of children with SCD.</div>

Exploring Preferences for a Digital Single‐Session Intervention for Adolescent Siblings of Youth With Cancer

Pediatric Blood & Cancer · 2026-03-13 · 1 citations

INTRODUCTION: Adolescent siblings of children with cancer are at elevated risk for psychosocial problems. Unfortunately, various barriers such as limited family time and resources, conflicting schedules, and psychosocial staffing constraints at cancer centers hinder sibling access to support. Digital, self-guided, single-session interventions (SSIs)-brief, low-cost, scalable mental health tools-have shown promise for surmounting such barriers and providing support to adolescents. This qualitative study aimed to gather perspectives from adolescent siblings, parents, and professionals to inform the development of a digital, self-guided Sibling SSI tailored to siblings' needs. METHODS: Semi-structured interviews were conducted with adolescent siblings (n = 22), parents (n = 21), and psychosocial providers and community leaders (n = 14). Drawing on existing evidence-based SSIs that use a solution-focused brief therapy framework, interviews explored content, language, timing, and delivery preferences for a Sibling SSI. Content analysis was used to summarize key recommendations. RESULTS: Participants viewed a digital Sibling SSI as a promising approach to promote coping. Content suggestions included acknowledging common cancer-related and adolescent struggles, highlighting siblings' existing coping strategies, and offering personalized action plans. Participants emphasized the importance of sibling-centric language and creating a space exclusively for siblings. Early dissemination within 3 months of diagnosis via psycho-oncology teams and community channels was recommended. DISCUSSION: Findings provide community-informed guidance to inform the development of a digital Sibling SSI aimed at surmounting barriers to care and enhancing psychosocial support for adolescent siblings of children with cancer.

PTSD Symptom Trajectories in the Wake of Traumatic Events: a Systematic Review and Meta-Analysis of 20 Years of Research

OSF Preprints (OSF Preprints) · 2026-01-29

All in the Family

The Journal of Cardiovascular Nursing · 2025-07-30

BACKGROUND: Congenital heart disease (CHD) can erode parental mental health and hamper adjustment in children with CHD; however, associations between family members are poorly understood. OBJECTIVE: The aim of this study was to test a model of associations of adjustment among family members of children with CHD. METHODS: Structural equation modeling was used to model 47 parent-sibling dyads. Parents completed measures of their mental health and patient adjustment (internalizing symptoms). Siblings self-reported symptoms of anxiety and depression. RESULTS: Parent mental health was associated with patient internalizing symptoms (P = .019) but not with depression (P = .793) or anxiety (P = .222) in siblings. Greater internalizing scores in patients were associated with more depression (P < .001) and anxiety (P = .042) in siblings. CONCLUSIONS: Our sample did not reveal expected associations between parental and sibling adjustment in families of children with CHD but did reveal that patient adjustment may play an important role. Family-centered care should include all members of the family and provide support as needed.