Gillette Children's Specialty Healthcare is a non-profit hospital located in St. Paul, Minnesota, USA. Through its 60-bed (staffed and licensed) hospital, 11 clinics, and nine greater Minnesota locations, Gillette treats more than 25,000 children each year who have complex conditions, rare disorders and traumatic injuries that affect the musculoskeletal and neurological systems.
ABSTRACT Objective Trofinetide is the first approved treatment for Rett syndrome (RTT) in the United States and Canada. Trofinetide improved the core symptoms of RTT in clinical trials, and real‐world evidence supports the findings of clinical trials. As RTT experts in the United States have now gained nearly 3 years of real‐world experience with trofinetide, a modified Delphi process was conducted in the United States to establish consensus recommendations for the practical use of trofinetide in the treatment of RTT. Methods A multidisciplinary steering group of five trofinetide‐experienced RTT experts practicing at an International Rett Syndrome Foundation (IRSF)‐designated center of excellence convened and developed 72 consensus statements across six domains: first‐line use, pre‐treatment assessment, initiation, benefit evaluation, tolerability management, and discontinuation strategies. The statements were then assigned a 4‐point Likert scale for testing via email by the Delphi panel identified by the steering group. Consensus was reached for individual statements if they met the pre‐specified consensus threshold of ≥ 75% agreement. Results Two rounds of assessments were completed by 25 respondents in each round, resulting in agreement ≥ 75% being reached across the final statement set. The consensus supports trofinetide as the standard of care for eligible patients with early initiation and an individualized approach to titration to maximize efficacy and tolerability. The panel reflected the steering group's view that trofinetide is efficacious. While side effects can emerge with trofinetide, they are manageable in most cases. Conclusions Recommendations highlight a flexible, patient‐centered approach to trofinetide use to optimize efficacy while addressing tolerability challenges and supporting adherence.
BACKGROUND:Stable osteochondritis dissecans (OCD) lesions are primarily treated nonoperatively. Nonoperative treatment includes several recommendations, including the use of an unloader brace. However, little is known about the clinical effectiveness of unloader bracing and its influence on OCD healing, return to sports, and patient-reported outcomes (PROs). The purpose of this investigation was to evaluate the impact of unloader bracing on patients with stable medial femoral condyle (MFC) OCD lesions treated nonoperatively. PURPOSE:To investigate the influence of valgus unloader bracing on the success of treating stable MFC OCD with nonoperative treatment strategies. STUDY DESIGN:Cohort study; Level of evidence, 2. METHODS:The authors used a prospective cohort of patients receiving nonoperative care for stable MFC knee OCD lesions. The primary variable of interest was the efficacy of bracing. The primary outcomes were transition from nonoperative care to surgical intervention, return-to-play (RTP) clearance, and PROs. Bivariate testing was used to determine if there were differences between groups. Chi-square testing was used to determine if there were differences in rates of transition to surgery by group. The timing of the transition by each group was assessed using a Wilcoxon rank-sum test, given the nonparametric nature of the data. Kaplan-Meier survival analysis was used to calculate time at risk, incidence rate, and survival times for each group. RESULTS:A total of 185 knees (bracing group: 123 [66.5%] knees; no-bracing group: 62 [33.5%] knees) were analyzed. Overall, 142 (76.8%) knees were successfully treated nonoperatively. Transition to surgery occurred in 43 (23.2%) knees. There were no differences in transition to surgery by groups, with no differences seen by group. Of the knees that did not transition to surgical care, 65 (71.4%) in the bracing group and 38 (74.5%) in the no-bracing group received RTP clearance (P = .693). PRO scores were similar between the 2 groups at the time of RTP clearance. CONCLUSION:The inclusion of an unloader brace in the nonoperative treatment for stable MFC OCD lesions did not decrease the likelihood of patients undergoing surgical intervention or improve their PRO scores at the time of RTP clearance. REGISTRATION:NCT02771496 (ClinicalTrials.gov identifier).
Background:The sphericity deviation score (SDS) is a continuous parameter to describe femoral head shape in patients with Perthes disease. The aims of this study were to evaluate the reproducibility of SDS and its association with the 3-group Stulberg classification. Methods:Anteroposterior and frog-leg lateral radiographs of 41 healed Perthes hips were assessed. Six observers with varying levels of experience, independently performed SDS measurements on two occasions, 2 weeks apart. Associations between SDS and the modified 3-group Stulberg classification were tested using ANOVA or Kruskal-Wallis tests and Spearman's rank correlation coefficient (rho). Intra- and interobserver agreement was assessed with Bland-Altman 95% limits of agreement (LoA), and reliability was analyzed by intraclass correlation coefficients (ICC). Results:The median SDS values in hips with a round femoral head were significantly higher when measured by inexperienced observers than those measured by experienced observers (30 vs 17; P < 0.001). The SDS values did not significantly correlate with the Stulberg classification among inexperienced observers. Among experienced observers, a strong correlation was found (Spearman's rho 0.670; P < 0.001); however, only 2 of 3 observers were able to distinguish between round and oval hips. Interobserver agreement for SDS was unacceptable (LoA >40) for inexperienced observers and marginal (LoA 20-40) for experienced observers. Interobserver reliability was poor for inexperienced observers (ICC 0.15; P = .02) and moderate for experienced observers (ICC 0.69; P < .001). Conclusions:SDS is a complex parameter that did not consistently differentiate between femoral head shapes when used by inexperienced observers. Even among experienced pediatric orthopaedic researchers, SDS showed marginal interobserver agreement. The method should be used only by experienced observers in scientific studies. Key Concepts:(1)The Sphericity Deviation Score (SDS) correlates significantly with the Stulberg classification only when measured by experienced pediatric orthopaedic researchers.(2)SDS is a complex and time-consuming parameter that often failed to distinguish clearly between adjacent Stulberg categories (e.g., round vs. ovoid hips).(3)Among inexperienced observers, the limits of agreement (LoA) of SDS measurements were unacceptable and the reliability (ICC) was poor.(4)SDS should be used only by experienced observers in scientific studies. Even among experienced researchers, interobserver LoA was marginal, indicating that further methodological refinements are desirable. Level of Evidence:III, retrospective cohort study.
