Understanding the reasons for delayed referral for intrathecal baclofen therapy in pediatric patients with severe spasticity.

OBJECTIVE: Intrathecal baclofen therapy (ITB) has been used in the treatment of spasticity and dystonia. In our pediatric movement disorder clinic, we noted a delay in referral of patients for consideration of ITB. Often, only after years of failed medical therapy, a baclofen pump is considered. This study attempts to investigate the prevalence, length and causes of the delay. METHODS: A retrospective, outcome analysis was performed. We conducted a survey of 30 pediatric patients who received baclofen pumps between the ages of 5 and 23. Patients were divided into two groups (before and after ITB approval by the US Food and Drug Administration in 1996) (FDA/Center for Drug Evaluation and Research, 2014; Ridley and Rawlins, J Neurosci Nurs 38:72-82, 2006; Medical Advisory Secretariat, Ont Health Technol Assess Ser 5:1-93, 2005). Information was collected regarding their onset of spasticity, attempted treatments, pump referral, satisfaction, and resulting change in the quality of life. RESULTS: There was a delay in referral in most cases investigated. Average time to baclofen pump implantation, after initial onset of spasticity, was 5.14 years (group A) and 11.7 years (group B). Out of the subjects who reported diminished effects or no effect of pharmacological treatment, 93 % of these respondents reported that ITB had a dramatic long-lasting effect on their spasticity. Of 30 patients, 28 reported effectiveness of ITB, and 26 of 30 subjects reported an improved quality and ease of life. CONCLUSION: Despite the limitations of this subjective retrospective analysis of outcomes and delay in referral, the opinions of the parents and caregivers should be considered. Earlier referral for ITB therapy may better treat severe spasticity in pediatric patients.

Topiramate for Weight Management in Children With Severe Obesity.

Background: Topiramate has been shown to result in significant weight loss compared to placebo in adults with obesity. However, there are no consensus guidelines on the acceptability, safety, and efficacy of topiramate for weight loss in children. We present a literature review and case series on topiramate use in young children with severe obesity. Methods: We performed a PubMed search from January 2000 to February 2022 utilizing keywords, "topiramate" and "obesity" and "children" and "adolescent." For our case series, children were identified through retrospective chart review from a multidisciplinary weight management program. Eligibility criteria: age ≤12 years, class II or III obesity, completed 16 weeks of topiramate therapy as adjunct to lifestyle modifications. Semistructured interviews were conducted with one parent to review side effects. Results: Literature search yielded nine articles. All studies reported trends toward BMI reduction and weight loss with topiramate monotherapy. Five children met case series eligibility (mean age 10 years 3 months ±1.5 years, 60% female). After 16 weeks of topiramate, all children had a decrease in BMI as a percentage of the 95th percentile (mean -12% [-5% to -18%]). Parents reported improvement in impulsive eating and decreased desire to overeat compared to baseline. Four out of five reported no side effects, one reported drowsiness which resolved by dosing at nighttime. Conclusions: Results suggest that topiramate is well tolerated and may be utilized for weight management in younger children. A randomized controlled trial investigating the impact of topiramate for weight management in this age group is warranted.

Glucagon-like Peptide-1 Receptor Agonists for the Treatment of Type 2 Diabetes in Youth.

Glucagon-like peptide-1 receptor agonists (GLP-1RAs) have gained traction for the management of type 2 diabetes and obesity. Unlike several classes of antidiabetic medications that contribute to weight gain, GLP-1RAs not only reduce haemoglobin A1c, but also promote weight loss. While there is a large body of evidence supporting its safety and efficacy in adults, paediatric clinical trial data have only emerged in recent years. This review will discuss the limited treatment options for paediatric type 2 diabetes and the mechanism of action of GLP-1RAs as it pertains to physiological pathways relevant for type 2 diabetes, obesity and their related comorbidities. The outcomes of paediatric trials evaluating liraglutide, exenatide, semaglutide and dulaglutide in paediatric type 2 diabetes and obesity will be closely examined, including differences compared with adult studies. Finally, potential barriers and strategies to expanding GLP-1RA access in adolescents will be discussed. Future studies are needed to determine if the cardio-and renal-protective benefits of GLP-1RAs apply to youth-onset type 2 diabetes.

Stoned-A Syndrome of D-Lactic Acidosis and Urolithiasis.

Short bowel syndrome (SBS) can lead to many complications related to the condition and its therapy. We describe 2 children with SBS who we believe are the second and third patients documented to have experienced both D-lactic acidosis and urolithiasis. We review aspects of these SBS complications and recent findings on the microbiome of patients with SBS that may predispose to these complications.

Machine Learning-Augmented Propensity Score-Adjusted Multilevel Mixed Effects Panel Analysis of Hands-On Cooking and Nutrition Education versus Traditional Curriculum for Medical Students as Preventive Cardiology: Multisite Cohort Study of 3,248 Trainees over 5 Years.

BACKGROUND: Cardiovascular disease (CVD) annually claims more lives and costs more dollars than any other disease globally amid widening health disparities, despite the known significant reductions in this burden by low cost dietary changes. The world's first medical school-based teaching kitchen therefore launched CHOP-Medical Students as the largest known multisite cohort study of hands-on cooking and nutrition education versus traditional curriculum for medical students. METHODS: This analysis provides a novel integration of artificial intelligence-based machine learning (ML) with causal inference statistics. 43 ML automated algorithms were tested, with the top performer compared to triply robust propensity score-adjusted multilevel mixed effects regression panel analysis of longitudinal data. Inverse-variance weighted fixed effects meta-analysis pooled the individual estimates for competencies. RESULTS: 3,248 unique medical trainees met study criteria from 20 medical schools nationally from August 1, 2012, to June 26, 2017, generating 4,026 completed validated surveys. ML analysis produced similar results to the causal inference statistics based on root mean squared error and accuracy. Hands-on cooking and nutrition education compared to traditional medical school curriculum significantly improved student competencies (OR 2.14, 95% CI 2.00-2.28, p < 0.001) and MedDiet adherence (OR 1.40, 95% CI 1.07-1.84, p = 0.015), while reducing trainees' soft drink consumption (OR 0.56, 95% CI 0.37-0.85, p = 0.007). Overall improved competencies were demonstrated from the initial study site through the scale-up of the intervention to 10 sites nationally (p < 0.001). DISCUSSION: This study provides the first machine learning-augmented causal inference analysis of a multisite cohort showing hands-on cooking and nutrition education for medical trainees improves their competencies counseling patients on nutrition, while improving students' own diets. This study suggests that the public health and medical sectors can unite population health management and precision medicine for a sustainable model of next-generation health systems providing effective, equitable, accessible care beginning with reversing the CVD epidemic.

Selective dorsal rhizotomy for spastic diplegia secondary to stroke in an adult patient.

BACKGROUND: Selective dorsal rhizotomy (SDR) is often recommended for children with spastic paraparesis and cerebral palsy. SDR reduces spasticity in the lower extremities for these children with spastic paraplegia. However, SDR is infrequently recommended for adults with spasticity. Spastic diplegia in adult patients can be due to stroke, brain or spinal cord injury from trauma, infection, toxic-metabolic disorders, and other causes. Although rarely considered, SDR is an option for adult patients with spastic diplegia as well. CASE DESCRIPTION: The authors describe a patient who underwent a SDR with a successful postoperative outcome. This man suffered a hypertensive and hemorrhagic stroke secondary to intravenous drug abuse at age 46. A SDR was performed after two failed intrathecal baclofen pump placements due to recurrent infections, likely resulting from his immunocompromised status. The patient underwent lumbar laminectomies and dorsal rhizotomies at levels L1-S1 bilaterally. Postoperatively, the patient's spasticity was significantly reduced. His Ashworth spasticity score decreased from 4/5 to 1/5, and the reduction in tone has been durable over 3 years. CONCLUSION: SDR in older patients with spastic paraparesis may be considered as a treatment option.