A semi-standardized music therapy intervention for girls with Rett syndrome: A descriptive essay

Rett syndrome (RTT) is a severe neurodevelopmental disorder resulting in a wide range of functional impairments and therefore greatly impacts the lives of both patients and their families. While genetic and medical aspects have been studied for several decades, rehabilitation intervention research is still in its infancy. In this study, the investigating researchers have presented a rehabilitative framework by using music therapy for girls with RTT. This model is founded upon the use of music therapy in light of Stern’s proposal of subjective experience and affect attunement; it also refers to Rosenbaum’s family-centered rehabilitation medicine perspective. This study both describes the theory behind this intervention and presents a newly developed outcome measure. This novel tool may have future clinical and research applications. Music therapy for patients with RTT has not been well researched yet, and as a result, is not universally recommended. However this study’s findings suggest that music therapy is an important component of multidisciplinary therapy. Further collaborative research should be encouraged in order to study and implement the use of music therapy in the treatment of severe disabilities. Projects such as the Enablin+program with the support from the European Commission constitute fundamental tools in promoting integrative medical research and international networks.

Pediatric spinal cord injury rehabilitation: A protocol for an international multicenter project (SINpedSCI)

PURPOSE: Children and adolescents (<18 years old) who sustain a spinal cord injury (SCI) should ideally be managed in specialized rehabilitation services. This project aims to describe the organization of pediatric SCI in ten rehabilitation units in seven countries and to qualitatively explore psychosocial aspects of adolescents living with SCI. METHODS: A multicenter cross-sectional project is planned, using quantitative (web survey) and qualitative (interview) methods in ten rehabilitation units from Norway, Sweden, United States, Israel, PR China, Russia and Palestine. Individual interviews will be conducted with ≥20 adolescents aged 13–17 years at least 6 months’ post rehabilitation. RESULTS: Units involved will be described and compared, according to funding, attachment to an acute SCI unit, catchment area, number of beds, admittance and discharge procedures, availability of services, staff/patient ratio, content and intensity of rehabilitation programs, length of stay, measurement methods, follow-up services, health promotion services, and pediatric SCI prevention acts. The semi-structured interview guide will include experiences from acute care and primary rehabilitation, daily life, school, contact with friends, leisure time activities, peers, physical and psychological health, and the adolescents’ plans for the future. CONCLUSION: Based on the present protocol, this project is likely to provide new insight and knowledge on pediatric SCI rehabilitation and increase the understanding of pediatric SCI in adolescents and their families internationally.

Safety and efficacy of repeat long-term incobotulinumtoxinA treatment for lower limb or combined upper/lower limb spasticity in children with cerebral palsy

PURPOSE: The open-label phase 3 ‘Treatment with IncobotulinumtoxinA in Movement Open-Label’ (TIMO) study investigated longer-term safety and efficacy of incobotulinumtoxin A in children/adolescents with cerebral palsy (CP). METHODS: Patients on standard treatment, with unilateral or bilateral lower limb (LL) or combined upper limb (UL)/LL spasticity received four incobotulinumtoxinA injection cycles (16 or 20 Units/kg bodyweight total [maximum 400 or 500 Units] per cycle depending on ambulatory status/clinical pattern treated), each followed by 12–16 weeks’ observation. Treatment for pes equinus was mandatory; flexed knee or adducted thigh were options for unilateral treatment and/or ULs for unilateral/bilateral treatment. The primary endpoint was safety; changes in Ashworth Scale and Gross Motor Function Measure-66 scores, and Global Impression of Change Scale scores at week 4 of each injection cycle were also evaluated. RESULTS: IncobotulinumtoxinA (≤500 Units for ≤98 weeks) was safe, well-tolerated, and effective across all endpoints for multipattern treatment of LL and combined LL/UL spasticity in ambulant/nonambulant children/adolescents with CP. Treatment effects increased with each injection cycle. No new/unexpected safety concerns were identified. CONCLUSION: IncobotulinumtoxinA showed a good safety and tolerability profile, with efficacy over multiple clinical presentations. As an adjunct treatment, it offers an effective, individualized treatment option for pediatric CP-related spasticity.

Primary care providers need education and resources to provide optimal care for children and adults with spina bifida

PURPOSE: The objective of this study was to describe the knowledge, attitudes, and practices related to caring for youth with SB within a statewide pediatric primary care medical home network. METHODS: Health care professionals who were members of the PA MHP statewide network were surveyed on sociological demographics, confidence in and knowledge of topics that impact the SB population, education on SB, designating responsibility of care, tasks completed in the office, and transition from pediatric to adult care from October 2016 through November 2016. RESULTS: Among 67 respondents, 78%believed counseling families/caregivers about the impact of SB on everyday life was a shared responsibility between PCPs and SB clinics. Eighty five percent of participants indicated they had exposure to SB through clinical courses or didactics in training but only 54%of participants felt knowledgeable on the impact of SB on everyday life. CONCLUSION: Findings suggest a need for increased educational efforts, improved awareness of SB, and access to evidence-based guidelines for PCPs to promote accessible and coordinated care to individuals with SB.

Association between race and in-hospital outcomes in children with spina bifida following inpatient urologic surgery

PURPOSE: Post-operative complication rates may vary among racial and/or ethnic groups and have not been previously described in individuals with spina bifida (SB) undergoing urologic surgery. The aim of this study was to compare in-hospital complication frequencies of individuals with SB following urologic surgery by race/ethnicity. METHODS: The Nationwide Inpatient Sample was used to identify pediatric patients with SB who underwent inpatient urologic procedures. A pediatric cohort (<18 years old) with SB that underwent urologic surgery were assessed. All analyses report weighted descriptive statistics, outcomes, and race/ethnicity was the primary predictor variable. The primary outcome of interest was post-operative complications which were defined using NSQIP ICD-9 code definitions. Secondary analysis included length of stay (LOS), and encounter cost was estimated using the cost-to-charge ratio files provided by the Healthcare Cost and Utilization Project. RESULTS: The unadjusted model showed no differences in complications, LOS, and cost. In the adjusted model there were no differences in complications, LOS, and cost between Black and White encounters. However, Hispanic ethnicity was associated with a 20%(95%CI: 4–40%) increase in LOS and 18%(95%CI: 2–35%, p = 0.02) increase in cost compared to White encounters. CONCLUSION: There was no evidence of variation for in-hospital complication rates among racial/ethnic groups undergoing urologic surgery. Hispanic ethnicity was associated with higher costs and longer LOS in pediatric SB encounters.

Disease and medication burden of medically complex adolescents seen in an interdisciplinary transitional care clinic

PURPOSE: Transitional care for adolescents with complex diseases, who are entering adulthood, is challenging. The purpose of this study is to quantify the disease and medication burden of this population, who are transitioning though an interdisciplinary specialty clinic. METHODS: This study is a retrospective observational study of all patients seen in a transitional care clinic between July 2012 and March 2015. The main outcomes assessed included disease state and medication burden. Descriptive statistics, along with the paired t-test and McNemar’s test, were used. RESULTS: The study cohort included 216 patients. The average patient age was 20.7 years, and the median number of clinic encounters was 6. Patients had at least 1 of 8 primary diagnoses. On average, patients took medications from 5 classes and used 3 dose forms. Among 163 patients who had medication reconciliation performed, the average number of medication classes increased by 0.44±1.51 (p = 0.003). There was an average increase of 3.70%(SD±36.31%; p = 0.27) in the number of required medication lab assessments ordered for patients who had medication reconciliation performed. CONCLUSION: There is a high disease and medication burden among adolescent patients with complex disease states who are to transition to adult care.

Spina bifida care, education, and research: A multidisciplinary community in a global context

Worldwide neural tube defects, such as encephalocele and spina bifida (SB), remain a substantial cause of the global burden of disease; and in the US, Latinos consistently have a higher birth prevalence of SB compared with other ethnic groups. From limited access and fragmented care, to scarcely available adult services, many are the challenges that besiege those living with SB. Thus, to provide inclusion and active involvement of parents of children and adults with SB from all communities, innovative approaches will be required, such as community-based participatory research and culturally competent learning collaboratives. Promisingly, the Spina Bifida Community-Centered Research Agenda was developed by the community of people living with SB through the Spina Bifida Association (SBA). Additionally, the SBA will host the Fourth World Congress on Spina Bifida Research and Care in March of 2023. Just as the SBA is clearly committed to this population, the Journal of Pediatric Rehabilitation Medicine will continue to serve as a catalyst for SB care, education, and research across the SB population in a global context.

Who is managing the bowels? A survey of clinical practice patterns in spina bifida clinics

PURPOSE: Neurogenic bowel dysfunction (NBD) affects 80% of individuals with spina bifida. Performing and disseminating research on NBD to reach the appropriate audience is difficult given the variability among medical specialties managing NBD. This study aimed to identify which medical specialties and types of providers are currently managing NBD in the United States. METHODS: A survey was developed and sent to 75 spina bifida clinics. Surveys queried which specialty was primarily responsible for medical and surgical management of NBD and any others that assist in NBD care. The license and certification level of the providers were collected. Descriptive statistics were performed to describe the results. RESULTS: Response rate was 68%. Urology was the leading specialty primarily responsible for NBD management (39%) followed by rehabilitation medicine and developmental pediatrics (22% and 20%, respectively). Physicians were the primary providers of care followed by nurse practitioners (54% vs 31%). Urology performs 65% of NBD surgeries. CONCLUSION: Multiple specialties and providers are involved in NBD management with variation among clinics. Development of improved NBD care should include a spectrum of specialties and providers. Dissemination of research should be aimed at multiple specialty groups.