Journal of Pediatric Rehabilitation Medicine - Volume 9, issue 1
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Journal of Pediatric Rehabilitation Medicine: An Interdisciplinary Approach is an international journal designed to parallel the multi-disciplinary team approach of caring for a child with an acute or chronic disease. The issues will primarily be themed and broad in scope including, but not be limited to cerebral palsy, traumatic brain injury, spinal cord injury, spina-bifida, amputation, muscular dystrophies of all types, stroke, cancer, mental retardation, developmental delays, and others.
The aim is to include a wide range of experts who care for children with the above diagnosis. Authorship by two different disciplines is requested though not required to encourage an interdisciplinary and collaborative approach. The goal is for the reader to obtain not just the medical perspective, but also nursing, occupational, physical and speech therapy, psychology, home care, etc., in providing the most comprehensive care for children. Manuscripts will be blinded and peer reviewed with appropriate feedback. Statistical analysis will be reviewed by the biostatistician. Readers are encouraged to submit and or suggest case reviews, commentaries, editorials, original research, conference schedules, or reviews.
Abstract: PURPOSE: We investigated the prognostic utility of onset age at first signs and symptoms (SS) to predict onset age at loss of ambulation (LOA) for childhood-onset Duchenne and Becker Muscular Dystrophies (DBMD). METHODS: Our cohort comprised male cases with DBMD ascertained by the population-based Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet ). Adjusted hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated using Cox proportional hazards models for associations between onset ages of first SS and LOA. Covariates controlled for were corticosteroid use, family history of DBMD, birth year, race/ethnicity, and MD STARnet…site. Onset age at first SS was considered as a continuous and as a categorical variable. RESULTS: A one-year increase in onset age at first SS was significantly associated with a 10% reduction in annual risk of LOA (HR = 0.90, CI = 0.87-0.94). Treating onset age at first SS as a categorical variable yielded a similar association (≥ 5 years: referent; ≥ 3 to < 5 years: HR = 1.36, CI = 1.02-1.81; 18 months to < 3 years: HR = 1.72, CI = 1.31-2.26; < 18 months: HR = 1.52, CI = 1.14-2.02). CONCLUSIONS: Earlier onset age at first SS is associated with earlier onset age at LOA and may have clinical utility in differentiating childhood-onset Duchenne and Becker muscular dystrophies.
Abstract: OBJECTIVE: To assess the effect of nutritional interventions on growth and on respiratory status in patients with congenital myopathy (CM), congenital muscular dystrophy (CMD), and congenital myasthenic syndrome (CMS). METHODS: Retrospective cohort study based on case-note review of 18 patients affected by CM, CMD, and CMS, followed at a single pediatric neuromuscular center, between 2006 and 2014. RESULTS: Seventy-two percent of patients required placement of a gastrostomy tube for bulbar weakness or for growth failure. Of those patients, 10 had 1 year follow up anthropometric data and 6 had 2 year follow up…anthropometric data. Height percentiles and z-scores were significantly improved in patients after 1 year, while weight and BMI percentiles and z-scores were not. Weight and height percentiles and z-scores were significantly improved in patients at 2 year follow up, while BMI percentiles and z-scores were not. The number of respiratory illnesses was not significantly different before or after placement of the feeding tube. Of the patients who did not have placement of a gastrostomy tube, 4 had 1 year follow up anthropometric data and 3 had 2 year follow up anthropometric data. Gastrostomy tube fed patients had significantly higher mean weight percentiles and z-scores compared to orally fed patients. There was no significant difference in height or BMI between the gastrostomy fed and orally fed groups. Individual growth curves highlight the effect of intervention on weight and height. CONCLUSIONS: This is a single multidisciplinary center experience describing the effect of nutritional interventions on growth in patients with congenital neuromuscular disorders. While the number of patients and their data in this report are limited, it highlights that the growth in this group of patients is unique but that the low weight and short stature respond to nutritional interventions with changes typically seen after 2 years of intervention.
Abstract: Scoliosis occurs in nearly all non-ambulatory children with spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Non-operative treatments have not been shown to be effective at preventing progression of scoliosis. Progressive scoliosis can impact the ability of patients to sit comfortably, be cosmetically unappealing, and in severe cases exacerbate pulmonary disease. The main goal of operative treatment is to improve sitting balance and prevent progression of scoliosis. Complication rates are high and there is little data on effect of operative treatment on quality of life in children with SMA and DMD. Comprehensive multi-disciplinary pre-operative…evaluations are vital to reduce the risks of operative treatment.
Keywords: Muscular dystrophy, spinal muscular atrophy, scoliosis, surgery
Abstract: Charcot Marie Tooth (CMT) disease is the most common hereditary sensorimotor neuropathy that has a slow onset. It presents usually in childhood, starting distally and from the lower limbs progressing to more proximal muscles. Due to the lack of curative medical treatments and the problematic outcomes of surgical intervention, rehabilitation continues to play a major role in treatment. This paper aims to summarize the rehabilitation approaches like aerobic, stretching and strengthening exercises. Orthotics is another important part of treatment that complete rehabilitative approaches. Orthotic devices that are currently being used and investigated in patients with CMT are also reviewed.…The evidence shows that exercise is effective in improving strength and general fitness. Stretching is somewhat effective in maintaining range of motion. Orthotic devices are the mainstay of maintaining mobility and ambulation and upper extremity function.
Abstract: PURPOSE: The consensus statement for standard of care in SMA recommends multidisciplinary medical care including physical therapy (PT) services. To date there are no reports regarding the implementation of these recommendations and the type of care or services received by individuals with SMA. The purpose of this study is to describe the PT services received by individuals with SMA. METHODS: Interviews were conducted with patients or their caregivers at the Pediatric Neuromuscular Clinical Research (PNCR) Network sites from October 2011 to September 2012. Questions included information about clinical status of the patient, sociodemographic profile of the…patient or caregiver, and PT services received in the past year, including the setting, frequency, duration and type of PT, and therapies administered by caregivers. RESULTS: Eighty-six percent of 105 participants reported receiving PT services, some in multiple settings: 62% in the neuromuscular clinic, 38% at school, 34% at home, and 13% in an outpatient clinic. Greater frequency of PT services received was associated with younger age and inability to walk, but not SMA type. CONCLUSION: This is the first multicenter study documenting PT services received by patients with SMA. Further research is needed to better understand the impact of PT services on the natural history of SMA.
Keywords: Spinal muscular atrophy, physical therapy, rehabilitation, standard of care
Abstract: PURPOSE: To document use of rehabilitative technology among individuals with Duchenne/Becker muscular dystrophy (DBMD) among sites of the Muscular Dystrophy Surveillance, Tracking, and Research network (MD STARnet ). METHODS: Data from 362 caregivers who participated in the MD STARnet caregiver interview between April 2006 and March 2012 (54.7% response rate) were analyzed to assess the type, frequency and duration of use of assistive technology. RESULTS: Caregiver reports of technology use by individuals with DBMD across five MD STARnet sites in the US demonstrated significant regional differences in the proportion of individuals…who had ever used night splints (36.9%-73.0%), standers (3.1%-22.2%) and scooters (10.7%-54.5%). Among individuals who used night splints 59.7% stopped using them at a mean age of 10.3 years after a mean duration of 2.9 years in spite of the current recommendation to continue using them through the non-ambulatory phase. DISCUSSION: Results of this comprehensive survey document the frequency of assistive device use by individuals with DBMD in the USA and also provides data on differences across the sites. Further research is needed to understand the reasons for and the impact of these differences on clinical outcomes and health related quality of life of individuals with DBMD.
Abstract: PURPOSE: To record the use and perceived benefits of mainstream allied health services, complementary therapies, nutritional supplements and structured physical activity in a paediatric population of males with Duchenne or Becker muscular dystrophy. METHOD: A questionnaire was distributed to 125 parents of males with a dystrophinopathy within a tertiary neuromuscular clinic population in Melbourne, Australia. RESULTS: Response rate to the survey was 41%. Most families (73%) reported use of allied health services: physiotherapy (65%), occupational therapy (47%), and psychology (25%). The most commonly used complementary therapy was massage (31%). Sixty-five percent of families…reported using nutritional supplements. Fifty-one and 38% of families reported participation in swimming and other organised sports, respectively. Physical and psychological benefits of sporting activities were identified by families. Participation in physical activity was lowest in those transitioning to full-time wheelchair use. CONCLUSIONS: Access to allied health services by boys with dystrophinopathies is variable and inconsistent with published international standards of care. There is frequent use of complementary therapies, despite a lack of proven efficacy. Studies of the effects of such therapies would support provision of evidence-based advice to families. Continued involvement in physical activity for those boys with declining function should be supported by clinicians.
Abstract: PURPOSE: Managing the stresses of parenting a child with cerebral palsy (CP) can be challenging. This study sought to identify factors that are associated with higher levels of caregiver stress. METHODS: A retrospective review of data from the Learn From Every Patient™ project conducted in an interdisciplinary CP clinic were used to compare caregiver responses on 2 subsets (financial and time/emotional) of the Assessment of Caregiver Experience in Neuromuscular Disorders (ACEND) and physical and medical characteristics of the child. RESULTS: The range of scores in both the financial and emotional subset was…large. The presence of behavior problems, seizures, and severity of CP showed the strongest associations with emotional stress and accounted for 14% of the variance in scaled scores (r= 0.392, adj R2 = 14.3, p< 0.01). The child's age was not significantly related to parental stress. The most highly reported areas of stress were worry about the child's pain, and the financial impact of lost wages. CONCLUSION: Caregiver experience varied widely and is associated with a range of factors among families caring for a child with CP. Further research is needed to test whether interventions to minimize the areas of greatest stress could make a meaningful difference in family functioning.