Quantitative measures of motor development in Angelman syndrome.

Angelman Syndrome is a rare neurodevelopmental disorder characterized by developmental delay, lack of speech, seizures, intellectual disability, characteristic behavior, and movement disorders. Clinical gait analysis provides the opportunity for movement quantification to investigate an observed maladaptive change in gait pattern and offers an objective outcome of change. Pressure-sensor-based technology, inertial and activity monitoring, and instrumented gait analysis (IGA) were employed to define motor abnormalities in Angelman syndrome. Temporal-spatial gait parameters of persons with Angelman Syndrome (pwAS) show deficiencies in gait performance through walking speed, step length, step width, and walk ratio. pwAS walk with reduced step lengths, increased step width, and greater variability. Three-dimensional motion kinematics showed increased anterior pelvic tilt, hip flexion, and knee flexion. PwAS have a walk ratio more than two standard deviations below controls. Dynamic electromyography showed prolonged activation of knee extensors, which was associated with a decreased range of motion and the presence of hip flexion contractures. Use of multiple gait tracking modalities revealed that pwAS exhibit a change in gait pattern to a flexed knee gait pattern.  Cross-sectional studies of individuals with AS show a regression toward this maladaptive gait pattern over development in pwAS ages 4-11. PwAS unexpectedly did not have spasticity associated with change in gait pattern. Multiple quantitative measures of motor patterning may offer early biomarkers of gait decline consistent with critical periods of intervention, insight into appropriate management strategies, objective primary outcomes, and early indicators of adverse events.

Reliability and construct validity of the Duchenne Video Assessment.

INTRODUCTION: The Duchenne Video Assessment (DVA) assesses quality of movement as an indication of Duchenne muscular dystrophy (DMD) disease severity. Caregivers video record patients performing home-based movement tasks using a mobile application, and physical therapists (PTs) rate the videos using scorecards with prespecified compensatory movement criteria. Reliability and construct validity of the DVA were tested using video and Pediatric Outcomes Data Collection Instrument (PODCI) data from patients with DMD and healthy controls from a separate study. METHODS: Fifteen PTs were trained and certified as DVA raters. All raters scored videos of five subjects performing each movement task; nine raters rescored the same videos four weeks later. Three raters scored videos from an average of 25 subjects for each movement task. Aggregate scores were used to test construct validity. An expert DMD clinician assigned each video to a severity group for known-groups analyses. Differences between rater scores across severity groups were tested and correlations between DVA and PODCI scores were calculated. RESULTS: Inter-rater reliability (intraclass correlation coefficient [ICC]) between all 15 raters ranged from 0.70 to 0.97 for all movement tasks. Mean intra-rater reliability ICC for nine raters ranged from 0.82 to 0.98 for all movement tasks. There were statistically significant differences between known severity groups for all movement tasks. The DVA correlated strongly with related PODCI constructs of physical function and weakly with unrelated constructs. DISCUSSION: The DVA was found to be a reliable and valid tool for measuring quality of movement as an indication of disease severity.

Feasibility and Acceptability of a Telephone-Based Intervention for Hispanic Children to Promote Treatment Adherence After Traumatic Brain Injury: A Pilot Study.

OBJECTIVE: To assess the feasibility and acceptability of a telephone-based education and navigation program for Hispanic parents of children hospitalized with traumatic brain injury (TBI). SETTING: Level I trauma hospital and pediatric inpatient rehabilitation unit in the Northwestern United States. PARTICIPANTS: Fourteen Hispanic parent-child dyads. Parents were 85% female, with a mean age of 35 years. Children were 58% male, with a mean age of 9.7 years, and had been hospitalized for complicated mild/moderate (n = 5) or severe (n = 9) TBI. DESIGN: Pilot prospective cohort design. MAIN MEASURES: Feasibility measures include recruitment, retention, and intervention adherence rates. Acceptability of intervention was measured by parents' use of educational materials and satisfaction with navigation program. We also evaluated study processes, including completion of baseline, 3, 6, and 12 months functional assessments of the child; assessment of parental health literacy and self-efficacy; and adherence to follow-up rehabilitation appointments. RESULTS: Eighty-two percent of approached potential participants were recruited into the study. One hundred percent of participants completed the intervention, and 85% had 1-year follow-up. Intervention acceptability was high: 90% reported satisfaction with navigator, and 92% used the educational manual. Assessments demonstrated significant improvement in parents' TBI caregiving and community self-efficacy; 92% attendance to follow-up rehabilitation appointments; and improvement in the child's functional measures, except communication skills. CONCLUSIONS: Findings support feasibility and acceptability of a culturally relevant program to facilitate transitions of care for Hispanic children with TBI. A future randomized trial is warranted to determine the efficacy of the intervention on long-term treatment adherence and the child's post-TBI function.

The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic.

The coronavirus disease 2019 (COVID-19) pandemic has resulted in the reorganization of health-care settings affecting clinical care delivery to patients with Duchenne and Becker muscular dystrophy (DBMD) as well as other inherited muscular dystrophies. The magnitude of the impact of this public health emergency on the care of patients with DBMD is unclear as they are suspected of having an increased risk for severe manifestations of COVID-19. In this article, the authors discuss their consensus recommendations pertaining to care of these patients during the pandemic. We address issues surrounding corticosteroid and exon-skipping treatments, cardiac medications, hydroxychloroquine use, emergency/respiratory care, rehabilitation management, and the conduct of clinical trials. We highlight the importance of collaborative treatment decisions between the patient, family, and health-care provider, considering any geographic or institution-specific policies and precautions for COVID-19. We advocate for continuing multidisciplinary care for these patients using telehealth.

Transforaminal intrathecal delivery of nusinersen using cone-beam computed tomography for children with spinal muscular atrophy and extensive surgical instrumentation: early results of technical success and safety.

BACKGROUND: Nusinersen, the only treatment approved by the United States Food and Drug Administration for spinal muscular atrophy (SMA), is delivered intrathecally. Many children with SMA have extensive spinal instrumentation and deformities, often precluding the use of standard approaches for gaining intrathecal access. Furthermore the anatomical distortion that often occurs with rotoscoliosis can complicate the use of fluoroscopic guidance. Compared to fluoroscopy, CT affords superior guidance for complex needle placements. This opens up alternatives to the posterior (interlaminar) technique, including transforaminal and caudal approaches. OBJECTIVE: This study describes the early results of technical success, complications and radiation dose of intrathecal delivery of nusinersen using cone-beam CT guidance with two-axis fluoroscopic navigational overlay. MATERIALS AND METHODS: We conducted a retrospective review of 15 consecutive nusinersen injections performed in four children with SMA and extensive spinal hardware precluding standard posterior lumbar puncture techniques. These children were treated using transforaminal thecal access employing cone-beam CT with navigational overlay. We analyzed results including technical success, complications and total fluoroscopy time. RESULTS: All procedures were technically successful. No major complications and one minor complication were reported; the minor complication was a post-procedural neuropathic headache that was attributed to procedural positioning and was treated successfully with gabapentin. The average procedural fluoroscopy time and air kerma were 1.9 min and 55.8 mGy, respectively. CONCLUSION: Cone-beam CT guidance with two-axis navigational overlay is a safe, effective method for gaining transforaminal intrathecal access in children with spinal abnormalities and hardware precluding the use of standard techniques.

Patient-centered benefit-risk assessment in duchenne muscular dystrophy.

INTRODUCTION: This study quantified caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregiver and patient differences were also explored. METHODS: A best-worst scaling survey (BWS) was administered to caregivers and patients. Across 9 profiles, respondents selected the best and worst attributes. Utility scores were estimated using mixed logistic regression. RESULTS: Respondents indicated greatest preference for therapies that maintain their current level of cough strength for 10 years or for 2 years. Preference scores for risks were low: 50% chance of diarrhea and 4 additional blood draws per year. CONCLUSION: There is a strong preference for pulmonary benefit and willingness to trade off risks and burden to achieve these benefits. In exchange for maintaining cough strength for 10 years, respondents were willing to tolerate high probabilities of diarrhea and additional blood draws. Muscle Nerve 55: 626-634, 2017.

Auditing Access to Outpatient Rehabilitation Services for Children With Traumatic Brain Injury and Public Insurance in Washington State.

OBJECTIVE: To identify insurance-based disparities in access to outpatient pediatric neurorehabilitation services. DESIGN: Audit study with paired calls, where callers posed as a mother seeking services for a simulated child with history of severe traumatic brain injury and public or private insurance. SETTING: Outpatient rehabilitation clinics. PARTICIPANTS: Sample of rehabilitation clinics (N=287): 195 physical therapy (PT) clinics, 109 occupational therapy (OT) clinics, 102 speech therapy (ST) clinics, and 11 rehabilitation medicine clinics. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Acceptance of public insurance and the number of business days until the next available appointment. RESULTS: Therapy clinics were more likely to accept private insurance than public insurance (relative risk [RR] for PT clinics, 1.33; 95% confidence interval [CI], 1.22-1.44; RR for OT clinics, 1.40; 95% CI, 1.24-1.57; and RR for ST clinics, 1.42; 95% CI, 1.25-1.62), with no significant difference for rehabilitation medicine clinics (RR, 1.10; 95% CI, 0.90-1.34). The difference in median wait time between clinics that accepted public insurance and those accepting only private insurance was 4 business days for PT clinics and 15 days for ST clinics (P≤.001), but the median wait time was not significantly different for OT clinics or rehabilitation medicine clinics. When adjusting for urban and multidisciplinary clinic statuses, the wait time at clinics accepting public insurance was 59% longer for PT (95% CI, 39%-81%), 18% longer for OT (95% CI, 7%-30%), and 107% longer for ST (95% CI, 87%-130%) than that at clinics accepting only private insurance. Distance to clinics varied by discipline and area within the state. CONCLUSIONS: Therapy clinics were less likely to accept public insurance than private insurance. Therapy clinics accepting public insurance had longer wait times than did clinics that accepted only private insurance. Rehabilitation professionals should attempt to implement policy and practice changes to promote equitable access to care.

Physical and Occupational Therapy for a Teenager with Acute Flaccid Myelitis: A Case Report.

AIMS: The purpose of this case report is to describe the inpatient rehabilitation program of a 13-year-old boy with acute flaccid myelitis (AFM), specific to physical and occupational therapy examination, treatment, and outcomes. CASE DESCRIPTION: AFM is a rare, acute neurologic illness in children and young adults who present with weakness and/or paralysis of unknown etiology. The teenager was admitted to the program, dependent for all mobility and self-care. Interventions focused on range of motion, transfer training, self-care, power wheelchair mobility, and environmental adaptations. OUTCOMES: Weekly re-evaluations and the WeeFIM were used at admission and discharge to measure the teenager's progress. At discharge, the teenager had made small gains in his passive and active range of motion. He was independent in directing his care and able to drive his power wheelchair with supervision. DISCUSSION: Due to the scarcity of published data describing AFM, this report describes an individual's response to a rehabilitation program and will hopefully add to future research in order to provide patients and families with expectations for their recovery and ultimate level of function.

Association Between Facility Type During Pediatric Inpatient Rehabilitation and Functional Outcomes.

OBJECTIVE: To compare functional outcomes between children receiving inpatient rehabilitation at children's hospitals and those at other facilities. DESIGN: Retrospective cohort study. SETTING: Inpatient rehabilitation facilities. PARTICIPANTS: Children (N=28,793) aged 6 months to 18 years who received initial inpatient rehabilitation. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Total, cognitive, and motor developmental functional quotients (DFQs; which is the WeeFIM score divided by age-adjusted norms and multiplied by 100) at discharge from inpatient rehabilitation and WeeFIM efficiency (the change in WeeFIM score from admission to discharge divided by the length of the rehabilitation stay), adjusting for age, sex, race, insurance, region, admission function, impairment type, discharge year, and length of stay. RESULTS: A total of 12,732 children received rehabilitation at 25 children's hospitals and 16,061 at 36 other facilities (general hospitals or freestanding rehabilitation hospitals). Adjusting for clustering by facility, patients at children's hospitals had a lower cognitive DFQ at admission (difference between children's hospitals and other facility types, -3.8; 95% confidence interval [CI], -7.7 to -0.1), a shorter length of stay (median, 16d vs 22d; P<.001), and a higher WeeFIM efficiency (difference, 0.63; 95% CI, 0.25-1.00) than did children at other facility types. Rehabilitation in a children's hospital was independently associated with a higher discharge cognitive DFQ (regression coefficient, 2.3; 95% CI, 0.3-4.2) and more efficient rehabilitation admissions (regression coefficient, 0.3; 95% CI, 0.1-0.6). CONCLUSIONS: Children who receive inpatient rehabilitation at children's hospitals have more efficient inpatient rehabilitation admissions, a shorter median length of stay, and a slight improvement in cognitive function than do children at other facility types.

Infra-Conus Single-Level Laminectomy for Selective Dorsal Rhizotomy: Technical Advance.

BACKGROUND/AIMS: Selective dorsal rhizotomy for spastic cerebral palsy is an effective and well-validated surgical approach. Multiple techniques have been described in the past including multiple laminectomies and a single-level laminectomy at the level of the conus. There is considerable technical challenge involved with a single-level laminectomy approach. METHODS: We report here a modification of the single-level laminectomy that selectively analyzes each individual nerve root with electromyography to separate dorsal and ventral nerve roots through comparison of stimulus responses. RESULTS: In 18 children with cerebral palsy who underwent this operation there was a mean improvement in the Modified Ashworth Scale of 2.0 with no reported incidence of muscle weakness, sensory loss, or neurogenic bladder. CONCLUSION: This approach allows for a modification of selective dorsal rhizotomy through a single-level laminectomy and tailors the selection of nerve root sectioning to the individual patient of interest while still maintaining its effectiveness.

Electrical impedance myography in Duchenne muscular dystrophy and healthy controls: A multicenter study of reliability and validity.

INTRODUCTION: Electrical impedance myography (EIM) is a non-invasive, painless, objective technique to quantify muscle pathology. METHODS: We measured EIM in 8 arm and leg muscles in 61 boys with Duchenne muscular dystrophy (DMD) and 31 healthy boys, ages 3-12 years, at 5 centers. We determined the reliability of EIM and compared results in boys with DMD to controls and to 6-minute walk distance (6MWD), North Star Ambulatory Assessment (NSAA), timed functional tests (TFTs), and strength (hand-held dynamometry). RESULTS: EIM was well tolerated and had good inter- and intrarater reliability (intraclass correlation coefficient 0.81-0.96). The averaged EIM phase value from all muscles was higher (P < 0.001) in controls (10.45 ± 2.29) than boys with DMD (7.31 ± 2.23), and correlated (P ≤ 0.001) with 6MWD (r = 0.55), NSAA (r = 0.66), TFTs (r = -0.56), and strength (r = 0.44). CONCLUSION: EIM is a reliable and valid measure of disease severity in DMD. Longitudinal studies comparing EIM with other assessments over time in DMD are warranted.

Functional independence after inpatient rehabilitation for traumatic brain injury among minority children and adolescents.

OBJECTIVE: To compare motor and cognitive functional independence scores between Hispanic, non-Hispanic black (NHB), and non-Hispanic white (NHW) children with traumatic brain injury (TBI) after discharge from inpatient rehabilitation. DESIGN: Retrospective cohort study using the Uniform Data System for Medical Rehabilitation national dataset from the years 2002 to 2012. SETTING: Inpatient rehabilitation units. PARTICIPANTS: Children (N=10,141) aged 6 months to 18 years who received inpatient rehabilitation for TBI. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Motor and cognitive functional independence after discharge from inpatient rehabilitation, adjusting for age, sex, admission function, length of stay, insurance, and region. RESULTS: Inpatient rehabilitation therapy improved functional independence for all children. Younger age, lower admission functional independence scores, and Medicaid insurance were associated with lower functional independence at discharge. Hispanic and NHB children had lower discharge cognitive scores compared with NHW children; however, differences were small and were partially explained by insurance status and region. Children who received rehabilitation therapy at pediatric facilities had greater cognitive improvement. CONCLUSIONS: While racial/ethnic disparities are small, minority children are more likely to be younger, to have Medicaid, and to be cared for at nonpediatric facilities, factors that increase their risk for lower functional outcomes.

Risk Factors for First Fractures Among Males With Duchenne or Becker Muscular Dystrophy.

BACKGROUND: Fractures are a significant concern for individuals with Duchenne/Becker muscular dystrophy with 21% to 44% of males experiencing a fracture. Factors that increase or decrease the risk for fracture have been suggested in past research, although statistical risk has not been determined. METHODS: In this retrospective cohort study, we used the Muscular Dystrophy Surveillance, Tracking and Research Network cohort, a large, population-based sample to identify risk factors associated with first fractures in patients with Duchenne or Becker muscular dystrophy. Our study cohort included males with Duchenne or Becker muscular dystrophy born between 1982 and 2006 who resided in Arizona, Colorado, Georgia, Iowa, and Western New York, retrospectively identified and followed through 2010. We utilized a multivariate Cox proportional hazard model to determine hazard ratios for relevant factors associated with first fracture risk including race/ethnicity, surveillance site, ambulation status, calcium/vitamin D use and duration, bisphosphonate use and duration, and corticosteroid use and duration. RESULTS: Of 747 cases, 249 had at least 1 fracture (33.3%). Full-time wheelchair use increased the risk of first fracture by 75% for every 3 months of use (hazard ratio=1.75, 95% confidence interval, 1.14, 2.68), but corticosteroid use, bisphosphonate use, and calcium/vitamin D use did not significantly affect risk in the final adjusted model. CONCLUSIONS: In this cohort, first fractures were common and full-time wheelchair use, but not corticosteroid use, was identified as a risk factor. The impact of prevention measures should be more thoroughly assessed. CLINICAL RELEVANCE: Fractures are a significant concern for individuals with dystrophinopathies, but the contribution of various risk factors has not been consistently demonstrated.

Sibling concordance for clinical features of Duchenne and Becker muscular dystrophies.

INTRODUCTION: The correlation of markers of disease severity among brothers with Duchenne or Becker muscular dystrophy has implications for clinical guidance and clinical trials. METHODS: Sibling pairs with Duchenne or Becker muscular dystrophy (n = 60) were compared for ages when they reached clinical milestones of disease progression, including ceased ambulation, scoliosis of ≥ 20°, and development of cardiomyopathy. RESULTS: The median age at which younger brothers reached each milestone, compared with their older brothers ranged from 25 months younger for development of cardiomyopathy to 2 months older for ceased ambulation. For each additional month of ambulation by the older brother, the hazard of ceased ambulation by the younger brother decreased by 4%. CONCLUSIONS: The ages when siblings reach clinical milestones of disease vary widely between siblings. However, the time to ceased ambulation for older brothers predicts the time to ceased ambulation for their younger brothers.

The Early Care (0-3 Years) In Duchenne Muscular Dystrophy Meeting Report.

Objective: This report summarizes the key discussions from the "Early Care (0-3 years) in Duchenne Muscular Dystrophy" meeting, which aimed to address the challenges and opportunities in the diagnosis and care of Duchenne muscular dystrophy (DMD) and female carriers within the 0-3-year age group. Methods: The meeting brought together experts and healthcare providers who shared insights, discussed advancements in DMD care, and identified research needs. Presentations covered diagnostic challenges, approved therapies, clinical trials, identification of young female carriers, and the importance of clinical care and support for families. Results: The meeting highlighted the importance of timely diagnosis and the lack of evidence-based guidelines for the care of children with DMD aged 0-3 years. Diagnostic challenges were discussed, including delays in receiving a DMD diagnosis and disparities based on ethnicity. The potential benefits and process of newborn screening were addressed.Approved therapeutic interventions, such as corticosteroids and exon-skipping drugs, were explored, with studies indicating the potential benefits of early initiation of corticosteroid therapy and the safety of exon-skipping drugs in DMD. Clinical trials involving infants and young boys were discussed, focusing on drugs like ataluren, vamorolone, and gene therapies.The meeting emphasized the importance of clinical care and support for families, including comprehensive information provision, early intervention services, and individualized support. The identification and care of young female carriers were also addressed. Conclusion: The meeting provided a platform for experts and healthcare providers to discuss and identify key aspects of early care for children with DMD aged 0-3 years. The meeting emphasized the need for early diagnosis, evidence-based guidelines, and comprehensive care and support for affected children and their families. Further research, collaboration, and the development of consensus guidelines are needed to improve early diagnosis, treatment, and outcomes in this population.

Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals.

BACKGROUND: Despite the increasing availability of clinical trials in Duchenne muscular dystrophy, racial/ethnic minorities and other populations facing health disparities remain underrepresented in clinical trials evaluating products for Duchenne. We sought to understand the barriers faced by Hispanic/Latino families specifically and underrepresented groups more generally to clinical trial participation in Duchenne. METHODS: We engaged two participant groups: Hispanic/Latino caregivers of children with Duchenne in the US, including Puerto Rico, and health professionals within the broader US Duchenne community. Caregiver interviews explored attitudes towards and experiences with clinical trials, while professional interviews explored barriers to clinical trial participation among socio-demographically underrepresented families (e.g., low income, rural, racial/ethnic minority, etc.). Interviews were analyzed aggregately and using a thematic analysis approach. An advisory group was engaged throughout the course of the study to inform design, conduct, and interpretation of findings generated from interviews. RESULTS: Thirty interviews were conducted, including with 12 Hispanic/Latina caregivers and 18 professionals. We identified barriers to clinical trial participation at various stages of the enrollment process. In the initial identification of patients, barriers included lack of awareness about trials and clinical trial locations at clinics that were less likely to serve diverse patients. In the prescreening process, barriers included ineligibility, anticipated non-compliance in clinical trial protocols, and language discrimination. In screening, barriers included concerns about characteristics of the trial, as well as mistrust/lack of trust. In consent and recruitment, barriers included lack of timely decision support, logistical factors (distance, time, money), and lack of translated study materials. CONCLUSIONS: Numerous barriers hinder participation in Duchenne clinical trials for Hispanic/Latino families and other populations experiencing health disparities. Addressing these barriers necessitates interventions across multiple stages of the clinical trial enrollment process. Recommendations to enhance participation opportunities include developing clinical trial decision support tools, translating prominent clinical trials educational resources such as ClinicalTrials.gov, fostering trusting family-provider relationships, engaging families in clinical trial design, and establishing ethical guidelines for pre-screening potentially non-compliant patients.

Challenges and Opportunities in Academic Physiatry: An Environmental Scan.

ABSTRACT: Environmental scans determine trends in an organization's or field's internal and external environment. The results can help shape goals, inform strategic decision making, and direct future actions. The Association of Academic Physiatrists convened a strategic planning group in 2020, composed of physiatrists representing a diversity of professional roles, career stages, race and ethnicity, gender, disability status, and geographic areas of practice. This strategic planning group performed an environmental scan to assess the forces, trends, challenges, and opportunities affecting both the Association of Academic Physiatrists and the entire field of academic physiatry (also known as physical medicine and rehabilitation, physical and rehabilitation medicine, and rehabilitation medicine). This article presents aspects of the environmental scan thought to be most pertinent to the field of academic physiatry organized within the following five themes: (1) Macro/Societal Trends, (2) Technological Advancements, (3) Diversity and Global Outreach, (4) Economy, and (5) Education/Learning Environment. The challenges and opportunities presented here can provide a roadmap for the field to thrive within the complex and evolving healthcare systems in the United States and globally.

Respiratory outcomes in children with congenital myotonic dystrophy.

PURPOSE: Congenital myotonic dystrophy (CDM) results in hypotonia and acute respiratory distress at birth. Previous studies show that prolonged periods of intubation (>4 weeks) correlate with increased mortality rates. The objective is to describe the use and duration of respiratory support in newborns with CDM and how these relate to mortality. METHODS: A retrospective chart review was performed at a tertiary pediatric hospital among children with confirmed diagnosis of CDM. The main outcome measures were: mortality, duration of invasive mechanical ventilation (IMV) and non-invasive partial pressure ventilation (NIPPV), along with long-term use of respiratory support and equipment. RESULTS: A total of 18 subjects met inclusion criteria, 83%.f which had documented respiratory distress at birth, 39%.equired NIPPV, and 50%.equired intubation in the neonatal period. The earliest NIPPV was initiated at day one of life, and the latest extubation to NIPPV was at 17 days of life. CONCLUSION: This cohort required IMV for shorter periods with earlier transitions to NIPPV which suggests a possible change in practice and earlier transition to NIPPV recently. Further data are needed to determine if there is a possible correlation between the need for NIPPV/IMV and mortality rates.

Palliative care services in families of males with Duchenne muscular dystrophy.

INTRODUCTION: Palliative care services that address physical pain and emotional, psychosocial, and spiritual needs may benefit individuals with Duchenne muscular dystrophy (DMD). METHODS: The objective of this study was to describe the palliative care services that families of males with DMD report they receive. A questionnaire was administered to families of males with DMD born prior to January 1, 1982. Thirty-four families responded. RESULTS: Most families (85%) had never heard the term palliative care. Only attendant care and skilled nursing services showed much usage, with 44% and 50% indicating receipt of these services, respectively. Receipt of other services was reported less frequently: pastoral care (27%); respite care (18%); pain management (12%); and hospice care (6%). Only 8 respondents (25%) reported having any type of directive document in place. CONCLUSION: The data suggest a need for improved awareness of palliative care and related services among families of young men with DMD.