Publications by authors named "Susan Apkon"

47 Publications

Respiratory outcomes in children with congenital myotonic dystrophy.

J Pediatr Rehabil Med 2021 Nov 14. Epub 2021 Nov 14.

Pediatric Rehabilitation Medicine, Children's Hospital Colorado, Denver, CO, USA.

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.
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http://dx.doi.org/10.3233/PRM-200726DOI Listing
November 2021

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.

J Comp Eff Res 2021 12 25;10(18):1337-1347. Epub 2021 Oct 25.

University of California Davis Health, Sacramento, CA, USA.

Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.
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http://dx.doi.org/10.2217/cer-2021-0018DOI Listing
December 2021

Steroid switching in dystrophinopathy treatment: a US chart review of patient characteristics and clinical outcomes.

J Comp Eff Res 2021 10 19;10(14):1065-1078. Epub 2021 Jul 19.

Children's Hospital Colorado, Aurora, CO 80045, USA.

To describe reasons for switching from prednisone/prednisolone to deflazacort and associated clinical outcomes among patients with Duchenne and Becker muscular dystrophy (DMD and BMD, respectively) in the USA. A chart review of patients with DMD (n = 62) or BMD (n = 30) who switched from prednisone to deflazacort (02/2017-12/2018) collected demographic/clinical characteristics, reasons for switching, outcomes and common adverse events. The mean ages at switch were 20.1 (DMD) and 9.2 (BMD) years. The primary physician-reported reasons for switching were 'to slow disease progression' (DMD: 83%, BMD: 79%) and 'tolerability' (67 and 47%). Switching was 'very' or 'somewhat' effective at addressing the primary reasons in 90-95% of patients. Physician-reported outcomes were consistent with deflazacort addressing patients' primary reasons for switching.
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http://dx.doi.org/10.2217/cer-2021-0110DOI Listing
October 2021

Reliability and construct validity of the Duchenne Video Assessment.

Muscle Nerve 2021 08 16;64(2):180-189. Epub 2021 Jun 16.

Casimir, Plymouth, Massachusetts, USA.

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.
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http://dx.doi.org/10.1002/mus.27335DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8361683PMC
August 2021

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

J Head Trauma Rehabil 2021 Jul-Aug 01;36(4):274-281

Departments of Anesthesiology and Pain Medicine (Dr Jimenez), Rehabilitation Medicine (Drs Fuentes and Apkon), Pediatrics (Drs Johnston and Rivara), Epidemiology (Dr Rivara), and Harborview Injury Prevention and Research Center (Drs Jimenez, Fuentes, and Rivara, Ms Virtue, and Mr Lopez), University of Washington, Seattle; Brain Injury Alliance of Washington, Seattle (Ms Crawley); and Center for Child Health, Behavior and Development, Seattle Children's Research Center, Seattle, Washington (Drs Jimenez, Fuentes, Zhou, and Rivara and Ms Alonso-Gonzalez).

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.
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http://dx.doi.org/10.1097/HTR.0000000000000658DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8249323PMC
October 2021

Children With Disabilities Must Be More Than an Afterthought in School Reopening.

JAMA Pediatr 2021 04;175(4):423-424

Physical Medicine & Rehabilitation and Pediatrics, University of Pittsburgh, Pittsburgh, Pennsylvania.

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http://dx.doi.org/10.1001/jamapediatrics.2020.5308DOI Listing
April 2021

Parent Project Muscular Dystrophy Females with Dystrophinopathy Conference, Orlando, Florida June 26 - June 27, 2019.

J Neuromuscul Dis 2021;8(2):315-322

Center for Gene Therapy, Nationwide Children's Hospital and Departments of Pediatrics and Neurology, Ohio State University, Columbus, Ohio, USA.

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http://dx.doi.org/10.3233/JND-200555DOI Listing
October 2021

Pediatric neuromuscular disorders: Care considerations during the COVID-19 pandemic.

J Pediatr Rehabil Med 2020;13(3):405-414

Department of Physical Medicine and Rehabilitation, Fischahs Chair in Pediatric Rehabilitation, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, USA.

COVID-19, the respiratory and frequently systemic disease caused by the novel SARS-COV-2 virus, was first recognized in December 2019 and quickly spread to become a pandemic and world-wide public health emergency over the subsequent 3-4 months. While COVID-19 has a very low morbidity rate across approximately 80% of the population, it has a high morbidity and mortality rate in the remaining 20% of the population.1 These numbers have put a significant strain on medical systems around the world. Patients with neuromuscular diseases such as those with Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), tend to be more medically fragile and have higher health care needs than the general population. Respiratory insufficiency, cardiac disease, obesity, and immunocompromised status due to chronic steroid treatments in certain patient populations with neuromuscular conditions are specific risk factors for severe COVID-19 disease. In general, the pediatric population has shown to be less severely impacted with lower infection rates and lower morbidity and mortality rates than the adult population, however, as expected, children with underlying medical conditions are at higher risk of morbidity from COVID-19 than their peers.2 Many patients with neuromuscular disease also rely heavily on caregiver support through their lifetime and thus maintaining the health of their primary caregivers is also a significant consideration in the health and well-being of the patients. This paper will address routine and emergency medical care, rehabilitation services, and other considerations for the pediatric patient with a neuromuscular condition during the COVID-19 pandemic.
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http://dx.doi.org/10.3233/PRM-200768DOI Listing
December 2020

School reopening during COVID-19 pandemic: Considering students with disabilities.

J Pediatr Rehabil Med 2020;13(3):425-431

Sholas Medical Consulting, LLC, New Orleans, LA, USA.

Over 80% of the children in the world have had their education impacted by COVID-19. For children with disabilities who receive special education services, access to in-person education and other resources at school is particularly important. The American Academy of Pediatrics advocates for students to attend school in person, without specifics for how children with disabilities can safely return to school. To appropriately plan and accommodate children with disabilities we must prioritize safety, allow for adherence to the Individuals with Disabilities Education Act, and preserve essential school staff. The less cumbersome default of confining students with disabilities to home is not acceptable. We provide an outline describing why Individual Education Plans and 504 plans are important, how they are related to the COVID-19 pandemic, and recommendations for measures to help with safe return to school for children with disabilities.
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http://dx.doi.org/10.3233/PRM-200789DOI Listing
December 2020

Transforaminal intrathecal delivery of nusinersen for older children and adults with spinal muscular atrophy and complex spinal anatomy: an analysis of 200 consecutive injections.

J Neurointerv Surg 2021 Jan 29;13(1):75-78. Epub 2020 May 29.

Radiology, University of Washington, Seattle, Washington, USA

Background: Nusinersen is the only approved treatment for all spinal muscular atrophy (SMA) subtypes and is delivered intrathecally. Distorted spinal anatomy and instrumentation preclude standard approaches for intrathecal access, necessitating alternative techniques for delivery. The purpose of this study is to report technical success and adverse events of transforaminal intrathecal delivery of nusinersen.

Methods: 28 patients, mean age 24.1±9.8 years (range 10.0-51.0 years), with intermediate or late onset SMA, underwent a combined 200 transforaminal nusinersen injections. All patients had osseous fusion or spinal instrumentation precluding standard posterior access routes. Patients who underwent nusinersen injections using a technique other than transforaminal lumbar puncture (n=113) were excluded. Technical success, adverse events (AEs) and radiation exposure were recorded.

Results: 200 (100%) procedures were technically successful; 6 (3%) required a second level of attempt for access. 187 (93.5%) interventions were completed using cone beam computed tomography (CBCT) with two-axis fluoroscopic navigational overlay. 13 (6.5%) procedures were performed with fluoroscopic-guidance only at subsequent sessions. There were 8 (4.0%) mild AEs and 2 (0.5%) severe AEs; one patient received antibiotics for possible traversal of the large bowel but did not develop meningitis, and one patient developed aseptic meningitis. Mean air kerma was 74.5±161.3 mGy (range 5.2-1693.0 mGy).

Conclusion: Transforaminal intrathecal delivery of nusinersen is feasible and safe for gaining access in patients with distorted spinal anatomy. The use of CBCT delineates anatomy and optimizes needle trajectory during the initial encounter, and may be used selectively for subsequent procedures.
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http://dx.doi.org/10.1136/neurintsurg-2020-016058DOI Listing
January 2021

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

Muscle Nerve 2020 07 5;62(1):41-45. Epub 2020 May 5.

Department of Neurology, University of Rochester Medical Center, Rochester, New York.

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.
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http://dx.doi.org/10.1002/mus.26902DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7264600PMC
July 2020

Restoration of Walking After Surgical Management of Equinus in a Non-ambulatory Child With Duchenne Muscular Dystrophy: A Case Presentation.

PM R 2019 11 26;11(11):1240-1243. Epub 2019 Apr 26.

Department of Rehabilitation, Seattle Children's Hospital, Seattle, Washington.

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http://dx.doi.org/10.1002/pmrj.12151DOI Listing
November 2019

Primary Care and Emergency Department Management of the Patient With Duchenne Muscular Dystrophy.

Pediatrics 2018 10;142(Suppl 2):S90-S98

MetroHealth Medical Center, Case Western Reserve University, Cleveland, Ohio.

Primary care providers (PCPs) are usually the first point of contact with the health care system for patients with Duchenne muscular dystrophy (DMD), and patients often present to emergency departments in which providers have little experience in dealing with this condition. With this article, we give primary care and emergency medicine providers a background in the common issues that affect people with DMD. By acquiring some specialized knowledge about the multisystem medical complications of DMD and by applying general principles of primary care, such as timely immunization, anticipatory safety counseling, behavioral screening, and routine nutritional and developmental assessments, the PCP can be a valued and effective medical provider to patients with DMD. The PCP can provide access to and effective coordination among the patient's specialty caregivers. Moreover, the PCP can become a trusted advisor to the patient and his family about important medical decisions, as well as issues in the psychosocial, behavioral, and educational domains. This article also contains a "pocket guide" used to assess and manage common urgent medical problems that cause patients with DMD to seek care in the emergency department. With the background information discussed in this article, both PCPs and emergency medicine physicians can skillfully care for patients with DMD in their respective settings, optimizing patient outcomes.
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http://dx.doi.org/10.1542/peds.2018-0333KDOI Listing
October 2018

Orthopedic and Surgical Management of the Patient With Duchenne Muscular Dystrophy.

Pediatrics 2018 10;142(Suppl 2):S82-S89

Shriner's Hospital for Children, Portland, Oregon.

Orthopedic care is an important aspect of the overall management of patients with Duchenne muscular dystrophy (DMD). In addition to progressive muscle weakness and loss of function, patients may develop joint contractures, scoliosis, and osteoporosis, causing fractures; all of these necessitate intervention by a multidisciplinary team including an orthopedic surgeon as well as rehabilitation specialists such as physio- and occupational therapists. The causes of these musculoskeletal complications are multifactorial and are related to primary effects on the muscles from the disease itself, secondary effects from weak muscles, and the related side effects of treatments, such as glucocorticoid use that affect bone strength. The musculoskeletal manifestations of DMD change over time as the disease progresses, and therefore, musculoskeletal management needs change throughout the life span of an individual with DMD. In this review, we target pediatricians, neurologists, orthopedic surgeons, rehabilitation physicians, anesthesiologists, and other individuals involved in the management of patients with DMD by providing specific recommendations to guide clinical practice related to orthopedic issues and surgical management in this setting.
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http://dx.doi.org/10.1542/peds.2018-0333JDOI Listing
October 2018

Rehabilitation Management of the Patient With Duchenne Muscular Dystrophy.

Pediatrics 2018 10;142(Suppl 2):S17-S33

Muscular Dystrophy Association of Queensland, Nundah, Australia.

Steadily improving management of Duchenne muscular dystrophy (DMD) continues to lead to improved physical and functional status, allowing increasingly successful transitions to independence and self-actualization in adulthood. Rehabilitation principles remain key to overall management for individuals with DMD with increasing options for ever more successful management, reflecting a changing natural history based on the use of glucocorticoids, more consistent comprehensive care, and the emergence of disease-modifying treatments. Advances and expansion in assessment, cardiorespiratory management, preventive management of contracture and deformity, assistive technology, "smart" technology, and robotics with increased emphasis on function, participation, self-advocacy, and independence in decision-making should allow individuals with DMD to experience childhood and transition to adulthood with support that allows for increasing success in the achievement of individual goals and fulfillment across the life span.
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http://dx.doi.org/10.1542/peds.2018-0333DDOI Listing
October 2018

The primary care pediatrician: key to improving neuromotor outcomes.

Dev Med Child Neurol 2018 09;60(9):851

Nationwide Children's Hospital - Complex Care, Columbus, OH, USA.

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http://dx.doi.org/10.1111/dmcn.13963DOI Listing
September 2018

Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan.

Lancet Neurol 2018 05 2;17(5):445-455. Epub 2018 Feb 2.

Division of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, and University of Ottawa, Ottawa, ON, Canada.

Improvements in the function, quality of life, and longevity of patients with Duchenne muscular dystrophy (DMD) have been achieved through a multidisciplinary approach to management across a range of health-care specialties. In part 3 of this update of the DMD care considerations, we focus on primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Many primary care and emergency medicine clinicians are inexperienced at managing the complications of DMD. We provide a guide to the acute and chronic medical conditions that these first-line providers are likely to encounter. With prolonged survival, individuals with DMD face a unique set of challenges related to psychosocial issues and transitions of care. We discuss assessments and interventions that are designed to improve mental health and independence, functionality, and quality of life in critical domains of living, including health care, education, employment, interpersonal relationships, and intimacy.
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http://dx.doi.org/10.1016/S1474-4422(18)30026-7DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5902408PMC
May 2018

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management.

Lancet Neurol 2018 04 3;17(4):347-361. Epub 2018 Feb 3.

Division of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, and University of Ottawa, Ottawa, ON, Canada.

A coordinated, multidisciplinary approach to care is essential for optimum management of the primary manifestations and secondary complications of Duchenne muscular dystrophy (DMD). Contemporary care has been shaped by the availability of more sensitive diagnostic techniques and the earlier use of therapeutic interventions, which have the potential to improve patients' duration and quality of life. In part 2 of this update of the DMD care considerations, we present the latest recommendations for respiratory, cardiac, bone health and osteoporosis, and orthopaedic and surgical management for boys and men with DMD. Additionally, we provide guidance on cardiac management for female carriers of a disease-causing mutation. The new care considerations acknowledge the effects of long-term glucocorticoid use on the natural history of DMD, and the need for care guidance across the lifespan as patients live longer. The management of DMD looks set to change substantially as new genetic and molecular therapies become available.
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http://dx.doi.org/10.1016/S1474-4422(18)30025-5DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5889091PMC
April 2018

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management.

Lancet Neurol 2018 03 3;17(3):251-267. Epub 2018 Feb 3.

Division of Endocrinology and Diabetes, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, USA.

Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in 2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved. In conjunction with improved patient survival, a shift to more anticipatory diagnostic and therapeutic strategies has occurred, with a renewed focus on patient quality of life. In 2014, a steering committee of experts from a wide range of disciplines was established to update the 2010 DMD care considerations, with the goal of improving patient care. The new care considerations aim to address the needs of patients with prolonged survival, to provide guidance on advances in assessments and interventions, and to consider the implications of emerging genetic and molecular therapies for DMD. The committee identified 11 topics to be included in the update, eight of which were addressed in the original care considerations. The three new topics are primary care and emergency management, endocrine management, and transitions of care across the lifespan. In part 1 of this three-part update, we present care considerations for diagnosis of DMD and neuromuscular, rehabilitation, endocrine (growth, puberty, and adrenal insufficiency), and gastrointestinal (including nutrition and dysphagia) management.
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http://dx.doi.org/10.1016/S1474-4422(18)30024-3DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5869704PMC
March 2018

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.

Pediatr Radiol 2018 03 13;48(3):392-397. Epub 2017 Nov 13.

Department of Radiology, Section of Interventional Radiology, Seattle Children's Hospital, University of Washington, 4800 Sand Point Way NE, M/S R-5417, Seattle, WA, 98105, USA.

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.
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http://dx.doi.org/10.1007/s00247-017-4031-6DOI Listing
March 2018

An algorithmic approach to the management of unrecognized hydrocephalus in pediatric candidates for intrathecal baclofen pump implantation.

Surg Neurol Int 2016 20;7:105. Epub 2016 Dec 20.

Department of Neurosurgery, Seattle Children's Hospital, Seattle, Washington, USA.

Background: Complications of intrathecal baclofen (ITB) pump implantation for treatment of pediatric patients with spasticity and dystonia associated with cerebral palsy remain unacceptably high. To address the concern that some patients may have underlying arrested hydrocephalus, which is difficult to detect clinically because of a low baseline level of neurological function, and may contribute to the high rates of postoperative cerebrospinal fluid leak, wound breakdown, and infection associated with ITB pump implantation, the authors implemented a standardized protocol including mandatory cranial imaging and assessment of intracranial pressure (ICP) by lumbar puncture prior to ITB pump implantation.

Methods: A retrospective case series of patients considered for ITB pump implantation between September 2012 and October 2014 at Seattle Children's Hospital is presented. All patients underwent lumbar puncture under general anesthesia prior to ITB pump implantation and, if the opening pressure was greater than 21 cmHO, ITB pump implantation was aborted and alternative management options were presented to the patient's family.

Results: Eighteen patients were treated during the study time period. Eight patients (44.4%) who had ICPs in excess of 21 cmHO on initial LP were identified. Eleven patients (61.1%) ultimately underwent ITB pump implantation (9/10 in the "normal ICP" group and 2/8 in the "elevated ICP" group following ventriculoperitoneal shunt placement), without any postoperative complications.

Conclusions: Given the potentially high rate of elevated ICP and arrested hydrocephalus, the authors advocate pre-implantation assessment of ICP under controlled conditions and a thoughtful consideration of the neurosurgical management options for patients with elevated ICP.
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http://dx.doi.org/10.4103/2152-7806.196236DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5223398PMC
December 2016

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

Arch Phys Med Rehabil 2017 09 23;98(9):1763-1770.e7. Epub 2017 Jan 23.

Seattle Children's Research Institute, Seattle, WA; Harborview Injury Prevention and Research Center, Seattle, WA; Department of Epidemiology, University of Washington School of Public Health, Seattle, WA; Department of Pediatrics, University of Washington School of Medicine, Seattle, WA.

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.
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http://dx.doi.org/10.1016/j.apmr.2016.12.013DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5522775PMC
September 2017

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

Phys Occup Ther Pediatr 2017 Oct 17;37(5):485-595. Epub 2017 Jan 17.

a Department of Rehabilitation , Seattle Children's Hospital , Seattle , Washington , USA.

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.
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http://dx.doi.org/10.1080/01942638.2016.1255289DOI Listing
October 2017

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

Muscle Nerve 2017 05 27;55(5):626-634. Epub 2017 Jan 27.

Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, USA.

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.
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http://dx.doi.org/10.1002/mus.25411DOI Listing
May 2017

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

Pediatr Neurosurg 2016 23;51(6):284-291. Epub 2016 Aug 23.

Department of Neurological Surgery, University of Washington, Seattle, Wash., USA.

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.
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http://dx.doi.org/10.1159/000448046DOI Listing
March 2017

Disparities in Functional Outcomes during Inpatient Rehabilitation between American Indian/Alaska Native and White Children.

J Health Care Poor Underserved 2016 ;27(3):1080-96

American Indian/Alaska Native (AI/AN) children have a high risk for poor health outcomes. Race/ethnicity and functional impairments are linked with health care disparities. While data exist for other race/ethnicity groups, little is known about outcomes for AI/AN children with functional impairments. In this study the Functional Independence Measure for Children (WeeFIM®) was used to determine differences in outcomes between AI/AN and White children receiving inpatient rehabilitation for functional impairments at one pediatric rehabilitation facility. American Indian/Alaska Native and White children had similar impairment types and functional levels at admission to inpatient rehabilitation. Both groups experienced functional improvement during rehabilitation. At discharge, AI/AN children had lower total functional scores and had less improvement in mobility scores compared with White children, with more impairment in physical mobility at discharge. This is one of the first studies to show a disparity in functional outcomes for AI/AN children compared with White children during inpatient rehabilitation.
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http://dx.doi.org/10.1353/hpu.2016.0143DOI Listing
April 2018

Functional outcomes during inpatient rehabilitation for American Indian and Alaska Native children with traumatic brain injury.

J Pediatr Rehabil Med 2016 05;9(2):133-41

Harborview Injury Prevention and Research Center, Seattle, WA, USA.

Purpose: American Indian/Alaska Native (AI/AN) people have the highest traumatic brain injury (TBI)-related mortality in the United States, but little is known about AI/AN children who survive traumatic brain injury (TBI). This study compares function and factors associated with discharge function between AI/AN and White children with TBI during inpatient rehabilitation.

Methods: Retrospective national cohort study of 114 AI/AN and 7,267 White children aged 6 months-18 years who received inpatient TBI rehabilitation between 2002-2012 at facilities utilizing the Uniform Data System for Medical Rehabilitation\scriptsize®. The outcome measure was developmental functional quotients (DFQ is the FIM\scriptsize® or WeeFIM\scriptsize® score divided by age norms x 100) at discharge.

Results: AI/AN race was not associated with motor (regression coefficient (β) 0.18, 95% confidence interval (CI) -2.39, 2.76) or cognitive (β -1.54, 95% CI -3.75, 0.67) function. Among a subgroup with loss of consciousness > 24 hours (AI/AN n= 13, White n= 643), AI/AN race was associated with lower motor DFQ (β -12.83, 95% CI -25.39, -0.34).

Conclusions: Overall, AI/AN race was not associated with inpatient rehabilitation function for children with TBI, but providers should not assume AI/ANs with more severe injuries have equitable outcomes.
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http://dx.doi.org/10.3233/PRM-160376DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5099074PMC
May 2016

Cases That "Stick" or Stay With Us.

PM R 2016 Apr;8(4):367-372

Rehabilitation Institute of Chicago and Northwestern University Feinberg School of Medicine, Donnelley Ethics Program, Physical Medicine & Rehabilitation, Medical Humanities, Rehabilitation Institute of Chicago, 345 Superior St Rm 164, Chicago, IL 60611(§). Electronic address:

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http://dx.doi.org/10.1016/j.pmrj.2016.02.007DOI Listing
April 2016

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

Arch Phys Med Rehabil 2016 09 26;97(9):1407-1412.e1. Epub 2016 Mar 26.

Seattle Children's Hospital, Seattle, WA; Harborview Injury Prevention and Research Center, Seattle, WA; Department of Pediatrics, University of Washington, Seattle, WA; Department of Epidemiology, University of Washington, Seattle, WA.

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.
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http://dx.doi.org/10.1016/j.apmr.2016.02.026DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5330160PMC
September 2016

Functional Outcomes and Unplanned Transfers of Pediatric Patients With Central Neurological Impairments Receiving Inpatient Rehabilitation Care With Cancer and Noncancer Diagnoses.

PM R 2016 06 26;8(6):529-35. Epub 2015 Oct 26.

Department of Rehabilitation Medicine, Seattle Children's Hospital, Seattle, WA; University of Washington, Seattle, WA(§).

Background: Functional impairments from central nervous system (CNS) dysfunction experienced by pediatric patients with cancer diagnoses are well documented. However, little is known of these patients' functional outcomes and potential complications while receiving inpatient rehabilitation services.

Objective: To compare functional outcomes and unplanned transfer rates of pediatric patients with impairments associated with CNS dysfunction between those with primary cancer diagnoses and noncancer diagnoses while receiving inpatient rehabilitation care.

Design: Retrospective comparison cohort study.

Setting: Inpatient rehabilitation unit within a regional tertiary care pediatric hospital.

Participants: Patients with CNS-based functional impairments with primary cancer diagnoses (n = 107) and noncancer diagnoses (n = 480), admitted to the inpatient rehabilitation unit between January 1, 2005 and April 1, 2012, who were aged 1 to 20 years at time of admission.

Intervention: Not applicable.

Main Outcome Measures: Pediatric Functional Independence Measure (WeeFIM) reflecting functional status was collected at admission and discharge with change score and WeeFIM efficiency calculated. Length of stay on the rehabilitation unit and unplanned transfer rates were also collected.

Results: No significant difference in total WeeFIM scores at admission was found between cancer and noncancer groups. Both groups had significant increases in WeeFIM scores at discharge (P < .001). The noncancer group had significantly higher WeeFIM change in self-care (P = .001), mobility (P = .009), and total score (P = .004) and had a greater length of stay (P < .001). A comparison of WeeFIM efficiency in all domains revealed no significant difference between cancer and noncancer groups. There was also no significant between-group difference in unplanned transfer rates.

Conclusions: Children with CNS-based functional impairments with cancer and noncancer diagnoses made functional gains with similar WeeFIM efficiencies after undergoing inpatient rehabilitation. However, patients with noncancer diagnoses made greater gains in self care, mobility, and total scores with longer stays on the rehabilitation service. No significant difference was found in unplanned transfer rates between cancer and noncancer groups for acute medical care.
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http://dx.doi.org/10.1016/j.pmrj.2015.10.007DOI Listing
June 2016
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