Rehabilitation Management Laura E. Case, PT, DPT, MS, PCS, C/NDT,a​ Susan D. Apkon, MD,​b Michelle Eagle, PhD,c​ ofAnn Gulyas, the MA, CCC-SLP, Patient BCS-S,d​ Laura Juel, With MS, OTR/L, ATP, DuchenneCDRS,​e Dennis Matthews, MD,​f g h RobbinMuscular A. Newton, MA, OTR/L, BCP,Dystrophy C/NDT,​ Helen F. Posselt, BA, Dip Physiotherapy abstract

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.

aDoctor of Physical Therapy Division, Department of Orthopedics, School of Medicine, Duke University, Durham, North Carolina; bDepartment of Rehabilitation Medicine, Seattle Children’s Hospital, Seattle, Washington; cNewcastle University, Newcastle upon Tyne, United Kingdom; dWestern Maryland Hospital System, Hagerstown, Maryland; eDuke University Health System and Lenox Baker Children’s Hospital, Durham, North Carolina; gDuke University Health System, Durham, North Carolina; fChildren’s Hospital Colorado, Aurora, Colorado; and hMuscular Dystrophy Association of Queensland, Nundah, Australia

The guidelines or recommendations in this article are not American Academy of Pediatrics policy, and publication herein does not imply endorsement.

Dr Case served as the chairperson of the Duchenne Muscular Dystrophy Care Considerations Rehabilitation Management Working Group as convened by the Centers for Disease Control and Prevention, drafted the initial manuscript, and reviewed and revised the manuscript; Drs Apkon, Eagle, and Matthews and Ms Gulyas, Ms Juel, Ms Newton, and Ms Posselt served in the Duchenne Muscular Dystrophy Care Considerations Rehabilitation Management Working Group as convened by the Centers for Disease Control and Prevention, contributed to the development of corresponding recommendations, and reviewed and revised the manuscript; and all authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work. DOI: https://​doi.​org/​10.​1542/​peds.​2018-​0333D Accepted for publication Jul 26, 2018 Address correspondence to Laura E. Case, PT, DPT, MS, PCS, C/NDT, Doctor of Physical Therapy Division, Department of Orthopedics, School of Medicine, DUMC Box 104002, Duke University, Durham, NC, 27708. E-mail: [email protected] PEDIATRICS (ISSN Numbers: Print, 0031-4005; Online, 1098-4275). Copyright © 2018 by the American Academy of Pediatrics FINANCIAL DISCLOSURE: Dr Case has received honoraria from the Genzyme Corporation of Sanofi; has participated in research supported by the Genzyme Corporation of Sanofi, PTC Therapeutics, the George and Mary Ann Leal Foundation, Families of Spinal Muscular Atrophy, Enobia Pharma and Alexion Pharmaceuticals, Amicus, the Robertson Foundation, the Marcus Foundation, GlaxoSmithKline, Eli Lilly and Company, the Cooperative International Neuromuscular Research Group, Valerion Therapeutics, Pfizer, Prosensa, BioMarin Pharmaceutical, Ionis Pharmaceuticals, Biogen, Ultragenyx Pharmaceutical, NS Pharma, Reveragen, Roivant Sciences, Therapeutic Research in Neuromuscular Disorders Solutions, Cytokinetics, and AveXis; has been awarded grant support by the National Skeletal Muscle Research Center; and is a member of the Pompe Registry North American Board of Advisors for the Genzyme Corporation of Sanofi. Dr Apkon is a

Downloaded from www.aappublications.org/news by guest on September 29, 2021 PEDIATRICS Volume 142, number s2, October 2018:e20180333D SUPPLEMENT ARTICLE ASSESSMENT

Duchenne Muscular Dystrophy by concern, change in status,

(DMD) Care– Considerations,1,2​ initially or specific needs as supported published3 in5 2010,​ ‍ were recently Multidisciplinary assessment across by professional85,86​ standards of updated. ‍ These considerations, the International Classification of care. ‍ supported by the Centers for Functioning, Disability, and Health INTERVENTION Disease Control and Prevention, and care continuum remains offer overall– recommendations for important in guiding rehabilitation management3 5 by the multidisciplinary interventions. Assessment tools Comprehensive, anticipatory, team. ‍‍ In this specialty article, we recommended in the original preventive rehabilitation offer in-depth descriptions of the (2010) Care Considerations remain management is focused on protecting current comprehensive, anticipatory, supported and expanded via newly fragile muscles; preserving and preventive rehabilitation management developed tools (Tables 3 and 4). maintaining optimal strength; of DMD including an updated Impairment-level measures minimizing the progression of understanding of the pathokinesiology include passive ranges of motion weakness when possible; preventing of disease progression, changing (ROM) and the assessment of and minimizing progressive natural history, recommendations alignment and posture, which is contracture and deformity; for assessment and intervention, critical in monitoring the success supporting optimal cardiorespiratory and future directions. Respiratory of musculoskeletal management care and function; optimizing energy rehabilitation management is and identifying needs for efficiency and energy conservation; covered in the article on respiratory additional physical therapy (PT), providing adaptive equipment and management6 that is part of this occupational therapy (OT), orthotic assistive technology; maintaining supplement. intervention, serial casting, seating skin integrity; preventing and system modification, supported minimizing pain; supporting function, DMD is characterized by well- standing, and adaptive equipment. functional independence, and known patterns of progressive Standardized functional assessments participation at– school,– – work, and in muscle degeneration and weakness, for DMD have been expanded family and social1 3,6​ life;9,20​ and31,108​ optimizing postural compensations, risk with the establishment of validity, quality of life. ‍ ‍ ‍ ‍ ‍ ‍ ‍ ‍ of progressive contracture and reliability, predictive potential, A multidisciplinary rehabilitation deformity, and functional– losses and minimal clinically important team is required that includes resulting from dystrophin1,2,​ 7​ 19 deficiency differences, which– are described (Tables 1 and 2). ‍ ‍‍ The natural in detail in the 2018 DMD Care physicians, physical therapists, 3 5 occupational therapists, speech- history of DMD has changed over Considerations. –‍ These assessments language pathologists, orthotists, the years with –more– comprehensive– should be used across the life span 32,41,​ 58​ 66 and providers of durable medical medical and therapeutic1 3,6​ 9,15,​ 16,​ 20​– 37 (Table 3). ‍ ‍ ‍ ‍ Use of the same management,​ ‍ ‍ ‍‍ ‍ ‍ ‍‍ the measures over time, including new equipment who coordinate with 38 40 those in pulmonary medicine, use of glucocorticoids,​ –‍‍ and assessments as appropriate, is orthopedics, cardiology, neurology, the advent of emerging disease- recommended to monitor– change and 38 43 genetics, social work, psychology, modifying treatments– . ‍ ‍ These support anticipatory management. 67 73 74 endocrinology, nutrition, and advances have resulted in prolonged Measurement– of pain,​ ‍ ‍ fatigue,​ 28,43​ 54 75 gastroenterology. Direct skilled PT, ambulation,​ ‍‍ decreases disability,69,75​ ​ 77 participation,– quality and/or delays in the development of life,​ ‍ ‍ and patient-reported OT, and speech-language therapy, 32,78​ 84 based on individual assessment, are of severe contracture– – and– outcomes ‍ ‍ ‍ are important deformity (including as is the increasing use of activity provided in outpatient, home, and 2,7​ 9,15,​ 16,​ 20​ 22,24​ 31,55,​ 56​ 59,60,​ 63​ school settings and in inpatient settings scoliosis),– ​ ‍ ‍ ‍ ‍ ‍ ‍ ‍ ‍‍ ‍ monitoring. ‍ Occupational during hospitalizations and should be improved1 4,6,​ cardiorespiratory36,​ 57​ therapist assessment of learning, status,​ ‍‍ ‍ ‍ and prolonged attentional, and sensory processing Musculoskeletalcontinued throughout Management adulthood. function into adulthood. Patients differences, fine motor function, and (Prevention of Contracture and have been supported by advancing activities of daily living (ADL) should Deformity) “ ” adaptive equipment, assistive begin early, guiding intervention and technology, and smart technology,– optimizing success. Multidisciplinary all of which promise ever-increasing1 3,31​ assessment across the International Anticipatory preventive participation in adulthood. ‍ ‍ Classification of Functioning, musculoskeletal management ‍Figure 1 includes an overall summary Disability, and Health should occur is focused on preserving muscle of rehabilitative care during the at least every 6 months, with more extensibility, joint mobility, and various clinical stages of DMD. frequent assessment being triggered symmetry to prevent and minimize Downloaded from www.aappublications.org/news by guest on September 29, 2021 S18 CASE et al TABLE 1 Pathokinesiology of Disease Progression Weakness Compensations Risk of Tightness (2-joint muscles get tight first) Early stage • Hip extensors (gluteus maximus) • Posterior trunk lean and increased lumbar • May see emerging tightness in the following: lordosis used to keep line of gravity behind hip joint (initially see less anterior pelvic tilt when hyperextension at hip joint in stance as long as quadriceps are strong enough to counteract movement into knee flexion) • Ankle dorsiflexors (anterior tibialis) • Lack of heel strike • Plantar flexors • Increased hip flexion during swing to clear foot • Foot may be pronated and averted • Hip abductors (gluteus medius) • May see "hip-waddling gait" due to inadequate • Hip flexors forward weight shift • Hip adductors • Increased UE abduction and lateral trunk sway • Iliotibial bands • Abdominals • Cadence decreases • Gowers maneuver

• Neck flexors (sternocleidomastoid) • Neck and UE weakness not usually noticeable functionally but apparent with testing; may see slight head lag when pulled up to sit; may "slide through your hands" when lifted up under shoulders • Shoulder depressors and extensors (lower trap and/or latissimus) • Shoulder abductors (deltoids) • Elbow extensors (triceps)

Transitional stage • Weakness progresses in muscles listed above • Must get line of gravity simultaneously in front • Iliotibial band and tensor fascia lata of knee joint and behind and lateral to hip joint; see: • Quadriceps weakness is key to gait • Anterior pelvic tilt • Hip flexors deterioration • Increased posterior and lateral trunk lean • Hamstrings during stance • Increased lumbar lordosis • Gastrocsoleus • Diminished hip extension in stance • Posterior tibialis • Base of support widens • Plantar fascia • For balance • Secondary to tight iliotibial bands • Ankle evertors (peroneals) • Increased ankle plantarflexion and equinus positioning to give torque that opposes knee flexion; begin to see increased falling; get inversion with posterior tibialis relatively stronger; leads to unstable subtalar joint and more falling due to “twisting of the ankle,”​ although most falling is due to weakness in quadriceps and “knee buckling”

Later stages • Weakness continues to progress in muscles • Before loss of ambulation, most compensations • Accelerated development of LE contractures listed above and becomes profound are used to maintain an upright posture and facilitate ambulation • UE weakness becomes more significant • After loss of ambulation, compensatory • Beginning development of UE contractures functionally and is imbalanced movements are primarily used to do the following: • Elbow extension weaker than flexion • Achieve support and stability in sitting • Tightness into elbow flexion and pronation • Forearm supination weaker than pronation • Assist UE function • Tightness in wrist and finger flexors and/or extensors, lumbricals, other intrinsic hand musculature • Wrist and finger extension weaker than • Compensatory movements include • Increased potential for deformity, such as swan flexion neck deformity • Leaning for stability

Downloaded from www.aappublications.org/news by guest on September 29, 2021 PEDIATRICS Volume 142, number s2, October 2018 S19 TABLE 1 Continued Weakness Compensations Risk of Tightness (2-joint muscles get tight first) • Distal hand function is relatively preserved at • Contralateral trunk leaning during UE least in long flexors but may be functionally function to substitute for shoulder girdle compromised by lack of proximal stability (deltoid) weakness in arm lifting and/or scoliosis requiring use of hands for sitting stability • Backward leaning and/or lurching to • Scoliosis: the development of scoliosis is a compensate for deltoid weakness in forward complication of the late or nonambulatory flexion and biceps weakness in elbow flexion stage, with natural history changing (decreasing scoliosis) with steroids but still requiring conservative preventative management with optimal wheelchair seating system support • Neck extensors, hamstrings, posterior • Leading with head (especially using neck • Hypoextensibility in cervical (neck) spinal extensors tibialis are relatively spared until later in extensors) to shift weight and compensate and rotator the disease for weak trunk musculature; using mouth to grab fingers and move arm to substitute for proximal UE musculature; pivoting forearm on elbow to substitute for elbow flexors • Decreased cervical rotation and lateral flexion With weakness and compensation, there is often no way to eliminate a compensatory position or pattern of movement without eliminating the function (ambulation and UE function), but intervention is used to prevent contractures that result from compensations, allowing compensations to be used to optimize function without leading to contracture that contributes to the self-perpetuating evolution of weakness, contracture, and/or functional loss that characterize DMD. Understanding the pathokinesiology below is required for optimal management.11,​ ‍14,‍20,​ ‍24,​ ​30

TABLE 2 Muscles and Structures Requiring Preventive Stretching LEs UEs joints and muscle groups known to be Stretching into isolated hip extension Elbow flexors at risk for hypoextensibility and those Stretching into isolated knee extension Forearm pronators Hip flexors Long wrist and finger flexors identified2 via assessment (Tables 1 Iliotibial bands Finger extensors and 2). When stretching, providers Hamstrings Lumbricals should include manual therapy Plantar flexors (with knee flexed and extended) Thumb adductors techniques, avoid the elicitation of Posterior tibialis Palm musculature Plantar fascia Shoulder musculature pain, and establish a preventative Toe flexors stretching program before decreases Other in passive ROM occur, with direct Neck extensors PT and OT accompanied by support Rib cage mobility and expansion of optimal positioning and the use of Any other structures identified by assessment splinting and orthotic intervention, custom seating systems, supported standing, and adaptive equipment. Preventive stretching of lower contracture and– – deformity– – and multiple coordinated interventions, extremities (LEs) should be initiated should be a component1 3,7​ 9,20​ 22,24​ of31 care including active and/or active- early in the ambulatory phase and at all stages. ‍‍ ‍ ‍ ‍ ‍‍ ‍‍ Joint assisted elongation; daily passive continued through adulthood. contractures and diminished muscle stretching of joints, muscles, and soft Preventive stretching of upper extensibility in DMD result from tissues at risk for tightness and those extremities (UEs) and the neck interacting factors, including a identified via assessment; prolonged becomes increasingly important in lack of full active joint ROM, static elongation; and support of optimal nonambulatory stages and throughout positioning, and imbalanced 2 positioning throughout the day with adulthood. Increased risk areas for muscle weakness across joints. splinting, orthotic intervention, contracture and deformity in adults Fibrotic changes in the muscle standing devices,– – custom– seating– in– with DMD (Tables 1 and 2) necessitate begin with early fibrosis in the mobility devices,1 3,7​ 13, 15,​and16,​ 20​ adaptive22,24​ 31,33,​ 110​ 112 detailed assessment and intervention newborn period, revealing the PTequipment and OT .Interventions‍ ‍ ‍‍ ‍ ‍ ‍‍ for‍ ‍‍ ‍ ‍ ‍ over time. The maintenance of Musculoskeletal Management importance of early– preventive– – chest wall mobility is important in management9, of13​ muscle16,20​ 22, flexibility24​ 31,85,​ 86,​ 108,​ and109​ respiratory management and the extensibility. ‍‍ ‍ ‍ ‍ ‍‍ ‍ ‍ ‍ prevention of scoliosis. Local care Effective maintenance of joint ROM, Stretching to prevent and minimize should be augmented– – by guidance– – muscle extensibility, and prevention contractures should be done a from specialists1 3,7,​ ​8,11​ every13,15,​ ​16,4 to20​ 22,24​ 31,33,​ 110​ of contracture and deformity requires minimum of 4 to 6 days per week for 6 months. ‍‍ ‍ ‍ ‍‍ ‍ ‍ ‍‍ ‍ ‍‍ ‍ ‍ Downloaded from www.aappublications.org/news by guest on September 29, 2021 S20 CASE et al FIGURE 1 Rehabilitation assessments and interventions across all disease stages for patients with DMD. a Areas typically at risk of contracture and deformity include hip flexors, iliotibial bands, hamstrings, plantar flexors, plantar fascia, elbow flexors, forearm pronators, long wrist and finger flexors and extensors, lumbricals, and cervical extensors; isolated joint contracture into hip and knee flexion and plantar flexion, varus at hindfoot and forefoot, elbow flexion, wrist flexion or extension, and finger joints; and deformity of the vertebral column and chest wall, including scoliosis, excessive kyphosis, lordosis, and decreased chest wall mobility. (Reproduced with permission from Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018.17[3]:257.)

Orthotic Intervention and Adaptive Orthoses Equipment for Musculoskeletal Management supported standing programs, and the use of adaptive equipment and assistive technology in addition to Resting or stretching ankle-foot – The prevention of contractures and manual –stretching programs– – orthoses (AFOs) are necessary, with nighttime use having been shown to deformity requires preventive splinting (see Supplemental1 3,7,​ 8,​ 13,​ 15,​ ​16, 20​Figs22, 224​ 11)31,108​ (Tables 4 and orthotic intervention, positioning, and 5). ‍ ‍ ‍ ‍ ‍ ‍ ‍‍ ‍ ‍‍ ‍ prevent and minimize progressive Downloaded from www.aappublications.org/news by guest on September 29, 2021 PEDIATRICS Volume 142, number s2, October 2018 S21 TABLE 3 Rehabilitation Assessments Standardized Functional Assessments Patient-Reported Outcomes and Patient-Reported Outcome Measures Disability Measures and Quality of Life Other Participation Scales North Star Ambulatory Assessment62,‍64,​ ‍65​ Pediatric Quality of Life Inventory75 Pain scales73 Timed functional tests58 10-m walk and/or run Pediatric Outcomes Data Collection Neuromuscular module Fatigue scales69 Instrument32 Supine to stand Canadian occupational Multidimensional fatigue scale Rate of perceived exertion87 performance measure78 4-stair climb Child health questionnaire79 NeuroQOL75 Borg Dyspnea Scale88 Sit to stand Pediatric Evaluation of Disability Health-related quality of life76 Activity monitoring60,‍89,​ ‍90​ Index80 Supine to sit Functional Independence Measure81 Questionnaire for neuromuscular DMD Upper Limb Patient-Reported diseases77 Outcome Measure91 6-min walk test58 Pediatric Functional Independence Life satisfaction index for Measure82 adolescents92,‍93​ Gait, stairs, Gower, chair66 School Function Assessment83 Children’s Assessment of Participation and Enjoyment and Preferences for Activities of Children94 Motor function measure95 Activities-Specific Scale for Kids96 Brooke Upper Extremity Scale97 Vignos Lower Extremity Scale97 Egen Klassifikation Scale98 9-hold peg test99 Jebsen-Taylor Hand Function Test100 Reachable work space101 Performance of upper limb102,‍103​ Bayley Scales of Infant Development, Third Edition (motor)104 Griffith Motor Scales104 Hammersmith Functional Motor Scale Extend (exploratory)104 Alberta Infant Motor Scale (exploratory)105 Gross Motor Function Measure (exploratory)106,‍107​ Impairment-level measures and/or assessments: passive ROM, muscle extensibility, strength, posture, and alignment. ADL Assessment includes ADL, instrumental ADL, access, adaptive equipment, and assistive technology. Falls Risk Assessment includes an assessment of learning, attention, and sensory processing. (Reproduced with permission from Birnkrant DJ, Bushby K, Bann CM et al; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, an update, part 1: Diagnosis, neuromuscular, rehabilitation, endocrine, and nutritional management [published correction appears in Lancet Neurol 2018;17(6):495]. Lancet Neurology 2018;17(3):251–267; supplemental appendix:2.)

plantarflexion contractures,– and wheelchair users to control plantar LE positioning in bed helps are 2,appropriate3,​ 7,​ 11,​ 13,​ 18,​ 24,​ 28​throughout31,109,​ 113​ varus if adequate medial-lateral minimize contracture. The use of life. ‍ ‍ ‍ ‍ ‍ ‍ ‍‍ ‍ ‍ AFOs positioning is maintained and resting hand splints, stretching should be custom molded, fabricated adequate dorsiflexion is supported by gloves, and oval-8 finger splints for comfort and optimum foot-ankle wheelchair footrests. Knee-ankle-foot is appropriate, continuing into alignment. They are typically best orthoses (KAFOs) (eg, long leg braces adulthood for stabilization, support, tolerated if started preventively or calipers) for supported standing, and musculoskeletal management. at young ages. Blanket lifter bars limited ambulation for therapeutic (Supplemental Fig 2). Standing Devices may ease bed mobility, increasing purposes, and the prevention of tolerance of nighttime AFO use. contracture and deformity– in late-

If nighttime tolerance cannot be ambulatory11,13,​ 20,​ and21,​ 27,​ early28,​ 44​ 54nonambulatory achieved, the use of stretching AFOs stages ‍ ‍ ‍ ‍ ‍ are evolving13 Supported standing devices during nonambulatory portions of and may not be tolerated at night. for individuals with no or mild the day is encouraged. Daytime AFO Adjustable knee extension splints LE contractures are necessary use can be appropriate for full-time can be considered for assistance in for late-ambulatory and early wheelchair users, extending into maintaining knee extension range nonambulatory stages, including adulthood. Lower-profile orthoses in nonambulatory individuals. standers and stand-and-drive may be considered for full-time Comfortable support of neutral motorized wheelchairs, extending Downloaded from www.aappublications.org/news by guest on September 29, 2021 S22 CASE et al TABLE 4 Orthotic Intervention, Splints, and/or Adaptive Equipment for Stretching • AFOs for stretching • Modified leaf-spring AFOs (lighter weight, less cumbersome, allow for some movement) for use at abduction and/or external rotation night or during daytime periods when not walking versus spinal extension). These • Articulating AFOs may offer more movement for increased tolerance at night, but can be bulkier differences present challenges • Adjustable-angle AFOs to try to gain increased range (a little bulkier) for use at night or during in wearing KAFOs, including an daytime periods when not walking increased risk of fracture as a result • Modular (3-piece) AFOs for use in combinations of night use and/or daytime use in nonambulatory stages of a higher risk of falls. Moreover, • Ankle height (supramalleolar during the day to maintain medial-lateral alignment if 90° technological options, including maintained by footrest) increasingly routine use of stand- • Taller AFO component to prevent plantar flexor tightness; may be rigid, flexible, or articulating and-drive motorized wheelchairs, (hinged) depending on need, tolerance, and preference may be more common than KAFOs • Inner liner for comfort (can be important in any AFO) to maintain supported standing • Serial casting • Knee extension splints mobility. However, reports of KAFO KAFOs (or long leg braces ) • “ ” use with glucocorticoids prolonging54 • Stander and/or stand-and-drive motorized wheelchair ambulation to even older ages,​ Hand and/or wrist splints to maintain length in long wrist and finger flexors • family satisfaction in spite of • Stretching gloves to maintain length in finger extensors 114 • Oval-8 finger splints to prevent hyperextension at proximal interphalangeal joint challenges,​ and situations in which KAFOs are the only means See also Supplemental Figs 2–11. of supported standing suggest that KAFOs continue to be an appropriate

– option in some contexts. Rapidly advancing technology in robotics benefits previously2,3,​ reported7,​ 8,​ 13,​ 16,​ 28,​ 44​with53,112 ​ if ambulation remains possible while the use of KAFOs. ‍ ‍ ‍ ‍ ‍ ‍ ‍ ‍ ‍ casted with sufficient quadriceps offers potentially improved future options. KAFO use should be viewed Supported standing for prolonged strength to avoid compromising55 passive elongation of LE musculature ambulation and losing function. as therapeutic rather than functional, should be considered when standing In nonambulatory individuals, with care taken to support safety to and walking in good alignment the risk of functional loss is less, minimize the risk of falls, and not used become difficult. The importance but cast weight may compromise exclusive of motorized mobility, which of initiating the preventive use transfers, necessitating lift use, is typically provided simultaneously of supported standing before and may be contraindicated with or earlier for safe, optimal, functional independence; mobility; and the development of contractures112 severe contractures. Skin integrity should be emphasized. Many and osteoporosis must also be Fallsparticipation and Fracture in all settings. Prevention advocate for the continued use of considered. An experienced team and Management supported standing devices and a is required for the successful use of powered stand-and-drive motorized serialKAFOs casting. wheelchair into late-nonambulatory Physical therapist collaboration stages and throughout adulthood with those in orthopedics to prevent if contractures do not limit falls and fractures and maintain positioning and if devices are The prolongation of ambulation or regain ambulation after a long tolerated. Motorized stand-and- for 2 to 4 years has historically bone fracture115 is increasingly drive wheelchairs obviate the need been reported with KAFOs, with emphasized. Extended ambulatory for transfers to use supported or without accompanying LE capacity, coupled with54 the risk of standing, decreasing the risk of falls surgery, and associated with low-trauma fractures,​ increases and increasing the number of hours decreases in scoliosis and LE the importance of fall prevention, per day of functional, comfortable, contracture, although with contextual including fall risk assessment supported standing (Supplemental qualifications, including individual, across settings, pool shoes for fall family, and team preferences, and prevention when walking on slippery FigSerial 5). Casting greater success– with experienced surfaces, and the early use of lift

teams and7,13,​ in28,​ the44​ 53 absence of and/or transfer equipment, especially 55,56​ obesity. ‍ ‍ ‍ ‍ ‍ Individuals with in bathrooms, where transfer Serial casting ‍ can be considered DMD, who are now walking longer challenges compromise safety. Rapid when stretching and orthotic use with glucocorticoids even without appropriate team management of have not maintained adequate ROM KAFOs, have differing height and long bone fractures with associated and/or when55,56​ surgery is not preferred body configurations at ambulation rehabilitation is essential and or chosen. ‍ Serial casting for loss (decreased height, increased may include the temporary use of ambulatory individuals is used only BMI, and increased relative hip assistive devices and other types of Downloaded from www.aappublications.org/news by guest on September 29, 2021 PEDIATRICS Volume 142, number s2, October 2018 S23 TABLE 5 Seating System, Power-Positioning Components, and Functional Components for Motorized Wheelchairs Components Purpose and Rationale Seating Sysytem Components • Solid seat with hip guides • To prevent pelvic asymmetry and obliquity; support a midline-level pelvic position to provide a level base of support in sitting for support of spinal symmetry; maintain symmetry of weight-bearing through ischial tuberosities for maintenance of skin integrity; and prevent pain • Pressure-relief cushion with adequate • For pressure relief, maintenance of skin integrity, prevention of pain, and to maximize sitting endurance stability over time while maintaining a level pelvis • Solid planar back with rigid lateral trunk • To maintain support at midline and maintain a midline, symmetrical, extended spinal alignment to supports prevent or minimize the development of scoliosis • Adductors • To support knees at midline and prevent positioning in excessive hip abduction and external rotation that leads to iliotibial band tightness; swing away, removable, and/or flip down to allow easier urinal use • Elbow supports • To maintain hand on joystick when driving, especially during power tilt and recline • Headrest • For safety and support of head control • Facial components on headrest (swing away) • To maintain midline head position and safety in the presence of decreased neck strength, decrease neck fatigue, and prevent neck pain • Gel overlays • Can be added to hip guides, trunk supports, adductors, arm rests, elbow supports, and headrest for pressure relief, maintenance of skin integrity, and tolerance of support • Mounts for smartphones and tablets • Access to support smartphone and tablets • Padded seatbelt • For safety and support of pelvic alignment Power-positioning components • Power tilt • To provide weight shift for the redistribution of pressure for pressure relief to maintain skin integrity (especially important if unable to shift weight independently); to allow resting intermittently to maximize functional endurance throughout the day • Power recline • To provide weight shift for the redistribution of pressure for pressure relief to maintain skin integrity, provide elongation of hip flexors for prevention and/or minimization of progressive hip flexor contractures, and allow resting intermittently to maximize functional endurance throughout the day • Power sliding (antishear) back • To maintain optimal positioning of seating system components, especially trunk supports, during position change with power-positioning components • Power-adjustable seat height • For functional access to the environment and to increase safety in transfers between surfaces • Separately elevating power-elevating leg • For elongation of hamstrings and assistance in preventing and/or minimizing progression of knee rests flexion contractures • Power stand and drive • To provide independent, supported standing intermittently throughout the day in optimal LE alignment with greater duration of standing than a separate stander can provide; minimize LE contracture and deformity; promote bone density, strength, and integrity and minimize the development of osteoporosis; provide support of function and/or ADL that are not possible in sitting; minimize risk of falls during supported standing by obviating the need for transfers to provide supported standing; and provide vibration during weight-bearing while driving in supported standing for increased bone density Functional components • Tray • For UE support and function • Swing-away and/or retractable joystick • Access • USB charger • For phone access for safety • Lights and blinkers • For safety in community mobility for participation • Group 4 base with high-speed package • Needed for safety on all terrain and crossing streets safely in community mobility • Bluetooth and infrared capabilities • For access and safety in environmental control and communication • Separate flip-up or swing-away footrests • Can be needed for safety in transfers • Aggressive tread-on tires • For safety and function on all terrains See also Supplemental Figs 2–11. USB, Universal Serial Bus.

Exercise and Activity Levels support to minimize the risk of loss of of contraction-induced– muscle – ambulation and prevent accelerated4,115​ injury and exercise-induced117 119,121,​ 123​ 126 contractureLearning, Attentional, and/or deformity and Sensory. ‍ Physical therapists prescribe, structural damage ‍ ‍ ‍ ‍ ‍ Processing Issues monitor, and guide exercise in related to strength,– – the duration

DMD on the basis of understanding of contraction,116 121, and123​ 126,the128,​ load131,​ 132​ potential effects of activity imposed. ‍ ‍ ‍ ‍ ‍ ‍ OT is important for early assessment and exercise on dystrophin- Other concerns include nitric oxide and intervention for learning, deficient muscle. Concern synthase dysfunction leading to 127 attentional, and sensory processing about exercise hastening– the increased ischemia during exercise issues, which are increasingly progression of weakness116 136 in DMD and cardiac concerns, including a lack understood as being important in is longstanding ‍ ‍ based on of correlation between skeletal57,137,​ and138​ DMD (Table 6). pathophysiology, including the risks cardiac muscle involvement ‍ Downloaded from www.aappublications.org/news by guest on September 29, 2021 S24 CASE et al TABLE 6 Areas for Specialized OT Assessment and Intervention Learning differences (specific differences aerobic-conditioning respiratory who appear likely to have a reduced in DMD are increasingly identified, exercise and support of gravity- exercise capacity even when including differences between verbal minimized movement, is highly functioning well. Individuals with and performance IQ and differences in recommended for the early DMD should have early referral to verbal memory, dyslexia, dyscalculia, and ambulatory through nonambulatory cardiology for the –identification– dysgraphia) Attentional issues stages and into adulthood2,3,​ 128,​ 132,​ as134​ long as and preventive management2 4,34​ 36,57,​ 142​ of Sensory processing issues it is medically safe. ‍ ‍ ‍ cardiomyopathyAssistive and/or. Adaptive‍ ‍ ‍ ‍ Devices‍ Fine motor Cycling is recommended as a for Function ADL submaximal aerobic form of activity, Computer access with benefits of assisted cycling Assistive technology Swallowing and oral motor dysfunction recently141 reported (Supplemental AFOs are not typically indicated Planning for academics and vocation in Fig 3). for use during ambulation because transition to adulthood Fibrosis (beginning in the newborn they tend to limit the compensatory movements needed for efficient period, before109 extensive cell necrosis),​ the proliferation of ambulation, add weight that can connective tissue, and increased compromise ambulation, and make with moderately to severely reduced stiffness, increases loads (resistance) it difficult to2, rise143​ from the floor and exercise capacity, as measured by against which muscles must work, climb stairs. ‍ During the late- cardiopulmonary exercise testing, further taxing contractile units ambulatory stage, KAFOs with locked even in the presence of normal or and potentially contributing to knees can prolong ambulation but mildly impaired139, 140​gross motor function ischemia and vascular and structural with use decreased, as described. and capacity. ‍ The balance impediments to regeneration. The During the early ambulatory stage, between beneficial versus harmful role of active movement, positive or lightweight manual mobility devices effects of muscle activity are not negative, on the fibrotic process and are appropriate for pushing the fully understood. Certain amounts vascularity is not clear. child on occasions when long- of muscle activity are assumed to Individualized assessment and distance mobility demands exceed be beneficial in preventing disuse monitoring of activity levels is endurance. In the late-ambulatory atrophy, maintaining residual important. Significant muscle pain stage and/or early nonambulatory strength, providing or maintaining or myoglobinuria in the 24-hour stages, an ultralightweight manual potential trophic influences period after a specific activity is a wheelchair with custom seating of active movement, and sign of overexertion and contraction- (Table 5) to support spinal symmetry maintaining functional status and 2,3,​ 128,​ 129,​ 132,​ 134​ induced injury, and if it occurs, the and LE alignment and swing-away flexibility,​ ‍ ‍ ‍ ‍ but eccentric 2 activity should be modified. footrests is necessary and may be muscle activity and maximal- or high- – – used in situations without access or resistance exercise are believed to be Cardiac concerns regarding 2,3,​ 123​ 126,128,​ 132​ 134 – transport for motorized mobility. detrimental ‍ ‍ ‍ ‍ ‍ ‍‍ and exercise include cardiomyopathy 2,4,​ 34​ 36,139,​ 142​ A variety of motorized mobility inappropriate across the life span and/or arrhythmias,​ ‍ ‍ ‍ ‍ ‍ 34 devices, including standing mobility because of potential contraction- abnormalities of calcium regulation,​ 34 devices, may be used intermittently induced muscle-fiber injury. Specifics and cardiac wall movement ; 34 34 for energy conservation and regarding the optimal type, frequency, fibrosis ; fatty infiltration ; – 138 independent long-distance mobility and intensity of exercise in DMD are not and conduction abnormalities. – 7,22,​ 33,​ 74,​ 116​ 136,139​ by individuals who continue to walk definitively known. ‍ ‍ ‍ ‍ ‍‍ These typically progress to dilated (Supplemental Figs 2 11). Participation in regular, gentle cardiomyopathy with arrhythmias,34 aerobic functional activity, such as including ventricular tachycardia. As functional community ambulation aquatics, cycling, and safe recreation- Early cardiac involvement can be becomes more difficult, a motorized based activity, is recommended by present even before139 overt clinical wheelchair is advocated. Custom some, especially early in the course of manifestations. Symptoms seating and power-positioning the disease, when residual strength may not present until cardiac components for the initial motorized is higher, with an emphasis on involvement is more advanced wheelchair are important standards moderation, lower-duration because of limited physical activity of care (Table 5), with power stand- activity, support of self-initiated and a lack of correlation between and-drive having been shown rests, and the avoidance– of cardiac and skeletal3,132,​ 134,​ muscle139​ to be used more successfully if overexertion2,3,​ 7,​ and74,​ 116​ overwork136,139​ involvement,​ ‍ ‍ ‍ supporting initiated before the development112 weakness. ‍ ‍ ‍ ‍ ‍ Aquatics, caution in prescribing exercise for of contracture and deformity. with potential benefits for individuals with dystrophinopathies, Custom seating for safety, support of Downloaded from www.aappublications.org/news by guest on September 29, 2021 PEDIATRICS Volume 142, number s2, October 2018 S25 ≥ optimal posture, and the prevention Upper Extremity Scale score 2), driverless cars promise ongoing of contracture and deformity robotics, miniature-proportional increases in independence. includes a solid seat with hip guides joysticks, microswitches, The early development of and a pressure relief cushion; a solid Bluetooth capabilities, software independent decision-making planar back with rigid lateral trunk and applications for computers, and respectful and responsible supports; flip-down, swing-away, fall detection systems with delegation skills in children with or removable knee adductors; and built-in Global Positioning System DMD, supported by the use of AT, a headrest. Facial components on detection, voice activation and “ self-advocacy skills, the hiring and headrests may be needed in older texting systems on smartphones ” directing of aides, the use of service individuals for adequate head and tablets, and smart home animals, and social participation support and symmetry. Swing- systems that are able to interface 2 foster transitions to independent away or flip-up footrests facilitate with motorized wheelchairs. Key adult function. transfers. Power-positioning pinch, microswitches, mouth call components include power tilt and buttons, and monitoring systems Funding for the support of AT and recline, power stand-and-drive, are considered if hand or voice support services is critical, as is the power-adjustable seat height, and weakness precludes the use of education of families about funding separately elevating power-elevating standard call buttons. Adaptive opportunities, including private leg rests. Elbow supports may be equipment for the support of ADL, insurance, Medicaid, Medicaid needed for UE support that keeps the safety, and the maintenance of skin Waiver programs, Medicare, hand on the joystick during position integrity include power-adjustable Independent Living, Vocational changes and on rough terrain. beds with pressure-relieving Rehabilitation, Supplemental Retractable, swing-away joystick mattresses; bathing and toileting Security Income, Achieving a Better hardware and trays may be needed equipment; lift and/or transfer Life Experience accounts, and for access. Lights and blinkers devices, including hydraulic and other funding sources, which vary are required for safety in evening motorized patient lifts; ceiling depending on the country, state, and community mobility. Ventilator lifts (hoists); slide sheets; and local environment. – holders are needed for those using environmental control options – ventilatory support (Supplemental (Supplemental Figs 2 11). Pain Management Figs 2 11). Assistive Technology AT assessment and intervention are focused on optimizing function and Pain, which is considered the fifth participation across the life span, vital sign, is important to assess which are particularly important – Referral to assistive technology in all individuals with DMD across during transitions between 67 73 (AT) specialists should be the life span. ‍ ‍ A more uniform functional levels and transitioning considered whenever function, assessment of pain, however, into adulthood. independence, and participation does not always lead to successful are compromised, and is necessary Extended ambulation, the management of pain, which is an when UE weakness affects importance of energy conservation important priority in transitions reach, fine motor skills, and for muscle preservation to adulthood and the 72management ADL. AT is helpful in optimizing and function, and increased of DMD in adulthood. Pain of fine motor skills, enhancing independence into adulthood varying types and intensities strategies for independence in require expanded technology may occur in DMD. Effective pain ADL, and improving access to for mobility, a greater variety of management requires an accurate alternative computer, mobility, or choices for different situations, and determination of the cause and environmental control (Table 7). increased AT options for mobility, may require comprehensive team Simple adaptations supporting UE driving, and community access that management. Postural correction, function include elevated lap trays are instrumental in participation, orthotic intervention, PT, adaptive and/or desks, adaptive straws, a employment, and avoidance of equipment, assistive technology, and hands-free water pouch, and/or social isolation. Vehicle adaptations pharmacological interventions may turntables if the hand cannot be increase options for community all be required. Adaptive equipment brought to the mouth or if biceps access, and adapted controls may and assistive technology should be strength is <3 in 5. More advanced allow for independent vehicle used to emphasize the prevention AT options include motorized driving. Skill development via and management of pain and and nonmotorized mobile arm accessible public transportation, optimize comfortable function and supports (considered at Brooke OT driving evaluation, and future movement with transfer, bathing, Downloaded from www.aappublications.org/news by guest on September 29, 2021 S26 CASE et al TABLE 7 Adaptive Equipment and Assistive Technology Equipment and Technology Purpose and Rationale • Adapted and/or assisted cycling: adapted tricycles (with stand for indoor • To provide supported submaximal activity and movement in UE and LE cycling use in inclement weather); power-assisted bicycles and/or tricycles; free- standing (stationary) power-assisted cycling • Lifts • For safe functional transfers in all settings; may need >1 piece of equipment for different settings • Stand-and-raise lifts: manual or motorized • Require reasonably good ankle ROM and tolerance of supported weight- bearing. Allow easier clean-up and clothing adjustment after toileting • Free-standing lift: motorized versus hydraulic with crank; can be portable • Portable and motorized offer most options for safe functional transfers in numerous settings; free standing lifts do not provide transfer into tub or to surface if cannot get legs of lift under object to which being transferred • Ceiling lifts • Do not take up any floor space, can be used throughout house and between rooms; can transfer down into tub and out • Wall-mounted lifts • Do not take up floor space; may be used if cannot use ceiling mount • Free-standing frames for “ceiling” lifts • If ceiling does not accommodate ceiling-mounted lift • Bath, shower, and/or commode chairs • For safe support during bathing, tub and/or shower transfers, hygiene; typically used with handheld shower • Tub benches padded with back support • Can go in tub • Hydraulic bath seats that descend into water • Can go in tub; descend into water for soaking, may be useful before stretching • Slider bath chairs • Can go in tub; provides mechanical sliding transfer • Roll-in shower chairs with tilt and seating system support • Roll-in shower required • Modified toilets: height, armrests, lift, and bidet • Safety, hygiene • Modified motorized beds, pressure-relieving mattresses, lateral rotation • Pressure relief, maintenance of skin integrity, function, prevention and/or mattresses and/or beds for position change and weight shift throughout minimization of pain the night • Stair lifts • Access, safety, energy conservation, protection of muscle • Platform and/or porch lifts • Access, safety, energy conservation, protection of muscles • Ramps: fixed, modular, portable, foldable, and threshold • Access, safety, energy conservation, protection of muscles • Modified vehicles with lifts, adapted controls • Access, safe transport, participation, independent driving • Adapted sports equipment • Safety, activity, participation • Smart home systems, Bluetooth, infrared • Environmental control, safety, independence • Evacuation chairs for school and/or work fire evacuation • Safety, access • Fine motor and communication: pencil grips, ergonomic pens and pencils; • Continuum of support in the presence of weakness, to decrease fatigue, “smart” pens, phones, tablets; computer adaptations, access; voice increase efficiency of function, support independence activation systems, call buttons; eye gaze systems • Mobile arm supports • Continuum of support in the presence of weakness, to decrease fatigue, increase efficiency of function, support independence • Robotics • Continuum of support in the presence of weakness, to decrease fatigue, increase efficiency of function, support independence

•• and toileting equipment. Power- especially if the patient is receiving plans for when stairclimbing •• positioning components offering glucocorticoids,– is an indication for becomes difficult; positional support and change, careful assessment2 4,144,​ 145​ for vertebral continuum of options for weight shift, and pressure relief on fractures. ‍ ‍ ‍ energy conservation and motorized wheelchairs and beds KEY ANTICIPATORY DISCUSSIONS FOR safe, functional, independent can be used as needed to maintain REHABILITATION mobility and participation in all skin integrity and pain prevention or •• settings; relief. Providers of pharmacological fall risk assessment and interventions must consider possible •• interactions with other medications Key anticipatory discussions should prevention; •• (eg, steroids and nonsteroidal occur before a time of crisis in each fracture management; anti-inflammatory drugs) and side of the following areas. Care team •• initiating supported standing; effects, particularly those that members can reassure the patient transfers and/or access at home, might negatively affect cardiac and family that rather than being at school, at work, and in the or respiratory function. Rarely, evidence of disease progression, •• community; orthopedic intervention might be these issues provide opportunities •• indicated for intractable pain that to discuss options for optimizing consideration of KAFO use; is amenable to surgery. Back pain, management: spine surgery; Downloaded from www.aappublications.org/news by guest on September 29, 2021 PEDIATRICS Volume 142, number s2, October 2018 S27 •• ABBREVIATIONS strategies for optimizing ADL and muscle recovery, optimizing and self-feeding compromised potential benefit versus damage by UE weakness or spine from specific types, durations, and ADL: activities of daily living •• fusion; and frequency of exercise. Robotics AFO: ankle-foot orthosis AT: assistive technology noninvasive ventilation and and AT advances are anticipated DMD: Duchenne muscular dystrophy tracheostomy. to provide ever increasing CONCLUSIONS functional independence, capacity KAFO: knee-ankle-foot orthosis for participation, and successful LE: lower extremity musculoskeletal management, and OT: occupational therapy As disease-modifying treatments it will be important to explore ways PT: physical therapy become available, questions that these developments can be ROM: ranges of motion will emerge regarding potential used to benefit individuals with UE: upper extremity increases in exercise capacity DMD. site principal investigator on several clinical trials sponsored by both Sarepta Therapeutics and PTC Therapeutics (funding is granted to her institution); the other authors have indicated they have no financial relationships relevant to this article to disclose. FUNDING: Supported in part by Cooperative Agreement, NU38OT000167, funded by the Centers for Disease Control and Prevention. POTENTIAL CONFLICT OF INTEREST: The authors have indicated they have no potential conflicts of interest to disclose.

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Downloaded from www.aappublications.org/news by guest on September 29, 2021 Rehabilitation Management of the Patient With Duchenne Muscular Dystrophy Laura E. Case, Susan D. Apkon, Michelle Eagle, Ann Gulyas, Laura Juel, Dennis Matthews, Robbin A. Newton and Helen F. Posselt Pediatrics 2018;142;S17 DOI: 10.1542/peds.2018-0333D

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