key: cord-1031380-lq4awi0i authors: Veerapandiyan, Aravindhan; Wagner, Kathryn R.; Apkon, Susan; McDonald, Craig M.; Mathews, Katherine D.; Parsons, Julie A.; Wong, Brenda L.; Eichinger, Katy; Shieh, Perry B.; Butterfield, Russell J.; Rao, Vamshi K.; Smith, Edward C.; Proud, Crystal M.; Connolly, Anne M.; Ciafaloni, Emma title: The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID‐19 pandemic date: 2020-05-05 journal: Muscle Nerve DOI: 10.1002/mus.26902 sha: a5ae762e9a68a3dad6da53548c6ca48a5e3673c1 doc_id: 1031380 cord_uid: lq4awi0i 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. ing, including avoiding public gatherings and public transport, limiting time in stores, and using remote technology platforms in place of in-person meetings and activities, such as use of telehealth for medical care if clinically appropriate. DBMD patients should continue their current treatments, and specifically should not discontinue existing medications, unless approved by their treating neurologist or neuromuscular specialist. We recommend that patients continue their current corticosteroid treatment. However, should they become ill, they should notify their neurologist or neuromuscular specialist, as their dose (amount or frequency) may need to be adjusted to prevent adrenal insufficiency. Stress dose corticosteroids should be considered in settings of acute sickness or hospitalization. 16 Patients and families should be aware of the risk of adrenal crisis during illness or with sudden cessation of steroid use, and they should discuss this with their health-care providers. Appropriate dosing of stress dose corticosteroids has been described in previous studies. 12, 16, 17 Consultation with an endocrinologist is recommended when steroid changes are being made during hospitalization. Vomiting is a rare symptom of COVID-19, but patients who cannot tolerate their regular doses of corticosteroids should seek medical attention for clinical assessment and parenteral steroid administration; alternatively, a family member should be prepared to deliver hydrocortisone intramuscularly. Exon-skipping agents, such as eteplirsen, golodirsen, and viltolarsen, are antisense oligonucleotides that restore expression of a shorter but functional dystrophin. These are given as intravenous infusions over 35 to 60 minutes, once a week. Patients who are receiving these exon-skipping agents are encouraged to continue their medications but should discuss with their neuromuscular specialist the risks vs benefits of continuing infusions during the pandemic. Home infusions should be considered as a potential measure to limit exposure to COVID-19. In the case of home infusions, or other home health care, it would be prudent to limit the number of visits to the necessary minimum, and ensure that providers wear appropriate personal protective equipment and are properly prescreened for symptoms of COVID-19. Many patients with DBMD are prescribed angiotensin-converting enzyme inhibitors or angiotensin receptor blockers for prophylaxis or treatment of cardiomyopathy. There has been some concern surrounding the use of these drugs due to the interplay of the SARS-CoV-2 virus and angiotensin-converting enzyme 2, which is a coreceptor for the virus. The American Heart Association, the American College of Cardiology, and the Heart Failure Society of America recently issued a joint statement that individuals should continue to take these medications in light of the known benefits to the heart and the uncertain risks of COVID-19. 18 We emphasize that treatment decisions should be individualized, and made jointly between the patient, family, and health-care provider, considering any geographic or institution specific policies and precautions for COVID-19. Patients should not be labeled as "terminal" and triaged for nontreatment simply on the basis of their disability and diagnosis. Comprehensive standard of care for patients with DBMD and other muscular dystrophies includes periodic assessments to monitor pulmonary, cardiac, and bone health, as well as side effects from medications or treatments. These can include blood work, echocardiograms, cardiac MRIs, pulmonary function tests, X-rays, and dualenergy X-ray absorptiometry for measuring bone mineral density. [11] [12] [13] Surging COVID-19 rates are placing a tremendous burden on healthcare systems, resulting in interruption of elective and/or nonemergent services and procedures. During this time, to enhance the safety of our patients, families, and medical staff, we recommend that standard practices be modified and individualized. Alternate options, such as home blood draws and home polysomnography, if clinically appropriate, should be considered to minimize exposure and risk. In some cases, delaying routine laboratory monitoring can be appropriate, but if management decisions necessitate acquisition of clinic-based laboratory studies, such as pulmonary function tests, these can be safely performed with appropriate personal protective equipment. We encourage continuation of comprehensive care using telemedicine, single-provider or multidisciplinary visits, for these patients. There has been widespread use of hydroxychloroquine for hospitalized COVID-19 patients. The efficacy of hydroxychloroquine against COVID-19 is unclear with some small uncontrolled studies suggesting benefit, and at least one controlled study showing no benefit. 21 There are potentially serious risks to skeletal and cardiac muscles. Hydroxychloroquine causes a vacuolar myopathy in a minority of patients 22, 23 and has also been associated with life-threatening cardiac arrhythmias. 24 Due to the uncertain benefits, and the potential risks to skeletal and cardiac muscle, hydroxychloroquine is not recommended for patients with DBMD. From a rehabilitation standpoint, the closures of schools and outpatient therapy facilities due to the COVID-19 pandemic has resulted in discontinuation of many therapy services. The clinical urgency of ongoing physical, occupational, and speech/language therapies should be evaluated on a case-by-case basis, and their suspension or continuation agreed upon by therapists, physicians, and patients. Tele-rehabilitation can be successfully implemented by therapy team members, but the ability to perform varies based on local regulatory and compliance requirements. We encourage families and caregivers to use home therapy regimens recommended by their therapy team, if time allows, understanding the impact of added burdens and responsibilities experienced by caregivers. Additional rehabilitation considerations include using intermittent bracing, such as resting ankle-foot orthoses/night splints or hand splints during planned daytime sedentary activities to help maintain a passive range of motion; increasing physical activity and ambulation in the home and neighborhood to prevent worsening contractures and disuse weakness; and therapeutic positioning, such as lying prone or standing with support to provide a passive stretch to the hip flexors, knee flexors, and ankle plantarflexors. 4, 25, 26 Orthotists and equipment specialists may have limited availability during a pandemic, which can limit necessary attention to bracing and equipment issues. The therapy team and physicians can work together to prioritize issues and recommend inperson vs video appointments or telephone calls with orthotists or equipment specialists. Children with DBMD have a spectrum of neurobehavioral manifestations, including intellectual disability, learning disabilities, anxiety, attention deficit hyperactivity, and autistic features. 27,28 DBMD patients and their family members are at increased risk of depression and anxiety. 13 Mental health effects can be compounded during this pandemic due to multiple factors, such as interrupted routines, school closures, and anxiety and fear about the situation, as well as limited availability or lack of mental health services. We strongly recommend that psychological care and behavioral support continue via telehealth. Numerous clinical trials investigating targeted treatments for DMD are currently in progress. The COVID-19 pandemic has impacted the conduct of clinical trials due to a variety of challenges such as quarantines, site closures, travel limitations, and interruptions in supply of investigational products. 29 These challenges affect adherence to protocol-specific procedures, protocol-mandated visits, and testing. Trial sponsors are actively pursuing alternate plans and telemedicine should be encouraged. Clinical trial sites have instituted policies pertaining to research conduct. We strongly recommend that considerations of participant and study staff safety remain the paramount concerns for any decisions regarding the need for in-person visits to the study site. Whenever possible, remote visits should replace in-person visits without compromising the collection of essen- Clinical characteristics of COVID-19 inchildren compared with adults in Shandong Province, China. 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