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Duchenne muscular dystrophy: Is survival really unchanged?
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Correspondence Duchenne Muscular Dystrophy: Is Survival Really Unchanged? To the Editor: In 1852, Meryon described a 16-year-old boy with what was to become known as Duchenne muscular dystrophy (DMD) who died from acute respiratory failure during a febrile episode with "profuse secretion of mucus from the trachea and larynx." One hundred-fifty years later, most patients with progressive neuromuscular disorders still die prematurely because of failure to aid their respiratory muscles. This is true despite the fact that life for DMD patients has been reported prolonged by I>7 years by providing intermittent positive pressure ventilation (IPPV) via an indwelling tracheostomy tube [ 1,2], as well as by use of noninvasive respiratory muscle aids, methods almost invariably preferred by patients for safety, convenience, appearance, comfort, and general acceptability [3]. In 1979 Alexander et al. demonstrated an extension of meaningful survival by at least 4.3 years for 10 individuals with DMD by the use of body ventilators [1]. They also reported successful use of glossopharyngeal breathing for ventilator-free breathing time and decanulating a tracheostomized DMD patient for conversion to strictly noninvasive methods of ventilatory support, including daytime use of mouthpiece IPPV. Curran and Colbert reported up to 24-hour body ventilator use by 23 individuals with DMD for a 6 year prolongation of survival to over 25 years of age [1]. Many of their patients also complemented nocturnal negative pressure body ventilator use with daytime mouthpiece IPPV. Splaingard et al. likewise described a significant prolongation of survival by body ventilator use that included daytime use of the intermittent abdominal pressure ventilator for 34 individuals with muscular dystrophy or spinal muscular atrophy [ 1]. One-half of their patients initially required endotracheal intubation for ventilatory support and could not be weaned. Although body ventilators can be helpful, ventilatory support has been demonstrated to be superior and more conveniently provided by noninvasive IPPV methods [3]. Twenty articles demonstrating that nocturnal-only use of nasal IPPV can delay the need for tracheostomy have recently been reviewed [1], and we have reviewed our use of 24-hour noninvasive IPPV for DMD patients as a long-term alternative to tracheostomy [3]. While the invasive nature of tracheostomy IPPV and tracheal suctioning diminishes patients' quality of life, many of our DMD patients have required 24-hour noninvasive IPPV for over 20 years, and five have gone on to require 24-hour long-term support without ever being hospitalized.
166 PEDIATRIC NEUROLOGY Vol. 16 No. 2
Thus, despite the fact that survival can be extended by providing ventilatory assistance, Boland et al. [4], and others before them [5,6] declared that the prognosis for DMD remains unchanged, and that DMD remains "untreatable." This is particularly ironic since medical treatments, more costly and less beneficial for function and survival than ventilator use, are now being widely used for many patients with neuromuscular diseases. Physicians treating patients with neuromuscular diseases need to become more familiar with convenient, noninvasive methods to keep them healthy. Yuka Ishikawa Department of Pediatrics National Yakumo Hospital and Department of Pediatrics Sapporo University of Medicine Hokkaido, Japan John R. Bach Department of Physical Medicine and Rehabilitation University of Medicine and Dentistry of New Jersey (UMDNJ)-New Jersey Medical School Jerry Lewis Muscular Dystrophy Association Clinic UMDNJ-New Jersey Medical School Center for Ventilatory Management Alternatives University Hospital, Newark and Kessler Institute for Rehabilitation West Orange, NJ
References 1. Bach JR. Conventional approaches to managing neuromuscular ventilatory failure. In: Bach JR (ed). Pulmonary rehabilitation: The obstructive and paralytic conditions. Philadelphia: Hanley & Belfus, 1996:285-301. 2. Baydur A, Gilgoff I, Prentice W, Carlson M, Fischer DA. Decline in respiratory function and experience with long-term assisted ventilation in advanced Duchenne's muscular dystrophy. Chest 1990;97:884-9. 3. Bach JR. Prevention of morbidity and mortality with the use of physical medicine aids. In: Bach JR (ed). Pulmonary rehabilitation: The obstructive and paralytic conditions. Philadelphia: Hanley & Belfus, 1996:303-29. 4. Boland BJ, Silbert PL, Groover RV, Wollan PC, Silverstein MD. Skeletal, cardiac, and smooth muscle failure in Duchenne muscular dystrophy. Pediatr Neurol 1996;14:7-12. 5. Hyser CL, Mendell JR. Recent advances in Duchenne and Becker muscular dystrophy. Neurol Clin 1988;6:429-38. 6. Matsuoka Y, Sakai M, lida M, Takahashi A. Advance of disability and prognosis in Duchenne muscular dystrophy--a comparison between institutionalized care and home care. Clin Neurol 1989;29:1000-3.
© 1997 by Elsevier Science Inc. All rights reserved. PII S0887-8994(97)00001-5 • 0887-8994/97/$17.00
Correspondence Duchenne Muscular Dystrophy: Is Survival Really Unchanged? To the Editor: In 1852, Meryon described a 16-year-old boy with what was to become known as Duchenne muscular dystrophy (DMD) who died from acute respiratory failure during a febrile episode with "profuse secretion of mucus from the trachea and larynx." One hundred-fifty years later, most patients with progressive neuromuscular disorders still die prematurely because of failure to aid their respiratory muscles. This is true despite the fact that life for DMD patients has been reported prolonged by I>7 years by providing intermittent positive pressure ventilation (IPPV) via an indwelling tracheostomy tube [ 1,2], as well as by use of noninvasive respiratory muscle aids, methods almost invariably preferred by patients for safety, convenience, appearance, comfort, and general acceptability [3]. In 1979 Alexander et al. demonstrated an extension of meaningful survival by at least 4.3 years for 10 individuals with DMD by the use of body ventilators [1]. They also reported successful use of glossopharyngeal breathing for ventilator-free breathing time and decanulating a tracheostomized DMD patient for conversion to strictly noninvasive methods of ventilatory support, including daytime use of mouthpiece IPPV. Curran and Colbert reported up to 24-hour body ventilator use by 23 individuals with DMD for a 6 year prolongation of survival to over 25 years of age [1]. Many of their patients also complemented nocturnal negative pressure body ventilator use with daytime mouthpiece IPPV. Splaingard et al. likewise described a significant prolongation of survival by body ventilator use that included daytime use of the intermittent abdominal pressure ventilator for 34 individuals with muscular dystrophy or spinal muscular atrophy [ 1]. One-half of their patients initially required endotracheal intubation for ventilatory support and could not be weaned. Although body ventilators can be helpful, ventilatory support has been demonstrated to be superior and more conveniently provided by noninvasive IPPV methods [3]. Twenty articles demonstrating that nocturnal-only use of nasal IPPV can delay the need for tracheostomy have recently been reviewed [1], and we have reviewed our use of 24-hour noninvasive IPPV for DMD patients as a long-term alternative to tracheostomy [3]. While the invasive nature of tracheostomy IPPV and tracheal suctioning diminishes patients' quality of life, many of our DMD patients have required 24-hour noninvasive IPPV for over 20 years, and five have gone on to require 24-hour long-term support without ever being hospitalized.
166 PEDIATRIC NEUROLOGY Vol. 16 No. 2
Thus, despite the fact that survival can be extended by providing ventilatory assistance, Boland et al. [4], and others before them [5,6] declared that the prognosis for DMD remains unchanged, and that DMD remains "untreatable." This is particularly ironic since medical treatments, more costly and less beneficial for function and survival than ventilator use, are now being widely used for many patients with neuromuscular diseases. Physicians treating patients with neuromuscular diseases need to become more familiar with convenient, noninvasive methods to keep them healthy. Yuka Ishikawa Department of Pediatrics National Yakumo Hospital and Department of Pediatrics Sapporo University of Medicine Hokkaido, Japan John R. Bach Department of Physical Medicine and Rehabilitation University of Medicine and Dentistry of New Jersey (UMDNJ)-New Jersey Medical School Jerry Lewis Muscular Dystrophy Association Clinic UMDNJ-New Jersey Medical School Center for Ventilatory Management Alternatives University Hospital, Newark and Kessler Institute for Rehabilitation West Orange, NJ
References 1. Bach JR. Conventional approaches to managing neuromuscular ventilatory failure. In: Bach JR (ed). Pulmonary rehabilitation: The obstructive and paralytic conditions. Philadelphia: Hanley & Belfus, 1996:285-301. 2. Baydur A, Gilgoff I, Prentice W, Carlson M, Fischer DA. Decline in respiratory function and experience with long-term assisted ventilation in advanced Duchenne's muscular dystrophy. Chest 1990;97:884-9. 3. Bach JR. Prevention of morbidity and mortality with the use of physical medicine aids. In: Bach JR (ed). Pulmonary rehabilitation: The obstructive and paralytic conditions. Philadelphia: Hanley & Belfus, 1996:303-29. 4. Boland BJ, Silbert PL, Groover RV, Wollan PC, Silverstein MD. Skeletal, cardiac, and smooth muscle failure in Duchenne muscular dystrophy. Pediatr Neurol 1996;14:7-12. 5. Hyser CL, Mendell JR. Recent advances in Duchenne and Becker muscular dystrophy. Neurol Clin 1988;6:429-38. 6. Matsuoka Y, Sakai M, lida M, Takahashi A. Advance of disability and prognosis in Duchenne muscular dystrophy--a comparison between institutionalized care and home care. Clin Neurol 1989;29:1000-3.
© 1997 by Elsevier Science Inc. All rights reserved. PII S0887-8994(97)00001-5 • 0887-8994/97/$17.00