Journal of the Neurological Sciences, 1987, 81:155-158 155

Elsevier

JNS 02885

Life spans of Duchenne muscular dystrophy

patients in the hospital care program in Japan
Masakuni Mukoyama ~, Kiyotaro Kondo 2, Kazuo Hizawa 3,
Hiroshi Nishitani 4 and the D M D R Group*
1National Institute of Neuroscience, NCPN, Ogawahigashicho, Kodaira, Tokyo (Japan), 2Department of Public
Health, Hokkaido University School of Medicine, Sapporo (Japan), 3Department of Pathology, School of
Medicine, Tokushima University, Tokushima (Japan), and 4Utano National Hospital, Kyoto (Japan)
(Received 27 March, 1987)
(Revised, received 25 May, 1987)
(Accepted 26 May, 1987)

SUMMARY

Analysis of 176 autopsy cases of Duchenne muscular dystrophy (DMD)

demonstrated that (1)hospitalized patients showed longer life spans than their non-
hospitalized affected maternal uncles, (2)patients hospitalized recently rived longer
than those hospitalized in the past, and (3)pulmonary infection has become a less
frequent cause of death in recent years, whereas dystrophic changes of the cardiac and
respiratory muscles are more closely related with recent fatal cases. These results
indicate the changing life span expectancy of patients with DMD and the changes in
cause of death over the last decade, probably owing to the benefits of a hospital care
program.

Key words: Duchenne muscular dystrophy; Life span; Cause of death; Hospital care

* The Duchenne Muscular Dystrophy Research Group consists of Ryoji Minami, Yakumo National
Sanatorium (NS), Yoshimi Akimoto, Iwaki NS, Gen Sato, Nishitaga NS, Tadayuki Ishihara, Higashisai-
tama NS, Imaharu Nakano, Shimoshizu NS, Isao Matsutani, Ioh NS, Mitsuo Iida, Suzuka NS, Hitoshi
Kameo, Hara NS, Yutaka Matsuka, Tokushima NS, Hiroshi Iwashita, ChikugoNS, and Noritoshi Shibuya.,
Kawatana NS, Japan.
Correspondence to: Dr. Masakuni Mukoyama, National Institute of Neuroseience, NCPN, 4-1-1,
Ogawahigashicho, Kodalra, Tokyo 187, Japan.

0022-510X/87/$03.50 © 1987 Elsevier Science Publishers B.V. (Biomedical Division)

156

INTRODUCTION

Patients with Duchenne muscular dystrophy (DMD) usually die in their late
adolescence, but statistical data on their life spans are scarce (Walton and Nattrass
1954; Emery and Skinner 1976). In 1964, the Japanese Ministry of Health and Welfare
established a national program which provides life-long institutionalized care, rehabili-
tation and schooling to young patients with various muscle diseases, without charge.
At present a total of 27 national sanatoria have special wards with a total of 2500 beds
for this program. Of an estimated total 2400 patients of D M D that survive in the
country, about half are currently hospitalized under this program (Kondo 1980). It is
our impression that the program is useful in providing high quality care, but to date, there
is no available information whether such efforts actually prolonged life spans of the
patients. In this study, we compared the life spans of D M D patients who were
hospitalized under national program with their 37 affected maternal uncles who died
at home before the introduction of this program, representing natural course of the
disease.

MATERIALS AND METHODS

Patients in this series are the total of 176 fatal cases with D M D who died in these
wards in the period from 1970 to 1984, and for whom adequate records were available.
Diagnosis is based on clinical observation for several years until death, involving
repeated E M G and serum enzyme studies. Muscle biopsy was not routinely done but
carried out in many cases. Atypical cases were excluded, The Becker type and so-called
Fukuyama type congenital muscular dystrophy were not included.
A collaborative research group was organized in 1978 to register fatal cases of
D M D who died in these wards. Detailed case reports were collected which included
agonal courses and pathological data, along with antemortem information. The present
materials, therefore, were considered to represent random autopsies of unquestionable
D M D cases, since nearly all patients in Japan are sooner or later admitted to these
wards.
Ages of death of those who died during 1970-1974, were compared with the data
of those who died during 1980-1984, and 37 affected maternal uncles.

RESULTS

All patients were males. The age of death in the 176 patients (Fig. 1) ranged from
10 to 29 years with a mode at 18 years and a mean of 18.28 + 3.55 years. The curve
was unimodal. Nine patients lived longer than 26 years of age. The age of death in 48
patients who died between 1980 and 1984 ranged from 14 to 28 years and averaged
19.96 + 3.90 years, whereas 63 patients who died between 1970 and 1974 ranged from
10 to 29 years and averaged 17.81 + 3.68 years. The former group lived significantly
longer than the latter (P < 0.005).
Thirty-seven affected maternal uncles had a life span of 5 to 30 years with an
 157

C88e8
20,

15. 18.28 + 3.55 years

15.°" cases )

o
1"0 1"5 2"0 2"5
Years

Alile of death
Fig. 1. Ages of death in 176 hospitalized patients with Duchenne muscular dystrophy.

average of 17.24 + 4.69years. This was lower than that of total 176 patients
(0.05 < P < 0.1), and was significantly lower than that of recent hospitalized group who
died between 1980 and 1984 (P < 0.005).
Pulmonary infection was a more frequent cause of demise (Table 1) in the group
of patients who died during the period of 1970 to 1974 than in those who died during
1980 to 1984 (25.4~ vs. 8.3 ~o). Respiratory insufficiency as a cause of death was more
common in the latter group (37.5~ vs. 9.5~o).

DISCUSSION

The present study indicated that (1) hospitalized patients had longer average life
spans than their affected uncles who died without proper care before the setting up of
the national program, (2) patients hospitalized recently lived longer than those

TABLE 1
CAUSES OF DEATH OF PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY
Percentages given in parentheses.

Causes ofdeath Patients died during

1970 to 1974 1980 to 1984

Pneumonia and bronchopneumonia 16 (25.4) 4 (8.3)

Cardiac failure 19 (30.2) 19 (39.6)
Respiratory insufficiency 6 (9.5) 18 (37.5)
Cardiopulmonary dysfunction 11 (17.5) 1 (2.1)
Others 11 (17.5) 6 (12.5)

Total 63 (100) 48 (100)

158

hospitalized in the past, sooner after the introduction of the program, and (3) while
pulmonary infection has become a less frequent cause of death more recently, respiratory
insufficiency is a more common cause of death in current cases. These changes have
occurred in correlation with severe dystrophic alterations of the diaphragm and inter-
costal muscles which were observed at autopsy (Mukoyama et al. 1984). The data in
Table 1 summarize reports by the pathologists from the different institutes at the
different dates. Pathological descriptions were somewhat arbitrary, but we believe that
they indicated a shift in the cause of death in DMD patients over the last decade, and
were reflecting the benefits of the program, in which complications, especially infections,
were maximally eliminated, and dystrophic changes themselves more directly caused the
death of patients.
It is likely that recent prolongation of life is due to better treatments. Controlled
nutrition, prevention of kyphoscoliosis (Siegel 1973), and management of cardiopul-
monary complications (Mukoyama and Kohno 1979; Matsuo 1983) are among the
factors that have contributed to this improvement. Whether medications, rehabilitation
or other aspects of management influence the course of the disease remains unevaluated.

ACKNOWLEDGEMENTS

This work was supported by Grant No. 62-3 from the National Center of
Neurology and Psychiatry (NCNP) of the Ministry of Health and Welfare, Japan. The
authors thank Professor Thoru Yamada of the Iowa University Hospital for assistance
in the preparation of this paper.

REFERENCES

Emery, A. E. H. and R. Skinner (1976) Clinical studies in benign (Becker type) X-linked muscular dystrophy,
Clin. Genet., 10: 189-201.
Kondo, K. (1980) Genetics of Duchenne muscular dystrophy and care program in Japan, Adv. Neurol. Sci.
(Tokyo), 24: 693-701.
Matsuo, S. (1983) Respiratory failure and its prevention in Duchenne muscular dystrophy, IR YO (Official
publication of the national hospitals and sanatoria of Japan), 37:474-481.
Mukoyama, M. and K. Kohno (1979) Pathology of the heart in Duehenne type muscular dystrophy.
Comparison with ECG findings, Abstract 2nd International Child Neurology Congress, p. 158.
Mukoyama, M., K. Hizawa, K. Hayashi and I. Sobue (I 984) The life span, causes of death and pathological
findings of Duchenne muscular dystrophy. Analysis of 124 autopsy cases, Neurol. Med. (Tokyo), 20:
377-380.
Siegel, I.M. (1973) Scoliosis in muscular dystrophy. Some comments about diagnosis, observations on
prognosis, and suggestions for therapy, Clin. Orthopaed., 93: 235-238.
Walton, J. N. and F. J. Nattrass (1954) On the classification, natural history and treatment of the myopathies,
Brain, 77: 169-231.