Paul J. Vignos

Steroid myopathy in connective tissue disease

In eight women with polymyositis (three patients), systemic lupus erythematosus (SLE) (three patients), rheumatoid arthritis (one patient) and shoulder-hand syndrome (one patient), weakness developed during high dose prednisone therapy. These women were studied using serial functional and manual muscle tests, determination of serum glutamic oxaloacetic transminase (SGOT), creatine phosphokinase (CPK) and serum aldolase levels, and urinary excretion of creatine. Insidious onset of weakness was characteristic. Myalgias were seen in five patients and unusual sudden weakness in two. Weakness was always most severe in the pelvic girdle muscles; there was a lesser involvement of shoulder girdle and distal muscles. Serum muscle enzyme levels were normal in all cases, but urinary creatine excretion was invariably increased and proved to be the most sensitive laboratory indicator for clinical diagnosis and for monitoring patient improvement. Serial urinary creatine excretion and serum enzyme studies were of value in differenting steroid myopathy from a flare of myositis in patients with connective tissue disease. Diagnosis and effective management were achieved by the use of readily available laboratory and clinical procedures without resorting to muscle biopsy.

Evaluation of a Program for Long-Term Treatment of Duchenne Muscular Dystrophy. Experience at the University Hospitals of Cleveland*

One hundred and forty-four boys who had Duchenne muscular dystrophy were managed at a single center between 1953 and 1994 and were followed for a mean of 8.9 years. The long duration of follow-up provided an opportunity to examine the effects of physical therapy and orthopaedic treatment on contractures of the lower extremities and on the duration of the ability to walk. Contractures of the lower extremities were controlled best when patients were managed with a combination of daily passive stretching exercises, prescribed periods of standing and walking, tenotomy of the Achilles tendon, posterior tibial-tendon transfer, and application of knee-ankle-foot orthoses. Approximately two years after bracing, the severity of the contracture of the heel cords was similar in the patients who had had an operation and those who had not. By the fourth year after bracing, however, the patients who had had an operation had less severe contractures than those who had had bracing alone. Five to seven years after the operation and bracing, control of contractures was still good, especially for the patients who had had posterior tibial-tendon transfer. Contracture of the knee was well controlled five to seven years after bracing in all patients who had had bracing, with or without an operation. The program enabled the patients who had been managed with bracing to walk until a mean age of 13.6 years. After loss of the ability to walk with bracing, the ability to stand continued for an additional two years with use of orthoses. The findings of the present study demonstrate the value of traditional methods of operative treatment and bracing for controlling contractures of the lower extremities in patients who have Duchenne muscular dystrophy and for prolonging their ability to walk.

Echocardiographic evaluation of left ventricular function in Duchenne's muscular dystrophy

Abstract Thirty-six patients with Duchennes muscular dystrophy were studied by echocardiography. The ejection fraction and percent shortening of left ventricular diameter (% ΔLVD) were significantly lower than that of an age-matched control group. The ejection fraction was less than 55 in 21 percent of the patients. There was no left ventricular enlargement, left ventricular hypertrophy or left atrial enlargement. Maximal diastolic endocardial velocity (MDEV) (105 ± 18 mm/sec) was lower than that in the control group (149 ±23 mm/sec); it was decreased in 90 percent of the patients and its severity showed poor correlation (r = 0.13) with abnormality in the ejection fraction. There was no correlation between abnormal MDEV and increased anterior forces on the electrocardiogram. Maximal systolic endocardial velocity (MSEV) was not significantly decreased. Electrocardiographic abnormalities were present in 92 percent of the patients. Patients were classified according to skeletal muscle functional capacity with group I patients having mild to moderate impairment and group II patients having severe disease with no significant difference between heart rate, ejection fraction, % ΔLVD, MDEV or MSEV of the two groups. Patients were subdivided into those less than 12 years old (group A); and those over 12 (group B). There was more severe skeletal muscle disability but no increased abnormality in parameters of left ventricular function in the older patients. Decreased MDEV is common in Duchennes muscular dystrophy but is a poor predictor of left ventricular dysfunction which occurs in the minority of patients and does not correlate with the severity of skeletal muscle weakness. Skeletal muscle disease progresses with age with no significant progression in left ventricular dysfunction.

Bracing for ambulation in childhood progressive muscular dystrophy.

1. Fifteen of seventeen patients with childhood muscular dystrophy who had ceased walking because of muscle weakness were able to walk independently again after correction of contractures and fitting of long lower-extremity braces. 2. The criterion for bracing is recent loss of the ability to walk independently or the ability to walk only with assistance in a patient who stands less than half an hour a day. 3. Contrary to previous opinion, there is enough residual muscle power for walking in braces when muscle weakness prevents independent walking. 4. When braces are needed to continue walking, the patient with muscular dystrophy has lost 60 per cent of his muscle mass. 5. It is usually necessary to correct heel-cord and iliotibial-band contractures surgically before application of braces. 6. The additional energy requirements for walking with braces have not caused any significant cardiovascular complications as far as can be determined through clinical observation aided by serial electrocardiograms and cardiac roentgenograms.

Assessment of Hand Function in Duchenne Muscular Dystrophy

Eighteen boys with Duchenne muscular dystrophy (DMD) were assessed for their ability to perform tasks involving wrist and hand function. Each subject was assessed using the Jebsen Test of Hand Function, range of motion measurements, and muscle strength tests. Writing and simulated page turning were performed successfully by boys in all age groups. Boys over age 15 had difficulty completing simulated feeding and picking up large and small objects. The muscle strength of the wrist extensors and the radial deviation range of motion at the wrist were found to be strongly correlated with six of the seven tasks assessed. These two clinical assessments appear to be good indicators of overall wrist and hand function. Life expectancy with DMD is increasing with advances in respiratory care making preservation of wrist and hand function, the major activity remaining with advanced disease, increasingly important.

Predicting the success of reambulation in patients with Duchenne muscular dystrophy.

We used biochemical and clinical variables to develop a method to predict the expected duration of independent walking following surgery and bracing in patients with Duchenne muscular dystrophy. Data from the records of fifty patients were analyzed by linear and multiple regression. The most useful factors, applied in combination, in predicting the duration of walking ability after bracing were: percentage of residual muscle strength, vital capacity, creatinine coefficient, motivation of the patient at the time of bracing, and decrease in creatinine coefficient in the two years prior to bracing. This system uses readily available variables to predict the response to bracing in patients with Duchenne muscular dystrophy. Improvement in the criteria for the selection of patients for surgery and bracing is important in view of the economic cost as well as the demands on the time and energy of these children and their parents.

The Excretion of the 3-Oxo-Conjugate of Aldosterone by Normal Adolescent Boys

Summary Excretion of the 3-oxo-conjugate of aldosterone by three of the ten normal adolescent boys studied was found to be substantially greater than that of normal adults. No correlation was observed between the values for urinary aldosterone and urinary sodium, nor between urinary aldosterone and urinary potassium for the group. A barely significant correlation was found between the values for urinary aldosterone and urinary potassium-sodium ratios.