Clinical Trials

Several of the following announcements, designated AAN, are from the 60th annual meeting of the American Academy of Neurology, held in April in Chicago.

boys nor the investigators knew who was on which schedule. (Boys on the two-day prednisone schedule received “dummy” pills on the no-prednisone days.)

Effects on strength maintenance were similar in the two groups, but time required to get up from the floor was better in the daily prednisone group.

Growth retardation, a known prednisone side effect, was less severe in the two-day, high-dose prednisone group, but weight gain, another serious side effect, was the same in both groups after a year. AAN

 

Two days a week of the corticosteroid prednisone at a high dose appears to be almost as beneficial as a daily moderate dose of the drug in boys with Duchenne muscular dystrophy (DMD), and some side effects may be less severe, investigators reported.

The yearlong, multicenter study, supported by MDA and the National Institutes of Health, was conducted by Diana Escolar at Children’s National Medical Center in Washington, with colleagues at many institutions.

The investigators analyzed data from 64 boys with DMD who were 4 to 10 years old, had not previously taken corticosteroids, and were still walking.

The boys were randomly assigned to take prednisone at 0.75 milligrams per kilogram every day, or to take prednisone at 10 milligrams per kilogram per day two days a week. Neither the

Two studies of the drug pentoxifylline in

Duchenne muscular dystrohy (DMD)

have yielded somewhat disappointing results, investigators reported. It had been hoped that pentoxifylline might slow disease progression by increasing blood flow, reducing scar tissue formation and countering inflammation.

A multicenter study in the United States conducted by Diana Escolar at Children’s National Medical Center in Washington, with many others at different clinics, tested pentoxifylline for a year in 17 boys with DMD who were between 4 and 9 years old and who hadn’t taken corticosteroids (like prednisone). Of 17 patients initially enrolled, only nine completed the study. Strength measurements didn’t show a significant change during the study.

Five of the eight participants who withdrew did so because of intolerable side effects, such as nausea and vomiting and reduction of white blood cell numbers.

The investigators said the lack of deterioration in strength in a year’s time suggests a possible beneficial effect on disease progression and warrants further study with a different formulation of pentoxifylline. AAN

A separate DMD study conducted at centers in the United States, Italy, Canada, Israel, Australia

High-dose prednisone two days a week appears almost as effective in DMD as daily prednisone and may interfere less with children’s growth.

and Argentina tested the effect of pentoxifylline plus prednisone. Diana Escolar and many others tested this drug

Diana Escolar at ^combination Children’s National against prednisone Medical Center in plus a placebo Washington received _{in 64 boys with} MDA support for the compressed prednisone ^{DMD, 57 of} trial and was also an whom completed investigator on trials of the study. The aver- pentoxifylline in DMD. age age was 10.

Strength measurements at one year were not significantly different between the two groups, but the pentoxifylline-treated patients had more coagulation abnormalities, skin problems and gastrointestinal side effects.

The investigators concluded that pentoxifylline was well tolerated in this group but had no effect in slowing DMD disease progression compared to prednisone alone.

AAN

 

Investigators reported the age of onset of cardiac problems in boys with Becker muscular dystrophy (BMD) can be approximately predicted from the location and type of mutation in the gene for the muscle protein dystrophin.

They say their analysis of 126 BMD patients identified dystrophin mutations that predispose boys to cardiac muscle deterioration (cardiomyopathy). The team included John Kissel and Jerry Mendell, co-directors of the MDA clinic at Nationwide Children’s Hospital in Columbus, Ohio.

AAN

A research group that included Jerry Mendell and John Kissel, co-directors of the MDA clinic at Nationwide Children’s

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