PHILADELPHIA — A drug that aids walking in people with multiple sclerosis may have a similar benefit in those with a rare form of paraplegia, a researcher said here,
In a small, proof-of-concept study ofdalfampridine (Ampyra), 50% of patients with hereditary spastic paraplegia improved on several measures of walking ability, according to Nicolas Collongues, MD, PhD, of the University of Strasbourg Hospital in Strasbourg, France.
The improvements were clinically meaningful as well as statistically significant, Collongues reported at the annual meeting here of the American Academy of Neurology.
“Patients all say they can walk with more facility and can climb stairs more easily,” Collongues told MedPage Today. “This is a real clinical improvement.”
But because of the rarity of the disease — about three people per 100,000 — large clinical trials will be difficult to organize, which might rule out formal approval to use the drug in patients, Collongues said.
He added that he and colleagues are trying to organize a larger study, using a cross-over design since a standard parallel group design would be impossible.
On the other hand, the drug is approved for use in multiple sclerosis, which opens the door to off-label use in hereditary spastic paraplegia, commented Babar Khohkar, MD, of Yale University, who was not part of the study but who moderated a session at which it was presented.
Large studies are easy to do in multiple sclerosis, he told MedPage Today, but “in this population you are probably going to have to do studies” like the one by Collongues and colleagues.
But, he added, “if I had a patient, and I saw these data, I would now consider (dalfampridine) off-label.” The question, he noted, is whether insurers would pay for it.
Collongues and colleagues enrolled 12 patients with the condition and gave them the drug — at 10 milligrams twice a day — for 15 days.
Patients were 52, on average, and had been diagnosed with the disease for nearly 16 years.
Before and after the drug treatment, the researchers tested patients’ ability to walk using three measures — the Timed-25-Foot Walk Test, the Spastic Paraplegia Rating Scale, and the 12-item Multiple Sclerosis Walking Scale.
They defined a responder as a patient whose score improved by at last 20% on any of the three.
In fact, Collongues reported, six of the 12 showed significant improvement on all three scales. Specifically, the responders:
Had an average baseline score on the paraplegia scale of 21, which fell to 17.3 after treatment. The 20.7% difference was significant at P=0.03.
Had an average baseline time of the 25-foot walk of 17.5 seconds, which fell to 13.5. The 21.1% improvement was also significant at P=0.03.
Had an average baseline score on the MS test 69.5, which fell to 48.3 after treatment. The 31.6% improvement was significant, again, at P=0.03.
Collongues reported that the drug was well tolerated and would likely be without significant adverse effects for an even longer treatment period.
“If it is well tolerated for 15 days, it is well tolerated after,” he said.
The issue is important, he noted, because there is almost no carry-over effect: “If you stop the drug,” he said, “one day later there is no effect.”
Article Courtesy of http://www.medpagetoday.com/MeetingCoverage/AAN/45552
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