Effect of Dalfampridine in Patients With Hereditary Spastic Paraplegia

• Dalfampridine 10 MG (Drug): The participants in the experimental group received dalfampridine administered as 10 mg extended-release tablets every twelve hours for 8 weeks.
• Placebo (Drug): Control group received a placebo drug with the same administration method (2 times per week for the total duration of 8 weeks).
• Physiotherapy (Behavioral): Conventional physiotherapy program including stretching and flexibility, strengthening, walking and balance exercises which were mainly focused on the lower limbs and improving walking. The program was applied 2 times per week for the total duration of 8 weeks.

Primary outcomes

• Timed 25-foot Walk Test [Change from baseline to week 8]: The Timed 25-foot walk test (T25FW) is considered the "best characterized objective measure of walking disability and can be used across a wide range of walking disabilities". For the T25-FW, patients were instructed to walk as fast as they could in a safemanner along amarked 25-foot course. The time in seconds to complete each test was recorded, and the test was immediately repeated.

Secondary outcomes

• Sit to Stand Test [Change from baseline to week 8]: 30 second Chair-Stand Test which is a reliable and valid measure used to assess lower extremity strength and endurance is used where the number of sitting and getting up within 30 seconds gives the score of the test.
• Timed Up and Go test [Change from baseline to week 8]: Functional mobility was evaluated using the Timed up and Go Test 'TUG' test, which is also reliable and valid test for people with Parkinson's disease. (Morris2001; Van2016). Upon issuing the command "Go," the participants stood up from a normal chair, walked 3 meters, turned, walked back to the chair, and sat. The time began with the command "Go" and ended when the participants sat back to the chair. This test was repeated three times, and the shortest performance time was recorded
• Modified Ashworth Scale [Change from baseline to week 8]: Modified Ashworth Scale (MAS) is one of the reliable and valid methods to measure muscle spasticity. The procedure to evaluate specific muscle groups; passively moved through the range of motion of a limb, and the resistance encountered during muscle stretch is rated on a five-point scale. Ashworth defines this rating as; 0 = no increase in tone, 1 = slight increase in tone at the end of the range of motion, 1+ = slight increase in tone throughout less than half the range of motion, 2 = increased muscle tone throughout the full range of motion, but passive movement is present. 3=tone movement that makes passive movement difficult, 4=rigidity .
• Muscle Length Measurement [Change from baseline to week 8]: Bilateral muscle length measurements were obtained using a standard goniometer. The patient positioned in supine position; the dorsiflexion has been tested for the gastrocnemius, the straight leg rise - hip angle has been measured to assess hamstrings, the Thomas test was used to assess iliopsoas and hip abduction angle was used for adductor group muscles.

Sex: All

Inclusion Criteria:

* Diagnosis of Hereditary Spastic Paraplegia at least 1 year ago

Exclusion Criteria:

* Having another neurological disorder
* An orthopedic deformity in the lower extremity
* Having a serious cognitive impairment

• Results publication: Selcuk Muhtaroglu F, Belgen Kaygisiz B, Usar Incirli S, Kahraman T. Dalfampridine as a promising agent in the management of hereditary spastic paraplegia: A triple-blinded, randomized, placebo-controlled pilot trial. J Clin Neurosci. 2023 Nov;117:136-142. doi: 10.1016/j.jocn.2023.09.026. Epub 2023 Oct 5. PMID 37804674

• Ferda Selcuk, Principal investigator, European University of Lefke