Marta Banach

A phenotypic‐genetic study of a group of Polish patients with spinal and bulbar muscular atrophy

We studied phenotype‐genotype correlation in a group of Polish males with spinal and bulbar muscular atrophy (SBMA) and in female carriers. Eleven males with suspected SBMA phenotype and three suspected female carriers were examined. Male patients presented with the predominant signs of progressive, symmetrical distal limb weakness with amyotrophy, facial muscular weakness with orofacial fasciculations, nasal voice and slight dysphagia, gynaecomastia, decreased potency, as well as hand tremor and distal peripheral sensory disturbances in a few cases. One of the carriers presented with a 30‐year history of fasciculations and minimal distal weakness and cramps in the legs, while the other two were asymptomatic. DNA analysis revealed expanded size of CAG repeats in Xq11‐12 in the AR gene in 10 out of 11 men (range 45–52 CAG repeats) and in the women (range 46–48 CAG repeats). There was no correlation between CAG repeat size and the age of disease onset and duration of the disease. A rare, predominantly distal distribution of weakness and amyotrophy was found in our group of the SBMA patients (8 out of 11 cases) from three unrelated kindreds and also in the remaining two sporadic cases. The extended CAG repeats within families were stable.

Efficacy and Safety of Botulinum Type A Toxin in Adductor Spasticity Due to Multiple Sclerosis

Objective: To assess the efficacy of Clostridium botulinum type A toxin-hemagglutinin complex [BoNT-A, Dysport®] in the treatment of adults with adductor muscle spasticity due to definite or probable multiple sclerosis [MS]. Methods: In this 12-week multinational, randomized, double-blind, placebo-controlled trial, patients received a single treatment of either BoNT-A 1,000–1,500 Ipsen units or placebo injected into the adductor muscles of each leg [500–750 Ipsen units per leg]. The primary outcome measure was a novel, patient-selected, functional outcome measure at week 4. Results: Fifty-five subjects were treated with BoNT-A, and 51 received the placebo. Treatment benefit in favor of BoNT-A, which tended toward significance, was seen for most endpoints but not the subjective patient-selected key outcome measure. As with a previous dose-ranging study of BoNT-A, pain was significantly reduced in both legs [P < 0.05 at weeks 8 and 12]. Lack of significance for the other endpoints was probably due to the low number of patients who received the full 1,500 Ipsen unit dose of BoNT-A, which has previously been shown to be effective. The majority of adverse events were mild-to-moderate in severity and resolved within a few days. Conclusion: The BoNT-A was shown to provide effective pain relief in patients with severe adductor spasticity due to MS, and the trend toward treatment benefit for the primary endpoint is in line with previously published literature.

Efficacy and safety of abobotulinumtoxinA liquid formulation in cervical dystonia: A randomized-controlled trial.

Approved botulinum toxin A products require reconstitution. AbobotulinumtoxinA solution for injection is a ready‐to‐use liquid formulation of abobotulinumtoxinA.

Efficacy and safety of abobotulinumtoxinA in spastic lower limb: Randomized trial and extension

Objective: To demonstrate single abobotulinumtoxinA injection efficacy in lower limb vs placebo for adults with chronic hemiparesis and assess long-term safety and efficacy of repeated injections. Methods: In a multicenter, double-blind, randomized, placebo-controlled, single-cycle study followed by a 1-year open-label, multiple-cycle extension, adults ≥6 months after stroke/brain injury received one lower limb injection (abobotulinumtoxinA 1,000 U, abobotulinumtoxinA 1,500 U, placebo) followed by ≤4 open-label cycles (1,000, 1,500 U) at ≥12-week intervals. Efficacy measures included Modified Ashworth Scale (MAS) in gastrocnemius–soleus complex (GSC; double-blind primary endpoint), physician global assessment (PGA), and comfortable barefoot walking speed. Safety was the open-label primary endpoint. Results: After a single injection, mean (95% confidence interval) MAS GSC changes from baseline at week 4 (double-blind, n = 381) were as follows: −0.5 (−0.7 to −0.4) (placebo, n = 128), −0.6 (−0.8 to −0.5) (abobotulinumtoxinA 1,000 U, n = 125; p = 0.28 vs placebo), and −0.8 (−0.9 to −0.7) (abobotulinumtoxinA 1,500 U, n = 128; p = 0.009 vs placebo). Mean week 4 PGA scores were as follows: 0.7 (0.5, 0.9) (placebo), 0.9 (0.7, 1.1) (1,000 U; p = 0.067 vs placebo), and 0.9 (0.7, 1.1) (1,500 U; p = 0.067); walking speed was not significantly improved vs placebo. At cycle 4, week 4 (open-label), mean MAS GSC change reached −1.0. Incremental improvements in PGA and walking speed occurred across open-label cycles; by cycle 4, week 4, mean PGA was 1.9, and walking speed increased +25.3% (17.5, 33.2), with 16% of participants walking >0.8 m/s (associated with community mobility; 0% at baseline). Tolerability was good and consistent with the known abobotulinumtoxinA safety profile. Conclusions: In chronic hemiparesis, single abobotulinumtoxinA (Dysport Ipsen) administration reduced muscle tone. Repeated administration over a year was well-tolerated and improved walking speed and likelihood of achieving community ambulation. Clinicaltrial.gov identifiers: NCT01249404, NCT01251367. Classification of evidence: The double-blind phase of this study provides Class I evidence that for adults with chronic spastic hemiparesis, a single abobotulinumtoxinA injection reduces lower extremity muscle tone.

Efficacy and Safety of AbobotulinumtoxinA (Dysport) for the Treatment of Hemiparesis in Adults With Upper Limb Spasticity Previously Treated With Botulinum Toxin: Subanalysis From a Phase 3 Randomized Controlled Trial

To assess the efficacy and safety of abobotulinumtoxinA in adults with upper limb spasticity previously treated with botulinum toxin A (BoNT‐A).

The Polish version of the Michigan Hand Outcomes Questionnaire: Cross-cultural adaptation, reliability, construct validity, and measurement error:

The aims of this study were to translate the Michigan Hand Outcomes Questionnaire into the Polish language and to test the measurement properties of its quality criteria. A total of 120 patients with hand complaints completed the Polish Michigan Hand Outcomes Questionnaire and the Disabilities of the Arm, Shoulder, and Hand questionnaire on the first assessment, along with the grip test, pinch test, and pain sore assessed using a visual analogue scale during activity. After 7 days, 76 patients completed the Michigan Hand Outcomes Questionnaire the second time. The Cronbach alpha of the Michigan Hand Outcomes Questionnaire subscales ranged from 0.79 to 0.96. The intraclass correlation coefficient varied from 0.82–0.97, and the Bland–Altman method indicated the Michigan Hand Outcomes Questionnaire total score limit of agreement was −13.2–12.3 and −9.18–9.62 for the right and left hand, respectively. The construct validity revealed a moderate to strong correlation between every subscale of the Polish Michigan Hand Outcomes Questionnaire and Disabilities of the Arm, Shoulder, and Hand, but they only correlated with the grip test and the visual analogue scale, and neither correlated with the pinch test. The study demonstrated properties similar to the original version, validating the belief that the use of this questionnaire in medical practice in Poland is justified.

Polish version of the Patient-Rated Ulnar Nerve Evaluation in preoperative patients: Translation and psychometric testing

Study Design: Cross‐sectional design. Introduction: This study examined the translated English to Polish version of the Patient‐Rated Ulnar Nerve Evaluation (PRUNE) for its internal consistency, test‐retest reliability, and construct validity. Methods: During the first assessment validity testing, a total of 39 consecutive patients with cubital tunnel syndrome completed the PRUNE, Michigan Hand Outcome Questionnaire, Disabilities of the Arm, Shoulder, and Hand questionnaire, and Patient Evaluation Measure in conjunction with the grip and key pinch tests and pain score (by Visual Analogue Scale). Cronbachs alpha (CA), intraclass correlation coefficient (ICC), and the Bland‐Altman plot were used to evaluate internal consistency, test‐retest reliability, and agreement, respectively. Analysis of variance compared the PRUNE score with the McGowan clinical stages. Results: After a 1‐day interval, 19 patients completed the PRUNE for the second time. The total PRUNE score was 44.4 ± 20.4, CA = 0.93, and ICC = 0.921. The total PRUNE score limits of agreement varied from −9.87 to 7.55 points. PRUNE subscale CA ranged from 0.79 to 092; the ICC varied from 0.738 to 0.911. The construct validity revealed a strong association with Michigan Hand Outcome Questionnaire (R = −0.83; P < .000), and moderate with Disabilities of the Arm, Shoulder, and Hand (R = 0.75; P < .000), Patient Evaluation Measure (R = 0.75; P < .000), and Visual Analogue Scale (R = 0.69; P < .000). The grip and pinch tests had low and no correlation with the total PRUNE score, respectively. Conclusion: The Polish version of PRUNE showed good psychometric properties for use in both clinical and research practice in patients with cubital tunnel syndrome of varying intensity.

Poster 292 Improvement of Spasticity Following AbobotulinumtoxinA (Dysport®) Injections in Shoulder Muscles in Hemiparetic Patients with Upper Limb Spasticity–Sub-Analysis of a Prospective, Long-Term, Open-Label Study with Single and Repeated Injection Cycles

Participants: A total of 134 patients (abobotulinumtoxinA, n1⁄489; placebo, n1⁄445) were randomized and 129 (abobotulinumtoxinA, n1⁄484; placebo, n1⁄445) completed the W4 primary endpoint evaluation. Interventions: CD patients were randomized (2:1) to abobotulinumtoxinA or placebo. Toxin-naı̈ve abobotulinumtoxinA patients received 500 units/2 mL in 2 affected neck muscles. AbobotulinumtoxinA CD subjects who had previously received botulinum treatment (non-naı̈ve) received 250-500 units/2 mL (2.5:1 abobotulinumtoxinA: previous onabotulinumtoxinA [Botox ] dose) into muscles injected during prior treatments. Main Outcome Measures: The primary endpoint was change from baseline to Week 4 (W4) in Toronto Western Spasmodic Torticollis Rating Oasis, The Online Abstract Submission System Scale (TWSTRS) total score. Safety was assessed over the 12-week study period. Results: Versus placebo, abobotulinumtoxinA patients experienced significantly greater changes from baseline in TWSTRS score at W4 (-2.5 versus -10.8, P<.001; based upon the modified intent-to-treat population). Adverse events (AEs) occurred in 41% and 22% of abobotulinumtoxinA and placebo patients, respectively. Dysphagia was reported in 9% of treated patients. Other AEs in treated patients were muscle weakness, neck pain, and headache, none of which were reported with placebo. Conclusions: Data from this study indicate a 2 mL dilution of abobotulinumtoxinA was significantly more effective than placebo in CD patients. No unexpected AEs were observed relative to previous studies that used the 1 mL dilution volume in this patient population. Level of Evidence: Level II