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Objective: This exploratory analysis of a large, randomized, double-blind study (NCT02106351) describes the effect of treatment with abobotulinumtoxinA followed by a tailored home exercises therapy programme in enabling children with upper limb spasticity due to cerebral palsy to achieve their functional goals using goal attainment scaling (GAS). Methods: Children with cerebral palsy and spasticity in ≥ 1 upper limb received up to 4 injection cycles of abobotulinumtoxinA (2 U/kg (cycle 1 only), 8U/kg and 16U/kg) into the elbow and wrist flexors and other upper limb muscles selected to support individual treatment goals. Children followed a home exercises therapy programme, which included stretches and exercises specifically chosen to facilitate goal achievement and engagement in activities. Results: For cycle 1, most children had active function goals set as their primary goal (69.7% vs 19.2% passive function goals). GAS T- scores and goal responder rates at week 16 indicated that most types of primary goal were achieved at least as expected during cycle 1 (all groups). Primary goal GAS T-scores were generally maintained for the first 3 abobotulinumtoxinA treatment cycles. Conclusion: Most children with upper limb spasticity treated with repeat cycles of abobotulinumtoxinA supported by an individualized home exercises therapy programme achieved their functional goals. LAY ABSTRACT Treatment with botulinum toxins (such as abobotulinumtoxinA; aboBoNT-A) is often recommended to treat spasticity of the upper limb to help children with cerebral palsy improve their hand skills. In this large study, children with cerebral palsy were treated with up to 4 aboBoNT-A injection cycles followed by a home exercises programme that was specifically made for each child to help them meet their treatment goals. This study aimed to explore the types of goals the children wanted to achieve with this treatment and the factors that influence goal attainment. Most children in this study had “active function” goals set, including “involving the affected arm more in daily activities” and improving “reaching”, “use of limb as a helping hand” and “grasp and release”. Combined treatment with aboBoNT-A and the exercise programme helped most children achieve their goals, but it was difficult to discern any differences in efficacy between the aboBoNT-A doses used in the study. The authors suggest that the high rates of goal achievement in this study indicate that botulinum toxin should not usually be given without a physiotherapy programme.

Oral sulphate solution (OSS: sodium sulphate, potassium sulphate and magnesium sulphate) is a low-volume osmotic agent for cleansing the intestines.Aim: in a multicentre, prospective, randomized, 3rd phase study with two parallel groups, the effectiveness, safety and tolerability of OSS was evaluated in comparison with Macrogol 4000 with electrolytes (a reference preparation for bowel cleansing in Russia) in adult patients who were scheduled for routine diagnostic colonoscopy.Methods. This study was conducted in three Russian research centres during the March–December, 2015 period. Men and women over the age of 18 scheduled to undergo routine diagnostic colonoscopy were randomly assigned either to the OSS group or to the Macrogol group with a fractional use mode before the colonoscopy. The colonoscopy researchers were not aware of which preparation had been taken by the patients. Anonymized video records were centrally analysed by three experts. The primary end point was the proportion of patients with a successful bowel preparation for colonoscopy ≥6 points, as determined by the Boston Bowel Preparation Scale of quality assessment (BBPS scale).Results. 296 patients were randomized in the study (147 patients were treated with OSS, 149 patients received Macrogol); 294 participants were included in the Intention to Treat population (ITT-population), and 274 participants were included in the population of patients who completed the study according to the protocol (Per-Protocol; PP-population) (139 patients received OSS, 135 patients received Macrogol). The proportion of patients with a successful bowel preparation (BBPS ≥6 scores) was high in both groups (OSS [PP-population]: 97.2 % (95 % confidence interval [CI] 89.5–99.3), Macrogol [PP-population]: 97.7 % (95 % CI: 90.7–99.4)). The corrected difference between the groups was -0.5 % (95 % CI: -4.2–3.3), thereby demonstrating “no less effective” of OSS as compared to Macrogol. Compliance with the drug use regime was higher in the OSS group than in the Macrogol group (95.7 % versus 82.3 %, respectively, p-value = 0.0011, ITT-population).The most common symptom reported in patients was nausea (27.9 % in the OSS group and 12.9 % in the Macrogol group). The proportion of patients who developed nausea was significantly higher in the OSS group than in the Macrogol group (25.2 % compared with 10.2 % when taking the first dose of the preparation (p = 0.0008) and 19.7 % compared with 6.8 % when taking the second dose of the preparation (p = 0.0016)). Differences in other symptoms (bloating, abdominal pain or abdominal discomfort) between the groups were not significant, with the severity of symptoms being generally mild. The safety profile of the investigated preparations in patients with inflammatory bowel disease (IBD) in remission did not differ from that in the general patient population.The differences in terms of secondary endpoints were not identified, including BBPS assessment for different sections of the colon, the level of polyp detection, the duration and completeness of colonoscopy, and the investigator’s satisfaction with the procedure. The analysis by subgroups also did not reveal any significant differences.Conclusion. In this study, the “not less effectiveness” of the sulphate solution was demonstrated as compared to Macrogol in a fractional use mode. Both preparations were well tolerated. Despite the higher incidence of nausea in the OSS group, the patients showed significantly higher compliance with the OSS mode as compared to that of Macrogol.This study is registered with the ClinicalTrials.gov Registry of Clinical Trials, No. NCT02321462.

Category: Bunion Introduction/Purpose: Hallux valgus (HV) is a progressive foot deformity affecting nearly 25% of adults, characterized by pain and functional disability, and morphological changes in foot appearance due to progressive lateral deviation of the hallux. HV is initially managed with orthotic interventions, which are often ineffective. Corrective surgery can be efficacious, but is associated with significant pain, long recovery times and, potentially, recurrence. AbobotulinumtoxinA (aboBoNT-A, Dysport ® ) injections may correct the underlying muscle imbalance (resulting from hypertonia of specific forefoot muscles governing the hallux) thought to cause deformity, thereby reducing pain and disability associated with HV. Therefore, the aim of this phase II, placebo-controlled, parallel-group, multicenter study (NCT03569098) was to evaluate the effect of aboBoNT-A on pain and disability associated with HV in adults. Methods: Patients were randomized (1:1:1) to receive aboBoNT-A 300U, 500U, or placebo in a ≥12-week double-blind (DB) phase, followed by a 24-week open-label (OL) phase (OL Cycle 1, 300U [all patients]; OL Cycle 2, 300U or 500U). Total doses were divided equally into four specific foot muscles involved in hallux function. Eligible patients were aged 18–75 years with an HV angle 15–30° and numeric pain rating scale (NPRS) score of ≥4 (0, no pain; 10, worst possible pain). NPRS score was self-reported for 7 days before baseline and before each post-baseline visit. The primary endpoint was change from baseline in NPRS score (7- day average) before DB Week 8. Two post-hoc endpoints were defined as the percentage of days patients’ NPRS scores were: 1) lower than their lowest NPRS score before baseline; and 2) ≥2 points lower than mean baseline NPRS score. Adverse events (AEs) were monitored throughout the study. Results: No statistically significant differences were observed for aboBoNT-A 300U (n=63) or 500U (n=60) versus placebo (n=63) in NPRS scores at DB Week 8 (least squares mean [standard error]: -1.7 [0.3], -2.4 [0.3], versus -2.0 [0.3], respectively). Scores showed a trend towards pain reduction at Week 12 for aboBoNT-A 500U (-2.4 [0.3]) versus placebo (-1.7 [0.3]; p=0.06). Scores reduced further in OL Cycle 1. Post-hoc analyses showed that patients treated with aboBoNT-A 500U spent significantly more days with reduced pain before DB Week 8 compared with those treated with placebo: analysis 1) 63% versus 38% of days, respectively (p