Incidence of hydrocephalus surgery increased significantly during 2004-2011, specifically in elderly patients. Surgical treatment of iNPH markedly improved functional independence, but the improvement rate was low compared to recent single- and multicentre studies. Thus, the potential for surgical improvement is likely lower than generally reported when treating patients as part of everyday clinical care.

Objective: To evaluate a group-based music intervention in patients with Parkinson’s disease. Design: Parallel group randomized controlled trial with qualitative triangulation. Setting: Neurorehabilitation in primary care. Subjects: Forty-six patients with Parkinson’s disease were randomized into intervention group ( n = 26), which received training with the music-based intervention, and control group ( n = 20) without training. Interventions: The intervention was delivered twice weekly for 12 weeks. Main measures: Primary outcome was Timed-Up-and-Go subtracting serial 7’s (dual-task ability). Secondary outcomes were cognition, balance, concerns about falling, freezing of gait, and quality of life. All outcomes were evaluated at baseline, post-intervention, and three months post-intervention. Focus groups and individual interviews were conducted with the intervention group and with the delivering physiotherapists. Results: No between-group differences were observed for dual-task ability. Between-group differences were observed for Falls Efficacy Scale (mean difference (MD) = 6.5 points; 95% confidence interval (CI) = 3.0 to 10.0, P = 0.001) and for Parkinson Disease Questionnaire-39 items (MD = 8.3; 95% CI = 2.7 to 13.8, P = 0.005) when compared to the control group post-intervention, but these were not maintained at three months post-intervention. Three themes were derived from the interviews: Expectations versus Results, Perspectives on Treatment Contents, and Key Factors for Success. Conclusion: Patient-reported outcomes and interviews suggest that the group-based music intervention adds value to mood, alertness, and quality of life in patients with Parkinson’s disease. The study does not support the efficacy in producing immediate or lasting gains in dual-tasking, cognition, balance, or freezing of gait.

ImportanceRituximab is a third-line option for refractory generalized myasthenia gravis (MG) based on empirical evidence, but its effect in new-onset disease is unknown.ObjectiveTo investigate the efficacy and safety of rituximab compared with placebo as an add-on to standard of care for MG.Design, Setting, and ParticipantsThis randomized, double-blind, placebo-controlled study took place throughout 48 weeks at 7 regional clinics in Sweden. Key inclusion criteria were age older than 18 years, onset of generalized symptoms within 12 months or less, and a Quantitative Myasthenia Gravis (QMG) score of 6 or more. Patients were screened from October 20, 2016, to March 2, 2020. Key exclusion criteria included pure ocular MG, suspected thymoma, previous thymectomy, and prior noncorticosteroid immunosuppressants or high doses of corticosteroids.InterventionsParticipants were randomized 1:1 without stratification to a single intravenous infusion of 500 mg of rituximab or matching placebo.Main Outcomes and MeasuresMinimal disease manifestations at 16 weeks defined as a QMG score of 4 or less with prednisolone, 10 mg or less daily, and no rescue treatment.ResultsOf 87 potentially eligible patients, 25 were randomized to rituximab (mean [SD] age, 67.4 [13.4] years; 7 [28%] female) and 22 to placebo (mean [SD] age, 58 [18.6] years; 7 [32%] female). Compared with placebo, a greater proportion with rituximab met the primary end point; 71% (17 of 24) in the rituximab group vs 29% (6 of 21) in the placebo group (Fisher exact test P = .007; probability ratio, 2.48 [95% CI, 1.20-5.11]). Secondary end points, comparing changes in Myasthenia Gravis Activities of Daily Living and Myasthenia Gravis Quality of Life at 16 weeks with QMG at 24 weeks did not differ between groups with censoring for rescue treatment (per-protocol analysis) but were in favor of active treatment when rescue treatment was taken into account by worst rank imputation (post hoc analysis). Rescue treatments were also more frequent in the placebo arm (rituximab: 1 [4%]; placebo, 8 [36%]). One patient in the placebo arm had a myocardial infarction with cardiac arrest and 1 patient in the active arm experienced a fatal cardiac event.Conclusions and RelevanceA single dose of 500 mg of rituximab was associated with greater probability of minimal MG manifestations and reduced need of rescue medications compared with placebo. Further studies are needed to address long-term benefit-risk balance with this treatment.Trial RegistrationClinicalTrials.gov Identifier: NCT02950155

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