Introduction Treatment adjustments in Parkinson's disease (PD) are in part dependent on motor assessments. The aim of this study was to evaluate the cost-effectiveness of home-based motor monitoring plus standard in-office visits versus in-office visits alone in patients with advanced PD. Methods The procedures consisted of a prospective, one-year follow-up, randomized, case-control study. A total of 40 patients with advanced PD were randomized into two groups: 20 patients underwent home-based motor monitoring by using wireless motion sensor technology, while the other 20 patients had in-office visits. Motor and non-motor symptom severities, quality of life, neuropsychiatric symptoms, and comorbidities were assessed every four months. Direct costs were assessed using a standardized questionnaire. Cost-effectiveness was assessed using the incremental cost-effectiveness ratio (ICER). Results Both groups of PD patients were largely comparable in their clinical and demographic variables at baseline; however, there were more participants using levodopa-carbidopa intestinal gel in the home-based motor monitoring group. There was a trend for lower Unified Parkinson's Disease Rating Scale functional status (UPDRS II) scores in the patients monitored at home compared to the standard clinical follow-up ( p = 0.06). However, UPDRS parts I, III, IV and quality-adjusted life-years scores were similar between both groups. Home-based motor monitoring was cost-effective in terms of improvement of functional status, motor severity, and motor complications (UPDRS II, III; IV subscales), with an ICER/UPDRS ranging from €126.72 to €701.31, respectively. Discussion Home-based motor monitoring is a tool which collects cost-effective clinical information and helps augment health care for patients with advanced PD.

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NCT01590589.

Objective Because patients homozygous for Huntington disease (HD) receive the gain-of-function mutation in a double dose, one would expect a more toxic effect in homozygotes than in heterozygotes. Our aim was to investigate the phenotypic differences between homozygotes with both alleles ≥36 CAG repeats and heterozygotes with 1 allele ≥36 CAG repeats. Methods This was an international, longitudinal, case-control study (European Huntington's Disease Network Registry database). Baseline and longitudinal total functional capacity, motor, cognitive, and behavioral scores of the Unified Huntington's Disease Rating Scale (UHDRS) were compared between homozygotes and heterozygotes. Four-year follow-up data were analyzed using longitudinal mixed-effects models. To estimate the association of age at onset with the length of the shorter and larger allele in homozygotes and heterozygotes, regression analysis was applied. Results Of 10,921 participants with HD (5,777 female [52.9%] and 5,138 male [47.0%]) with a mean age of 55.1 ± 14.1 years, 28 homozygotes (0.3%) and 10,893 (99.7%) heterozygotes were identified. After correcting for multiple comparisons, homozygotes and heterozygotes had similar age at onset and UHDRS scores and disease progression. In the multivariate linear regression analysis, the longer allele was the most contributing factor to decreased age at HD onset in the homozygotes (p < 0.0001) and heterozygotes (p < 0.0001). Conclusions CAG repeat expansion on both alleles of the HTT gene is infrequent. Age at onset, HD phenotype, and disease progression do not significantly differ between homozygotes and heterozygotes, indicating similar effect on the mutant protein. Classification of evidence This study provides Class II evidence that age at onset, the motor phenotype and rate of motor decline, and symptoms and signs progression is similar in homozygotes compared to heterozygotes.

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AIMTo investigated the real-world effectiveness and safety of various regimens of interferon-free treatments in patients infected with hepatitis C virus (HCV).METHODSWe performed an observational study to analyze different antiviral treatments administered to 462 HCV-infected patients, of which 56.7% had liver cirrhosis. HCV RNA after 4 wk of treatment and at 12 wk after treatment sustained virologic response (SVR) as well as serious adverse events (SAEs) was analyzed first for the whole cohort and then separately in patients who met or did not meet the inclusion criteria of a clinical trial (CT-met and CT-unmet, respectively).RESULTSThe most frequently prescribed treatment was simeprevir/sofosbuvir (36.4%), followed by sofosbuvir/ledipasvir (24.9%) and ombitasvir/paritaprevir/ritonavir (r)/dasabuvir (19.9%). Ribavirin (RBV) was administered in 198 patients (42.9%). SVRs occurred in 437/462 patients (94.6%). The SVRs ranged between 93.3% and 100% for genotypes 1-4. SVRs were achieved in 96.2% patients in the CT-met group vs 91.9% patients in the CT-unmet group (P = 0.049). Undetectable HCV RNA at week 4 occurred in 72.9% of the patients. In the univariate analysis, the factors associated with SVRs were lower liver stiffness, absence of cirrhosis, higher platelet count, higher albumin levels, no RBV dose reduction, undetectable HCV RNA at week 4 and CT-met group. In the multivariate analysis, only albumin was an independent predictor of treatment failure (P = 0.04). Eleven patients (2.4%) developed SAEs; 5.2% and 0.7% of the patients in the CT-unmet and CT-met groups, respectively (P = 0.003).CONCLUSIONA high proportion of patients with HCV infection achieved SVRs. For patients who did not meet the CT criteria, treatment regimens must be optimized.

Background: Because of rapid demographic changes, the prevalence of movement disorders (MDs) is expected to increase in Africa. The objective of this study was to estimate the prevalence of MDs in an inpatient/outpatient-based study of rural and urban health care centers in Cameroon. Methods: In this retrospective medical chart review, the inpatient/outpatients settings covered an urban population (3,000,000) and a rural population (380,276). Neurological diseases were classified according to the International Statistical Classification of Diseases-Related Health Problems, 10th revision (ICD-10). Crude prevalence was calculated per 100 with 95% confidence intervals (CIs). Results: Of 20,131 medical charts reviewed (13% from the rural area), 4187 patients (20.8%) with neurological complaints were identified. MDs were diagnosed exclusively from urban centers in 134 patients (3.2%): the mean patient age was 48.6 AE 18.6 years, and 54.7% were women. The most prevalent MDs were hyperkinetic movements (tremor, myoclonus, and drug-induced MDs [ICD-10 code G25]; prevalence, 1.19%; 95% CI, 1.192-1.194%), Parkinson's disease (ICD-10 code G20; prevalence, 0.78%; 95% CI, 0.785-0.787%), dystonia (ICD-10 code G24; prevalence, 0.61%; 95% CI, 0.612-0.613%), secondary parkinsonism (ICD-10 code G21; prevalence, 0.56%; 95% CI, 0.564-0.565%), Huntington's disease (ICD-10 code G10; prevalence, 0.09%; 95% CI, 0.091-0.092%), and ataxia (ICD-10 code R29; prevalence, 0.04%; 95% CI, 0.0451-0.0456). Conclusion: Although the burden of MDs is expected to increase, MDs are likely underdiagnosed in rural areas. High-quality movement disorder training is essential to tackle this need.