Current medical research and opinion
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Meta Analysis
The impact of clinical heterogeneity on conducting network meta-analyses in transthyretin amyloidosis with polyneuropathy.
Objective: The comparative safety and efficacy of tafamidis, patisiran and inotersen treatments for transthyretin amyloidosis with polyneuropathy (ATTR-PN) has not been evaluated in clinical trials. In the absence of head-to-head evidence, indirect treatment comparisons such as network meta-analyses (NMAs) can be performed to evaluate relative effects of treatments. This study aims to assess the feasibility of conducting an NMA of available therapies for ATTR-PN patients. ⋯ Neuropathy outcomes were not evaluated consistently between trials. Conclusions: An NMA of ATTR-PN treatments was not feasible, given the observed cross-trial heterogeneity. This decision highlights the importance of careful consideration for clinical heterogeneity that may threaten the validity of indirect comparisons.
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Review
Non-coronary atherosclerotic cardiovascular disease in patients with familial hypercholesterolaemia.
Objective: Familial hypercholesterolaemia (FH) is a common autosomal dominant inherited disease, affecting 1 in 200-500 individuals worldwide. FH is characterized by elevated circulating low-density lipoprotein cholesterol (LDL-C) concentrations. Its association with increased risk of coronary heart disease (CHD) (>10-fold, compared with patients without FH) is well documented. ⋯ A key question is whether statins or other LDL-C-lowering therapies, provide an additional reduction in the risk of these less-recognized vascular and non-vascular complications in FH patients. Conclusions: Heterozygous FH is associated with increased risk for stroke, PAD and AoVC. Clinicians should take these non-CHD ASCVD aspects into consideration for optimal management of FH patients.
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Objective: To compare the rates of successfully treated patients (STPs) with vortioxetine versus venlafaxine in major depressive disorder (MDD), using dual endpoints that combine improvement of mood symptoms with optimal tolerability or functional remission, and conduct a simplified cost-effectiveness analysis. Methods: The 8-week SOLUTION study (NCT01571453) assessed the efficacy and safety of vortioxetine (10 mg/day) versus venlafaxine XR (150 mg/day) in adult Asian patients with MDD. Rates were calculated post-hoc of STP Mood and Tolerability (≥50% reduction from baseline in Montgomery-Åsberg Depression Rating Scale [MADRS] total score and no treatment-emergent adverse events) and STP Mood and Functioning (≥50% reduction from baseline in MADRS total score and Sheehan Disability Scale total score ≤6). ⋯ Conclusions: Higher rates of dual treatment success were seen with vortioxetine versus venlafaxine. Although vortioxetine was not dominant in the base case, the incremental cost per STP for vortioxetine versus venlafaxine were overall within acceptable ranges. These results support the benefits previously reported with vortioxetine versus other antidepressants in broad efficacy, tolerability profile and cost-effectiveness.
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Objective: Rituximab is used as an off-label treatment for relapsing-remitting multiple sclerosis (RRMS); however, the comparative efficacy and safety of rituximab versus currently licensed disease-modifying drugs (DMDs) for RRMS is unknown. A systematic literature review was conducted to evaluate the available data pertaining to efficacy and safety of rituximab in adult patients with RRMS and highly active relapsing multiple sclerosis (HA-RMS); data quality was critically assessed via risk of bias (RoB) assessment. Methods: Biomedical literature databases were searched until mid-2018 and key proceedings were searched from 2016 to 2018. ⋯ Two-thirds of the non-randomized RRMS studies were associated with critical/serious RoB; the single RCT was associated with low RoB. Furthermore, all of the non-randomized HA-RMS studies were associated with critical/serious RoB. Conclusions: Available evidence of off-label rituximab use for the treatment of patients with RRMS suggests generally favorable efficacy versus placebo and interferons/glatiramer acetate; however, the poor quality of the included studies limits any robust conclusions.
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Objective: To assess the real-world use of home health services (HHS) among patients with major depressive disorder (MDD) with and without treatment-resistant depression (TRD). Methods: Adults (18-64 years) from a commercial claims database (07/2009 to 03/2015) were categorized into three cohorts: "TRD"(N = 6411), "non-TRD MDD"(N = 33,068), "non-MDD"(N = 149,884) stratified based on use of HHS (HHS vs. no-HHS). Healthcare resource utilization (HRU) and costs were evaluated up to two years following the first antidepressant pharmacy claim using descriptive statistics. ⋯ Patients without MDD who used HHS had annual healthcare costs of $22,340 while non-MDD patients who did not use HHS had healthcare costs of $3479 PPPY. However, among HHS users, HHS costs represented a relatively small proportion of total healthcare costs. Conclusions: The high proportion of TRD patients using HHS suggests it is a utilized healthcare delivery pathway by TRD patients.