Current medical research and opinion
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Randomized Controlled Trial Multicenter Study
IncobotulinumtoxinA for upper- and lower-limb spasticity in Japanese patients.
Introduction: The safety and tolerability of incobotulinumtoxinA 400 U for upper- and lower-limb post-stroke spasticity was assessed in a small cohort of Japanese patients during the open-label lead-in tolerability periods (LITP) of two phase 3 studies (CTI-153029 and CTI-153030; Japan Pharmaceutical Information Centre). Methods: Adult patients received a single incobotulinumtoxinA injection session (total dose of 400 U) in the upper (J-PURE) or lower limb (J-PLUS). Adverse events (AEs) were assessed at 1, 4, 8 and 12 weeks post-injection during the 12 week follow-up. ⋯ Non-serious, transient AEs of special interest reported by two patients in J-PURE comprised muscular weakness and eyelid ptosis. No patient discontinued due to AEs. Conclusion: Preliminary results in this small population suggest that incobotulinumtoxinA 400 U is well tolerated for treating upper- or lower-limb post-stroke spasticity in Japanese patients.
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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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Observational Study
Distance to glycemic goal at the time of treatment intensification in patients with type 2 diabetes mellitus failing metformin monotherapy in the United States.
Background: A substantial proportion of patients with type 2 diabetes mellitus (T2DM) do not reach their target HbA1c level on metformin. The objective of this retrospective observational cohort study is to better characterize the distance between HbA1c target and patient's actual HbA1c level (the distance to goal), using a target HbA1c of 7.0% (53 mmol/mol), in patients with T2DM who have started metformin monotherapy. Methods: We used data from the GE Centricity Electronic Medical Record database by IQVIA in 2016 in the United States (US) to identify adults with T2DM who started metformin monotherapy (MM) and received at least 90 days of treatment. ⋯ The mean post-MM HbA1c for those who failed MM and did not receive intensified treatment was 8.0% (64 mmol/mol) (median 7.5%, 58 mmol/mol) and the mean distance to goal was 1.0% (median 0.5%). Conclusion: A proportion of US T2DM patients do not achieve glycemic control (target HbA1c < 7.0%) despite 90 days of MM. Patients who failed MM and eventually received intensified treatment did so when their HbA1c distance to goal exceeded the level at which one add-on therapy alone might be sufficient to bring them to goal.
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Objective: To determine the pattern of antidiabetic drug use, with a particular focus on the metformin dose, among patients with type 2 diabetes mellitus (T2DM) in a Japanese working population. Methods: We used an administrative claims database linked to yearly health check-up data from large corporations. Data were collected for T2DM patients who began using an antidiabetic drug between 2014 and 2017 (n = 20,401). ⋯ SGLT2is were infrequently prescribed. Metformin was prescribed at a daily dose of ≤500 mg in many patients. Greater efforts are needed to assess the comparative effectiveness of these treatment strategies.
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Background: Current healthcare professional consensus-generating methodologies work by forcing consensus, which risks corrupting original opinions and often fails to assess prior expert knowledge awareness. Experience gained with a novel method in a progressive life-long rare disease, X-linked hypophosphataemia, which addresses these risks is presented here. Methods: Four case-studies are reported, presenting a novel methodology comprised of two survey rounds. ⋯ Less frequent Round 1 items had various degrees of prompted agreement consensus; some did not reach the consensus threshold of >50% participant agreement. Conclusions: Observed proportional group awareness and consensus is quicker than the Delphi technique and its variants, providing objective assessments of expert knowledge and standardized categorization of items regarding awareness, consensus and prompting. Further, it offers tailored management of each item in terms of educational need and further investigation.