Current medical research and opinion
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Objective: Describe the development of a claims-based classifier utilizing machine learning to identify patients with probable Lennox-Gastaut syndrome (LGS) from six state Medicaid programs. Methods: Patients were included if they had ≥2 medical claims ≥30 days apart for specified or unspecified epilepsy, excluding those with ≥1 claim for petit mal status. The LGS classifier utilized a random forest algorithm, a compilation of thousands of binary decision trees in which machine-generated predictor variables split the data set into branches that predict the presence or absence of LGS. ⋯ The random forest methodology outperformed logistic regression and single tree methodology. Most of the important LGS predictor characteristics identified by the classifier were statistically significantly associated with LGS status (p < .05). Conclusions: The claims-based LGS classifier showed high sensitivity and specificity, outperformed single tree and logistic regression methodologies and identified a prevalence of probable LGS that was similar to previously published estimates.
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Objective: To describe the cost-effectiveness of pembrolizumab plus chemotherapy (carboplatin and paclitaxel or nab-paclitaxel; P + C) in metastatic, squamous, non-small-cell lung cancer (NSCLC) patients in the US. Methods: A model comparing P + C versus C alone is developed utilizing partitioned survival analysis. Primary clinical efficacy, treatment utilization, health utility and safety data are derived from the KEYNOTE-407 trial and projected over 20 years. ⋯ Conclusions: Across all eligible patients, the addition of pembrolizumab to chemotherapy is projected to approximately double life expectancy, yielding an extension to a point not previously seen in metastatic squamous NSCLC. Overall, and within all relevant PD-L1 subgroups, use of P + C yields an ICER below $100,000/QALY, and can be a cost-effective first-line treatment for eligible metastatic squamous NSCLC patients for whom chemotherapy is currently administered. In the PD-L1 ≥ 50% subgroup, additional follow-up within trials of pembrolizumab plus chemotherapy and pembrolizumab monotherapy are needed to better define cost-effectiveness between these comparators.
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Normalizing serum thyroid stimulating hormone (TSH) levels by lifelong treatment with levothyroxine (LT4) remains the primary goal of therapy for patients with hypothyroidism. The reference ranges for TSH are derived from populations with (supposedly) normal thyroid function. But, TSH results are affected by a number of factors including alterations in TSH levels with age, concurrent illnesses, circadian rhythm, inter- and intra-assay differences, and some commonly used medications that interfere with thyroid function or the TSH test. ⋯ However, the new LT4 formulations are associated with manifold increases in the number of self-reported adverse events. Therefore, patients with hypothyroidism as well as the clinicians managing them need to better understand the utility as well as the limitations of the widely used TSH measurement. In addition, both pharmaceutical companies and the prescribing clinician need to take greater care when patients are switched from older to newer formulations.
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Objectives: Atopic dermatitis (AD) is a chronic, relapsing skin condition, with signs and symptoms that impact patients' lives and are best measured from the patient perspective. Therefore, there is a need for AD-specific questionnaires that are consistent with Food and Drug Administration guidance and best measurement practices, assessing sign and symptom severity and associated impacts, to support treatment efficacy in regulated trials. The objectives were to develop patient-reported outcome (PRO) questionnaires assessing sign and symptom severity, as well as impacts of moderate-to-severe adult AD. ⋯ During the patient interviews (n = 15), 19 sign and symptom and 41 impact concepts were reported, the majority of which were evaluated by the ADerm-SS and ADerm-IS, thus substantiating the content of both questionnaires. Additionally, patients interpreted both questionnaires as intended by the developers. Conclusions: The ADerm-SS and ADerm-IS can be regarded as content-valid PRO questionnaires for moderate-to-severe AD.