Background: Huntington’s disease (HD) is a rare, genetic, neurodegenerative disease. Obtaining population-level data on epidemiology and disease management is challenging. Objective: To investigate the epidemiology, clinical manifestations, treatment, and healthcare utilization of patients with HD in Israel. Methods: Retrospective population-based cohort study, including 20 years of routinely collected data from Maccabi Healthcare Services, an insurer and healthcare provider for one-quarter of the Israeli population. Results: The study cohort included 109 adult patients (aged ≥18 years) diagnosed with HD, with mean age of 49.9 years and 56%females. The most common HD-related conditions were anxiety (40%), behavioral problems (34%), sleep disorders (21%), and falls (13%). Annual incidence rates for HD ranged from 0.17 to 1.34 per 100,000 from 2000 to 2018; the 2018 crude prevalence in adults was 4.36 per 100,000. Median survival from diagnosis was approximately 12 years (95%CI: 10.4–15.3). The most frequent symptomatic treatments were antidepressants (69%), antipsychotics (63%), and tetrabenazine (63%), the only drug approved for the treatment of HD chorea in Israel during the examined period. Patterns of healthcare utilization changed as disease duration increased, reflected by increased frequency of emergency department visits and home visits. Conclusion: This retrospective population-based study provides insights into the prevalence, incidence, clinical profile, survival, and resource utilization of patients with HD in ethnically diverse Israel. The findings in this study are generally consistent with the international literature and demonstrate the value of routinely collected healthcare data as a complementary resource in HD research.
Aim: Generating direct comparative evidence in prospective randomized trials is difficult for rare diseases. Real-world cohorts may supplement control populations. Methods: Entrectinib-treated adults with advanced ROS1 fusion-positive NSCLC (n = 94) from Phase I/II trials (ALKA-372-001 [EudraCT2012-00148-88], STARTRK-1 [NCT02097810], and STARTRK-2 [NCT02568267]) were compared with a real-world crizotinib-treated cohort (n = 65). Primary end point, time-to-treatment discontinuation (TTD); secondary end points, PFS and OS. Results: Median (95% CI) weighted TTD: 12.9 (9.9–17.4) months for entrectinib; 8.8 (6.2–9.9) months for crizotinib (weighted hazard ratio, 0.72 [0.51–1.02]). Median OS with entrectinib was not reached, weighted median OS with crizotinib was 18.5 (15.1–19.9) months. Conclusion: Entrectinib administered in clinical trials may be associated with longer TTD than a real-world crizotinib population.
The search yielded 350 citations with two additional citations identified through examination of reference lists of the included publications. After applying the selection criteria, six articles that described eight different study cohorts were included in the qualitative data synthesis. Seven study cohorts were retrospective analyses and one was a physician panel survey. Six study cohorts included patients from Europe, and two included patients from the USA. Among studies that followed patients from primary GBM diagnosis, 41-64% received systemic treatment at recurrence. Treatment regimens varied by year of diagnosis and country. Bevacizumab-containing regimens were the most common in USA-based cohorts (representing 80-86% of treatment received). Bevacizumab-containing therapy, nitrosourea-containing therapy, and temozolomide were the most common treatment regimens in Spain, Italy, and France, respectively. ConClusions: The studies in this qualitative systematic review demonstrate that few treatment options are available for rGBM patients. In Europe, where bevacizumab is not approved for rGBM, the treatment paradigm varies between countries and several options are used (including regimens without bevacizumab). In the USA, where bevacizumab is indicated for rGBM, bevacizumab-containing regimens have become the standard of care. PCN313 Real WoRld aNaPlastiC lymPhoma KiNase (alK) ReaRRaNgemeNt testiNg PatteRNs, tReatmeNt sequeNCes aNd suRvival of alK-iNhibitoR tReated PatieNts
Summary
There is an increasing expectation that computational approaches may supplement existing human decision-making. Frontloading of models for cardiac safety prediction is no exception to this trend, and ongoing regulatory initiatives propose use of high-throughput
in vitro
data combined with computational models for calculating proarrhythmic risk. Evaluation of these models requires robust assessment of the outcomes. Using FDA Adverse Event Reporting System reports and electronic healthcare claims data from the Truven-MarketScan US claims database, we quantify the incidence rate of arrhythmia in patients and how this changes depending on patient characteristics. First, we propose that such datasets are a complementary resource for determining relative drug risk and assessing the performance of cardiac safety models for regulatory use. Second, the results suggest important determinants for appropriate stratification of patients and evaluation of additional drug risk in prescribing and clinical support algorithms and for precision health.
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