Dataset Information

Epidemiology of AL amyloidosis: a real-world study using US claims data.

ABSTRACT: Amyloid light-chain (AL) amyloidosis is a rare disease caused by extracellular deposition of misfolded immunoglobulin light chains. This study aimed to provide an up-to-date estimate of prevalence and incidence of AL amyloidosis in the United States. Using claims databases from years 2007 to 2015, adults ?18 years old with AL amyloidosis were included if they had (1) at least 1 inpatient or 2 outpatient claims consistent with AL amyloidosis and (2) received 1 AL-specific treatment. Prevalence was calculated as the number of AL patients divided by the number of enrollees on June 30th of each calendar year. Incidence was calculated as the number of patients with AL who were disease-free and enrolled with a health plan for 1 year prior, divided by the number of enrollees with enrollment from July 1st of the previous year to June 30th of each calendar year. The prevalence of AL amyloidosis increased significantly between 2007 and 2015, from 15.5 cases per million in 2007 to 40.5 in 2015, an annual percentage change (APC) of 12% (P < .001). The incidence ranged from 9.7 to 14.0 cases per million person-years (APC, 3%; P = .114) with no statistically significant increase. There was an increase in AL amyloidosis prevalence over a 9-year period coupled with stable incidence rates. Although there is no diagnosis code specific to AL amyloidosis and no validated method for identifying this condition using claims data, extrapolating from our data, there are at least 12?000 adults in the United States living with AL amyloidosis, and the number seems likely to rise.

SUBMITTER: Quock TP

PROVIDER: S-EPMC5965052 | biostudies-literature | 2018 May

REPOSITORIES: biostudies-literature

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Epidemiology of AL amyloidosis: a real-world study using US claims data.

Quock Tiffany P TP Yan Tingjian T Chang Eunice E Guthrie Spencer S Broder Michael S MS

Blood advances 20180501 10

Amyloid light-chain (AL) amyloidosis is a rare disease caused by extracellular deposition of misfolded immunoglobulin light chains. This study aimed to provide an up-to-date estimate of prevalence and incidence of AL amyloidosis in the United States. Using claims databases from years 2007 to 2015, adults ≥18 years old with AL amyloidosis were included if they had (1) at least 1 inpatient or 2 outpatient claims consistent with AL amyloidosis and (2) received 1 AL-specific treatment. Prevalence wa ...[more]

PMID: 29748430

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Project description:BACKGROUND:Huntington's disease (HD) is a rare, genetic, neurodegenerative and ultimately fatal disease with no cure or progression-delaying treatment currently available. HD is characterized by a triad of cognitive, behavioural and motor symptoms. Evidence on epidemiology and management of HD is limited, especially for Germany. This study aims to estimate the incidence and prevalence of HD and analyze the current routine care based on German claims data. METHODS:The source of data was a sample of the Institute for Applied Health Research Berlin (InGef) Research Database, comprising data of approximately four million insured persons from approximately 70 German statutory health insurances. The study was conducted in a retrospective cross-sectional design using 2015 and 2016 as a two-year observation period. At least two outpatient or inpatient ICD-10 codes for HD (ICD-10: G10) during the study period were required for case identification. Patients were considered incident if no HD diagnoses in the 4 years prior to the year of case identification were documented. Information on outpatient drug dispensations, medical aids and remedies were considered to describe the current treatment situation of HD patients. RESULTS:A 2-year incidence of 1.8 per 100,000 persons (95%-Confidence interval (CI): 1.4-2.4) and a 2-year period prevalence of 9.3 per 100,000 persons (95%-CI: 8.3-10.4) was observed. The prevalence of HD increased with advancing age, peaking at 60-69?years (16.8 per 100,000 persons; 95%-CI: 13.4-21.0) and decreasing afterwards. The most frequently observed comorbidities and disease-associated symptoms in HD patients were depression (42.9%), dementia (37.7%), urinary incontinence (32.5%), extrapyramidal and movement disorders (30.5%), dysphagia (28.6%) and disorders of the lipoprotein metabolism (28.2%). The most common medications in HD patients were antipsychotics (66.9%), followed by antidepressants (45.1%). Anticonvulsants (16.6%), opioids (14.6%) and hypnotics (9.7%) were observed less frequently. Physical therapy was the most often used medical aid in HD patients (46.4%). Nursing services and speech therapy were used by 27.9 and 22.7% of HD patients, respectively, whereas use of psychotherapy was rare (3.2%). CONCLUSIONS:Based on a representative sample, this study provides new insights into the epidemiology and routine care of HD patients in Germany, and thus, may serve as a starting point for further research.

Project description:BackgroundBiological therapies (BTs) including infliximab (IFX), adalimumab (ADL), secukinumab (SCK) and ustekinumab (UST) are approved in Japan for the treatment of psoriasis. Although the persistence rates and medical costs of BTs treatment have been investigated in multiple foreign studies in recent years, few such studies have been conducted in Japan and the differences between patients who adhered to treatment and those who did not have not been reported. This study is aimed at investigating the persistence rates and medical costs of BTs in the treatment of psoriasis in Japan, using the real-world data from a large-scale claims database.MethodsClaims data from the JMDC database (August 2009 to December 2016) were used for this analysis. Patient data were extracted using the ICD10 code for psoriasis and claims records of BT injections. Twelve-month and 24-month persistence rates of BTs were estimated by Kaplan-Meier methodology, and 12-month-medical costs before and after BT initiation were compared between persistent and non-persistent patient groups at 12 months.ResultsA total of 205 psoriasis patients treated with BTs (BT-naïve patients: 177) were identified. The 12-month/24-month persistence rates for ADL, IFX, SCK, and UST in BT-naïve patients were 46.8%?±?16.6%/46.8?±?16.6%, 53.0%?±?14.9%/41.0%?±?15.5%, 55.4%/55.4% (95% CI not available) and 79.4%?±?9.9%/71.9%?±?12.2%, respectively. Statistically significant differences in persistence were found among different BT treatments, and UST was found to have the highest persistence rate. The total medical costs during the 12 months after BT initiation in BT-naïve patients were (in 1000 Japanese Yen): 2218 for ADL, 3409 for IFX, 465 for SCK, 2824 for UST (average: 2828). Compared with the 12-month persistent patient group, the total medical costs in the persistent group was higher (?:+?118), but for some medications such as IFX or UST cost increases were lower for persistent patients.ConclusionsUST was found to have the highest persistence rate among all BTs for psoriasis treatment in Japan. The 12-month medical costs after BT initiation in the persistent patient group may not have increased as much as in the non-persistent patient group for some medications.

Project description:BACKGROUND:Real-World Data (RWD) studies provide important insights in disease epidemiology, in real clinical populations, with long follow-up periods. The aim of the present study was to describe the epidemiology of schizophrenia spectrum disorders (SD) during an 8-year period in Spain. METHODS:A retrospective cohort of subjects aged 15 to 64?years was followed-up using electronic healthcare databases of the Valencia region (2008-2015). SD cases included outpatient and inpatient settings (ICD 9 codes 295.XX). Prevalence of SD was assessed. Incidence rate (IR) in the subpopulation aged between 15 and 34?years was also provided. Healthcare utilization (HCU) rates, including outpatient, specialists, hospitalizations and antipsychotic dispensations were estimated. RESULTS:The cohort included 3,976,071 subjects; 24,749 of them had a prevalent diagnosis of SD. The overall prevalence for SD was 6.2 per 1000 persons. SD were 76% more prevalent in men than women. IR in the subpopulation aged between 15 and 34?years was 50.25 per 100,000 persons years and was more than 2 times higher for men than for women. 83.4% of the overall outpatient visits from the cohort of patients were related to SD. The 21,095 overall hospitalizations with the SD code resulted in 286,139?days of hospitalization, with a median of 4?days (IQR: 1.6-9.2) per person-year. 93.2% of subjects diagnosed with SD were ever treated with some antipsychotic drug during the study period, and 70% of the patients were ever treated with antipsychotic polypharmacy. CONCLUSIONS:This large population-based study using RWD provides novel and recent information SD in a southern European country. The prevalence and IR of SD showed is greater than previously published and higher in men than in women. The fact of having used a large arsenal of electronic data (including outpatient and inpatient) for 8?years may have influenced. SD represents high burden and healthcare utilization. Contrary to guidelines recommendations the majority of patients were ever treated with antipsychotic polypharmacy.

Project description:IntroductionTriptans, the most commonly prescribed acute treatments for migraine attacks are, per FDA labeling, contraindicated in cardiovascular (CV) disease patients and have warnings and precautions for those with CV risk factors.MethodsHeadache specialists, cardiologists, and health economics and outcomes researchers convened to identify diagnostic codes for: (1) CV diseases contraindicating triptans based on FDA labeling; (2) conditions comprising "other significant underlying CV disease"; and (3) CV risk factors included as warnings and precautions for triptans. A retrospective, cross-sectional analysis of commercially insured adult US migraine patients in the 2017 Optum® Clinformatics® Data Mart (CDM) and the 2017 IBM® Watson Health MarketScan® Commercial Claims database was used to estimate the proportion of migraine patients with CV contraindications and warnings and precautions to triptans.ResultsOf the 56,662 migraine patients analyzed from Optum CDM, 13.5% had ≥1 CV disease as specified in triptan labeling and an additional 8.5% had ≥1 "other CV disease" judged by the panel to constitute a "significant underlying CV disease" (total: 22.0% migraine patients). Of 176 724 migraine patients analyzed from MarketScan, 12.2% had ≥1 CV disease as specified in the labeling and an additional 8.0% had ≥1 "other significant underlying CV disease" (total: 20.2% of migraine patients). An additional 25.4% and 25.1% of migraine patients had ≥2 CV risk factors in Optum CDM and MarketScan. In total, 47.4% and 45.3% of migraine patients in both databases had a CV disease specified as a contraindication, an "other CV disease" endorsed as significant, or ≥2 CV risk factors identified as warnings and precautions to triptans.ConclusionsAnalyses of more than 230,000 people with migraine showed that ≥20% of commercially insured US migraine patients have a CV condition that specifically contraindicates triptan treatment, and an additional 25% have ≥2 CV risk factors identified as warnings and precautions to triptans.

Project description:BackgroundThe randomized, dose-optimization, open-label ReDOS study in US patients with metastatic colorectal cancer (CRC) showed that, compared with a standard dosing approach, initiating regorafenib at 80 mg/day and escalating to 160 mg/day depending on tolerability increased the proportion of patients reaching their third treatment cycle and reduced the incidence of adverse events without compromising efficacy. Subsequently, the ReDOS dose-escalation strategy was included as an alternative regorafenib dosing option in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines. A retrospective analysis was conducted using a US claims database to assess whether inclusion of this dose-escalation strategy in NCCN Guidelines has influenced the use of flexible dosing in routine US clinical practice, and to describe clinical outcomes pre- and post-inclusion in NCCN Guidelines.MethodsPatients with CRC in the Optum's de-identified Clinformatics® Data Mart database initiating regorafenib for the first time between January 2016 and June 2020 were stratified based on whether they initiated regorafenib pre- or post-inclusion of ReDOS in NCCN Guidelines, and in two groups: flexible dosing (< 160 mg/day; < 84 tablets in the first treatment cycle) and standard dosing (160 mg/day; ≥ 84 tablets in the first treatment cycle). The primary endpoints were the proportion of patients who initiated their third treatment cycle and the mean number of treatment cycles per group.Results703 patients initiated regorafenib during the study period, of whom 310 (44%) initiated before and 393 (56%) initiated after inclusion of ReDOS in NCCN Guidelines. After inclusion in the guidelines, the proportion of patients who received flexible dosing increased from 21% (n = 66/310) to 45% (n = 178/393), the proportion who received standard dosing decreased from 79% (n = 244/310) to 55% (n = 215/393), the proportion who initiated their third treatment cycle increased from 36% (n = 113/310) to 46% (n = 179/393), and the mean (standard deviation) number of treatment cycles increased from 2.6 (2.9) to 3.2 (3.1).ConclusionsFollowing inclusion of ReDOS in NCCN Guidelines, real-world data suggest that US clinicians have markedly increased use of flexible dosing in clinical practice, potentially maximizing clinical benefits and safety outcomes for patients with metastatic CRC receiving regorafenib.

Project description:PurposeUsing real-world data (RWD)-based trial simulation approach, we aim to simulate colorectal cancer (CRC) trials and examine both effectiveness and safety end points in different simulation scenarios.MethodsWe identified five phase III trials comparing new treatment regimens with an US Food and Drug Administration-approved first-line treatment in patients with metastatic CRC (ie, fluorouracil, leucovorin, and irinotecan) as the standard-of-care (SOC) control arm. Using Electronic Health Record-derived data from the OneFlorida network, we defined the study populations and outcome measures using the protocols from the original trials. Our design scenarios were (1) simulation of the SOC fluorouracil, leucovorin, and irinotecan arm and (2) comparative effectiveness research (CER) simulation of the control and experimental arms. For each scenario, we adjusted for random assignment, sampling, and dropout. We used overall survival (OS) and severe adverse events (SAEs) to measure effectiveness and safety.ResultsWe conducted CER simulations for two trials, and SOC simulations for three trials. The effect sizes of our simulated trials were stable across all simulation runs. Compared with the original trials, we observed longer OS and higher mean number of SAEs in both CER and SOC simulation. In the two CER simulations, hazard ratios associated with death from simulations were similar to that reported in the original trials. Consistent with the original trials, we found higher risk ratios of SAEs in the experiment arm, suggesting potentially higher toxicities from the new treatment regimen. We also observed similar SAE rates across all simulations compared with the original trials.ConclusionIn this study, we simulated five CRC trials, and tested two simulation scenarios with several different configurations demonstrated that our simulations can robustly generate effectiveness and safety outcomes comparable with the original trials using real-world data.

Dataset Information

Epidemiology of AL amyloidosis: a real-world study using US claims data.

Publications

Epidemiology of AL amyloidosis: a real-world study using US claims data.

Similar Datasets

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