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BACKGROUND: Patients with obstructive hypertrophic cardiomyopathy (oHCM) have a substantial humanistic, clinical, and economic burden due to the array of symptoms and complications associated with the disease. The objective of this review was to identify key evidence gaps related to oHCM, specifically in Europe, North America, and Japan. METHODS: A targeted literature review was conducted using PubMed to identify English-language studies published between 2012 and 2022 assessing patients with HCM/oHCM in France, Germany, Italy, Spain, the United Kingdom (UK), the United States (US), Canada, and Japan. Outcomes of interest were epidemiology, natural history, pathophysiology, management, and clinical, economic, and humanistic burden. Identified studies were assessed qualitatively to characterize evidence gaps. RESULTS: Among 2,262 abstracts and 531 full-text articles screened, 178 articles were included from PubMed searches. An additional 16 unique studies were identified via a supplemental Google Scholar search initially conducted in January 2023 and updated in July 2024. Disease natural history, pathophysiology, and management were well documented globally. Significant evidence gaps were noted for the epidemiology, treatment, and burden of oHCM. Although multiple US studies were identified on the clinical, economic, and humanistic burden of oHCM, and one clinical burden study was found for Japan, there was a lack of evidence for France, Germany, Italy, Spain, the UK, and Canada. CONCLUSIONS: Major evidentiary gaps exist for the epidemiology, treatment, and burden of oHCM. Future research should address these gaps, with a specific focus on generating real-world evidence for Canada and European countries that will support the evaluation of emerging therapies in these regions.
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Cardiomiopatia Hipertrófica , Humanos , Cardiomiopatia Hipertrófica/epidemiologia , Cardiomiopatia Hipertrófica/fisiopatologia , Cardiomiopatia Hipertrófica/terapia , Cardiomiopatia Hipertrófica/diagnóstico , Custos de Cuidados de Saúde , Efeitos Psicossociais da Doença , Europa (Continente)/epidemiologia , MasculinoRESUMO
BACKGROUND AND AIMS: Nonalcoholic steatohepatitis (NASH) may progress to advanced liver disease (AdvLD). This study characterized comorbidities, healthcare resource utilization (HCRU) and associated costs among hospitalized patients with AdvLD due to NASH in Italy. METHODS AND RESULTS: Adult nonalcoholic fatty liver disease (NAFLD)/NASH patients from 2011 to 2017 were identified from administrative databases of Italian local health units using ICD-9-CM codes. Development of compensated cirrhosis (CC), decompensated cirrhosis (DCC), hepatocellular carcinoma (HCC), or liver transplant (LT) was identified using first diagnosis date for each severity cohort (index-date). Patients progressing to multiple disease stages were included in >1 cohort. Patients were followed from index-date until the earliest of disease progression, end of coverage, death, or end of study. Within each cohort, per member per month values were annualized to calculate all-cause HCRU or costs() in 2017. Of the 9,729 hospitalized NAFLD/NASH patients identified, 97% were without AdvLD, 1.3% had CC, 3.1% DCC, 0.8% HCC, 0.1% LT. Comorbidity burden was high across all cohorts. Mean annual number of inpatient services was greater in patients with AdvLD than without AdvLD. Similar trends were observed in outpatient visits and pharmacy fills. Mean total annual costs increased with disease severity, driven primarily by inpatient services costs. CONCLUSION: NAFLD/NASH patients in Italy have high comorbidity burden. AdvLD patients had significantly higher costs. The higher prevalence of DCC compared to CC in this population may suggest challenges of effectively screening and identifying NAFLD/NASH patients. Early identification and effective management are needed to reduce risk of disease progression and subsequent HCRU and costs.
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Recursos em Saúde/economia , Custos Hospitalares , Hepatopatia Gordurosa não Alcoólica/economia , Hepatopatia Gordurosa não Alcoólica/terapia , Demandas Administrativas em Assistência à Saúde , Adolescente , Adulto , Idoso , Assistência Ambulatorial/economia , Carcinoma Hepatocelular/economia , Carcinoma Hepatocelular/epidemiologia , Carcinoma Hepatocelular/terapia , Comorbidade , Bases de Dados Factuais , Progressão da Doença , Custos de Medicamentos , Feminino , Recursos em Saúde/tendências , Custos Hospitalares/tendências , Humanos , Itália/epidemiologia , Cirrose Hepática/economia , Cirrose Hepática/epidemiologia , Cirrose Hepática/terapia , Neoplasias Hepáticas/economia , Neoplasias Hepáticas/epidemiologia , Neoplasias Hepáticas/terapia , Transplante de Fígado/economia , Masculino , Pessoa de Meia-Idade , Hepatopatia Gordurosa não Alcoólica/diagnóstico , Hepatopatia Gordurosa não Alcoólica/epidemiologia , Admissão do Paciente/economia , Prevalência , Prognóstico , Estudos Retrospectivos , Fatores de Risco , Fatores de Tempo , Adulto JovemRESUMO
BACKGROUND: In a phase 3 randomized, double-blind, placebo-controlled trial, treatment with idelalisib, a phosphoinositol-3 kinase δ inhibitor, + bendamustine/rituximab improved progression-free survival (PFS) and overall survival (OS) in adult patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL). Here we report the results of health-related quality of life (HRQL) analyses from this study. METHODS: From June 15, 2012 to August 21, 2014, 416 patients with R/R CLL were enrolled; 207 patients were randomized to the idelalisib arm and 209 to the placebo arm. In the 416 patients randomized to receive bendamustine/rituximab and either oral idelalisib 150 mg twice-daily or placebo, HRQL was assessed at baseline and throughout the blinded part of the study using the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) and EuroQoL Five-Dimension (EQ-5D) visual analogue scale (VAS) questionnaires. The assessments were performed at scheduled patient visits; every 4 weeks for the first 6 months from the initiation of treatment, then every 8 weeks for the next 6 months, and every 12 weeks thereafter until end of study. Least-squares mean changes from baseline were estimated using a mixed-effects model by including treatment, time, and treatment-by-time interaction, and stratification factors as fixed effects. Time to first symptom improvement was assessed by Kaplan-Meier analysis. RESULTS: In mixed-effects model analysis, idelalisib + bendamustine/rituximab treatment led to clinically meaningful improvements from baseline in leukemia-associated symptoms. Moreover, per Kaplan-Meier analysis, the proportion of patients with symptom improvement was higher and time to improvement was shorter among patients in the idelalisib-containing arm compared with those who did not receive idelalisib. The physical and social/family FACT-Leu subscale scores, along with the self-rated health assessed by EQ-VAS, showed improvement with idelalisib over placebo, but the difference did not reach statistical significance. The functional and emotional FACT-Leu subscale scores remained similar to placebo. CONCLUSIONS: Addition of idelalisib to bendamustine/rituximab, apart from improving PFS and OS, had a neutral to beneficial impact on HRQL in patients with R/R CLL, particularly by reducing leukemia-specific disease symptoms. TRIAL REGISTRATION: Clinicaltrials.gov NCT01569295. Registered April 3, 2012.
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Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Cloridrato de Bendamustina/administração & dosagem , Leucemia Linfocítica Crônica de Células B/tratamento farmacológico , Purinas/administração & dosagem , Qualidade de Vida , Quinazolinonas/administração & dosagem , Rituximab/administração & dosagem , Adulto , Idoso , Método Duplo-Cego , Feminino , Humanos , Estimativa de Kaplan-Meier , Masculino , Pessoa de Meia-IdadeRESUMO
Objective: Quantify proportion of human immunodeficiency virus (HIV)-infected patients with specific comorbidities receiving healthcare coverage from commercial, Medicaid, and Medicare payers. Methods: Data from MarketScan research databases were used to select adult HIV-infected patients from each payer. Treated HIV-infected patients were matched to HIV-negative controls. Cross-sectional analyses were performed between 2003 and 2013 among HIV-infected patients to quantify the proportion with individual comorbidities over the period, by payer. Results: Overall, 36298 HIV-infected patients covered by commercial payers, 26246 covered by Medicaid payers, and 1854 covered by Medicare payers were identified between 2003 and 2013. Essential hypertension (31.4%, 39.3%, and 76.2%, respectively), hyperlipidemia (29.2%, 22.1%, and 49.6%), and endocrine disease (21.8%, 27.2%, and 54.0%) were the most common comorbidities. Comparison of data from 2003 to data from 2013 revealed significant increases across payers in the percentage of patients with the comorbidities specified above (P < .05). Across all payers, the proportions of treated HIV-infected patients with deep vein thrombosis, hepatitis C, renal impairment, thyroid disease, and liver disease from 2003 to 2013 was significantly greater (P < .05) than for matched controls. Conclusions: Comorbidities are common among the aging HIV-infected population and have increased over time. There should be a consideration in treatment choices for HIV infection, including the choices of antiretroviral regimens.
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Comorbidade , Infecções por HIV/complicações , Adulto , Idoso , Idoso de 80 Anos ou mais , Estudos Transversais , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Estados Unidos/epidemiologiaRESUMO
BACKGROUND: Several major ESRD-related regulatory and reimbursement changes were introduced in the United States in 2011. In several large, national datasets, these changes have been associated with decreases in erythropoiesis stimulating agent (ESA) utilization and hemoglobin concentrations in the ESRD population, as well as an increase in the use of red blood cell (RBC) transfusions in this population. Our objective was to examine the use of RBC transfusion before and after the regulatory and reimbursement changes implemented in 2011 in a prevalent population of chronic dialysis patients in a large national claims database. METHODS: Patients in the Truven Health MarketScan Commercial and Medicare Databases with evidence of chronic dialysis were selected for the study. The proportion of chronic dialysis patients who received any RBC transfusion and RBC transfusion event rates per 100 patient-months were calculated in each month from January 1, 2007 to March 31, 2012. The results were analyzed overall and stratified by primary health insurance payer (commercial payer or Medicare). RESULTS: Overall, the percent of chronic dialysis patients with RBC transfusion and RBC transfusion event rates per 100 patient-months increased between January 2007 and March 2012. When stratified by primary health insurance payer, it appears that the increase was driven by the primary Medicare insurance population. While the percent of patients with RBC transfusion and RBC transfusion event rates did not increase in the commercially insured population between 2007 and 2012 they did increase in the primary Medicare insurance population; the majority of the increase occurred in 2011 during the same time frame as the ESRD-related regulatory and reimbursement changes. CONCLUSIONS: The regulatory and reimbursement changes implemented in 2011 may have contributed to an increase in the use of RBC transfusions in chronic dialysis patients in the MarketScan dataset who were covered by Medicare plus Medicare supplemental insurance.
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Transfusão de Eritrócitos/tendências , Hematínicos/uso terapêutico , Falência Renal Crônica/terapia , Medicare/tendências , Mecanismo de Reembolso/tendências , Diálise Renal/tendências , Estudos de Coortes , Transfusão de Eritrócitos/economia , Feminino , Hematínicos/economia , Humanos , Falência Renal Crônica/economia , Masculino , Medicare/economia , Mecanismo de Reembolso/economia , Diálise Renal/economia , Estudos Retrospectivos , Estados UnidosRESUMO
BACKGROUND: For symptomatic obstructive hypertrophic cardiomyopathy (oHCM), limited evidence exists on healthcare resource utilization (HRU) and cost for patients with symptomatic oHCM by treatment categories. We evaluated whether HRU and costs vary by initial treatment in symptomatic oHCM. METHODS: This is a retrospective study of medical and pharmacy claims from 2016 to 2021 to identify (per International Classification of Disease Tenth Revision diagnosis codes) adult patients in the USA with symptomatic oHCM. Patients included in the study cohort were required to be treatment naïve (≥ 12 months' activity before first treatment) and symptomatic (fatigue, chest pain, syncope, dyspnea, heart failure, or palpitations within 3 months of index date). Patients were grouped by first index treatment [beta blocker (BB), calcium channel blockers (CCB), disopyramide, combination therapy], and HRU and costs [per person per year (PPPY), in USD] by initial treatment were reported. RESULTS: Among 7334 patients with symptomatic oHCM, initial treatment included BB (65.8%), CCB (21.1%), disopyramide (1.2%), or BB + CCB (11.9%). Overall, 87.2% were prescribed monotherapy. Outpatient visits were the main driver of all-cause HRU (mean 11.5 PPPY), and varied by initial treatment (BB: 11.0, CCB: 10.5, disopyramide: 7.2, combination therapy: 12.1). All-cause urgent care visits were more frequent than inpatient visits (means: 5.4 and 0.83 PPPY, respectively). All-cause incurred costs were $46,628 PPPY overall and varied by treatment (BB: $47,029, CCB: $42,124, disopyramide: $27,007, combination therapy: $54,024). CONCLUSIONS: In this large, US-based cohort of patients with symptomatic oHCM, initial therapy was most commonly BB and CCB monotherapy. Costs and HRU were high for most patients, but greater for those treated initially with combination therapy.
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AIMS: Patients with obstructive hypertrophic cardiomyopathy (oHCM) experience significant clinical burden which is associated with a high economic burden. Peak oxygen uptake (pVO2), measured by cardiopulmonary exercise testing, is used to quantify functional capacity, and has been studied as a primary endpoint in recent clinical trials. This study aimed to gather evidence to consolidate the prognostic value of pVO2 in oHCM and to assess whether it is feasible to predict health outcomes in an economic model based on changes in pVO2. METHODS: A targeted literature review was conducted in MEDLINE (via PubMed) and Embase databases to identify evidence on the prognostic value of pVO2 as a surrogate health outcome to support future oHCM economic model development. Following screening, study characteristics, population characteristics, and pVO2 prognostic association data were extracted. RESULTS: A total of 4,687 studies were identified. In total, 3,531 and 538 studies underwent title/abstract and full-text screening, respectively, of which 151 were included and nine of these were in hypertrophic cardiomyopathy (HCM); only three studies focused on oHCM. The nine HCM studies consisted of one systematic literature review and eight primary studies reporting on 27 potentially predictive relationships from a pVO2-based metric with clinical outcomes including all-cause mortality, cardiovascular mortality, sudden cardiac death, transplant, paroxysmal, and permanent atrial fibrillation. pVO2 was described as a predictor of single and composite endpoints, in three and six studies, respectively, with one study reporting on both. LIMITATIONS: This study primarily uses systemic literature review methods but does not qualify as one due to not entailing parallel reviewers during title-abstract and full-text stages of review. CONCLUSION: The findings of this study suggest pVO2 is predictive of multiple health outcomes, providing a rationale to use pVO2 in the development of an economic model.
Obstructive hypertrophy cardiomyopathy (oHCM) is a condition where the heart muscle thickens, obstructing blood flow and potentially impacting health. Peak oxygen uptake (pVO2) measures the highest amount of oxygen consumption during peak exercise and serves as an indicator of fitness. pVO2 can be used to assess heart health and predict severe conditions and death, acting as a surrogate endpoint. Surrogate endpoints are valuable in drug investigations since they allow earlier decisions on drug approval and funding before longer-term patient follow-up is available.This study reviewed evidence on the relationship between pVO2 values in patients with heart disease and the risk of becoming sicker or dying. Our goal was to assess if these relationships had been established and whether it is feasible to use them to predict future treatment benefits and support economic evaluations of new treatments. Our review found that most studies reported on patients with heart failure, with only nine focusing on HCM. Evidence indicates that low pVO2 values in patients with heart disease are linked to an increased risk of developing other heart conditions, needing a heart transplant, or dying.
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Cardiomiopatia Hipertrófica , Teste de Esforço , Modelos Econômicos , Humanos , Consumo de Oxigênio , PrognósticoRESUMO
PURPOSE: Anemia in cancer patients can be treated with red blood cell (RBC) transfusions. The patient burden associated with a treatment in terms of total time spent is an important factor to consider when measuring the benefits and challenges of a therapy. This study estimates the time-related patient burden associated with outpatient RBC transfusion. METHODS: A retrospective chart review of outpatient cancer patients receiving a RBC transfusion was conducted at 10 US centers. RBC transfusion time was measured as time elapsed from pre- to post-transfusion vital sign assessment and from transfusion start to stop time. Elapsed time from hemoglobin level testing and blood draw for cross-match to transfusion, estimated travel time and distance, and clinical and demographic data were also collected. RESULTS: Data from 110 patients (48.2 % male; mean age 64 ± 12 years) showed that the mean elapsed time between pre- and post-vital sign assessment was 4.2 h (95 % confidence interval (CI), 3.64-4.81) including 3.6 h (95 % CI, 3.0-4.1) on average to receive the actual RBC transfusion treatment. Hemoglobin level testing (mean Hg level, 8.33 g/dL ± 0.67) and blood drawn for cross-match were completed in an average of 31.2 h (95 % CI, 17.0-45.5) and 18.2 h (95 % CI, 12.1-24.2) prior to transfusion, respectively. Patient one-way travel time averaged 30.0 min (95 % CI, 25.9-34.3). CONCLUSIONS: In the US, CIA patients experience an important time burden when being treated with RBC transfusion in addition to the burden already added by chemotherapy.
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Anemia/terapia , Antineoplásicos/administração & dosagem , Transfusão de Eritrócitos/métodos , Neoplasias/tratamento farmacológico , Adulto , Idoso , Idoso de 80 Anos ou mais , Assistência Ambulatorial/métodos , Anemia/etiologia , Estudos Transversais , Feminino , Hemoglobinas/metabolismo , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Fatores de Tempo , Transporte de PacientesRESUMO
PURPOSE: The erythropoiesis-stimulating agents (ESAs), darbepoetin alfa (DA), and epoetin alfa (EA) differ with respect to dosing schedule in chemotherapy-induced anemia. DA can be administered less frequently than EA, which may increase synchronicity between chemotherapy and ESA schedules. This study compared DA and EA with respect to frequency of synchronization and frequencies of total and ESA healthcare visits in current clinical practice. METHODS: A retrospective analysis of ESA utilization during ESA episodes of care was conducted on all cancer patients identified in the SDI health oncology electronic medical records database who underwent chemotherapy and received ESA therapy from July 1, 2007 to March 31, 2010 (n = 6522 DA, n = 3,439 EA). RESULTS: The frequency of synchronization (chemotherapy and ESA therapy on the same day) was higher with DA (67 %) than EA (58 %) (p < 0.001). The odds that an ESA administration was synchronized with chemotherapy were higher with DA compared with EA (odds ratio = 1.46, 95 % CI: 1.37, 1.54). Compared with EA, DA patients had 2.3 fewer visits with an ESA administration (p < 0.001) and 3.0 fewer total visits (p < 0.001). CONCLUSIONS: Compared with patients receiving EA, DA patients were more likely to have an ESA administration on the same healthcare visit as chemotherapy and had fewer visits for any cause or for ESA administration. These results suggest that through greater synchronization of ESA and chemotherapy administrations, DA may reduce patient and practice burden and healthcare utilization.
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Anemia/tratamento farmacológico , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Agendamento de Consultas , Eritropoese/efeitos dos fármacos , Hematínicos/administração & dosagem , Neoplasias/tratamento farmacológico , Adolescente , Adulto , Idoso , Instituições de Assistência Ambulatorial/estatística & dados numéricos , Anemia/induzido quimicamente , Antineoplásicos/efeitos adversos , Antineoplásicos/uso terapêutico , Darbepoetina alfa , Bases de Dados Factuais , Esquema de Medicação , Epoetina alfa , Eritropoetina/administração & dosagem , Eritropoetina/efeitos adversos , Eritropoetina/análogos & derivados , Feminino , Hematínicos/efeitos adversos , Humanos , Masculino , Pessoa de Meia-Idade , Proteínas Recombinantes/administração & dosagem , Proteínas Recombinantes/efeitos adversos , Estudos Retrospectivos , Adulto JovemRESUMO
OBJECTIVES: To characterize erythropoiesis-stimulating agent (ESA) usage initiated in hospital outpatient oncology centers that employ weekly (QW) and every-3-week (Q3W) ESA dosing regimens; describe the frequency of ESA dosing, transfusions, hemoglobin determinations, and anemia-related visits between these 2 regimens; and compare the rates at which inpatient ESA doses are administered on QW versus Q3W schedules. METHODS: This was a retrospective, observational record review evaluating ESA usage in 641 patients from 8 outpatient oncology clinics throughout the United States. Adult patients who initiated myelosuppressive chemotherapy for a documented solid tumor between August 1, 2007 and June 30, 2009 and received their first 3 consecutive outpatient ESA doses on a QW or Q3W schedule were eligible for study inclusion. During a single course of chemotherapy, ESA administrations were recorded as long as ESA therapy was continued on the initial regimen. ESA doses were captured until termination of ESA therapy, until 9 months had elapsed since the first ESA dose, until the patient was switched to another ESA regimen, or until death. ESA administration during inpatient admissions was also recorded. RESULTS: ESA utilization varied between the dosing groups, with fewer ESA doses administered per follow-up month in patients receiving Q3W versus QW ESA therapy (mean, 1 vs 2 doses). Compared to weekly administration, extended-dose ESA therapy also reduced the number of hemoglobin determinations and anemia-related visits without chemotherapy required per follow-up month. Neither the number of transfusions nor the number of packed red blood cell units administered per follow-up month differed between treatment groups. Compared to weekly ESA therapy, Q3W administration reduced costs associated with ESA prescribing and utilization. CONCLUSION: Extended-dose ESA therapy (Q3W dosing) may improve practice efficiency and may be associated with reduced frequencies of hemoglobin determinations and ESA doses required. Q3W dosing may also reduce inpatient ESA utilization by reducing the number of ESA doses required for previously maintained outpatients.
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Alquilantes/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Linfoma Folicular/tratamento farmacológico , Purinas/uso terapêutico , Quinazolinonas/uso terapêutico , Rituximab/uso terapêutico , Adulto , Fatores Etários , Idoso , Idoso de 80 Anos ou mais , Esquema de Medicação , Feminino , Humanos , Linfoma Folicular/mortalidade , Linfoma Folicular/patologia , Masculino , Pessoa de Meia-Idade , Segurança do Paciente , Recidiva , Fatores de Risco , Análise de Sobrevida , Resultado do TratamentoRESUMO
BACKGROUND: Nonalcoholic fatty liver disease (NAFLD) has reached high prevalence, paralleling the obesity pandemic. The aggressive form of the disease, nonalcoholic steatohepatitis (NASH), is characterized by fatty infiltration and inflammation of the liver, can progress to compensated cirrhosis (CC) and end-stage liver disease (ESLD: decompensated cirrhosis [DCC] and hepatocellular carcinoma [HCC]), and may ultimately require liver transplantation (LT). Real-world data on the burden of NAFLD/NASH are limited. This study aimed to evaluate the clinical and economic burden of NAFLD/NASH to the French hospital system. METHODS: This retrospective cohort study used data from the French PMSI-MCO database. Adults with NAFLD/NASH diagnosis identified between 2009 and 2015 were categorized into disease severity cohorts (NAFLD/NASH, CC, DCC, HCC, and LT). Demographic and clinical data were assessed at the index (diagnosis) date. Hospitalization resource utilization and costs were assessed in the pre- and post-index periods. Rates of liver disease progression and death were evaluated for each cohort. FINDINGS: During the median follow-up of 34.8 months, of the 131,656 patients included, 1491 patients developed CC (1.1%), 7846 developed DCC (5.9%), 1144 developed HCC (0.9%), and 52 required LT (0.04%). The diagnosis of NAFLD/NASH was associated with increasing annual costs: 7736 vs 5076 before the diagnosis. Rates of comorbidities, hospitalization resource utilization, and costs increased with disease progression. The rate of death at seven-year follow-up was 7.9% in NAFLD/NASH, CC: 18.0%, DCC: 34.9%, and HCC: 48.8%. INTERPRETATION: NAFLD/NASH is associated with high economic burden and imparts substantial risk of negative clinical outcomes and mortality at all stages of disease.
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The rising prevalence of nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) presents many public health challenges, including a substantial impact on healthcare resource utilization and costs. There are important regional differences in the burden of NAFLD/NASH, and Spain-specific data are lacking. This retrospective, observational study examined the impact of liver disease severity, comorbidities, and demographics on healthcare resource utilization and costs in Spain.NAFLD/NASH patients in the Spanish National Health System's Hospital Discharge Records Database (1/1/2006 to 4/30/2017) were categorized into disease severity cohorts as NAFLD/NASH overall, NAFLD/NASH non-progressors, compensated cirrhosis (CC), decompensated cirrhosis (DCC), liver transplant (LT), or hepatocellular carcinoma (HCC). Patients were followed from index date until the earliest of 6 months, disease progression, end of coverage, death, or end of study. Within each cohort, pre- and post-index healthcare resource utilization and costs per patient per month (PPPM) were calculated.A total of 8,205 patients (mean age 58.4; 54% male) were identified; 5,984 (72.9%) were non-progressors, 139 (1.7%) progressed to CC, 2,028 (24.7%) to DCC, 115 (1.4%) to LT, and 61 (0.7%) to HCC. Pre-index comorbidity burden was high across disease cohorts, and the frequency of comorbidities increased with disease severity. From pre- to post-index, average length of stay (LOS) increased significantly (23%-41%) as did all-cause PPPM costs (44%-46%), with significantly longer LOS and costs in patients with increasing disease severity.Progression of NAFLD/NASH was associated with significantly higher costs and longer LOS. A coordinated approach is needed to manage resources and costs in Spain.
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Hepatopatia Gordurosa não Alcoólica/epidemiologia , Índice de Gravidade de Doença , Adolescente , Adulto , Idoso , Estudos de Coortes , Bases de Dados Factuais , Feminino , Custos de Cuidados de Saúde , Humanos , Estudos Longitudinais , Masculino , Pessoa de Meia-Idade , Hepatopatia Gordurosa não Alcoólica/economia , Hepatopatia Gordurosa não Alcoólica/patologia , Estudos Retrospectivos , Espanha/epidemiologia , Adulto JovemRESUMO
BACKGROUND: Risk factors and timing associated with disease progression and mortality in nonalcoholic fatty liver disease (NAFLD) are poorly understood. AIMS: To evaluate the impact of disease severity, demographics and comorbidities on risk of mortality and time to progression in a large, real-world cohort of diagnosed NAFLD patients. METHODS: Claims data from a 20% Medicare representative sample between 2007 and 2015 were analysed retrospectively. Adults were categorised into disease severity groups: NAFLD/nonalcoholic steatohepatitis (NASH) alone, compensated cirrhosis, decompensated cirrhosis, liver transplant or hepatocellular carcinoma. Cumulative incidence of mortality and disease progression were calculated for each group and multivariate analyses performed adjusting for demographics, comorbidities and disease severity. RESULTS: A total of 10 826 456, patients were assessed and the prevalence of NAFLD was 5.7% (N = 621 253). Among patients with NAFLD, 71.1% had NAFLD/NASH alone and 28.9% had NAFLD cirrhosis. Overall, 85.5% of patients had hypertension, 84.1% dyslipidemia, 68.7% had cardiovascular disease and 55.5% diabetes. The cumulative risk of progression of NAFLD to cirrhosis, and compensated cirrhosis to decompensated cirrhosis was 39% and 45%, respectively, over 8 years of follow-up. The independent predictors of progression included cardiovascular disease, renal impairment, dyslipidemia and diabetes. The cumulative risk of mortality for NAFLD, NAFLD cirrhosis, decompensated cirrhosis and hepatocellular carcinoma was 12.6%, 31.1%, 51.4% and 76.2%, respectively. CONCLUSIONS: The present report (a) demonstrates that NAFLD is grossly underdiagnosed in real-world clinical settings and (b) provides new evidence on the progression rates of NAFLD and risk factors of mortality across the spectrum of severity of NAFLD and cirrhosis.
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Cirrose Hepática/epidemiologia , Cirrose Hepática/patologia , Falência Hepática/epidemiologia , Falência Hepática/mortalidade , Hepatopatia Gordurosa não Alcoólica/epidemiologia , Hepatopatia Gordurosa não Alcoólica/patologia , Adulto , Idoso , Carcinoma Hepatocelular/epidemiologia , Carcinoma Hepatocelular/mortalidade , Estudos de Coortes , Comorbidade , Progressão da Doença , Feminino , Humanos , Cirrose Hepática/mortalidade , Falência Hepática/patologia , Neoplasias Hepáticas/epidemiologia , Neoplasias Hepáticas/mortalidade , Masculino , Medicare/estatística & dados numéricos , Pessoa de Meia-Idade , Mortalidade , Hepatopatia Gordurosa não Alcoólica/complicações , Hepatopatia Gordurosa não Alcoólica/mortalidade , Prevalência , Estudos Retrospectivos , Estados Unidos/epidemiologiaAssuntos
Hepatopatia Gordurosa não Alcoólica , Idoso , Progressão da Doença , Fibrose , Humanos , Cirrose Hepática , Medicare , Estados UnidosRESUMO
BACKGROUND: Coformulated elvitegravir, cobicistat, emtricitabine, and tenofovir disoproxil fumarate (EVG/COBI/FTC/TDF; Stribild(®)) is a recommended integrase inhibitor-based regimen in treatment guidelines from the US Department of Health and Human Services and the British HIV Association. The purpose of this analysis was to determine the change in patient-reported symptoms over time among HIV-infected adults who switch to Stribild(®) versus those continuing on a protease inhibitor (PI) with FTC/TDF. METHODS: A secondary analysis was conducted on the STRATEGY-PI study (GS-US-236-0115, ClinicalTrials.gov NCT01475838), a randomized, open-label, phase 3b trial of HIV-infected adults taking a PI with FTC/TDF who were randomly assigned (2:1) either to Stribild(®) (switch) or continuation of their existing regimen (no-switch). Logistic regressions and longitudinal modeling were conducted to evaluate the relationship of treatment with bothersome symptoms. RESULTS: At week 4 as compared with baseline, the switch group experienced a statistically significantly lower prevalence in five symptoms (diarrhea/loose bowels, bloating/pain/gas in stomach, pain/numbness/tingling in hands/feet, nervous/anxious, and trouble remembering). The lower prevalence of diarrhea/loose bowels, bloating/pain/gas in stomach, and pain/numbness/tingling in hands/feet observed at week 4 was maintained over time. While there were no significant differences between groups in the prevalence of sad/down/depressed and problems with sex at week 4 or week 48, longitudinal models indicated the switch group had a statistically significantly decreased prevalence in both symptoms from week 4 to week 48. As compared with the no-switch group, higher levels of satisfaction with treatment were experienced by patients in the switch group at the first follow-up visit and at week 24. CONCLUSIONS: In this study sample, a switch from a ritonavir-boosted PI, FTC, and TDF regimen to coformulated EVG/COBI/FTC/TDF was associated with more treatment satisfaction and a reduction in the prevalence of patient-reported diarrhea/loose bowel symptoms, which was maintained over the 48-week study period.
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Terapia Antirretroviral de Alta Atividade/normas , Combinação Elvitegravir, Cobicistat, Emtricitabina e Fumarato de Tenofovir Desoproxila/efeitos adversos , Combinação Elvitegravir, Cobicistat, Emtricitabina e Fumarato de Tenofovir Desoproxila/uso terapêutico , Emtricitabina/uso terapêutico , Infecções por HIV/tratamento farmacológico , Ritonavir/uso terapêutico , Tenofovir/uso terapêutico , Adulto , Fármacos Anti-HIV/efeitos adversos , Fármacos Anti-HIV/uso terapêutico , Terapia Antirretroviral de Alta Atividade/efeitos adversos , Combinação de Medicamentos , Emtricitabina/efeitos adversos , Feminino , Humanos , Modelos Logísticos , Masculino , Avaliação de Resultados da Assistência ao Paciente , Satisfação do Paciente , Inibidores de Proteases/efeitos adversos , Inibidores de Proteases/uso terapêutico , Ritonavir/efeitos adversos , Tenofovir/efeitos adversosRESUMO
OBJECTIVE: To explore the relative efficiency of dialysis facilities in the United States and identify factors that are associated with efficiency in the production of dialysis treatments. DATA SOURCES/STUDY SETTING: Medicare cost report data from 4,343 free-standing dialysis facilities in the United States that offered in-center hemodialysis in 2010. STUDY DESIGN: A cross-sectional, facility-level retrospective database analysis, utilizing data envelopment analysis (DEA) to estimate facility efficiency. DATA COLLECTION/EXTRACTION METHODS: Treatment data and cost and labor inputs of dialysis treatments were obtained from 2010 Medicare Renal Cost Reports. Demographic data were obtained from the 2010 U.S. Census. PRINCIPAL FINDINGS: Only 26.6 percent of facilities were technically efficient. Neither the intensity of market competition nor the profit status of the facility had a significant effect on efficiency. Facilities that were members of large chains were less likely to be efficient. Cost and labor savings due to changes in drug protocols had little effect on overall dialysis center efficiency. CONCLUSIONS: The majority of free-standing dialysis facilities in the United States were functioning in a technically inefficient manner. As payment systems increasingly employ capitation and bundling provisions, these institutions will need to evaluate their efficiency to remain competitive.
Assuntos
Instituições de Assistência Ambulatorial/estatística & dados numéricos , Instituições de Assistência Ambulatorial/normas , Eficiência Organizacional , Diálise Renal , Idoso , Estudos Transversais , Humanos , Estudos Retrospectivos , Estados UnidosRESUMO
BACKGROUND AND OBJECTIVES: Red blood cell transfusion was previously the principle therapy for anemia in CKD but became less prevalent after the introduction of erythropoiesis-stimulating agents. This study used adaptive choice-based conjoint analysis to identify preferences and predictors of transfusion decision-making in CKD. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: A computerized adaptive choice-based conjoint survey was administered between June and August of 2012 to nephrologists, internists, and hospitalists listed in the American Medical Association Masterfile. The survey quantified the relative importance of 10 patient attributes, including hemoglobin levels, age, occult blood in stool, severity of illness, eligibility for transplant, iron indices, erythropoiesis-stimulating agents, cardiovascular disease, and functional status. Triggers of transfusions in common dialysis scenarios were studied, and based on adaptive choice-based conjoint-derived preferences, relative importance by performing multivariable regression to identify predictors of transfusion preferences was assessed. RESULTS: A total of 350 providers completed the survey (n=305 nephrologists; mean age=46 years; 21% women). Of 10 attributes assessed, absolute hemoglobin level was the most important driver of transfusions, accounting for 29% of decision-making, followed by functional status (16%) and cardiovascular comorbidities (12%); 92% of providers transfused when hemoglobin was 7.5 g/dl, independent of other factors. In multivariable regression, Veterans Administration providers were more likely to transfuse at 8.0 g/dl (odds ratio, 5.9; 95% confidence interval, 1.9 to 18.4). Although transplant eligibility explained only 5% of decision-making, nephrologists were five times more likely to value it as important compared with non-nephrologists (odds ratio, 5.2; 95% confidence interval, 2.4 to 11.1). CONCLUSIONS: Adaptive choice-based conjoint analysis was useful in predicting influences on transfusion decisions. Hemoglobin level, functional status, and cardiovascular comorbidities most strongly influenced transfusion decision-making, but preference variations were observed among subgroups.