Inhibitors and mortality in persons with nonsevere hemophilia A in the United States.
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SUBMITTER: Lim MY
PROVIDER: S-EPMC7556148 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:BackgroundJoint bleeding in hemophilia may eventually lead to joint damage. In nonsevere hemophilia, joint bleeds occur infrequently. Currently, knowledge on the joint status of patients with nonsevere hemophilia using objective imaging is limited.ObjectiveTo investigate the joint status in patients with nonsevere hemophilia A.MethodsThis cross-sectional study included patients with nonsevere hemophilia A aged 24-55 years. Joint status was assessed by magnetic resonance imaging (MRI) of the elbows, knees, and ankles and International Prophylaxis Study Group (IPSG) scores were calculated. Lifetime joint bleeding history was collected from medical files. The contribution of factors to joint outcome was explored using multivariable linear regression analysis.ResultsIn total, 51 patients were included, of whom 19 (37%) had moderate and 32 (63%) had mild hemophilia. Patients had a median age of 43 years (interquartile range [IQR] 32-50), a median factor VIII activity of 10 IU/dl (IQR 4-16) and a median annual joint bleeding rate (AJBR) of 0.0 (IQR 0.0-0.2). Soft-tissue changes (IPSG subscore > 0) in the elbows, knees, and ankles were present in 19%, 71%, and 71% of patients, respectively. Osteochondral changes (IPSG subscore > 0) in the elbows, knees, and ankles were present in 0%, 20%, and 35% of patients, respectively. In 14% of bleed-free joints, hemosiderin depositions were observed. Age and AJBRs were most strongly associated with the IPSG score.ConclusionThis study demonstrates that a substantial proportion of adults with nonsevere hemophilia has joint changes on MRI despite low joint bleeding rates.
| S-EPMC9314729 | biostudies-literature
Project description:BackgroundDesmopressin increases plasma factor VIII and von Willebrand factor levels in persons with nonsevere hemophilia A. Patients' perspectives on desmopressin are relevant to increase and optimize its suboptimal use. However, patients' views on desmopressin are not reported.ObjectivesTo evaluate the perspectives of persons with nonsevere hemophilia A on desmopressin use, barriers for its use, side effects, and their knowledge about desmopressin's efficacy and side effects.MethodsPersons with nonsevere hemophilia A were included in a cross-sectional, national, multicenter study. Questionnaires were filled out by adult patients and children aged ≥12 years themselves. Caretakers filled out questionnaires for children aged <12 years.ResultsIn total, 706 persons with nonsevere hemophilia A were included (544 mild, 162 moderate, [age range, 0-88 years]). Of 508 patients, 234 (50%) patients reported previous desmopressin use. Desmopressin was considered as at least moderately effective in 171 of 187 (90%) patients. Intranasal administration was the modality of choice for 138 of 182 (76%) patients. Flushing was the most reported side effect in 54 of 206 (26%) adults and 7 of 22 (32%) children. The most frequently reported advantage and disadvantage were the convenience of intranasal, out-of-hospital administration by 56% (126/227) and side effects in 18% (41/227), respectively. Patients' self-perceived knowledge was unsatisfactory or unknown in 28% (63/225).ConclusionOverall, desmopressin was most often used intranasally and considered effective, with flushing as the most common side effect. The most mentioned advantage was the convenience of intranasal administration and disadvantage was side effects. More information and education on desmopressin could answer unmet needs in patients with current or future desmopressin treatment.
| S-EPMC10439392 | biostudies-literature
Project description:BackgroundA range of treatments for patients with severe hemophilia A (HA) have been developed over the last decade, allowing for reduced frequency of administration and improved outcomes (joint health and breakthrough bleeding rates). While clinically effective, the cost effectiveness of these treatments has not been established.ObjectiveThis study presents a cost-effectiveness analysis of contemporary rFVIII treatments for severe HA patients without inhibitors.MethodsA published semi-Markov model was used to compare three different prophylaxis regimens: (1) extended half-life (EHL) recombinant Factor VIII (rFVIII) Fc-fusion protein (rFVIIIFc, Eloctate®, Sanofi), (2) EHL PEGylated rFVIII (PEG-rFVIII, Adynovate®, Takeda), and (3) standard half-life (SHL) rFVIII (antihemophilic factor [recombinant], Advate®, Takeda), used as a proxy for all SHL rFVIII treatments. Acquisition costs were included based on published dosing and weight data. Benefits were incorporated through published annualized bleeding rates, rates of target joint development/resolution, and improvements in the modified hemophilia joint health score. Results were presented as total, discounted costs, and quality-adjusted life-years (QALYs).ResultsrFVIIIFc was shown to provide the most QALYs (27.922) compared with both PEG-rFVIII (27.454) and SHL rFVIII (27.071), at lower costs. Discounted lifetime costs were estimated at US$18.235m (rFVIIIFc), US$20.198m (PEG-rFVIII), and US$18.285m (SHL rFVIII), and were predominantly affected by model settings related to acquisition costs, patient weight, and dosing.ConclusionsrFVIIIFc may offer a cost-effective option for severe HA patients. Uncertainties owing to the limited evidence base is the main limitation of the study.
| S-EPMC8611150 | biostudies-literature
Project description:Background:Age-associated conditions are increasingly common among persons living with human immunodeficiency virus (HIV) (PLWH). A longitudinal investigation of their accrual is needed given their implications on clinical care complexity. We examined trends in the co-occurrence of age-associated conditions among PLWH receiving clinical care, and differences in their prevalence by demographic subgroup. Methods:This cohort study was nested within the North American AIDS Cohort Collaboration on Research and Design. Participants from HIV outpatient clinics were antiretroviral therapy-exposed PLWH receiving clinical care (ie, ≥1 CD4 count) in the United States during 2000-2009. Multimorbidity was irreversible, defined as having ≥2: hypertension, diabetes mellitus, chronic kidney disease, hypercholesterolemia, end-stage liver disease, or non-AIDS-related cancer. Adjusted prevalence ratios (aPR) and 95% confidence intervals (CIs) comparing demographic subgroups were obtained by Poisson regression with robust error variance, using generalized estimating equations for repeated measures. Results:Among 22969 adults, 79% were male, 36% were black, and the median baseline age was 40 years (interquartile range, 34-46 years). Between 2000 and 2009, multimorbidity prevalence increased from 8.2% to 22.4% (Ptrend < .001). Adjusting for age, this trend was still significant (P < .001). There was no difference by sex, but blacks were less likely than whites to have multimorbidity (aPR, 0.87; 95% CI, .77-.99). Multimorbidity was the highest among heterosexuals, relative to men who have sex with men (aPR, 1.16; 95% CI, 1.01-1.34). Hypertension and hypercholesterolemia most commonly co-occurred. Conclusions:Multimorbidity prevalence has increased among PLWH. Comorbidity prevention and multisubspecialty management of increasingly complex healthcare needs will be vital to ensuring that they receive needed care.
| S-EPMC5889007 | biostudies-other
Project description:BACKGROUND: Despite the endemic nature of Echinococcus granulosus and Echinococcus multilocularis infection in regions of the United States (US), there is a lack of data on echinococcosis-related mortality. To measure echinococcosis-associated mortality in the US and assess possible racial/ethnic disparities, we reviewed national-death certificate data for an 18-year period. METHODOLOGY/PRINCIPAL FINDINGS: Echinococcosis-associated deaths from 1990 through 2007 were identified from multiple-cause-coded death records and were combined with US census data to calculate mortality rates. A total of 41 echinococcosis-associated deaths occurred over the 18-year study period. Mortality rates were highest in males, Native Americans, Asians/Pacific Islanders, Hispanics and persons 75 years of age and older. Almost a quarter of fatal echinococcosis-related cases occurred in residents of California. Foreign-born persons accounted for the majority of echinococcosis-related deaths; however, both of the fatalities in Native Americans and almost half of the deaths in whites were among US-born individuals. CONCLUSIONS/SIGNIFICANCE: Although uncommon, echinococcosis-related deaths occur in the US. Clinicians should be aware of the diagnosis, particularly in foreign-born patients from Echinococcus endemic areas, and should consider tropical infectious disease consultation early.
| S-EPMC3274497 | biostudies-other
Project description:The clinical presentation and outcome of sarcoidosis varies by race. However, the race difference in mortality outcome remains largely unknown.We studied mortality related to sarcoidosis from 1999 through 2010 by examining data on multiple causes of death from the National Center for Health Statistics. We compared the comorbid conditions between sarcoidosis-related deaths with deaths caused by car accidents (previously healthy control subjects) and rheumatoid arthritis (chronic disease control subjects) in both African Americans and Caucasians.From 1999 through 2010, sarcoidosis was reported as an immediate cause of death in 10,348 people in the United States with a combined overall mean age-adjusted mortality rate of 2.8 per 1 million person-years. Of these, 6,285 were African American and 3,984 Caucasian. The age-adjusted mortality rate for African Americans was 12 times higher than for Caucasians. African Americans died at an earlier age than Caucasians. African Americans living in the District of Columbia and North Carolina and Caucasians living in Vermont had higher mortality rates. Although the total sarcoidosis age-adjusted mortality rate had not changed over the 12 year period studied, this rate increased for Caucasians (R = 0.747, P = .005) but not for African Americans. Compared with the control groups, pulmonary hypertension was significantly more common in individuals with sarcoidosis.This nationwide population-based study exposes a significant difference in ethnicity and sex among people dying of sarcoidosis in the United States. Pulmonary hypertension investigation should be considered in all patients with sarcoidosis, especially African Americans.
| S-EPMC4314818 | biostudies-other
Project description:The ongoing COVID-19 pandemic has spotlighted the role of America's overcrowded prisons as vectors of ill health, but robust analyses of the degree to which high rates of incarceration impact population-level health outcomes remain scarce. In this paper, we use county-level panel data from 2927 counties across 43 states between 1983 and 2014 and a novel instrumental variable technique to study the causal effect of penal expansion on age-standardised cause-specific and all-cause mortality rates. We find that higher rates of incarceration have substantively large effects on deaths from communicable, maternal, neonatal, and nutritional diseases in the short and medium term, whilst deaths from non-communicable disease and from all causes combined are impacted in the short, medium, and long run. These findings are further corroborated by a between-unit analysis using coarsened exact matching and a simulation-based regression approach to predicting geographically anchored mortality differences.
| S-EPMC8193150 | biostudies-literature
Project description:OBJECTIVE:To compare cognitive performance among Japanese and American persons, aged 68?years and older, using two nationally representative studies and to examine whether differences can be explained by differences in the distribution of risk factors or in their association with cognitive performance. DESIGN:Nationally representative studies with harmonized collection of data on cognitive functioning. SETTING:Nihon University Japanese Longitudinal Study of Aging and the US Health and Retirement Study. PARTICIPANTS:A total of 1953 Japanese adults and 2959 US adults, aged 68?years or older. MEASUREMENTS:Episodic memory and arithmetic working memory are measured using immediate and delayed word recall and serial 7s. RESULTS:Americans have higher scores on episodic memory than Japanese people (0.72 points on a 20-point scale); however, when education is controlled, American and Japanese people did not differ. Level of working memory was higher in Japan (0.36 on a 5-point scale) than in the United States, and the effect of education on working memory was stronger among Americans than Japanese people. There are no differences over the age of 85?years. CONCLUSION:Even with large differences in educational attainment and a strong effect of education on cognitive functioning, the overall differences in cognitive functioning between the United States and Japan are modest. Differences in health appear to have little effect on national differences in cognition. J Am Geriatr Soc 68:354-361, 2020.
| S-EPMC7002215 | biostudies-literature
Project description:Hemophilia B is a rare congenital blood disorder characterized by factor IX deficiency. Clinical profiles of hemophilia B range from mild to severe forms of the disease. The objective of this study was to characterize the economic burden associated with differing clinical profiles of hemophilia B from a US health system perspective. Using the IBM MarketScan database (June 2011-February 2019), a claims-based algorithm was developed to identify 4 distinct profiles (mild, moderate, moderate-severe, and severe) in adult males with hemophilia B based on the frequency of hemorrhage events and factor IX replacement claims. Mean annual health care resource use (HRU) and costs were statistically compared between patients with hemophilia B (N = 454) and 1:1 demographic-matched controls (N = 454), both overall and with stratification by clinical profile. Compared with matched controls, patients with hemophilia B had a significantly higher comorbidity burden (Charlson Comorbidity Index, mean ± standard deviation [SD]: 0.9 ± 1.7 vs 0.3 ± 0.9, P < .001). Across all clinical profiles, patients with hemophilia B had significantly higher HRU vs matched controls (mean ± SD: 0.3 ± 0.6 vs 0.1 ± 0.3 inpatient admissions; 0.6 ± 1.2 vs 0.2 ± 0.6 emergency department visits; 17.7 ± 22.9 vs 8.0 ± 11.0 outpatient visits; all P < .001). Annual total health care costs per patient among patients with hemophilia B were more than 25-fold higher vs matched controls (mean ± SD: $201 635 ± $411 530 vs $7879 ± $29 040, respectively, P < .001). Annual total health care costs per patient increased with increasing severity (mean ± SD: mild, $80 811 ± $284 313; moderate, $137 455 ± $222 021; moderate-severe, $251 619 ± $576 886; severe, $632 088 ± $501 270). The findings of this study highlight the substantial burden of illness associated with hemophilia B.
| S-EPMC8045501 | biostudies-literature
Project description:BackgroundTreatment of patients with hemophilia has advanced over the past decades, but it is unknown whether this has resulted in a normal life expectancy in the Netherlands.ObjectiveThis observational cohort study aimed to assess all-cause and cause-specific mortality in patients with hemophilia in the Netherlands between 2001 and 2018 and to compare mortality and life expectancy with previous survival assessments from 1973 onward.Patients/methodsAll 1066 patients with hemophilia who participated in a nationwide survey in 2001 were followed until July 2018.ResultsInformation on 1031 individuals (97%) was available, of whom 142 (14%) deceased during follow-up. Compared with the general Dutch male population, mortality of patients with hemophilia was still increased (standardized mortality ratio: 1.4, 95% confidence interval: 1.2-1.7). Intracranial bleeding and malignancies were the most common causes of death. Estimated median life expectancy of patients with hemophilia was 77 years, 6 years lower than the median life expectancy of the general Dutch male population (83 years). Over the past 45 years, death rates of patients with hemophilia have consistently decreased, approaching the survival experience of the general population. Over the past decades, mortality due to human immunodeficiency virus and hepatitis C virus infections has decreased, death due to intracranial hemorrhages has increased, and death due to ischemic heart disease has remained consistently low over time.ConclusionsSurvival in patients with hemophilia in the Netherlands has improved over time but is still lower than that of the general population.
| S-EPMC7986360 | biostudies-literature