Background
Data on the prevalence and disease management of chronic urticaria (CU) and chronic spontaneous urticaria (CSU) in the pediatric population are scarce. This study assessed the prevalence ...of CU and CSU, and disease management among pediatric patients (0‐17 years).
Methods
A physician‐based online survey was conducted in 5 European countries (United Kingdom, Germany, Italy, France, and Spain) assessing the annual diagnosed prevalence, disease characteristics, and treatment patterns in the target population. Results are based on physician responses and analyzed using descriptive statistics. Prevalence estimates were calculated based on the number of CU/CSU pediatric patients diagnosed, seen, and treated by the respondents and extrapolated to the total pediatric population from each country.
Results
Across 5 European countries, the one‐year diagnosed prevalence of CU and CSU in pediatric patients was 1.38% (95% CI, 0.94‐1.86) and 0.75% (95% CI, 0.44‐1.08), respectively. Angioedema was reported in 6%‐14% of patients. A large proportion of CSU pediatric patients (40%‐60%) were treated with H1‐antihistamines at approved dose and 16%‐51% received H1‐antihistamines at higher doses. Approximately 1/3 of pediatric CSU patients remained uncontrolled with H1‐antihistamines at approved/higher doses. Other prescribed treatments were oral corticosteroids (10%‐28%) and topical creams (15%‐26%).
Conclusions
This study revealed a prevalence of CSU among pediatric population comparable to adults and also suggested an unmet need for approved treatments for inadequately controlled pediatric CSU patients. It is truly of concern that harmful (oral steroids) or insufficient (topical creams) treatments were frequently used despite better and guideline‐recommended alternatives.
To explore the published evidence on the link between treatment satisfaction and patients' compliance, adherence, and/or persistence.
Articles published from January 2005 to November 2010 assessing ...compliance, adherence, or persistence and treatment satisfaction were identified through literature searches in Medline, Embase, and PsycInfo. Abstracts were reviewed by two independent researchers who selected articles for inclusion. The main attributes of each study examining the link between satisfaction and adherence, compliance, or persistence were summarized.
The database searches yielded 1278 references. Of the 281 abstracts that met the inclusion criteria, 20 articles were retained. In the articles, adherence and compliance were often used interchangeably and various methods were used to measure these concepts. All showed a positive association between treatment satisfaction and adherence, compliance, or persistence. Sixteen studies demonstrated a statistically significant link between satisfaction and compliance or persistence. Of these, ten demonstrated a significant link between satisfaction and compliance, two showed a significant link between satisfaction and persistence, and eight demonstrated a link between either a related aspect or a component of satisfaction (eg, treatment convenience) or adherence (eg, intention to persist). An equal number of studies aimed at explaining compliance or persistence according to treatment satisfaction (n = 8) and treatment satisfaction explained by compliance or persistence (n = 8). Four studies only reported correlation coefficients, with no hypothesis about the direction of the link. The methods used to evaluate the link were varied: two studies reported the link using descriptive statistics, such as percentages, and 18 used statistical tests, such as Spearman's correlation or logistic regressions.
This review identified few studies that evaluate the statistical association between satisfaction and adherence, compliance, or persistence. The available data suggested that greater treatment satisfaction was associated with better compliance and improved persistence, and with lower regimen complexity or treatment burden.
Chronic idiopathic (also called spontaneous) urticaria (CIU/CSU) is the most common form of chronic urticaria and has been associated with impairment to health outcomes, although the effect has never ...been assessed using a nationally representative sample in the United States.
To assess the burden of CIU/CSU from the patients' perspective in terms of health related quality of life, impairment to work and nonwork activities, and health care resource use.
Data were obtained from the US National Health and Wellness Survey. Current use of a prescription for the treatment of chronic hives was used as a proxy for CIU/CSU. Patients with CIU/CSU in the proxy group were matched 1:4 to respondents without chronic hives using survey year, sex, age, and race. Generalized linear models were adjusted for comorbidities, smoking, body mass index, and health insurance status. Outcome measures included the Medical Outcomes Study 12-Item and 36-Item Short Form Health Surveys; self-reported depression, anxiety, and sleep difficulties; the Work Productivity and Activity Impairment questionnaire, and health care resource use.
After matching and adjustment for covariates, those currently using a prescription for chronic hives had mental component summary scores 5.7 points lower, physical component summary scores 6.5 points lower, and health utility scores 0.11 points lower than controls, as well as higher adjusted odds of reporting depression, anxiety, and sleep difficulties. Mean adjusted work impairment was approximately double in prescription-treated chronic hives relative to controls, as was frequency of health care visits.
Chronic hives substantially affects quality of life, nonwork activities, capacity to work, and health care use, providing further evidence of a high burden of CIU/CSU across multiple health outcomes and unmet need for effective treatment.
Background
The Urticaria Activity Score summed over 7 days (UAS7) assesses the itch severity and hive count in chronic spontaneous urticaria (CSU) using once- or twice-daily diary-based ...documentation.
Objective
The aim of this study was to evaluate the comparability of twice-daily versus once-daily versions of the UAS and the resulting UAS7 values.
Methods
Data came from the ASSURE-CSU study. The twice-daily and once-daily UAS7 was calculated from morning and evening ratings, as well as from exact 24-h evening ratings of hive count and itch severity, respectively. Three UAS7 scores were computed: UAS7 twice daily (UAS7
TD
), UAS7 once daily for maximum itch (UAS7
OD1MAX
), and UAS7 once daily for average itch (UAS7
OD2AVG
). UAS7 values were assigned to five score bands (0, 1–6, 7–15, 16–27, 28–42), reflecting urticaria-free to severe disease activity. The score values and score band ratios of the UAS7
TD
and UAS7
OD
versions were compared and assessed for correlation by weighted Cohen’s kappa statistics.
Results
Data from 614 patients were analyzed. All three versions of the UAS7 yielded very similar results, with a mean (standard deviation) UAS7
TD
, UAS7
OD1MAX
, and UAS7
OD2AVG
of 17.3 (10.49), 17.7 (8.90), and 16.2 (8.68), respectively. Correlation coefficients between UAS7
TD
and UAS7
OD1MAX
, UAS7
TD
and UAS7
OD2AVG
, and UAS7
OD1MAX
and UAS7
OD2AVG
were 0.94, 0.95, and 0.99, respectively, showing very high positive pairwise correlation. The weighted kappa coefficient,
κ
(95% confidence interval) was 0.78 (0.75–0.82) for UAS7
TD
versus UAS7
OD1MAX
, and 0.82 (0.78–0.85) for UAS7
TD
versus UAS7
OD2AVG
, demonstrating substantial agreement.
Conclusions
The once- and twice-daily UAS7 scores were highly consistent, supporting the use of either version when evaluating CSU activity.
Background
Chronic spontaneous urticaria (CSU) is associated with considerable burden, but data from European patients are limited.
Methods
This study is a retrospective, cross-sectional analysis of ...National Health and Wellness Survey data from the five largest EU countries (5EU: France, Germany, Italy, Spain, and the UK) collected between 2010 and 2013. Burden of disease for patients with CSU was estimated by comparing individuals currently treated for chronic urticaria (proxy CSU cases) with controls selected from respondents without chronic urticaria. Matching and regression models were used to quantify the impact of chronic urticaria on health-related quality of life, self-reported psychological complaints, work and activity impairment, and healthcare use.
Results
The sample included 175,923 respondents. Prevalence of diagnosed chronic urticaria was 0.5 and 0.2 % were treating the condition with a prescription. Cases (
N
= 369) had substantially lower (worse) regression-adjusted mean Mental Component Summary (40.2 vs. 45.4), Physical Component Summary (44.6 vs. 49.9), and SF-6D health utility scores (0.63 vs. 0.71; all
p
< 0.001) relative to controls (
N
= 1476), differences that exceed accepted minimally important differences for these measures. Depression, anxiety, and sleep difficulties were approximately twice as prevalent among those currently receiving treatment for chronic urticaria (all
p
< 0.001). Cases also had elevated presenteeism (31 vs. 17 %), overall work impairment (37 vs. 20 %), and impairment in non-work activities (42 vs. 26 %; all
p
< 0.01) relative to controls. Physician visits (9.1 vs. 4.9), emergency room visits (0.8 vs. 0.3), and hospitalizations (0.3 vs. 0.2) were more frequent than in controls (all
p
< 0.01).
Conclusions
This research adds to the existing evidence showing significant burden of CSU.
Abstract
Objective
To develop a computer model to predict patients with nonalcoholic steatohepatitis (NASH) using machine learning (ML).
Materials and Methods
This retrospective study utilized two ...databases: a) the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) nonalcoholic fatty liver disease (NAFLD) adult database (2004-2009), and b) the Optum® de-identified Electronic Health Record dataset (2007-2018), a real-world dataset representative of common electronic health records in the United States. We developed an ML model to predict NASH, using confirmed NASH and non-NASH based on liver histology results in the NIDDK dataset to train the model.
Results
Models were trained and tested on NIDDK NAFLD data (704 patients) and the best-performing models evaluated on Optum data (~3,000,000 patients). An eXtreme Gradient Boosting model (XGBoost) consisting of 14 features exhibited high performance as measured by area under the curve (0.82), sensitivity (81%), and precision (81%) in predicting NASH. Slightly reduced performance was observed with an abbreviated feature set of 5 variables (0.79, 80%, 80%, respectively). The full model demonstrated good performance (AUC 0.76) to predict NASH in Optum data.
Discussion
The proposed model, named NASHmap, is the first ML model developed with confirmed NASH and non-NASH cases as determined through liver biopsy and validated on a large, real-world patient dataset. Both the 14 and 5-feature versions exhibit high performance.
Conclusion
The NASHmap model is a convenient and high performing tool that could be used to identify patients likely to have NASH in clinical settings, allowing better patient management and optimal allocation of clinical resources.
The NASHmap model is a non-invasive tool using 14 variables (features) collected in standard clinical practice to classify patients as probable nonalcoholic steatohepatitis (NASH) or non-NASH, and ...here we have explored its performance and prediction accuracy. The National Institute of Diabetes and Digestive Kidney Diseases (NIDDK) NAFLD Adult Database and the Optum Electronic Health Record (EHR) were used for patient data. Model performance metrics were calculated from correct and incorrect classifications for 281 NIDDK (biopsy-confirmed NASH and non-NASH, with and without stratification by type 2 diabetes status) and 1,016 Optum (biopsy-confirmed NASH) patients. NASHmap sensitivity in NIDDK is 81%, with a slightly higher sensitivity in T2DM patients (86%) than non-T2DM patients (77%). NIDDK patients misclassified by NASHmap had mean feature values distinct from correctly predicted patients, particularly for aspartate transaminase (AST; 75.88 U/L true positive vs 34.94 U/L false negative), and alanine transaminase (ALT; 104.09 U/L vs 47.99 U/L). Sensitivity was slightly lower in Optum at 72%. In an undiagnosed Optum cohort at risk for NASH (n = 2.9 M), NASHmap predicted 31% of patients as NASH. This predicted NASH group had AST and ALT mean levels above normal range of 0-35 U/L, and 87% had HbA1C levels > 5.7%. Overall, NASHmap demonstrates good sensitivity in predicting NASH status in both datasets, and NASH patients misclassified as non-NASH by NASHmap have clinical profiles closer to non-NASH patients.
H1-antihistamines (H1AH) are the first-line treatment for chronic spontaneous urticaria (CSU), but 50% of patients have inadequate disease control at standard doses.
To assess the comorbidity burden ...and healthcare resource utilization (HRU) associated with non-response to H1AH-based treatments; to identify predictors of non-response.
Optum® de-identified Electronic Health Record dataset (2007-2020) was used to identify adult patients with CSU who initiated a H1AH, alone or in combination with other oral non-biologics (index treatment). Based on twelve-month treatment patterns observed after index treatment initiation, patients were categorized as responders (continued index treatment or had only 1 next H1AH treatment without corticosteroids) or non-responders (continued corticosteroids or had 2 or more treatment switches). Patient characteristics and HRU were assessed in the 12 months before (baseline) and ≥12 months after (follow-up) index treatment initiation. Baseline predictors associated with non-response were identified using machine learning.
There were 17 062 patients who met inclusion criteria, and 14824 (86.9%) were classified as non-responders. A higher proportion of non-responders had records of CSU-related symptoms, comorbidities, polypharmacy, and certain laboratory tests than responders at baseline. A higher proportion of non-responders than responders visited an allergist or dermatologist during follow-up (59.5% vs 53.0%). Non-responders had a larger increase in hospitalizations (15.7% vs -2.4%) than responders during follow-up vs baseline. Predictors of non-response included index and baseline treatment classes, types of specialists seen, chronic pulmonary disease, depression, and female sex.
A large proportion of CSU patients treated with H1AH-based therapies had uncontrolled disease, contributing to increased HRU and patient burden. Non-responders had more comorbidities and HRU at baseline and follow-up, with steep increases in follow-up hospitalizations relative to baseline, highlighting an urgent need for early disease control.
Real-World Burden of Nonalcoholic Steatohepatitis Geier, Andreas; Rinella, Mary E.; Balp, Maria-Magdalena ...
Clinical gastroenterology and hepatology,
05/2021, Letnik:
19, Številka:
5
Journal Article
Recenzirano
Odprti dostop
Nonalcoholic steatohepatitis (NASH) is associated with an increase in healthcare resource use and poor health-related quality of life (HRQoL). We assessed the humanistic and economic burden of NASH, ...disease management, and patient journey.
We performed a cross-sectional analysis of data, collected from July through November 2017, from the Growth from Knowledge Disease Atlas Real-World Evidence program, reported by physicians in United States, France, and Germany. We extracted demographic and medical data from medical records. Some patients voluntarily completed a survey that provided information on disease history, treatment satisfaction, and patient-reported outcomes.
We analyzed data from 1216 patients (mean age, 54.9±12.3 years; 57.5% male; mean body mass index, 31.7±6.9); 64.6% had biopsy-confirmed NASH and comorbidities were recorded for 41.3%. Treatments included lifestyle modification (64.6%) or use of statins (25.0%), vitamin E (23.5%), or metformin (20.2%). Patients with biopsy-confirmed NASH reported more physician (4.5 vs 3.7) and outpatient visits (1.8 vs1.4) than patients with suspected NASH not confirmed by biopsy. Among the 299 patients who completed the survey, 47.8% reported various symptoms associated to their NASH. Symptomatic patients reported significantly lower HRQoL than patients without symptoms.
In an analysis of data from 3 countries, we found NASH to be associated with regular use of medical resources; patients with symptoms of NASH had reduced HRQoL. The burden of NASH appears to be underestimated. Studies are needed to determine the burden of NASH by fibrosis stage and disease severity.
Background
Chronic spontaneous urticaria (CSU) negatively impacts patients' sleep, thereby reducing health‐related quality of life (HRQoL). Half of patients with inadequately controlled CSU report ...sleep interference often or every night, which can lead to depression, anxiety, social, and work‐related problems.
Methods
This randomized, double‐blind, placebo‐controlled Phase 2b core study (NCT02477332) included adult patients ≥18 years with moderate to severe CSU inadequately controlled with H1‐antihistamines. The current analysis includes patients randomized to receive ligelizumab 72 or 240 mg, omalizumab 300 mg or placebo every 4 weeks (q4w) for five injections over 20 weeks with treatment‐free follow‐up for 24 weeks. Patients could enter the open‐label extension study (NCT02649218) from Week 32 onwards if their weekly urticaria activity score was ≥12, which included an open‐label treatment (52 weeks of ligelizumab 240 mg q4w) and a 48‐week post‐treatment follow‐up. Weekly Sleep Interference Scores (SIS7, range 0 no interference–21 substantial interference), Weekly Activity Interference Score (AIS7), Dermatology Life Quality Index (DLQI) scores, and Overall Work Impairment were assessed.
Results
Mean baseline SIS7 scores were balanced between the treatment arms for ligelizumab 72 mg (n = 84) and 240 mg (n = 85), omalizumab 300 mg (n = 85), and placebo (n = 43). By Week 12, patients experienced large improvements in sleep interference, with least square mean (standard error) changes from baseline (CFB) in SIS7 of −7.84 (0.58), −7.55 (0.61), −6.98 (0.60), and −5.85 (0.81), respectively. By Week 12, CFB in AIS7 were −8.25 (0.57), −8.25 (0.59), −7.30 (0.60), and −5.62 (0.79), DLQI scores were −9.79 (0.77), −9.93 (0.81), −8.35 (0.79), and −6.99 (1.11), and Overall Work Impairment scores were −28.96 (3.73), −30.76 (3.71), −25.74 (3.91), and −20.13 (5.10) for ligelizumab 72 and 240 mg, omalizumab 300 mg and placebo, respectively. Improvements in each patient‐reported outcome were sustained with ligelizumab 240 mg treatment during the extension study.
Conclusions
Ligelizumab showed effective and sustained responses in managing sleep interference in patients with CSU, and numerically higher responses than with omalizumab and placebo. Treating the symptoms of CSU with ligelizumab improved disease burden, HRQoL, and markedly improved sleep quality.
PDF
