UNASSIGNED: To understand treatment patterns, healthcare resource utilization (HCRU), and economic burden of diffuse large B-cell lymphoma (DLBCL) in elderly adults in the US.
UNASSIGNED: This retrospective database analysis utilized US Centers for Medicare and Medicaid Services Medicare fee-for-service administrative claims data from 2015 to 2020 to describe DLBCL patient characteristics, treatment patterns, HCRU, and costs among patients aged ≥66 years. Patients were indexed at DLBCL diagnosis and required to have continuous enrollment from 12 months pre-index until 3 months post-index. HCRU and costs (USD 2022) are reported as per-patient per-month (PPPM) estimates.
UNASSIGNED: A total of 11,893 patients received ≥1-line (L) therapy; 1633 and 391 received ≥2L and ≥3L therapy, respectively. Median (Q1, Q3) age at 1L, 2L, and 3L initiation, respectively, was 76 (71, 81), 77 (72, 82), and 77 (72, 82) years. The most common therapy was R-CHOP (70.9%) for 1L and bendamustine ± rituximab for 2L (18.7%) and 3L (17.4%). CAR T was used by 14.8% of patients in 3L. Overall, 39.6% (1L), 42.1% (2L), and 47.8% (3L) of patients had all-cause hospitalizations. All-cause mean (median [Q1-Q3]) costs PPPM during each line were $22,060 ($20,121 [$16,676-$24,597]) in 1L, $30,027 ($20,868 [$13,416-$31,016]) in 2L, and $47,064 ($25,689 [$15,555-$44,149]) in 3L, with increasing costs driven primarily by inpatient expenses. Total all-cause 3L mean (median [Q1-Q3]) costs PPPM for patients with and without CAR T were $153,847 ($100,768 [$26,534-$253,630]) and $28,466 ($23,696 [$15,466-$39,107]), respectively.
UNASSIGNED: No clear standard of care exists in 3L therapy for older adults with relapsed/refractory DLBCL. The economic burden of DLBCL intensifies with each progressing line of therapy, thus underscoring the need for additional therapeutic options.

BACKGROUND: Patients with mucopolysaccharidosis (MPS) often face delayed diagnoses, limited treatment options and high healthcare costs, that may significantly affect patients\' quality of life. The objective of this study was to understand medical service utilization related to diagnosis and treatment, economic burden during diagnosis period, and health-related quality of life among MPS patients in China.
METHODS: A series of patients diagnosed with MPS registered in the national patient organization were recruited for a cross-sectional survey from May to July 2019. Information were collected from patients or their parents via phone interview, including demographic data, utilization of services related to diagnosis and treatment, total cost during the period of MPS diagnosis and health-related quality of life (HRQoL). HRQoL was assessed by PedsQL 4.0 Generic Core Scale (PedsQL) and 36-item short-form health survey (SF-36) depending on the age of patients with MPS and compared with the general Chinese population.
RESULTS: A total of 180 MPS patients (50, 67, 15, 46, 1 and 1 for type I, II, III, IV, VI and VII), with a mean age of 9.54 years and 137 (76.11%) males, were included in analysis. The mean age at first visit to a medical doctor for MPS related symptoms was 3.65 ± 2.58 years old, while only 12 patients (6.67%) were diagnosed on their first visit. The mean diagnostic delay, which is defined as the time between the first visit to a medical doctor for MPS related symptoms and the final diagnosis, was 9.42 months, with no significant difference between types. The average number of misdiagnosis was 4.56. Before the confirmed diagnosis, the patients made an average of 6.31 visits and visited 4.3 hospitals. During diagnosis period, the mean of ¥81,086.72 direct medical costs accounted for 63.75% of the total cost. Only 32.78% of the patients had ever received specific treatments. The mean scores of PedsQL and SF-36 of patients were significantly lower than the Chinese norms. Household annual income per person, specific treatment use and MPS subtype were significantly associated HRQoL of patients.
CONCLUSIONS: The results highlight challenges faced by MPS patients in terms of diagnosis, access to specific treatments, economic burden and low HRQoL. There is an urgent need to improve early detection and diagnosis, create fair and consistent mechanisms to increase access to specialized treatment and reduce the economic burden of MPS patients in China.

BACKGROUND: Schizophrenia is a disorder associated with neurocognitive deficits that adversely affect daily functioning and impose an economic burden. Cognitive rehabilitation interventions, particularly during the early phases of illness, have been shown to improve cognition, functionality, and quality of life. The Feuerstein Instrumental Enrichment (FIE) program, based on the Mediated Learning Experience and the Structural Cognitive Modifiability theory, has been applied in various disorders, but its applicability in schizophrenia has not yet been clarified.
OBJECTIVE: This study aims to investigate the effects of the FIE program on the functionality of patients with first-episode schizophrenia.
METHODS: In total, 17 patients will be recruited for an open-label intervention consisting of twice-weekly sessions for 10 weeks. The primary outcome measure will be changes in the Goal Achievement Scale score. Maze task performance from the Measurement and Treatment Research to Improve Cognition in Schizophrenia (MATRICS) battery will serve as a secondary outcome measure. At the same time, changes in Positive and Negative Syndrome Scale scores and other MATRICS domains will be analyzed as exploratory outcomes. Assessments will be administered before and after the intervention, with a follow-up period of 6 months.
RESULTS: This trial was preregistered in The Brazilian Registry of Clinical Trials (RBR-4gzhy4s). By February 2024, 11 participants were enrolled in the training. Recruitment is expected to be completed by May 2024. Data analysis will be conducted between May and September 2024. The results are expected to be published in January 2025.
CONCLUSIONS: This study may establish a protocol for the FIE program that uses mediation techniques for individuals in the early stages of schizophrenia. The results will add to the knowledge about strategies to promote cognitive skills and functional impairment in daily life.
UNASSIGNED: DERR1-10.2196/57031.

BACKGROUND: The substantial economic burden of acute myeloid leukemia (AML) could be reduced with post-remission maintenance therapies that delay relapse. Real-world healthcare resource utilization (HCRU) data and costs among patients with AML receiving oral azacitidine (Oral-AZA) maintenance therapy or no maintenance are not well understood. We characterize HCRU and costs among these patients in clinical practice in the USA.
METHODS: Data from IQVIA PharMetrics® Plus (January 1, 2016-June 30, 2022) were used. Patients ≥ 18 years who were newly diagnosed with AML, received first-line systemic induction therapy, and attained disease remission were eligible. Patients receiving Oral-AZA maintenance and those receiving no maintenance (\"watch and wait\" [W&W]) were matched 1:3 on baseline characteristics using propensity score matching (PSM) and followed until hematopoietic stem cell transplantation or end of continuous insurance enrollment, whichever occurred first. Outcomes included treatment patterns, inpatient and outpatient visits, and costs.
RESULTS: After PSM, the Oral-AZA cohort included 43 patients and the W&W cohort 129. Of the 43 patients receiving Oral-AZA, 88.4% started at the recommended dose of 300 mg and 11.6% at 200 mg. The Oral-AZA cohort had significantly (p = 0.0025) longer median (95% CI) time to relapse from the index maintenance date (median not reached [NR; 9.0 months-NR] vs 3.3 months [0.8 months-NR]), and fewer per person per month (PPPM) hospitalizations (0.23 vs 0.61; p = 0.0005) and overall outpatient visits (5.77 vs 7.58; p = 0.0391) than the W&W cohort. Despite higher AML drug costs PPPM in the Oral-AZA cohort ($16,401 vs $10,651 for W&W), total healthcare costs PPPM were lower ($25,786 vs $38,530 for W&W; p < 0.0001).
CONCLUSIONS: Patients with newly diagnosed AML treated with Oral-AZA maintenance in clinical practice had prolonged remission and lower HCRU and costs than patients receiving no maintenance therapy. These findings underscore the clinical and economic value of Oral-AZA in clinical practice.

UNASSIGNED: Neurofibromatosis type 1 (NF1) is a rare genetic disorder, with lack of evidence of disease burden in China. We aimed to describe the economic burden, health-related quality of life (HRQL), and caregiver burden of NF1 patients in China.
UNASSIGNED: We conducted an online cross-sectional survey employing the China Cloud Platform for Rare Diseases, with 223 caregivers of NF1 pediatric patients (patients under 18), and 226 adult patients. Economic burden was estimated using direct and indirect costs related to NF1 in 2021, and the Work Productivity and Activity Impairment Questionnaire: General Health V2.0 (WPAI-GH). HRQL measures included EQ-5D-Y proxy version and PedsQL™ 4.0 Generic Core Scales (PedsQL GCS) proxy version for pediatric patients, and EQ-5D-5L and PedsQL™ 3.0 Neurofibromatosis Module (PedsQL NFM) for adult patients. Caregiver burden was estimated by Zarit Burden Interview (ZBI).
UNASSIGNED: For pediatric patients, the average direct cost in 2021 was CNY 33,614 (USD 4,879), and employed caregivers\' annual productivity loss was 81 days. EQ-5D-Y utility was 0.880 ± 0.13 and VAS score was 75.38 ± 20.67, with 52.6% patients reporting having problems in \"pain/discomfort\" and 42.9% in \"anxiety/depression.\" PedsQL GCS total score was 68.47 ± 19.42. ZBI score demonstrated that 39.5% of caregivers had moderate-to-severe or severe burden. For adult patients, average direct cost in 2021 was CNY 24,531 (USD 3,560). Patients in employment reported an absenteeism of 8.5% and presenteeism of 21.6% according to the results of WPAI-GH. EQ-5D-5L utility was 0.843 ± 0.17 and VAS score was 72.32 ± 23.49, with more than half of patients reporting having problems in \"pain/discomfort\" and \"anxiety/depression\" dimensions. PedsQL NFM total score was 68.40 ± 15.57.
UNASSIGNED: Both pediatric and adult NF1 patients in China had a wide-ranging economic burden and low HRQL, especially in the psychological dimension. Caregivers for NF1 pediatric patients experienced considerable caregiver burden. More attention and support from policymakers and stakeholders are required to relieve NF1 patients\' and caregivers\' distress.

BACKGROUND: Non-communicable diseases (NCDs) predispose households to exorbitant healthcare expenditures in health systems where there is no access to effective financial protection for healthcare. This study assessed the economic burden associated with the rising burden of type-2 diabetes (T2D) and hypertension comorbidity management, and its implications for healthcare seeking in urban Accra.
METHODS: A convergent parallel mixed-methods study design was used. Quantitative sociodemographic and cost data were collected through survey from a random community-based sample of 120 adults aged 25 years and older and living with comorbid T2D and hypertension in Ga Mashie, Accra, Ghana in November and December 2022. The monthly economic cost of T2D and hypertension comorbidity care was estimated using a descriptive cost-of-illness analysis technique from the perspective of patients. Thirteen focus group discussions (FGDs) were conducted among community members with and without comorbid T2D and hypertension. The FGDs were analysed using deductive and inductive thematic approaches. Findings from the survey and qualitative study were integrated in the discussion.
RESULTS: Out of a total of 120 respondents who self-reported comorbid T2D and hypertension, 23 (19.2%) provided complete healthcare cost data. The direct cost of managing T2D and hypertension comorbidity constituted almost 94% of the monthly economic cost of care, and the median direct cost of care was US$19.30 (IQR:10.55-118.88). Almost a quarter of the respondents pay for their healthcare through co-payment and insurance jointly, and 42.9% pay out-of-pocket (OOP). Patients with lower socioeconomic status incurred a higher direct cost burden compared to those in the higher socioeconomic bracket. The implications of the high economic burden resulting from self-funding of healthcare were found from the qualitative study to be: 1) poor access to quality healthcare; (2) poor medication adherence; (3) aggravated direct non-medical and indirect cost; and (4) psychosocial support to help cope with the cost burden.
CONCLUSIONS: The economic burden associated with healthcare in instances of comorbid T2D and hypertension can significantly impact household budget and cause financial difficulty or impoverishment. Policies targeted at effectively managing NCDs should focus on strengthening a comprehensive and reliable National Health Insurance Scheme coverage for care of chronic conditions.

UNASSIGNED: Recent trials have confirmed the effectiveness of promising dengue control technologies - two vaccines and Wolbachia. These would generally be applied at the municipal level. To help local officials decide which, if any, control strategy to implement, they need affordable, timely, and accurate data on dengue burden. Building on our previous work in Mexico, Indonesia, and Thailand, we developed a streamlined prospective method to estimate dengue burden at the municipal level quickly, accurately, and efficiently.
UNASSIGNED: The method entails enrolling and repeatedly interviewing 100 patients with laboratory-confirmed dengue. They will be selected after screening and testing about 1,000 patients with clinical dengue. The method will capture both acute and chronic effects relating to disease, economic burden, and psychological impacts (presenteeism). The total time requirements are 1.5 years, comprised of 0.25 years for planning and approvals, 1 year for data collection (a full dengue cycle), and 0 .25 years for data cleaning and analysis. A collaboration with municipal and academic colleagues in the city of Semarang, Central Java, Indonesia shows how the method could be readily applied in Indonesia\'s eighth largest city (population 1.8 million).
UNASSIGNED: Many surveillance studies gather only information on numbers of cases. This proposed method will provide a comprehensive picture of the dengue burden to the health system, payers, and households at the local level.

OBJECTIVE: This study aimed to determine the direct and indirect costs of sickle cell disease (SCD) in Saudi Arabia.
METHODS: Data were collected from 217 participants aged ≥18 years in the eastern region of Saudi Arabia, using a prevalence-based cost-of-illness approach. The Institute for Medical Technology Assessment Medical Consumption Questionnaire and Institute for Medical Technology Assessment Productivity Cost Questionnaire were used to assess costs. A multistage process was used, encompassing patient data collection over 3 months, cost calculation from clinic visits and drug prices, and extrapolation for annual estimates.
RESULTS: The study revealed substantial societal costs of SCD, with an average per-patient cost of SAR181 899 (US$48 506), covering healthcare and productivity losses. Healthcare costs, including hospitalization, informal care, and medication, averaged SAR80 306 (US$21 415). In addition, productivity costs, including unpaid work and presenteeism, averaged SAR101 594 (US$27 092). Obtaining higher levels of education, such as a diploma and BSc degree or higher, has been found to significantly decrease the costs associated with SCD (P = .016, P = .001). Furthermore, when comparing different employment statuses, students (B = -0.301, P = .058) were found to have marginally lower SCD costs, suggesting that their expenses were lower than those of individuals in other employment categories. The predictive model used in this study explained 11.2% of the variation in costs.
CONCLUSIONS: Our study highlights a significant economic burden of SCD in Saudi Arabia and highlights the need for targeted strategies to alleviate financial challenges and improve patient well-being.

Idiopathic hypersomnia (IH) is a rare neurological sleep disorder, characterized by excessive daytime sleepiness despite normal sleep duration, that can significantly impact patient\'s lives. The burden of IH goes beyond excessive daytime sleepiness, pervading all aspects of everyday life. Characteristic and burdensome symptoms of IH include sleep inertia/drunkenness, long sleep duration, and daytime cognitive dysfunction. This systematic review assessed current knowledge regarding IH diagnostic challenges and burden of illness. Literature searches for original epidemiological, clinical, humanistic, or economic research relevant to IH published between 2012 and 2022 in MEDLINE, Embase, Cochrane, gray literature (diagnostic criteria and treatment guidelines), conferences (2019-2022), and clinical trial databases yielded 97 articles. Findings indicate that IH remains a poorly defined diagnosis of exclusion that is difficult to distinguish from narcolepsy type 2 because of symptom overlap and inadequacies of objective testing. Consequently, individuals with IH endure diagnostic delays of up to 9 years. The economic burden of IH has not been characterized to any appreciable extent. Pharmacological treatment options can improve symptoms and functional status, but rarely restores normal levels of functioning. These findings highlight the need to reclassify central disorders of hypersomnolence. Further collaboration is now required between research groups to identify and validate objective markers to help redefine diagnostic criteria for IH. This would move IH into a position that could benefit from future targeted therapeutic interventions. The study was funded by Takeda Development Center Americas, Inc.

OBJECTIVE: To compare the clinical, economic, and health utility outcomes associated with alternative cystoscopic surveillance regimens for high-risk non-muscle-invasive bladder cancer (HRNMIBC).
METHODS: We performed real-world clinical data-driven microsimulations of a hypothetical cohort of 100 000 patients diagnosed with HRNMIBC at age 70 years. The cohort was simulated to undergo alternative surveillance regimens recommended by five guidelines, and two hypothetical regimens-surveillance intensity escalation and de-escalation-which had a surveillance intensity moderately higher and lower, respectively, than the guideline-recommended regimens. We evaluated the 10-year cumulative incidence of muscle-invasive bladder cancer (MIBC), cancer-specific survival (CSS), overall survival (OS), and cost-effectiveness from a United States healthcare payer perspective.
RESULTS: The guideline-recommended surveillance regimens led to an estimated 10-year cumulative incidence of MIBC ranging from 11.0% to 11.6%, CSS 95.0% to 95.2%, and OS 69.7% to 69.8%. Surveillance intensity escalation resulted in a 10-year cumulative incidence of MIBC of 10.5% (95% confidence interval [CI] 10.3-10.7%), CSS of 95.4% (95% CI 95.2-95.5%), and OS of 69.9% (95% CI 69.6-70.1%), vs 11.9% (95% CI 11.7-12.1%), 94.9% (95% CI 94.8-95.1%), and 69.6% (95% CI 69.3-69.9%), respectively, from surveillance intensity de-escalation. By increasing surveillance intensity, the number-needed-to-treat to prevent one additional MIBC progression over 10 years was ≥80, and ≥257 to avoid one additional cancer-related mortality. Compared to surveillance intensity de-escalation, higher-intensity regimens incurred an incremental cost of ≥$336 000 per incremental quality-adjusted life year gained, which well exceeded conventional willingness-to-pay thresholds, ≥$686 000 per additional MIBC progression prevented, and ≥$2.2 million per additional cancer-related mortality avoided.
CONCLUSIONS: In microsimulations testing a wide range of cystoscopic surveillance intensity for patients newly diagnosed with HRNMIBC, moderate surveillance de-escalation appears associated with an insignificant change in 10-year OS and furthermore is cost-effective vs higher-intensity surveillance regimens. These results suggest that moderate surveillance de-escalation can reduce costs of care without compromising life expectancy for many patients.