Myelofibrosis and anemia: A German claims data analysis to describe epidemiology and current treatment.

OBJECTIVES: There is limited data on the incidence, prevalence, and treatments for myelofibrosis (MF) in Germany. This retrospective study examined claims data from 3.3 million insured individuals, spanning from 2010 to 2021. METHODS: Four sensitivity scenarios were explored to identify cases of MF. Point prevalence and cumulative incidence of MF were determined as of December 31, 2021, and within 2021, respectively. A cross-sectional analysis used the main scenario definition of MF to identify cases and evaluate the period prevalence of patients receiving treatment for symptoms and/or splenomegaly, including first-line (1L) Janus kinase inhibitor (JAKi), second-line, or further (2L+) MF-related treatment therapies during 2021. The prevalence of anemia treatment was also reported. RESULTS: The estimated standardized point prevalence of MF on December 31, 2021, was 9.9-12.4 cases per 100 000 persons, and cumulative incidence in 2021 was 1.2-1.8 cases per 100 000 persons. Standardized period prevalence in 2021 for MF patients receiving 1L JAKi and/or 2L+ MF-related treatment was 4.0 cases per 100 000. Among these patients, 47.1%-53.7% required treatment for anemia, resulting in a period prevalence of 1.9-2.2 cases per 100 000 individuals. CONCLUSION: The data reveal gaps in MF treatments and the need to improve patient quality of life.

Impact of disease progression on health-related quality of life of advanced ovarian cancer patients - Pooled analysis from the PRIMA trial.

OBJECTIVE: Progression-free survival (PFS) is an important early efficacy endpoint in ovarian cancer (OC) and its relevance to patients should be assessed. PRIMA, a phase III trial, assessed niraparib in patients with OC; this post hoc analysis examined the relationship between disease progression in OC and health-related quality of life (HRQoL). METHODS: The PRIMA trial randomized patients with advanced OC responsive to first-line platinum-based chemotherapy to once daily maintenance oral niraparib or placebo. This post hoc analysis evaluated the impact of disease progression on HRQoL by comparing HRQoL at the last visit pre-progression to end of treatment (EoT), and after 4, 8, 12, and 24 weeks. Assessments included the Functional Assessment of Cancer Therapy-Ovarian Symptom Index (FOSI), the European Quality of Life Five Dimension Five Level questionnaire (EQ-5D-5L) and EQ Visual Analogue Scale (EQ-VAS), the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (EORTC-QLQ-C30), and the EORTC Quality of Life Questionnaire Ovarian Cancer module (EORTC-QLQ-OV28). RESULTS: This post hoc analysis included 733 patients. Mean FOSI, EQ-5D-5L, and EQ-VAS scores deteriorated from last visit pre-progression to EoT and remained low up to 24-week follow-up. Least squares mean changes from last visit pre-progression to EoT were -2.1 (95% confidence interval -2.4, -1.7) for FOSI, -4.6 (-5.6, -3.5) for the EQ-5D-5L index, and -7.9 (-9.6, -6.3) for EQ-VAS. CONCLUSIONS: Disease progression negatively impacted HRQoL in patients with OC. PFS is clinically relevant, and prolonging PFS may preserve HRQoL.

Costs of Public Health Screening of Children for Presymptomatic Type 1 Diabetes in Bavaria, Germany.

OBJECTIVE: We sought to evaluate costs associated with public health screening for presymptomatic type 1 diabetes in 90,632 children as part of the Fr1da study in Bavaria and in forecasts for standard care. RESEARCH DESIGN AND METHODS: We report on resource use and direct costs for screening-related procedures in the Fr1da study coordination center and laboratory and in participating pediatric practices and local diabetes clinics. Data were obtained from Fr1da study documents, an online survey among pediatricians, and interviews and records of Fr1da staff members. Data were analyzed with tree models that mimic procedures during the screening process. Cost estimates are presented as they were observed in the Fr1da study and as they can be expected in standard care for various scenarios. RESULTS: The costs per child screened in the Fr1da study were €28.17 (95% CI 19.96; 39.63) and the costs per child diagnosed with presymptomatic type 1 diabetes were €9,117 (6,460; 12,827). Assuming a prevalence of presymptomatic type 1 diabetes of 0.31%, as in the Fr1da study, the estimated costs in standard care in Germany would be €21.73 (16.76; 28.19) per screened child and €7,035 (5,426; 9,124) per diagnosed child. Of the projected screening costs, €12.25 would be the costs in the medical practice, €9.34 for coordination and laboratory, and €0.14 for local diabetes clinics. CONCLUSIONS: This study provides information for the planning and implementation of screening tests for presymptomatic type 1 diabetes in the general public and for the analysis of the cost-effectiveness of targeted prevention strategies.

Different information needs in subgroups of people with diabetes mellitus: a latent class analysis.

BACKGROUND: Current evidence suggests that the information needs of people with diabetes mellitus differ across patient groups. With a view to being able to provide individualized information, this study aims to identify (i) the diabetes-related information needs of people with diabetes mellitus; (ii) different subgroups of people with specific information needs; and (iii) associated characteristics of the identified subgroups, such as sociodemographic characteristics, diabetes-related comorbidities, and well-being. METHODS: This cross-sectional study was based on data from 837 respondents with diabetes mellitus who participated in the population-based KORA (Cooperative Health Research in the Augsburg Region) Health Survey 2016 in Southern Germany (KORA GEFU 4 study) (45.6% female, mean age 71.1 years, 92.8% Type 2 diabetes). Diabetes-related information needs were assessed with a questionnaire asking about patients' information needs concerning 11 diabetes-related topics, e.g. 'long-term complications' and 'treatment/therapy'. Subgroups of people with different information needs and associated characteristics were identified using latent class analysis. RESULTS: We identified the following four classes of people with different information needs: 'high needs on all topics', 'low needs on all topics', 'moderate needs with a focus on complications and diabetes in everyday life', and 'advanced needs with a focus on social and legal aspects and diabetes research'. The classes differed significantly in age, years of education, type of diabetes, diabetes duration, diabetes-related comorbidities, smoking behaviour, diabetes education, current level of information, and time preference. CONCLUSIONS: Knowledge about different patient subgroups can be useful for tailored information campaigns or physician-patient interactions. Further research is needed to analyse health care needs in these groups, changes in information needs over the course of the disease, and prospective health outcomes.

Status quo bias and health behavior: findings from a cross-sectional study.

BACKGROUND: Status quo bias (SQB) has often been referred to as an important tool for improving public health. However, very few studies were able to link SQB with health behavior. METHODS: Analysis were based on data from the population-based KORA S4 study (1999-2001, n = 2309). We operationalized SQB through two questions. The first asked whether participants switched their health insurance for financial benefits since this was enabled in 1996. Those who did were assigned a 'very low SQB' (n = 213). Participants who did not switch were asked a second hypothetical question regarding switching costs. We assigned 'low SQB' to those who indicated low switching costs (n = 1035), 'high SQB' to those who indicated high switching costs (n = 588), and 'very high SQB' to those who indicated infinite switching costs (n = 473). We tested the association between SQB and physical activity, diet, smoking, alcohol consumption, the sum of health behaviors, and body mass index (BMI) using logistic, Poisson and ordinary least square regression models, respectively. Models were adjusted for age, sex, education, income, satisfaction with current health insurance and morbidity. RESULTS: SQB was associated with a higher rate of physical inactivity [OR = 1.22, 95% CI (1.11; 1.35)], a higher sum of unhealthy lifestyle factors [IRR = 1.05, 95% CI (1.01; 1.10)] and a higher BMI [ß = 0.30, 95% CI (0.08; 0.51)]. CONCLUSION: A high SQB was associated with unfavorable health behavior and higher BMI. Targeting SQB might be a promising strategy for promoting healthy behavior.

Costs and health-related quality of life in Alpha-1-Antitrypsin Deficient COPD patients.

BACKGROUND: Alpha-1-Antitrypsin Deficiency (AATD) is an economically unexplored genetic disease. METHODS: Direct and indirect costs (based on self-reported information on healthcare utilization) and health-related quality of life (HRQL, as assessed by SGRQ, CAT, and EQ-5D-3 L) were compared between 131 AATD patients (106 with, 25 without augmentation therapy (AT)) and 2,049 COPD patients without AATD participating in the COSYCONET COPD cohort. The medication costs of AT were excluded from all analyses to reveal differences associated with morbidity profiles. The association of AATD (with/without AT) with costs or HRQL was examined using generalized linear regression modelling (GLM) adjusting for age, sex, GOLD grade, BMI, smoking status, education and comorbidities. RESULTS: Adjusted mean direct annual costs were €6,099 in AATD patients without AT, €7,117 in AATD patients with AT (excluding costs for AT), and €7,460 in COPD patients without AATD. AATD with AT was significantly associated with higher outpatient (+273%) but lower inpatient (-35%) and medication costs (-10%, disregarding AT) compared with COPD patients without AATD. There were no significant differences between groups regarding indirect costs and HRQL. CONCLUSION: Apart from AT costs, AATD patients tended to have lower, though not significant, overall costs and similar HRQL compared to COPD patients without AATD. AT was not associated with lower costs or higher HRQL. TRIAL REGISTRATION: NCT01245933.