Transfusion-related cost offsets and time burden in patients with myelofibrosis on momelotinib vs. danazol from MOMENTUM.

Aim: To estimate projected US-based cost and time burden for patients with myelofibrosis and anemia treated with momelotinib compared with danazol.Methods: Cost and time burden were calculated based on the transfusion status of patients in the MOMENTUM trial and estimates extracted from previous studies.Results: Reductions in transfusion associated with momelotinib are projected to result in cost and time savings compared with danazol in transfusion-dependent and transfusion-independent/requiring patients with myelofibrosis, respectively: annual medical costs ($53,143 and $46,455 per person), outpatient transfusion costs ($42,021 and $8,370 per person) and annual time savings (173 and 35 h per person).Conclusion: Fewer transfusions with momelotinib are projected to result in cost and time savings in patients with myelofibrosis and anemia compared with danazol.

Estimated cost & time savings in patients with the blood cancer myelofibrosisMyelofibrosis is a rare blood cancer often associated with bone marrow damage, too few of some types of blood cells and symptoms including tiredness, night sweating, itching and feelings of fullness and pain because of increased spleen size. Patients with anemia (too few red blood cells) may require regular blood transfusions and this is one sign that myelofibrosis is getting worse. MOMENTUM was a Phase III clinical trial showing that the drug momelotinib was safe and effective in patients with myelofibrosis who were previously treated with a type of drug called a JAK inhibitor. In particular, the trial showed that momelotinib reduced the need for transfusions compared with danazol, another drug typically used to treat patients with anemia. Based on this transfusion information from MOMENTUM and other publicly available information about estimated medical costs and patients' time spent in receiving transfusions, the analysis described here shows that a reduction in the number of transfusions with momelotinib compared with danazol is estimated to lead to cost savings as well as reduced patient time spent in transfusion-related travel, preparing and waiting for transfusions and receiving and recovering from transfusions.

The Validity, Time Burden, and User Satisfaction of the FoodImage™ Smartphone App for Food Waste Measurement Versus Diaries: A Randomized Crossover Trial.

The FoodImage™ smartphone app transmits to researchers users' photographs of food selection and food waste, and includes user-tagged information about waste reasons and destination. Twenty-four participants were trained to record food waste using FoodImage, food waste diaries requiring visual estimation of waste quantities, and diaries requiring scale weights. Participants used each method during three staged food-waste scenarios (food preparation, eating, and clean-out) in a randomized crossover trial. Two participants had extreme values for the weighed diary method; therefore, accuracy results are reported with and without these two participants' data. Error was calculated as waste estimated with the experimental method minus directly weighed waste. Mean absolute error from FoodImage was significantly smaller than or equal to the error from both diary methods in each scenario. Furthermore, the mean values from FoodImage were equivalent to directly weighed values in two out of the three tasks; while weighed diaries were equivalent in two tasks only when the two participants with extreme values were removed. Visually estimated diaries were equivalent for only one task. All 24 participants preferred FoodImage to diaries and all rated FoodImage as less time consuming. Over one week, FoodImage would require ~24 fewer minutes of users' time to record all data. Unlike food waste diaries, FoodImage also transmits data to researchers in real-time and provides detailed data on food selection and intake. ClinicalTrials.gov Identifier: NCT03309306.

Patient-Reported Factors in Treatment Satisfaction in Patients with Relapsed/Refractory Multiple Myeloma (RRMM).

BACKGROUND: Therapy choices in relapsed/refractory multiple myeloma (RRMM) should consider patient satisfaction with treatment, because it is associated with adherence to therapy, health outcomes, and medical safety. The primary objective of this pilot cross-sectional observational study was to ascertain factors associated with patient-reported treatment satisfaction in RRMM. PATIENTS AND METHODS: Patients with a self-reported diagnosis of RRMM recruited from PatientsLikeMe, MyelomaCrowd, and Facebook were administered an electronic survey that included questions on demographics and clinical history, treatment experience, economic burden, and standardized patient-reported outcome measures, including the Treatment Satisfaction Questionnaire for Medication, Eastern Cooperative Oncology Group performance status (ECOG PS) measure, and Work Productivity and Activity Impairment Questionnaire: Specific Health Problem V2.0. Univariable and multivariable analyses were used to identify predictors of patient-perceived treatment satisfaction. RESULTS: One hundred sixty patients with RRMM participated in the study, with a median of two prior relapses and 66.3% reporting the most recent relapse within the last 12 months. ECOG PS ≥2 was associated with lower patient-reported global satisfaction and perceived effectiveness of current treatment. In addition to shorter time spent receiving therapy, orally administered treatment was the strongest predictor of higher satisfaction with treatment convenience. For patients receiving an injectable drug-containing regimen versus an all-oral regimen, respectively, time spent receiving multiple myeloma-directed therapy was higher (12.6 vs. 4.0 hours per month), and total monthly indirect costs were $1,033 and $241. CONCLUSION: Poor ECOG PS was linked to reduced treatment satisfaction and perceived effectiveness of current therapy, whereas an all-oral regimen was associated with increased treatment convenience satisfaction. IMPLICATIONS FOR PRACTICE: This study suggests that attributes including better Eastern Cooperative Oncology Group performance status, less time spent receiving treatment, and oral route of treatment administration lead to higher patient-perceived satisfaction with relapsed/refractory multiple myeloma (RRMM) treatment. Oral route of administration was also associated with less time spent receiving treatment and reduced economic burden for patients. Increased attention to these factors in shared treatment decision making is warranted to help identify individual patient needs, preferences, and expectations for RRMM treatments, to resolve dissatisfaction issues, and to improve the experience of patients with RRMM.

Measuring the burden of treatment for chronic disease: implications of a scoping review of the literature.

BACKGROUND: Although there has been growing research on the burden of treatment, the current state of evidence on measuring this concept is unknown. This scoping review aimed to provide an overview of the current state of knowledge as well as clear recommendations for future research, within the context of chronic disease. METHODS: Four health-based databases, Scopus, CINAHL, Medline, and PsychInfo, were comprehensively searched for peer-reviewed articles published between the periods of 2000-2016. Titles and abstracts were independently read by two authors. All discrepancies between the authors were resolved by a third author. Data was extracted using a standardized proforma and a comparison analysis was used in order to explore the key treatment burden measures and categorize them into three groups. RESULTS: Database searching identified 1458 potential papers. After removal of duplications, and irrelevant articles by title, 1102 abstracts remained. An additional 22 papers were added via snowball searching. In the end, 101 full papers were included in the review. A large number of the studies involved quantitative measures and conceptualizations of treatment burden (n = 64; 63.4%), and were conducted in North America (n = 49; 48.5%). There was significant variation in how the treatment burden experienced by those with chronic disease was operationalized and measured. CONCLUSION: Despite significant work, there is still much ground to cover to comprehensively measure treatment burden for chronic disease. Greater qualitative focus, more research with cultural and minority populations, a larger emphasis on longitudinal studies and the consideration of the potential effects of "identity" on treatment burden, should be considered.

Risk factors for family time burdens providing and arranging health care for children with special health care needs: Lessons from nonproportional odds models.

We identify need, enabling, and predisposing factors for high family time burdens associated with the health care of chronically-ill children, using data from the U.S. 2009-2010 National Survey of Children with Special Health Care Needs (NS-CSHCN), a population-based survey of 40,242 children with special health care needs (CSHCN). We estimate generalized ordered logistic multivariable regressions of time spent (1) providing health care for the child at home, (2) arranging/coordinating health care, and (3) combined time. Factors associated with higher time burdens included child's functional limitations, severe or unstable health conditions, public health insurance, lack of a medical home, low family income, low adult education, and non-white race. Nonproportional odds models revealed associations between risk factors and time burden that were obscured by binary and standard ordered logistic models. Clinicians and policymakers can use this information to design interventions to alleviate this important family stressor.

Quantitative Comparison of Liver Volume, Proton Density Fat Fraction, and Time Burden between Automatic Whole Liver Segmentation and Manual Sampling MRI Strategies for Diagnosing Metabolic Dysfunction-associated Steatotic Liver Disease in Obese Patients.

BACKGROUND: The performance of automatic liver segmentation and manual sampling MRI strategies needs be compared to determine interchangeability. OBJECTIVE: To compare automatic liver segmentation and manual sampling strategies (manual whole liver segmentation and standardized manual region of interest) for performance in quantifying liver volume and MRI-proton density fat fraction (MRI-PDFF), identifying steatosis grade, and time burden. METHODS: Fifty patients with obesity who underwent liver biopsy and MRI between December 2017 and November 2018 were included. Sampling strategies included automatic and manual whole liver segmentation and 4 and 9 large regions of interest. Intraclass correlation coefficient (ICC), Bland-Altman, linear regression, receiver operating characteristic curve, and Pearson correlation analyses were performed. RESULTS: Automatic whole liver segmentation liver volume and manual whole liver segmentation liver volume showed excellent agreement (ICC=0.97), high correlation (R2=0.96), and low bias (3.7%, 95% limits of agreement, -4.8%, 12.2%) in liver volume. There was the best agreement (ICC=0.99), highest correlation (R2=1.00), and minimum bias (0.84%, 95% limits of agreement, -0.20%, 1.89%) between automated whole liver segmentation MRI-PDFF and manual whole liver segmentation MRI-PDFF. There was no difference of each paired comparison of receiver operating characteristic curves for detecting steatosis (P=0.07-1.00). The minimum time burden for automatic whole liver segmentation was 0.32 s (0.32-0.33 s). CONCLUSION: Automatic measurement has similar effects to manual measurement in quantifying liver volume, MRI-PDFF, and detecting steatosis. Time burden of automatic whole liver segmentation is minimal among all sampling strategies. Manual measurement can be replaced by automatic measurement to improve quantitative efficiency.

Which components of medical homes reduce the time burden on families of children with special health care needs?

OBJECTIVES: To examine which components of medical homes affect time families spend arranging/coordinating health care for their children with special health care needs (CSHCNs) and providing health care at home. DATA SOURCES: 2009-2010 National Survey of Children with Special Health Care Needs (NS-CSHCN), a population-based survey of 40,242 CSHCNs. STUDY DESIGN: NS-CSHCN is a cross-sectional, observational study. We used generalized ordered logistic regression, testing for nonproportional odds in the associations between each of five medical home components and time burden, controlling for insurance, child health, and sociodemographics. DATA COLLECTION/EXTRACTION METHODS: Medical home components were collected using Child and Adolescent Health Measurement Initiative definitions. PRINCIPAL FINDINGS: Family-centered care, care coordination, and obtaining needed referrals were associated with 15-32 percent lower odds of time burdens arranging/coordinating and 16-19 percent lower odds providing health care. All five components together were associated with lower odds of time burdens, with greater reductions for higher burdens providing care. CONCLUSIONS: Three of the five medical home components were associated with lower family time burdens arranging/coordinating and providing health care for children with chronic conditions. If the 55 percent of CSHCNs lacking medical homes had one, the share of families with time burdens arranging care could be reduced by 13 percent.