Treatment sequences and drug costs from diagnosis to death in multiple myeloma.

Novel therapies for multiple myeloma (MM) have improved patient survival, but their high costs strain healthcare budgets. End-of-life phases of treatment are generally the most expensive, however, these high costs may be less justifiable in the context of a less pronounced clinical benefit. To manage drug expenses effectively, detailed information on end-of-life drug administration and costs are crucial. In this retrospective study, we analysed treatment sequences and drug costs from 96 MM patients in the Netherlands who died between January 2017 and July 2019. Patients received up to 16 lines of therapy (median overall survival: 56.5 months), with average lifetime costs of €209 871 (€3111/month; range: €3942-€776 185) for anti-MM drugs. About 85% of patients received anti-MM treatment in the last 3 months before death, incurring costs of €20 761 (range: €70-€50 122; 10% of total). Half of the patients received anti-MM treatment in the last 14 days, mainly fully oral regimens (66%). End-of-life treatment costs are substantial despite limited survival benefits. The use of expensive treatment options is expected to increase costs further. These data serve as a reference point for future cost studies, and further research is needed to identify factors predicting the efficacy and clinical benefit of continuing end-of-life therapy.

From qualitative data to a measurement instrument: A clarification and elaboration of choices made in the development of the Patient Scale of the Patient and Observer Scar Assessment Scale (POSAS) 3.0.

PURPOSE: To clarify and elaborate on the choices that were made in the development of the Patient Scale of the Patient and Observer Scar Assessment Scale 3.0 (POSAS 3.0), based upon the rich information obtained from patients during focus groups and pilot tests. METHODS: The discussions described in this paper are a reflection of the focus group study and pilot tests that were conducted in order to develop the Patient Scale of the POSAS3.0. The focus groups took place in the Netherlands and Australia and included 45 participants. Pilot tests were performed with 15 participants in Australia, the Netherlands, and the United Kingdom. RESULTS: We discussed the selection, wording and merging of 17 included items. Additionally, the reason for exclusion of 23 characteristics are given. CONCLUSION: Based upon the unique and rich material of patient input obtained, two versions of the Patient Scale of the POSAS3.0 were developed: the Generic version, and the Linear scar version. The discussions and decisions taken during the development are informative for a good understanding of the POSAS 3.0 and are indispensable as a background for future translations and cross-cultural adaptations.

Development of the Patient Scale of the Patient and Observer Scar Assessment Scale (POSAS) 3.0: a qualitative study.

PURPOSE: The Patient and Observer Scar Assessment Scale (POSAS) is widely used for measurements of scar quality. This encompasses visual, tactile and sensory characteristics of the scar. The Patient Scale of previous POSAS versions was lacking input from patients. Therefore, the aim of this study was to develop the POSAS3.0, Patient Scale with involvement of adults patients with all scar types, complying with the highest clinimetric standards. METHODS: From February 2018 to April 2019, a series of six focus group interviews were performed in the Netherlands and Australia to identify scar quality characteristics that adults with scars consider to be important. All focus groups were transcribed, anonymized and analysed using a thematic analysis. Relevant characteristics were formulated into items, resulting in a Dutch and English version of the Patient Scale. These drafts were pilot tested in Australia, the Netherlands and the United Kingdom, and refined accordingly. RESULTS: A total of 21 relevant scar quality characteristics were identified during the focus groups. Two distinct versions of the POSAS3.0, Patient Scale were developed. The Generic version contains 16 items and can be used for all scar types, except linear scars. The Linear Scar version of the Patient Scale contains the same 16 items, with an extra item referring to the widening of scar margins. All included items are rated on a verbal rating scale with five response options. CONCLUSION: Two versions of the POSAS3.0 Patient Scale were developed. Further field tests are being performed to establish the measurement properties and scoring algorithm of the scales.

Cost-utility of an eHealth application 'Oncokompas' that supports cancer survivors in self-management: results of a randomised controlled trial.

PURPOSE: The eHealth self-management application 'Oncokompas' was developed to support cancer survivors in monitoring health-related quality of life (HRQOL) and symptoms, and obtaining personalized feedback and options for supportive care. The aim of this study was to assess the cost-utility of Oncokompas compared with care as usual (CAU) among cancer survivors. METHODS: Survivors were randomly allocated to the intervention or control group. Direct (non-)medical, indirect non-medical costs, and HRQOL were measured at 3- and 6-month follow-up, using iMTA Medical Consumption and Productivity Costs and the EuroQol-5D questionnaires. Mean cumulative costs and quality-adjusted life-years (QALYs) were compared between both groups. RESULTS: In total, 625 survivors were randomized into intervention (n = 320) or control group (n = 305). Base case analysis showed that incremental costs from a societal perspective were - €163 (95% CI, - 665 to 326), and incremental QALYs were 0.0017 (95% CI, - 0.0121 to 0.0155) in the intervention group compared with those in the control group. The probability that, compared with CAU, Oncokompas is more effective was 60%, less costly 73%, and both more effective and less costly 47%. Sensitivity analyses showed that incremental costs vary between - €40 and €69, and incremental QALYs vary between - 0.0023 and - 0.0057. CONCLUSION: Oncokompas is likely to be equally effective on utilities, and not more expensive than CAU, and will therefore contribute to sustainable cancer survivorship care in a (cost-)effective manner. IMPLICATIONS FOR CANCER SURVIVORS: Oncokompas seems to improve HRQOL and reduces the burden of several tumour-specific symptoms, while costs from a societal perspective are similar to CAU.

Tools for a systematic appraisal of integrated community-based approaches to prevent childhood obesity.

BACKGROUND: Evaluation and monitoring methods are often unable to identify crucial elements of success or failure of integrated community-wide approaches aiming to tackle childhood overweight and obesity, yet difficult to determine in complex programmes. Therefore, we aimed to systematically appraise strengths and weaknesses of such programmes and to assess the usefulness of the appraisal tools used. METHODS: To identify strengths and weaknesses of the integrated community-based approaches two tools were used: the Good Practice Appraisal tool for obesity prevention programmes, projects, initiatives and intervention (GPAT), a self-administered questionnaire developed by the WHO; and the OPEN tool, a structured list of questions based on the EPODE theory, to assist face-to-face interviews with the principle programme coordinators. The strengths and weaknesses of these tools were assessed with regard to practicalities, quality of acquired data and the appraisal process, criteria and scoring. RESULTS: Several strengths and weaknesses were identified in all the assessed integrated community-based approaches, different for each of them. The GPAT provided information mostly on intervention elements whereas through the OPEN tool information on both the programme and intervention levels were acquired. CONCLUSION: Large variability between integrated community-wide approaches preventing childhood obesity in the European region was identified and therefore each of them has different needs. Both tools used in combination seem to facilitate comprehensive assessment of integrated community-wide approaches in a systematic manner, which is rarely conducted. Nonetheless, the tools should be improved in line to their limitations as recommended in this manuscript.

A multigene assessment of phylogenetic relationships within the dasyurid marsupial subfamily Sminthopsinae.

We report sequences of the mitochondrial cytochrome b (1146 bp) and 12S rRNA (961 bp) genes, as well as the nuclear protamine P1 (608 bp) gene, from 13 species representing all four genera of the dasyurid marsupial subfamily Sminthopsinae. Mitochondrial sequences are partitioned into five categories (three codon positions in cytochrome b, and stems and loops in 12S rRNA) with distinct substitution rates, transition biases, and base compositions. We extract estimates of these biases from the sequences and employ them to calculate two overall distances based on the DNAML model. Phylogenetic analyses using distance and parsimony methods yield trees with different topologies for mtDNA and protamine. These trees are compatible with respect to highly resolved nodes, but incompatible with respect to length differences in a parsimony framework. The tree from combined-data analysis is dominated by the larger data set (mtDNA). The balance of evidence favors a basal separation of Planigale from other sminthopsines. Within Planigale, Pl. maculata is sister to the remaining species. Although the precise intergeneric affinities of Antechinomys are unresolved, A. laniger does not appear to be part of the genus Sminthopsis as suggested by morphological data. The 12S rRNA resolves Ningaui ridei and N. yvonnae as sister species.

Assessment of painful episode frequency in sickle-cell disease.

Frequency of painful episodes in sickle-cell disease is considered to be related to clinical severity and possibly to other aspects of the disease. Measurements of frequency often include only hospital-related or more severe, longer-lasting episodes. Since painful episodes, however, may regularly occur in nonhospital settings or be shorter-lasting with possible different pathologic effects, we measured all painful episodes in 10 adults with sickle-cell disease for 1.0-3.8 years, using a daily questionnaire. The results were related to other indices of disease severity and to possible precipitating factors, such as cold weather and menses. Sixty-one percent (on average) of the total number of episodes (243) were nonhospital-related, and 33% (on average) were shorter-lasting. Episode frequencies, whether determined as total, hospital-related, nonhospital-related, or shorter-lasting, were not related to each other or to other indicators of disease severity. The highest incidence of episode frequency occurred in the winter. The association of episodes with menses was moderately close in individual patients. The findings suggest that nonhospital-related painful episodes and shorter-lasting episodes may contribute significantly to episode frequency. Measurement of frequency of all painful episodes would require consideration when evaluating episode frequency and its relationship to disease severity, to possible precipitating factors of episodes, and to treatment of the disease, and for study of the natural course of the disease.

Assessment of the anemia of chronic disease by digital image processing of erythrocytes.

Eighteen patients with the anemia of chronic disease were studied to determine the usefulness and accuracy of image processing analysis of erythrocytes for diagnosis. Diagnostic tests used for comparison included estimates of serum ferritin, serum iron and iron-binding capacity, stainable marrow iron, and erythrocyte morphology as determined by standard methods. The findings show that the analyses obtained by image processing were diagnostic for 83% of the patients with chronic disease. Serum ferritin levels were supportive of diagnosis for 33% of the patients, and serum iron levels were useful for approximately 25% of the patients. Cell indices and marrow iron were of limited value. The study demonstrates that quantitative information obtained by digital image processing of erythrocytes can be very useful for the diagnosis of the anemia of chronic disease.