Systematic methodological review of health state values in glaucoma cost-utility analyses.

IMPORTANCE: Describing the characteristics and sources of health state utility values and reporting practice in the literature of cost-utility analyses facilitates an understanding of the level of the transparency, validity, and generalizability of cost-utility analyses. Improving the quality of reporting will support investigators in describing the incremental value of emerging glaucoma interventions. OBJECTIVE: To describe the state of practice among published glaucoma cost-utility analysis studies, focusing on valuation of health and the quality of reporting. EVIDENCE REVIEW: We searched several databases including Medline, CINHAL, Embase, Web of Science, Scopus, Biosis previews, the Health Economic Evaluations Database, and the NHS Economic Evaluation Database (NHS EED). We included full-text, English, published cost-utility analyses of glaucoma interventions with quality-adjusted life years (QALYs) as the primary outcome measure to calculate incremental cost-utility ratios. Excluded studies were non-English language, reviews, editorials, protocols, or other types of economic studies (cost-benefit, cost-minimization, cost-effectiveness). Study characteristics, operational definitions of glaucoma health states and health state utilities were extracted. The original source of the health utility was reviewed to determine the scale of measurement and the source of preference weighting. Items from the Systematic Review of Utilities for Cost-Effectiveness (SpRUCE checklist) were used to assess the reporting and quality of health utilities in glaucoma CUA. FINDINGS: 43 CUAs were included, with 11 unique sources of health utilities. A wide range of health utilities for the same Hodapp-Parrish-Anderson glaucoma health states were reported; ocular hypertension (0.84-0.95), mild (0.68-0.94), moderate (0.57-0.92), advanced (0.58-0.88), severe/blind (0.46-0.76), and bilateral blindness (0.26-0.5). Most studies reported the basis for using health utilities (34, 79%) and any assumptions or adjustments applied to the health utilities (22, 51%). Few studies reported a framework for assessing the relevance of health utilities to a decision context (8, 19%). Even fewer (3, 7%) applied a systematic search strategy to identify health utilities and used a structured assessment of quality for inclusion. Overall, reporting has not improved over time. CONCLUSIONS AND RELEVANCE: This review describes that few CUAs describe important rationale for using health state utility values. Including additional details on the search, appraisal, selection, and inclusion process of health utility values improves transparency, generalizability and supports the assessment of the validity of study conclusions. Future investigations should aim to use health utilities on the same scale of measurement across health states and consider the source and relevance to the decision context/purpose of conducting that cost-utility study.

Resurrecting Multiattribute Utility Function: Developing a Value Set for Health Utility for Glaucoma.

OBJECTIVES: This study aimed to develop a scoring function to calculate health utilities for health states described by the Health Utility for Glaucoma (HUG-5) based on the preferences of the general population in the United States. METHODS: Preferences for HUG-5 health states were elicited using the standard gamble and visual analog scale through an online survey. Quota-based sampling was used to recruit a representative sample of the US general population in terms of age, sex, and race. A multiple attribute disutility function (MADUF) approach was adopted to derive scoring for the HUG-5. Model fit was assessed using mean absolute error associated with 5 HUG-5 marker health states that describe mild/moderate and severe glaucoma. RESULTS: Of 634 respondents completing the tasks, 416 were included in the estimation of the MADUF; 260 respondents (63%) considered worst possible HUG-5 health state better than death. The preferred scoring function generates the utilities ranging from 0.05 (worst HUG-5 health state) to 1 (best HUG-5 health state). The correlation between mean elicited and estimated values for marker states was strong (R2 = 0.97) with mean absolute error = 0.11. CONCLUSIONS: The MADUF for HUG-5 is used to measure health utilities on the scale of perfect health and death, which can be used to estimate quality-adjusted life-years for economic evaluations of glaucoma interventions.

Generation, Selection, and Face Validation of Items for a New Generic Measure of Quality of Life: The EQ-HWB.

OBJECTIVES: This article aims to describe the generation and selection of items (stage 2) and face validation (stage 3) of a large international (multilingual) project to develop a new generic measure, the EQ-HWB (EQ Health and Wellbeing), for use in economic evaluation across health, social care, and public health to estimate quality-adjusted life-years. METHODS: Items from commonly used generic, carer, social care, and mental health quality of life measures were mapped onto domains or subdomains identified from a literature review. Potential terms and items were reviewed and refined to ensure coverage of the construct of the domains/subdomain (stage 2). Input on the potential item pool, response options, and recall period was sought from 3 key stakeholder groups. The pool of candidate items was tested in qualitative interviews with potential future users in an international face validation study (stage 3). RESULTS: Stage 2 resulted in the generation of 687 items. Predetermined selection criteria were applied by the research team resulting in 598 items being dropped, leaving 89 items that were reviewed by key stakeholder groups. Face validation (stage 3) tested 97 draft items and 4 response scales. A total of 47 items were retained and 14 were modified, whereas 3 were added to the candidate pool of items. This resulted in a 64-item set. CONCLUSIONS: This international multiculture, multilingual study with a common methodology identified many items that performed well across all countries. These were taken to the psychometric testing along with modified and new items for the EQ-HWB.

Biomechanical Evaluation Comparing Pulvertaft Weave and Side-to-Side Tenorrhaphy Using Porcine Tendons.

Background: Pulvertaft Weave (PTW) is an established method of tenorrhapy in tendon transfers. Previous studies have suggested that a Side-to-Side (STS) tenorrhapy is easier to perform has the same advantages and has greater load to failure, ultimate load and stiffness compared to PTW. However, there is insufficient data comparing behaviour of STS and PTW during cyclical loading. The aim of this study is to compare these two methods in terms of creep after cyclic landing. Methods: Fresh porcine flexor digitorum tendons were used. Ten tendon PTW and ten STS repairs were performed. Cross sectional diameter was measured. The tendon repairs were tested by applying tension up to 25N for 100 cycles, followed by tension up to 75N for 100 cycles, followed by loading to failure. Force-displacement data was used to determine the creep of the repaired tendon. Results: All tendons survived 100 cycles of loading at 25N. After 1 cycle of loading, the mean cyclic creep in the PTW group was almost 3 mm larger than in the STS group (p = 0.046). After 100 cycles, the mean cyclic creep in the PTW group was 4.4 mm larger (p = 0.008). The cyclic creep rate was significantly larger in the PTW group (p < 0.001). All STS but only four PTW repairs survived after cyclic loading at 75N (p = 0.01). After 1 cycle and 100 cycles, mean creep of the surviving PTW samples was almost 7 mm (p = 0.006) and almost 9 mm (p = 0.004) larger than the STS group. The mean load to failure was four times larger in the STS group than the PTW group (p = 0.004). Conclusions: STS repairs have a significantly smaller permanent elongation after cyclic loading at 25N and 75N, a significantly smaller cyclic creep rate, require a significantly larger load to fail. This implies that STS repairs are less likely to elongate after cyclic loading and can withstand greater loads. These properties can be valuable in allowing patients to commence mobilisation immediately after surgery.

Developing a Valuation Function for the Preference-Based Multiple Sclerosis Index: Comparison of Standard Gamble and Rating Scale.

OBJECTIVE: The standard gamble (SG) and rating scale (RS) are two approaches that can be employed to elicit health state preferences from patients in order to inform decision making. The objectives of this study were: (i) to contribute evidence towards the similarities and differences in the SG and the RS to reflect patient preferences, and (ii) to develop a multi-attribute utility function (MAUF) (i.e., scoring algorithm) for the PBMSI. STUDY DESIGN: Two samples were recruited for the study. The first sample provided cross-sectional data to generate the preference weights which were then used to develop (D) the MAUFD. The distribution of SG and RS were compared across levels of perceived difficulty. The second sample provided additional data to validate (V) the MAUF, termed MAUFV. RESULTS: The mean RS values ranged from 0.39 to 0.65, whereas the mean SG values were much higher ranging from 0.80 to 0.91. Correlations between the two methods were very low ranging from -0.29 to 0.15. Bland-Altman plots revealed the extent of differences in values produced by the two methods. CONCLUSION: In contemplating trade-offs in the selection of a preference-based elicitation approach for a MAUF that could guide clinical decision making, results suggest the RS is preferable in terms of feasibility and validity for MS patients. The PBMSI with patient preferences shows promise as a measure of health-related quality of life for MS.

Using existing data to identify candidate items for a health state classification system in multiple sclerosis.

PURPOSE: In multiple sclerosis (MS), the use of preference-based measures is limited to generic measures such as Health Utilities Index Mark 2 and 3, the EQ-5D and the SF-6D. However, the challenge of using such generic preference-based measures in people with MS is that they may not capture all domains of health relevant to the disease. Therefore, the main aim of this paper is to describe the development of a health state classification system for MS patients. The specific objectives are: (1) to identify items best reflecting the domains of quality of life important to people with MS and (2) to provide evidence for the discriminative capacity of the response options by cross-walking onto a visual analog scale of health rating. METHODS: The data come from an epidemiologically sampled population of people with MS diagnosed post-1994. The dataset consisted of 206 items relating to impairments, activity limitations, participation restrictions, health perception and quality of life. Important domains were identified from the responses to the Patient Generated Index, an individualized measure of quality of life. The extent to which the items formed a uni-dimensional, linear construct was estimated using Rasch analysis, and the best item was selected using the threshold map. RESULTS: The sample was young (mean age 43) and predominantly female (n = 140/189; 74%). The P-PBMSI classification system consisted of five items, with three response levels per item, producing a total of 243 possible health states. Regression coefficient values consistently decreased between response levels and the linear test for trend were statistically significant for all items. The linear test for trend indicated that for each item the response options provided the same discriminative ability within the magnitude of their capacity. A scoring algorithm was estimated using a simple additive formula. The classification system demonstrated convergent validity against other measures of similar constructs and known-groups validity between different clinical subgroups. CONCLUSION: This study produced a health state classifier system based on items impacted upon by MS, and demonstrated the potential to discriminate the health impact of the disease.

Pharmacoeconomics of hematopoietic stem cell mobilization: an overview of current evidence and gaps in the literature.

Adequate hematopoietic stem cell (HSC) mobilization and collection is required prior to proceeding with high dose chemotherapy and autologous hematopoietic stem cell transplant. Cytokines such as G-CSF, GM-CSF, and peg-filgrastim, alone or in combination with plerixafor, and after chemotherapy have been used to mobilize HSCs. Studies have shown that the efficiency of HSC mobilization and collection may vary when different methods of mobilization are used. No studies have shown that survival is significantly affected by the method of mobilization, but some studies have suggested that cost and resource utilization may be different between different mobilization techniques. After the FDA approval of plerixafor with G-CSF to mobilize HSCs many transplant centers became concerned about the cost of HSC mobilization. A panel of experts was convened ant this paper reviews the current literature on the pharmacoeconomics of HSC mobilization.

LupusQoL-US benchmarks for US patients with systemic lupus erythematosus.

OBJECTIVE: The LupusQoL-US instrument was recently validated in the US. We studied the benchmarks for a US patient cohort with systemic lupus erythematosus (SLE) and relevant demographic and disease correlates. METHODS: LupusQoL-US was administered to 185 patients with SLE. Demographic data (age, sex, ethnicity, marital status) and disease features (duration, disease activity and damage) were assessed simultaneously. Descriptive statistics were obtained. LupusQoL-US domain scores were calculated, and compared by sex, ethnicity, and marital status using nonparametric tests. Correlation between LupusQoL-US domains and age, disease duration, disease activity, and disease damage were obtained. RESULTS: Mean age of patients was 42.2 +/- 14.5 years; 94% of subjects were women. African American patients comprised 60% of the study cohort. The most affected domains were Fatigue and Physical Health. The least affected was Intimate Relationships. Age correlated with Physical Health, Pain, and Body Image (r = 0.15-0.18). Differences were observed based on sex and marital status, but not by ethnicity; there the LupusQoL-US correlated inversely with disease activity (r = -0.001 to -0.36) and damage (r = -0.003 to -0.40). CONCLUSION: All domains of the LupusQoL-US based health related quality of life (HRQOL) were affected adversely. HRQOL varied by age, sex, and marital status in our SLE cohort.

Treatment of scaphoid nonunion with combined vascularized distal radius graft and iliac crest bone graft.

The optimal treatment of scaphoid nonunion is a controversial and challenging task in hand surgery. Although advances in internal fixation techniques have resulted in improved union rates, fractures with impaired vascularity have less than satisfactory results with conventional grafting techniques. We describe the technique of using a tricortical iliac crest graft and a vascularized distal radius graft in cases of nonunion with avascular necrosis. The principle of our technique is to improve union rates over previously described procedures by addressing the issues of stability using a tricortical graft with compression through internal fixation and by improving biology using a cancellous vascularized graft.

Literacy, race, and PSA level among low-income men newly diagnosed with prostate cancer.

OBJECTIVES: Among men with newly diagnosed prostate cancer, prostate-specific antigen (PSA) levels are higher and the cancer stage more advanced for African Americans than for whites. An earlier study found that after adjustment for literacy, race was no longer a significant predictor of advanced stage at presentation. We investigated whether, after adjusting for literacy, race was a significant independent predictor of greater PSA levels among men with newly diagnosed prostate cancer. METHODS: Consecutive patients with newly diagnosed prostate cancer from four outpatient care facilities in Chicago were interviewed and given a literacy assessment (n = 308). The PSA level at diagnosis was obtained from the medical charts. Logistic regression models were used to identify predictors of high PSA levels (greater than 20 ng/mL) at presentation. RESULTS: African-American men were three times more likely to have low literacy skills (sixth grade or less: 22.9% versus 7.1%; P <0.001) than were white men. In turn, men with low literacy skills were more than twice as likely to have a PSA level greater than 20 ng/mL at diagnosis (33.3% versus 13.5%; P = 0.009). On multivariate analyses, significant predictors of high PSA levels included low literacy (adjusted odds ratio 2.5, 95% confidence interval 1.5 to 4.2) and older age (age 65 to 74 years, adjusted odds ratio 2.6, 95% confidence interval 2.1 to 3.1 versus older than 74 years, adjusted odds ratio 3.4, 95% confidence interval 1.8 to 6.6), but not African-American race. CONCLUSIONS: In the current era in which PSA testing is common, low literacy may be an important and potentially overlooked factor associated with higher PSA levels at prostate cancer diagnosis among African-American and white men.

Granulocyte colony--stimulating factor for chemotherapy-induced neutropenia in patients with small cell lung cancer : the 40% rule revisited.

Recombinant granulocyte colony-stimulating factor (G-CSF) [filgrastim and lenograstim] and pegylated G-CSF (pegfilgrastim) have been shown to reduce the severity and duration of chemotherapy-associated febrile neutropenia (FN) when administered prophylactically to cancer patients receiving chemotherapeutic regimens. The American Society of Clinical Oncology (ASCO) evidence-based clinical guidelines published in 1994, 1996 and 1997 recommended primary prophylaxis with G-CSF for cancer patients. The 2000 ASCO update, with the same recommendation, highlights the importance of economic considerations in decision making for CSFs. This paper reviews the available cost-effectiveness evidence on the use of G-CSF as primary prophylaxis against FN in patients with small cell lung cancer (SCLC).Cost-effectiveness ratios from a healthcare payer perspective supported the use of filgrastim as primary prophylaxis for people with SCLC, on the basis of both clinical and economic benefits, treated with chemotherapeutic regimens that have an FN rate in the range of 40-60%. However, when indirect and patient out-of-pocket costs attributable to severe FN are included, available evidence suggests that the risk threshold may be reduced by more than half. Given that FN rates associated with chemotherapeutic regimens for SCLC are generally <40%, then few circumstances would warrant the use of G-CSFs (filgrastim and lenograstim) under the current rule. However, inclusion of indirect costs would lower the cost-effectiveness threshold. Future cost-effectiveness studies of medications such as pegfilgrastim should attempt to capture the societal perspective by incorporating productivity-related costs and using base-case rates of FN reported in the literature.