Objectives To develop and provide initial validation for a multivariate, claims-based prediction model for disability status (DS), a proxy measure of performance status (PS), among older adults. The model was designed to augment information on health status at the point of cancer diagnosis in studies using insurance claims to examine cancer treatment and outcomes. Materials and Methods We used data from the 2001–2005 Medicare Current Beneficiary Survey (MCBS), with observations randomly split into estimation and validation subsamples. We developed an algorithm linking self-reported functional status measures to a DS scale, a proxy for the Eastern Cooperative Oncology Group (ECOG) PS scale. The DS measure was dichotomized to focus on good [ECOG 0–2] versus poor [ECOG 3–4] PS. We identified potential claims-based predictors, and estimated multivariate logistic regression models, with poor DS as the dependent measure, using a stepwise approach to select the optimal model. Construct validity was tested by determining whether the predicted DS measure generated by the model was a significant predictor of survival within a validation sample from the MCBS. Results and Conclusion One-tenth of beneficiaries met the definition for poor DS. The base model yielded high sensitivity (0.79) and specificity (0.92); positive predictive value=48.3% and negative predictive value=97.8%, c-statistic=0.92 and good model calibration. Adjusted poor claims-based DS was associated with an increased hazard of death (HR=3.53, 95% CI 3.18, 3.92). The ability to assess DS should improve covariate control and reduce indication bias in observational studies of cancer treatment and outcomes based on insurance claims.

Background In prior research, we developed a claims-based prediction model for poor patient disability status (DS), a proxy measure for performance status, commonly used by oncologists to summarize patient functional status and assess ability of a patient to tolerate aggressive treatment. In this study, we implemented and validated the DS measure in 4 cohorts of cancer patients: early and advanced non-small cell lung cancers (NSCLC), stage IV estrogen-receptor (ER-) negative breast cancer, and myelodysplastic syndromes (MDS). Data and methods 1999–2007 SEER-Medicare data for the four cohorts of cancer patients. Bivariate and multivariate logistic regression tested the association of the DS measure with designated cancer-directed treatments: early NSCLC (surgery), advanced NSCLC (chemotherapy), stage IV ER- breast cancer (chemotherapy), and MDS (erythropoiesis-stimulating agents). Treatment model fit was compared across model iterations. Results In both unadjusted and adjusted results, predicted poor DS was strongly associated with a lower likelihood of cancer treatment receipt in all four cohorts [early NSCLC (N=20,280), advanced NSCLC (N=31,341), stage IV ER- breast cancer (N=1,519), and MDS (N=6,058)] independent of other patient, contextual, and disease characteristics, as well as the Charlson Comorbidity Index (CCI). Inclusion of the DS measure into models already controlling for other variables did not significantly improve model fit across the cohorts. Conclusions The DS measure is a significant independent predictor of cancer-directed treatment. Small changes in model fit associated with both DS and the CCI suggest that unobserved factors continue to play a role in determining cancer treatments.

Aims: Iron overload adversely affects patients with myelodysplastic syndromes (MDS), but benefits of iron chelation therapy have not been clearly demonstrated. We examined the association between deferasirox (DFX) therapy and mortality in transfusion-receiving Medicare patients. Patients & methods: MDS patients from 2005 to 2008 were identified using ICD-9 codes from 100% Medicare claims. Patients receiving ≥20 blood units were observed until death or end of study. Marginal structural models were used for estimation. Results: 3926 patients (10.1% used DFX) were observed for a mean of 48.8 weeks. Each incremental week of DFX was associated with a significant reduction in mortality risk (hazard ratio [HR]: 0.989; 95% CI: 0.983-0.996; p = 0.001). Conclusion: DFX therapy is associated with a reduced mortality risk among older MDS patients who received a minimum transfusion threshold.

Erythropoiesis-stimulating agents (ESA) are used commonly to reduce symptomatic anemia in patients with myelodysplastic syndromes (MDS).We assessed population-based patterns of ESA use relative to treatment guidelines using data from the Surveillance, Epidemiology, and End Results (SEER) registries, with linked Medicare claims providing detailed treatment data from 2001 through 2005. The study found widespread use (62%) of ESA in Medicare beneficiaries with MDS. Similar ESA use rates regardless of risk status, low frequency (45%) of serum erythropoietin determination prior to ESA initiation, and high prevalence (60.4%) of short-duration ESA episodes suggest clinically important discrepancies between actual practice and guideline-recommended therapy.

Patient and physician characteristics associated with use of erythropoiesis-stimulating agents in myelodysplastic syndrome patients have not yet been described. Myelodysplastic syndrome patients diagnosed from 2001 to 2005 were identified from the Surveillance Epidemiology and End ResultsMedicare database. Multivariate regressions examined the association between patient and physician characteristics and the probability of receiving any erythropoiesis-stimulating agents, and of receiving therapeutic-length (≥8 week) treatment episodes. Among the 6,588 myelodysplastic syndrome patients studied, 65% received erythropoiesis-stimulating agents. Use of erythropoiesis-stimulating agents was lower for blacks compared to whites (OR 0.78; 95% CI:0.61-0.99), single persons compared to married (OR 0.77; 95% CI:0.62-0.97), Medicaid recipients (OR 0.66; 95% CI:0.55-0.79), and those living in census tracts with lower educational attainment.Patients who did not consult a hematology-oncology specialist were less likely to receive erythropoiesis-stimulating agents. Specialist access, financial resources and mobility are key determinants of receipt of erythropoiesis-stimulating agents among myelodysplastic syndrome patients.Key words: erythropoiesis-stimulating agent, myelodysplastic syndromes, patient, physician, characteristics. Citation ESA usePatients were classified as ESA users if they had at least one claim for an ESA during follow up. Among ESA users, we also determined if the patient received a therapeutic-length treatment episode (TTE), defined as eight weeks or over. This is the minimum duration suggested by the NCCN treatment guideline 9 and it has been used as an assessment cut-off point in several trials. 10,11 Episodes were counted separately for epoetin alfa and darbepoetin alfa. The first treatment episode began at the first week there was a claim for an ESA and continued weekly until there was a gap in treatment of three weeks for epoetin alfa or six weeks for darbepoetin alfa. Additional treatment episodes began at the week of the first prescription claim following a gap in treatment. One week was added to the episode length of each darbepoetin episode to account for the extended half-life of darbepoetin. Each treatment episode was then classified by length as TTE or not TTE. Measurement of key study variablesSociodemographic characteristics included patient age, race, sex, marital status, pre-diagnosis enrollment in Medicaid or a Medicare Savings Program (MSP; where state Medicaid programs pay for Medicare Part B premiums), census track-level median household income and proportion of adults with less than a high school education (quartile ranges), whether less than 5% of households reported difficulty speaking English, size of metropolitan area, region and year of diagnosis.Patient health status indicators included MDS diagnostic category, history of a different primary cancer within five years prior to MDS diagnosis, transfusions prior to MDS diagnosis, acute or chronic medical conditions, indicators of poo...

Introduction:High quality research regarding treatment effectiveness, quality, and value is critical for improving the U.S. health care system. Recognition of this has led federal and state officials to better leverage existing data sources such as medical claims and survey data, but access must be balanced with privacy concerns.Methods:We reviewed and catalogued data access policies for a selection of publicly-funded federal and state datasets to investigate how such policies may be promoting or limiting research activities.Results:We found significant variation in data access policies across federal agencies and across state agencies, including variation for multiple datasets available from the same agency. We also observed numerous indirect hurdles to use of data, including complex data use application procedures, high user fees, and prolonged wait times for data delivery.Conclusions:Policy makers and data owners should consider making changes to data access policies to maximize the utility and availability of these valuable resources.

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These results suggest a mixed pattern of change in the face of the FDA safety warnings and CMS NCD in MDS and highlight the importance of monitoring for unintended consequences of policy changes.