Treatment Patterns and Outcomes in Patients with KRAS Wild-Type Metastatic Colorectal Cancer Treated in First Line with Bevacizumab- or Cetuximab-Containing Regimens.

PURPOSE: Patients with Kirsten rat sarcoma viral oncogene wild-type (KRAS WT) metastatic colorectal cancer (mCRC) treated in first line with bevacizumab (B) or cetuximab (C) plus standard chemo backbones had comparable outcomes in phase III Cancer and Leukemia Group B (CALGB) 80405. We examined comparative effectiveness of B and C regimens in real-world community settings. METHODS: This retrospective study examined progression-free survival (PFS) and OS in a US community sample of KRAS WT mCRC patients treated with first-line B (n = 254) or C (n = 146) regimens. Medical records from the Vector Oncology Data Warehouse were used. Disease progression was determined from patient charts. OS was measured from the start of first-line treatment until death. RESULTS: There were no significant difference in either PFS or OS respectively between B-treated compared to C-treated patients (HR = 1.324, 95% CI 0.901, 1.947; HR = 1.080, 95% CI 0.721, 1.617). More B patients received oxaliplatin backbones (74.8 vs. 36.3%), and more C patients received irinotecan backbones (51.4 vs. 20.1%), ps < 0.001. Multivariate survival analyses showed a significant difference indicating a greater risk for death among C-treated patients with right-sided tumors vs. left-sided tumors (HR = 2.263, 95% CI 1.394, 3.673, p = 0.0009), but not for B-treated patients (HR = 1.209, 95% CI 0.825, 1.771, p = 0.3297). CONCLUSIONS: Consistent with CALGB 80405, median PFS and OS for these community oncology KRAS WT mCRC patients treated with first-line B or C regimens did not differ significantly.

Progression-Free Survival in Patients Receiving Chemotherapy Alone (C) or Chemotherapy with Bevacizumab (CB) for First-Line Treatment of KRAS Mutant Metastatic Colorectal Cancer in Community Oncology Settings.

PURPOSE: Bevacizumab is a standard first-line (L1) treatment for metastatic colorectal cancer (mCRC) patients regardless of RAS status. This retrospective study examined treatment patterns and outcomes in a community oncology sample of KRAS mutant mCRC patients treated with chemotherapy (C) or C plus bevacizumab (CB) in L1. METHODS: This study used medical records from the Vector Oncology Data Warehouse. Eligible patients were confirmed KRAS mutant mCRC and received L1 C or CB. Kaplan-Meier analysis assessed L1 progression-free survival (PFS) and overall survival (OS). Cox regression models examined the interaction of tumor location (R/L) with treatment. RESULTS: CB (n = 264) compared to C (n = 109) patients were younger, less likely performance status (PS) impaired, and more likely with liver metastases. Median unadjusted PFS was 10.41 months (95% CI 9.0-11.3) in CB and 7.66 months (95% CI 6.5-9.1) in C patients (p = 0.174). Median unadjusted OS was 26.91 months (95% CI 24.3-29.3) in CB and 23.33 months (95% CI 19.7-29.2) in C patients (p = 0.571). For patients with right- vs. left-sided tumors, C (but not CB)-treated patients had higher adjusted risk for progression (HR = 1.715, 95% CI 1.108, 2.653; p = 0.015). CONCLUSIONS: CB- vs. C-treated KRAS mutant mCRC patients may have a meaningful PFS benefit. Patients with right-sided tumors treated with C were at higher risk for disease progression than patients with left-sided tumors. Tumor location had no significant effect on outcomes in the CB cohort.

Corrigendum to "Prevalence of Fracture Risk Factors in Postmenopausal Women Enrolled in the POSSIBLE US Treatment Cohort".

[This corrects the article DOI: 10.1155/2013/715025.].

Real-world Direct Health Care Costs for Metastatic Colorectal Cancer Patients Treated With Cetuximab or Bevacizumab-containing Regimens in First-line or First-line Through Second-line Therapy.

BACKGROUND: The present study examined real-world direct health care costs for metastatic colorectal cancer (mCRC) patients initiating first-line (1L) bevacizumab (BEV)- or cetuximab (CET)-containing regimen in 1L or 1L-through-second-line (1L-2L) therapy. PATIENTS AND METHODS: Using a large US insurance claims database, patients with mCRC initiating 1L BEV- or 1L CET-containing regimen from January 1, 2008 to September 30, 2014 were identified. The per-patient per-month (PPPM) all-cause health care costs (2014 US dollars) were measured during 1L therapy and, for patients continuing to a 2L biologic-containing regimen, 1L-2L therapy. Multivariable regression analyses were used to compare PPPM total health care costs between patients initiating a 1L BEV- versus 1L CET-containing regimen. RESULTS: A total of 6095 patients initiating a 1L BEV- and 453 initiating a 1L CET-containing regimen were evaluated for 1L costs; 2218 patients initiating a 1L BEV- and 134 initiating a 1L CET-containing regimen were evaluated for 1L-2L costs. In 1L therapy, 1L CET had adjusted PPPM costs that were $3135 (95% confidence interval [CI], $1174-$5040; P < .001) greater on average than 1L BEV. In 1L-2L therapy, 1L BEV-2L CET had adjusted PPPM costs that were $1402 (95% CI, $1365-$1442; P = .010) greater than those for 1L BEV-2L BEV, and 1L CET-2L BEV had adjusted PPPM costs that were $4279 (95% CI, $4167-$4400; P = .001) greater on average than those for 1L BEV-2L BEV. The adjusted PPPM cost differences for 1L BEV-2L other biologic or 1L CET-2L other biologic agent were numerically greater but statistically insignificant. CONCLUSION: PPPM total health care costs for 1L and 2L therapy tended to be greater for patients treated with 1L CET-containing regimens than for 1L BEV-containing regimens. Also, continuing treatment with BEV-containing regimens 1L-2L was less costly than switching between BEV and CET. The cost differences between BEV and CET hold important implications for treatment decisions of mCRC patients in real-world clinical practice.

Big data analysis of treatment patterns and outcomes among elderly acute myeloid leukemia patients in the United States.

Over half of patients diagnosed with acute myeloid leukemia (AML) are 65 years or older. We examined patient characteristics, treatment patterns, and survival among elderly patients in routine clinical practice. We utilized a retrospective cohort analysis of first primary AML patients in the linked Surveillance, Epidemiology, and End Results (SEER)-Medicare database. Patients were diagnosed between January 1, 2000 and December 31, 2009, >66 years, and continuously enrolled in Medicare Part A and B in the year prior to diagnosis. Kaplan-Meier curves and Cox proportional hazards regression assessed overall survival by treatment. There were 3327 (40 %) patients who received chemotherapy within 3 months of diagnosis. Treated patients were more likely younger, male, and married, and less likely to have secondary AML and poor performance indicators and comorbidity score compared to untreated patients. In multivariate survival analysis, treated patients exhibited a significant 33 % lower risk of death compared to untreated patients. Significant survival benefits were noted with receipt of intensive and hypomethylating agent (HMA) therapies compared to no therapy. A survival benefit with allogeneic hematopoietic stem cell transplantation was seen in younger Medicare patients. This real-world study showed that about 60 % of elderly AML patients remain untreated following diagnosis. Use of anti-leukemic therapy was associated with a significant survival benefit in this elderly cohort.

Survival and Racial Differences of Non-Small Cell Lung Cancer in the United States Military.

BACKGROUND: Lung cancer is the leading cause of cancer-related death in the United States (US) Military and worldwide, with non-small cell lung cancer (NSCLC) accounting for 87 % of cases. OBJECTIVES: Using a US military cohort who receives equal and open access to healthcare, we sought to examine demographic, clinical features and outcomes with NSCLC. DESIGN AND PARTICIPANTS: We conducted a retrospective cohort analysis of 4,751 patients, aged ≥ 18 years and diagnosed with a first primary NSCLC between 1 January 2003 and 31 December 2013 in the US Department of Defense (DoD) cancer registry. MAIN MEASURES: Differences by patient and disease characteristics were compared using Chi-square and t-test. Kaplan Meier curves and Cox proportional hazards regression assessed overall survival. RESULTS: The mean age at diagnosis was 66 years, 64 % were male, 72 % were Caucasian, 41 % were diagnosed at early stage, 77 % received treatment and 82 % had a history of tobacco use. Mean age at diagnosis was highest among Caucasians (67 years) and lowest among African Americans (AA; 62 years). Asian/Pacific Islanders (PI) were more likely to be female (p < 0.0001), have adenocarcinoma histology (p = 0.0003) and less likely to have a history of tobacco use (p < 0.0001) compared to other racial/ethnic groups. In multivariable survival analysis, older age, male gender, increasing stage, not receiving treatment, and tobacco history were associated with higher mortality risk. Untreated patients exhibited a 39 % higher mortality risk compared to treated patients (HR = 1.39; 95%CI = 1.23-1.57). Compared to Caucasian patients, Asian/PIs demonstrated a 20 % lower risk of death (HR = 0.80; 95%CI = 0.66-0.96). There was no difference in mortality risk between AAs and Hispanics compared to Caucasians. CONCLUSION: The lack of significant outcome disparity between AAs and Caucasians and the earlier stage at diagnosis than usually seen in civilian populations suggest that equal access to healthcare may play a role in early detection and survival.

Treatment patterns and comparative effectiveness in elderly diffuse large B-cell lymphoma patients: a surveillance, epidemiology, and end results-medicare analysis.

BACKGROUND: The incidence of diffuse large B-cell lymphoma (DLBCL) occurs disproportionately in elderly patients. We evaluated real-world treatment patterns and outcomes in elderly DLBCL patients in the U.S. MATERIALS AND METHODS: A retrospective cohort analysis of 9,333 DLBCL patients from the linked Surveillance, Epidemiology, and End Results (SEER)-Medicare database was conducted. Patients were diagnosed between January 1, 2000, and December 31, 2007; were aged >66 years, and were continuously enrolled in Medicare Part A and B in the year prior to diagnosis. Within 3 months of diagnosis, 4,565 (49%) received rituximab plus chemotherapy (R+chemo), 2,181 (23%) received chemotherapy only, and 467 (5%) received rituximab monotherapy (R-mono). Cox proportional hazards regression assessed overall survival between R+chemo versus chemotherapy only and R-mono versus no treatment. RESULTS: Overall, 23% of patients received no treatment, and the proportion was higher among those aged >80 years (33%). Patients receiving R+chemo were younger and more likely white compared with those receiving chemotherapy only. Patients receiving R-mono were older and more likely female compared with those not treated. In multivariate analysis, patients receiving chemotherapy only had a twofold increased mortality risk versus R+chemo, and this was confirmed in a subanalysis of patients aged >80 years. A 91% higher mortality risk was noted with receipt of fewer than six cycles versus six cycles of chemotherapy or chemoimmunotherapy. Patients receiving R-mono had a 69% decreased mortality risk compared with patients who were not treated. CONCLUSION: This real-world analysis of elderly DLBCL patients confirmed that 23% do not receive treatment. Overall survival is higher for patients receiving R+chemo and R-mono relative to chemotherapy only and no treatment, respectively. Suboptimal durations of therapy with curative intent (fewer than six cycles) were associated with poorer outcomes.

Treatment patterns and outcomes among patients with metastatic melanoma treated in community practice.

BACKGROUND: The primary systemic treatments for advanced melanoma have been chemotherapy and immunotherapy. New agents are currently in development. OBJECTIVES: This study aimed to characterize treatment patterns and outcomes across several lines of therapy and to illustrate the treatment landscape prior to the approval of new therapies. The study endpoints were progression-free survival (PFS), overall survival (OS), and best overall response within line of therapy. METHODS: A retrospective chart analysis was conducted at 11 community oncology practices in the USA. Data for patients aged ≥18 years and diagnosed with stage IV and/or metastatic melanoma during 2006-2010 were analyzed. Primary endpoints were PFS within line of therapy and OS from the diagnosis of metastasis. RESULTS: Data on a total of 202 patients were collected. The sample was mostly male (60%) and Caucasian (88%), with a mean age of 61.3 years. Of the 202 patients, 56 (28%) never received any systemic therapy. In the remaining 146 patients, systemic therapies included temozolomide-based regimens (n = 68), platinum-based regimens without temozolomide (n = 16), other regimens (n = 23), and research regimens (n = 39). Of the 146 patients who received systemic therapy, not all did so immediately after the diagnosis of metastasis: 102 (51%) patients did so shortly after diagnosis and before first disease progression, and 44 (22%) did so after first disease progression. Response rates were very low (≤5%) and did not differ across treatment groups. Progressive disease was the most frequent best overall response category identified, with rates of 83, 78, and 89% in the first to third lines of treatment, respectively. In 146 patients receiving first-line systemic therapy, median PFS was 3.25 months. Median OS in the entire sample was 7.66 months. CONCLUSIONS: Findings provided little evidence for any beneficial effects of the treatments available in the timeframe referred to in this study. Few patients (≤5%) responded to treatment, PFS among treated patients was short (3.25 months in first-line treatments, less in later lines), and there was no evidence of a differential effect of treatment regimens on PFS. There was no evidence of shorter survival in patients who never received systemic therapy. The high proportion of patients who did not receive any systemic therapy highlights the lack of effective therapies and underscores the unmet medical need in this patient population.

Treatment practice in the elderly patient with chronic lymphocytic leukemia-analysis of the combined SEER and Medicare database.

The median age at diagnosis of chronic lymphocytic leukemia (CLL) is 72, but patients enrolled in randomized trials are often a decade younger. Therapy selection and outcomes in the older, comorbid population are less understood. We evaluated treatment patterns and outcomes among 2,985 first primary CLL patients from the linked Surveillance, Epidemiology, and End Results-Medicare database. There were 151 chlorambucil (CLB), 594 rituximab monotherapy (R-mono), 696 rituximab + intravenous chemotherapy (R + IV Chemo), and 1,544 IV chemo-only patients. Patients administered CLB and R-mono were the oldest and had the highest comorbidity burden while patients receiving R + IV Chemo were the youngest and had the lowest comorbidity burden (p < 0.0001). In the multivariate survival analysis, receipt of R + IV Chemo was associated with significantly lower mortality risk vs. IV Chemo-only (hazard ratio (HR) = 0.73; 95 % confidence interval (CI) 0.62-0.87) and a non-significant mortality risk reduction with R-mono vs. CLB (HR = 0.47; 95 % CI: 0.21-1.05). Older age and increasing comorbidity score were significantly associated with higher mortality. These findings suggest that chemoimmunotherapy is more effective than chemotherapy in an elderly population with a high prevalence of comorbidity, and this extends the conclusions from clinical trials in younger, medically fit patients.

Influence of patient perceptions and preferences for osteoporosis medication on adherence behavior in the Denosumab Adherence Preference Satisfaction study.

OBJECTIVE: This study aims to evaluate patient perceptions of subcutaneous denosumab or oral alendronate in postmenopausal women with or at risk for osteoporosis and how these perceptions influence adherence. METHODS: Postmenopausal women with low bone mass were randomized to denosumab 60 mg every 6 months for 1 year (treatment period 1 [TP1]) followed by alendronate 70 mg once weekly for 1 year (treatment period 2 [TP2]), or vice versa. Beliefs about Medicines Questionnaire data were collected at baseline and at 6, 12, 18, and 24 months; a necessity-concerns differential (NCD) was calculated for each time point. Logistic regression analyses were performed to evaluate the influences of baseline characteristics on nonadherence. RESULTS: Participants included 250 women (alendronate/denosumab, n = 124; denosumab/alendronate, n = 126). During TP1, the NCD at month 6 was higher with denosumab than with alendronate (P = 0.0076). In TP2, the NCD was higher for women switched to denosumab than for women switched to alendronate at 6 months (P = 0.0126) and 12 months (P = 0.4605). Denosumab was preferred to alendronate regardless of treatment sequence (P < 0.0001). Covariate analysis revealed that higher TP2 baseline necessity scores were associated with lower odds of nonadherence (P = 0.0055), whereas higher concerns about medication scores were associated with higher odds of nonadherence (P = 0.0247). Higher NCD scores were also associated with lower odds of nonadherence (P = 0.0015). CONCLUSIONS: Participants preferred denosumab to alendronate while on treatment and had more positive perceptions of denosumab than alendronate. These perceptions were associated with better adherence.

Referral and treatment patterns among patients with stages III and IV non-small-cell lung cancer.

PURPOSE: Little is known about how referrals to different cancer specialists influence cancer care for non-small-cell lung cancer (NSCLC). Among Medicare enrollees, we identified factors of patients and their primary care physician that were associated with referrals to cancer specialists, and how the types of cancer specialists seen correlated with delivery of guideline-based therapies (GBTs). METHODS: Data from patients with stages III and IV NSCLC included in the SEER-Medicare database were linked to their physicians in the American Medical Association Masterfile database. Using logistic regression, we (1) identified patient and physician factors that were associated with referrals to cancer specialists (medical oncologists, radiation oncologists, and surgeons); (2) identified the types of referral to cancer specialists that predicted greater likelihood of receiving GBT (per National Comprehensive Cancer Network guidelines). RESULTS: A total of 28,977 patients with NSCLC diagnosed from January 1, 2000 to December 31, 2005 met eligibility criteria. Younger age, white race, higher income, and primary physician specialty other than family practice predicted higher likelihood of referrals to medical oncologists (P < .01 for all predictors). Seeing the three types of cancer specialists predicted higher likelihood of GBT (stage IIIA: odds ratio [OR] = 20.6; P < .001; IIIB: OR = 77.2; P < .001; and IV: OR = 1.2; P = .011), compared with seeing a medical oncologist only. Use of GBTs increased over the study period (42% to 48% from 2000 to 2005; P < .001). CONCLUSION: Referrals to all types of cancer specialists increased the likelihood of treatment with standard therapies, particularly in stage III patients. However, racial and income disparities still prevent optimal referrals to cancer specialists.

Prevalence of Fracture Risk Factors in Postmenopausal Women Enrolled in the POSSIBLE US Treatment Cohort.

Subject- and physician-reported data from 4,429 postmenopausal women receiving osteoporosis treatment in the Prospective Observational Scientific Study Investigating Bone Loss Experience (POSSIBLE US) were used to assess the prevalence of risk factors (RFs) and on-study fracture. RFs assessed at study entry were age >70 years; fracture since age 50; minimum T-score (hip/spine) ≤-2.5 at diagnosis; body mass index <18.5 kg/m(2); rheumatoid arthritis; parental history of hip fracture; current smoking; and recent oral glucocorticoid use. Data were collected with semiannual self-administered questionnaires. Results were stratified by physician-reported osteoporosis/osteopenia diagnosis. Low T-score and age >70 years were the most common RFs in the osteoporosis group, and age >70 years and prior fracture were the most common risk factors in the osteopenia group. Multiple RFs were more common than a single RF in osteoporotic women (54.2% versus 34.6%; P < 0.0001) but not osteopenic women (13.8% versus 33.6%; P < 0.0001). Women with multiple RFs had more on-study osteoporosis-related fractures than women with a single RF (osteoporosis group: 9.9% versus 6.2%; P = 0.0092; osteopenia group: 11.2% versus 4.7%; P < 0.0001). In postmenopausal women receiving osteoporosis treatment, multiple RFs increased fracture risk. RFs, in addition to bone mineral density, can help identify candidates for osteoporosis treatment.

The burden of metastatic melanoma: treatment patterns, healthcare use (utilization), and costs.

Using a large national claims database, this study investigates the differences in survival and healthcare costs of metastatic melanoma patients by the number of metastatic sites. An analysis was carried out using data obtained from January 2007 to March 2010. Patients included had at least two claims for metastatic disease at least 30 days apart, at least two claims for melanoma at least 30 days apart, or at least one claim for cancer-related treatment with a diagnosis of melanoma and evidence of anticancer systemic therapy. The index date was the first metastatic diagnosis date. Patients were characterized as having evidence of lymph node (LN) involvement only, 1-3 distant metastatic sites, or 4+ distant metastatic sites. Average per-patient per-month (PPPM) costs and mortality were examined. There were 431 metastatic melanoma patients: most were male (65%) with mean baseline Charlson's comorbidity index of 3.52. The mean (SD) total unadjusted costs PPPM in the follow-up period were lower for patients with metastases to LN only ($6773 [$5521]) than for those who had 1-3 ($10 999 [$11 319]) or 4+ ($15 762 [$12 377]) distant metastases (P<0.001). When compared with patients with LN metastases only, patients having 1-3 [cost ratio (CR): 1.739, P<0.001] or 4+ (CR: 2.375, P<0.001) distant metastatic sites had higher all-cause healthcare costs. Among the entire study cohort, 42% died with a median survival time of 270 days. Mortality varied by cohort: 3% in LN only; 37% in 1-3 non-LN, and 64% in >3 non-LN (P<0.001). In conclusion, patients with metastatic melanoma with a greater number of metastatic sites have increased healthcare costs and significantly worse survival times.

The association of rash severity with overall survival: findings from patients receiving erlotinib for pancreatic cancer in the community setting.

OBJECTIVES: This retrospective study examined pancreatic cancer patients who received combination gemcitabine and erlotinib to determine if the association between rash and outcomes observed in clinical trials would be observed in 'real-world' community oncology settings. METHODS: Medical records from 10 community oncology practices were used to identify eligible patients. Rash severity was classified as High (moderate/severe) versus Low (absent/mild) based on medical record review. Kaplan-Meier analysis assessed progression-free survival (PFS) and overall survival (OS) by rash status from a landmark of 42 days after treatment initiation. Cox regression with time-varying covariates tested whether high-severity rash predicted longer OS and PFS. RESULTS: The High Severity group (n = 34) had longer median OS from the landmark than the Low Severity group (n = 134; 7.58 months vs 5.03 months, P = 0.0339). Cox regression analysis (n = 174) confirmed a reduced risk of death with High Rash Severity (hazard ratio [HR] = 0.67, P = 0.0389). Progression-free survival results showed a similar pattern (median PFS 2.37 months from landmark vs 2.04 months for High vs Low Severity groups, P = 0.0485). CONCLUSIONS: Results from this community sample were consistent with findings from randomized clinical trials, showing that longer OS is predicted by high-severity rash in erlotinib-treated pancreatic cancer patients.

Comparative effectiveness of chemotherapy in elderly patients with metastatic colorectal cancer.

PURPOSE: Treatment advances have improved outcomes in clinical trials of patients with metastatic colorectal cancer (mCRC). Less is known about these effects for patients in real-world settings. This study evaluated treatment patterns and survival in older, demographically diverse patients with mCRC. METHODS: A retrospective cohort analysis was performed for 4,250 patients from January 1, 2000 to December 31, 2007 using linked Surveillance, Epidemiology, and End Results-Medicare database. Patients were ≥ 66 years, enrolled in Medicare parts A and B, and received first-line treatment with fluorouracil and leucovorin (5-FU/LV), capecitabine (CAP), 5-FU/LV plus oxaliplatin (FOLFOX), or CAP and oxaliplatin (CAPOX). Cox regression with backward elimination and propensity score-weighted Cox regression estimated relative risk of death. Date of last follow-up was December 2009. Statistical comparisons were made between 5-FU/LV vs. CAP and FOLFOX vs. CAPOX. RESULTS: Compared to 5-FU/LV, patients treated with CAP were older (mean age 78 vs. 76; P<0.0001) and more likely female (61 vs. 54 %; P=0.0017), while patients receiving CAPOX and FOLFOX were similar in age (mean age 74 vs. 73; P=0.0924). Complications requiring medical resource utilization following initiation of therapy were significantly higher among patients administered with 5-FU/LV (54 %) vs. CAP (17 %; P<0.0001) and FOLFOX (75 %) vs. CAPOX (57 %; P<0.0001). The multivariate analysis revealed no significant differences in survival between 5-FU/LV and CAP and between FOLFOX and CAPOX. CONCLUSIONS: Overall survival was comparable between CAP and 5-FU/LV and between CAPOX and FOLFOX with fewer complications requiring medical resource utilization associated with CAP and CAPOX, thus confirming clinical trial results.

Cost-effectiveness of rituximab as maintenance therapy in patients with follicular non-Hodgkin lymphoma after responding to first-line rituximab plus chemotherapy.

A recent phase III trial demonstrated that maintenance rituximab® therapy after response to first-line treatment with rituximab plus chemotherapy (R-chemo) increases progression-free survival (PFS) for follicular non-Hodgkin lymphoma (f-NHL). A cost-effectiveness analysis of R-maintenance versus observation was conducted from a US payer perspective to estimate PFS and overall survival (OS) over a representative patient's lifetime. Primary outcomes were cost per life-year gained (LYG) and cost per quality-adjusted life-year (QALY) gained. Compared with observation, R-maintenance increased mean PFS by 1.50 years, OS by 1.21 years and QALYs gained by 1.11 years. The incremental cost of maintenance therapy was $38,545. The costs per LYG and QALY gained were $31,934 and $34,842, respectively. Within the limitations of modeling long-term outcomes, R-maintenance therapy in patients who received R-chemo for previously untreated f-NHL compared with observation alone after R-chemo for first-line treatment for f-NHL is cost-effective from the US payer perspective.

Costs and clinical outcomes among patients with second-line non-small cell lung cancer in the outpatient community setting.

INTRODUCTION: A comparison of clinical and economic outcomes among patients receiving second-line monotherapy with erlotinib, docetaxel, and pemetrexed for non-small cell lung cancer was conducted using a large network of outpatient community clinics. METHODS: We identified 610 patients with advanced non-small cell lung cancer who received 2L treatment from July 1, 2006, to June 30, 2008, and were followed up through July 1, 2009, to evaluate progression-free survival (PFS), overall survival (OS), costs, and health resource utilization. Cox proportional hazards regression were used to compare PFS and OS across treatment cohorts. Economic outcomes were calculated per patient per month (PPPM) during a 12-month follow-up period. RESULTS: There were 73 patients who received erlotinib, 87 received docetaxel, and 450 received pemetrexed. The median age was 67 years, and 55% were men. No significant differences in stage, baseline performance status, hemoglobin level, or body mass index were observed by treatment. The median OS was 132 days for docetaxel, 132 days for pemetrexed, and 155 days for erlotinib (p = 0.39). Adjusting for age, gender, stage, performance status, and hemoglobin level, there was no significant association between treatment type and OS (p = 0.36) or PFS (p = 0.26). Relative to pemetrexed, total adjusted costs PPPM was $1579 lower for docetaxel and $1584 lower for erlotinib (p < 0.05). Outpatient visits, laboratory procedures, and acute care visits were also less frequent with erlotinib relative to pemetrexed (-2.6 PPPM, p < 0.05). CONCLUSIONS: We observed no significant differences in OS and PFS between patients receiving erlotinib, docetaxel, and pemetrexed. Nevertheless, erlotinib and docetaxel were associated with a statistically significant lower costs and resource use relative to pemetrexed.

Cost-effectiveness of adding rituximab to fludarabine and cyclophosphamide for the treatment of previously untreated chronic lymphocytic leukemia.

A recent phase III trial demonstrated improved progression-free survival (PFS) and overall survival (OS) associated with adding rituximab to fludarabine and cyclophosphamide (R-FC) compared to FC in treatment of previously untreated chronic lymphocytic leukemia (CLL). A cost-effectiveness analysis of R-FC over FC was performed from a US third-party payer perspective over a lifetime horizon in the base case. One-way, two-way and probabilistic sensitivity analyses were conducted to assess the robustness of the results. A secondary analysis was performed by also considering a societal perspective. R-FC was associated with an incremental 1.15 quality-adjusted life-years (QALYs) compared to FC and resulted in an incremental cost-effectiveness ratio of $23 530 per QALY in the base case and $31 513 per QALY considering a societal perspective. Results were most sensitive to time horizon, discount rate and unit drug cost for rituximab. Within the limitations of modeling long-term outcomes, R-FC is cost-effective for previously untreated CLL.

Development, reliability, and validity of a new Preference and Satisfaction Questionnaire.

OBJECTIVES: Existing questionnaires that assess preference and/or satisfaction with postmenopausal bone loss treatments were reviewed and determined to be inadequate for the assessment of an oral pill versus a subcutaneous injection. The Preference and Satisfaction Questionnaire (PSQ) was developed to assess preference, satisfaction, and bother with a weekly oral tablet versus a once every 6 months subcutaneous injection for treatment of postmenopausal bone loss. METHODS: Questions were developed based on literature review and expert input. Content validity of the PSQ in this patient population was assessed among current or previous bisphosphonate users in group interviews, and item comprehension and readability were also evaluated. Reliability, validity, and structure of the questionnaire were assessed in two phase 3 randomized clinical trials. RESULTS: Twenty-four women participated in cognitive interviews and found the PSQ understandable and acceptable. Subsequently, 1583 trial participants took the PSQ. Interitem correlations, ranging from 0.50 to 0.97 for preference items, 0.85 to 0.94 for pill-satisfaction items, and 0.84 to 0.92 for injection-satisfaction items, and a well-fitting confirmatory factor analysis (root mean square error of approximation 0.04, nonnormed fit index 0.99, and root mean square residual 0.08) supported the structure of the instrument. Cronbach's alpha reliability values for pill satisfaction, injection satisfaction, pill bother, and injection bother were 0.93, 0.89, 0.82, and 0.61, respectively. Discriminative validity was indicated with better satisfaction and bother scores being related to adherence and the absence of adverse events. CONCLUSIONS: The PSQ is a valid and reliable measure and may be a valuable tool to assess patient preference and satisfaction with a weekly oral tablet and 6-month subcutaneous injection for postmenopausal bone loss.

Impact of medication adherence on health care utilization and productivity: self-reported data from a cohort of postmenopausal women on osteoporosis therapy.

BACKGROUND: Many pharmacologic agents are approved for the prevention and treatment of osteoporosis, which is common among postmenopausal women. Evidence exists relating treatment persistence to fracture risk. Less is known about treatment persistence and the use of health care service and individual productivity. OBJECTIVE: This study was undertaken to describe health care use and productivity loss relative to osteoporosis medication persistence using women's self-reported data from the Prospective Observational Scientific Study Investigating Bone Loss Experience (POSSIBLE US™), a large, longitudinal (October 2004-December 2009) osteoporosis cohort study of postmenopausal women. METHODS: Analyses included women on pharmacologic osteoporosis therapy (alendronate, risedronate, ibandronate, calcitonin, raloxifene, or teriparatide) who provided health care use/productivity data collected using semiannual questionnaires over 1 year of follow-up. Participant characteristics, use, and productivity metrics were summarized. Logistic regression models and generalized linear models were used to examine use, time missed from usual activities, number of days spent in bed, and lost work time relative to treatment persistence, adjusting for potential confounders. RESULTS: At entry, of the 2528 women studied (91% white, 3.1% Hispanic/Latino, 2.3% African American/black, 1.1% Asian, and 2.1% American Indian/Native Alaskan, Native Hawaiian/Pacific Islander, or other; mean age, 64.6 [range, 37-97] years), 43.1% had osteoporosis and 23.4% had a previous fracture. After adjustment, subjects who switched therapies during follow-up were more likely to have had any kind of diagnostic testing (95.2% of switchers vs 91.2% of persistent subjects and 88.9% of discontinuers, P < 0.05). Discontinuers were less likely than persistent subjects to visit their primary care physicians (92.0% vs 94.4%, P = 0.0337). Variations in the number of days spent in bed, time missed from usual activities, and work loss (n = 852 employed subjects) by treatment persistence were not significant. CONCLUSIONS: Use of diagnostic testing differed significantly by osteoporosis treatment status. Compared with women who persisted with treatment, primary care provider visits were less common among those who discontinued treatment. Treatment persistence was not associated with significant differences in productivity measures.