Use of the metastatic breast cancer progression (MBC-P) questionnaire to assess the value of progression-free survival for women with metastatic breast cancer.

While overall survival (OS) has historically been the primary endpoint for clinical trials in oncology, progression-free survival (PFS) has gained acceptance as a valuable surrogate endpoint. However, there are no known published reports about the value of PFS from the patient's perspective. We developed a questionnaire that included items regarding quality of life (QoL) and the importance of different treatment outcomes and presented hypothetical scenarios for which respondents were asked to indicate their preferences concerning treatments as they relate to PFS. 282 women with metastatic breast cancer (MBC), ranging in age from 21 to 80 years completed an online version of this questionnaire. The majority of women (66 %) had been diagnosed with MBC within the previous 3 years and 56 % had been told their MBC had progressed. When asked to rank five treatment characteristics from most important to least important, respondents ranked "extending PFS" as the second most important treatment outcome after OS. When presented with a hypothetical scenario of two women receiving different treatments, respondents preferred the treatment that resulted in longer PFS (16 vs. 12 months), even when OS and side effects were assumed to be equal. Specifically, when asked to consider which woman within the hypothetical scenario had better QoL, physical functioning, and emotional well-being, respondents more often chose the woman who experienced longer PFS (QoL: 40 vs. 6 %; physical functioning: 32 vs. 8 %; emotional well-being: 58 vs. 6 %) compared to the woman within the hypothetical scenario who had a shorter time of progression. Respondents rated their own QoL highest after being told their MBC was responding to treatment (mean score 76.6) versus after the initial diagnosis of breast cancer and MBC (68.5 and 60.3). These findings suggest that extending PFS is an important treatment outcome and, from a patient perspective, improves overall QoL, physical functioning, and emotional well-being

Identification and cost of adverse events in metastatic breast cancer in taxane and capecitabine based regimens.

PurposeWe sought to compare the economic impact of treatment-related adverse events (AEs) in patients with metastatic breast cancer (mBC) using taxane- or capecitabine-based treatment regimens as either first- or second-line (FL or SL) therapy in the US.MethodsWe used healthcare claims data from the Truven Health Analytics MarketScan® Commercial Databases to conduct a retrospective cohort study comparing the economic impact of AEs amongst taxane- and capecitabine-treated mBC patients in the US. We selected women diagnosed with mBC between 2008-2010 who received a taxane or capecitabine as first- or second-line (FL or SL) chemotherapy. Costs related to hospitalization, outpatient services, emergency department visits, chemotherapy and other medications were tabulated and combined to determine total healthcare costs. The incremental monthly costs associated with the presence of AEs compared to no AEs were estimated using generalized linear models, controlling for age and Charlson Comorbidity Index.ResultsWe identified 15,443 mBC patients meeting inclusion criteria. Adjusted total monthly costs were significantly higher in those who experienced AEs than in those without AEs in both lines of treatment (FL incremental cost: taxanes $1,142, capecitabine$1,817; SL incremental cost: taxanes $1,448, capecitabine$4,437). Total costs increased with the number of AEs and were primarily driven by increased hospitalization amongst those with AEs.ConclusionsAdverse events in taxane- or capecitabine-treated mBC patients are associated with significant increases in costs. Selecting treatment options associated with fewer AEs may reduce costs and improve outcomes in these patients

Assessing the discordance rate between local and central HER2 testing in women with locally determined HER2-negative breast cancer.

BackgroundThe importance of human epidermal growth factor receptor 2 (HER2) as a prognostic and predictive marker in invasive breast cancer is well established. Accurate assessment of HER2 status is essential to determine optimal treatment options.MethodsBreast cancer tumor tissue samples from the VIRGO observational cohort tissue substudy that were locally HER2-negative were retested centrally with both US Food and Drug Administration (FDA)-approved immunohistochemistry (IHC) and fluorescence in situ hybridization (FISH) assays, using FDA-approved assay cutoffs; results were compared.ResultsOf the 552 unique patient samples centrally retested with local HER2-negative results recorded, tumor samples from 22 (4.0%) patients were determined to be HER2-positive (95% confidence interval [CI] = 2.5%-5.7%). Of these, 18 had been tested locally by only one testing methodology; 15 of 18 were HER2-positive after the central retesting, based on the testing methodology not performed locally. Compared with the 530 patients with centrally confirmed HER2-negative tumors, the 22 patients with centrally determined HER2-positive tumors were younger (median age 56.5 versus 60.0 years) and more likely to have ER/PR-negative tumors (27.3% versus 22.3%). These patients also had shorter median progression-free survival (6.4 months [95% CI = 3.8-15.9 months] versus 9.1 months [95% CI = 8.3-10.3 months]) and overall survival (25.9 months [95% CI = 13.8-not estimable] versus 27.9 months [95% CI = 25.0-32.9 months]).ConclusionsThis study highlights the limitations of employing just one HER2 testing methodology in current clinical practice. It identifies a cohort of patients who did not receive potentially efficacious therapy because their tumor HER2-positivity was not determined by the test initially used. Because of inherent limitations in testing methodologies, it is inadvisable to rely on a single test to rule out potential benefit from HER2-targeted therapy

The impact of site of care (office clinic versus hospital outpatient) on treatment patterns in HER2-positive breast cancer.

e11077 Background: This study examines treatment (tx) patterns in patients (pts) with breast cancer (BC) receiving trastuzumab (T) in a physician clinic (MD) &amp; outpatient hospital (HOSP) setting. Methods: Pts ≥18 yrs with BC (≥2 claims with ICD-9-CM 174.xx, ≥30 days apart) and ≥2 T claims from 1/1/2006 to 12/31/2010 were identified from a large US commercial insurance claims database. Pts continuously enrolled for ≥ 6mths before &amp; after index date (date of 1st T claim) were included. Pts receiving T at both sites of care, receiving T before the index date &amp;/or pts with &gt; 1 primary cancer were excluded. Metastatic (mBC) vs. early stage (ESBC) tx was identified by presence of ≥2 claims of metastases after index date. Pts were followed from index date to 30 days after the last infusion prior to a gap of ≥ 90 days, death, disenrollment, or end of study period. Pts were stratified by site of care- MD or HOSP. Descriptive &amp; multivariate analyses were conducted to examine differences in tx patterns (duration and doses/mo) &amp; number of missed doses during the tx period. Results: 2,823 BC pts receiving T were identified; 480 (17%) were treated for mBC. Most patients received T in the MD setting (94% ESBC pts and 87% of MBC pts). Compared to the MD setting, pts treated in the HOSP were more likely to be older (ESBC: 61 vs 53 yrs; MBC: 64 vs 54 yrs; each p &lt;0.001) &amp; have Medicare benefits (ESBC: 54% vs 7%; MBC: 63% vs 9%; each p &lt;0.001). There was no difference in mean baseline Charlson comorbidity index in any group. Mean T duration in HOSP was significantly shorter vs MD among ESBC pts (303 &amp; 333 days, p&lt;0.05) but not MBC pts (313 for HOSP vs 355 days for MD, p&gt;0.05). Compared to the MD setting, HOSP treated patients had more treatment gaps of 30-59 days (ESBC: 58% vs 25%; MBC 56% vs 27%; each p&lt;0.001) and fewer infusions/month (ESBC: mean 1.45 vs 1.99; MBC 1.54 vs 2.07; each p&lt;0.001). In multivariate analyses, infusion counts remained lower among HOSP treated patients (ESBC: IRR 0.74, CI 0.67-0.79; MBC: IRR 0.76, CI 0.66-0.87). Conclusions: MBC and ESBC pts treated in the MD setting were younger and received more infusions with fewer treatment gaps than HOSP pts. Further research assessing the impact of these differences in tx patterns on clinical outcomes is needed. </jats:p

Treatment patterns and outcomes with HER2-directed therapy for the treatment of HER2-positive metastatic breast cancer.

e11063 Background: The goal of this study was to compare treatment patterns and outcomes in patients receiving a HER2 directed therapy for the treatment of HER2+ metastatic breast cancer (mBC). Methods: Female mBC patients (with ≥2 claims with ICD9 diagnosis codes for mBC), age ≥18 years receiving treatment with trastuzumab (T) or lapatinib (L) between 1/1/06 and 8/31/11 were identified in the HealthCore Integrated Research Database (HIRDSM). The earliest mBC claim was defined as index date. Patients with &lt;12 months preindex and ≥6 months postindex continuous eligibility were excluded. Two cohorts were identified: T group received T only; TL group received both L and T (sequentially or together). Treatment duration, metastasis site (mets) at mBC diagnosis, no. of chemotherapy agents, no. of hospitalizations, and total cost of care were assessed in the two groups. Results: Of 1449 patients who received a HER2 directed agent, 1165 were in the T group (mean age 56 yrs) and 284 in the TL group (mean age 54 yrs). T group mets included bone (n=553, 48%), liver (n=336, 29%) and brain (n=283, 24%) mets; TL group had bone mets (n=225, 79%), brain mets (n=167, 59%) and liver mets (n=153, 54%). Mean no. of chemotherapy agents received was lower in T group 3.3 (SD 1.7) compared to TL group 5.8 (SD 2.1) (p=0.0002). The time from 1st mBC diagnosis to treatment with T or L was 117 (SD 217) days in T group and 107 (SD 208) days in TL group. For TL group, time between index date and treatment was 114 (SD 214) days for T and 690 (SD 400) days for L. The mean treatment duration was shorter in T group 417 (SD 366) days compared to TL group 724 (SD 433) days (p&lt;0.0001). TL patients received 579 (SD 402) days of T and 205 (SD 234) days with L. Number of hospitalizations and total cost of care (mean monthly) were 0.1 (SD 0.2) and $12561 (SD$10,975), respectively for T group and 0.1 (SD 0.1) and $13,532 (SD$7,220), respectively for TL group. Conclusions: Mean treatment duration and mean number of chemotherapy agents suggest that the TL group may have received more lines of treatment than the T group. There was no difference in mean monthly costs between the two groups. Medical chart data will be used to confirm treatment patterns and evaluate treatment outcomes in both groups. </jats:p

Use of the metastatic breast cancer progression (MBC-P) questionnaire to assess the value of progression-free survival for women with metastatic breast cancer.

While overall survival (OS) has historically been the primary endpoint for clinical trials in oncology, progression-free survival (PFS) has gained acceptance as a valuable surrogate endpoint. However, there are no known published reports about the value of PFS from the patient's perspective. We developed a questionnaire that included items regarding quality of life (QoL) and the importance of different treatment outcomes and presented hypothetical scenarios for which respondents were asked to indicate their preferences concerning treatments as they relate to PFS. 282 women with metastatic breast cancer (MBC), ranging in age from 21 to 80 years completed an online version of this questionnaire. The majority of women (66 %) had been diagnosed with MBC within the previous 3 years and 56 % had been told their MBC had progressed. When asked to rank five treatment characteristics from most important to least important, respondents ranked "extending PFS" as the second most important treatment outcome after OS. When presented with a hypothetical scenario of two women receiving different treatments, respondents preferred the treatment that resulted in longer PFS (16 vs. 12 months), even when OS and side effects were assumed to be equal. Specifically, when asked to consider which woman within the hypothetical scenario had better QoL, physical functioning, and emotional well-being, respondents more often chose the woman who experienced longer PFS (QoL: 40 vs. 6 %; physical functioning: 32 vs. 8 %; emotional well-being: 58 vs. 6 %) compared to the woman within the hypothetical scenario who had a shorter time of progression. Respondents rated their own QoL highest after being told their MBC was responding to treatment (mean score 76.6) versus after the initial diagnosis of breast cancer and MBC (68.5 and 60.3). These findings suggest that extending PFS is an important treatment outcome and, from a patient perspective, improves overall QoL, physical functioning, and emotional well-being