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Etanercept therapy improves PROs in patients with psoriasis and makes a meaningful difference to their lives. These results support the efficacy profile of physician-reported clinical measures while providing a more complete understanding of the benefits experienced by patients with psoriasis treated with etanercept.

Adult chronic immune thrombocytopenic purpura (ITP) is a disorder manifested by varying degrees of purpura and mucosal bleeding, rarely including intracranial hemorrhage. Therapy is aimed at increasing the patient's platelet count to safe levels and includes a wide variety of treatments. While the diagnosis, treatment, and prognosis of chronic ITP have been extensively discussed, the effect of ITP and its treatment on patient quality of life has not been evaluated in adults. In this study, the Short-Form 36 questionnaire was used to evaluate the health-related quality of life (HRQOL) of 73 adult ITP patients compared with that of the general U.S. population and of patients with six other relatively common chronic disorders. This study shows that the HRQOL of adult patients with ITP is significantly worse than that of the general U.S. population. It is also worse than that of patients with hypertension, arthritis, or cancer; similar to that of patients with diabetes; but better than that of patients with congestive heart failure or a missing or paralyzed limb. Future studies need to address the effects of treatment not only on the platelet count and bleeding but also on HRQOL.

BACKGROUND.Adding trastuzumab to adjuvant chemotherapy provides significant clinical benefit in patients with human epidermal growth factor receptor 2 (HER2)‐positive breast cancer. A cost‐effectiveness analysis was performed to assess clinical and economic implications of adding trastuzumab to adjuvant chemotherapy, based upon joint analysis of NSABP B‐31 and NCCTG N9831 trials.METHODS.A Markov model with 4 health states was used to estimate the cost utility for a 50‐year‐old woman on the basis of trial results through 4 years and estimates of long‐term recurrence and death based on a meta‐analysis of trials. From 6 years onward, rates of recurrence and death were assumed to be the same in both trastuzumab and chemotherapy‐only arms. Incremental costs were estimated for diagnostic and treatment‐related costs. Analyses were from payer and societal perspectives, and these analyses were projected to lifetime and 20‐year horizons.RESULTS.Over a lifetime, the projected cost of trastuzumab per quality‐adjusted life year (QALY; discount rate 3%) gained was $26,417 (range, $9104‐$69,340 under multiway sensitivity analysis). Discounted incremental lifetime cost was $44,923, and projected life expectancy was 3 years longer for patients who received trastuzumab (19.4 years vs 16.4 years). During a 20‐year horizon, the projected cost of adding trastuzumab to chemotherapy was $34,201 per QALY gained. Key cost‐effectiveness drivers were discount rate, trastuzumab price, and probability of metastasis. The cost‐effectiveness result was robust to sensitivity analysis.CONCLUSIONS.Trastuzumab for adjuvant treatment of early stage breast cancer was projected to be cost effective over a lifetime horizon, achieving a cost‐effectiveness ratio below that of many widely accepted oncology treatments. Cancer 2007. © 2007 American Cancer Society.

Background and Purpose— Despite the availability of results from multiple newer clinical trials and changing healthcare costs, the cost-effectiveness of recombinant tissue-type plasminogen activator (r-tPA) for treatment of acute ischemic stroke within 0 to 3 hours of symptom onset was last evaluated in 1998 for the United States Using current evidence, we evaluate the long-term cost-effectiveness of r-tPA administered 0 to 3 hours after acute ischemic stroke onset versus no r-tPA. Methods— A disease-based decision model to project lifetime outcomes of patients after acute ischemic stroke by r-tPA treatment status from the US payer perspective was developed. Model inputs were derived from a recent meta-analysis of r-tPA trials, cohort studies, and health state preference studies. Cost data, inflated to 2013 dollars, were based on drug wholesale acquisition cost and the literature. To compare r-tPA to no r-tPA, we calculated incremental total direct costs, incremental quality-adjusted life years, and incremental cost-effectiveness ratios. We performed 1-way and probabilistic sensitivity analyses to evaluate uncertainty in the results. Results— r-tPA resulted in a gain of 0.39 quality-adjusted life years (95% confidence range, 0.16–0.66) on average per patient and a lifetime cost-saving of $25 000 (95% confidence range, −$42 500 to −$11 000) compared with no r-tPA. In probabilistic sensitivity analyses, r-tPA was dominant compared with no r-tPA in ≈100% of simulations. The model was sensitive to inputs for r-tPA efficacy, healthcare costs for disabled patients, mortality rates for disabled and nondisabled patients, and quality of life estimates. Conclusions— Our analysis supports earlier economic evaluations that r-tPA is a cost-effective method to treat stroke. Appropriate use of r-tPA should be prioritized nationally.

BACKGROUND This report describes the results of an analysis of patient‐reported outcomes from EMILIA (TDM4370g/BO21977), a randomized phase 3 study of the antibody–drug conjugate trastuzumab emtansine (T‐DM1) versus capecitabine and lapatinib in human epidermal growth factor receptor 2 (HER2)–positive locally advanced or metastatic breast cancer. METHODS A secondary endpoint of the EMILIA study was time to symptom worsening (time from randomization to the first documentation of a ≥ 5‐point decrease from baseline) as measured by the Trial Outcome Index Physical/Functional/Breast (TOI‐PFB) subset of the Functional Assessment of Cancer Therapy–Breast questionnaire. Predefined exploratory patient‐reported outcome endpoints included proportion of patients with a clinically significant improvement in symptoms (per TOI‐PFB) and proportion of patients with diarrhea symptoms (per Diarrhea Assessment Scale). RESULTS In the T‐DM1 arm, 450 of 495 patients had a baseline and ≥ 1 postbaseline TOI‐PFB score versus 445 of 496 patients in the capecitabine‐plus‐lapatinib arm. Time to symptom worsening was delayed in the T‐DM1 arm versus the capecitabine‐plus‐lapatinib arm (7.1 months versus 4.6 months, respectively; hazard ratio = 0.796; P = .0121). In the T‐DM1 arm, 55.3% of patients developed clinically significant improvement in symptoms from baseline versus 49.4% in the capecitabine‐plus‐lapatinib arm (P = .0842). Although similar at baseline, the number of patients reporting diarrhea symptoms increased 1.5‐ to 2‐fold during treatment with capecitabine and lapatinib but remained near baseline levels in the T‐DM1 arm. CONCLUSIONS Together with the EMILIA primary data, these results support the concept that T‐DM1 has greater efficacy and tolerability than capecitabine plus lapatinib, which may translate into improvements in health‐related quality of life. Cancer 2014;120:642–651. © 2013 American Cancer Society.

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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. Cancer 2014;120:2657–2664.