The cancer journal
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This prospective phase II pilot study evaluated safety and efficacy of transarterial chemoembolization (TACE) with drug-eluting beads (DEBs) loaded with doxorubicin in patients with unresectable hepatocellular carcinoma (HCC). ⋯ DEB-TACE is safe and effective in achieving local tumor control in patients with unresectable HCC.
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In the twenty-first century, melphalan-prednisone can no longer be regarded as the standard treatment of multiple myeloma patients not eligible for high-dose melphalan followed by autologous stem cell transplantation. The introduction of thalidomide, lenalidomide, and bortezomib has improved the arsenal of therapeutic options in multiple myeloma. ⋯ In this review, we discuss the role of novel therapies in multiple myeloma in elderly multiple myeloma patients. Important aspects, such as toxicity and the role of prognostic factors, are also addressed.
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Phase II oncologic clinical trials for agents that are mainly growth inhibitory and benefit only a selected patient population are challenging. The randomized discontinuation trial design is one approach by which this can be accomplished. ⋯ The primary end point is the fraction of patients who remain progression free after an additional postrandomization period or the time to progression after randomization. By enriching for a possible sensitive population and then testing whether this was due to the agent or selection of an indolent disease group, the randomized discontinuation trial design efficiently assesses the putative growth inhibitory properties of an investigational agent and furthermore minimizes the number of patients exposed to placebo.
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The emergence of many newer, molecularly targeted anticancer drugs requires that we rethink the way that we conduct phase II trials in oncology. In particular, we can no longer afford to advance drugs (or combinations) to phase III trials with a high risk of failure to improve on outcomes. ⋯ We use the example of advanced nonsmall cell lung cancer to demonstrate how randomized phase II trials have already made an impact in oncology, whereas single-arm phase II trials have led to negative phase III trials in the same disease. Finally, we make the case that randomized phase II trials are feasible, as long as reasonable statistical standards are applied.
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Since 1992, the Food and Drug Administration has allowed for accelerated approval of cancer drugs in cases where a statistically significant and clinically meaningful improvement in survival or side effect over alternative therapies is clearly demonstrated in controlled randomized trials. To this effect, endpoints other than overall survival (OS) are accepted as surrogate markers for survival. A lack of consensus exists regarding the validity of progression-free survival (PFS) as a true measure of outcome due to treatment. ⋯ The magnitude of the improvement in PFS is the magnitude of the improvement in OS. PFS is simply a measure of a drug's effect on tumor growth while it is administered and is not a surrogate for OS. Although PFS should not replace OS in regulatory approval consideration should be given to studies that treat beyond current definitions of progressive disease as a strategy to augment OS.