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Neo Adjuvant Clinical Trials

Posted 3/4/2014

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  First, a definition. "Neo-adjuvant" refers to treatment that is delivered after diagnosis and before definitive surgery. In breast cancer, this means chemotherapy, or, occasionally hormonal therapy, that is given after cancer has been diagnosed via a biopsy, but prior to more breast surgery. This may be the recommendation in several instances: if the cancer looks especially aggressive, so the wish is to begin chemotherapy asap, if the tumor is large and the hope is to shrink it so that smaller surgery (a lumpectomy vs a mastectomy) is possible, or sometimes for other reasons relevant to the particular situation. This has been a standard practice, in these instances, for some years now, so there is accumulating data about its value.

  Today's offering is a "Comment" from The Lancet that looks at how to define success in neoadjuvant clinical trials. The results seem to be more difficult to interpret than those from other trials, partly because the perspectives vary and interpretations come from various positions and questions. A basic one is whether cPr (complete pathological response, meaning that the tumor seems to vanish after treatment) is related to long-term survival. That is, does the seemingly vanished tumor in the breast mean that the woman lives longer or is it just a reassuring artifact that sadly does not change the course of metastatic disease.

  Here is the beginning of the article and a link to read more. If this situation is relevant to you, this is a very good piece that makes it quite understandable.

Defining success in neoadjuvant breast cancer trials

Improvements in treatment of early-stage breast cancer depend on clinical trials in the adjuvant and neoadjuvant settings. Adjuvant trials address the definitive endpoints of relapse and survival, but are large and expensive, and require years of follow-up. Neoadjuvant trials capture intermediate endpoints, such as pathological complete response to treatment (PCR), with the hope that these endpoints will translate into meaningful improvements in definitive ones. Compared with adjuvant trials, neoadjuvant trials need smaller sample sizes and are less expensive, and the endpoint of PCR is achieved in months, rather than years. For these reasons, the number of neoadjuvant randomized clinical trials has increased greatly. In 2013, the US Food and Drug Administration awarded accelerated approval to pertuzumab on the basis of data from neoadjuvant trials. The agency still insists on efficacy confirmation from large adjuvant trials for full approval, but has opened the door to neoadjuvant registration approaches.


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