lunes, 26 de octubre de 2015

Exceptional Responders in Oncology

Medscape Internal Medicine > George Lundberg: At Large at Medscape
COMMENTARY
Exceptional Responders in Oncology: A Response Worth Sharing

George D. Lundberg, MD
Disclosures | October 22, 2015



Those of us active in the field were defining, developing, and maturing evidence-based medicine (EBM) in the late 1980s and all through the 1990s and 2000s. I credit Dr David Eddy[1] with having first published the term in the Journal of the American Medical Association [now called JAMA] and Dr David Sackett[2] with doing the lion's share of early development and promotion.

EBM considers large, randomized, controlled, preferably double-blinded, clinical trials—with big enough numbers to boast statistical power—to be the "Holy Grail," the sine qua non, the absolute essential for establishing medical research information as close to "truth" as you can get.

Of course, the results of such clinical trials were often in conflict with the results of other similar trials. Thus, the field of meta-analysis came to be, to endeavor to get even closer to truth. But there were always those pesky outliers.

Then came some results from the Human Genome Project.[3] We learned—almost in disbelief, and to our group consternation—that often, especially with cancer but also with common cardiovascular and other diseases, the essential "apples-with-apples" comparisons that were so laboriously sought were flawed. When viewed genomically, cancers with like names were, as often as not, mixtures of apples with oranges, and with grapefruit. And maybe even pomegranates. Oh my!

Medians and means were still medians and means. But clinical trials that were considered "failed" statistically may well have been successful for some patients, even those far removed from the mean or median—those outliers.

Now we know that cancer in fact often is many, varied genomic, proteomic, metabolomic diseases wrapped into what was once considered, by location, morphology, and patterns, to be a single cancer diagnosis. That anatomical diagnosis—non-small cell lung cancer, or pancreatic adenocarcinoma, or malignant glioma—must now be divided into types, and those into subtypes, and those into sub-subtypes.

Alexander Pope said, "The proper study of mankind is man."[4] I would further say, "The proper study of me is me," and "The proper study of my cancer would be my cancer."

Most cancer treatment in the United States is not provided in comprehensive cancer centers or within controlled clinical trials. Most cancer patients are treated by practicing, community clinical oncologists. These treatment results are not part of studies, and thus often are not captured and shared with the broader medical community.

Cancer patients may experience "exceptional responses" to therapies. Many cancer researchers are now thinking that some of these cases might be of serious intellectual and scientific value, over and above the obvious clinical value for individual patients.

Cancer Commons, a not-for-profit organization based in Palo Alto, California, is endeavoring to change that by collecting and sharing such valuable therapeutic, experiential information so that others can learn from it. Other possible similar examples of clinical therapeutic responses could then be grouped and compared. (Full disclosure: I was a founder of the organization in 2011. I remain an unpaid advisor and strongly believe in its mission.)

In order to encourage the collection and sharing of information about "exceptional responders," thereby heightening awareness and increasing the opportunity for study, another Palo Alto entity, the Cureus journal of medicine, has teamed up with Cancer Commons to promote the literary competition known as "Exceptional Responders in Oncology". (Disclosure: I am an unpaid executive advisor to Cureus and strong advocate for post-publication peer review).

Please submit your case reports of "exceptional responders" (positive or negative) to Cureus between October 22 and November 23, 2015. All published case reports will be judged by reader/reviewer "crowdsourced" post-publication peer review. There is a monetary prize of $3000 for the winning author(s).

This is an exercise intended to actually find exceptional responders—the further study of whom might unlock some cancer mysteries—as well as to call widespread attention to the importance of the concept of practicing physicians now being able to easily share their important clinical observations with their fellow physicians via true open-access publishing.

Give it a try. Help us improve the world of cancer care.

That's my opinion. I'm Dr George Lundberg, At Large for Medscape.

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