I have blogged extensively about biomarkers, which are becoming a critical component in the evolution of predictive medicine. One of the goals of research in predictive medicine is to identify biomarkers that can be used to identify individuals at high risk for developing a disease in early detection trials. I have also written extensively about the early health model whereby diseases are identified and treated in their preclinical, presymptomatic states using panels of biomarkers. The presumed advantage of the early health model, not yet proven, is that early diagnosis and treatment can be more effective for the patient and less costly.
In many cases, chronic lymphoid leukemia (CLL) is a slowly progressing and rather indolent disease. However, a recent report (see: Researchers Find Predictive Tests and Early Treatment Delay Progression of Blood Cell Cancer) discusses the identification of early-stage, but high-risk, chronic lymphoid leukemia (CLL) with predictive biomarkers. For me, this illustrates another facet of the early health model. Below is an excerpt from the article with boldface emphasis mine:
Mayo Clinic researchers say they have moved closer to their goal of providing personalized care for a common blood cell cancer. They have found that the use of predictive biomarkers along with two targeted treatments significantly delays the need for conventional chemotherapy in patients with early-stage, but high-risk, chronic lymphoid leukemia (CLL). Their study,...found that in a small group of patients the use of the new tools delayed the need for standard chemotherapy treatment to about four years after cancer diagnosis....However, they say that because of these promising findings, all CLL patients at Mayo Clinic now undergo the predictive tests, whose results can be used to risk-stratify therapy, including enrollment in ongoing experimental treatments if appropriate. The two targeted therapies studied, alemtuzumab and rituximab, are widely used in advanced-stage CLL....“The standard of care [up to this time for CLL] patients is to watch and wait until patients develop more advanced disease and then to treat them with chemotherapy and monoclonal antibodies,” [says an author of the study]. “We believe this new approach is better for patients because it identifies those who will develop aggressive CLL sooner than later and helps delay need for more toxic treatments....Researchers at Mayo Clinic and other centers have developed a number of different biomarker tests to predict, with reasonable certainty, which patients are at high-, low- or moderate-risk for progression of their cancer. They have found that testing CLL cells for overproduction of CD38 and ZAP-70 proteins, and analyzing the altered state of several genes as well as chromosomal defects in the cells can significantly predict cancer that will grow faster.
This work suggests that the definition of predictive medicine and predictive tests is now being expanded to include the risk-stratification of patients with early forms of a disease such as chronic lymphoid leukemia (CLL) using biomarkers. The overproduction of CD38 and ZAP-70 proteins identifies those patients who will go on to develop the more aggressive form of the disease. Rather than waiting until such patients develop more aggressive disease and require treatment with both chemotherapy and monoclonal antibodies, they are treated earlier with the latter type of agents. Such treatment delays the need for standard chemotherapy by about four years.
Look for more work along these same lines, which is to say the use of biomarkers for risk-stratification and earlier treatment for those with the worst prognosis. One example of this approach is the expanding use of biomarkers in nomograms which can be to predict patient outcomes and thereby aid in therapeutic decision-making (see: The Ingredients for Prostate Cancer Nomograms: The Addition of Biomarkers Sets the Table for Future Recipes).
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