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Connections in Common


I read with interest the article by Doug Birch in the Winter 2017 issue concerning ivacaftor treatment for a cystic fibrosis patient with a rarer CF mutation [“A Genetically Unique CF Patient”]. It is wonderful that ivacaftor provided benefit to this patient who does not carry the G551D mutation. 

Johns Hopkins has an additional connection with ivacaftor. I was chairman of the clinical pharmacology department at Vertex Pharmaceuticals and was a primary developer on the ivacaftor program. Together with my colleagues Juihong Zha and John Mondrick from the Metrum Research Institute, we modeled the dose response of ivacaftor and determined the dose and schedule. Dr. Zha and I wrote the clinical pharmacology sections of the first label and the documents for the regulatory filings.

Johns Hopkins was one of over 100 clinical trial sites where the drug was studied. The large number of sites was necessary because the particular CF mutation for which the drug is approved is not a common one among CF patients in a disease that is, itself, not common.

Karen Kumor, M.D. ’75

(Biochemistry 1969-74; House Staff, Pediatrics, 1975-77; Fellow, Clinical Pharmacology, 1977-79)

Vaccines Save Lives

The HPV vaccine has been licensed for use in adolescents and young adult women for over a decade and subsequently approved for HPV disease prevention in boys and young men. It is wrong to suggest that the mechanism of action and effects have not been well studied and/or that mass vaccination is not a cost-effective strategy to prevent HPV-related disease [Letters, Winter 2017]. 

In fact, prior to licensure, HPV vaccines were tested on approximately 75,000 people, according to the Centers for Disease Control and Prevention (CDC). Since licensure, CDC reports nearly 90 million doses of vaccine have been distributed. There is no evidence that HPV vaccine causes autoimmune diseases. There is considerable evidence, however, that HPV infections cause genital warts and cervical and anogenital cancers and that HPV vaccination substantially reduces the risk for infection. 

While Dr. Holtzman is correct that the HPV vaccine does not obviate the need for Pap testing to detect cervical cancer, his argument fails to account for the full range of HPV-related diseases prevented by vaccination. Further, Pap testing is designed to detect disease that has already occurred, not to prevent it. Also of concern is that screening strategies for other HPV-related cancers are not well established.  

While the CDC will continue to monitor reports of adverse events and investigate causal links to vaccines, including HPV, to quote a recent statement from the American Academy of Pediatrics: “Vaccines are safe. Vaccines are effective. Vaccines save lives.” As pediatricians, if we are not clear on these facts, we contribute to the public mistrust that undermines the progress that has been made in vaccine preventable diseases.

Krishna K. Upadhya, M.D., M.P.H.

Assistant Professor, School of Medicine

Hitting the Bull’s Eye

I read with interest the article about Antony Rosen and others working in “precision medicine” [Winter 2017]. First, let me agree with Rosen who states that a “one-size-fits-all approach to health care” no longer exists.

If the clinical decision is “precise” but not necessarily accurate, it is not very useful.  Consider a target on a rifle range; if the shots have a tight grouping anywhere on the target, they are precise but they are only precise and accurate if that tight grouping is in the bull’s eye.

In order to make the right clinical decision for the right patient at the right time, judgment is required. To do this properly requires knowledge of multiple parameters, including risk profiles; clinical trials data; family history; factors such as obesity, diabetes and smoking; imaging studies; blood tests; functional studies; genetics; and common sense (often related to the experience of the physician).

I am all for being as precise and accurate as possible when making decisions about individual patients. Some believe that precision medicine will hit the bull’s eye. To me that is a statement of hope—and I hope those who think that way are correct. In my opinion, [physician] judgment (which also includes experience, risk factor profiles and clinical trials) is still necessary to make clinical decisions for the individual patient, and it will stay that way for a long time. I certainly agree with Sir William Osler, who said, “Care more for the individual patient than for the special features of his disease.”

C. Richard Conti ’60, M.D.

(Osler House Staff, 1960-61,1964-65), Professor of Medicine (Cardiology), University of Florida, College of Medicine

Setting the Record Straight

I appreciate being mentioned in Class Notes [Winter 2017] for having received Pediatric Hospital Medicine’s first Lifetime Achievement Award. But I should set the record straight: I am identified as “chairman emeritus of pediatrics” at the University of North Carolina but am professor emeritus of pediatrics, not chairman emeritus.

Kenneth B. Roberts, MD ’69