The “big, big, big mistakes” we make in drug therapy with sick infants


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The big news in medicine today is the announcement of a promising new gene-based therapy for certain leukemia patients – children and young adults aged 3 to 25 – who were facing death because every other treatment had failed: 52 of 63 patients who received the therapy last year went into remission – “a high rate for such a severe disease.”

The reports come on the heels of yesterday’s Food & Drug Administration hearing aimed at carefully assessing how safe and effective the therapy is. The FDA advisory committee wasn’t concerned about effectiveness; instead, they zeroed-in on reports of the side-effects – raging fever, crashing blood pressure, lung congestion, infertility, and the possibility of causing secondary cancers. After hearing the evidence the committee unanimously recommended that the FDA approve the drug. The agency will announce their decision in October.

And that’s how drug approval is done – except for, of all people, neonates: premature and full-term infants less than 28 days old – babies so tiny, their little hands can’t wrap around their parents’ index fingers.

Infants admitted to a neonatal intensive care unit may receive up to 60 medications – antibiotics, anesthetics, narcotics, diuretics – in their first month of life. Yet according to a report in the online medical journal STAT, 90% percent of these drugs have not been approved by the FDA for use in newborns.

Instead, doctors make treatment decisions by scaling down from how medications are used in adults: “We take it right out of the vial of an adult drug, dilute it down, and give it to the babies,” says Dr. Jonathan Davis, chief of newborn medicine at Tufts Medical Center. Doctors make decisions based on little more than anecdotes and intuition – essentially treating each sick newborn as an uncontrolled, unapproved study of one.

Result: “There have been some big, big, big mistakes in neonatology through the years when it comes to drugs,” said neonatologist Dr. Matthew Laughon of the University of North Carolina at Chapel Hill. Examples of what’s been admitted to publicly are the sudden deaths of preemies due to too-large doses of the antibiotic chloramphenicol in the 1950s; the fatal poisoning of infants from large amounts of benzyl alcohol, a preservative used to flush catheters, in the 1980s; and deaths from a preservative, propylene glycol, in a multivitamin given orally to premature infants in a dose intended for adults.

This happens because how drugs affect people is strongly age-dependent: infants absorb, metabolize, and excrete drugs differently than adults. “Yet we haven’t done the studies to know exactly what those differences are,” said Catherine Sherwin, chief of pediatric clinical pharmacology at the University of Utah School of Medicine. “We just know they’re different.”

Why is there no medical evidence and no FDA approval process for 90% of the drugs used on newborns? Fear and fear of lawyers: Pharmaceutical companies shy away from studying infants because they are fragile, cannot spare many blood samples, and are vulnerable to permanent injuries – injuries that, in the past, have been awarded large malpractice verdicts. And second, money: Newborns are a small market, so pharmaceutical companies aren’t likely to make money by getting drugs approved for neonate use.

So where does that leave us? “We’ve got to do something,” says Dr. Davis. Without drug data for newborns, he said, “we can’t be certain which drugs, in which doses, to use when.”



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