Doctors are one step closer to having a risk biomarker to alert them to which of their pediatric stem cell transplant patients are likely to experience a potentially deadly side effect called sinusoidal obstruction syndrome (SOS).
There is a drug, defibrotide, approved to treat SOS. Paczesny hopes the results of the biomarker study will encourage defibrotide’s manufacturer to conduct a multicenter clinical trial testing whether giving it to patients who test positive for the risk biomarkers before they begin to show symptoms can prevent SOS from developing.
In this study, conducted at four academic medical centers across the country, the researchers wanted to see at what point the potential risk biomarkers would start showing unusual levels. They tested 80 patients’ plasma at two points in time: three days after the stem cell transplant and seven days after the stem cell transplant. SOS typically occurs within a month of transplant, so those two points in time would presumably give doctors enough time to act. And, in fact, the researchers found that measuring those biomarkers on day three following the transfer of hematopoietic cells did indicate which patients would go on to develop SOS.
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