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`Medical )1 press
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`;,
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`Home / Oncology & Cancer
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`(J) DECEMBER 22, 2014
`Test predicts response to treatment for complication of
`leukemia stem cell treatment
`
`by The Mount Sinai Hospital
`
`A new test may reveal which patients will respond to treatment for graft versus host disease (GVHD), an
`often life-threatening complication of stem cell transplants (SCT) used to treat leukemia and other blood
`disorders, according to a study led by researchers at the Icahn School of Medicine at Mount Sinai and
`in print int e.January issue.
`published online today int e journa Lancet Haemato ogyan
`
`Patients with fatal blood cancers like leukemia often require allogenic stem cell SCT to survive. Donor
`stem cells are transplanted to a recipient, but not without the risk of developing GVHD, a life-threatening
`complication and major cause of death after SCT The disease, which can be mild to severe, occurs when
`the transplanted donor cells (known as the graft) attack the patient (referred to as the host). Symptom
`severity, however, does not accurately define how patients will respond to treatment and patients are
`often treated alike with high-dose steroids. Although SCT cures cancer in 50 percent of the patients, 25
`percent die from relapsed cancer and there remaining go into remission but later succumb to effects of
`GVHD.
`
`"High dose steroids is the only proven treatment for GVHD," said James L. M. Ferrara, MD, DSc, Ward(cid:173)
`Coleman Chair in Cancer Medicine Professor at the Icahn School of Medicine at Mount Sinai, Director of
`Hematologic Malignancies Translational Research Center at Tisch Cancer Institute at Mount Sinai. "Those
`with low-risk GVHD are often over-treated and face significant side-effects from treatment. Patients with
`high risk GVHD are undertreated and the GVHD progresses, often with fatal consequences. Our goal is to
`provide the right treatment for each patient. We hope to identify those patients at higher risk and design
`an aggressive intervention while tailoring a less-aggressive approach for those with low-risk."
`
`Dr. Ferrara, along with a multi-center team of researchers, developed and tested this new scoring system
`using almost 500 patient blood samples with newly diagnosed GVHD in varying grades from two different
`centers. They used three validated biomarkers TNFR1, ST2 and Reg3a to create an algorithm that
`calculated the probability of non-relapse mortality (usually caused by GVHD) that provided three distinct
`risk scores to predict the patient's response to GVHD treatment.
`
`The acid test was to evaluate the algorithm in a validation set of 300 addit ional patients from twenty
`different SCT centers throughout the US. The algorithm worked perfectly, and the cumulative incidence of
`non-relapse mortality significantly increased as the GVHD score increased, and so the response rat e to
`primary GVHD treatment decreased.
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`https ://med i calxpress.com/news/2014-12-response-treatment-com plication-leukemia-stem. html#
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`Pharmacyclics Exhibit 2043
`Sandoz v. Pharmacyclics
`JPR201 9-00865
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`1/2
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`Test predicts response to treatment for complication of leukemia stem cell treatment
`12/4/2019
`''This new scoring system will help identify patient who may not respond to standard treatments, and may
`require an experimental and more aggressive approach," said Dr. Ferrara. "And it will also help guide
`treatment for patients with lower-risk GVHD who may be over-treated. This will allow us to personalize
`treatment at the onset of the disease. Future algorithms will prove increasingly useful to develop
`precision medicine for all SCT patients."
`
`In order to capitalize on this discovery, Dr. Ferrara has created the Mount Sinai Acute GVHD International
`Consortium (MAGIC) which consists of a group of ten SCT centers in the US and Europe who will
`collaborate to use this new scoring system to test new treatments for acute GVHD. Dr. Ferrara and
`colleagues have also written a protocol to treat high-risk GVHD that has been approved by the FDA.
`
`Provided by The Mount Sinai Hospital
`
`Citation: Test predicts response to treatment for complication of leukemia stem cell treatment (2014,
`December 22) retrieved 4 December 2019 from https://medicalxpress.com/news/2014-12-response(cid:173)
`treatment-complication-leukemia-stem.html
`
`This document is subject to copyright. Apart from any fair dealing for the purpose of private study or
`research, no part may be reproduced without the written permission. The content is provided for
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