by Christian Morrow, Courier Staff Writer
In the U.S., there are about 100,000 people, almost all African Americans, who live with Sickle Cell Disease. And while medical advances have increased their life expectancies, a result is increased risk for organ damage, such as cardiopulmonary complications, and kidney or liver failure.
One cause is iron toxicity, a side effect of transfusions used to give Sickle Cell Disease (SCD) patients more healthy red blood cells. One way some doctors have addressed the problem in high-risk patients is via a technique called “Red Cell Exchange Transfusion,” where a patient’s sickled cells are removed as new healthy cells are introduced.
However, the process takes longer, is more expensive, and there is disagreement in the medical community on the technique’s effectiveness, and when it should be used, if at all.
That may soon change.
On July 9, the University of Pittsburgh and UPMC announced they would lead the largest-ever clinical trial of the technique, thanks to a $19.2 million grant from the National Institutes of Health.
Co-principal investigator Darrell Triulzi, M.D., professor of pathology and director of the division of transfusion medicine at the Pitt School of Medicine, told the New Pittsburgh Courier there is no consensus on the treatment’s value and the only studies on it have been small. This trial will enroll 150 patients at 22 sites over five years—each will receive the treatment for 12 months, patients will randomly be assigned to receive either the standard of care alone or in combination with monthly red blood cell exchange treatments.
“One of the reasons it is so large is we wanted a definitive study,” Dr. Triulzi said. “There will be enough patients in the study to answer whether or not it is beneficial. We’ve had great success getting young patients to survive into adulthood, but once there, they begin to suffer from organ damage—and there is no other treatment for this population.”
So, only patients exhibiting pulmonary hypertension, heart failure or chronic kidney disease will be eligible. Most will be patients already enrolled in the UPMC Sickle Cell program.
Of the approximately 700 sickle cell patients in Southwestern Pennsylvania, around 500 are enrolled at UPMC, but only about 30 percent show the organ damage the trial is targeting.
This study will count deaths and hospitalizations for organ damage complications in both the control and experimental groups. If there is a significant difference, it could change the standard of practice.
“If the trial shows efficacy it will become the de facto standard of care,” said Dr. Triulzi. “This is extremely important because it can give patients hope that a treatment will soon be available. We also want the community’s help, support and involvement.”
For patients not enrolled in the UPMC program who may fit the criteria and would like to participate, Dr. Triulzi said they should contact the UPMC Department of Hematology/Oncology so they can be evaluated.
The Pitt Graduate School of Public Health will serve as the data coordinating center for the trial, with principal investigator Maria Mori Brooks, Ph.D., professor of epidemiology, leading the development and administration of the data collection and statistical analyses.
Some of the participating sites outside Pittsburgh include the University of Illinois at Chicago—which helped design the study protocols—Duke University, Emory University, University of California San Francisco Benioff Children’s Hospital, University of Alabama at Birmingham, Imperial College London (in the United Kingdom), and Henri Mondor Hospital in Paris, France.
Like us at https://www.facebook.com/pages/New-Pittsburgh-Courier/143866755628836?ref=hl
Follow @NewPghCourier on Twitter https://twitter.com/NewPghCourier
About Post Author
