Sickle cell blood study begun
Pitt, UPMC get major fed grant
Researchers from the University of Pittsburgh and UPMC are launching an international study funded by a $ 19.2 million grant from the National Institutes of Health that will assess an experimental treatment for sickle cell anemia that drains patients of their blood.
Don’t worry — not all of the blood is removed, and it gets replenished.
The procedure is called red cell exchange or erythrocytapheresis ( pheresis), and it’s a two- hour transfusionlike process that replaces 70% of a patient’s blood with donor blood. The procedure has been around since the 1980s, but physicians don’t agree on how effective it is at treating sickle cell anemia.
“There’s never been a really good study on this,” said Dr. Mark Gladwin, Pitt Department of Medicine chair, who is one of three principal investigators on the Pittsburgh team with Dr. Darrell Triulzi and Maria Brooks, a Pitt professor of epidemiology.
The grant is designed to conduct a better study and create consensus among doctors on treatment efficacy.
Sickle cell anemia or sickle cell disease — a blood disease that affects 100,000 mostly black Americans ( and 700 Western Pennsylvanians) — is caused by a mutation in the gene for hemoglobin ( the blood protein that carries oxygen). With it, red blood cells break apart and take on a crescent or sickle shape.
Sickled cells are sticky and don’t flow easily through tiny blood vessels. When they clog, patients experience extreme pain.
“When [ the pain] is really bad, it’s consuming,” said Gale Langhorn- Cox, 60, one of 30 sickle cell patients currently being treated with the red cell exchange at UPMC but who is not part of the study. “You feel it everywhere.”
Even though she faces daily pain, Ms. LanghornCox is outliving most others who have dealt with the illness. While medications like hydroxyurea have extended the lives of those with sickle cell, on average men die at 42 and women at 48, Dr. Gladwin said.
Because sickled cells can’t carry adequate amounts of oxygen, organs don’t receive enough of it. They become damaged and eventually fail, leading to a patient’s death.
“We don’t know how to keep them from dying,” Dr. Gladwin said.
According to Dr. Triulzi, not knowing is what motivated the grant proposal — the team wanted to find out.
The grant will fund a 7year clinical trial led by the Pittsburgh researchers that will involve 150 patients receiving treatment at 22 medical sites in the U. S., United Kingdom and France. The University of Illinois at Chicago, Duke University and Imperial College London, as well as Pitt and UPMC, are among the sites.
The treatment that participants in the Sickle Cell Disease and Cardiovascular Risk — Red Cell Exchange trial receive will be randomized. Some will undergo the red cell exchange while others instead will receive “the best standard of [ alternative] care,” according to Dr. Gladwin.
He expects that those treated with red blood cell exchange will show improved health and organ function. Ms. Langhorn- Cox said the procedure makes her feel better and more energized.
She has been receiving the treatments since 2013. When she first started them, Ms. Langhorn- Cox underwent red cell exchange once every four weeks. Now she visits the hospital once every six weeks to have it done. Patients receiving the treatment in the trial will get it monthly.
But Dr. Triulzi said there are downsides to the procedure as well: Recipients can develop antibodies against donor blood, iron can build up in their bodies, and infections and blood clots can develop. Also, each exchange treatment requires a lot of blood ( 6 to 8 maximum donations/ units), time and money.
Doctors at UPMC are uncertain of actual patient cost, but Ms. Langhorn- Cox said her insurance covers the cost of the procedure.
If the study shows that the exchange doesn’t work, there are alternatives to it. Bone marrow transplants have been successful in curing the disease, but they aren’t a universal solution, Dr. Gladwin said.