Georgia four-year old tests new drug to prevent bleeding

ATLANTA - Visits to Children's Healthcare of Atlanta are a family affair for Jackie and Greg Hogan and their 3 boys.

Patrick, the youngest, is the patient.

"Patrick is wild and funny," his father says. "He's sweet, too," Jackie Hogan adds.

The 4-year old, diagnosed as a newborn with a severe form of hemophilia A, has a port in his chest for medication, and he used to wear a helmet. Because the slightest bump or bruise could trigger dangerous internal bleeding in Patrick.

"He'd hit his head, and it was the size of a softball," Greg Hogan says."My mom sewed pads into all his pants, with the helmet, and we had gates all over our house. Just to kind of keep him in safe zones," Patrick's mother says.

The Hogans learned how to give Patrick infusions several times a week of medication, called a bypassing agent, to help his blood clot. But, then his body developed an inhibitor that blocked that medicine, making bleeds much more difficult to stop.

"Unfortunately, kids with inhibitors can develop spontaneous bleeding events, in which they can just spontaneously bleed into their abdomen, into organs, into their brains," says Dr. Robert Sidonio, Jr, a hemophilia specialist at Children's Healthcare, and a paid scientific advisor to the drugmaker Genentech.

He's part of a clinical trial to test the drug emicizumab or HEMLIBRA , designed to reduce the number of dangerous bleeding episodes in hemophiliacs. And Patrick is the first Georgia child to receive the drug.

"And, it's a quick stick in the thigh, and it's over, and we're done for week," Greg Hogan says.

"But, you also hold your breath," Jackie Hogan says. "Because there are claims, but it's a clinical trial, which can be a little bit scary for our kid."

But a year later, Patrick's bleeds have nearly stopped.

Dr. Sidonio says the drug won't completely stop the bleeds but can dramatically improve them.

"These kids were often developing up to 20 to 30 bleeds a year, and with the drug, they're having as little as zero to 1 to 2 bleeds a year," Dr. Sidonio says.

It means the Hogans can relax, and Patrick can act like a kid.

"When he falls now, I'm not running and checking, 'Okay, is this an ER visit or not?' That's not the case anymore," Greg Hogan says.  "He falls, 'Okay, you fell, let's get up. Let's put a Band-Aid on it.' You move on."

In late 2017, The Food and Drug Administration granted approval for emicizumab to be used in adults and children with hemophilia A with inhibitors.

Patrick Hogan will soon finish his part of the clinical trial.

He will be allowed to stay on the medication until his insurance provider begins to cover the medication.