• By Vivien Williams

Mayo Clinic goes off-label to treat severe bleeding disorder

April 26, 2019

For people with a rare bleeding disorder called "hereditary hemorrhagic telangiectasia," or HHT, simple issues such as a nosebleed can be life-threatening. The disease has no cure. But Dr. Vivek Iyer, a Mayo Clinic pulmonary and critical care physician, found that a drug commonly used for cancer can stop the bleeding and restore quality of life.

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When you watch Neeta Pai volunteering at a hospital gift shop, you'd never guess that not long ago, she almost died from a debilitating bleeding disease.

"I like to do charity work. It brings me a lot of joy," Pai says.

Her first symptom was a severe nosebleed.

"My nosebleeds were so severe. It would be just gushing like you would turn on the tap," Pai says.

They happened constantly and for no reason. Neeta would faint from blood loss.

"Any minor things would make my nose bleed. If I pinched my nose hard, the blood would go up into my ears, up in my head, and, of course, down the throat to the stomach. And my mouth was full of blood. Nobody could stop it," Pai says.

For years, she suffered, with no answers to her questions of why.

"Oh, they were terrible. I had no life. Absolutely no life," Pai says.

The nosebleeds worsened, and after one extreme episode, Neeta went into cardiac arrest. Her heart stopped.

"After that experience, I didn't want to live."

Hope came to Neeta when she met Dr. Iyer at Mayo Clinic. He diagnosed her with HHT.

"HHT stands for hereditary hemorrhagic telangiectasia," Dr. Iyer says.

People with the disorder develop tufts or tangles in their blood vessels.

"It basically affects blood vessels in almost every part of the body, and the most major areas of concern are the brain, the lungs, the nose, and the intestines and the liver," Dr. Iyer says.

And these tangles tend to bleed spontaneously. In fact, Dr. Iyer and his colleagues found that bleeding is the most important reason for hospitalization among HHT patients in the U.S. Dr. Iyer says frequent nosebleeds in these patients reduce quality of life and can be life-threatening.

"They can be catastrophic and can lead to death or severe disability," Dr. Iyer says.

But Dr. Iyer and his team have used a medication to help stop the bleeding. It's called bevacizumab (Avastin) — a cancer drug that prevents tumors from growing new blood vessels.  

"We took that idea from the cancer world … and it has worked out really, really well," Dr. Iyer says.

Dr. Iyer tested the drug in a study, and results show it can be life-changing for patients like Neeta. It's administered via infusion. Neeta says the medication has saved her.

"Since then, I have had no bleeds. It's given me my life back," Pai says.

Neeta pays it forward with her volunteer work, and she appreciates every single moment.

"I'm so full of gratitude. I count my blessings every day."

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