New Options Emerging for Patients with Cardiac Amyloidosis

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Multidisciplinary UH team manages these patients

Innovations in Cardiovascular Medicine & Surgery | Summer 2019

Promising new therapies are causing cardiologists to take a second look at cardiac amyloidosis – and how they can diagnose patients earlier in the disease’s progression.

“Cardiac amyloidosis is much more common than previously appreciated,” says Guilherme Oliveira, MD, Director of the Advanced Heart Failure and Transplant Center at UH Cleveland Medical Center; and Professor of Medicine, Case Western Reserve University School of Medicine. “For a number of years, it was relegated to an unimportant diagnosis because we had no treatments. But now, with the treatments that have become available, it needs to come to the forefront of people’s minds.” 

Guilherme Oliveira, MDGuilherme Oliveira, MD

“In the past, most diagnoses of cardiac amyloidosis were grouped with HFpEF – heart failure with preserved ejection fraction,” Dr. Oliveira adds. “People didn’t try to segment those patients because the treatment was basically the same. Now, because there are disease-modifying treatments for cardiac amyloidosis that have been shown to increase survival, it’s important to identify patients with amyloidosis within the HFpEF population.”

Two new therapies have emerged in the last year or so to treat transthyretin (ATTR) amyloidosis – distinct from the light chain (AL) amyloidosis that can be a consequence of clonal plasma cell disorders such as multiple myeloma.

Patisiran (Onpattro) was approved by the U.S. Food and Drug Administration (FDA) in August 2018 for patients with the hereditary form of ATTR cardiac amyloidosis who also have peripheral neuropathy.

“It’s a RNA silencer – it prevents the transcription of the protein in the liver,” Dr. Oliveira says.

In a trial of 225 patients reported in The New England Journal of Medicine, patisiran was shown to improve several clinical manifestations of hereditary ATTR amyloidosis, including quality of life, body mass index and gait speed.

Even more recently, in early May 2019, a small molecule called tafamidis earned  FDA approval. In the ATTR-ACT trial, also reported in The New England Journal of Medicine, tafamidis was shown to reduce all-cause mortality and cardiovascular-related hospitalizations in ATTR cardiac amyloidosis patients, compared with placebo. It also slowed decline in patients’ functional capacity and quality of life. 

“Both of these new drugs have shown significant clinical benefit,” Dr. Oliveira says. “In the future, this disease is going to be treated by multiple drugs given at multiple stages or perhaps a cocktail of drugs. You will give one drug that will slow down the transcription in the liver, another drug that will stabilize the circulating protein in the blood, another one that will prevent deposition and yet another one that will scavenge and dissolve the amyloid that’s already been deposited. Now that tafamidis is approved, I intend to use it in conjunction with patisiran in my cardiac amyloidosis patients.”

According to Dr. Oliveira, there are several clinical clues that can alert a cardiologist that their patient may have cardiac amyloidosis.

“On history and physical exam, a lot of patients with HFpEF that have cardiac amyloid also will have had a history of carpal tunnel syndrome or spinal stenosis and back pain,” he says. “These other diseases occur about five to 10 years before a diagnosis of cardiac amyloidosis is made. On echocardiogram, there is also large left ventricular hypertrophy (LVH), but on EKG, LVH is absent. Cardiac amyloidosis is an infiltrative disease, and it replaces the myocardial muscle, so it doesn’t conduct electricity. The QRS instead of being very tall, which is typical with LVH because of muscle, actually becomes very short or low-voltage. It’s what we call electro-echocardiographic LVH dissociation.”

“When you see LVH on the echo, you can do two things,” he adds. “You can order a pyrophosphate scan. It will only light up if there is transthyretin amyloid in the heart. You can also order an echocardiogram with strain and look at the pattern of strain deposition. If you find something that has been described as an apical sparing pattern, that would suggest a diagnosis of cardiac amyloidosis.  If you find an LVH patient and order a simple troponin and the troponin is positive, that also has a 90 percent sensitivity for the diagnosis of cardiac amyloidosis.”

At UH, cardiac amyloidosis patients are managed by a multidisciplinary team that includes Dr. Oliveira, Ehsan Malek, MD, from Hematology/Oncology, Arash Rashidi, MD, from Nephrology, and cardiac amyloid nurse Mary Okorn, RN. UH heart failure specialist Monique Robinson, MD, also manages a number of cardiac amyloidosis patients using CardioMEMS -- the miniaturized, wireless sensor implanted to continuously monitor pulmonary artery (PA) pressures.

“These patients are very hard to manage from a fluid standpoint once they develop heart failure, but we’ve found that CardioMEMS has really been incredibly helpful in keeping these people out of the hospital and keeping them fluid-balanced,” Dr. Oliveira says.

For more information about treatment options for cardiac amyloidosis at UH or to make a referral, please call 216-844-3800.

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