Amyloidosis Treatment

Besides treatment with chemotherapy-based regimens, researchers at NYP are involved in studying novel therapies for amyloidosis including medications that are used for the treatment of multiple myeloma. In addition, some patients may be eligible for stem cell transplantation. Because amyloidosis is a progressive disease, doctors at NYP will offer an eligible patient a transplant immediately. If a patient with amyloidosis is eligible and in good condition (eg, < 3 organs affected, good cardiac function, and good laboratory criteria), a bone marrow transplant specialist at NYP can harvest the stem cells and perform the transplant. If the patient has more advanced disease, the team at NYP will pretreat the patient with a chemotherapy regiment to downstage the amyloidosis, and then perform the stem cell transplant. Bone marrow transplant specialists at NYP have found this procedure to be very promising for patients with amyloidosis.

Cardiac Amyloidosis

When there is abnormal accumulation of amyloid protein in the heart, patients may experience symptoms such as shortness of breath, dizziness, and edema. Accumulation of amyloid protein in the heart reduces the ability of the heart to fill and can result in damage to the heart's electrical system. If left untreated, cardiac amyloidosis can lead to heart failure. Therefore, doctors at NYP place a high priority on intervening early before this major organ is affected.

Doctors at NYP typically begin with a physical exam, followed by blood and urine tests and an electrocardiogram. Next, an echocardiogram test is performed. If the tests are consistent and there is suspicion of cardiac amyloidosis, a definitive diagnosis can be confirmed by a biopsy of the heart. The procedure is safe if performed at an experienced institution. Doctors at NYP have significant expertise in performing a endomyocardial biopsy because NYP has the one of the largest heart failure and transplant programs in the country and they routinely perform more than 20 heart biopsies per week.

Recently, researchers at NYP have been successfully employing cardiac magnetic resonance imaging (CMRI) – a less invasive method – to accurately diagnose cardiac amyloidosis. Doctors at NYP have also developed a novel scanning technique using technetium pyrophosphate that can accurately distinguish between the two most common forms of cardiac amyloidosis – AL and ATTR.

If the patient has a confirmed diagnosis of amyloid in the heart, the next step is for NYP doctors to determine the type of cardiac amyloidosis – primary (AL) or hereditary (ATTR). About half of patients who present at NYP with amyloid in the heart have AL and the other half have ATTR amyloidosis. The most common form of cardiac ATTR is wild type, or senile cardiac amyloidosis. NYP cardiologists collaborate with hematologists-oncologists to devise the best treatment course for patients with cardiac amyloidosis.

Typical treatment for primary (AL) cardiac amyloidosis includes chemotherapy to limit the accumulation of protein in the heart, as well as diuretics and blood thinners among other therapies. As the population continues to age, TTR has become more commonplace. Doctors at NYP are heavily involved in research studies targeting patients with TTR amyloid.

Kidney Amyloidosis

Amyloid protein may deposit in the kidneys and lead to organ dysfunction. Patients may experience symptoms such as foamy urine or leg edema. Doctors at NYP will conduct a simple urine test to assess for the presence of amyloid protein in the urine. Occasionally, a biopsy may need to be taken from the kidney to confirm the diagnosis.

Typically, the treating nephrologist at NYP will collaborate with a hematologist-oncologist to first treat a potentially underlying plasma cell dyscrasia. If the treatment is effective in limiting further amyloid production, kidney function can be stabilized and may even improve. The nephrologist may then treat any lingering symptoms of kidney amyloid, such as fluid retention.

Kidney amyloidosis inhibits the kidneys from properly removing waste from the body, and if left untreated can lead to kidney failure. Therefore, doctors at NYP place a high priority on intervening early before the kidney is seriously affected. Once the amyloid is under control, a kidney and bone marrow transplant is also an option for eligible patients.