What is amyloidosis and what causes it?

Amyloidosis is the name for a group of rare, serious conditions caused by a build-up of an abnormal protein called amyloid in organs and tissues throughout the body. The build-up of amyloid proteins (deposits) can make it difficult for the organs and tissues to work properly.

How is AL amyloidosis treated?

Treatment

1. Melphalan – A chemotherapy drug used in high doses as part of a stem cell transplant.
2. Bortezomib – A new agent that blocks the proteasome, an enzyme in all cells, found to be very helpful in patients with multiple myeloma and most recently in patients with AL Amyloidosis.

Is Daratumumab approved for amyloidosis?

Subcutaneous Daratumumab Approved in Europe for Newly Diagnosed Light-Chain Amyloidosis, Pretreated Myeloma. The European Commission has granted marketing authorization for the expanded use of subcutaneous daratumumab for use in 2 new indications.

What blood tests indicate amyloidosis?

There is no specific blood test and results of investigations vary greatly from patient to patient. The diagnosis of amyloidosis starts when a doctor becomes suspicious of the patient’s symptoms. A definitive diagnosis of amyloidosis can only be made through a biopsy.

How fast does AL amyloidosis progress?

Median OS improved from 1.4 years in the 1980s to 4.6 years in the 2010s. This finding is in line with a study of patients with AL amyloidosis from the U.K., which reported an increase in survival from 17 months (1.4 years) in 1987–2005 to 51 months (4.25 years) in 2011–2015 [24].

What is Darzalex approved for?

The U.S. Food and Drug Administration (FDA) has approved Darzalex Faspro (daratumumab and hyaluronidase), in combination with Kyprolis (carfilzomib) and the corticosteroid dexamethasone, for the treatment of adults with multiple myeloma who have received one to three lines of therapy.

Does Darzalex treat amyloidosis?

DARZALEX FASPRO® in combination with bortezomib, cyclophosphamide, and dexamethasone is indicated for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis. This indication is approved under accelerated approval based on response rate.

What are amyloid spells?

TFNEs, also called amyloid spells, occur commonly in CAA as brief, recurrent, stereotypical episodes including both positive seizure-like and negative TIA-like phenomena. CAA is characterized by progressive deposition of amyloid-β in cortical and leptomeningeal vessel walls.

What are warning signs of amyloidosis?

Signs and symptoms of amyloidosis may include:

• Swelling of your ankles and legs.
• Severe fatigue and weakness.
• Shortness of breath with minimal exertion.
• Unable to lie flat in bed due to shortness of breath.
• Numbness, tingling or pain in your hands or feet, especially pain in your wrist (carpal tunnel syndrome)