Christina T. Loguidice
Dialysis-related amyloidosis (DRA) is a rare complication of kidney failure that most commonly affects adults older than 60 years who have been on hemodialysis for more than 5 years. The condition results in a buildup of beta 2-microglobulin in the blood. As deposits collect in the bones, joints, and tendons, patients may experience pain and stiffness, and they become prone to sustaining fractures and other injuries. Deposits can also collect in the heart, lungs, and other vital organs, interfering with their function. Treatments have been limited, but the US Food and Drug Administration (FDA) recently approved the first device to treat this disabling and potentially life-threatening disorder: the Lixelle Beta 2-microglobulin Apheresis Column. The Lixelle Column is manufactured by Kaneka Corporation in Osaka, Japan, and is distributed in the United States by its subsidiary, Kaneka Pharma America.
“While DRA affects only a small population of patients on dialysis, there are not many treatment options for these patients and some options may not be available to patients in all areas,” said William Maisel, MD, MPH, deputy director for science, chief scientist, and acting director of the Office of Device Evaluation, Center for Devices and Radiological Health, in an FDA press release. “The Lixelle Beta 2-microglobulin Apheresis Column may provide this patient population with an option for relieving some of the debilitating symptoms of DRA.”
The Lixelle Column is added to hemodialysis equipment, and the patient’s blood passes through the Lixelle Column before entering the dialysis filter. The Lixelle Column contains porous cellulose beads that remove beta 2-microglobulin from the blood as it passes over the beads.
The FDA granted the Lixelle Column a Humanitarian Use Device designation based on data supporting its safety and probable benefit in approximately 100 patients in Japan with DRA who were treated with the device, as well as postmarket safety data from another approximately 200 patients in Japan, where the device is already approved. The data indicate that patients generally experienced an improvement in symptoms following use of the device and that the treatment was well tolerated. The most common adverse events included temporary hypotension and a lower hematocrit level, which are also common following dialysis or any extracorporeal therapy.
As part of its approval, Kaneka must conduct a postmarket study in the United States to ascertain the device’s benefits, risks, and adverse events. The Lixelle Column is expected to be particularly helpful to patients with DRA-related symptoms who lack access to extended dialysis therapies or are ineligible for kidney transplantation.
FDA authorizes use of first device to treat patients with dialysis-related amyloidosis [press release]. Bethesda, MD; US Food and Drug Administration; March 6, 2015. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm436864.htm?source=govdelivery&utm_medium=email&utm_source=govdeliveryamyloidosis/article/402237 Accessed April 6, 2015.