The Vanderbilt Amyloid Multidisciplinary Program (VAMP) is a new approach to treating a rare and misunderstood disease that can kill its victims before they understand what they have been diagnosed with.
Vanderbilt formally established VAMP a year ago to advance the understanding, treatment and research of amyloidosis, joining Boston University and the Mayo Clinic as one of the few places to offer a multidisciplinary approach to the often fatal disease, said Stacey Goodman, M.D., professor of Medicine and director of the VA Medical Center Bone Marrow Transplant Program.
“There has been an interest for a long time in developing a multidisciplinary program that deals with this very rare disease that is under-recognized. We have a group of providers at Vanderbilt with expertise in both their subspecialty and organ transplantation who also have an interest in amyloidosis,” she said.
The most common form of the disease is light chain (AL) amyloidosis, a plasma cell disorder that originates in the bone marrow and creates abnormal proteins called amyloid that build up in the body’s organs causing them to malfunction. It is a rare disease with only 1,200 to 3,200 new cases reported each year in the United States. VAMP cared for 50 patients over the past year.
It is a group of diseases that requires a team of specialists to treat it, including cardiologists, hematologists, gastrointestinal specialists, hepatologists, neurologists, pathologists and pulmonologists.
“We established VAMP to standardize and to commit to the process of providing structured and systematic multidisciplinary care to these patients,” Goodman said. “The first step is to maximize regular medical management of organ dysfunction, getting them clinically better and stronger and then treat the underlying form of amyloidosis.”
Amyloid can deposit in the kidney, liver, spleen, heart and bone marrow. Patients often undergo bone marrow transplant to get rid of amyloid and transplantation to replace the diseased organ. Many of the patients first present to Cardiology when they begin to experience signs of heart failure.
Charlotte Haffner, 61, was active and healthy when she began to experience shortness of breath, fatigue and swelling in 2008. She received a diagnosis of amyloidosis from Vanderbilt Heart cardiologist Joseph Fredi, M.D. She underwent a heart transplant and a stem cell transplant and now leads a support group for other patients with amyloidosis. The group meets monthly in The Vanderbilt Clinic.
“I decided to start the amyloidosis support group because I had no one to turn to when I went through my heart and stem cell transplants. Having a rare disease is difficult, but having no one to talk to about what to expect makes it even harder,” Haffner said.
“The support group is very important to me. It gives me a chance to give back, to share my experiences and maybe help smooth the way for someone who is confused and afraid.”
Aside from streamlining clinical management of these patients, VAMP is dedicated to improving diagnosis, disease monitoring and treatment through research, including studies of potential biomarkers, noninvasive imaging and drug trials.
For more information about VAMP, please call 936-8422. For a complete list of VAMP providers go to http://www.vanderbilthealth.com/hematology/37821.
