Melphalan plus Stem-Cell Transplantation Improves Kidney Function in Patients with Primary Amyloidosis. Ann Intern Med. 2001;134:S91. doi: 10.7326/0003-4819-134-9_Part_1-200105010-00007
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Published: Ann Intern Med. 2001;134(9_Part_1):S91.
Primary (AL) amyloidosis is an uncommon condition in which abnormal bone marrow cells make proteins that form thin bands or fibrils of a substance called amyloid. The amyloid fibrils can settle in many organs of the body and lead to organ failure and death. One of the most common organs affected is the kidney. Patients with kidneys affected by amyloid typically lose large amounts of protein in their urine because the amyloid interferes with the filtering mechanism of the kidney. These patients can eventually develop kidney failure and require dialysis. Approximately 20% of people with AL amyloidosis have some improvement if they take chemotherapy drugs, such as melphalan. However, it is rare for this treatment to stop new amyloid production completely. A newer therapy allows the use of much higher doses of melphalan to more effectively destroy the abnormal bone marrow cells. To allow the bone marrow to recover from the chemotherapy, stem cells are collected from the patient's blood before chemotherapy is administered. The stem cells are given back to the patient after chemotherapy and allow the bone marrow to recover normal functioning.
To see whether high-dose melphalan plus autologous stem-cell transplantation improves kidney function in patients with kidneys affected by AL amyloidosis.
65 patients with AL amyloidosis who were losing large amounts of protein in their urine.
The researchers took stem cells from the patients and then gave the patients high doses of melphalan intravenously on 2 days. One to 3 days after administering melphalan, the researchers returned the patients' own stem cells through intravenous infusions. The researchers then followed patients for 1 to 2 years to see if bone marrow and kidney function improved.
In 65 patients, the abnormal bone marrow cells disappeared, and there was no evidence of continued production of amyloid. Fifty of the 65 patients lived longer than 12 months. Kidney function improved in about one third of the 50 survivors and in more than two thirds of the 23 persons who no longer had evidence of amyloid production. While this therapy was successful in some patients, more than 20% had serious side effects from the intensive therapy. Side effects included immediately worsening kidney function, fluid in the lungs, infection, and bleeding.
The study had no comparison group. Therefore, it is not possible to tell whether the intensive therapy is better than, the same as, or worse than other, less intensive therapies used for AL amyloidosis.
Intensive therapy with high-dose melphalan followed by autologous stem-cell transplantation is difficult and toxic. However, it may improve both bone marrow and kidney function in some patients with AL amyloidosis.
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