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A Phase II Study of Risk-Adapted IV Melphalan in Patients with AL Amyloidosis AL amyloidosis is a rare and devastating disease. It is a bone marrow disorder where abnormal proteins (free light chains) are produced and deposit in the tissues, disrupting organ function and ultimately leading to death if left untreated. The amyloid protein may be deposited in any organ of the body but most often is found in the kidneys, heart, liver, bowel, and nerves. The care of patients with AL amyloidosis has often been difficult due to its rarity, the diversity of organ (and therefore specialist) involvement, and until recently a lack of treatments options. This trial is examining treatment with a type of chemotherapy called melphalan. It is known that melphalan delivered at intermediate-dose or at high-dose with stem cell transplantation has considerable activity in AL amyloidosis, although the toxicity of the transplant procedure remains concerning. Therefore, there is a pressing need to identify the optimal mode of delivery of melphalan for individual patients. The trial is evaluating a risk-adapted melphalan dosing strategy to deliver a safe stem cell transplant to those who can tolerate the procedure reserving the less toxic intermediate-dose melphalan for patients who are not candidates for transplantation. The reduction in free light chains measured six months after treatment will be the main outcome measure of this strategy as patients who achieve a reduction in their free light chains are known to live longer. Attached to the trial are other scientific studies which will help us learn more about amyloidosis. This study is funded by the Leukaemia Foundation, Amgen Australia and the Princess Alexandra Hospital Foundation. It is being conducted under the auspices of the Australasian Leukaemia and Lymphoma Study Group and is currently enrolling new patients on the trial. Further information can be obtained from the Trial Coordinator (Jan Alexander 07 3240 5112) or from the Principle Investigator (Dr Peter Mollee 07 3240 2396). |