The role of PBSCT in treatment of AL amyloidosis is far from settled
2016
Our experience with M-Dex is similar, with a good response rate, which is probably a little bit lower than with high-dose therapy but with fewer treatment-related deaths, especially in a multicentric setting. Moreover, responses with M-Dex can be very rapid, with a complete response after 1 course for some patients, and most responses occurring before 6 months. Thus, in contrast to Mehta, we believe that benefit/risk ratios of high-dose versus an effective conventional therapy like M-Dex must be compared in a randomized fashion in patients with primary amyloidosis. In our randomized trial, which is ongoing, M-Dex is compared to a high-dose regimen using melphalan (200 or 140 mg/m 2 , depending on age and clinical status) supported with autologous blood stem cells previously collected after mobilization with granulocyte colony-stimulating factor (G-CSF) alone. More than 80 of the 100 patients planned have been included already in 25 centers, demonstrating that such a study is realizable. We hope that it will be completed at the end of this year and will help to solve the still-persisting issue of dose intensity inAL.
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