Guido Tricot, MD, PhD

In the early 1980s, McElwain andcolleagues demonstrated thathigh-dose melphalan (Alkeran,100–400 mg/m2) was very effectivein patients with aggressive (plasmacell leukemia) or refractory myeloma.[1] Other researchers subsequentlyconfirmed these results.[2-4]Unfortunately, the duration of cytopeniaassociated with such treatmentwas excessive (3 to 4 weeks), leadingto a treatment-related mortalityrate of 10% to 20%.

Multiple myeloma is a multistep malignancy, starting with an indolent phase (monoclonal gammopathy of unknown significance [MGUS] or smoldering myeloma), progressing to overt myeloma (which is typically restricted to the bone marrow), and