What is the treatment for younger people with myeloma?

There are multiple systemic options for patients with myeloma. Symptomatic patients may be divided into two main categories: those who are eligible for high-dose chemotherapy with bone marrow transplantation (BMT; the minority) and those who are ineligible (the majority). In general, high-dose chemotherapy followed by BMT is reserved for younger patients in very good to excellent condition. For relatively young, healthy patients, a clear survival benefit has been shown in randomized controlled trials (RCTs) for this aggressive treatment over standard conventional dose chemotherapy. A couple of these RCTs showed that high-dose chemotherapy with BMT improved patients' survival by a full year or more, which is quite a difference for one treatment type over another.

Currently, initial treatment regimens for patients prior to BMT include combinations of the steroid dexamethasone with thalidomide, lenalidomide, and bortezomib with or without thalidomide. Thalidomide and lenalidomide (a derivative of thalidomide) act by decreasing new blood vessel formation from tumors, thereby preventing their growth. Bortezomib is an example of a newer class of biologic agent called a proteosome inhibitor. A major trial from the Netherlands revealed that incorporating thalidomide into initial treatment, called "induction," as well as after high-dose chemotherapy as "maintenance" therapy improved patients' outcomes. In the United States, lenalidomide is replacing thalidomide as maintenance treatment for patients with myeloma since it's generally better tolerated with less side effects.