As the myeloma community continues to investigate whether stem cell transplantation or novel agents is the best way to treat newly diagnosed myeloma patients, recent results from a Phase 3 clinical trial indicate that stem cell transplantation may lead to longer disease-free survival.

The results from this study show that more multiple myeloma patients who received a stem cell transplant were alive and disease-free after 24 months compared to patients treated with Revlimid in combination with conventional chemotherapy.

The transplanted patients, however, also experienced more severe side effects compared to non-transplanted patients.

Dr. Antonio Palumbo, the lead author of the study from the University of Torino in Italy, presented the results at the 16^th Congress of the European Hematology Association (EHA) in London on Sunday.

Novel agents have improved myeloma treatment in recent decades, prompting researchers to investigate whether they may be as effective as stem cell transplantation in myeloma patients.

According to Dr. Palumbo, these results are the first to show that stem cell transplantation may be superior to novel agents for progression-free survival in myeloma patients.

Dr. Palumbo’s study included 402 newly diagnosed myeloma patients under the age of 65. All patients received four 28-day cycles of initial therapy that included 25 mg Revlimid ^[1] (lenalidomide) daily for three weeks and low-dose dexamethasone ^[2] (Decadron) weekly.

After their initial treatment, half of the patients continued with six 28-day cycles of conventional chemotherapy plus novel therapy, which included melphalan ^[3] (Alkeran) and prednisone ^[4] plus 10 mg Revlimid daily for three weeks (MPR).

The other half received two courses of high-dose melphalan, plus cyclophosphamide ^[5] (Cytoxan) and granulocyte colony-stimulating factor to increase the number of stem cells prior to collection.  This was then followed by stem cell transplantation.

After a follow-up of 12 months, interim results of the Phase 3 trial presented last June suggested that conventional chemotherapy in combination with Revlimid may be as effective as stem cell transplantation (see related Beacon ^[6] news).

After 24 months of follow-up, patients who received MPR achieved a similar complete response rate (20 percent) compared to patients who received a stem cell transplant (25 percent).

However, fewer patients who received MPR were disease-free at 24 months (59 percent), compared to patients who received a transplant (75 percent).

Overall survival remained high in both the MPR group (95 percent) and the transplanted group (97 percent) after 24 months.

The most common severe side effect was low white blood cell counts, which affected 55 percent of patients who received MPR and 89 percent of patients who received a stem cell transplant.

Patients who received a stem cell transplant also experienced higher rates of infection (17 percent versus 0 percent) and stomach and intestinal side effects (21 percent versus 0 percent), compared to patients who received MPR.

For more information, please see abstract 508 ^[7] on the EHA ^[8] website.