Overall and event-free survival are not improved by the use of myeloablative therapy following intensified chemotherapy in previously untreated patients with multiple myeloma: a prospective randomized phase 3 study

Blood. 2003 Mar 15;101(6):2144-51. doi: 10.1182/blood-2002-03-0889. Epub 2002 Nov 27.

Abstract

We compared the efficacy of intensified chemotherapy followed by myeloablative therapy and autologous stem cell rescue with intensified chemotherapy alone in patients newly diagnosed with multiple myeloma. There were 261 eligible patients younger than 66 years with stage II/III multiple myeloma who were randomized after remission induction therapy with vincristine, adriamycin, dexamethasone (VAD) to receive intensified chemotherapy, that is, melphalan 140 mg/m(2) administered intravenously in 2 doses of 70 mg/m(2) (intermediate-dose melphalan [IDM]) without stem cell rescue (n = 129) or the same regimen followed by myeloablative therapy consisting of cyclophosphamide, total body irradiation, and autologous stem cell reinfusion (n = 132). Interferon-alpha-2a was given as maintenance. Of the eligible patients, 79% received both cycles of IDM and 79% of allocated patients actually received myeloablative treatment. The response rate (complete remission [CR] plus partial remission [PR]) was 88% in the intensified chemotherapy group versus 95% in the myeloablative treatment group. CR was significantly higher after myeloablative therapy (13% versus 29%; P =.002). With a median follow-up of 33 months (range, 8-65 months), the event-free survival (EFS) was not different between the treatments (median 21 months versus 22 months; P =.28). Time to progression (TTP) was significantly longer after myeloablative treatment (25 months versus 31 months; P =.04). The overall survival (OS) was not different (50 months versus 47 months; P =.41). Intensified chemotherapy followed by myeloablative therapy as first-line treatment for multiple myeloma resulted in a higher CR and a longer TTP when compared with intensified chemotherapy alone. However, it did not result in a better EFS and OS.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage
  • Cause of Death
  • Chromosome Aberrations
  • Combined Modality Therapy
  • Cyclophosphamide / administration & dosage
  • Dexamethasone / administration & dosage
  • Disease-Free Survival
  • Doxorubicin / administration & dosage
  • Female
  • Granulocyte Colony-Stimulating Factor / administration & dosage
  • Humans
  • Leukocyte Count
  • Male
  • Melphalan / administration & dosage
  • Melphalan / adverse effects
  • Middle Aged
  • Multiple Myeloma / mortality*
  • Multiple Myeloma / therapy*
  • Myeloablative Agonists / therapeutic use*
  • Prognosis
  • Prospective Studies
  • Quality of Life
  • Remission Induction
  • Stem Cell Transplantation
  • Survival Rate
  • Transplantation, Autologous
  • Vincristine / administration & dosage
  • Whole-Body Irradiation

Substances

  • Myeloablative Agonists
  • Granulocyte Colony-Stimulating Factor
  • Vincristine
  • Dexamethasone
  • Doxorubicin
  • Cyclophosphamide
  • Melphalan

Supplementary concepts

  • VAD protocol