A randomized phase III trial of thalidomide and prednisone as maintenance therapy following autologous stem cell transplantation (ASCT) in patients with multiple myeloma (MM) with a quality of life assessment: NCIC CTG MY.10 Trial
Mené sur 332 patients atteints d'un myélome multiple, cet essai de phase III évalue, du point de vue de la survie globale, une combinaison thalidomide-prednisone en traitement d'entretien après une greffe autologue de cellules souches (durée médiane de suivi : 4,1 ans)
We conducted a randomized controlled trial comparing thalidomide-prednisone as maintenance therapy with observation in 332 patients who had undergone autologous stem cell transplantation (ASCT) with melphalan 200 mg/m2. The primary endpoint was overall survival (OS); secondary endpoints were myeloma-specific progression-free survival (MS-PFS), PFS, incidence of venous thromboembolism (VT) and health-related quality of life (HRQoL). With a median follow-up of 4.1 years, no differences in OS between thalidomide-prednisone and observation were detected (respective 4-yr estimates of 68% vs. 60%; HR=0.77; P=0.18); thalidomide-prednisone was associated with superior MS-PFS and PFS (for both outcomes 4-yr estimates 32% vs. 14%; HR=0.56; P<0.0001) and more frequent VT (7.3% vs none; P=0.0004). Median survival after first disease recurrence was 27.7 months with thalidomide-prednisone and 34.1 months in those observed. Nine second malignancies were observed with thalidomide-prednisone versus six in observation. Those allocated to thalidomide-prednisone reported worse HRQoL with respect to cognitive function, dyspnea, constipation, thirst, leg swelling, numbness, dry mouth, and balance problems. We conclude that maintenance therapy with thalidomide-prednisone following ASCT improves the duration of disease control but is associated with worsening of patient-reported HRQoL and no detectable overall survival benefit. Conducted by the NCIC Trials Group (Canada) and the Eastern Cooperative Oncology Group (ECOG). ClinicalTrials.gov Number: NCT00049673