Long-term survival of stage I multiple myeloma given chemotherapy just after diagnosis or at progression of the disease: a multicentre randomized study

Abstract

We conducted a randomized trial to evaluate whether melphalan-prednisone (MPH-P) treatment administered just after diagnosis improves survival of stage I multiple myeloma (MM). Between January 1987 and March 1993, 145 consecutive previously untreated patients with stage I MM were randomized between treatment with MPH-P (administered for 4 days every 6 weeks) just after diagnosis and treatment only at disease progression. Survival was not influenced by MPH-P treatment either administered just after diagnosis or at disease progression (64 vs 71 months respectively). Comparing the first with the second group the odds ratio of death is 1.17 (95% confidence interval 0.57–2.42;P = 0.64). Disease progression occurred within a year in about 50% of patients who were initially untreated. Response rate was similar in both groups, but duration of response was shorter in patients who were treated at disease progression (48 vs 79 months, P = 0.044). Patients actually treated at disease progression (34/70) survived shorter than those who had neither disease progression nor treatment (56 vs > 92 months;P = 0.005). Starting MPH-P just after diagnosis does not improve survival and response rate in stage I MM, with respect to deferring therapy until disease progression. However, patients with stage I MM randomized to have treatment delayed and who actually progressed and were treated had shorter survival than those with stable disease and no treatment. Biologic or other disease features could identify these subgroups of patients. © 2000 Cancer Research Campaign

Management of Newly Diagnosed Symptomatic Multiple Myeloma: Updated Mayo Stratification of Myeloma and Risk-Adapted Therapy (mSMART) Consensus Guidelines 2013

“…25 Although SMM has a much higher risk of progression to symptomatic myeloma (10% per year in the first 5 years), many of these patients can be observed for years before any active therapy is required. 26 Given the lack of any demonstrated benefit for initiating therapy for this early disease stage, 27,28 it is important to distinguish this presymptomatic phase from symptomatic myeloma that requires therapy. However, availability of more active drugs has once again raised the question of early intervention to prevent progression of SMM, and current trials are evaluating this issue.…”

Beta‐2‐microglobulin is an independent predictor of progression in asymptomatic multiple myeloma

“…Prognostic models for predicting asymptomatic MM progression, 8,12,15 including the model described in the current study, would allow the identification of low-risk patients who harbor a very indolent disease similar to MGUS and high-risk patients who might benefit from close follow-up and who may be optimal candidates for experimental therapies aimed at delaying the progression of MM. [35][36][37][38][39][40][41] …”

Multiple myeloma: the disease and its treatment

“…Patients with inactive or SMM should be closely monitored without instituting therapy, as no survival benefit/ advantage has been demonstrated by treating asymptomatic multiple myeloma. 50,51 A long term plan for managing the disease should be formulated before instituting therapy for symptomatic disease (Figure 2). In general MM therapy includes symptomatic supportive care & systemic therapy.…”