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. 2011 Sep 22;118(12):3205-11.
doi: 10.1182/blood-2011-06-297853. Epub 2011 Jul 25.

Approach to the treatment of multiple myeloma: a clash of philosophies

S Vincent Rajkumar  1 , Gösta Gahrton, P Leif Bergsagel
Affiliations

Approach to the treatment of multiple myeloma: a clash of philosophies

S Vincent Rajkumar et al. Blood. .

Abstract

In this Perspective, we summarize some of the most contentious issues surrounding diagnosis and treatment of myeloma. We outline how a fundamental clash of philosophies, cure versus control, may be at the heart of many of the controversies. From the very definition of the disease to risk stratification to the validity of current clinical trial endpoints, we highlight the major areas of debate and provide alternative viewpoints that have implications for trial design and interpretation, as well as clinical practice.

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Figures

Figure 1
Figure 1
Interpretation of randomized trials. (A) Trials in which a new drug shows improvement in PFS over the control arm can be reasonably interpreted as proving the clinical benefit of the given drug because patients in the control group will not have access to such therapy in real life. (B) In contrast, in trials in which the early use of a given drug is being tested, an improvement in PFS may or may not translate into clinical benefit because patients in the control arm do have access to the given drug at the time of relapse in real life.
Figure 2
Figure 2
Interpretation of randomized trials of lenalidomide. Regulatory trials that showed the value of lenalidomide as a new active agent in myeloma used PFS as the primary endpoint. (A) In this instance, improvement in PFS over the control arm can be reasonably interpreted as proving the clinical benefit of the given drug because patients in the control group will not have access to lenalidomide in real life in the absence of regulatory approval. (B) In contrast, in trials in which the early use of lenalidomide as maintenance therapy was tested, an improvement in PFS may or may not translate into clinical benefit because patients in the control arm do have access to lenalidomide at the time of relapse in real life.
Figure 3
Figure 3
Word cloud generated from a recent review of MM illustrating the relative importance given to different concepts. A word cloud gives greater prominence to words that appear more frequently in a given article. We think a greater importance needs to be given to OS relative to PFS and CR.
See this image and copyright information in PMC

References

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    1. Fermand JP, Ravaud P, Chevret S, et al. High-dose therapy and autologous peripheral blood stem cell transplantation in multiple myeloma: up-front or rescue treatment? Results of a multicenter sequential randomized clinical trial. Blood. 1998;92(9):3131–3136. - PubMed
    1. Barlogie B, Kyle RA, Anderson KC, et al. Standard chemotherapy compared with high-dose chemoradiotherapy for multiple myeloma: final results of phase III US Intergroup Trial S9321. J Clin Oncol. 2006;24(6):929–936. - PubMed

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