Multiple myeloma is an incurable haematological cancer characterized by the accumulation of monoclonal plasma cells inside the bone marrow. Melphalan and Thalidomide are two drugs that have dramatically improved patient survival. However, a significant subpopulation of myeloma patients does not respond to these drugs; in addition, some patients are predisposed to drug-related toxicities, which remains unpredictable. Using samples from the MY.10 randomized clinical trial with an observation arm, the present study found several single nucleotide polymorphism (SNP) genetic biomarkers to be predictive of thalidomide treatment benefit and thalidomide related toxicity (peripheral neuropathy). In addition, the present study also confirmed previous findings where several SNPs were also found to be predictive of melphalan response. Lastly, novel SNPs were found to be associated with myeloma prognosis. These findings contribute to the growing body of evidence that genetic biomarkers can be useful in predicting drug response and prognosis in myeloma. Further studies are needed to confirm our findings.
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