The primary objective of this observational study is to identify the molecular profiles and clinical characteristics that define subsets of myeloma patients during the course of the disease.
Understanding the molecular basis of cancer is a critical step toward devising the most effective treatment of the patient as an individual. The promise of molecular targeted therapeutics and personalized cancer care has been demonstrated in breast and lung cancer and chronic myeloid leukemia. However, similar examples of success in multiple myeloma have not been achieved despite extensive basic research as well as clinical advances. What is well understood is that myeloma is a heterogeneous disease with great genetic and epigenetic complexity.22, 23 Therefore, there remains a critical need to understand myeloma patient biology in the context of current patient care.24 The objective of this longitudinal study is to identify patient subgroups and phenotypes defined by molecular profiling and clinical features. These profiles will enable a better understanding of mechanisms of disease, drug response and patient relapse. Ultimately the study is intended to drive successful drug development and patient care in multiple myeloma.
Study Type
OBSERVATIONAL
Enrollment
1,154
Molecular profiles and clinical characteristics that define subsets of myeloma patients at initial diagnosis and at relapse of disease.
Standard clinical and laboratory assessments. Genomic tests (DNA and RNA sequencing, etc.) on bone marrow aspirates obtained at baseline, suspected complete response, and relapse/progression.
Time frame: Baseline to 8 years.
Response rates
IMWG criteria: stringent complete response, complete response, very good partial response, partial response, no response.
Time frame: Up to one year after baseline.
Survival rates
Progression-free survival and overall survival
Time frame: Five to eight years after baseline
Bone disease assessed radiographically
Time frame: Baseline and during five to eight years of follow-up
Health-related quality of life
EORTC QLQ-C30 and QLQ-MY20
Time frame: Baseline and during five to eight years of follow-up
Resource utilization
Hospitalizations and ER visits
Time frame: Baseline and during five to eight years of follow-up
Severe adverse events
Severe/CTCAE grade 3-4 adverse events (checklist)
Time frame: Five to eight years
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