Abstract
BACKGROUND: Thrombocytopenia is less common but shows high risk of early mortality in newly diagnosed multiple myeloma (NDMM) patients. In the era of novel agents-based induction therapy (NAIT), it is unclear whether NAIT can overcome the poor prognosis associated with thrombocytopenia. OBJECTIVES: To evaluate the prognostic implications of thrombocytopenia in NDMM patients. METHODS: We retrospectively analyzed 1363 NDMM patients baseline characteristics, treatment response and survival, further performed regression analysis, constructed a nomogram model to predict progression free survival (PFS), and further internally validated this model. RESULTS: Overall, 211 (15.48%) NDMM patients were harboring thrombocytopenia, with advanced disease stages and worse outcomes. Their PFS (15 months vs 21.5 months, p < 0.001)and overall survival (47 months vs 77 months, p < 0.001) were significantly inferior compared with patients without thrombocytopenia. In NDMM receiving NAIT, the overall response (87.8% vs 92.4%, p = 0.33) but not deep response or survival could be improved between patients with and without thrombocytopenia. Five important variables (thrombocytopenia, R-ISS stage III, NAIT, deep response and autologous stem-cell transplantation) in multivariate Cox analysis were incorporated in the nomogram, which was further validated by internal datasets. The Calibration curve and time-dependent Receiver operating characteristic showed that the model accurately predicted the 12- and 24- months PFS of NDMM patients. CONCLUSIONS: Thrombocytopenia has an indispensable prognostic effect in decreasing responses to induction therapy and survival in NDMM patients. Thrombocytopenia might need to be regarded as an independent prognostic factor in risk stratification of Chinese NDMM patients.