Effect of hematologic response on outcome of patients undergoing transplantation for primary amyloidosis: Importance of achieving a complete response

Our objective was to determine whether the goal of high-dose therapy should be a partial hematologic response or a complete response. We analyzed 282 consecutive stem cell transplant patients. A partial hematologic response was achieved in 108 patients (38%), and 93 (33%) achieved a complete hematologic response. Survival rates of patients with complete, partial, or no response were significantly different (p<0.001), even after eliminating bias from early death. The degree of response was affected by the intensity of chemotherapy conditioning, septal thickness, and cardiac biomarkers. Hematologic response translates to longer survival.