High-dose melphalan followed by autologous hematopoietic cell transplant (AHCT) can improve long-term outcome of patients with primary amyloidosis. Historically, it has been associated with high transplant related mortality (TRM). However, improvement in patient selection has resulted in decreased TRM and improved outcomes. The most common approach, if patients are determined to be eligible, is to proceed directly to ASCT. However, at our institution, patients uniformly receive bortezomib-based induction therapy to obtain best response prior to transplant. We performed a retrospective analysis to evaluate the outcomes of AL amyloidosis patients undergoing ASCT at our institution.
From 6/2007 to 3/2014, 24 patients underwent AHCT, receiving either melphalan 200mg/m2 or 140mg/m2 as a single dose. The median age at transplant was 60 years (range 35 to 72), with 54% male patients. Karnofsky Performance Status was 80% or higher in 87% of patients. Only 13% had known cardiac involvement. All patients had normal troponin levels and the median NT-pro-BNP 253 (range 62 to 7580). The majority of patients had either intermediate (21%) or high (47%) risk co-morbidity index scores. The median time from diagnosis to transplant was 8.3 months, with the majority of patients receiving at least 1 line of prior therapy (92%). For disease response, following induction therapy, 58% were transplanted in first complete response (CR) or partial response (PR), 8% in second PR, and 33% with refractory disease. Patients were mobilized with either G-CSF alone (79%), G-CSF + plerixafor (17%), or G-CSF + cyclophosphamide (4%). The median cell dose was 5.5 x 106 CD34+ cells/kg (range 2.86 to 12.3 x 106) No patient died within 100 days of transplant. The median follow up was 27 months (range 1.5 to 79.8). The median overall survival at 1 year, 3 years, and 5 years was 87%, 77%, and 77%. Relapse free survival at 1 year, 3 years, and 5 years was 78%, 68%, and 68%.
In conclusion, bortezomib-based therapy followed by ASCT is well tolerated and can be performed with low TRM, resulting in durable OS and RFS.