Improved Treatment Related Mortality in Patients with Primary Systemic Amyloidosis (AL Amyloidosis) undergoing Autologous Hematopoietic Stem Cell Transplant (aHSCT).

Abstract

To date, there is no standard of care for patients with newly diagnosed Primary (AL) amyloidosis. Autologous hematopoietic stem cell transplant (aHSCT) is a reasonable option, but has been limited in its use due to increase in treatment-related mortality (TRM). We retrospectively analyzed the outcomes of 42 newly diagnosed consecutive AL amyloidosis patients transplanted at our center. The median age at aHSCT was 57.5 (range 26-71). Twenty one (50%) had involvement of at least two organs and 40 (97%) patients had cardiac stage I or II. Patients received high dose Melphalan 140(n=4) or 200(n=38) mg/m². Median times to neutrophil and platelet engraftments were 12 and 18 days, respectively. Three months hematologic response were complete response in 21 patients (50%), very good partial response in 4 (10%), partial response in 5 (12%) and Minimal/Stable disease in 6(15%). The respective 1, 3, and 5 year progression-free survival were 79%, 67% and 57%, and overall survival from Transplant 81%, 73% and 66%. Day 100 and 1 year TRM were 4.8% and 7.1% respectively. Our results show that aHSCT is a safe and reasonable option for patients with AL amyloidosis. Day 100 and 1 year TRM compares favorably to multiple myeloma patients undergoing aHSCT.