title: Long-term event-free and overall survival after risk-adapted melphalan and SCT for systemic light chain amyloidosis.
abstract: Stem cell transplantation (SCT), an effective therapy for amyloid light chain (AL) amyloidosis patients, is associated with low treatment-related mortality (TRM) with appropriate patient selection and risk-adapted dosing of melphalan (RA-SCT). Consolidation after SCT increases hematologic complete response (CR) rates and may improve overall survival (OS) for patients with <CR. We retrospectively analyzed outcomes for 143 patients who underwent RA-SCT with or without consolidation. Melphalan was administered at 100 (14%), 140 (52%) and 200 (34%) mg/m2. The TRM rate at 100 days was 5%. RA-SCT resulted in CR in 24% (3 months) and 48% (12 months) of patients. The CR rate was particularly high (62%) in patients offered bortezomib consolidation. With a median follow-up among survivors of 7.7 years, median event-free survival (EFS) with RA-SCT was 4.04 years (95% confidence interval (CI): 3.41-5.01 years); median OS was 10.4 years (95% CI: 7.3-not achieved). Patients with CR at 12 months after SCT had significantly longer EFS (P=0.01) and OS (P=0.04). In a multivariate analysis, melphalan dose had no impact on EFS (P=0.26) or OS (P=0.11). For selected patients, RA-SCT was safe and was associated with extended long-term survival. With the availability of novel agents for consolidation, RA-SCT remains a very effective and important backbone treatment for AL amyloidosis.
authors: Department of Medicine, New York-Presbyterian Hospital/Weill Cornell Medical College, New York, NY, USA.|Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors: H
authors: H
authors: Landau
authors: Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors: M
authors: M
authors: Smith
authors: Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
authors: C
authors: C
authors: Landry
authors: Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors: J F
authors: JF
authors: Chou
authors: Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors: S M
authors: SM
authors: Devlin
authors: Department of Medicine, New York-Presbyterian Hospital/Weill Cornell Medical College, New York, NY, USA.|Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors: H
authors: H
authors: Hassoun
authors: Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors: C
authors: C
authors: Bello
authors: Department of Medicine, New York-Presbyterian Hospital/Weill Cornell Medical College, New York, NY, USA.|Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors: S
authors: S
authors: Giralt
authors: Department of Medicine, Tufts Medical Center, Boston, MA, USA.
authors: R L
authors: RL
authors: Comenzo
mesh_terms: D000686:Amyloidosis / Q000188:drug therapy* / Q000401:mortality* / Q000628:therapy; D000069286:Bortezomib / Q000627:therapeutic use; D060830:Consolidation Chemotherapy / Q000379:methods; D018572:Disease-Free Survival; D005500:Follow-Up Studies; D006801:Humans; D007147:Immunoglobulin Light Chains; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D012074:Remission Induction; D012189:Retrospective Studies; D020379:Risk Adjustment; D033581:Stem Cell Transplantation; D015996:Survival Rate
chemical_list: D007147:Immunoglobulin Light Chains; D000069286:Bortezomib; D008558:Melphalan