Outcomes From Autologous Hematopoietic Cell Transplantation Versus Chemotherapy Alone for the Management of Light Chain (AL) Amyloidosis

• Published in: Biology of blood and marrow transplantation 2017-09, Vol.23 (9), p.1473-1477
• Main Authors: Oke, Oluchi, MD, Sethi, Tarsheen, MD, Goodman, Stacey, MD, Phillips, Sharon, MSPH, Decker, Ilka, MD, Rubinstein, Samuel, MD, Concepcion, Beatrice, MD, Horst, Sarah, MD, Jagasia, Madan, MD, Kassim, Adetola, MD, Harrell, Shelton L, NP, Langone, Anthony, MD, Lenihan, Daniel, MD, Rawling, Kyle T, Slosky, David, MD, Cornell, R. Frank, MD, MS
• Format: Article
• Language: English
• Subjects: Adult; Aged; Antineoplastic Agents - therapeutic use; Bortezomib; Bortezomib - therapeutic use; Female; Hematology, Oncology and Palliative Medicine; Hematopoietic Stem Cell Transplantation - methods; Humans; Immunoglobulin Light-chain Amyloidosis - immunology; Immunoglobulin Light-chain Amyloidosis - mortality; Immunoglobulin Light-chain Amyloidosis - pathology; Immunoglobulin Light-chain Amyloidosis - therapy; Induction Chemotherapy - methods; Light chain amyloidosis; Male; Middle Aged; Multivariate Analysis; Natriuretic Peptide, Brain - blood; Organ response, chemotherapy; Proportional Hazards Models; Proteinuria - immunology; Proteinuria - mortality; Proteinuria - pathology; Proteinuria - therapy; Retrospective Studies; Stroke Volume - physiology; Transplantation; Transplantation, Autologous; Treatment Outcome
• ISSN: 1083-8791; 1523-6536
• DOI: 10.1016/j.bbmt.2017.05.020

Abstract Light chain amyloidosis (AL) results in tissue deposition of misfolded proteins causing organ dysfunction. In an era of modern therapies such as bortezomib, reassessment of the benefit of autologous hematopoietic cell transplantation (AHCT) should be considered. This study compares the difference in outcomes between patients with AL receiving chemotherapy alone (CT) vs. AHCT. Seventy-four patients with AL were retrospectively analyzed. Two cohorts of patients were studied, those receiving CT (n=31) or AHCT (n=43). Of those receiving AHCT, 29 received induction chemotherapy prior to AHCT while 14 patients proceeded straight to AHCT without induction therapy. Patients in the AHCT cohort were younger, had higher ejection fractions, lower brain natriuretic peptide levels and higher proteinuria compared with the CT cohort. Among patients receiving chemotherapy, the majority (87%) received bortezomib-based treatment. Transplant related mortality (TRM) was 7%. Patients receiving AHCT were more likely to achieve complete or very good partial response (p=0.048). The median progression free survival (PFS) and overall survival (OS) were superior in the AHCT cohort compared with CT (not reached vs. 9 months (mo), p