Long-term event-free and overall survival after risk-adapted melphalan and SCT for systemic light chain amyloidosis Journal Article

   

Authors:	Landau, H.; Smith, M.; Landry, C.; Chou, J. F.; Devlin, S. M.; Hassoun, H.; Bello, C.; Giralt, S.; Comenzo, R. L.
Article 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/m 2. 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. © 2017 Macmillan Publishers Limited, part of Springer Nature.
Journal Title:	Leukemia
Volume:	31
Issue:	1
ISSN:	0887-6924
Publisher:	Nature Publishing Group
Date Published:	2017-01-01
Start Page:	136
End Page:	142
Language:	English
DOI:	10.1038/leu.2016.229
PROVIDER:	scopus
PMCID:	PMC5220129
PUBMED:	27560108
DOI/URL:	https://www.scopus.com/inward/record.uri?eid=2-s2.0-84989229100&doi=10.1038%2fleu.2016.229&partnerID=40&md5=60277bd4f2e6222db8a9d0225c24961a
Notes:	Article -- Export Date: 2 February 2017 -- Source: Scopus

https://synapse.mskcc.org/synapse/works/has_related_data_chk/104297