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

Autor: Landau H; 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., Smith M; Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Landry C; Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, USA., Chou JF; Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Devlin SM; Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Hassoun H; 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., Bello C; Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Giralt S; 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., Comenzo RL; Department of Medicine, Tufts Medical Center, Boston, MA, USA.
Jazyk: angličtina
Zdroj: Leukemia [Leukemia] 2017 Jan; Vol. 31 (1), pp. 136-142. Date of Electronic Publication: 2016 Aug 18.
DOI: 10.1038/leu.2016.229
Abstrakt: 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 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.
Competing Interests: MS, CL, JFC, SMD, CB and SG have no conflicts of interest to disclose. HL has received honoraria from Spectrum, Janssen and Prothena, and research funding from Onyx, Celgene, Millennium Pharmaceuticals, The Amyloidosis Research Fund, the Werner and Elaine Dannheiser Fund for Research on the Biology of Aging of the Lymphoma Foundation and Takeda. HH has received honoraria and research grant support from Celgene, Novartis and Takeda. RLC has received honoraria from Takeda-Millennium and Janssen and research support from Janssen, Takeda-Millennium, Prothena and Karyopharm.
Databáze: MEDLINE
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