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

被引:43
作者
Landau, H. [1 ,2 ]
Smith, M. [2 ]
Landry, C. [3 ]
Chou, J. F. [2 ]
Devlin, S. M. [2 ]
Hassoun, H. [1 ,2 ]
Bello, C. [2 ]
Giralt, S. [1 ,2 ]
Comenzo, R. L. [4 ]
机构
[1] New York Presbyterian Hosp, Dept Med, Weill Cornell Med Coll, New York, NY USA
[2] Mem Sloan Kettering Canc Ctr, Dept Med, 1275 York Ave, New York, NY 10021 USA
[3] Icahn Sch Med Mt Sinai, Dept Med, New York, NY 10029 USA
[4] Tufts Med Ctr, Dept Med, Boston, MA USA
关键词
STEM-CELL TRANSPLANTATION; HIGH-DOSE MELPHALAN; AL AMYLOIDOSIS; ORGAN INVOLVEMENT; CONSENSUS OPINION; INDUCTION THERAPY; STAGING SYSTEM; PHASE-II; DEXAMETHASONE; BORTEZOMIB;
D O I
10.1038/leu.2016.229
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
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.
引用
收藏
页码:136 / 142
页数:7
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