Efficacy of bortezomib, cyclophosphamide and dexamethasone in treatment-naive patients with high-risk cardiac AL amyloidosis (Mayo Clinic stage III)

被引：104

作者：

Jaccard, Arnaud ^{[1
,2
]}

Comenzo, Raymond L. ^{[3
]}

Hari, Parameswaran ^{[4
]}

Hawkins, Philip N. ^{[5
]}

Roussel, Murielle ^{[6
]}

Morel, Pierre ^{[7
]}

Macro, Margaret ^{[8
]}

Pellegrin, Jean-Luc ^{[9
]}

Lazaro, Estibaliz ^{[9
]}

Mohty, Dania ^{[1
,10
]}

Mercie, Patrick ^{[9
]}

Decaux, Olivier ^{[11
]}

Gillmore, Julian ^{[5
]}

Lavergne, David ^{[1
,2
]}

Bridoux, Frank ^{[12
]}

Wechalekar, Ashutosh D. ^{[5
]}

Venner, Christopher P. ^{[5
,13
]}

机构：

[1] CHU Limoges, Natl Amyloidosis Ctr, Limoges, France

[2] CHU Limoges, Hematol Unit, Limoges, France

[3] Tufts Med Ctr, Boston, MA USA

[4] Med Coll Wisconsin, Milwaukee, WI 53226 USA

[5] UCL, Sch Med, Ctr Amyloidosis & Acute Phase Prot, London WC1E 6BT, England

[6] CHU Toulouse, Hematol Unit, Toulouse, France

[7] CH Lens, Hematol Unit, Lille, France

[8] CHU Caen, Hematol Unit, Caen, France

[9] CHU Bordeaux, Internal Med Units, Bordeaux, France

[10] CHU Limoges, Cardiol Unit, Limoges, France

[11] CHU Rennes, Internal Med Unit, Rennes, France

[12] CHU Poitiers, Nephrol Unit, Poitiers, France

[13] Univ Alberta, Cross Canc Inst, Edmonton, AB, Canada

来源：

HAEMATOLOGICA | 2014年 / 99卷 / 09期

关键词：

LIGHT-CHAIN AMYLOIDOSIS; TWICE-WEEKLY BORTEZOMIB; PRIMARY SYSTEMIC AMYLOIDOSIS; DIAGNOSED MULTIPLE-MYELOMA; HIGH RESPONSE RATES; PHASE; 1/2; LENALIDOMIDE; INVOLVEMENT; MELPHALAN; SURVIVAL;

D O I：

10.3324/haematol.2014.104109

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Bortezomib is an active agent in AL amyloidosis and responses to this drug in combination with cyclophosphamide and dexamethasone are both rapid and deep. Here we present an international, multicenter series of 60 patients with Mayo Clinic stage III cardiac amyloidosis to assess the impact of this regimen in improving outcomes in this poor-risk group. The median follow-up for the entire cohort is 11.8 months. The overall response rate was 68%. In a landmark analysis, examining patients who survived more than 3 months, the overall response rate was 86%. A cardiac response was seen in 32% of patients. The estimated 1-year survival rate for the whole cohort was 57% and 24 patients (40%) died while on therapy. Although unable to save the poorest risk patients, the combination of bortezomib, cyclophosphamide and dexamethasone can achieve a high number of hematologic and cardiac responses, likely improving overall survival and justifying a prospective trial.

引用

页码：1479 / 1485

页数：7

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