采用口服美法仑、地塞米松和沙利度胺治疗晚期心脏 AL 淀粉样变性患者。

Treatment of patients with advanced cardiac AL amyloidosis with oral melphalan, dexamethasone, and thalidomide.

作者信息

Palladini Giovanni, Russo Paola, Lavatelli Francesca, Nuvolone Mario, Albertini Riccardo, Bosoni Tiziana, Perfetti Vittorio, Obici Laura, Perlini Stefano, Moratti Remigio, Merlini Giampaolo

机构信息

Biotechnology Research Laboratories, Center for Amyloidosis, Department of Biochemistry, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico San Matteo and University of Pavia, Piazzale Golgi, 19, 27100 Pavia, Italy.

出版信息

Ann Hematol. 2009 Apr;88(4):347-50. doi: 10.1007/s00277-008-0600-y. Epub 2008 Sep 9.

DOI:10.1007/s00277-008-0600-y

PMID:18779964

Abstract

Patients with primary (AL) amyloidosis and heart failure have a very poor prognosis and cannot tolerate aggressive therapy, such as autologous stem cell transplantation and high-dose dexamethasone-based regimens. We prospectively treated 22 patients with advanced cardiac amyloidosis combining oral melphalan, thalidomide, and reduced intensity dexamethasone (MTD). Six patients died due to cardiac amyloidosis before completing cycle 3. Early death was associated with reduced ejection fraction. Eight patients achieved a hematological response and four achieved a durable improvement of cardiac dysfunction. Treatment with MTD is feasible in patients with advanced cardiac AL amyloidosis and effective in subjects with preserved systolic function.

摘要

原发性（AL）淀粉样变性合并心力衰竭的患者预后很差，无法耐受积极的治疗，如自体干细胞移植和基于高剂量地塞米松的治疗方案。我们前瞻性地对22例晚期心脏淀粉样变性患者联合使用口服美法仑、沙利度胺和低强度地塞米松（MTD）进行治疗。6例患者在完成第3周期治疗前因心脏淀粉样变性死亡。早期死亡与射血分数降低有关。8例患者获得血液学缓解，4例患者心脏功能障碍得到持久改善。MTD治疗对晚期心脏AL淀粉样变性患者是可行的，对收缩功能保留的患者有效。