Lacy Martha Q, Dispenzieri Angela, Hayman Suzanne R, Kumar Shaji, Kyle Robert A, Rajkumar S Vincent, Edwards Brooks S, Rodeheffer Richard J, Frantz Robert P, Kushwaha Sudhir S, Clavell Alfredo L, Dearani Joseph A, Sundt Thoralf M, Daly Richard C, McGregor Christopher G A, Gastineau Dennis A, Litzow Mark R, Gertz Morie A
Division of Hematology, Mayo Clinic College of Medicine, Rochester, Minnesota 55905, USA.
J Heart Lung Transplant. 2008 Aug;27(8):823-9. doi: 10.1016/j.healun.2008.05.016. Epub 2008 Jul 3.
Historically, patients with AL amyloidosis and overt congestive heart failure have had an ominous prognosis with median survival of approximately 6 months.
Between 1994 and 2005, 11 patients underwent sequential orthotopic heart transplantation (HT) followed by autologous peripheral blood stem cell transplantation (SCT) for treatment of AL amyloidosis. Patients were accepted for this approach if they had heart-dominant AL with minimal/no other organ impairment and no evidence of multiple myeloma. Conditioning chemotherapy consisted of melphalan 200 mg/m(2) (6 patients) or melphalan 140 mg/m(2) (5 patients).
Two patients died of complications from the SCT (18% transplant-related mortality). Nine patients survived both the HT and the SCT. Three patients subsequently died from progressive amyloidosis at 66, 56.7 and 55 months after SCT. The 1- and 5-year survival for HT was 82% and 65%. The median survival was 76 months from HT and 57 months from SCT.
These data suggest that aggressive treatment of the underlying plasma cell clone after HT may improve long-term outcomes in patients with cardiac amyloid. HT followed by SCT is feasible and offers the possibility of remission for carefully selected patients with cardiac amyloidosis.
从历史上看，患有AL淀粉样变性和明显充血性心力衰竭的患者预后不佳，中位生存期约为6个月。
1994年至2005年期间，11例患者接受了序贯原位心脏移植（HT），随后进行自体外周血干细胞移植（SCT），以治疗AL淀粉样变性。如果患者以心脏为主的AL且极少/无其他器官损害且无多发性骨髓瘤证据，则接受这种治疗方法。预处理化疗包括美法仑200mg/m²（6例患者）或美法仑140mg/m²（5例患者）。
2例患者死于SCT并发症（移植相关死亡率18%）。9例患者在HT和SCT后均存活。3例患者随后在SCT后66、56.7和55个月死于进行性淀粉样变性。HT的1年和5年生存率分别为82%和65%。HT后的中位生存期为76个月，SCT后的中位生存期为57个月。
这些数据表明，HT后积极治疗潜在的浆细胞克隆可能改善心脏淀粉样变性患者的长期预后。HT后进行SCT是可行的，为精心挑选的心脏淀粉样变性患者提供了缓解的可能性。
