Massachusetts General Hospital, Boston, MA, USA.
Transplantation. 2010 Oct 27;90(8):905-11. doi: 10.1097/TP.0b013e3181f10edb.
Patients with light chain (AL) amyloidosis who present with severe heart failure due to cardiac involvement rarely survive more than 6 months. Survival after cardiac transplantation is markedly reduced due to the progression of amyloidosis. Autologous stem-cell transplantation (ASCT) has become a common therapy for AL amyloidosis, but there is an exceedingly high treatment-related mortality in patients with heart failure.
We developed a treatment strategy of cardiac transplant followed by ASCT. Twenty-six patients were evaluated, and of 18 eligible patients, nine patients underwent cardiac transplantation. Eight of these patients subsequently received an ASCT.
Six of seven evaluable patients achieved a complete hematologic remission, and one achieved a partial remission. At a median follow-up of 56 months from cardiac transplant, five of seven patients are alive without recurrent amyloidosis. Their survival is comparable with 17,389 patients who received heart transplants for nonamyloid heart disease: 64% in nonamyloid vs. 60% in amyloid patients at 7 years (P=0.83). Seven of eight transplanted patients have had no evidence of amyloid in their cardiac allograft.
This demonstrates that cardiac transplantation followed by ASCT is feasible in selected patients with AL amyloidosis and heart failure, and that such a strategy may lead to improved overall survival.
由于心脏受累而出现严重心力衰竭的轻链（AL）淀粉样变性患者的生存期通常不超过 6 个月。由于淀粉样变性的进展，心脏移植后的存活率明显降低。自体干细胞移植（ASCT）已成为 AL 淀粉样变性的常见治疗方法，但心力衰竭患者的治疗相关死亡率极高。
我们制定了心脏移植后再进行 ASCT 的治疗策略。评估了 26 名患者，其中 18 名符合条件的患者中有 9 名接受了心脏移植。其中 8 名患者随后接受了 ASCT。
7 名可评估患者中有 6 名达到完全血液学缓解，1 名达到部分缓解。在心脏移植后中位随访 56 个月时，7 名患者中有 5 名无复发性淀粉样变性存活。他们的存活率与 17389 名因非淀粉样心脏病接受心脏移植的患者相当：7 年时非淀粉样组为 64%，淀粉样组为 60%（P=0.83）。接受移植的 8 名患者中有 7 名在心脏移植物中没有发现淀粉样蛋白。
这表明心脏移植后再进行 ASCT 在有选择的 AL 淀粉样变性和心力衰竭患者中是可行的，这种策略可能会提高总体生存率。
