Department of Cardiothoracic Science, Azienda Sanitaria Universitaria Integrata di Udine.
Haematology Department, Azienda sanitaria Universitaria Integrata, DAME.
J Cardiovasc Med (Hagerstown). 2021 Apr 1;22(4):261-267. doi: 10.2459/JCM.0000000000001094.
The prognosis for patients affected by light-chain cardiac amyloidosis and acquired transthyretin-related (TTR) amyloidosis is poor. Heart transplantation (HTx) could improve prognosis also enabling autologous stem cell transplantation (ASCT) in the first group.
A total of 36 patients affected by systemic amyloidosis have been referred to our centre from 2009 to 2019. Of these, nine had cardiac involvement: seven by light-chain amyloidosis and two by acquired TTR amyloidosis. None died while waiting for HTx. A specific internal protocol useful to select candidates and to monitor the organ involvement after HTx was developed. Median age at diagnosis was 54 years and 66% were male. The most common short-term complication after HTx was renal failure (44%), followed by acute cardiac rejection more than 2R (22%). ASCT was performed in six out of seven light-chain cardiac amyloidosis patients, with a median time of 6 months after HTx. Two patients affected by light-chain cardiac amyloidosis died due to amyloidosis relapse: one before undergoing ASCT. After a median follow-up of 31 (7-124) months, 1- and 5-year survival was 88 and 66% in the cardiac light-chain amyloidosis group. Conversely, 1- and 5-year survival was 100% in the acquired TTR amyloidosis group.
HTx may represent a valuable option in carefully selected patients. ASCT after HTx is an effective treatment that could decrease amyloidosis relapse in light-chain cardiac amyloidosis patients. A multidisciplinary approach is mandatory to select the best candidates and to obtain the most effective results with a specific surveillance follow-up protocol.
轻链心脏淀粉样变性和获得性转甲状腺素蛋白相关（TTR）淀粉样变性患者的预后较差。心脏移植（HTx）可以改善预后，也使第一组患者能够进行自体干细胞移植（ASCT）。
2009 年至 2019 年期间，共有 36 例系统性淀粉样变性患者被转诊至我们中心。其中 9 例有心脏受累：7 例为轻链淀粉样变性，2 例为获得性 TTR 淀粉样变性。在等待 HTx 期间，没有患者死亡。我们制定了一个特定的内部方案，用于选择候选者并监测 HTx 后的器官受累情况。诊断时的中位年龄为 54 岁，66%为男性。HTx 后最常见的短期并发症是肾衰竭（44%），其次是 2R 以上的急性心脏排斥反应（22%）。7 例轻链心脏淀粉样变性患者中有 6 例接受了 ASCT，HTx 后中位时间为 6 个月。2 例轻链心脏淀粉样变性患者因淀粉样变性复发而死亡：1 例在接受 ASCT 之前。中位随访 31（7-124）个月后，轻链心脏淀粉样变性组的 1 年和 5 年生存率分别为 88%和 66%。相反，获得性 TTR 淀粉样变性组的 1 年和 5 年生存率均为 100%。
在精心挑选的患者中，HTx 可能是一种有价值的选择。HTx 后 ASCT 是一种有效的治疗方法，可以降低轻链心脏淀粉样变性患者的淀粉样变性复发率。需要多学科方法来选择最佳候选者，并通过特定的监测随访方案获得最有效的结果。
