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9例心脏淀粉样变性自体干细胞移植的临床分析

[Clinical analysis of autologous stem cell transplantation for nine cases of cardiac amyloidosis].

作者信息

Ogura Mizuki, Sekine Rieko, Nishiyama Sayuri, Abe Yu, Iizuka Hiromitsu, Kusaka Sayaka, Nakagawa Yasuhiro, Suzuki Kenshi

机构信息

Department of Hematology, Japanese Red Cross Medical Center.

出版信息

Rinsho Ketsueki. 2012 Jul;53(7):710-5.

Abstract

Long-term survival remains poor for patients with cardiac amyloidosis. High-dose melphalan (MEL) with stem cell transplantation (HDM/SCT) is an effective treatment for AL amyloidosis, but patients with cardiac involvement are ineligible because of high therapy-related mortality. Here we report detailed HDM/SCT outcomes of 9 patients with cardiac failure. Their median age was 56 years (range, 45∼66). After a median follow-up of 15 months (range 9∼32), three died of multiorgan failure within the early phase after HDM/SCT, and the other six including poor risk patients are alive at present. Their symptoms of cardiac decompensation have improved. Decreases in interventricular septum thickness were confirmed in 4 patients 6∼12 months after HDM/SCT by echocardiography. One-year overall survival rate was 67%, longer than previously reported rates. HDM/SCT may lead to improvements in quality of life and extended survival in cardiac amyloidosis patients. Meanwhile, the median dosage of MEL in our procedure was 103 mg/m(2) (range 68∼180), less than the recommended dose, and patients were maintained on miscellaneous therapies. Further studies are required to clarify an effective MEL dose and to refine selection criteria for patients undergoing HDM/SCT.

摘要

心脏淀粉样变性患者的长期生存率仍然很低。高剂量美法仑(MEL)联合干细胞移植(HDM/SCT)是治疗AL淀粉样变性的有效方法,但心脏受累的患者因治疗相关死亡率高而不符合条件。在此,我们报告9例心力衰竭患者的HDM/SCT详细结果。他们的中位年龄为56岁(范围45至66岁)。中位随访15个月(范围9至32个月)后,3例在HDM/SCT后的早期死于多器官衰竭,其他6例包括高危患者目前仍存活。他们的心脏失代偿症状有所改善。4例患者在HDM/SCT后6至12个月通过超声心动图证实室间隔厚度减小。1年总生存率为67%,高于先前报道的比率。HDM/SCT可能会改善心脏淀粉样变性患者的生活质量并延长生存期。同时,我们手术中MEL的中位剂量为103 mg/m²(范围68至180),低于推荐剂量,并且患者接受了多种治疗。需要进一步研究以明确有效的MEL剂量并完善HDM/SCT患者的选择标准。

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