VAD with or without subsequent high-dose melphalan followed by autologous stem cell support in AL amyloidosis: Japanese experience and criteria for patient selection
VAD with or without subsequent high-dose melphalan followed by autologous stem cell support in AL amyloidosis: Japanese experience and criteria for patient selection
复制标题
AL 淀粉样变性中 VAD 联合或不联合后续大剂量马法兰，随后进行自体干细胞支持：日本经验和患者选择标准
DOI：
10.1080/13506120412331336907
复制
发表时间：
2004
期刊：
影响因子：
5.5
通讯作者：
S. Ikeda
中科院分区：
文献类型：
--
作者：
T. Gono;M. Matsuda;Y. Shimojima;W. Ishii;J. Koyama;K. Sakashita;K. Koike;Y. Hoshii;S. Ikeda
AL淀粉样变性患者接受VAD（长春新碱、多柔比星和地塞米松）联合或不联合高剂量美法仑治疗，随后根据疾病严重程度的合格标准接受自体PBSCT，并前瞻性研究治疗获益和并发症。本研究入组了31例患者。仅在符合所有合格性标准的患者中进行VAD和随后的高剂量美法仑联合auto-PBSCT。在不适合这种治疗的患者中，在一般状况令人满意的患者中单独进行VAD。11例患者符合合格性标准，其中7例接受VAD治疗，随后接受高剂量美法仑联合auto-PBSCT。7例患者单独接受VAD治疗，其余17例患者接受支持治疗。在接受化疗的14例患者中，9例（其中5/7例接受VAD和大剂量美法仑治疗，4/7例仅接受VAD治疗）显示完全血液学缓解，淀粉样变性相关临床症状明显改善。化疗的严重并发症分别为巨细胞病毒感染和卡氏肺孢子虫肺炎1例和2例。这些化学疗法可有效减少M蛋白，并且还可用于改善淀粉样变性诱导的器官功能障碍。对于不能耐受高剂量美法仑的患者，单独使用VAD是一种有效的治疗选择，尽管可能对心脏和外周神经有有害影响。
Patients with AL amyloidosis were treated with VAD (vincristine, doxorubicin and dexamethasone) with or without high-dose melphalan followed by auto-PBSCT according to eligibility criteria based on disease severity, and prospectively investigated the therapeutic benefits and complications. Thirty-one patients were enrolled in this study. VAD and subsequent high-dose melphalan with auto-PBSCT were performed only in patients who met all of the eligibility criteria. Among patients ineligible for this treatment, VAD alone was performed in those with satisfactory general status. Eleven patients met the eligibility criteria, and of these, 7 were treated with VAD and subsequent high-dose melphalan with auto-PBSCT. Seven patients received VAD alone, and the remaining 17 were treated with the supportive therapy. Among the 14 patients treated with chemotherapy, 9 (5 of the 7 treated with VAD and high-dose melphalan, and 4 of the 7 treated with VAD alone) showed complete hematological response with apparent improvement of amyloidosis-related clinical symptoms. Serious complications of chemotherapy were cytomegalovirus infection and pneumocystis carinii pneumonia seen in 1 and 2 patients, respectively. These chemotherapies may be effective for reduction of M-protein and are also useful in improving of amyloidosis-induced organ dysfunction. In patients who cannot tolerate high-dose melphalan, VAD alone is a potent therapeutic option, although there are possible harmful effects on the heart and peripheral nerve.
影响因子：
3.6
作者：
R. Kyle;M. Gertz
通讯作者：
R. Kyle;M. Gertz