自体外周血造血干细胞移植治疗POEMS综合征的初步研究

目的 评价自体外周血造血干细胞移植(auto-PBSCT)治疗POEMS综合征的疗效和安全性.方法 9例POEMS综合征患者用大剂量马法兰预处理后进行auto-PBSCT治疗.男6例,女3例,移植时中位年龄44(39～48)岁.起病至移植的中位时间为12(5～60)个月.1例单用粒细胞集落刺激因子(G-CSF)进行外周血干细胞动员,8例采用化疗后注射G-CSF进行动员.2例马法兰剂量为140 mg/m2,7例为200 mg/m2.回输的中位单个核细胞(MNC)数量为3.75(1.05～8.33)×108/kg,中位CD34+细胞为5.37(1.32～10.90)×106/kg.结果 1例为序贯移植,其余患者为单次移植.1例患者移植后第9天死于严重感染和急性肾功能衰竭,其余8例均成功植入.8例患者可评价短期疗效,中位ANC≥0.5×109/L、BPC≥20×109/L的时间分别为10(9～11)d和11.5(9～14)d.2例分别在动员和移植后血清免疫固定电泳检测M蛋白转阴,其他患者移植后均未获得血液学完全缓解.6例伴有下肢水肿和皮肤病变的患者,除1例外,余均有不同程度的好转,1例伴淋巴结肿大患者移植后淋巴结缩小,3例伴视乳头水肿患者2例缓解.7例患者在移植后周围神经症状缓慢改善.结论 大剂量马法兰预处理后行auto-PBSCT治疗POEMS综合征有一定疗效,且安全性高,移植相关死亡率低.

Preliminary study of autologous peripheral blood stem cell transplantation in patients with POEMS syndrome

Objective To evaluate the efficacy and safety of autologous peripheral blood stem cell transplantation(auto-PBSCT) after high dose melphalan in patients with POEMS syndrome. Methods Nine patients including 6 males and 3 females received 10 auto-PBSCT after high dose melphalan in our hospital from June 2005 to October 2009. The median age at transplantation was 44 (39 -48 )years. The median time from onset of disease to transplantation was 12(5 -60)months. Peripheral stem cells were mobilized by GCSF alone in one patient and 8 patients by G-CSF plus chemotherapy. Two patients were conditioned by melphalan 140 mg/m2 and 7 by melphalan 200 mg/m2. The median number of MNC was 3.75 ( 1.05 -8.33) ×108/kg, and that of CD34 + cell was 5.37( 1.32 - 10.90) × 106/kg. Result One patient received tandem auto-PBSCT and others received single one. Stem cell engrafted in all but 1 patient who died of severe infection and acute renal failure on day 9 after transplantation. Eight patients were evaluable for response. The median time to ANC≥0.5 × 109/L and platelet ≥20 × 109/L was 10(9 - 11 ) and 1l.5(9 - 14)days respectively. Two patient reached negative immunofixation electrophoresis (IFE) after stem cell mobilization and transplantation respectively, and the other 6 remained IFE postive after auto-PBSCT. Skin changes and edema of lower extremities were improved in 5 of 6 patients, lymphadenopathy relieved in 1 and papilledema improved in 2 of 3 patients. All but 1 patient achieved gradual neurologic improvement after transplantation.Conclusion PBSCT is an effective and safe therapy for POEMS syndrome patients with low treatment related mortality.