Abstract：
BACKGROUND: Autologous hematopoietic cell transplantation (ASCT) improves immunologic homeostasis in autoimmune diseases. ASCT-treated refractory lupus nephritis ( LN ) has been reported. Nevertheless, the long-term outcome of patients with refractory LN after ASCT remains unknown. This study reports the outcomes of 20 refractory lupus patients with 10-year of follow-up after receiving ASCT.
METHODS: Twenty-two patients with LN refractory to immunosuppressive therapy were enrolled. Twenty patients were examined closely and two cases died within 100 days after ASCT. Hematopoietic cell mobilization with cyclophosphamide and granulocyte colony-stimulating factor (G-CSF) was followed by collection of CD34+ positively selected cells. The conditioning regimen consisted of intravenous cyclophosphamide, rabbit antithymocyte globulin, methylprednisolone, and G-CSF. All immunosuppressive therapies were discontinued at the start of mobilization and corticosteroids were tapered rapidly after ASCT.
RESULTS: Data was collected from 22 patients with refractory LN treated by ASCT. 59% were female, duration of lupus before ASCT was 46 (33-71) months, and median duration of follow-up after ASCT was 89.5 (56-108) months. 20 long-term followed up patients had an average follow-up time of 92 months ( 63.25-109.5 ). Eighteen patients achieved complete remission, one patient reached partial remission, one patient without remission started peritoneal dialysis at month 12, and one patient received short-term renal replacement therapy before ASCT started hemodialysis at 84 months after transplantation. Nine patients relapsed 10 times during the follow-up, and three patients received rituximab. Two patients relapsed during pregnancy after complete response and the Apgar scores of infants were 9 and 10, respectively. All nine patients received glucocorticoids and immunosuppressive medication after relapse and responded again. The 10-year overall survival, 10-year disease-free survival rate, and 10-year renal survival were 100%, 35%, and 90%, respectively. The rate of relapse was 45%. Complications included hypocytosis, infection, B-type insulin resistance syndrome, and monoclonal immunoglobulinemia.
CONCLUSIONS: This study suggests ASCT is effective and safety in treating refractory LN and is beneficial to improve their long-term outcomes.
METHODS: Twenty-two patients with LN refractory to immunosuppressive therapy were enrolled. Twenty patients were examined closely and two cases died within 100 days after ASCT. Hematopoietic cell mobilization with cyclophosphamide and granulocyte colony-stimulating factor (G-CSF) was followed by collection of CD34+ positively selected cells. The conditioning regimen consisted of intravenous cyclophosphamide, rabbit antithymocyte globulin, methylprednisolone, and G-CSF. All immunosuppressive therapies were discontinued at the start of mobilization and corticosteroids were tapered rapidly after ASCT.
RESULTS: Data was collected from 22 patients with refractory LN treated by ASCT. 59% were female, duration of lupus before ASCT was 46 (33-71) months, and median duration of follow-up after ASCT was 89.5 (56-108) months. 20 long-term followed up patients had an average follow-up time of 92 months ( 63.25-109.5 ). Eighteen patients achieved complete remission, one patient reached partial remission, one patient without remission started peritoneal dialysis at month 12, and one patient received short-term renal replacement therapy before ASCT started hemodialysis at 84 months after transplantation. Nine patients relapsed 10 times during the follow-up, and three patients received rituximab. Two patients relapsed during pregnancy after complete response and the Apgar scores of infants were 9 and 10, respectively. All nine patients received glucocorticoids and immunosuppressive medication after relapse and responded again. The 10-year overall survival, 10-year disease-free survival rate, and 10-year renal survival were 100%, 35%, and 90%, respectively. The rate of relapse was 45%. Complications included hypocytosis, infection, B-type insulin resistance syndrome, and monoclonal immunoglobulinemia.
CONCLUSIONS: This study suggests ASCT is effective and safety in treating refractory LN and is beneficial to improve their long-term outcomes.
摘要：
背景：自体造血细胞移植（ASCT）可改善自身免疫性疾病的免疫稳态。已经报道了ASCT治疗的难治性狼疮性肾炎(LN)。然而，ASCT术后难治性LN患者的长期结局尚不清楚.本研究报告了20例难治性狼疮患者接受ASCT后10年随访的结果。
方法：纳入对免疫抑制治疗无效的LN患者22例。对20例患者进行了仔细检查，其中2例在ASCT后100天内死亡。用环磷酰胺和粒细胞集落刺激因子（G-CSF）动员造血细胞，然后收集CD34阳性选择的细胞。调理方案包括静脉注射环磷酰胺，兔抗胸腺细胞球蛋白，甲基强的松龙,和G-CSF.动员开始时停止所有免疫抑制疗法，ASCT后皮质类固醇迅速减少。
结果：数据来自接受ASCT治疗的22例难治性LN患者。59%是女性，ASCT前狼疮的持续时间为46（33-71）个月，ASCT后的中位随访时间为89.5(56-108)个月.20例长期随访患者平均随访时间为92个月(63.25～109.5)。18例患者完全缓解,一名患者达到部分缓解,1例没有缓解的患者在移植后第12个月开始腹膜透析,1例患者在ASCT开始血液透析前接受短期肾脏替代疗法.9例患者在随访期间复发10次,三名患者接受了利妥昔单抗治疗。两名患者在完全反应后在怀孕期间复发，婴儿的Apgar评分分别为9和10。所有9名患者在复发后接受了糖皮质激素和免疫抑制药物治疗，并再次缓解。10年总生存率,10年无病生存率，10年肾生存率为100%,35%,90%,分别。复发率为45％。并发症包括细胞减少，感染,B型胰岛素抵抗综合征,和单克隆免疫球蛋白血症。
结论：本研究提示ASCT治疗难治性LN有效且安全，有利于改善其长期预后。
方法：纳入对免疫抑制治疗无效的LN患者22例。对20例患者进行了仔细检查，其中2例在ASCT后100天内死亡。用环磷酰胺和粒细胞集落刺激因子（G-CSF）动员造血细胞，然后收集CD34阳性选择的细胞。调理方案包括静脉注射环磷酰胺，兔抗胸腺细胞球蛋白，甲基强的松龙,和G-CSF.动员开始时停止所有免疫抑制疗法，ASCT后皮质类固醇迅速减少。
结果：数据来自接受ASCT治疗的22例难治性LN患者。59%是女性，ASCT前狼疮的持续时间为46（33-71）个月，ASCT后的中位随访时间为89.5(56-108)个月.20例长期随访患者平均随访时间为92个月(63.25～109.5)。18例患者完全缓解,一名患者达到部分缓解,1例没有缓解的患者在移植后第12个月开始腹膜透析,1例患者在ASCT开始血液透析前接受短期肾脏替代疗法.9例患者在随访期间复发10次,三名患者接受了利妥昔单抗治疗。两名患者在完全反应后在怀孕期间复发，婴儿的Apgar评分分别为9和10。所有9名患者在复发后接受了糖皮质激素和免疫抑制药物治疗，并再次缓解。10年总生存率,10年无病生存率，10年肾生存率为100%,35%,90%,分别。复发率为45％。并发症包括细胞减少，感染,B型胰岛素抵抗综合征,和单克隆免疫球蛋白血症。
结论：本研究提示ASCT治疗难治性LN有效且安全，有利于改善其长期预后。
