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儿童血液系统恶性肿瘤患者异基因外周血干细胞移植的长期结局:西班牙儿童血液和骨髓移植工作组(GETMON)及西班牙异基因外周血移植组(GETH)的报告

Long-term outcome of allogeneic PBSC transplantation in pediatric patients with hematological malignancies: a report of the Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON) and the Spanish Group for Allogeneic Peripheral Blood Transplantation (GETH).

作者信息

Diaz M A, Gonzalez-Vicent M, Gonzalez M E, Verdeguer A, Martinez A, Perez-Hurtado M, Badell I, de la Rubia J, Bargay J, de Arriba F, Diez J L, Caballero D, Madero L, Brunet S

机构信息

Department of Pediatrics, Division of Pediatric Hematology Oncology, Hospital Infantil Niño Jesus, Madrid, Spain.

出版信息

Bone Marrow Transplant. 2005 Nov;36(9):781-5. doi: 10.1038/sj.bmt.1705135.

Abstract

We analyzed the clinical outcome in 90 children undergoing allogeneic PBSC transplantation from HLA-identical relative for leukemia. GvHD prophylaxis was CsA+ methotrexate in 50 and CsA+/-steroids in 40. Median CD34+ cells infused were 6 x 10(6)/kg (range, 1.4-32). Median follow-up was 60 months (range, 6-115). CI of transplant-related mortality (TRM) was 18.4+/-4%. On multivariate analysis, high Lansky score (>80) at transplantation was associated with lower TRM (HR, 0.9; P<0.0002). Relapse incidence (RI) was 33.6+/-6%. On multivariate analysis, high Lansky score at transplantation and cGvHD were associated with lower RI (HR, 0.04; P<0.0005 and HR, 0.23; P<0.03, respectively). Disease-free survival (DFS) was 57.8+/-5%. Disease status at transplantation (HR, 0.33; P<0.02), steroid treatment at day +90 (HR, 5.61; P<0.005) and cGvHD (HR, 0.23; P<0.005) had a significant impact on DFS in multivariate analysis. CI of cGvHD was 63.7+/-7%. Patients with cGvHD had better DFS (65+/-5%) because of lower RI (15.7+/-6%) and similar TRM (27.4+/-4%). These data suggest acceptable long-term outcomes after allogeneic PBSC transplantation in children despite the high incidence of cGvHD. These patients had a lower risk of relapse and a better DFS.

摘要

我们分析了90例接受来自 HLA 相同亲属的异基因外周血干细胞移植治疗白血病的儿童的临床结局。50例患者采用环孢素A(CsA)+甲氨蝶呤预防移植物抗宿主病(GvHD),40例采用CsA+/-类固醇。输注的CD34+细胞中位数为6×10⁶/kg(范围1.4 - 32)。中位随访时间为60个月(范围6 - 115)。移植相关死亡率(TRM)的置信区间为18.4±4%。多因素分析显示,移植时高兰斯基评分(>80)与较低的TRM相关(风险比[HR],0.9;P<0.0002)。复发率(RI)为33.6±6%。多因素分析显示,移植时高兰斯基评分和慢性移植物抗宿主病(cGvHD)与较低的RI相关(HR分别为0.04;P<0.0005和HR,0.23;P<0.03)。无病生存率(DFS)为57.8±5%。多因素分析中,移植时的疾病状态(HR,0.33;P<0.02)、+90天时的类固醇治疗(HR,5.61;P<0.005)和cGvHD(HR,0.23;P<0.005)对DFS有显著影响。cGvHD的置信区间为63.7±7%。患有cGvHD的患者DFS较好(65±5%),因为RI较低(15.7±6%)且TRM相似(27.4±4%)。这些数据表明,尽管cGvHD发病率较高,但儿童异基因外周血干细胞移植后的长期结局是可以接受的。这些患者复发风险较低,DFS较好。

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