Frangoul Haydar, Nemecek Eneida R, Billheimer Dean, Pulsipher Michael A, Khan Shakila, Woolfrey Ann, Manes Becky, Cole Catherine, Walters Mark C, Ayas Mouhab, Ravindranath Yaddanapudi, Levine John E, Grupp Stephan A
Department of Pediatrics, Vanderbilt University, Nashville, TN 37232-2573, USA.
Blood. 2007 Dec 15;110(13):4584-7. doi: 10.1182/blood-2007-07-101071. Epub 2007 Sep 7.
A prospective multicenter trial was conducted to evaluate the safety and feasibility of granulocyte colony-stimulating factor (G-CSF)-primed bone marrow (G-BM) in children receiving allogeneic bone marrow transplantation (BMT). A total of 42 children with a median age of 9.8 years (range, 0.8-17 years) were enrolled. Donors with median age of 9.2 years (range, 1.1-22 years) received 5 microg/kg per day of subcutaneous G-CSF for 5 consecutive days. BM was harvested on the fifth day. No donor experienced complications related to G-CSF administration or marrow har-vest. Median nucleated (NC) and CD34 cells infused was 6.7 x 10(8)/kg (range, 2.4-18.5 x 10(8)/kg) and 7.4 x 10(6)/kg (range, 2-27.6 x 10(6)/kg), respectively. Neutrophil and platelet engraftment was at a median of 19 days (range, 13-28 days) and 20 days (range, 9-44 days), respectively. A total of 13 (32%) patients developed grade 2 graft-versus-host disease (GVHD), and 5 (13%) of 40 evaluable patients developed chronic GVHD (3 limited and 2 extensive). Higher cell dose was not associated with increased risk of acute or chronic GVHD. Overall survival and event-free survival at 2 years were 81% and 69%, respectively. Collection of G-BM from pediatric donors is safe, and can result in high NC and CD34 cell doses that facilitate engraftment after myeloablative BMT without a discernable increase in the risk of GVHD.
开展了一项前瞻性多中心试验，以评估粒细胞集落刺激因子（G-CSF）预处理的骨髓（G-BM）在接受异基因骨髓移植（BMT）儿童中的安全性和可行性。共纳入42名儿童，中位年龄为9.8岁（范围0.8 - 17岁）。中位年龄为9.2岁（范围1.1 - 22岁）的供者连续5天接受每日5μg/kg的皮下G-CSF。在第5天采集骨髓。没有供者出现与G-CSF给药或骨髓采集相关的并发症。输注的中位有核细胞（NC）和CD34细胞分别为6.7×10⁸/kg（范围2.4 - 18.5×10⁸/kg）和7.4×10⁶/kg（范围2 - 27.6×10⁶/kg）。中性粒细胞和血小板植入的中位时间分别为19天（范围13 - 28天）和20天（范围9 - 44天）。共有13名（32%）患者发生2级移植物抗宿主病（GVHD），40名可评估患者中有5名（13%）发生慢性GVHD（3例局限性和2例广泛性）。较高的细胞剂量与急性或慢性GVHD风险增加无关。2年时的总生存率和无事件生存率分别为81%和69%。从儿科供者采集G-BM是安全的，并且可以产生高剂量的NC和CD34细胞，这有助于在清髓性BMT后植入，而不会明显增加GVHD风险。
