Howrey R P, Martin P L, Driscoll T, Szabolcs P, Kelly T, Shpall E J, Bearman S I, Slat-Vasquez V, Rubinstein P, Stevens C E, Kurtzberg J
Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Bone Marrow Transplant. 2000 Dec;26(11):1251-4. doi: 10.1038/sj.bmt.1702697.
A 15-year-old female received an unrelated three of six HLA antigen matched umbilical cord blood (UCB) transplant for refractory, relapsed T-cell ALL. Conditioning consisted of TBI, melphalan, and anti-thymocyte globulin (ATG), with cyclosporin A (CsA) and solumedrol for GVHD prophylaxis. She engrafted and a day 34 bone marrow aspirate showed 100% donor cells and no evidence of leukemia. The post-transplant course was complicated by mild grade I acute GVHD involving skin, and limited chronic GVHD of the gut which resolved with the addition of 1 mg/kg/day of steroids to her CsA prophylaxis. One hundred and ninety days after transplantation the patient developed pancytopenia and was subsequently found to have a leukemic relapse. Immunosuppression was discontinued and she was started on G-CSF and erythropoietin. Moderate skin and gut GVHD developed which was treated with both topical and low-dose oral steroids. Over the next few weeks she became transfusion independent and a follow-up bone marrow aspirate showed complete remission. She continued in complete remission for 4 months, at which time localized leukemic relapse was found in a soft tissue breast mass in spite of continued bone marrow remission. While the patient ultimately died of progressive disease, this case demonstrates that mismatched UCB in conjunction with G-CSF is capable of generating a GVL effect that can induce a complete remission.
一名15岁女性因难治性、复发性T细胞急性淋巴细胞白血病接受了6个HLA抗原中3个配型相合的非亲属脐带血移植。预处理方案包括全身照射、美法仑和抗胸腺细胞球蛋白（ATG），并使用环孢素A（CsA）和甲泼尼龙预防移植物抗宿主病（GVHD）。她成功植入，移植后第34天的骨髓穿刺显示100%为供体细胞，且无白血病迹象。移植后的病程出现了并发症，包括轻度I级急性GVHD累及皮肤，以及肠道局限性慢性GVHD，在CsA预防方案中加用1mg/kg/天的类固醇后病情缓解。移植190天后，患者出现全血细胞减少，随后被发现白血病复发。停用免疫抑制剂，开始使用粒细胞集落刺激因子（G-CSF）和促红细胞生成素。出现了中度皮肤和肠道GVHD，采用局部和低剂量口服类固醇治疗。在接下来的几周里，她不再需要输血，后续的骨髓穿刺显示完全缓解。她持续完全缓解4个月，此时尽管骨髓持续缓解，但在乳腺软组织肿块中发现了局部白血病复发。尽管患者最终死于疾病进展，但该病例表明，不相合的脐带血联合G-CSF能够产生移植物抗白血病（GVL）效应，从而诱导完全缓解。
