非清髓性造血细胞移植。通过移植物抗肿瘤效应替代大剂量细胞毒性疗法。
Nonmyeloablative hematopoietic cell transplantation. Replacing high-dose cytotoxic therapy by the graft-versus-tumor effect.
作者信息
Feinstein L, Sandmaier B, Maloney D, McSweeney P A, Maris M, Flowers C, Radich J, Little M T, Nash R A, Chauncey T, Woolfrey A, Georges G, Kiem H P, Zaucha J M, Blume K G, Shizuru J, Niederwieser D, Storb R
机构信息
Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N., D1-100, P.O. Box 19024, Seattle, Washington 98109-1024, USA.
出版信息
Ann N Y Acad Sci. 2001 Jun;938:328-37; discussion 337-9.
Conventional allografting produces considerable regimen-related toxicities that generally limit this treatment to patients younger than 55 years and in otherwise good medical condition. T cell-mediated graft-versus-tumor (GVT) effects are known to play an important role in the elimination of malignant disease after allotransplants. A minimally myelosuppressive regimen that relies on immunosuppression for allogeneic engraftment was developed to reduce toxicities while optimizing GVT effects. Pre-transplant total-body irradiation (200 cGy) followed by post-transplant immunosuppression with cyclosporine (CSP) and mycophenolate mofetil (MMF) permitted human leukocyte antigen (HLA)-matched sibling donor hematopoietic cell engraftment in 82% of patients (n = 55) without prior high-dose therapy. The addition of fludarabine (90 mg/m2) facilitated engraftment in all 28 subsequent patients. Overall, fatal progression of underlying disease occurred in 20% of patients after transplant. Non-relapse mortality occurred in 11% of patients. Toxicities were low. Grade 2-4 acute graft-versus-host disease (GVHD) associated with primary engraftment developed in 47% of patients, and was readily controlled in all but two patients. Donor lymphocyte infusions (DLI) were not very effective at converting a low degree of mixed donor/host chimerism to full donor chimerism; however, the addition of fludarabine reduced the need for DLI. With a median follow-up of 244 days, 68% of patients were alive, with 42% of patients in complete remission, including molecular remissions. Remissions occurred gradually over periods of weeks to a year. If long-term efficacy is demonstrated, such a strategy would expand treatment options for patients who would otherwise be excluded from conventional allografting.
传统的同种异体移植会产生相当多与治疗方案相关的毒性,这通常将这种治疗方法局限于55岁以下且身体状况良好的患者。已知T细胞介导的移植物抗肿瘤(GVT)效应在同种异体移植后消除恶性疾病中起重要作用。为了减少毒性同时优化GVT效应,开发了一种依赖免疫抑制实现异基因植入的最小骨髓抑制方案。移植前全身照射(200 cGy),随后用环孢素(CSP)和霉酚酸酯(MMF)进行移植后免疫抑制,使得82%(n = 55)未接受过先前高剂量治疗的患者成功植入了人类白细胞抗原(HLA)匹配的同胞供体造血细胞。随后的28例患者中,添加氟达拉滨(90 mg/m2)促进了植入。总体而言,20%的患者在移植后出现潜在疾病的致命进展。11%的患者发生非复发死亡率。毒性较低。47%的患者出现与初次植入相关的2 - 4级急性移植物抗宿主病(GVHD),除两名患者外,其他患者均能得到有效控制。供体淋巴细胞输注(DLI)在将低程度的混合供体/宿主嵌合体转化为完全供体嵌合体方面效果不佳;然而,添加氟达拉滨减少了对DLI的需求。中位随访244天,68%的患者存活,42%的患者完全缓解,包括分子缓解。缓解在数周至一年的时间内逐渐出现。如果能证明其长期疗效,这样的策略将为那些原本被排除在传统同种异体移植之外的患者扩大治疗选择。