Enright H, Davies S, McGlave P
University of Minnesota Bone Marrow Transplant Program, Minneapolis, USA.
Clin Transpl. 1994:283-93.
Marrow transplant is potentially curative therapy for CML. Allogeneic sibling-donor marrow transplant may be the therapy of choice for younger patients early in the course of disease. Early transplant is an important influence on disease-free survival and relapse after related-donor transplant therapy, although additional patient characteristics influencing outcome can be identified and may have cumulative adverse effects. The 5-year disease-free survival of patients transplanted within one year of diagnosis and without signs of advanced disease is greater than 65%. Significant problems remain, however, including early mortality (primarily from infection, pneumonia, and pneumonitis) and relapse of CML following transplant, including late relapse occurring more than 5 years posttransplant. For patients without a matched, related donor, unrelated-donor marrow transplant may be a treatment option and can result in successful outcome for patients with CML. Relapse following unrelated-donor marrow transplant is rare. However, the use of an unrelated donor is associated with significant toxicity, including early mortality, engraftment failure, and ongoing morbidity and mortality associated with acute and chronic graft-versus-host disease. For patients who lack an available matched-related or -unrelated donor, autologous marrow transplant has been developed as an alternative approach to therapy. Long-term survival following autologous marrow transplant is possible and may even approach the survival for allogeneic related-donor recipients, although cure of disease is not achieved.
骨髓移植是慢性粒细胞白血病(CML)的潜在治愈性疗法。对于病程早期的年轻患者,异基因同胞供体骨髓移植可能是首选治疗方法。早期移植对相关供体移植治疗后的无病生存率和复发有重要影响,尽管可以确定影响预后的其他患者特征,且这些特征可能具有累积不良影响。诊断后一年内且无晚期疾病迹象的患者,其5年无病生存率大于65%。然而,仍存在重大问题,包括早期死亡率(主要源于感染、肺炎和肺炎)以及移植后CML复发,包括移植后5年以上发生的晚期复发。对于没有匹配的相关供体的患者,无关供体骨髓移植可能是一种治疗选择,并且可以使CML患者获得成功的治疗结果。无关供体骨髓移植后的复发很少见。然而,使用无关供体与显著的毒性相关,包括早期死亡率、植入失败以及与急慢性移植物抗宿主病相关的持续发病率和死亡率。对于缺乏可用的匹配相关或无关供体的患者,自体骨髓移植已被开发为一种替代治疗方法。自体骨髓移植后有可能实现长期生存,甚至可能接近异基因相关供体接受者的生存率,尽管无法治愈疾病。