Lucarelli G
Divisione Ematologica, Ospedale di Pesaro, Italy.
J Intern Med Suppl. 1997;740:49-52.
For all patients with a histocompatibility antigen (HLA) identical donor we are actually using two protocols to whom the patients is assigned. This is based on which class the patients belongs to at the time of bone-marrow transplant and is independent from the patient's age. For 116 patients in Class 1 and for 271 patients in Class 2 prepared for the transplant with busulfan 14 mg/kg, cyclophosphamide 200 mg/kg and cyclosporin alone, the probabilities of survival and of event-free survival are 95% and 90% for Class 1 and 85% and 81% for Class 2. For 125 Class 3 patients prepared for the transplant with busulfan 14 mg/kg, cyclophosphamide reduced to 120-160 mg/kg, cyclosporin and 'short' methotrexate, the probabilities of survival and of event-free survival are 78% and 54%. For 108 adult patients aged between 17 and 35 years, who underwent the transplant after preparation with the same protocol used for the Class 2 or Class 3 patients, the probabilities of survival are 67% and of event-free survival are 63%. Bone marrow transplantation remains the only form of radical treatment of thalassaemia in those patients with an HLA identical donor.
对于所有拥有组织相容性抗原（HLA）匹配供体的患者，我们实际上采用了两种方案并将患者分配至相应方案。这是基于患者在骨髓移植时所属的类别，且与患者年龄无关。对于116名1类患者和271名2类患者，使用白消安14mg/kg、环磷酰胺200mg/kg及单独使用环孢素进行移植预处理，1类患者的生存概率和无事件生存概率分别为95%和90%，2类患者分别为85%和81%。对于125名3类患者，使用白消安14mg/kg、环磷酰胺减至120 - 160mg/kg、环孢素及“短疗程”甲氨蝶呤进行移植预处理，生存概率和无事件生存概率分别为78%和54%。对于108名年龄在17至35岁之间的成年患者，采用与2类或3类患者相同的方案进行预处理后接受移植，生存概率为67%，无事件生存概率为63%。对于那些拥有HLA匹配供体的患者，骨髓移植仍然是地中海贫血唯一的根治方法。
