Giardini C, Angelucci E, Lucarelli G, Galimberti M, Polchi P, Baronciani D, Bechelli G
Divisione Ematologica di Muraglia, Centro Trapianto Midollo Osseo, Ospedale di Pesaro, Italy.
Am J Pediatr Hematol Oncol. 1994 Feb;16(1):6-10.
We reviewed the results of transplanting allogeneic marrow from HLA-identical donors in patients with beta-thalassemia. Among the 484 consecutive patients who have received transplants since 1981, survival and disease-free survival rates leveled off at approximately 1 year after transplantation, at 82 and 75%, respectively.
Clinical characteristics of patients before transplant have been studied to determine their impact on survival, disease-free survival, and graft rejection. By multivariate analysis, portal fibrosis, hepatomegaly, and a history of inadequate chelation therapy were identified as risk factors. The patients were then divided into three classes of risk.
The rate of prolonged disease-free survival was 98% and 87% for class 1 and class 2 patients. This rate of disease-free survival is 70% with the use of our last conditioning protocol for class 3 patients. Older patients (17-32 years) have a 79% probability of prolonged disease-free survival.
We conclude that for patients with thalassemia major, transplantation of bone marrow from a human leukocyte antigen-identical donor offers a high probability of disease-free survival, particularly for those patients in early stages of their disease.
我们回顾了β地中海贫血患者接受来自人类白细胞抗原（HLA）相合同胞供者的异基因骨髓移植的结果。自1981年以来接受移植的484例连续患者中，移植后约1年的生存率和无病生存率分别稳定在82%和75%。
研究了移植前患者的临床特征，以确定其对生存、无病生存和移植物排斥的影响。通过多变量分析，门静脉纤维化、肝肿大和螯合治疗不足史被确定为危险因素。然后将患者分为三个风险等级。
1级和2级患者的长期无病生存率分别为98%和87%。使用我们最后一种预处理方案时，3级患者的无病生存率为70%。年龄较大的患者（17至32岁）长期无病生存的概率为79%。
我们得出结论，对于重型地中海贫血患者，来自人类白细胞抗原相合同胞供者的骨髓移植提供了较高的无病生存概率，特别是对于疾病早期的患者。
