Gaziev Javid, Sodani Pietro, Polchi Paola, Andreani Marco, Lucarelli Guido
Mediterranean Institute of Hematology, International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Policlinico Tor Vergata, Rome, Italy.
Ann N Y Acad Sci. 2005;1054:196-205. doi: 10.1196/annals.1345.024.
Current regular blood transfusion programs and chelation treatment have considerably improved survival of patients with thalassemia, which resulted in a larger proportion of adult patients. However, disease- and treatment-related complications in these patients progress over time, causing severe morbidity and shortened life expectancy. Stem cell transplantation still remains the only cure currently available for patients with thalassemia. This study updates transplant outcomes in 107 adult patients with median age of 22 years (range, 17-35 years) who received bone marrow transplantation (BMT) from human leukocyte antigen (HLA)-identical related donors between 1988 and 1996 (group A) and describes the results of BMT in 15 adult patients with median age of 21 years (range, 17-31 years) who were treated with a new treatment protocol (Protocol 26) between 1997 and 2003 (group B). The probability of survival, event-free survival, nonrejection mortality, and rejection for group A patients were 66%, 62%, 37%, and 4%, respectively, with a median follow-up of 12 years (range, 8.3-16.2 years). Group B patients treated with the new protocol had some improvement in thalassemia-free survival (67%) and lower transplant-related mortality (27%) than that of previous protocols. However, transplant-related mortality in these high-risk patients remains elevated. Current myeloablative BMT in adult patients is characterized by higher transplant-related toxicity due to an advanced phase of disease. Although this new approach to transplant adult patients with a reduced-dose intensity-conditioning regimen has improved thalassemia-free survival, transplant-related mortality in these high-risk patients remains elevated.
目前的常规输血方案和螯合疗法显著提高了地中海贫血患者的生存率，使得成年患者的比例有所增加。然而，这些患者与疾病和治疗相关的并发症会随着时间的推移而进展，导致严重的发病率和预期寿命缩短。干细胞移植仍然是目前地中海贫血患者唯一可用的治愈方法。本研究更新了1988年至1996年间接受来自人类白细胞抗原（HLA）匹配的相关供者的骨髓移植（BMT）的107例中位年龄为22岁（范围17 - 35岁）的成年患者的移植结果（A组），并描述了1997年至2003年间接受新治疗方案（方案26）治疗的15例中位年龄为21岁（范围17 - 31岁）的成年患者的BMT结果（B组）。A组患者的生存概率、无事件生存概率、非排斥死亡率和排斥率分别为66%、62%、37%和4%，中位随访时间为12年（范围8.3 - 16.2年）。接受新方案治疗的B组患者在无地中海贫血生存方面有一定改善（67%），且移植相关死亡率（27%）低于先前方案。然而，这些高危患者的移植相关死亡率仍然较高。目前成年患者的清髓性BMT的特点是由于疾病处于晚期，移植相关毒性较高。尽管这种采用降低剂量强度预处理方案的新方法改善了成年患者的无地中海贫血生存，但这些高危患者的移植相关死亡率仍然较高。
