骨髓移植治疗地中海贫血。美国的经验。

M C Walters, K M Sullivan, R J O'Reilly, F Boulad, J Brockstein, K Blume, M Amylon, F L Johnson, M Klemperer, J Graham-Pole
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引用次数: 0

摘要

目的:我们回顾了在美国接受骨髓移植治疗的30例重度地中海贫血患者的结果。患者和方法:通过国际骨髓移植登记处的一项调查,确定了在西雅图接受移植的10名患者和来自美国其他5个中心的20名患者。这些移植手术于1981年11月至1992年4月进行,患者具有不同的种族背景,年龄从6个月到14岁(中位4.0岁)。在30名患者中,27名患者接受了来自人类白细胞抗原(HLA)相同的兄弟姐妹或其他家庭成员的骨髓,1名患者接受了来自无关供者的HLA匹配的骨髓,2名患者接受了来自相关供者的单倍相同但HLA不匹配的骨髓。细胞减少(准备)治疗因机构和移植前风险类别而异。一般情况下,患者给予丁硫丹(12-24 mg/kg)或二甲酰基胺(5 mg/kg)联合环磷酰胺(120-240 mg/kg)。一部分患者接受720 cGy剂量的全身照射(TBI),随后接受环磷酰胺(120 mg/kg)照射。结果:27例接受hla相同家族成员骨髓移植的患者中有16例(59%)是无事件幸存者,移植后随访时间从2个月到> 10年不等。27例患者中有6例(22%)地中海贫血复发,5例(19%)死亡。估计地中海贫血的精算复发率为24%,无事件生存率为57%。接受hla不相同或无亲缘关系供者骨髓的3名患者中,只有1名存活。移植前未常规行肝活检。因此,将患者分为Lucarelli危险组是不可能的。采用肝大小和铁状态,通过螯合规律和血清铁蛋白水平评估,设计了改良的风险分类。使用这种分类,移植风险组间无事件生存率无显著差异。结论:在这一小部分患者中观察到的结果证实了地中海贫血可以通过骨髓移植治愈。虽然大多数患者是无事件幸存者,但相当多的患者经历了疾病复发。美国移植中心的多中心合作试验可能是必要的,以评估骨髓移植对地中海贫血的应用,并确定最佳治疗方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Bone marrow transplantation for thalassemia. The USA experience.

Purpose: We have reviewed the results of bone marrow transplantation in 30 patients with thalassemia major who were treated in the United States.

Patients and methods: Ten patients who underwent transplantation in Seattle and 20 patients from five other U.S. centers were identified through a survey of the International Bone Marrow Transplant Registry. These transplants were performed between November 1981 and April 1992 in patients with diverse ethnic backgrounds and ranged in age from 6 months to 14 years (median 4.0 years). Twenty-seven of the 30 patients received marrow from a human leukocyte antigen (HLA)-identical sibling or other family member, one patient received HLA-matched marrow from an unrelated donor, and two patients were given haploidentical but HLA-mismatched marrow from a related donor. Cytoreductive (preparative) therapy varied among institutions and pretransplant risk categories. In general, patients were given busulfan (12-24 mg/kg) or dimethylmyleran (5 mg/kg) in combination with cyclophosphamide (120-240 mg/kg). A subset of patients were given total body irradiation (TBI) at a dose of 720 cGy followed by cyclophosphamide (120 mg/kg).

Results: Sixteen of 27 patients (59%) who received marrow from an HLA-identical family member are event-free survivors, with a duration of follow-up ranging from 2 months to > 10 years after transplantation. Six of these 27 patients (22%) had recurrence of thalassemia and five (19%) died. The estimated actuarial rate of thalassemia recurrence was 24% and the rate of event-free survival was 57%. Only one of the three patients who received marrow from HLA-nonidentical or unrelated donors survives event-free. Liver biopsies were not routinely performed before transplant. Thus, classification of patients into Lucarelli risk groups was not possible. A modified risk classification was devised by using liver size and iron status assessed by the regularity of chelation and the serum ferritin level. With use of this classification, there was no significant difference in event-free survival between transplant risk groups.

Conclusions: The findings observed in this small series of patients confirms that thalassemia can be cured with bone marrow transplantation. Although most patients are event-free survivors, a significant number experienced recurrence of their disease. A cooperative multicenter trial of U.S. transplant centers may be necessary to evaluate the use of marrow transplantation for thalassemia and to determine optimal treatment.

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