Mollee P, Woodward N, Durrant S, Lockwood L, Gillett E A, Morton J, Rowell J
Bone Marrow Transplant Unit, Royal Brisbane Hospital, Queensland, Herston, Australia.
Intern Med J. 2001 Aug;31(6):337-42. doi: 10.1046/j.1445-5994.2001.00078.x.
In severe aplastic anaemia, the treatment of choice for young patients with a human leucocyte antigen-matched sibling is now established as allogeneic bone marrow transplantation (BMT). In older patients and in those without a matched sibling donor, immunosuppressive therapy is the usual first option. 'Alternative' marrow donors are emerging as an option for those without a matched sibling donor.
To review 10 years of local experience in treating severe aplastic anaemia with BMT and immunosuppressive therapy with emphasis on long-term outcomes.
A retrospective analysis was performed of all patients with severe aplastic anaemia presenting to the Royal Brisbane and Royal Children's Hospitals between 1989 and 1999. Data were abstracted regarding patient demographics, pretreatment characteristics and outcome measures, including response rates, overall survival and long-term complications.
Twenty-seven consecutive patients were identified, 12 treated with immunosuppression alone and 15 with BMT. In these two groups, transfusion independence was attained in 25% and 100%, respectively, with overall survival being 36% and 100%, respectively. Those treated with immunosuppression were significantly older (median 41.5 versus 22 years, P = 0.008). Long-term survivors of either treatment had extremely low morbidity. Three patients carried pregnancies to term post-transplant. Three patients received alternative donor BMT with correspondingly excellent survival.
Patients treated with allogeneic BMT for severe aplastic anaemia enjoyed extremely good long-term survival and minimal morbidity. Patients treated with immunosuppressive therapy had a poorer outcome reflecting their older age and different usage of therapies over the past decade. Optimal treatment strategies for severe aplastic anaemia remain to be determined.
在重型再生障碍性贫血中，对于有人类白细胞抗原匹配同胞供者的年轻患者，目前公认的治疗选择是异基因骨髓移植（BMT）。对于老年患者以及没有匹配同胞供者的患者，免疫抑制治疗通常是首选。“替代”骨髓供者正成为没有匹配同胞供者患者的一种选择。
回顾10年中应用BMT和免疫抑制治疗重型再生障碍性贫血的本地经验，重点关注长期疗效。
对1989年至1999年间在皇家布里斯班医院和皇家儿童医院就诊的所有重型再生障碍性贫血患者进行回顾性分析。提取有关患者人口统计学、预处理特征和结局指标的数据，包括缓解率、总生存率和长期并发症。
共确定27例患者，12例仅接受免疫抑制治疗，15例接受BMT。在这两组中，分别有25%和100%的患者实现了输血独立，总生存率分别为36%和100%。接受免疫抑制治疗的患者年龄明显较大（中位年龄41.5岁对22岁，P = 0.008）。两种治疗的长期存活者发病率极低。3例患者移植后足月妊娠。3例患者接受了替代供者BMT，生存率相应良好。
接受异基因BMT治疗的重型再生障碍性贫血患者长期生存率极高，发病率极低。接受免疫抑制治疗的患者结局较差，这反映了他们的年龄较大以及过去十年中治疗方法的不同使用情况。重型再生障碍性贫血的最佳治疗策略仍有待确定。
