Fattizzo Bruno, Gurnari Carmelo, Giammarco Sabrina, Ricchiuti Antony, Awada Hussein, Bortolotti Marta, Galli Nicole, Pedone Giacinto Luca, Versino Francesco, Consonni Dario, Trikha Roochi, Gandhi Shreyans, Sica Simona, Maciejewski Jaroslaw P, Kulasekararaj Austin, Barcellini Wilma
Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy.
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Am J Hematol. 2025 Apr;100(4):584-591. doi: 10.1002/ajh.27611. Epub 2025 Jan 29.
We retrospectively analyzed a large international cohort of 1113 patients with aplastic anemia to evaluate treatment choice and outcome in elderly patients as compared with a younger population. Overall, 319 (29%) patients were > 60 years old at diagnosis (60-64 years (n = 85), 106 65-69 years (n = 106), and 128 > 70 years (n = 128)). Elderly patients showed a more severe thrombocytopenia at onset and a significantly lower overall response (complete plus partial) to first-line therapy at 6 months as compared to younger patients (47% vs. 65%, p < 0.0001), irrespective of treatment modality (ATG or CyA combinations, eltrombopag, or androgens); 27 (8%) received transplant as second line therapy and 11 (41%) died, mainly due to transplant complications. The rate of evolution to MDS was greater in elderly patients (12% vs. 7% in younger AA, p = 0.002), whilst the rate of evolution to AML was similar (1.8 vs. 1.3%). By multivariable analysis, older age remained the main factor associated with mortality [HR 1.64 (95% CI 1.5-1.7), p < 0.001], followed by disease severity by Camitta classification [HR 2.24 (95% CI 1.6-3.1) for severe AA; HR 3.8 (95% CI 2.4-6) for very severe AA], and male gender [1.45 (95% CI 1.1-1.8), p < 0.001]. In this large study, elderly AA was associated with inferior outcome even in the TPO-RA era, highlighting the need for further optimization of clinical management.
我们回顾性分析了一个由1113例再生障碍性贫血患者组成的大型国际队列，以评估老年患者与年轻人群相比的治疗选择和预后。总体而言，319例（29%）患者诊断时年龄>60岁（60 - 64岁（n = 85），65 - 69岁（n = 106），>70岁（n = 128））。与年轻患者相比，老年患者发病时血小板减少更严重，且6个月时对一线治疗的总体缓解率（完全缓解加部分缓解）显著更低（47%对65%，p < 0.0001），无论治疗方式如何（抗胸腺细胞球蛋白或环孢素联合、艾曲泊帕或雄激素）；27例（8%）接受了二线移植治疗，11例（41%）死亡，主要死于移植并发症。老年患者发展为骨髓增生异常综合征的比例更高（老年再生障碍性贫血患者为12%，年轻患者为7%，p = 0.002），而发展为急性髓系白血病的比例相似（1.8%对1.3%）。多变量分析显示，高龄仍然是与死亡率相关的主要因素[风险比1.64（95%置信区间1.5 - 1.7），p < 0.001]，其次是Camitta分类的疾病严重程度[重度再生障碍性贫血的风险比2.24（95%置信区间1.6 - 3.1）；极重度再生障碍性贫血的风险比3.8（95%置信区间2.4 - 6）]，以及男性[1.45（95%置信区间1.1 - 1.8），p < 0.001]。在这项大型研究中，即使在血小板生成素受体激动剂时代，老年再生障碍性贫血患者的预后也较差，这突出表明需要进一步优化临床管理。
