Abstract：
Aplastic anemia (AA) is a rare, life-threatening hematological disease, with a poorly defined incidence. As the data available on AA varies substantially worldwide, a multicenter, ambispective, observational study was carried out between 2010 and 2019 to assess the incidence, clinical management and survival of AA at seven Spanish hospitals. The incidence of AA was 2.83 per million inhabitants per year, consistent with that reported previously in Europe, with a median age at diagnosis of 61 years-old (range 12-86), and a similar number of males and females. The initial diagnosis was severe or very severe AA in 55.8% of cases and 93.7% required transfusion. The most frequent first line therapy was anti-thymocyte globulin (ATG) plus cyclosporin A (CsA, 44.2%), followed by other CsA-based regimes (46.3%), with hematopoietic stem cell transplantation an infrequent 1st line therapy. The 6-month response rate was 68.2%, which then increased over a median follow-up of 3.9 years. The 5-year overall survival (5OS) was 73.6%, similar in severe (78.6%) and very severe AA patients (74.6%) but lower in moderate AA (MAA) patients (68.4%). The 5OS was 100% in 0-25 year-old patients but dropping to 58.3% in patients ≥ 60 years-old. At the last contact, 75.8% of the patients were alive. In conclusion, the incidence, characteristics and management of AA in our study are consistent with that reported previously. In terms of survival, although the global long-term OS rate was good, there is room for improvement, particularly in older patients. Finally, what appears to be a worse long-term survival of MAA patients, as reported previously, reinforces the importance of not underestimating this condition when diagnosed as MAA.
摘要：
再生障碍性贫血（AA）是一种罕见的，危及生命的血液病,发病率定义不清。由于AA的可用数据在全球范围内差异很大，一个多中心，两性平等,2010年至2019年进行了观察性研究，以评估发病率，西班牙七家医院AA的临床管理和生存率。AA的发病率为每年每百万居民2.83人,与先前在欧洲报道的一致，诊断时的中位年龄为61岁（范围12-86岁），男性和女性数量相似。在55.8％的病例中，最初诊断为严重或非常严重的AA，并且93.7％需要输血。最常见的一线治疗是抗胸腺细胞球蛋白（ATG）加环孢菌素A（CsA，44.2%)，其次是其他基于CsA的政权(46.3%)，造血干细胞移植是一种罕见的一线治疗方法。6个月的反应率为68.2%，然后在3.9年的中位随访时间内增加。5年总生存率（5OS）为73.6%，重度AA患者（78.6%）和极重度AA患者（74.6%）相似，但中度AA（MAA）患者较低（68.4%）。在0-25岁的患者中5OS为100％，但在≥60岁的患者中降至58.3％。在最后一次接触时，75.8%的患者存活。总之,发病率，我们研究中AA的特征和管理与以前报道的一致。在生存方面,尽管全球长期OS率很好，还有改进的空间，尤其是老年患者。最后,MAA患者的长期生存率似乎更糟，如前所述，加强了诊断为MAA时不要低估这种情况的重要性。
