Data_Sheet_1_Pure Red Cell Aplasia and Other Haematological Diseases Associated With Thymoma: A Case Series and Systematic Review.pdf

Background: Thymoma-associated haematological diseases (HDs), such as pure red cell aplasia (PRCA) and Good's syndrome, are extremely rare, and due to the paucity of large-scale studies, the characteristics, remission after thymectomy, and long-term evaluation remain undetermined.
Methods: We retrospectively assessed patients with thymoma and associated HDs from Jan 2005 to Dec 2020. All patients received thymectomy and/or additional treatments for HDs. A comparison with thymoma-associated myasthenic gravis (MG), and a systematic review from PubMed/MEDLINE and Embase were conducted.
Results: In the median follow-up of 56 months, 130 patients were enrolled. Patients with thymoma-associated MG (n = 46) and HDs [n = 8; PRCA (n = 5), PRCA and Good's syndrome (n = 2) and autoimmune haemolytic anaemia (n = 1)] were evaluated. Patients with MG had a significantly higher remission rate after thymectomy (50 vs. 17%; p = 0.0378) as compared to those with other autoimmune diseases. Two of seven patients with PRCA experienced remission with thymectomy alone, and an additional two patients achieved remission with thymectomy plus immunosuppressive therapy (IST). In the systematic review, 60 studies (case reports, n = 46; case series including the present study, n = 14) were evaluated. Forty-four percent of patients were diagnosed with PRCA after thymoma, and 61% achieved remission with thymectomy plus IST; however, Good's syndrome was unaffected.
Conclusions: Our study indicates that patients with thymoma-associated autoimmune diseases other than MG have a lower remission rate than those with MG. Remission of thymoma-associated PRCA can be achieved by thymectomy and IST. This study provides insight into extremely rare but puzzling autoimmune manifestations.

背景：胸腺瘤相关血液系统疾病（haematological diseases, HDs），如纯红细胞再生障碍性贫血（pure red cell aplasia, PRCA）与古德综合征（Good's syndrome），临床极为罕见。由于目前缺乏大规模研究，此类疾病的临床特征、胸腺切除术后缓解情况及长期预后评估仍未明确。
方法：本研究回顾性分析了2005年1月至2020年12月期间收治的胸腺瘤合并相关血液系统疾病患者。所有患者均接受胸腺切除术及/或针对血液系统疾病的额外治疗。此外，本研究与胸腺瘤相关重症肌无力（myasthenic gravis, MG）患者队列进行对照，并对PubMed/MEDLINE及Embase数据库开展了系统综述。
结果：本研究中位随访时长为56个月，共纳入130例患者。其中包括46例胸腺瘤相关重症肌无力（MG）患者，以及8例合并血液系统疾病的患者[具体为：纯红细胞再生障碍性贫血（PRCA）5例、PRCA合并古德综合征2例、自身免疫性溶血性贫血1例]。与合并其他自身免疫性疾病的患者相比，重症肌无力患者胸腺切除术后缓解率显著更高（50% vs. 17%；p=0.0378）。7例纯红细胞再生障碍性贫血患者中，2例仅通过胸腺切除术即获得缓解，另有2例患者经胸腺切除术联合免疫抑制治疗（immunosuppressive therapy, IST）实现缓解。系统综述共纳入60项研究（其中个案报告46项，包含本研究在内的病例系列研究14项）。结果显示，44%的患者在确诊胸腺瘤后被检出PRCA，61%的患者经胸腺切除术联合免疫抑制治疗（IST）获得缓解，但古德综合征患者未观察到缓解效果。
结论：本研究表明，除重症肌无力外，其他胸腺瘤相关自身免疫性疾病患者的术后缓解率低于重症肌无力患者。胸腺瘤相关PRCA可通过胸腺切除术联合免疫抑制治疗实现缓解。本研究为这类极为罕见却令人困惑的自身免疫性临床表现提供了新的研究视角。