Service de Médecine Interne, Hôpital Estaing, Centre Hospitalier Universitaire de Clermont-Ferrand, Clermont-Ferrand, France.
Service de Médecine Interne, Centre Hospitalier Universitaire Lyon Sud, Hospices Civils de Lyon, Pierre-Bénite, France.
Rheumatology (Oxford). 2021 Dec 24;61(1):355-366. doi: 10.1093/rheumatology/keab363.
To characterize the clinical and biological course, management and response to treatment in SLE-associated pure red cell aplasia (PRCA).
This was a nationwide, multicentre, retrospective cohort study. From 2006 to 2018, we included adults with a diagnosis of PRCA supported by bone marrow examination and SLE or biologic manifestations of SLE after ruling out parvovirus B19 infection.
We enrolled 24 patients (20 women). SLE was diagnosed before PRCA for 14 patients (median delay 81 months). At PRCA diagnosis, mean age, haemoglobin level, and reticulocyte and differential erythroblast count were 39.2 (13.2) years, 62 ( 20) g/l, 9.1 (7.6) × 109/l and 2.8 ( 2.5)%, respectively. Eleven (45%) patients experienced multiple PRCA flares (median 6, range 2-11). CS therapy resulted in only three complete sustained responses, and 19 (79%) patients required immunosuppressive agents with highly variable regimens. After a median follow-up of 76 months (range 13-173), 17 (71%) patients showed complete response for PRCA, 5 (21%) partial response and 2 (8%) treatment failure. In total, 21 (87%) patients required red blood cell transfusion; 5 had a diagnosis of transfusion-related iron overload. Eighteen (75%) patients experienced severe infectious events requiring hospitalization.
SLE-associated PRCA is a severe condition. Repeated red blood cell transfusions and several lines of immunosuppressant therapy are mostly required, with high risk of severe infectious events and iron overload. Despite sustained response for PRCA and SLE obtained in most patients, the best therapeutic strategy remains to be determined.
描述与系统性红斑狼疮（SLE）相关的纯红细胞再生障碍性贫血（PRCA）的临床和生物学过程、治疗方法及疗效。
这是一项全国性的、多中心、回顾性队列研究。2006 年至 2018 年，我们纳入了骨髓检查支持 PRCA 诊断且排除微小病毒 B19 感染的 SLE 患者或具有 SLE 生物标志物的成年人。
我们共纳入了 24 例患者（20 例女性）。14 例患者（中位潜伏期 81 个月）在 PRCA 之前被诊断为 SLE。PRCA 诊断时，患者的平均年龄、血红蛋白水平、网织红细胞计数和有核红细胞计数分别为 39.2（13.2）岁、62（20）g/L、9.1（7.6）×109/L 和 2.8（2.5）%。11 例（45%）患者经历了多次 PRCA 发作（中位数 6 次，范围 2-11 次）。环磷酰胺（CS）治疗仅导致 3 例完全持续缓解，19 例（79%）患者需要使用不同方案的免疫抑制剂。中位随访 76 个月（范围 13-173 个月）后，17 例（71%）患者的 PRCA 完全缓解，5 例（21%）部分缓解，2 例（8%）治疗失败。总共有 21 例（87%）患者需要输血；5 例患者诊断为输血相关性铁过载。18 例（75%）患者发生严重感染事件需要住院治疗。
与 SLE 相关的 PRCA 是一种严重的疾病。大多数患者需要反复输血和多线免疫抑制剂治疗，严重感染事件和铁过载的风险较高。尽管大多数患者获得了 PRCA 和 SLE 的持续缓解，但最佳治疗策略仍有待确定。
