非严重再生障碍性贫血患者对单药环孢素的反应

Munazza Nabi Awan, Nighat Shahbaz, Irsa Hidayat, Asghar Ali Kerio, Hashim Khan, Awais Siddiq
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摘要

研究目的研究设计:横断面观察研究。研究地点和时间:研究地点和时间:巴基斯坦拉瓦尔品第武装部队骨髓移植中心临床血液学部,2022 年 1 月至 2023 年 12 月:方法:共纳入 51 名非小细胞肺癌患者,根据 "改良卡米塔标准",这些患者被归类为不符合重度和极重度标准的再生障碍性贫血。结果根据生存率(OS)和反应进行评估。根据英国标准血液学委员会(BCSH)的标准,在 3 个月、6 个月和 12 个月时将反应评估为完全反应(CR)、部分反应(PR)、总体反应(ORR)和无反应(NR):在51名患者中,男性34人(67%),女性17人(33%)。确诊时的中位年龄为25岁(IQR为26岁)。随访12个月时,OS为86.3%。3、6和12个月时对环孢素单药治疗的总体反应率分别为49%、57%和59%。基线血红蛋白与6个月和12个月时的应答相关,3、6和12个月时的输血依赖性与总生存率之间存在显著关联(p=0.01、0.005和0.04)。时间定义点的反应也对OS有显著影响(3个月Plog-rank = 0.046,6个月Plog-rank = 0.01,12个月Plog-rank = 0.008):3个月、6个月和12个月的总体反应率表明,CSA有可能成为一种可行的治疗方案,尤其是在资源有限的情况下。尽管一些患者出现了与治疗相关的并发症,但CSA的安全性总体上是可以耐受的:环孢素 A 非重型再生障碍性贫血 存活率 反应率 完全反应 部分反应
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Response to Single Agent Cyclosporin in Patients with Non-Severe Aplastic Anaemia.

Objective: To determine the effectiveness of cyclosporin A (CSA) monotherapy in treating patients with non-severe aplastic anaemia (NSAA).

Study design: A cross-sectional observational study. Place and Duration of the Study: Department of Clinical Haematology, Armed Forces Bone Marrow Transplant Center, Rawalpindi, Pakistan, from January 2022 till December 2023.

Methodology: A total of 51 patients of NSAA, classified as aplastic anaemia not satisfying criteria for severe and very severe disease as per Modified Camitta Criteria, were included. Results were evaluated in terms of survival rate (OS) and responses. Responses were assessed as complete response (CR), partial response (PR), overall response (ORR), and no response (NR) by using standard British Committee for standard Haematology (BCSH) response criteria at 3, 6, and 12 months.

Results: Out of 51 patients, 34 (67%) were males and 17 (33%) were females. Median age at the time of diagnosis was 25 (IQR 26) years. At follow-up of 12 months, OS was 86.3%. Overall response rates to cyclosporin monotherapy at 3, 6, and 12 months were 49%, 57%, and 59%, respectively. Baseline haemoglobin was associated with responses at 6 and 12 months and a significant association was found between transfusion dependency at 3, 6, and 12 months with overall survival (p = 0.01, 0.005, and 0.04, respectively). Responses at time-defined points also had significant impact on OS (3 months Plog-rank = 0.046, 6 months Plog-rank = 0.01, and 12 months Plog-rank = 0.008).

Conclusion: Overall response rates at 3, 6, and 12 months indicate the potential of CSA as a viable treatment option, particularly in resource-constrained settings. Despite some patients experiencing treatment-related complications, CSA demonstrated a generally tolerable safety profile.

Key words: Cyclosporin A, Non-severe aplastic anaemia, Survival rate, Response rate, Complete response, Partial response.

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