Campagna Alessia, De Benedittis Daniela, Fianchi Luana, Scalzulli Emilia, Rizzo Lorenzo, Niscola Pasquale, Piccioni Anna Lina, Di Veroli Ambra, Mancini Stefano, Villivà Nicoletta, Martini Tiziano, Mohamed Sara, Carmosino Ida, Criscuolo Marianna, Fenu Susanna, Aloe Spiriti Maria Antonietta, Buccisano Francesco, Mancini Marco, Tafuri Agostino, Breccia Massimo, Poloni Antonella, Latagliata Roberto
Hematology, Sant'Andrea Hospital, Sapienza University, 00185 Rome, Italy.
Hematology, Policlinico Umberto I, Sapienza University, 00185 Rome, Italy.
J Clin Med. 2022 May 5;11(9):2596. doi: 10.3390/jcm11092596.
Aims: To define the peculiar features of patients with the deletion of the chromosome 20 long arm (del20q), data from 69 patients with myelodysplastic syndromes (MDSs) and isolated del20q, followed by the Gruppo Romano-Laziale Sindromi Mielodisplastiche (GROM-L) and Ospedale Torrette of Ancona, were collected and compared with those of 502 MDS patients with normal karyotype (NK-MDS). Results: Compared to the NK-MDS group, patients with del20q at diagnosis were older (p = 0.020) and mainly male (p = 0.006). They also had a higher rate of bone marrow blast < 5% (p = 0.004), a higher proportion of low and int-1 risk according to IPSS score (p = 0.023), and lower median platelet (PLT) count (p < 0.001). To date, in the del20q cohort, 21 patients (30.4%) received no treatment, 42 (61.0%) were treated with erythropoiesis-stimulating agents (ESA), 3 (4.3%) with hypomethylating agents, and 3 (4.3%) with other treatments. Among 34 patients evaluable for response to ESA, 21 (61.7%) achieved stable erythroid response according to IWG 2006 criteria and 13 (38.2%) were resistant. Nine patients (13.0%) progressed to acute myeloid leukaemia (AML) after a median time from diagnosis of 28 months (IR 4.1−51.7). The median overall survival (OS) of the entire cohort was 60.6 months (95% CI 54.7−66.4). the 5-year cumulative OS was 55.9% (95% CI 40.6−71.2). Conclusion: According to our results, we hypothesize that MDSs with isolated del 20q may represent a distinct biological entity, with peculiar clinical and prognostic features. The physio-pathological mechanisms underlying the deletion of the chromosome 20 long arm are still unclear and warrant future molecular analysis.
为明确20号染色体长臂缺失(del20q)患者的独特特征,收集了来自罗马-拉齐奥骨髓增生异常综合征研究组(GROM-L)和安科纳托雷特医院的69例骨髓增生异常综合征(MDS)合并孤立性del20q患者的数据,并与502例核型正常的MDS患者(NK-MDS)的数据进行比较。结果:与NK-MDS组相比,诊断时del20q患者年龄更大(p = 0.020),且以男性为主(p = 0.006)。他们骨髓原始细胞<5%的比例也更高(p = 0.004),根据国际预后评分系统(IPSS)评分,低危和中危-1的比例更高(p = 0.023),血小板(PLT)中位数更低(p < 0.001)。迄今为止,在del20q队列中,21例患者(30.4%)未接受治疗,42例(61.0%)接受促红细胞生成素(ESA)治疗,3例(4.3%)接受去甲基化药物治疗,3例(4.3%)接受其他治疗。在34例可评估对ESA反应的患者中,根据2006年国际工作组(IWG)标准,21例(61.7%)达到稳定的红系反应,13例(38.2%)耐药。9例患者(13.0%)在诊断后中位时间28个月(四分位间距4.1 - 51.7)进展为急性髓系白血病(AML)。整个队列的中位总生存期(OS)为60.6个月(95%置信区间54.7 - 66.4)。5年累积总生存率为55.9%(95%置信区间40.6 - 71.2)。结论:根据我们的结果,我们推测孤立性del20q的MDS可能代表一种独特的生物学实体,具有独特的临床和预后特征。20号染色体长臂缺失的生理病理机制仍不清楚,值得未来进行分子分析。