■髓样肉瘤(MS)是一种由未成熟骨髓细胞组成的髓外恶性肿瘤。它发生在急性髓系白血病(AML)患者中,骨髓增生异常综合征(MDS),或慢性粒细胞白血病(CML)。MS可能与疾病诊断一致,或先于骨髓受累数月甚至数年;它也可以代表复发的髓外表现(1,2)。
■本研究的目的是描述波兰诊断为MS的儿童的临床特征,并分析诊断方法,治疗,结果包括总生存率(OS),无复发生存率(RFS),和无事件生存(EFS)。该研究还试图确定决定治疗结果的因素。
■研究组包括43名患者(F=18,M=25),年龄0-18岁(中位年龄,10.0岁;平均年龄,8.8年)根据肿瘤活检和免疫组织化学或根据影像学发现鉴定潜在的骨髓疾病和髓外肿瘤而诊断为MS。
■分析研究组的临床资料和使用的诊断和治疗方法。使用STATISTICAv.13(StatSoft,Inc.,塔尔萨,OK,美国),并使用MedCalc11.5.1(MedCalc软件,奥斯坦德,比利时)。在p<0.05时考虑统计学显著性。
■在研究组中,MS最常伴有AML。最常见的受累部位是皮肤,其次是轨道区域。MS的皮肤表现多见于<10岁的年龄组。最常见的遗传异常是t(8;21)(q22;q22)易位。5年OS概率(pOS),5年RFS概率(pRFS),5年EFS概率(pEFS)分别为0.67±0.08、0.79±0.07和0.65±0.08。在患有孤立性MS和患有AML/MDS并发骨髓受累的患者中,pOS值分别为0.56±0.12和0.84±0.09(p=0.0251),分别,pEFS值分别为0.56±0.12和0.82±0.08(p=0.0247),分别。在有和没有t(8;21)(q22;q22)易位的患者中,pEFS值分别为0.90±0.09和0.51±0.14(p=0.0490),分别。
■MS是一种具有高度可变的临床病程的疾病。与AML/MDS并发骨髓受累的患者相比,孤立性MS患者的治疗结果更差。发现具有t(8;21)(q22;q22)易位的患者具有显著较高的pEFS。MS位置,年龄组,化疗方案,手术,和/或放疗对治疗结局无显著影响.进一步探讨MS患儿的预后因素。
UNASSIGNED: Myeloid sarcoma (MS) is an extramedullary malignant tumor composed of immature myeloid cells. It occurs in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myeloid leukemia (CML). MS may coincide with disease diagnosis or precede bone marrow involvement by months or even years; it can also represent the extramedullary manifestation of a relapse (1, 2).
UNASSIGNED: The aim of this
study is to describe clinical characteristics of children diagnosed with MS in Poland as well as to analyze diagnostic methods, treatment, and outcomes including overall survival (OS), relapse-free survival (RFS), and event-free survival (EFS). The
study also attempted to identify factors determining treatment outcomes.
UNASSIGNED: The
study group comprised 43 patients (F=18, M=25) aged 0-18 years (median age, 10.0 years; mean age, 8.8 years) diagnosed with MS based on tumor biopsy and immunohistochemistry or identification of underlying bone marrow disease and extramedullary tumor according to imaging findings.
UNASSIGNED: The clinical data and diagnostic and therapeutic methods used in the
study group were analyzed. A statistical analysis of the treatment outcomes was conducted with STATISTICA v. 13 (StatSoft, Inc., Tulsa, OK, USA) and analysis of survival curves was conducted with MedCalc 11.5.1 (MedCalc Software, Ostend, Belgium). Statistical significance was considered at p<0.05.
UNASSIGNED: In the
study group, MS was most frequently accompanied by AML. The most common site of involvement was skin, followed by orbital region. Skin manifestation of MS was more common in the age group <10 years. The most frequent genetic abnormality was the t(8;21)(q22;q22) translocation. The 5-year OS probability (pOS), 5-year RFS probability (pRFS), and 5-year EFS probability (pEFS) were 0.67 ± 0.08, 0.79 ± 0.07, and 0.65 ± 0.08, respectively. In patients with isolated MS and those with concurrent bone marrow involvement by AML/MDS, pOS values were 0.56 ± 0.12 and 0.84 ± 0.09 (p=0.0251), respectively, and pEFS values were 0.56 ± 0.12 and 0.82 ± 0.08 (p=0.0247), respectively. In patients with and without the t(8;21)(q22;q22) translocation, pEFS values were 0.90 ± 0.09 and 0.51 ± 0.14 (p=0.0490), respectively.
UNASSIGNED: MS is a disease with a highly variable clinical course. Worse treatment outcomes were observed in patients with isolated MS compared to those with concurrent bone marrow involvement by AML/MDS. Patients with the t(8;21)(q22;q22) translocation were found to have significantly higher pEFS. MS location, age group, chemotherapy regimen, surgery, and/or radiotherapy did not have a significant influence on treatment outcomes. Further exploration of prognostic factors in children with MS is indicated.