Department of Pediatric Oncology, Children's Cancer Hospital Egypt (CCHE 57357), Cairo, Egypt.
Department of Pediatric Oncology, Children's Cancer Hospital Egypt (CCHE 57357), Cairo, Egypt; Department of Pediatric Oncology, National Cancer Institute, Cairo University, Cairo, Egypt.
Clin Lymphoma Myeloma Leuk. 2019 Mar;19(3):e142-e152. doi: 10.1016/j.clml.2018.12.011. Epub 2018 Dec 20.
Acute megakaryoblastic leukemia is a rare subtype of pediatric acute myeloid leukemia (AML) with poor outcomes in patients with non-Down syndrome. The reported outcomes have been poor, and the prognostic factors have not been clearly determined.
To evaluate the prognostic significance of various cytogenetic abnormalities and minimal residual disease status determined by flow cytometry after induction I, we retrospectively analyzed the data of 80 patients with non-Down syndrome with a diagnosis of M7 AML treated at Children's Cancer Hospital Egypt (CCHE-57357) from July 2007 through December 2016.
Of the 80 patients, 15 died during induction I and were excluded from the survival analysis. The overall survival, event-free survival, and cumulative incidence of relapse at 2 years was 52.6% ± 12.7%, 45.2% ± 12.3%, and 31.8% ± 11.5% respectively. Of the 90 patients, 61 had cytogenetic abnormalities, including trisomy 19,13q, trisomy 8, complex karyotype, t(1;22), KMT2A rearrangements, and trisomy 21. None of these had an effect on the outcomes. In addition, 34 patients had minimal residual disease < 0.1% after induction I, but the difference did not reach statistical significance. Patients with a delayed time to recovery (possibly due to myelofibrosis) had worse outcomes compared with those with early recovery (47% ± 19.2% vs. 63.2% ± 21.9%, respectively).
Acute megakaryoblastic leukemia in patients with non-Down syndrome has a poor outcome with no clearly defined prognostic factors. However, future directions to risk stratify and tailor therapy should include assessment of the tumor biology according to the molecular pathways and study of the pathogenesis of myelofibrosis in this disease, which could affect the prognosis.
急性巨核细胞白血病是一种罕见的儿童急性髓系白血病（AML）亚型，非唐氏综合征患者的预后较差。已有报道的结果较差，且预后因素尚未明确。
为了评估诱导 I 后通过流式细胞术确定的各种细胞遗传学异常和微小残留病状态的预后意义，我们回顾性分析了 2007 年 7 月至 2016 年 12 月在埃及儿童癌症医院（CCHE-57357）接受诊断为 M7 AML 治疗的 80 例非唐氏综合征患者的数据。
在 80 例患者中，有 15 例在诱导 I 期间死亡，被排除在生存分析之外。总的 2 年生存率、无事件生存率和复发累积发生率分别为 52.6%±12.7%、45.2%±12.3%和 31.8%±11.5%。90 例患者中有 61 例存在细胞遗传学异常，包括 19 三体、13q 三体、8 三体、复杂核型、t（1;22）、KMT2A 重排和 21 三体。这些均未对结果产生影响。此外，34 例患者在诱导 I 后微小残留病<0.1%，但差异无统计学意义。恢复时间延迟（可能由于骨髓纤维化）的患者预后较差，与恢复早期的患者相比（分别为 47%±19.2%和 63.2%±21.9%）。
非唐氏综合征患者的急性巨核细胞白血病预后较差，且无明确的预后因素。然而，未来分层风险和制定个体化治疗的方向应包括根据分子途径评估肿瘤生物学，并研究该疾病中骨髓纤维化的发病机制，这可能会影响预后。
