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首页> 外文期刊>Pediatric blood & cancer >Characteristics and outcomes of children with acute myeloid leukemia and Down syndrome who are ineligible for clinical trials due to severe comorbidities
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Characteristics and outcomes of children with acute myeloid leukemia and Down syndrome who are ineligible for clinical trials due to severe comorbidities

机译：急性髓性白血病儿童的特征和结果，患有严重的合并症导致的临床试验的唐氏综合征

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Abstract Background High survival rates of 80‐90% have been reported in recent clinical trials of reduced‐intensity chemotherapies for children with acute myeloid leukemia and Down syndrome (AML‐DS). However, a certain number of children with AML‐DS have complicating comorbidities, including congenital heart disease (CHD), and are therefore ineligible for enrolment in clinical trials. Methods We retrospectively analyzed the clinical characteristics and outcomes of children with AML‐DS who were excluded from Japanese clinical trials conducted between 2000 and 2015. Results Twelve children (six males and six females) were identified and were ineligible for CHD ( n ?=?8) and other comorbidities, including hyperleukocytosis complicated with coagulopathy, severe hemophagocytosis, pulmonary fibrosis, and hypoxic‐ischemic encephalopathy ( n ?=?1 each). The median age at the diagnosis was 14 months (range, 5 months to 11.5 years). Among all cases, 11 patients were treated with curative intent. Four patients were considered intolerant to intensive chemotherapy and received only low‐dose cytarabine‐based chemotherapy: three failed to achieve remission and died of disease, while one successfully achieved remission but eventually died of infection. Seven cases underwent regular‐intensive chemotherapy for AML‐DS: six were alive and in remission; one had relapsed disease. One patient who received the best supportive care died of disease. Finally, six patients remained in continuous complete remission, while six died. The 5‐year overall survival rate was 51%. Conclusions The prognosis of AML‐DS patients who received insufficient treatment due to severe complication was poor. The optimal dose intensity of curative chemotherapy for such cases should be explored.
机译：80-90％的摘要背景的高存活率已报告的急性髓细胞白血病和唐氏综合症（AML-DS）儿童减低强度的化疗最近的临床试验。然而，与AML-DS孩子的一定数目的复杂合并症，包括先天性心脏疾病（CHD），并因此适用于临床试验注册资格。方法回顾性分析谁被排除从2000年至2015年业绩十二个孩子（SIX男六名女）之间进行的日本临床试验的临床特点及与AML-DS儿童的成果鉴定，并没有资格对CHD（N？=？ 8）和其他合并症，包括高白细胞与凝血病并发，严重的吞噬血细胞，肺纤维化和缺氧缺血性脑病（N 2 =θ1的每个）。年龄中位数在诊断为14个月（5个月至11.5岁）。在所有的情况下，11例患者有治愈意图治疗。四名病人被认为不耐受强烈化疗和仅接受低剂量阿糖胞苷化疗：三次失败，以达到缓解和死于疾病，而一个成功实现了缓解，但最终死于感染。七例行常规密集化疗的AML-DS：六位还活着，并在缓解;一人复发的疾病。谁收到的最好的支持治疗一个病人死于疾病。最后，六名患者仍连续完全缓解，而年去世。 5年生存率为51％。结论谁因严重并发症接受治疗不足AML-DS患者的预后较差。疗效化疗这样的情况下的最佳剂量强度应该探讨。

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