SIMULTANEOUS OCCURRENCE OF ADVANCED NEUROBLASTOMA AND ACUTE MYELOID LEUKEMIA

The authors report the case of a 4-year-old boy with a diagnosis of stage IV neuroblastoma (NB), who had been treated with 6 cycles of cyclophosphamide, doxorubicin, cisplatin, and etoposide for 12 months. The patient reached partial remission and presented a diagnosis of acute myelomonocytic leukemia (M4 AML), confirmed by immunophenotyping. After 2 months of therapy for leukemia, the child died with both malignancies in activity. A necropsy histologically confirmed the simultaneity of the two diseases. The authors review the possibilities of this association. The review leads to the conclusion that AML can occur as a secondary malignancy after the onset of the neuroblastoma, or be suggested by a misdiagnosis. The simultaneous occurrence of both as described here is not, however, found in the literature, to the best of the authors' knowledge.     

ANTITHROMBIN III SUPPLEMENTATION IN CHILDHOOD ACUTE LYMPHOBLASTIC LEUKEMIA TREATED WITH L-ASPARAGINASE

The change of plasma antithrombin III (AT) levels after supplementation of AT concentrates was examined in ALL children with acquired AT deficiency following L-asparaginase (ASP) administration. The patients received AT concentrates of 34.5 &#45 7.6 U/kg. The increase of plasma AT activity and antigen was 2.07 &#45 0.62% and 0.70 &#45 0.16 mg/dL per unit AT infused per kilogram of body weight, respectively. The activity decreased to 62.0 &#45 7.7% of the peak values by 48 hours after supplementation. The administration of AT concentrates constantly increased the plasma AT activity in ALL children treated with ASP, which may minimize the acquired prothrombotic state.     

应用维奈克拉桥接清髓性预处理方案的挽救性异基因造血干细胞移植治疗原发诱导治疗失败的急性髓系白血病一例

目的:探讨维奈克拉在难治急性髓系白血病（AML）患者移植中的应用。方法:回顾性分析2020年3月苏州大学附属第一医院收治的1例诱导治疗失败后使用维奈克拉和去甲基化药物桥接清髓性预处理方案后行异基因造血干细胞移植（allo-HSCT）的难治AML患者诊治过程。结果:患者，女性，28岁，诊断为难治AML。初始给予IA（去甲氧柔红霉素+阿糖胞苷）（3+7）方案诱导化疗未缓解，CLAG（克拉屈滨+阿糖胞苷+粒细胞集落刺激因子）方案再诱导化疗未缓解，使用维奈克拉与去甲基化药物桥接清髓性预处理方案化疗后，进行挽救性单倍体allo-HSCT。复查骨髓缓解，植入成功，随访100 d，持续缓解，无移植并发症发生。结论:对于原发诱导治疗失败的难治AML，使用维奈克拉与去甲基化药物桥接清髓性预处理可作为挽救性allo-HSCT的优选方案。     

治疗相关性白血病异基因造血干细胞移植疗效分析

目的:探讨异基因造血干细胞移植（allo-HSCT）对治疗相关性白血病（TRL）的临床效果。方法:回顾性分析2012年4月至2020年2月于航天中心医院接受allo-HSCT的14例TRL患者的临床资料，分析其疗效及生存情况。结果:14例患者中，男性5例，女性9例；中位年龄35岁（12~59岁）；急性髓系白血病12例，慢性淋巴细胞白血病/小细胞淋巴瘤1例，急性淋巴细胞白血病1例。移植时4例骨髓完全缓解，3例骨髓部分缓解，其余7例均未缓解。亲缘全相合移植5例，单倍型移植9例，均采用清髓性预处理方案。14例患者均顺利植活，中位粒细胞植活时间为16 d（10~24 d），中位血小板植活时间为13 d（10~34 d）。7例发生Ⅰ~Ⅱ级急性移植物抗宿主病（GVHD），6例发生慢性GVHD，2例发生Ⅲ级肠道GVHD。中位随访时间32个月（4~97个月），14例患者中5例死亡。结论:allo-HSCT可以改善TRL患者的预后，提高长期生存率。     

Association Between NR3C1 Gene Polymorphisms and Toxicity Induced by Glucocorticoids Therapy in Saudi Children with Acute Lymphoblastic Leukemia

Background: Glucocorticoids (GCs) are key hormones used for the treatment of acute lymphoblastic leukemia(ALL) in children, but their cytotoxic effects are not well defined. The aim of this study was to evaluate the associationbetween polymorphisms in NR3C1 encoding for protein involved in the GCs metabolism and its role in the developmentof ALL and the toxicity outcome, in terms of liver toxicity, glucose abnormality and infections, in ALL Saudi children.Methods: The following polymorphisms BCII rs41423247, ER22/23 EK rs6189 and rs6190 and N363S rs6195 inNR3C1 were analyzed in 70 children with ALL treated according to the ALL 2000 study protocol in comparison to60 control subjects. Treatment toxicities and their association with genotypes were evaluated according to CommonToxicity Criteria (NCI-CTC). Results: This study demonstrated that the NR3C1 did not contribute to the developmentof childhood ALL. Homozygous ER22/23EK polymorphism was not found in both ALL patients and in control groupwhereas the heterozygous polymorphism was only observed in the control group (6.66%). The toxicology data inthis study showed a significant difference between ALL patients carrying N363S polymorphism and wild type (40%and 6.51% respectively, P= 0.009) and a high-risk factor in the toxicity of glucose abnormality (OR=10.167; 1.302-79.339).BCII shows increased risk factors towards the liver toxicity (OR=2.667; 0.526-7.330) as well as the glucoseabnormality (OR=7.5; 1.039-54.116). Conclusion: This study suggested that the polymorphisms in NR3C1 were notassociated with the development of ALL in children. N363S polymorphism was sensitive to glucocorticoids and it maycontribute to the glucose abnormality for these patients.     

MicroRNAs in hematological malignancies

MicroRNAs (miRNAs) have become one of the hottest topics in biology over recent years, but remarkably have only been formally recognized for just over 10 years. These endogenously produced short (19–24 nt) non-coding RNAs have introduced an entirely new paradigm in our understanding of gene control and it is now evident that miRNAs play a crucial regulatory role in many, if not all, physiological and pathological processes. In this review we provide an overview of the role and potential clinical utility for miRNAs in hematological malignancies and their function in normal hematopoiesis. Although still in its infancy, the miRNA field has already added much to our understanding of hematological processes, and provides us with novel tools as both biomarkers and therapeutic agents for hematological malignancies.     

整体及重点部位护理预防白血病患者化疗期感染的效果

目的探讨整体护理合并重点部位护理预防白血病患者化疗期间感染的效果。方法将2008年4月至2013年4月收治的190例患者按就诊时间分为对照组和试验组,对照组采用白血病化疗传统护理,试验组采用整体护理合并重点部位护理。比较两组患者的感染率、感染部位和焦虑量表(SAS)评分。结果对照组患者总感染率显著高于试验组,分别为26.31%和9.47%,差异有统计学意义(P<0.05)。两组患者感染率最高的部位为呼吸道,其次为口腔、胃肠道和肛周。对照组各部位感染率均显著高于试验组(P<0.05)。SAS评分显示,对照组患者焦虑程度显著高于试验组(P<0.05)。结论整体护理合并重点部位护理能够显著降低白血病化疗患者的感染率,减轻患者身心痛苦,提高患者的生活质量,值得临床推广。     

Effect of sodium dichloroacetate on apoptotic gene expression in human leukemia cell lines

Background

Sodium dichloroacetate (DCA) is an agent with anticancer properties against solid tumors. DCA also seems to have antileukemic activity. In order to affirm it we investigate the effect of DCA on cell viability and apoptotic gene expression profiles in leukemia cell lines: CEM/C1, CCRF/CEM, HL-60, HL-60/MX2.

Clinical Benefit-Risk Profile of Lenalidomide in Patients With Lower-risk Myelodysplastic Syndromes Without del(5q): Results of a Phase III Trial

BackgroundIn the phase III MDS-005 study of patients with lower-risk, non-del(5q) myelodysplastic syndromes, lenalidomide was associated with a higher rate of ≥ 8 weeks red blood cell transfusion independence (RBC-TI) compared with placebo, but also with a higher risk of hematologic adverse events (AEs).Patients and MethodsThis analysis evaluated the ratio of clinical benefit-risk in patients treated with lenalidomide or placebo, and assessed the effect of lenalidomide dose reductions on response. Clinical benefit was a composite endpoint defined as RBC-TI, transfusion reduction ≥ 4 units packed red blood cells, hemoglobin increase ≥ 1.5 g/dL, or cytogenetic response.ResultsThe rate of clinical benefit was higher with lenalidomide than with placebo (31.9% vs. 3.8%). The ratio of response (RBC-TI and clinical benefit) to risk (hematologic AEs) favored lenalidomide over placebo. Patients who underwent ≥ 1 lenalidomide dose reduction had a longer duration of treatment, received a higher cumulative dose, and were more likely to experience clinical benefit versus patients without dose reductions.ConclusionDespite the occurrence of hematologic AEs, the overall benefit-risk profile supported lenalidomide treatment. Appropriate management of hematologic AEs by dose reductions may help patients with myelodysplastic syndromes to remain on treatment and achieve clinical benefit.