This study aimed to investigate the effectiveness of continuous renal replacement therapy and hemoperfusion in acute severe organophosphorus pesticide poisoning patients. Fifty-eight acute severe organophosphorus pesticide poisoning patients who accepted therapy in Nangjing First Hospital, Nangjing Medical University from January 2018 to December 2022 were chosen as the study participants. They were randomly divided into a control group and a research group. Both groups received conventional treatment, while the research group received continuous renal replacement therapy combined with hemoperfusion therapy. Relative to the control group, the research group had higher total effectiveness rate, shorter coma time, cholinase recovery time, reduced stay in intensive care unit, less atropine dosage, lower levels of inflammatory factors and lower sequential organ failure assessment scores. Our study suggests that continuous renal replacement therapy combined with hemoperfusion can improve the clinical therapeutic effect in patients with acute severe organophosphorus pesticide poisoning, effectively control the inflammatory response, and shorten the clinical rescue and treatment time of patients.

BACKGROUND AND OBJECTIVES:Vitamin A is vital for the growth and health of children. This study aimed to estimate the current vitamin A status and the prevalence of vitamin A deficiency (VAD) among preschool children and explore the correlation between serum vitamin A concentration and changes in hematological parameters.METHODS AND STUDY DESIGN:The study included 697 children aged 1-6 years, presenting for routine checkups at the Department of Pediatrics, Peking University Shougang Hospital, Beijing, from April 2017 to December 2020. We obtained the complete laboratory test data of 630 children.RESULTS:The mean serum vitamin A concentration among preschool children was 0.29±0.08 mg/L, with a median of 0.29 mg/L. The proportion of children with VAD and marginal VAD (MVAD) was 9.84% and 43.49%, respectively. The highest prevalence of VAD and MVAD was in the 3- to 4-year age group. Compared with the normal vitamin A serum concentration group, other groups had lower mean corpuscular volume, mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, and higher red blood cell distribution width. The mean serum vitamin A concentration among anemic children was significantly lower (0.27±0.07 mg/L) than among those children who were not anemic (p<0.05).CONCLUSIONS:VAD constitutes a public health problem in northern China. The prevalence of VAD is highest, and the serum vitamin A concentration was the lowest among preschool children aged 3-4 years. Vitamin A serum concentration was associated with red blood cell indices. We should attach more importance to those children aged 3-4 years.

Abstract. Background:. The role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with high-risk (HR) T-cell acute lymphoblastic leukemia (T-ALL) in first complete remission (CR1) is still under evaluation. Moreover, relapse is the main factor affecting survival. This study aimed to explore the effect of allo-HSCT (especially haploidentical HSCT [haplo-HSCT]) on improving survival and reducing relapse for HR childhood T-ALL in CR1 and the prognostic factors of childhood T-ALL in order to identify who could benefit from HSCT. Methods:. A total of 74 newly diagnosed pediatric T-ALL patients between January 1, 2012 and June 30, 2018 were enrolled in this retrospective study. Patients were stratified into the low-risk chemotherapy cohort (n = 16), HR chemotherapy cohort (n = 31), and HR transplant cohort (n = 27). Characteristics, survival outcomes, and prognostic factors of all patients were then analyzed. Results:. Patient prognosis in the HR chemotherapy cohort was significantly worse than that in the low-risk chemotherapy cohort (5year overall survival [OS]: 58.5% vs. 100%, P = 0.003; 5-year event-free survival [EFS]: 54.1% vs. 83.4%, P = 0.010; 5-year cumulative incidence of relapse [CIR]: 45.2% vs. 6.3%, P = 0.011). In HR patients, allo-HSCT improved the 5-year EFS and CIR compared to that of chemotherapy (5-year EFS: 80.1% vs. 54.1%, P = 0.041; 5-year CIR: 11.6% vs. 45.2%, P = 0.006). The 5-year OS was higher in the HR transplant cohort than that in the HR chemotherapy cohort (81.0% vs. 58.5%, P = 0.084). Minimal residual disease re-emergence was an independent risk factor for 5-year OS, EFS, and CIR; age ≥10 years was an independent risk factor for OS and EFS; and high white blood cell count was an independent risk factor for EFS and CIR. Conclusion:. Allo-HSCT, especially haplo-HSCT, could effectively reduce relapse of children with HR T-ALL in CR1.

Background The presence of minimal residual disease (MRD) is an independent risk factor for poor prognosis in patients with acute lymphoblastic leukemia (ALL). Moreover, the role of chimeric antigen receptor T-cell (CAR-T) therapy in patients with MRD is currently unclear. Methods We conducted a prospective study to investigate the role of CAR-T therapy in patients with persistent/recurrent MRD-positive ALL in first remission. Results A total of 77 patients who had persistent/recurrent MRD were included. Of these patients, 43 were enrolled in the CAR-T group, 20 received chemotherapy as a bridge to allogeneic hematopoietic cell transplantation (allo-HSCT), and 14 patients received intensified chemotherapy. MRD negativity was achieved in 90.7% of the patients after CAR-T infusion. Patients who received CAR-T therapy had a higher 3-year leukemia-free survival (LFS) than patients who did not (77.8% vs. 51.1%, P = 0.033). Furthermore, patients in the CAR-T group had a higher 3-year LFS than those in the chemotherapy bridge-to-allo-HSCT group [77.8% (95% CI, 64.8–90.7%) vs. 68.7% (95% CI, 47.7–89.6%), P = 0.575] and had a significantly higher 3-year LFS than those in the intensified chemotherapy group [77.8% (95% CI, 64.8–90.7%) vs. 28.6% (95% CI, 4.9–52.3%), P = 0.001]. Among the patients who received CAR-T therapy, eight were not bridged to allo-HSCT, and six (75%) remained in remission with a median follow-up of 23.0 months after CAR-T infusion. Conclusions Our findings show that CAR-T therapy can effectively eliminate MRD and improve survival in patients with a suboptimal MRD response.

Purpose: To assess the prognostic effect of different levels of IKZF1 gene deletions in pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL).Methods: IKZF1 Δ2-8/ALB deletions were quantified using multiplex real-time quantitative PCR in newly diagnosed pediatric BCP-ALL patients. Seventy-four patients with IKZF1 deletions ≥ 0.01% were included. Clinical characteristics, laboratory data, and treatment outcomes were analysed.Results: The patients were divided into two groups: IKZF1 deletions < 1% (group A) and ≥ 1% (group B). Group B patients had a higher BCR-ABL1 positive rate than did group A patients. The proportions of patients who had an age at onset ≥10 years old, and white blood cell count ≥ 50×109/L were significantly higher in group B than in group A. The 3-year overall survival (OS) and 3-year event-free survival (EFS) rates in group B were 79 ± 8.8% and 62.4 ± 9.7%, respectively, being significantly lower than those in group A (97.7 ± 2.2% and 83.2 ± 5.8%, respectively). The level of IKZF1 deletions ≥ 1% and the central nervous system leukemia were independent risk factors of EFS.Conclusions: Pediatric BCP-ALL patients with high levels of IKZF1 gene deletions have a poorer prognosis than those with low levels.

急性髓系白血病(AML)是由于造血干细胞分化障碍及增殖过度所致异常骨髓造血前体细胞聚集的恶性克隆性疾病.近年来随着危险度分层的合理应用、靶向药物的不断研发、支持治疗的进步、造血干细胞移植技术的日渐成熟,儿童AML的生存率已经较前有明显提高.文章综述儿童AML的治疗进展.

Acute lymphoblastic leukemia (ALL) is a heterogeneous disease whose prognostic factors include minimal residual disease (MRD) and cytogenetic abnormalities. To explore the significance of MRD in ALL subtypes, we analyzed the outcomes of 1126 children treated with risk-stratified therapy based on sequential MRD monitoring. MRD distributions and treatment outcomes differed between distinct leukemia subtypes. Patients with ETV6-RUNX1 or hyperdiploidy had the best prognosis (5-year OS: 97 ± 1.5% and 89.2 ± 2.7%). However, hyperdiploidy patients with MRD ≥ 10% on day 15 had a higher risk of relapse (36.4%) than those with ETV6-RUNX1. TCF3-PBX1 patients had the fastest disease clearance (negative MRD rate on day 33: 92.1%), but the overall prognosis was intermediate (5-year OS: 82.5%). Patients with high-risk characteristics and ALL-T had inferior outcomes: even with undetectable MRD on day 33, cumulative incidence of relapse was 19.9% and 23.4%, respectively. Moreover, those with poor early-treatment response and detectable week-12 MRD had a worse prognosis. After adjusting for other risk factors, re-emergent MRD was the most significant adverse prognostic indicator overall. Sequential MRD measurement is important for MRD-guided therapy, and integration of MRD values at different timepoints based on leukemia subtype could allow for more refined risk stratification.

目的 了解儿童白血病中性粒细胞缺乏合并草绿色链球菌脓毒症的危险因素及临床特征.方法 回顾分析111例白血病中性粒细胞缺乏合并脓毒症患儿的临床资料.结果 111例患儿共发生136例次脓毒症,检测到138株菌株,其中25株为草绿色链球菌.25例次草绿色链球菌脓毒症中,男12例次、女13例次,脓毒症发生时中位年龄98(66～138)月,距末次化疗结束中位时间9(6～10) d,中性粒细胞缺乏已持续中位时间5(4～6) d,脓毒症发生时中性粒细胞中位值0.01(0～0.01)×109/L.25例次均有发热,热峰(39.3±0.7) ℃,中位发热持续时间1.5(1.0～2.8)d;11例次存在口腔感染,7例次合并上呼吸道感染,3例次继发肺部感染,1例次合并胃肠炎,1例次合并感染中毒性脑病;5例次除发热外无其他感染症状.将138株菌株分为草绿色链球菌组(25株)、非草绿色链球菌革兰阳性菌组(14株)及革兰阴性菌组(99株) 3组,三组间年龄、急性髓系白血病所占比例、热峰、口腔感染发生率差异均有统计学意义(P<0 . 05);草绿色链球菌组急性髓系白血病及口腔感染比例较高.草绿色链球菌对万古霉素、利奈唑胺及氯霉素100 %敏感,对大环内酯类及青霉素类耐药率较高.结论 草绿色链球菌是白血病中性粒细胞缺乏合并脓毒症的常见病原体,急性髓系白血病及口腔感染是感染该菌高危因素,万古霉素及利奈唑胺可作为粒细胞缺乏发热合并口腔感染患儿的经验用药.

OBJECTIVE To explore the impact of induction treatment response on the prognosis of pediatric core binding factor-acute myeloid leukemia (CBF-AML). METHODS The result of induce reaction and survival data of 157 pediatric CBF-AML patients in our hospital from September 2008 to December 2018 were retrospectively analyzed．The survival rate of the patients with different degrees of morphological remission after induction chemotherapy was comparative analyzed. RESULTS Among the 157 children with CBF-AML, 113 (72.4%) patients achieved morphologic leukemia-free state (MLFS) after the first course of induction chemotherapy, 153 (98.1%) patients achieved MLFS after the second course of induction chemotherapy. The 5-year event-free survival (EFS) rate and 5-year overall survival (OS) rate of patients with non-remission (NR) status after the first course of induction of chemotherapy was significantly lower than the patients achieved MLFS and the patients achieved partial remission (PR). The 5-year EFS rate and 5-year OS rate of the patients with PR status after the second course of induction chemotherapy were lower than the patients achieved MLFS, but the difference was not statistically significant. Multivariable analyze showed that NR after the first course of induction chemotherapy and myeloid sarcoma were the independent risk factors affecting EFS of the patients. There were six patients with NR status after the first course of induction chemotherapy, in which all of them harbored t(8;21), three of them with sex chromosome deletion, two of them with myeloid sarcoma. CONCLUSION NR status after the first course of induction chemotherapy was the independent risk factor affecting EFS and OS of CBF-AML patients, it can be taken as an indicator for higher risk stratification. PR status after the first course of induction chemotherapy may not be used as a diagnostic criterion for primary drug resistance.

背景 儿童复发/难治性急性淋巴细胞白血病(ALL)临床治疗较困难、预后差.硼替佐米联合化疗显示出一定的有效性,目前国内相关报道较少.目的 探讨硼替佐米联合化疗对复发难治及高危儿童ALL的疗效及安全性.设计病例系列报告.方法 纳入2017年9月至2019年9月北京大学人民医院儿科收治的接受硼替佐米联合化疗的复发难治及高危ALL患儿,分析其临床特征、实验室检查、治疗情况和预后.主要结局指标总反应率(ORR)、2年无事件生存(EFS)、总生存(OS)、不良反应.结果 11例患儿纳入分析,男9例,女2例,诊断中位年龄10(3～15)岁.7例T-ALL,2例T淋巴母细胞淋巴瘤Ⅳ期,2例B-ALL.复发2例,难治4例,高危5例.经硼替佐米联合化疗1个疗程后,8例(B-ALL 1例,T-ALL 7例)治疗有反应,ORR 72.7％.治疗前骨髓形态未缓解3例,部分缓解4例,治疗后完全缓解率85.7％(6/7);治疗前骨髓微小残留病(MRD)阳性10例,治疗后均有下降,其中1例MRD转阴.不良反应总体可耐受,主要为骨髓抑制、感染、胃肠道反应、肝功能异常等,无化疗相关死亡病例.9例行异基因造血干细胞移植治疗,其中7例无病存活,2例死于移植后复发;2例患儿继续化疗,长期随访均死亡.中位随访时间15(2～29)个月,2年EFS为(45.5±15)％,OS为(63.6±14.5)％.结论 硼替佐米联合化疗是复发难治及高危ALL患儿的一种有效、耐受性良好的治疗选择.

Although chimeric antigen receptor T-cell (CAR-T) therapy produces a high complete remission rate among patients with relapsed/refractory B-cell acute lymphoblastic leukemia, relapse remains an urgent issue. It is uncertain whether consolidative haploidentical-allogeneic hematopoietic stem cell transplantation (haplo-HSCT) is suitable for achieving sustainable remission. Therefore, we aimed to assess the efficacy and safety of bridging CAR-T therapy to haplo-HSCT. Fifty-two patients with relapsed/refractory Philadelphia chromosome-negative B-cell acute lymphoblastic leukemia who underwent haplo-HSCT after CAR-T therapy were analyzed. The median time from CAR-T therapy to haplo-HSCT was 61 days. After a median follow-up of 24.6 months, the 1-year probabilities of event-free survival, overall survival, and cumulative incidence of relapse were 80.1% (95% confidence interval (CI), 69.0–90.9), 92.3% (95% CI, 85.0–99.5), and 14.1% (95% CI, 10.7–17.4), respectively, while the corresponding 2-year probabilities were 76.0% (95% CI, 64.2–87.7), 84.3% (95% CI, 74.3–94.3), and 19.7% (95% CI, 15.3–24.0), respectively. No increased risk of 2-year cumulative incidence of graft-versus-host disease, treatment-related mortality, or infection was observed. A pre-HSCT measurable residual disease-positive status was an independent factor associated with poor overall survival (hazard radio: 4.201, 95% CI: 1.034–17.063; P = 0.045). Haplo-HSCT may be a safe and effective treatment strategy to improve event-free survival and overall survival after CAR-T therapy.

Background: In pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL), the IKZF1 gene deletions is an molecular marker of poor prognosis. We aimed to assess the prognostic effect of different levels of IKZF1 gene deletions in pediatric BCP-ALL. Procedure: IKZF1 Δ2-8/ALB deletions were quantified using multiplex real-time quantitative PCR (RQ-PCR) in newly diagnosed pediatric BCP-ALL patients between June 2014 and January 2018. Seventy-four patients with IKZF1 deletions of ≥ 0.01% were included. Clinical characteristics, laboratory data, and treatment outcomes were analyzed. Results: The patients were divided into two groups: IKZF1 deletions of < 1% (Group A) and of ≥ 1% (Group B). Patients in group B had a higher BCR-ABL1 positive rate than those in group A (P = 0.001). The proportions of patients who had an age at onset of ≥10 years old, and white blood cell count ≥ 50×109/L were significantly higher in group B than in group A (P < 0.05). The 3-year overall survival (OS) and 3-year event-free survival (EFS) rates in group B were 79 ± 8.8% and 62.4 ± 9.7%, respectively, which were significantly lower than the 3-year OS (97.7 ± 2.2%, P = 0.022) and 3-year EFS (83.2 ± 5.8%, P = 0.019) in group A. Multivariate analysis revealed that the level of IKZF1 deletions of ≥ 1% and CNSL were independent risk factors of EFS. Conclusions: Pediatric BCP-ALL patients with high levels of IKZF1 gene deletions have a poorer prognosis than those with low levels.

ABSTRACT Objective: Malignancy-associated hemophagocytic lymphohistiocytosis (M-HLH) in children is a relatively rare but life-threatening secondary hemophagocytic lymphohistiocytosis (sHLH). Until now, only a limited number of cases regarding children with M-HLH has been reported. Methods: We conducted a retrospective study of 27 children with M-HLH, who admitted to our center between July 2007 and October 2019. The clinical data and laboratory data were analyzed. Results: The median age of the children with M-HLH was 7 years. Underlying diseases included myeloid malignancy (n = 6), lymphoid malignancy (n = 18) and unknown type lymphoma (n = 3). The one-year mortality rate was 56%. All patients had persistent fever. The clinical manifestations included hepatomegaly (89%), splenomegaly (67%) and central nervous system symptoms (56%). Thirteen children (48%) had Epstein–Barr virus (EBV) infection. No significant differences were observed between EBV-positive and negative M-HLH patients in terms of most clinical indicators. However, EBV-positive M-HLH patients showed prolonged activated partial thromboplastin time (APTT) and more hemophagocytosis in the bone marrow (BM) in contrast to EBV-negative patients. Eighteen patients (67%) received the HLH-94/04 regimen as the initial treatment. There were no significant differences in the overall survival (OS) between EBV-positive and negative patients. Patients with prolonged APTT had a significantly poorer OS than other patients (p = 0.012). Conclusions: The M-HLH children with EBV infection are more likely to have prolonged APTT and more hemophagocytosis in BM. The M-HLH children had a poor prognosis, especially those with prolonged APTT.

Purpose: To investigate the effect of tyrosine kinase inhibitors (TKIs) on the statural growth in children with acute lymphoblastic leukemia (ALL). Methods: We retrospectively collected data from 344 children with ALL younger than 17 years old at diagnosis identified in pediatric department of Peking University People's Hospital. The children were divided into three groups: conventional chemotherapy group, imatinib group and dasatinib group. Height was expressed as standard deviation score(HtSDS). In the three groups, we compared the HtSDS and Delta HtSDS at the start of treatment and during follow-up period and also compared the adult height and median parental height(MPH). We further compared the HtSDS classified by age and gender in imatinib group. At last, univariate analysis was used to analyze the influencing factors on the deceleration of height growth by imatinib. Results: There were 298 children in conventional chemotherapy group, 39 in imatinib group and 7 in dasatinib group. In imatinib group, the mean HtSDS of children at follow-up time was significantly lower than that at the start of treatment (P< 0.05), regardless of age and gender. In imatinib group, the decrease of HtSDS in girls was more obvious than in boys(P = 0.031). The HtSDS gradually decreased in the first and the second year in imatinib group. After discontinuation of imatinib, the HtSDS had no obvious change. Multivariate analysis showed that the HtSDS at the start of imatinib was negatively correlated with severe growth impairment on imatinib therapy. The HtSDS in dasatinib group and conventional chemotherapy group maintained a high degree of consistency. Conclusion: Imatinib can affect growth velocity in children with ALL, regardless of age and gender. With the discontinuation of imatinib, the inhibitory effect will not continue. The lower HtSDS at the start of imatinib therapy, the more obvious effect of imatinib on growth impairment will be, and the effect will be more obvious in girls than boys.

Background Pediatric acute myeloid leukemia (AML) with t(8;21) (q22;q22) is classified as a low-risk group. However, relapse is still the main factor affecting survival. We aimed to investigate the effect of allogeneic hematopoietic stem cell transplantation (allo-HSCT) on reducing recurrence and improving the survival of high-risk pediatric t(8;21) AML based on minimal residual disease (MRD)-guided treatment, and to further explore the prognostic factors to guide risk stratification treatment and identify who will benefit from allo-HSCT. Methods Overall, 129 newly diagnosed pediatric t(8;21) AML patients were included in this study. Patients were divided into high-risk and low-risk group according to RUNX1-RUNX1T1 transcript levels after 2 cycles of consolidation chemotherapy. High-risk patients were divided into HSCT group and chemotherapy group according to their treatment choices. The characteristics and outcomes of 125 patients were analyzed. Results For high-risk patients, allo-HSCT could improve 5-year relapse-free survival (RFS) rate compared to chemotherapy (87.4% vs. 61.9%; P = 0.026). Five-year overall survival (OS) rate in high-risk HSCT group had a trend for better than that in high-risk chemotherapy group (82.8% vs. 71.4%; P = 0.260). The 5-year RFS rate of patients with a c-KIT mutation in high-risk HSCT group had a trend for better than that of patients with a c-KIT mutation in high-risk chemotherapy group (82.9% vs. 75%; P = 0.400). Extramedullary infiltration (EI) at diagnosis was associated with a high cumulative incidence of relapse for high-risk patients (50% vs. 18.4%; P = 0.004); allo-HSCT can improve the RFS ( P = 0.009). Conclusions allo-HSCT can improve the prognosis of high-risk pediatric t(8;21) AML based on MRD-guided treatment. Patients with a c-KIT mutation may benefit from allo-HSCT. EI is an independent prognostic factor for high-risk patients and allo-HSCT can improve the prognosis.

Early death (ED) and treatment-related toxicity emerge as two major barriers for curing paediatric acute promyelocytic leukaemia (APL) patients. This study aims to investigate the effect of idarubicin on controlling hyperleukocytosis in induction therapy and the efficacy and safety of a risk-adapted attenuated consolidation chemotherapy. We summarised the characteristics and long-term outcomes of 73 paediatric APL patients treated at our institution from February 2002 to October 2018, during which treatment protocols evolved over three periods and were defined as protocol A, B and C chronologically. All of the patients received an all-trans retinoic acid (ATRA)-arsenic trioxide (ATO) combination remission induction therapy, with hydroxyurea (group A) or idarubicin (group B and C) to control hyperleukocytosis. Consolidation chemotherapy was modified with risk-adapted attenuated intensity and minimised cumulative doses of anthracyclines for group C (144 mg/m2 and 288 mg/m2 of daunorubicin equivalents for standard- and high-risk patients, respectively). The median initial WBC, platelet count, and fibrinogen were 2.9 × 109/L (range 0.9–158.3 × 109/L), 32 × 109/L (range 4–226 × 109/L), and 160 mg/dL (range 53–549 mg/dL), respectively. High-risk and standard-risk were seen in 20.5% and 79.5% of patients, respectively. Three patients (4.1%) suffered early haemorrhagic death. At the end of induction therapy, 68 (93.2%) patients achieved haematologic complete remission (HCR). At a median follow-up of 91.97 months, the estimated 5-year overall survival (OS) and event-free survival (EFS) rates for the whole cohort were 95.9 ± 2.3% and 88.7 ± 3.8%, respectively. A comparison of HCR rates and documented instances of toxicity between groups A and B + C showed no significant differences. However, idarubicin significantly reduced the peak WBC count (Z = − 3.292, P = 0.001) and duration of hyperleukocytosis (Z = − 2.827, P = 0.005). Estimated 3-year EFS (91.7 ± 8.0%) and OS (100%) rates for group C were not significantly different from those for group B, whereas the risk of treatment-related infections was significantly reduced (χ2 = 5.515, P = 0.019). Idarubicin (8–10 mg/m2/day for 2 days) for hyperleukocytosis control in induction therapy is safe and effective for paediatric APL. Risk-adapted attenuated consolidation chemotherapy is advocated.

The prognosis of myeloid sarcoma (MS) is controversial. Many reports indicated that orbital-MS has a good prognosis and is closely related to t(8;21), but the prognostic role of MS in pediatric t(8;21) AML is unclear. We retrospectively analyzed data from 127 patients with pediatric t(8;21) AML diagnosed between January 2010 and June 2018. We compared patients with (n = 30) and without MS (n = 97). The median follow-up time was 52.6 months. The proportion of t(8;21) AML patients with MS was 23.6%. Males were more likely to have MS than females. The complete remission rate after the first course of induction chemotherapy and the 3-year relapse-free survival (RFS) among patients with MS were lower than those among patients without MS (60% vs. 78.4%,p = 0.045) (68.8 +/- 8.8% vs. 88.0 +/- 3.4%,p = 0.004). The female sex and a higher level ofRUNX1/RUNX1T1transcripts after consolidation were risk factors for poor RFS among patients with MS. Our data showed that MS was an independent risk factor in pediatric t(8;21) AML. Close monitoring of measurable residual disease of the bone marrow and extramedullary lesions is needed to guide stratified treatment.

OBJECTIVE:To study the influence of dasatinib treatment on body height in children with acute myeloid leukemia (AML).METHODS:A retrospective analysis was performed for the clinical data of 86 AML children aged <17 years. According to the treatment regimen, these children were divided into a conventional chemotherapy group and a dasatinib chemotherapy group. The 57 children in the conventional chemotherapy group were given conventional chemotherapy drugs without tyrosine kinase inhibitor, and the 29 children in the dasatinib chemotherapy group were given conventional chemotherapy drugs and dasatinib. The two groups were compared in terms of height standard deviation score (HtSDS) at the beginning of treatment and after treatment, as well as the change in HtSDS after 1 and 2 years of treatment.RESULTS:There was no significant difference in HtSDS between the conventional and dasatinib chemotherapy groups before treatment. Within the first two years of treatment, the dasatinib chemotherapy group had a similar change trend of HtSDS as the conventional chemotherapy group. Four children in the dasatinib chemotherapy group reached the final adult height during follow-up, which was significantly lower than the target height (P=0.044). In the conventional chemotherapy group, there was no significant difference between final adult height and target height. In the dasatinib chemotherapy group, the children in adolescence had a significant change in HtSDS after treatment (P=0.032).CONCLUSIONS:Dasatinib treatment may affect the final height of children with AML, and the use of dasatinib after the beginning of adolescence may lead to growth disorder, but dasatinib treatment has little effect on body height in the short-term treatment.

We retrospectively analyzed characteristics and outcomes of low-risk acute myeloid leukemia. The incidence rates of extramedullary infiltration, myeloid sarcoma, and central nervous system leukemia in this study were 24.76%, 20.56%, and 5.61%, respectively. Patients with extramedullary infiltration had worse 3-year relapse-free survival (67.0% +/- 6.6% vs. 86.9% +/- 2.9%; P = .000) and worse 3-year overall survival (76.1% +/- 6.2% vs. 87.8% +/- 2.8%; P=.026). Myeloid sarcoma was an independent risk factor for poor relapse-free survival (hazard ratio, 2.640; 95% confidence interval, 1.281-5.440; P = .008) and overall survival (hazard ratio, 2.079; 95% confidence interfal, 0.969-4.460; P = .049). Background: The impact of extramedullary infiltration (EMI) on the clinical outcomes of pediatric patients with acute myeloid leukemia (AML) are controversial. Patients and Methods: A total of 214 pediatric patients with low-risk AML were classified as having EMI (central nervous leukemia [CNSL] and/or myeloid sarcoma [MS]) and not having EMI. Patients with isolated MS before AML diagnosis by bone marrow examination were confirmed with histopathologic examination. For patients diagnosed with AML by bone marrow examination, a thorough physical examination and radiologic imaging were used to confirm MS. Results: Male gender, a high white blood cell count, the FAB-M5 subtype, t(8;21) and t(1;11) abnormalities, and c-KIT mutations were associated with EMI. The presence of MS was associated with a low complete remission rate (63.6% vs. 79.4%; P = .000) and poor 3-year relapse-free survival (RFS) (62.6% +/- 7.5% vs. 87.0% +/- 2.8%; P = .000) and 3-year overall survival (73.5% +/- 7% vs. 88.8% +/- 2.6%; P = .011). Multivariate analysis revealed that MS was a poor prognostic factor for RFS and overall survival. Bone infiltration was an independent risk factor for inferior RFS with MS. Patients with CNSL had a low complete remission rate (58.3% vs. 77.2%; P = .045); however, CNSL did not significantly affect the survival of low-risk patients with AML. Conclusion: MS should be considered an independent risk factor to guide stratified treatment. (C) 2020 Elsevier Inc. All rights reserved.

Three patients with B-cell acute lymphocytic leukemia with relapsed central nervous system leukemia (CNSL) received CD19-specific chimeric antigen receptor modified T cell (CAR-T) therapy and clinical response was evaluated. They experienced a complete remission in cerebrospinal fluid by CAR-T infusion with no life-threatening complications. CAR-T infusion may be an effective and safe treatment for patients with relapsed/refractory CNSL. Background: chimeric antigen receptor-modified T cell (CAR-T) therapy is an effective and promising treatment for refractory and multiply relapsed B-cell acute lymphoblastic leukemia (B-ALL). Because of its side effects and poor responses such as neurotoxicity and cytokine release syndrome, patients with central nervous system leukemia were excluded in most previous clinical trials of CAR-T treatment. Patients and Methods: We enrolled 3 B-ALL patients with central nervous system leukemia relapse. They were infused with CD19-specific CAR-Ts, and their clinical responses were evaluated by bone marrow smear, flow cytometry, and cytogenetic alterations detected by quantitative PCR, interleukin-6, and the expansion and persistence of circulating CAR-Ts in peripheral blood and cerebrospinal fluid. Results: After CAR-T infusion, 2 of the 3 patients experienced bone marrow minimal residual disease-negative complete remission, and all patients tested negative for residual leukemia cells in cerebrospinal fluid tested by flow cytometry. These 3 patients experienced grade 2 or 3 cytokine release syndrome, which resolved completely after symptomatic treatment. None experienced neurotoxicity or needed further intensive care. Conclusion: CAR-T infusion is a potentially effective treatment for relapsed/refractory B-ALL patients with central nervous system involvement. (C) 2020 Elsevier Inc. All rights reserved.