BACKGROUND:Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an important curative treatment for pediatric patients with SAA. The chimeric anti-CD20 monoclonal antibody rituximab (RTX) is the most widely used agent to deplete circulating B cells for preventing the complication of donor-specific human leukocyte antigen antibodies and treating Epstein-Barr virus (EBV) reactivation in patients who undergo allo-HSCT. However, RTX efficacy and safety in allo-HSCT recipients with SAA remain unclear in pediatric patients. METHODS:This study included 105 pediatric patients with SAA who underwent allo-HSCT at Wuhan Children's Hospital. The patients' basic characteristics, stem cell transplantation, survival, GVHD occurrence, CMV and EBV reactivation and amounts of lymphocytes was compared in the patients with RTX (RTX+) and without RTX (RTX-) treatment. RESULTS:Compared with the RTX-, the RTX+ showed a significantly higher incidence of GVHD (16.6% vs 4%; P = 0.02). The RTX+ was associated with a markedly higher rate of CMV reactivation (76.7% vs 38.7%; P < 0.001) and lower rate of EBV reactivation (60.0% vs 89.3%; P < 0.001). Among 1 month after HSCT, the RTX+ exhibited lower T-cell (P = 0.04), B-cell (P < 0.001), and NK-cell (P = 0.01) counts, which exhibit no significant difference in the long-term period. CONCLUSION:The pre-treatment of RTX exhibits beneficial effects on the overall and long-term response, indicated by 100% successful implantation, lower risk of EBV reactivation, and little influence on lymphocyte regeneration. More emphasis should be placed on preventing CMV reactivation among RTX-treated pediatric SAA patients undergoing allo-HSCT.

OBJECTIVE:To summarize the clinical characteristics, treatment outcomes, and prognosis of two children with STAT3 gene mutation-associated hyper-IgE syndrome(HIES) who underwent allogeneic hematopoietic stem cell transplantation(allo-HSCT). METHODS:A retrospective analysis was performed on two pediatric patients with STAT3-mutated HIES (STAT3-HIES) who received allo-HSCT. Data included baseline clinical features, transplant protocols, post-transplant outcomes, complications (graft-versus-host disease, infection, virus reactivation), serum IgE levels, and long-term follow-up. RESULTS:The two patients (one male, one female; ages 2 years 10 months and 10 years 10 months at transplant) received peripheral blood stem cells from HLA 9/10-matched unrelated donors and were conditioned with the classic BUCY regimen(busulfan+cyclophosphamide+antithymocyte globulin). Neutrophils and platelets engraftment were achieved on day+11 and day +9/+11, respectively. Complete donor chimerism(100%) was confirmed on day+15 post-transplant. No grade II-IV acute GVHD or viral reactivation occurred. Serum IgE levels decreased markedly at 30 days post-transplant compared to pre-transplant. Pulmonary CT findings (e.g., infection foci,cavitation) improved significantly. By the last follow-up (April 30, 2025), both children survived, and their quality of life improved significantly (infection control, resolution of skin eczema, and improvementin organ function). CONCLUSION:For children with STAT3-mutated HIES who experience recurrent severe respiratory infections impairing quality of life, allo-HSCT with a suitable donor can effectively control infection, improve immune abnormalities, and long-term outcomes.

OBJECTIVE:To investigate the clinical characteristics, pathogenic distribution, and risk factors of bloodstream infection (BSI) in children with aplastic anemia (AA) following allogeneic hematopoietic stem cell transplantation (allo-HSCT), to provide clinical evidence for the early identification of high-risk patients and optimization of empirical antibacterial therapy regimens. METHODS:The clinical data of 113 children with AA who underwent allo-HSCT at the Pediatric Hematology and Oncology Center of Wuhan Children's Hospital from August 2016 to December 2024 were retrospective collected. The incidence of bloodstream infection, pathogenic distribution, and identify relevant risk factors in these patients was analyzed. RESULTS:Among the 113 children with AA, 23 cases (20.4%) developed bloodstream infection after allo-HSCT. A total of 26 pathogenic strains were isolated, including 13 Gram-negative strains (50.0%), 10 Gram-positive strains (38.5%), and 3 fungal strains (11.5%). The most commonly detected pathogenic bacteria were Klebsiella pneumoniae, Escherichia coli, and Pseudomonas aeruginosa, with the detection rate of carbapenem-resistant Gram-negative organisms (CRO) reaching 26.1%. Multivariate analysis indicated that active infection prior to transplantation (HR=3.749, 95%CI:1.398-10.055, P=0.009) was an independent risk factor for post-transplant BSI. Moreover, the OS rate of children with agranulocytosis duration >17 days was significantly lower than that of those with agranulocytosis duration ≤17 days. CONCLUSION:Gram-negative bacteria are the predominant pathogens causing bloodstream infection in children with AA following allo-HSCT. Additionally, the presence of active infection prior to transplantation was identified as an independent risk factor for post-transplant BSI.

Donor cell leukemia is a rare but severe complication following an allogeneic hematopoietic stem cell transplantation (allo-HSCT), particularly in patients with non-malignant diseases, due to its aggressive clinical progression and poor prognosis. In recent years, with the increasing number of umbilical cord blood stem cell transplantation cases, umbilical cord blood–derived donor cell leukemia has garnered growing attention. Although umbilical cord blood cells may inherently carry tumor precursor characteristics, host environmental factors, immunosuppression, and chemotherapy during transplantation can exacerbate their transformation into leukemia. Herein, we report a clinical case of a pediatric patient with severe aplastic anemia who developed umbilical cord blood–derived early T-cell precursor acute lymphoblastic leukemia 2 years and 5 months after receiving an allo-HSCT. Despite multiple chemotherapy regimens, the patient ultimately failed to achieve remission and succumbed to the disease. Although this outcome is regrettable and highlights the refractory nature of post-transplant donor cell leukemia, further investigation into its molecular biological mechanisms is warranted to develop early prevention strategies and more effective therapeutic approaches.

OBJECTIVES:To investigate the prognostic value of molecular minimal residual disease (Mol-MRD) monitored before and after allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric acute myeloid leukemia (AML). METHODS:Clinical data of 71 pediatric AML patients who underwent HSCT between August 2016 and December 2023 were analyzed. Mol-MRD levels were dynamically monitored in MRD-positive patients, and survival outcomes were evaluated. RESULTS:No significant difference in the 3-year overall survival (OS) rate was observed between patients with pre-HSCT Mol-MRD ≥0.01% and <0.01% (77.3% ± 8.9% vs 80.4% ± 7.9%, P=0.705). However, patients with pre-HSCT Mol-MRD <1.75% had a significantly higher 3-year OS rate than those with Mol-MRD ≥1.75% (86.6% ± 5.6% vs 44.4% ± 16.6%, P=0.020). The median Mol-MRD level in long-term survivors was significantly lower than in non-survivors [0.61% (range: 0.04%-51.58%)] vs 10.60% (range: 1.90%-19.75%), P=0.035]. Concurrent flow cytometry-based MRD positivity was significantly higher in non-survivors (80% vs 24%, P=0.039). There was no significant difference in the 3-year overall survival rate between patients with Mol-MRD ≥0.01% and those with <0.01% at 30 days post-HSCT (P=0.527). For children with Mol-MRD <0.22% at 30 days post-HSCT, the 3-year overall survival rate was 80.4% ± 5.9%, showing no significant difference compared to those with molecular negativity (87.0% ± 7.0%) (P=0.523). CONCLUSIONS:Patients with pre-HSCT Mol-MRD <1.75% or post-HSCT Mol-MRD <0.22% may achieve long-term survival outcomes comparable to Mol-MRD-negative cases through HSCT and targeted interventions.

IntroductionITPR3 encodes a subunit of the inositol 1,4,5-trisphosphate receptor (IP3R), which forms a Ca2+ channel on the endoplasmic reticulum (ER) membrane responsible for ER Ca2+ release. Recently, both autosomal dominant and recessive ITPR3 mutations have been reported in association with combined immunodeficiency (CID), accompanied by multisystem manifestations including neurological involvement. MethodsWe retrospectively analyzed the clinical characteristics of two patients with ITPR3 deficiency accompanied by hemophagocytic lymphohistiocytosis (HLH) at our center. Through a literature review, we further compared their clinical manifestations with those of combined immunodeficiency (CID) patients who presented with HLH.Results and DiscussionsOur two CID patients with multisystem disorders harboring germline heterozygous ITPR3 mutations (c.7570C>G, p.Arg2524Gly and c.7570C>T, p.Arg2524Cys). Both patients developed severe Epstein-Barr virus (EBV)-associated HLH, and one patient succumbed to disease-related complications. Our study demonstrates that ITPR3-associated CID confers a susceptibility to EBV-driven pathologies, particularly HLH, which warrants heightened clinical vigilance. Therefore, early hematopoietic stem cell transplantation (HSCT) should be considered to improve survival outcomes in these patients.

BACKGROUND:Blinatumomab has been approved for the treatment of pediatric B-cell acute lymphoblastic leukemia (B-ALL). This study aimed to investigate the association between leukemia burden and the efficacy of blinatumomab in real-world applications. METHODS:A real-world study was conducted by enrolling patients aged 0-18 years who were diagnosed with CD19-positive B-ALL and treated with blinatumomab between January 2021 and May 2023 from 14 centers in China. RESULTS:A total of 304 patients were enrolled in this analysis. In the patients with > 5% blasts before blinatumomab (non-complete remission, NCR group), 75.9% achieved complete remission (CR) and 69.0% of the NCR patients achieved minimal residual disease (MRD) negativity. Among the patients with ≤ 5% blasts but multiparametric flow cytometry MRD (MFC MRD) positive (MRD+ group), 98.9% achieved MRD negativity. Of the MFC MRD negative patients (MRD- group), the quantitative polymerase chain reaction MRD (qPCR MRD) and next-generation sequencing MRD (NGS MRD) clearance rate was 60.0% (12/20) and 65.5% (19/29), respectively. Additionally, patients in the MRD- and MRD+ groups had significantly better outcomes than those in the NCR group, with 30-month overall survival (OS) rates of 95.3% (95% CI: 91.4%-99.3%), 91.2% (95% CI: 85.0%-97.8%), and 77.6% (95% CI: 67.4%-89.4%), respectively (p < 0.001), and 30-month event-free survival (EFS) rates of 93.9% (95% CI: 89.6%-98.3%), 90.8% (95% CI: 85.0%-97.1%), and 56.7% (95% CI: 41.0%-78.6%), respectively (p < 0.001). In this study, 41.4% of patients experienced grade ≥ 3 adverse events (AEs), with hematological toxicity being the most common (33.2%). The severe adverse events, such as cytokine release syndrome (CRS) and neurotoxicity, occurred at a low rate, particularly grade ≥ 3, at 3.6% and 2.6%, respectively. CONCLUSIONS:Overall, these results indicate that blinatumomab is effective and well tolerated. Patients with a lower leukemia burden before blinatumomab administration tend to have better OS and EFS with fewer AEs.

ANCA-vasculitis (AAV) is a small-vessel vasculitis characterized by the presence of autoantibodies against proteinase-3 (PR3) or myeloperoxidase (MPO). The dynamics of the T-cell response within tissues is studied best in animal models. It was the aim to analyze the lesional T-cell dynamics in the experimental autoimmune vasculitis model. Female Wistar Kyoto-rats were immunized with human MPO emulsified in complete Freund's adjuvant. Control animals received complete Freund's adjuvant without MPO. Selected groups received anti-IL17A treatment. Lesional T-cells from kidneys were assessed by flow cytometry (FACS), realtime polymerase chain reaction (PCR) and EliSpot. All animals immunized with MPO developed signs of vasculitis. At week six, lung damage expressed as petechial bleeding score and renal damage quantified by albuminuria were highest. As analyzed by FACS, the fraction of renal Th17 cells peaked at week six in MPO rats equaling the proportion of Th1 cells. MPO-specific renal Th1 and Th17 cells were detectable by EliSpot at weeks four and six post-immunization in MPO-immunized rats being absent in control rats. Neutralization of IL-17A did not affect the development of humoral and cellular anti-MPO immunity. Likewise, pulmonary and renal vasculitis were not ameliorated. In summary, the dynamics of the lesional T-cell response in the EAV model shows a major participation of MPO-specific Th17 and Th1 cells in renal vasculitis. Simple cytokine neutralization was not efficacious in this disease model so that combined neutralization approaches should be studied further.

OBJECTIVE:To analyze the factors associated with mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with severe aplastic anemia (SAA). METHODS:The clinical data of 90 children with SAA who received allo-HSCT in the Department of Hematology, Wuhan Children's Hospital Affiliated to Tongji Medical College, Huazhong University of Science and Technology from August 2016 to July 2023 were collected. The clinical features and causes of death were analyzed retrospectively. Cox proportional hazards model was used to screen the risk factors of death. RESULTS:Only 9 children died with a median time of 6.3(2.6, 8.3) months among the 90 children with SAA after allo-HSCT. Among the 5 deaths due to infection, 3 were pulmonary infection, including 2 cases of cytomegalovirus pneumonia. One case developed septic shock due to gastrointestinal infection. One case experienced graft failure, which was complicated by bloodstream infection, and developed septic shock. Three cases died of transplantation-associated thrombotic microangiopathy (TA-TMA). One case died of gastrointestinal graft-versus-host disease (GVHD). The results of multivariate analysis showed that post-transplant +60 d PLT≤30×109/L (HR=7.478, 95%CI : 1.177-47.527, P =0.033), aGVHD Ⅲ-Ⅳ (HR=7.991, 95%CI : 1.086-58.810, P =0.041), and TA-TMA occurrence (HR=13.699, 95%CI : 2.146-87.457, P =0.006) were independent risk factors for post-transplant mortality. CONCLUSION:Allo-HSCT is an effective therapy for SAA in children. Post-transplant +60 d PLT≤30×109/L, aGVHD Ⅲ-Ⅳ, and TA-TMA occurrence are independently associated with post-transplant mortality, which may be helpful for early detection of potential high-risk children and optimization of clinical diagnostic and treatment strategies.

Objective:To investigate the clinical features and risk factors associated with cutaneous chronic graft-versus-host disease(cGVHD)after allogeneic hematopoietic stem cell transplantation(allo-HSCT)in children.Methods:A retrospective analysis was conducted on the clinical data of children who underwent allo-HSCT in the Wuhan Children's Hospital from August 1,2016,to December 31,2023,and were regularly followed up for 1 year or more.The differences in clinical features between children with and without cutaneous cGVHD were compared,and the risk factors affecting the occurrence of cutaneous cGVHD were analyzed.Results:During the study period,296 children received allo-HSCT.Until December 31,2024,follow-up showed that 20 children(6.8％)developed cutaneous cGVHD,which manifested as cutaneous lichenification,hyperpigmentation,keratosis pilaris,sclerotic changes,and hair or nail involvement.According to their skin lesion area and degree of grading,5 cases were mild,10 cases were moderate,and 5 cases were severe.Multivariate logistic regression analysis revealed that female donors and previous acute GVHD were risk factors for the development of cutaneous cGVHD after allo-HSCT.All 20 children were treated with glucocorticoid±calcineurin inhibitors(tacrolimus/cyclosporine)as first-line therapeutic agents.Only 1 child improved after first-line treatment.The remaining 19 children treated with a second-line regimen of combination interventions based on individualized status,including 10 children who could not tolerate hormonotherapy or first-line treatment,and showed no significant improvement after 3 months,as well as 9 children with multi-organ cGVHD.After comprehensive second-line treatment,17 children showed improvement in cutaneous symptoms.There were 3 deaths,including 1 due to primary disease recurrence and 2 due to pulmonary infections.Conclusion:The skin is the first manifestation and most common organ involved in cGVHD in children.Cutaneous cGVHD severely affects the daily activities of transplanted children and requires prolonged immunosuppressive therapy,but has a favorable prognosis.First-line treatments for adults are not applicable to children who usually require a combination treatment with multiple drugs.

OBJECTIVE:To analyze the molecular genetic spectrum of children with acute myeloid leukemia (AML), and explore its correlation with clinical characteristics and prognosis. METHODS:The clinical and molecular genetic data of 116 children with newly diagnosed AML in Wuhan Children's Hospital from September 2015 to August 2022 were retrospectively analyzed. The Fisher's exact test was used to analyze the correlation of gene mutations with clinical features, and Kaplan-Meier curve was used to analyze the influences of gene mutations on the prognosis. RESULTS:NRAS (22%), KRAS (14.9%), and KIT (14.7%) mutations were the most common genetic abnormalities in 116 children with AML. Children with KIT, CEBPA and GATA2 mutations showed a higher median onset-age than those without mutations (all P < 0.05). Children with FLT3-ITD mutation exhibited a higher white blood cell count at initial diagnosis compared to those without mutations (P < 0.05). Children with ASXL2 mutation had lower platelet count and hemoglobin at initial diagnosis than those without mutations (both P < 0.05). KIT mutations were often co-occurred with t(8;21)(q22;q22). There was no significant relationship between gene mutation and minimal residual disease (MRD) remission rate after the first and second induction therapy (P >0.05). KIT and NRAS mutations were not associated with prognosis significantly (P >0.05). The overall survival (OS) rates of children with CEBPA and FLT3-ITD mutations were superior to those without mutations, but the differences were not statistically significant (P >0.05). The 3-year OS rate of 61 children treated by allogeneic hematopoietic stem cell transplantation was 89.8%, which was significantly higher than 55.2% of those only treated by chemotherapy (P < 0.001). CONCLUSIONS:Gene mutations are common in children with AML, and next-generation sequencing can significantly improve the detection rate of gene mutations, which can guide the risk stratification therapy. In addition, FLT3-ITD and KIT mutations may no longer be poor prognostic factors.

This study aims to explore the association between plasma human terminal complement complex C5b-9 (sC5b-9) levels and the occurrence of haematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA) in children. The study retrospectively analysed the data of 131 patients who underwent allogeneic haematopoietic stem cell transplantation (allo-HSCT) at the Department of Haematology-Oncology between May 2020 and July 2023. The clinical data included dynamic data on plasma sC5b-9 concentrations and analysed the potential of pre- and post-transplantation levels for the prediction and diagnosis of TA-TMA after allo-HSCT. The median age of the cohort was 5.9 [3.4, 10.8] years, and it comprised 73 (55.8%) male and 58 (44.2%) female patients. The primary diseases were haematologic malignancy in 57 cases (43.5%), aplastic anaemia in 39 cases (29.8%) and Mediterranean anaemia in 13 cases (9.9%). Out of the 131 patients, 21 (14.7%) developed TA-TMA, and the median time of onset was 88 [59, 136] days after transplantation. The 1-year overall survival rate was significantly higher in the non-TA-TMA group (92.7% ± 2.5%) than in the TA-TMA group (47.6% ± 10.7%) (χ 2  = 35.71, p  < 0.05). During TA-TMA, sC5-9 levels are significantly elevated ( p  < 0.05). However, the baseline levels of sC5b-9 before allo-HSCT were not related to the development of TA-TMA after the procedure. The plasma sC5b-9 concentration after allo-HSCT in children is closely related to the occurrence of TA-TMA, and 306.4 ng/mL is an optimal clinical cut-off level above which TA-TMA is indicated. This cut-off level was associated with a sensitivity of 90.5% and a specificity of 86.6%. Nonetheless, the diagnosis of TA-TMA should be established in conjunction with clinical manifestations and corroborated by additional laboratory test results.

Background:Chalazion is common in children which can lead to cosmetically disfiguring, ptosis, astigmatism, and affect the quality of life. The aim of this study was to investigate the distribution patterns of serum total immunoglobulin E (T-IgE) and specific immunoglobulin E (sIgE) levels in children with chalazia, further to explore the clinical significance of immunoglobulin E (IgE) in the development of chalazion. Methods:This retrospective cross-sectional study enrolled children with chalazion or allergic conjunctivitis (AC) in Hubei, China, from June 2020 to December 2021. The food and inhaled sIgE were summarized. Meanwhile, the correlations between serum T-IgE, the numbers of positive sIgE, grades of sIgE, and the stages of chalazion in children were analyzed. Results:A total of 224 children with chalazion were included, among whom 98 (43.8%) were males, with average age of 3.5±2.0 years (range, 0.5 to 11.2 years). Compared to AC, the sIgE positive rate of epithelial materials of dogs was higher in chalazia children (9.4% vs. 0.6%, P=0.001). The positive rate of food allergens-sIgE in preschool children with chalazion (71.4%) was higher than that in school children (50.0%) (P=0.008). Furthermore, the numbers and the grade of sIgE had a positive correlation with the stages of chalazia, respectively (R=0.269, 0.245, P<0.001). Moreover, there was also a positive correlation between T-IgE and the stages of chalazia (R=0.238, P=0.001). Conclusions:Compared to AC, children with chalazion are more sensitive to the epithelial materials of dogs in Hubei province. Meanwhile, children with high level of T-IgE and more positive allergens-sIgE associated with more severe stage of chalazia.

Objective:To summarize the clinical features of reversible posterior encephalopathy syndrome(PRES)after allogeneic hematopoietic stem cell transplantation(allo-HSCT)in children.Methods:The clinical data of six children who developed PRES after undergoing allo-HSCT in the Department of Hematology of Wuhan Children's Hospital from June 2016 to December 2022 were retrospectively analyzed,and their clinical characteristics,imaging examination,laboratory examination,and treatment regression were summarized.Results:Among 281 children underwent allo-HSCT,6 cases(2.14％)developed PRES,with a median age of 5.1(1.5-9.7)years old.4 cases underwent related haploidentical donor transplantation,and 2 cases underwent sibling allografting and unrelated donor allografting donor transplantation,respectively.All six children had an acute onset of illness,with clinical manifestations of nausea and vomiting,seizures,psychiatric disorders,visual disturbances.The five cases elevated blood pressure.All children with PRES were treated with oral immunosuppressive drugs during seizures,and 3 cases were combined with different degrees of graft-versus-host disease.Most of the children showed effective improvement in clinical symptoms and imaging after adjusting/discontinuing suspected medications(cyclosporine,etc.)and symptomatic supportive treatments(oral antihypertensive,diazepam for antispasmodic,mannitol to lower cranial blood pressure),and one of them relapsed more than 8 months after the first seizure.Conclusion:PRES is rare after hematopoietic stem cell transplantation in children,and its onset may be related to hypertension,cytotoxic drugs,graft-versus-host disease,etc.Most of them can be recovered after active treatment,but not completely reversible,and the prognosis of those who combined with TMA is poor.

To investigate the clinical significance of dynamic monitoring ecotropic virus integration site-1 (EVI1) expression in childhood acute myeloid leukemia (AML). A retrospective analysis was conducted on 113 pediatric AML patients of Wuhan Children’s Hospital from 2014 to 2022. The correlation between EVI1 expression levels and clinical indicators including clinical characteristics, first complete remission (CR1), relapse, and overall survival (OS) was analyzed. Receiver operating characteristic (ROC) curve analysis was carried out to comprehend the influence of EVI1 expression on relapse. A total of 78 AML children with EVI1 expression at initial diagnosis were eligible, divided into EVI1-positive (EVI1high) and EVI1-negative (EVI1low) groups. FAB classification (P = 0.047) and abnormal karyotype (P = 0.009) showed significant differences between the two groups. The proportion of EVI1high in individuals with complex and/or monomeric karyotypes was significantly higher than in other cases (P = 0.032). When completing the first induction therapy, the EVI1high group showed a significantly lower CR1 rate than the EVI1low group (P = 0.015). Among 51 cases with EVI1 expression dynamically monitored, those with EVI1 overexpression more than twice had significantly shorter OS (P < 0.05). Among 19 non-HSCT patients undergoing three EVI1 assessments during induction therapy, those with EVI1 overexpression over once had higher relapse rates (P = 0.045). In addition, EVI1 expression level ≥ 83.38

OBJECTIVE:To investigate the safety and efficacy of mitoxantrone liposome in the treatment of children with high-risk acute myeloid leukemia (AML). METHODS:The children with high-risk AML who received the mitoxantrone liposome regimen at Wuhan Children's Hospital from January 2022 to February 2023 were collected as the observation group, and the children with high-risk AML who received idarubicin regimen were enrolled as controls, and their clinical data were analyzed. Time to bone marrow recovery, the complete remission rate of bone marrow cytology, the clearance rate of minimal residual disease, and treatment-related adverse reactions were compared between the two groups. RESULTS:The patients treated with mitoxantrone liposome showed shorter time to recovery of leukocytes(17 vs 21 day), granulocytes(18 vs 24 day), platelets(17 vs 24 day), and hemoglobin(20 vs 26 day) compared with those treated with idarubicin, there were statistical differences (P <0.05). The effective rate and MRD turning negative rate in the observation group were 90.9% and 72.7%, respectively, while those in the control group were 94.1% and 76.4%, with no statistical difference (P >0.05). The overall response rate of the two groups of patients was similar. CONCLUSION:The efficacy of mitoxantrone liposome is not inferior to that of idarubicin in children with high-risk AML, but mitoxantrone liposome allows a significantly shorter duration of bone marrow suppression and the safety is better.

Objective:To investigate the prognostic value of minimal residual disease(MRD)detected by multi-parameter flow cytometry(MFC)in pediatric patients with acute myeloid leukemia(AML)after induction chemotherapy.Methods:A retrospective study was conducted on 97 pediatric patients initially diagnosed with AML at Wuhan Children's Hospital from August 2015 to December 2022.The study analyzed the results of MRD detection using MFC after the first and second cycles of induction chemotherapy,and its association with prognosis were analyzed.Results:Following the first cycle of induction treatment,57 of the 97 patients tested positive for MRD(MRD1+,58.8％).Subsequently,19 patients remained MRD positive(MRD2+,19.6％)after the second cycle of induction treatment.Kaplan-Meier survival analysis showed that the estimated 3-year overall survival(OS)rate of the 37(64.9％)MRD1+patients who underwent transplantation was significantly higher than that of the 20(35.1％)MRD1+patients who did not undergo transplantation(84.6％vs 40.0％,P=0.0001).Among the 35 MRD1+MRD2-patients,the 3-year OS rate of the 25 children who underwent transplantation was higher than that of the 10 children who did not undergo transplantation(87.2％vs 70.0％,P=0.3229).The 3-year OS rate of the 19 MRD1+MRD2+patients was lower than that of the 35 MRD1+MRD2-patients(57.4％vs 81.8％,P=0.059).In the 19 MRD2+patients,the 3-year OS rate of the 12 children who underwent transplantation was significantly higher than that of the 7 children who did not undergo transplantation(80.8％vs 14.3％,P=0.0007).There was no significant difference in 3-year OS between the 12 MRD1+MRD2+patients and 25 MRD1+MRD2-patients,both treated with transplantation(80.8％vs 87.2％,P=0.8868).In those not treated with transplantation,the 7 MRD1+MRD2+patients had a significantly lower 3-year OS compared with the 10 MRD1+MRD2-patients(14.3％vs 70.7％,P=0.0114).Further multivariate analysis indicated that MRD2 positivity and transplantation were both independent prognostic factors(P=0.031,0.000),while MRD1 positivity was not significantly associated with the overall prognosis of 97 patients(P=0.902).Conclusion:MRD positivity following the second cycle of induction chemotherapy is an independent risk factor for unfavorable outcomes in children with AML.MRD2 positivity indicates a poorer prognosis and can help to identify the candidates requiring transplantation.MRD2 positivity is not a contraindication for transplantation in pediatric patients,and early transplantation significantly improves the prognosis of high-risk patients.

The influence of ABO incompatibility on transplant results in juvenile aplastic anemia (SAA) children receiving hematopoietic stem cell transplantation (HSCT) is debated, and no published data are available. This study involved 85 children with AA who received hematopoietic stem cell transplants from donors who were ABO-compatible (n = 38), major ABO-incompatible (n = 17), minor ABO-incompatible (n = 21), and bi-directionally incompatible (n = 9). Except for a statistical difference in acute graft-versus-host disease (aGVHD), the four groups did not have significantly different chronic GVHD, overall survival, and neutrophil and platelet implantation rates, and no pure red cell aplasia or passenger lymphocyte syndrome. Therefore, we inferred that ABO blood group-incompatible donors do not significantly affect HSCT outcomes and are not a barrier.

BackgroundFor children with severe aplastic anemia, if the first immunosuppressive therapy (IST) fails, it is not recommended to choose a second IST. Therefore, for patients without matched sibling donor (MSD) and matched unrelated donor (MUD), haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) can be chosen as a salvage treatment. This article aims to explore the comparison between upfront Haplo-HSCT and salvage Haplo-HSCT after IST.Methods29 patients received salvage Haplo-HSCT, and 50 patients received upfront Haplo-HSCT. The two groups received Bu (Busulfan, 3.2mg/kg/d*2d on days -9 to-8), CY (Cyclophosphamide, 60mg/kg/d*2d on days -4 to-3), Flu (fludarabine, 40mg/m2/d*5d on days -9 to -5) and rabbit ATG (Anti-thymocyte globulin, total dose 10mg/kg divided into days -4 to -2).ResultsThe OS of the salvage Haplo-HSCT group showed no difference to the upfront Haplo-HSCT group (80.2 ± 8.0% vs. 88.7 ± 4.8%, p=0.37). The FFS of the salvage Haplo-HSCT group also showed no difference to the frontline Haplo-HSCT group (75 ± 8.2% vs. 84.9 ± 5.3%, p=0.27). There was no significant difference in the incidence of other complications after transplantation between the two groups, except for thrombotic microangiopathy (TMA). In the grouping analysis by graft source, the incidence of II-IV aGVHD in patients using PBSC ± BM+UCB was lower than that in the PBSC ± BM group (p=0.010)ConclusionUpfront Haplo-HSCT and salvage Haplo-HSCT after IST in children with acquired severe aplastic anemia have similar survival outcomes. However, the risk of TMA increases after salvage Haplo-HSCT. This article provides some reference value for the treatment selection of patients. In addition, co-transplantation of umbilical cord blood may reduce the incidence of GVHD.