Objective:To evaluate the efficacy and safety of cord blood stem cell transplantation (CBSCT) in pediatric recipients with mucopolysaccharidosis type Ⅱ (MPS Ⅱ, Hunter syndrome).Methods:Clinical data of five male children with MPS Ⅱ who underwent CBSCT at the Department of Hematology, Children's Hospital of Soochow University between March 2018 and July 2023 were retrospectively analyzed. Post-transplantation clinical outcomes and enzymatic activity were observed. Literature was searched in the China National Knowledge Infrastructure (CNKI), Wanfang, and PubMed databases using the keywords "mucopolysaccharidosis type Ⅱ" "MPS Ⅱ" "IDS gene" and "Hunter syndrome" in both English and Chinese. Articles describing clinical manifestations, genetic diagnosis, and hematopoietic stem cell transplantation (HSCT) in MPS II were screened.Results:All five patients were male, with a median age at diagnosis of 4.3(2.5-5.5) years and a median age at transplantation of 4.6(2.8-6.5) years. At diagnosis, all exhibited coarse facial features, hepatosplenomegaly, skeletal deformities or abnormalities, abnormal head MRI findings, and Mongolian spots; four had joint stiffness, three had valvular heart disease, and two had airway obstruction, short stature, and intellectual disability. Three recipients received single-unit cord blood, and two received double-unit cord blood. Myeloablative conditioning regimens consisted of busulfan, cyclophosphamide, anti-thymocyte globulin ± fludarabine. The median neutrophil engraftment and platelet engraftment times were 19(14-21) days and 26(15-44) days, respectively. Complete donor chimerism was achieved at 1 month post-transplantation. Complications included peri-engraftment syndrome in 5 cases, acute graft-versus-host disease (GVHD) in 2 cases (1 with grade Ⅳ skin and grade Ⅱ intestinal involvement; 1 with grade Ⅱ skin involvement), limited chronic GVHD in 1 case (moderate intestinal involvement), cytomegalovirus (CMV) infection in 3 cases, Epstein-Barr virus (EBV) infection in 1 case, and capillary leak syndrome in 1 case; all were successfully managed. At the last follow-up in December 2023, all patients were alive, and enzyme activity had normalized by 3 months post-transplantation. Most clinical symptoms and signs improved; however, neurocognitive function showed no significant improvement, and some recipients exhibited progressive brain parenchymal changes on MRI. Literature review included 7 English and 5 Chinese studies, indicating that CBSCT and other HSCT modalities can improve multi-system clinical manifestations in MPS Ⅱ children, including restoration of enzyme activity, organ function improvement (such as liver and spleen shrinkage, adenoid reduction), enhanced motor function, and stabilization of neurocognitive function. Some studies suggest superior efficacy compared with enzyme replacement therapy, particularly in delaying disease progression and improving daily living abilities.Conclusion:CBSCT effectively restores enzymatic activity and improves multi-system manifestations in children with MPS Ⅱ, although its effect on neurological symptoms remains controversial. It is a safe and feasible therapeutic option for this condition.

Objective:To explore the efficacy and feasibility of hypomethylating agent (HMA) as preventive therapy in children with high-risk acute myeloid leukemia (AML) after allogeneic hematopoietic stem cell transplantation (allo-HSCT).Methods:A retrospective cohort study. Data from 173 children who underwent allo-HSCT for high-risk AML at Children′s Hospital of Soochow University between August 2019 and April 2023 were analyzed. Participants were categorized into a trial group receiving HMA and a control group. Further classification was based on HMA courses:≥4 and <4 courses. The efficacy and safety of HMA preventive treatment after allo-HSCT were evaluated. Survival analysis was performed using the Kaplan-Meier method with Log-Rank testing, the Fine-Gray model was used to assess cumulative relapse rates and Cox regression was used to identify prognostic factors. Adverse events during HMA were descriptively analyzed.Results:Among 173 patients, there were 100 males (57.8%) and 73 females (42.2%), with the age of 81 (34,127) months. The starting time of HMA was 123 (91, 191) d post-transplant, continuing 4.0 (3.0, 6.5) courses and the follow-up period was 24 (13, 32) months. The trial group (53 cases) showed better 2-year overall survival (OS) rate ((88.6±5.6)% vs. (76.6±4.3)%, χ 2=5.00, P=0.025) and relapse-free survival (RFS) rate ((89.2±4.7)% vs. (56.2±4.8)%, χ 2=15.75, P<0.001) than control group (120 cases). The 2-year OS rates and RFS rates were similar between ≥4 courses group (31 cases) and <4 courses group (22 cases)(both P>0.05). The cumulative relapse rate in the trial group was significantly lower ((10.8±0.2)% vs. (35.2±0.2)%, χ 2=10.84, P=0.001) than control group. Among children with molecular relapse, 8 cases (8/30, 26.7%) in the control group had hematological relapse compared to 1 case (1/2) in the trial group ( χ 2=0.81, P=0.369). The differences in incidence of acute and chronic graft-versus-host disease (GVHD) were not statistically significant (all P>0.05). Cox regression analysis revealed that minimal residual disease (MRD) positivity detected by flow cytometry before allo-HSCT and chronic GVHD were independent risk factors for OS (both P<0.05).The HMA preventive treatment was an independent protective factor for RFS, while age ≥10 years and MRD positivity detected by PCR before allo-HSCT were independent risk factors for RFS (all P<0.05). In trial group, 38 cases experienced grade 3 to 4 adverse events (71.7%). Conclusion:HMA is safe as preventive treatment in post-transplant children with high-risk AML, which can reduce the relapse risk and doesn't increase the risk of GVHD.

Objective:To evaluate the efficacy and safety of cord blood stem cell transplantation (CBSCT) in pediatric recipients with mucopolysaccharidosis type Ⅱ (MPS Ⅱ, Hunter syndrome).Methods:Clinical data of five male children with MPS Ⅱ who underwent CBSCT at the Department of Hematology, Children's Hospital of Soochow University between March 2018 and July 2023 were retrospectively analyzed. Post-transplantation clinical outcomes and enzymatic activity were observed. Literature was searched in the China National Knowledge Infrastructure (CNKI), Wanfang, and PubMed databases using the keywords "mucopolysaccharidosis type Ⅱ" "MPS Ⅱ" "IDS gene" and "Hunter syndrome" in both English and Chinese. Articles describing clinical manifestations, genetic diagnosis, and hematopoietic stem cell transplantation (HSCT) in MPS II were screened.Results:All five patients were male, with a median age at diagnosis of 4.3(2.5-5.5) years and a median age at transplantation of 4.6(2.8-6.5) years. At diagnosis, all exhibited coarse facial features, hepatosplenomegaly, skeletal deformities or abnormalities, abnormal head MRI findings, and Mongolian spots; four had joint stiffness, three had valvular heart disease, and two had airway obstruction, short stature, and intellectual disability. Three recipients received single-unit cord blood, and two received double-unit cord blood. Myeloablative conditioning regimens consisted of busulfan, cyclophosphamide, anti-thymocyte globulin ± fludarabine. The median neutrophil engraftment and platelet engraftment times were 19(14-21) days and 26(15-44) days, respectively. Complete donor chimerism was achieved at 1 month post-transplantation. Complications included peri-engraftment syndrome in 5 cases, acute graft-versus-host disease (GVHD) in 2 cases (1 with grade Ⅳ skin and grade Ⅱ intestinal involvement; 1 with grade Ⅱ skin involvement), limited chronic GVHD in 1 case (moderate intestinal involvement), cytomegalovirus (CMV) infection in 3 cases, Epstein-Barr virus (EBV) infection in 1 case, and capillary leak syndrome in 1 case; all were successfully managed. At the last follow-up in December 2023, all patients were alive, and enzyme activity had normalized by 3 months post-transplantation. Most clinical symptoms and signs improved; however, neurocognitive function showed no significant improvement, and some recipients exhibited progressive brain parenchymal changes on MRI. Literature review included 7 English and 5 Chinese studies, indicating that CBSCT and other HSCT modalities can improve multi-system clinical manifestations in MPS Ⅱ children, including restoration of enzyme activity, organ function improvement (such as liver and spleen shrinkage, adenoid reduction), enhanced motor function, and stabilization of neurocognitive function. Some studies suggest superior efficacy compared with enzyme replacement therapy, particularly in delaying disease progression and improving daily living abilities.Conclusion:CBSCT effectively restores enzymatic activity and improves multi-system manifestations in children with MPS Ⅱ, although its effect on neurological symptoms remains controversial. It is a safe and feasible therapeutic option for this condition.

Objective:To explore the efficacy and feasibility of hypomethylating agent (HMA) as preventive therapy in children with high-risk acute myeloid leukemia (AML) after allogeneic hematopoietic stem cell transplantation (allo-HSCT).Methods:A retrospective cohort study. Data from 173 children who underwent allo-HSCT for high-risk AML at Children′s Hospital of Soochow University between August 2019 and April 2023 were analyzed. Participants were categorized into a trial group receiving HMA and a control group. Further classification was based on HMA courses:≥4 and <4 courses. The efficacy and safety of HMA preventive treatment after allo-HSCT were evaluated. Survival analysis was performed using the Kaplan-Meier method with Log-Rank testing, the Fine-Gray model was used to assess cumulative relapse rates and Cox regression was used to identify prognostic factors. Adverse events during HMA were descriptively analyzed.Results:Among 173 patients, there were 100 males (57.8%) and 73 females (42.2%), with the age of 81 (34,127) months. The starting time of HMA was 123 (91, 191) d post-transplant, continuing 4.0 (3.0, 6.5) courses and the follow-up period was 24 (13, 32) months. The trial group (53 cases) showed better 2-year overall survival (OS) rate ((88.6±5.6)% vs. (76.6±4.3)%, χ 2=5.00, P=0.025) and relapse-free survival (RFS) rate ((89.2±4.7)% vs. (56.2±4.8)%, χ 2=15.75, P<0.001) than control group (120 cases). The 2-year OS rates and RFS rates were similar between ≥4 courses group (31 cases) and <4 courses group (22 cases)(both P>0.05). The cumulative relapse rate in the trial group was significantly lower ((10.8±0.2)% vs. (35.2±0.2)%, χ 2=10.84, P=0.001) than control group. Among children with molecular relapse, 8 cases (8/30, 26.7%) in the control group had hematological relapse compared to 1 case (1/2) in the trial group ( χ 2=0.81, P=0.369). The differences in incidence of acute and chronic graft-versus-host disease (GVHD) were not statistically significant (all P>0.05). Cox regression analysis revealed that minimal residual disease (MRD) positivity detected by flow cytometry before allo-HSCT and chronic GVHD were independent risk factors for OS (both P<0.05).The HMA preventive treatment was an independent protective factor for RFS, while age ≥10 years and MRD positivity detected by PCR before allo-HSCT were independent risk factors for RFS (all P<0.05). In trial group, 38 cases experienced grade 3 to 4 adverse events (71.7%). Conclusion:HMA is safe as preventive treatment in post-transplant children with high-risk AML, which can reduce the relapse risk and doesn't increase the risk of GVHD.

OBJECTIVES: Augmented renal clearance (ARC), in contrast to renal dysfunction, refers to enhanced renal elimination of circulating solutes compared to the expected baseline. Although patients may present with normal serum creatinine (Scr) levels, the incidence of ARC is high in intensive care unit (ICU) settings. ARC is associated with subtherapeutic exposure and treatment failure of renally cleared antibiotics. However, limited research exists on the incidence and risk factors of ARC in the ICU, and even fewer data are available specifically for neurological ICU (NICU). This study aims to determine the incidence and risk factors of ARC in neurocritically ill patients. METHODS: We retrospectively analyzed all available Scr data of neurocritical care patients admitted to the NICU of the Second Xiangya Hospital of Central South University between December 2020 and January 2023. Creatinine clearance (CrCl) was calculated using the Cockcroft-Gault equation. ARC was defined as a CrCl≥130 mL/(min·1.73 m²) sustained for more than 50% of the duration of the NICU stay. A total of 208 neurocritically ill patients were assigned into an ARC group (n=52) and a non-ARC (N-ARC) group (n=156). Clinical characteristics were compared between the 2 groups. Variables with P<0.05 in univariate analysis were included in binary Logistic regression to identify independent risk factors for ARC. RESULTS: The incidence of ARC among neurocritically ill patients was 25.00%. Of the 74 patients with normal CrCl, 20 (27.03%) gradually developed ARC during hospitalization. Compared with the N-ARC group, the patients of the ARC group were younger (P<0.001), with a higher proportion of females (P=0.048) and a lower admission mean arterial pressure (MAP) (P=0.034). Moreover, patients of the ARC group were commonly complicated with severe bacterial infections compared with the patients of the N-ARC group (P<0.001). In binary Logistic regression analysis, younger age (OR=0.903, 95% CI 0.872 to 0.935) and severe bacterial infections (OR=6.270, 95% CI 2.568 to 15.310) were significant predictors of ARC. CONCLUSIONS ARC is relatively common in the NICU. A considerable number of patients with initially normal renal function developed ARC during hospitalization. Younger age and concurrent severe bacterial infection are important risk factors of ARC in neurocritically ill patients.
Humans ; Risk Factors ; Female ; Male ; Retrospective Studies ; Creatinine/blood* ; Middle Aged ; Intensive Care Units ; Aged ; Adult ; Incidence ; Critical Illness ; Renal Elimination ; Kidney/metabolism*

Objective:To understand the current situation of robot-assisted patient rehabilitation training in neurological disease-related departments of ClassⅢ medical institutions across the country, so as to provide guidance and basis for promoting the improvement of robot-assisted training.Methods:In September 2021, convenience sampling was used to select neurological disease-related departments in Class Ⅲ hospitals from all provinces and cities across the country to conduct a survey on the status of robot-assisted rehabilitation training. A self-designed Hospital Robot-Assisted Training Questionnaire was used for investigation. The survey was conducted using an electronic questionnaire. A total of 250 questionnaires were distributed, and 236 valid questionnaires were recovered, with a valid recovery rate of 94.4%.Results:Among the 236 neurological disease-related departments, a total of 93 (39.4%) departments used robot-assisted rehabilitation training. The robot type was mainly wearable robot, which was mainly used to train lower limb motor function, upper limb motor function, gait ability and so on. A total of 55.9% (52/93) of departments believed that robot-assisted rehabilitation training was more effective than conventional rehabilitation training. Hospital type, department bed turnover times, and daily training time were the main factors affecting the effectiveness of robot-assisted training, and the differences were statistically significant ( P<0.05) . Among the 93 departments that had used robots, 22.6% (21/93) of the departments had not experienced any negative events in the robot-assisted rehabilitation training. The 143 departments that had not yet used robots had a positive attitude towards robots, and the main reasons for not using robots were that robots were too expensive, lacked the guidance of technicians, and were afraid to use them without professional training. Conclusions:Robot-assisted training has been concerned and tried to use by neurological disease-related departments, but the safety still needs to be further enhanced. Medical institutions should formulate robot-assisted rehabilitation training guidelines as soon as possible to guide the application and development of robot-assisted training.

Objective:To summarize the best evidence for the prevention and management of aspiration pneumonia in elderly patients to provide a basis for clinical nursing work.Methods:We systematically searched the domestic and foreign guide nets, websites of relevant institutions and databases on the relevant evidence for the prevention and management of aspiration pneumonia, including guidelines, expert consensus, systematic reviews, randomized controlled trials (RCT) , and the search time limit was from January 1, 2010, to June 30, 2020, and the language was not limited. Two researchers evaluated the quality of the included literature, and extracted, analyzed, and integrated evidence that met the quality evaluation criteria.Results:A total of 8 guidelines, 5 expert consensus, 10 systematic reviews, and 19 RCTs were included. The evidence was collected and extracted to form a prevention and management strategy for elderly patients with aspiration pneumonia including 11 first-level indicators and 32 second-level indicators.Conclusions:Medical and nursing staff should take effective measures in risk assessment, posture management, oral management, airway management, medication management, functional exercise and so on to reduce the risk of aspiration. Medical and nursing staff should also strengthen training for their own, elderly patients and main caregivers, and reduce the incidence of aspiration in elderly patients by applying the best evidence, so as to reduce the incidence of aspiration pneumonia.

Objective:To summarize the best evidence for prevention and management of aspiration in neurosurgical ICU patients and provide evidence for clinical nursing work.Methods:Relevant evidence on prevention and management of aspiration in neurosurgical ICU patients, including guidelines, consensus, system reviews, and randomized controlled trials (RCTs) which were published from 1st January, 2010 to 30th June, 2019 in any language was retrieved from guideline websites, relevant websites and databases. Two researchers evaluated the quality of the included literature and performed evidence extraction on those literatures that met the quality standards.Results:Totally 9 clinical practice guidelines, 8 expert consensus, 13 Meta analysis / systematic reviews and 10 RCTs were included, which were collected and extracted to form the final version of best evidence for aspiration prevention and management including 10 primary indicators and 29 secondary indicators.Conclusions:Medical staff should take effective measures in early assessment of aspiration risks, artificial airway management, position management, enteral nutrition management, sedation and analgesia management, etc., and reduce the incidence of aspiration in neurosurgical ICU patients by applying the best evidence. In addition, hospitals should provide various forms of education and training to medical staff, form transdisciplinary cooperation teams, and strengthen the prevention and management of aspiration.

Objective To explore how much impact that the educational level may exert on the Montreal Cognitive Assessment Scale (MoCA) for screening mild cognitive impairment (MCI) among the old people.Methods 3 230 participants aged 60+ years in 6 districts of Xiamen were selected using multi-stage stratified randomized sampling.One-way ANOVA and Scheffe were performed using SPSS 23.0 to compare the MoCA scores among different educational level groups.Binary logistic regression was used to analyze differential item functioning (DIF).If the P＜0.000 3 125 (Bonferroni correction),the corresponding item is considered to have DIF.Results The educational level of the elderly in Xiamen is generally lower,with 39.63％(1 133/2 858) illiterate and 28.04％(801/2 858) educated only at primary school,respectively.MoCA scores of elderly with different educational backgrounds were quite different with statistical significance(x2=413.73,P＜0.01),but Scheffe showed that there was no significant difference in MoCA scores between high school educated and undergraduate.Age,gender and MoCA scores of the elderly were significantly correlated (P＜0.01).The items that had DIF included:trail making test,copy cube,numbers of clock,hands of clock,abstraction 1 and 2,and word 1,2,3,and 5 of delayed recall,adding up to 11 items.The items of trail making test and copy cube only have DIF in the people above junior high school education level,while the 2 items of abstraction dimension only show DIF in the people above senior high school education level.Conclusion Most items of MoCA scale are suitable for screening people with all educational levels except when applying trail making test,copy cube and the 2 items of abstract dimension.

Objective To explore how much impact that the educational level may exert on the Montreal Cognitive Assessment Scale (MoCA) for screening mild cognitive impairment (MCI) among the old people.Methods 3 230 participants aged 60+ years in 6 districts of Xiamen were selected using multi-stage stratified randomized sampling.One-way ANOVA and Scheffe were performed using SPSS 23.0 to compare the MoCA scores among different educational level groups.Binary logistic regression was used to analyze differential item functioning (DIF).If the P＜0.000 3 125 (Bonferroni correction),the corresponding item is considered to have DIF.Results The educational level of the elderly in Xiamen is generally lower,with 39.63％(1 133/2 858) illiterate and 28.04％(801/2 858) educated only at primary school,respectively.MoCA scores of elderly with different educational backgrounds were quite different with statistical significance(x2=413.73,P＜0.01),but Scheffe showed that there was no significant difference in MoCA scores between high school educated and undergraduate.Age,gender and MoCA scores of the elderly were significantly correlated (P＜0.01).The items that had DIF included:trail making test,copy cube,numbers of clock,hands of clock,abstraction 1 and 2,and word 1,2,3,and 5 of delayed recall,adding up to 11 items.The items of trail making test and copy cube only have DIF in the people above junior high school education level,while the 2 items of abstraction dimension only show DIF in the people above senior high school education level.Conclusion Most items of MoCA scale are suitable for screening people with all educational levels except when applying trail making test,copy cube and the 2 items of abstract dimension.