1.Exploring Vascular Recruitment in Tracheobronchial Adenoid Cystic Carcinoma from Perspective of Abnormal Collateral
Jun TENG ; Lei LI ; Junyan XIA ; Yi LUO ; Qinyan HONG ; Shuiping CHEN ; Hongwu WANG
Chinese Journal of Experimental Traditional Medical Formulae 2025;31(6):260-269
Tracheobronchial adenoid cystic carcinoma (TACC) is a low-grade malignant tumor originating from the airway mucosa. Despite its slow progression,it is characterized by high invasiveness,frequent recurrence,and a strong tendency for metastasis. Preclinical studies have shown that vascular-targeted therapy holds significant potential. However,an effective systemic treatment for TACC has not been established yet. This study explored TACC from the perspective of "Feiji" in traditional Chinese medicine (TCM) as the starting point. It deeply investigated the mechanisms of abnormal collaterals and tumor vascular recruitment and further elaborated on the theoretical connection between abnormal collaterals and tumor vascular recruitment. Firstly,collateral hyperactivity led to disordered and erratic pulmonary collaterals. Their abnormal structures were similar to the disorderly and tortuous nature of tumor (pseudo)angiogenesis. This resulted in imbalances in the functions of circulation,perfusion,and reverse injection of the pulmonary collaterals,and then led to unrestrained collateral dysfunction and the accumulation of pathogenic factors. Secondly,the remodeling of the extracellular matrix (ECM) and epithelial-mesenchymal transition (EMT) in TACC were critical processes in vascular co-option (VCO),representing the micro-level manifestation of the displacement of nutrient and defense. During this process,ECM remodeling made TACC cells more likely to hijack normal blood vessels,creating a complex vascular microenvironment conducive to tumor growth. In terms of treatment,this study proposed a TCM strategy of "regulating collaterals to expel pathogenic factors and nourishing collaterals to strengthen the healthy Qi",and listed potential TCM. These were intended to regulate the Qi and blood in the collaterals,repair the functions of abnormal collaterals,and intervene in the vascular recruitment process of TACC. Future research should focus on improving the TCM clinical syndrome characteristics of TACC. Through modern molecular biology techniques,it is necessary to deeply analyze the micro-level pattern of vascular recruitment in TACC. This would enrich the understanding of the profound connection between abnormal collaterals and tumor vascular recruitment,providing empirical evidence for TCM-targeted therapies for vascular recruitment in TACC.
2.Effects of Yunpi Tongchang Formula on intestinal mucosal barrier damage via TLR4/MyD88/NF-κB signaling pathway in rats with opioid-induced constipation of Spleen-Kidney Yang Deficiency Syndrome
Lu-mei ZHANG ; Zhi-ming ZHANG ; Zhong-yang SONG ; Xin WANG ; Qian XU ; Xia YANG ; Xin-yu LI ; Yan-yun SHEN ; Hai-hong ZHAO ; Zhi-gang WANG
Chinese Traditional Patent Medicine 2025;47(7):2205-2212
AIM To investigate the effects of Yunpi Tongchang Formula on intestinal mucosal barrier damage in rats with opioid-induced constipation(OIC)of Spleen-Kidney Yang Deficiency Syndrome.METHODS In contrast to the 10 rats of the blank group,the 50 rats of the modeling group were induced into models of OIC of Spleen-Kidney Yang Deficiency Pattern by 7 days consecutive administration of both subcutaneous loperamide injection and alternating gavage of activated carbon ice water and vinegar.Following successful modeling,rats were randomly allocated into the model group,the mosapride citrate tablet group(1.35 mg/kg),and the high-dose,medium-dose,and low-dose Yunpi Tongchang Formula groups(15.12,7.56,3.78 g/kg),with 8 mice in each group.Upon the completion of the 14 days treatment,the rats had their TCM Syndrome scores assessed;their fecal water content,initial black stool excretion time,and small intestine propulsion rate measured;their colon tissue morphology observed by HE staining;their serum levels of IL-6,TNF-α,and IL-1β detected by ELISA;their expressions of occludin and zonula occludens-1(ZO-1)in colon tissues detected by immunohistochemistry;their mRNA expressions of MyD88,TLR4 and NF-κB p65 in the colon tissues detected by RT-qPCR;and their protein expressions of MyD88,TLR4 and NF-κB p65 in the colon tissues detected by Western blot.RESULTS Compared to the blank group,the model group had higher TCM Syndrome scores(P<0.01);lower fecal water content and small intestine propulsion rate(P<0.05,P<0.01);longer initial black stool excretion time(P<0.01);more mucosal edema in colon tissue,obvious inflammatory infiltration,and glandular disorder;increased serum levels of IL-6,TNF-α and IL-1 β(P<0.05);decreased colon expressions of ZO-1 and occludin(P<0.01);and increased mRNA and protein expressions of TLR4,MyD88 and NF-κB p65(P<0.01).Compared to the model group,both the medium-dose Yunpi Tongchang Formula group and the mosapride citrate tablet group demonstrated effectively reduced TCM syndrome scores(P<0.01);increased fecal water content and small intestine propulsion rate(P<0.05,P<0.01);and shorter initial black stool excretion time(P<0.01);improved colon mucosal edema and inflammatory infiltration;decreased serum levels of IL-6,TNF-α and IL-1β(P<0.01);upregulated protein expressions of ZO-1 and occludin(P<0.01);and downregulated mRNA and protein expressions of TLR4,MyD88 and NF-κB p65(P<0.05,P<0.01).CONCLUSION Yunpi Tongchang Formula significantly ameliorates constipation symptoms in OIC rat models of Spleen-Kidney Yang Deficiency Syndrome because of its efficacy in attenuating intestinal inflammation and preserving the integrity of intestinal epithelial barrier structure,with its mechanistic action in downregulating TLR4/MyD88/NF-κB signaling pathway activation.
3.Consensus on informed consent for orthodontic treatment
Yang CAO ; Bing FANG ; Zuolin JIN ; Hong HE ; Yuxing BAI ; Lin WANG ; Haiping LU ; Zhihe ZHAO ; Tianmin XU ; Weiran LI ; Min HU ; Jinlin SONG ; Jun WANG ; Fang JIN ; Ding BAI ; Xianglong HAN ; Yuehua LIU ; Bin YAN ; Jie GUO ; Jiejun SHI ; Yongming LI ; Zhihua LI ; Xiuping WU ; Jiangtian HU ; Linyu XU ; Lin LIU ; Yi LIU ; Yanqin LU ; Wensheng MA ; Shuixue MO ; Liling REN ; Shuxia CUI ; Yongjie FAN ; Jianguang XU ; Lulu XU ; Zhijun ZHENG ; Peijun WANG ; Rui ZOU ; Chufeng LIU ; Lunguo XIA ; Li HU ; Weicai WANG ; Liping WU ; Xiaoxing KOU ; Jiali TAN ; Yuanbo LIU ; Bowen MENG ; Yuantao HAO ; Lili CHEN
Chinese Journal of Stomatology 2025;60(12):1327-1336
This consensus was developed by the Orthodontic Society of the Chinese Stomatological Association to provide a systematic, scientific, and practical guideline for informed consent in orthodontic care. Orthodontic treatment is typically lengthy, highly individualized, and involves multiple factors such as growth and development, occlusal function, and facial esthetics. Rapid technological advances and diverse risk profiles make the traditional reliance on orthodontist experience or institutional templates insufficient to ensure patients′ full understanding and autonomous decision-making. To address this, the expert panel conducted extensive reviews of domestic and international guidelines, analyzed representative dispute cases, and performed multicenter patient-clinician surveys. Using a multi-round Delphi method, the group established a standardized informed consent framework covering the initial consultation, treatment, and retention phases. The consensus emphasizes that informed consent is not only a fundamental legal and ethical requirement but also a key step in building trust, improving patient compliance, and enhancing treatment satisfaction. Orthodontists should clearly and comprehensively explain treatment plans, potential risks, uncertainties, and associated costs, while respecting the autonomy of patients or guardians, and maintain continuous communication and dynamic evaluation throughout the treatment process. The release of this consensus provides unified and authoritative guidance for clinical orthodontics, helping to standardize informed consent, enhance its transparency, safeguard patient rights, reduce medical risks, and promote high-quality, sustainable development of orthodontic practice.
4.Guideline for Adult Weight Management in China
Weiqing WANG ; Qin WAN ; Jianhua MA ; Guang WANG ; Yufan WANG ; Guixia WANG ; Yongquan SHI ; Tingjun YE ; Xiaoguang SHI ; Jian KUANG ; Bo FENG ; Xiuyan FENG ; Guang NING ; Yiming MU ; Hongyu KUANG ; Xiaoping XING ; Chunli PIAO ; Xingbo CHENG ; Zhifeng CHENG ; Yufang BI ; Yan BI ; Wenshan LYU ; Dalong ZHU ; Cuiyan ZHU ; Wei ZHU ; Fei HUA ; Fei XIANG ; Shuang YAN ; Zilin SUN ; Yadong SUN ; Liqin SUN ; Luying SUN ; Li YAN ; Yanbing LI ; Hong LI ; Shu LI ; Ling LI ; Yiming LI ; Chenzhong LI ; Hua YANG ; Jinkui YANG ; Ling YANG ; Ying YANG ; Tao YANG ; Xiao YANG ; Xinhua XIAO ; Dan WU ; Jinsong KUANG ; Lanjie HE ; Wei GU ; Jie SHEN ; Yongfeng SONG ; Qiao ZHANG ; Hong ZHANG ; Yuwei ZHANG ; Junqing ZHANG ; Xianfeng ZHANG ; Miao ZHANG ; Yifei ZHANG ; Yingli LU ; Hong CHEN ; Li CHEN ; Bing CHEN ; Shihong CHEN ; Guiyan CHEN ; Haibing CHEN ; Lei CHEN ; Yanyan CHEN ; Genben CHEN ; Yikun ZHOU ; Xianghai ZHOU ; Qiang ZHOU ; Jiaqiang ZHOU ; Hongting ZHENG ; Zhongyan SHAN ; Jiajun ZHAO ; Dong ZHAO ; Ji HU ; Jiang HU ; Xinguo HOU ; Bimin SHI ; Tianpei HONG ; Mingxia YUAN ; Weibo XIA ; Xuejiang GU ; Yong XU ; Shuguang PANG ; Tianshu GAO ; Zuhua GAO ; Xiaohui GUO ; Hongyi CAO ; Mingfeng CAO ; Xiaopei CAO ; Jing MA ; Bin LU ; Zhen LIANG ; Jun LIANG ; Min LONG ; Yongde PENG ; Jin LU ; Hongyun LU ; Yan LU ; Chunping ZENG ; Binhong WEN ; Xueyong LOU ; Qingbo GUAN ; Lin LIAO ; Xin LIAO ; Ping XIONG ; Yaoming XUE
Chinese Journal of Endocrinology and Metabolism 2025;41(11):891-907
Body weight abnormalities, including overweight, obesity, and underweight, have become a dual public health challenge in Chinese adults: overweight and obesity lead to a variety of chronic complications, while underweight increases the risks of malnutrition, sarcopenia, and organ dysfunction. To systematically address these issues, multidisciplinary experts in endocrinology, sports science, nutrition, and psychiatry from various regions have held multiple weight management seminars. Based on the latest epidemiological data and clinical evidence, they expanded the guideline to include assessment and intervention strategies for underweight, in addition to the core content of obesity management. This guideline outlines the etiological mechanisms, evaluation methods, and multidimensional management strategies for overweight and obesity, covering key areas such as diagnosis and assessment, medical nutrition therapy, exercise prescription, pharmacological intervention, and psychological support. It is intended to provide a scientific and standardized approach to weight management across the adult population, aiming to curb the rising prevalence of obesity, mitigate complications associated with abnormal body weight, and improve nutritional status and overall quality of life.
5.Role and mechanism of miR-183 targeting PTEN to regulate the AKT signaling pathway in the progression of diabetic nephropathy
Yaping XIE ; Xiu WANG ; Ying YE ; Hong XIA ; Feng YU ; Shuhong ZHANG ; Ying XIONG
Chinese Journal of Nephrology 2025;41(10):749-758
Objective:To investigate the mechanism by which microRNA-183 (miR-183) regulates the progression of diabetic nephropathy (DN) through targeting phosphatase and tensin homolog deleted on chromosome 10 (PTEN) and modulating the AKT signaling pathway, and to identify potential therapeutic targets for DN.Methods:(1) Bioinformatic analysis of miRNA expression: MiRNA expression datasets from diabetic nephropathy (DN) and control samples were obtained from the Gene Expression Omnibus database. Differential expression analysis was performed, and differentially expressed miRNAs (DEmiRNAs) were identified using thresholds of an absolute log 2 (fold changes) >1 and an adjusted P-value<0.05. The results were visualized in a volcano plot and a heatmap. (2) Animal model establishment and in vivo interventional studies: A DN rat model was induced by administration of a high-fat/high-sucrose diet combined with an intraperitoneal injection of streptozotocin. Rats were randomly assigned into four groups ( n=10 per group) using a random number table: control group, DN model group, miR-183 inhibitor negative control (NC) group, and miR-183 inhibitor group. The latter two groups received tail vein injections of the miR-183 inhibitor NC or the miR-183 inhibitor, respectively, for eight consecutive weeks. Parameters including fasting blood glucose, 24-hour urinary protein excretion, urinary albumin excretion rate (UAER), serum creatinine, and blood urea nitrogen (BUN) were measured. Renal histopathological changes were assessed by HE and PAS staining. Furthermore, the expression of candidate miRNAs from patient data was validated, and the mechanism of action of miR-183 was investigated using quantitative real-time PCR and Western blotting. (3) In vitro mechanistic investigations in cultured podocytes: Mouse podocyte clone-5 (MPC5) cells were cultured in vitro and subjected to the following conditions: normal glucose (5.3 mmol/L glucose), high glucose (30 mmol/L glucose), and osmotic control (5.3 mmol/L glucose+19.5 mmol/L mannitol). Cells in the logarithmic growth phase were transfected with the miR-183 inhibitor (100 nmol/L), miR-183 mimic (50 nmol/L), or their corresponding negative controls. A dual-luciferase reporter assay was conducted to validate the binding interaction between miR-183 and the 3'-untranslated region (3'UTR) of PTEN. The effects of miR-183 on the AKT signaling pathway, apoptosis-related proteins, and cell viability were evaluated by quantitative real-time PCR, Western blotting, and the cell counting kit-8 assay, respectively. Results:MiR-183 expression was markedly upregulated in renal tissues from DN patients and DN model rats (both P<0.05). Inhibition of miR-183 significantly reduced renal miR-183 levels by 90.2% ( P<0.01), decreased fasting blood glucose by 65.3% ( P<0.01), and improved renal function parameters, including reductions in urinary protein (40.3%), blood urea nitrogen (32.1%), urinary albumin excretion rate (22.5%), and serum creatinine (40.2%) (all P<0.01). Histological analyses showed attenuation of glomerular lesions and glycogen accumulation. Bioinformatic prediction and experimental validation identified PTEN as a direct target of miR-183, confirmed by dual-luciferase assays. In vitro, miR-183 inhibition increased PTEN expression, reduced AKT phosphorylation, promoted podocyte proliferation, and suppressed apoptosis (upregulation of Bcl-2 and downregulation of cleaved-caspase-3). These effects were abolished upon PTEN knockdown. Conclusions:miR-183 aggravates DN by targeting PTEN and activating the AKT signaling pathway. Inhibition of miR-183 improves renal function and reduces podocyte apoptosis, suggesting miR-183 as a potential therapeutic target for DN.
6.Analysis of rapeutic effect of musculoskeletal ultrasound-guided acupuncture for the treatment of myofascial pain syndrome after rotator cuff suture under shoulder arthroscopy.
Xiao LIANG ; Jing WANG ; Xiao-Hui XIA ; Hong-Tao WANG
China Journal of Orthopaedics and Traumatology 2025;38(6):601-607
OBJECTIVE:
To compare clinical efficacy of musculoskeletal ultrasound-guided acupuncture treatment and radiation extracorporeal shock wave therapy in myofascial pain syndrome after rotator cuff suture under shoulder arthroscopy.
METHODS:
From June 2021 to April 2022, 75 patients with myofascial pain syndrome after rotator cuff suture under shoulder arthroscopy were admitted and divided into musculoskeletal ultrasound group and extracorporeal shock wave group according to different treatment methods. There were 39 patients in musculoskeletal ultrasound group, including 12 males and 27 females, aged from 43 to 77 years old with an average of (56.33±9.45) years old;11 patients on the left side and 28 patients on the right side;the course of disease with a median of 7.00(4.00, 12.00) weeks;acupuncture treatment was performed under the guidance of musculoskeletal ultrasound. There were 36 patients in extracorporeal shock wave group, including 16 males and 20 females, aged from 46 to 72 years old with a median of (58.94±8.94) years old;12 patients on the left side and 24 patients on the right side;the course of disease with an average of 5.50(4.00, 8.00) weeks;extracorporeal shock wave therapy with radiation were performed. Visual analogue scale (VAS) and American shoulder and elbow surgeons score (ASES) were compared between two groups to evaluate improvement of shoulder joint pain and function before treatment and 1, 3, 6, 12, and 24 months after treatment.
RESULTS:
Both of two groups were followed up for 24 to 27 months with an average of (24.68±0.89) months. No complications such as infection and vascular and nerve injury occurred during follow-up period. At 6 months after treatment, VAS of musculoskeletal ultrasound group (2.00±1.19) was lower than that of extracorporeal shock wave group (3.08±1.02), and the difference was statistically significant (P<0.05). At 1, 3, 12 and 24 months after treatment, there were no statistically significant difference in VAS between two groups (P>0.05). At 3, 6 and 12 months after treatment, ASES scores of musculoskeletal ultrasound group were (77.44±11.56), (86.06±6.11), and (89.44±4.66) respectively, which were higher than those of extracorporeal shock wave group (55.23±12.76), (58.10±10.25), (84.03±7.36), the differences were statistically significant (P<0.05);there were no statistically significant difference in ASES between two groups at 1 and 24 months after treatment(P>0.05).
CONCLUSION
Musculoskeletal ultrasound-guided acupuncture treatment has advantages of faster pain relief and more rapid improvement of shoulder joint function for myofascial pain syndrome after rotator cuff suture under shoulder arthroscopy compared with radioactive extracorporeal shock wave therapy.
Humans
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Male
;
Female
;
Middle Aged
;
Acupuncture Therapy/methods*
;
Adult
;
Aged
;
Arthroscopy/adverse effects*
;
Myofascial Pain Syndromes/etiology*
;
Ultrasonography
;
Rotator Cuff/surgery*
7.Preliminary efficacy observation of 3D printed functional spinal external fixation brace combined with McKenzie therapy in the treatment of lumbar disc herniation.
Ning-Xia WANG ; Ping CHEN ; Hai-Dong WANG ; Jing JI ; Fang-Hong NIAN ; Xin LIU ; Chong-Fei JIN ; Duo-Ming ZHAO ; Hao-Lin LI ; Wei-Gang CHENG ; Gui-Lin LAI ; Guo-Biao WU
China Journal of Orthopaedics and Traumatology 2025;38(10):1047-1054
OBJECTIVE:
To observe the clinical efficacy of 3D printing spinal external fixator combined with McKenzie therapy for patients with lumbar dics herniation (LDH).
METHODS:
Sixty patients with LDH between January 2022 and January 2023 were enrolled. Among them, 30 patients were given McKinsey training. According to different treatment methods, all patients were divided into McKenzie group and McKenzie + 3D printing group, 30 patients in each group. The McKenzie group provided McKenzie therapy. The McKenzie + 3D printing group were treated with 3D printing spinal external fixation brace on the basis of McKenzie therapy. Patients in both groups were between 25 and 60 years of age and had their first illness. In the McKenzie group, there were 19 males and 11 females, with an average age of (48.57±5.86) years old, and the disease duration was (7.03 ±2.39) months. The McKenzie + 3D printing group, there were 21 males and 9 females, with an average age of (48.80±5.92) years old, and the disease duration was(7.30±2.56) months. Pain was evaluated using the visual analogue scale (VAS), and lumbar spine function was assessed using the Oswestry disability index (ODI) and the Japanese Orthopaedic Association (JOA) score. VAS, ODI and JOA scores were compared between two groups before treatment and at 1, 3, 6, 9 and 12 months after treatment.
RESULTS:
All patients were followed up for 12 months. The VAS for the McKenzie combined with 3D printing group before treatment and at 1, 3, 6, 9, and 12 months post-treatment were(6.533±0.860), (5.133±1.008), (3.933±0.868), (2.900±0.759), (2.067±0.640), (1.433±0.504), respectively. In the McKenzie group, the corresponding scores were (6.467±0.860), (5.067±1.048), (4.600±0.968), (3.533±1.008), (2.567±0.728), (1.967±0.809), respectively. The ODI of the McKenzie group before treatment and at 1, 3, 6, 9, and 12 months post-treatment were (41.033±6.810)%, (37.933±6.209)%, (35.467±6.962)%, (27.567±10.081)%, (20.800±7.531)%, (13.533±5.158)%, respectively. For the McKenzie combined with 3D printing group, the corresponding ODI were(38.033±5.605)%, (33.000±6.192)%, (28.767±7.045)%, (22.200±5.517)%, (17.700±4.836)%, (11.900±2.771)%, respectively. The JOA scores of the McKenzie combined with 3D printing group before treatment and at 1, 3, 6, 9, and 12 months post-treatment were(8.900±2.074), (13.133±2.330), (15.700±3.583), (20.400±3.480), (22.267±3.084), (24.833±2.640), respectively. In the McKenzie group, the corresponding scores were(9.200±2.091), (12.267±2.406), (15.333±3.198), (18.467±2.240), (20.133±2.751), (22.467±2.849), respectively. Before the initiation of treatment, no statistically significant differences were observed in the VAS, ODI, and JOA scores between two groups (P>0.05). At 3, 6, 9, and 12 months post-treatment, the VAS in the McKenzie combined with 3D printing group was significantly lower than that in the McKenzie group, and the difference was statistically significant (P<0.05). The comparison of ODI between two groups at 1, 3, 6, 9, and 12 months post-treatment revealed statistically significant differences (P<0.05). At 6, 9, and 12 months post-treatment, the JOA score in the McKenzie combined with 3D printing group was significantly higher than that in the McKenzie-only group, and the difference was statistically significant (P<0.05).
CONCLUSION
The combination of 3D printed functional spinal external fixation brace with McKenzie therapy can significantly improve and maintain lumbar function in patients with LDH.
Humans
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Male
;
Female
;
Middle Aged
;
Printing, Three-Dimensional
;
Intervertebral Disc Displacement/surgery*
;
External Fixators
;
Lumbar Vertebrae/surgery*
;
Adult
;
Braces
;
Treatment Outcome
8.Clinical analysis of nutritional components in children with acute leukemia.
Li-Xia SHI ; Ming-Zhong ZHAO ; Fei-Fei WANG ; Yu-Qian XING ; Hong-Yan JI ; Ping ZHAO
Chinese Journal of Contemporary Pediatrics 2025;27(3):300-307
OBJECTIVES:
To assess the changes in body composition and nutritional risks faced by children with different stages of acute leukemia (AL).
METHODS:
Bioelectrical impedance analysis combined with anthropometric measurements was used to detect body composition. This prospective study was conducted from August 2023 to July 2024 at Shandong Provincial Hospital, examining the body composition and physical balance of children with various stages of AL and healthy children.
RESULTS:
The non-fat components of children with AL and healthy children both showed a linear increase with age. In the younger age group, there were no significant differences in body composition between children with AL and healthy children. However, in the older age group, the body composition of children undergoing chemotherapy for AL was significantly lower than that of healthy children (P<0.05), and muscle mass recovered first after the completion of AL chemotherapy. The proportion of children with increased trunk fat in AL children who completed chemotherapy was significantly lower than that in healthy children (P<0.05), while the incidence rate of severe left-right imbalance in body composition was significantly higher (P<0.05). Muscle distribution in children with AL primarily showed insufficient limb and overall muscle mass, whereas healthy children mainly exhibited insufficient upper limb muscle mass.
CONCLUSIONS
The body composition of children with AL varies at different treatment stages, indicating that nutritional status is affected by both the disease itself and the treatment. Early screening can provide a basis for reasonable nutritional intervention.
Humans
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Child
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Male
;
Female
;
Child, Preschool
;
Body Composition
;
Prospective Studies
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Adolescent
;
Leukemia/metabolism*
;
Infant
;
Nutritional Status
;
Acute Disease
;
Electric Impedance
9.Thiotepa-containing conditioning for allogeneic hematopoietic stem cell transplantation in children with inborn errors of immunity: a retrospective clinical analysis.
Xiao-Jun WU ; Xia-Wei HAN ; Kai-Mei WANG ; Shao-Fen LIN ; Li-Ping QUE ; Xin-Yu LI ; Dian-Dian LIU ; Jian-Pei FANG ; Ke HUANG ; Hong-Gui XU
Chinese Journal of Contemporary Pediatrics 2025;27(10):1240-1246
OBJECTIVES:
To evaluate the safety and efficacy of thiotepa (TT)-containing conditioning regimens for allogeneic hematopoietic stem cell transplantation (HSCT) in children with inborn errors of immunity (IEI).
METHODS:
Clinical data of 22 children with IEI who underwent HSCT were retrospectively reviewed. Survival after HSCT was estimated using the Kaplan-Meier method.
RESULTS:
Nine patients received a traditional conditioning regimen (fludarabine + busulfan + cyclophosphamide/etoposide) and underwent peripheral blood stem cell transplantation (PBSCT). Thirteen patients received a TT-containing modified conditioning regimen (TT + fludarabine + busulfan + cyclophosphamide), including seven PBSCT and six umbilical cord blood transplantation (UCBT) cases. Successful engraftment with complete donor chimerism was achieved in all patients. Acute graft-versus-host disease occurred in 12 patients (one with grade III and the remaining with grade I-II). Chronic graft-versus-host disease occurred in one patient. The incidence of EB viremia in UCBT patients was lower than that in PBSCT patients (P<0.05). Over a median follow-up of 36.0 months, one death occurred. The 3-year overall survival (OS) rate was 100% for the modified regimen and 88.9% ± 10.5% for the traditional regimen (P=0.229). When comparing transplantation types, the 3-year OS rates were 100% for UCBT and 93.8% ± 6.1% for PBSCT (P>0.05), and the 3-year event-free survival rates were 100% and 87.1% ± 8.6%, respectively (P>0.05).
CONCLUSIONS
TT-containing conditioning for allogeneic HSCT in children with IEI is safe and effective. Both UCBT and PBSCT may achieve high success rates.
Humans
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Retrospective Studies
;
Transplantation Conditioning/methods*
;
Thiotepa/therapeutic use*
;
Hematopoietic Stem Cell Transplantation/adverse effects*
;
Male
;
Female
;
Child, Preschool
;
Infant
;
Child
;
Transplantation, Homologous
;
Graft vs Host Disease
;
Adolescent
10.Clinical Characteristics and Prognostic Analysis of Newly Diagnosed Acute Myeloid Leukemia Patients with NRAS and KRAS Gene Mutations.
Zhang-Yu YU ; Bo CAI ; Yi WANG ; Yang-Yang LEI ; Bing-Xia LI ; Yu-Fang LI ; Yan-Ping SHI ; Jia-Xin CHEN ; Shu-Hong LIU ; Chang-Lin YU ; Mei GUO
Journal of Experimental Hematology 2025;33(3):682-690
OBJECTIVE:
To retrospectively analyze the clinical characteristics, co-mutated genes in newly diagnosed acute myeloid leukemia (AML) patients with NRAS and KRAS gene mutations, and the impact of NRAS and KRAS mutations on prognosis.
METHODS:
The clinical data and next-generation sequencing results of 80 newly diagnosed AML patients treated at our hospital from December 2018 to December 2023 were collected. The clinical characteristics, co-mutated genes of NRAS and KRAS , and the impact of NRAS and KRAS mutations on prognosis in newly diagnosed AML patients were analyzed.
RESULTS:
Among 80 newly diagnosed AML patients, NRAS mutations were detected in 20 cases(25.0%), and KRAS mutations were detected in 9 cases(11.3%). NRAS mutations predominantly occurred at codons 12 and 13 of exon 2, as well as codon 61 of exon 3, while KRAS mutations were most commonly occurred at codons 12 and 13 of exon 2, all of which were missense mutations. There were no statistically significant differences observed in terms of age, sex, white blood cell count(WBC), hemoglobin(Hb), platelet count(PLT), bone marrow blasts, first induction chemotherapy regimen, CR1/CRi1 rates, chromosome karyotype, 2022 ELN risk classification and allogeneic hematopoietic stem cell transplantation(allo-HSCT) among the NRAS mutation group, KRAS mutation group and NRAS/KRAS wild-type group (P >0.05). KRAS mutations were significantly correlated with PTPN11 mutations (r =0.344), whereas no genes significantly associated with NRAS mutations were found. Survival analysis showed that compared to the NRAS/KRAS wild-type group, patients with NRAS mutation had a relatively higher 5-year overall survival (OS) rate and relapse-free survival (RFS) rate, though the differences were not statistically significant (P =0.097, P =0.249). Compared to the NRAS/KRAS wild-type group, patients with KRAS mutation had a lower 5-year OS rate and RFS rate, with no significant differences observed (P =0.275, P =0.442). There was no significant difference in the 5-year RFS rate between the KRAS mutation group and NRAS mutation group (P =0.157), but the 5-year OS rate of patients with KRAS mutation was significantly lower than that of patients with NRAS mutation (P =0.037).
CONCLUSION
In newly diagnosed AML patients, KRAS mutation was significantly correlated with PTPN11 mutation. Compared to patients with NRAS/KRAS wild-type, those with NRAS mutation showed a more favorable prognosis, while patients with KRAS mutation showed a poorer prognosis; however, these differences did not reach statistical significance. Notably, the prognosis of AML patients with KRAS mutation was significantly inferior compared to those with NRAS mutation.
Humans
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Leukemia, Myeloid, Acute/diagnosis*
;
Mutation
;
Prognosis
;
Proto-Oncogene Proteins p21(ras)/genetics*
;
GTP Phosphohydrolases/genetics*
;
Retrospective Studies
;
Membrane Proteins/genetics*
;
Female
;
Male
;
Middle Aged
;
Adult
;
Aged

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