1.Effect of Yang-Reinforcing and Blood-Activating Therapy on the Long-Term Prognosis for Dilated Cardio-myopathy Patients with Yang Deficiency and Blood Stasis Syndrome:A Retrospective Cohort Study
Shiyi TAO ; Jun LI ; Lintong YU ; Ji WU ; Yuqing TAN ; Xiao XIA ; Fuyuan ZHANG ; Tiantian XUE ; Xuanchun HUANG
Journal of Traditional Chinese Medicine 2026;67(1):53-59
ObjectiveTo evaluate the impact of yang-reinforcing and blood-activating therapy on the long-term prognosis for patients with dilated cardiomyopathy (DCM) of yang deficiency and blood stasis syndrome. MethodsA retrospective cohort study was conducted involving 371 DCM patients with yang deficiency and blood stasis syndrome. The yang-reinforcing and blood-activating therapy was defined as the exposure factor. Patients were categorized into exposure group (186 cases) and non-exposure group (185 cases) according to whether they received yang-reinforcing and blood-activating therapy combined with conventional western medicine for 6 months or longer. The follow-up period was set at 48 months, and the Kaplan-Meier survival analysis was used to assess the cumulative incidence of major adverse cardiovascular events (MACE) in both groups. Cox regression analysis was used to explore the impact of yang-reinforcing and blood-activating therapy on the risk of MACE, and subgroup analysis was performed. Changes in traditional Chinese medicine (TCM) syndrome score, left ventricular ejection fraction (LVEF), left ventricular fractional shortening (LVFS), left ventricular end-diastolic diameter (LVEDD), and Minnesota Living with Heart Failure Questionnaire (MLHFQ) score were compared between groups at the time of first combined use of yang-reinforcing and blood-activating therapy (before treatment) and 1 year after receiving the therapy (after treatment). ResultsMACE occurred in 31 cases (16.67%) in the exposure group and 47 cases (25.41%) in the non-exposure group. The cumulative incidence of MACE in the exposure group was significantly lower than that in the non-exposure group [HR=0.559, 95%CI(0.361,0.895), P=0.014]. Cox regression analysis showed that yang-reinforcing and blood-activating therapy was an independent factor for reducing the risk of MACE in DCM patients [HR=0.623, 95%CI(0.396,0.980), P=0.041], and consistent results were observed in different subgroups. Compared with pre-treatment, the exposure group showed decreased TCM syndrome score and MLHFQ score, reduced LVEDD, and increased LVEF and LVFS after treatment (P<0.05); in the non-exposure group, TCM syndrome score decreased, LVEF and LVFS increased, and LVEDD reduced after treatment (P<0.05). After treatment, the exposure group had higher LVEF and LVFS, smaller LVEDD, and lower TCM syndrome score and MLHFQ score compared with the non-exposure group (P<0.05). ConclusionCombining yang-reinforcing and blood-activating therapy with conventional western medicine can reduce the risk of MACE in DCM patients with yang deficiency and blood stasis syndrome, meanwhile improving their clinical symptoms, cardiac function, and quality of life.
2.Short-Term Efficacy and Long-Term Recurrence Rate of Traditional Chinese Medicine Versus Western Surgical Treatment for Mixed Hemorrhoids:A Multi-Center Retrospective Cohort Study Based on Real-World Data
Kang DING ; Zhimin FAN ; Xiaojie ZHOU ; Xiaoxiao WANG ; Yuanyuan GE ; Huiting ZHU ; Yuxin ZHU ; Xia YANG ; Jun DU ; Shicai HUANG ; Yang ZHANG
Journal of Traditional Chinese Medicine 2026;67(7):747-754
ObjectiveTo observe the short-term and long-term efficacy of traditional Chinese medicine (TCM) surgical operations in treating mixed hemorrhoids. MethodsA multi-center retrospective cohort study was conducted, collecting clinical data from 17,831 mixed hemorrhoid surgery patients in 8 top-tier TCM hospitals in Jiangsu Province. Standardized and structured datasets were obtained through artificial intelligence models. Patients who underwent western surgical treatment were categorized into the western surgery group (11,646 cases), and those receiving TCM surgical operations were categorized into the TCM surgery group (6185 cases). Propensity score matching (1∶1 matching) was used to balance baseline data between groups. The primary outcome was the one-year recurrence rate, and secondary outcomes included the main symptoms (rectal bleeding, degree of prolapse) and secondary symptoms (anal distension, anal edema, wound secretion and exudation, anal stenosis, residual skin tags, perianal itching, and anal pain) measured on days 7, 28, and 60 after discharge. ResultsAfter matching, 2194 patients were included in each group. Symptom scores showed that at 28 days after discharge, the TCM surgical group had superior improvement in rectal bleeding [OR=5.786, 95%CI (3.092,10.827)], degree of prolapse [OR=4.510, 95%CI (1.649,12.333)], and anal edema [OR=3.188, 95%CI (1.295,7.845)] compared to the western surgical group. At 60 days post-discharge, the TCM group still showed advantages in improving rectal bleeding [OR=5.237, 95%CI (1.077,25.464)] and anal pain [OR=11.697, 95%CI (1.186,115.336)] (P<0.05). Long-term follow-up showed that the one-year recurrence rate in the TCM surgery group was 1.1% (8/726), while that in the western surgery group was 2.3% (10/444), with no statistically significant difference between the two groups (P>0.05). ConclusionBased on real-world data, TCM surgical treatment for mixed hemorrhoids shows significant short-term symptom improvement, particularly in terms of hemostasis, reducing swelling, and alleviating prolapse of anal masses.
3.Expression and clinical significance of KIF15, EGFR, and HER2 in gallbladder cancer
Jun WANG ; Lubing WANG ; Gangfeng HU ; Bo ZHANG ; Xia HUANG ; Lei HUANG
Journal of Surgery Concepts & Practice 2025;30(5):409-416
Objective To investigate the expression of kinesin family member 15(KIF15), epidermal growth factor receptor(EGFR), and human epidermal growth factor receptor 2(HER2) in gallbladder cancer(GBC) and their clinical and pathological significance. Methods Immunohistochemical staining was employed to detect the expression of KIF15, EGFR, and HER2 proteins in GBC tissue microarrays. The correlation between protein expression levels and various clinical and pathological characteristics of GBC patients was analyzed. Results The positive expression rates of KIF15, EGFR, and HER2 proteins in GBC tissues were 71.6%, 62.2%, and 51.4% respectively, compared to 16.7%, 0, and 0 in para-carcinoma tissues (all P=0.000). KIF15 expression was correlated with cancer differentiation grade (P=0.006), while EGFR expression was associated with lymph node metastasis(P=0.026) and the number of metastatic lymph nodes(P=0.012). HER2 expression was correlated with cancer differentiation grade(P=0.018), tumor size(P=0.005), and T stage(P=0.032). GBC patients with co-positive expression of KIF15 and HER2 had significantly lower overall survival compared to those with negative expression. Positive HER2 expression, lymph node metastasis, and the number of metastatic lymph nodes were identified as independent risk factors for overall survival in GBC patients. Conclusions Positive expression of KIF15, EGFR, and HER2 in GBC tissues are correlated with various clinical indicators and poorer prognosis. These proteins may serve as potential factors for predicting prognosis and evaluating therapeutic efficacy in GBC.
4.Study on the Correlation between the Expression Level of Serum Developmental Endothelial Locus-1 and Inflammatory Response and Disease Severity in Patients with Chronic Obstructive Pulmonary Disease
Jun WANG ; Changming HUANG ; Xia WU ; Ran ZHANG
Journal of Modern Laboratory Medicine 2025;40(2):160-163,168
Objective To explore the relationship between developmental endothelial locus-1(Del-1)and inflammatory response and disease severity in patients with chronic obstructive pulmonary disease(COPD).Method 82 COPD patients admitted to Huai'an Fifth People's Hospital from January 2023 to July 2023 were selected as the COPD group.Among them,40 patients with acute exacerbation were included in the acute exacerbation group,and 42 patients in the stable phase were included in the stable group.Another 40 healthy volunteers who underwent physical examinations during the same period were selected as the control group.The levels of serum Del-1,C-reactive protein(CRP),interleukin-1β(IL-1β)and neutrophil to lymphocyte ratio(NLR)in all study subjects were detected.Pearson's method was used to analyze the correlation between serum Del-1 and inflammatory markers.The predictive value of serum Del-1 for acute exacerbation in COPD patients was analyzed using receiver operating characteristic(ROC)curves.Result The serum CRP,IL-1β and NLR levels in the COPD group were higher than those in the control group(t=12.047,13.107,9.404),the serum Del-1 level in the COPD group was 386.87±42.34 pg/ml,lower than that in the control group 512.63±60.21 pg/ml(t=13.343),and the differences were statistically significant(all P<0.05).The serum CRP,IL-1β and NLR levels in the acute exacerbation group were higher than those in the stable group(t=4.170,5.292,5.919),the serum Del-1 level in the acute exacerbation group was 347.64±40.69 pg/ml,lower than that in the stable group 424.23±48.31 pg/ml(t=7.746),and the differences were statistically significant(all P<0.05).Pearson analysis showed that the serum Del-1 level in COPD patients were negatively correlated with serum CRP,IL-1β and NLR levels(r=-0.534,-0.587,-0.612,all P<0.05).ROC analysis showed that serum Del-1 had a high predictive value for acute exacerbation in COPD patients,with an AUC(95%CI)of 0.886(0.812~0.960).When the optimal cutoffvalue was 380.65 pg/ml,the sensitivity and specificity were 82.50%and 85.00%,respectively.Conclusion Serum Del-1 is low expression in COPD patients and is ngeatively correlated with their inflammatory response and disease severity,it is expected to become a new biomarker for predicting the risk of acute exacerbation of COPD.
5.The Impact of Early Cleavage on Pregnancy and Obstetric Outcomes after Sin-gle Cleavage Stage Embryo and Single Blastocyst Transfer
Min YU ; Leizhen XIA ; Zhihui HUANG ; Qiongfang WU ; Jun TAN
Journal of Practical Obstetrics and Gynecology 2025;41(2):136-142
Objective:To investigate the impact of early cleavage on the pregnancy and obstetric outcomes of fresh cycle single cleavage embryo and single blastocyst transfer.Methods:A retrospective study was conducted to analyze 794 patients who underwent single cleavage embryo transfer and 1466 patients who underwent single blastocyst transfer in the reproductive medicine center of our hospital from August 2018 to December 2022 during the fresh cycle treatment of in vitro fertilization/intracytoplasmic sperm injection and embryo transfer(IVF/ICSI-ET).The patients were divided into two groups according to whether the transferred embryos underwent normal early cleavage.In the single cleavage embryo transfer,there were 383 cases in the normal early cleavage group(early cleavage group)and 411 cases in the non normal early cleavage group(non-early cleavage group);in the single blastocyst transfer,there were 815 cases in the early cleavage group and 651 cases in the non-early cleavage group.The pregnancy and obstetric outcomes of the early cleavage group and non-early cleavage group were compared in single cleavage embryo transfer and single blastocyst transfer.Results:In the single cleavage embryo transfer,the human chorionic gonadotropin(hCG)positive rate,clinical pregnancy rate,and live birth rate in the early cleavage group were significantly higher than those in the non-early cleavage group(64.0%vs.55.7%,P=0.018;54.6%vs.47.0%,P=0.032;46.7%vs.38.7%,P=0.022).There were no significant differ-ences in biochemical abortion rate,multiple pregnancy rate,abortion rate,early abortion rate,premature delivery rate,low birth weight rate,macrosomia rate,and birth defect rate compared with the non-early cleavage group(P>0.05);in the single blastocyst transfer,there were no significant differences in pregnancy outcomes and obstetric indicators between the early cleavage group and the non-early cleavage group(P>0.05).Multivariate Logistic regression analysis showed that after adjusting for confounding factors on pregnancy outcomes,in the single cleavage embryo transfer,the hCG positive rate(aOR 1.54,95%CI 1.12-2.11,P=0.008),clinical preg-nancy rate(aOR 1.49,95%CI 1.09-2.04,P=0.012),and live birth rate(aOR 1.53,95%CI 1.12-2.09,P=0.008)in the early cleavage group were significantly higher than those in the non-early cleavage group;in the sin-gle blastocyst transfer,there were no significant differences for these indicators between the early cleavage group and the non-early cleavage group(P>0.05).Conclusions:Transplantation of single cleavage embryo with nor-mal early cleavage can significantly improve the clinical pregnancy rate and live birth rate.Early cleavage during single blastocyst transplantation had no significant effect on clinical pregnancy outcome.Early cleavage had no significant effect on the obstetric outcomes of single cleavage embryo and single blastocyst transfer.
6.Factors influencing ethical sensitivity of oncology nurses: a qualitative study
Yun HUANG ; Yingxin WU ; Jun MEI ; Yuqi YANG ; Hui LI ; Xia XIANG
Chinese Journal of Modern Nursing 2025;31(32):4422-4427
Objective:To explore the factors influencing ethical sensitivity of oncology nurses.Methods:Using purposive sampling, 15 oncology nurses from 13 tertiary general hospitals in Foshan, Guangdong Province were selected as research participants from July to September 2023. In-depth semi-structured interviews were conducted, and Colaizzi's seven-step content analysis method was applied to organize and analyze the collected data.Results:A total of 4 themes were summarized: insufficient clinical experience and knowledge reserve; lack of ethical education and management system processes; high-pressure working environment and communication barriers; and the constraints of the cultural concept of "valuing life over death".Conclusions:Oncology nurses have insufficient ethical sensitivity. Nursing managers should establish a systematic ethical education system, improve ethical management systems, optimize the clinical working environment, and integrate cultural sensitivity education to jointly enhance nurses' communication skills. Attention should also be paid to the inheritance of clinical experience from senior nurses, so as to improve the ethical sensitivity of oncology nurses in a multidimensional way.
7.Efficacy of anrikefon versus tegileridine for analgesia in patients with moderate-to-severe pain after abdominal surgery with general anesthesia
Ziyuan LI ; Wenjie SU ; Meirong WANG ; Jun LI ; Daolin XIA ; Yuanliang CHEN ; Guiming HUANG ; Liang DONG ; Jia DENG ; Kaiming DUAN
Chinese Journal of Anesthesiology 2025;45(10):1291-1297
Objective:To compare the efficacy of anrikefon and tegileridine for analgesia in patients with moderate-to-severe pain after abdominal surgery with general anesthesia.Methods:In this multicenter, randomized, double-blind, active-controlled clinical trial, 101 patients with moderate to severe pain (numeric pain rating scale [NRS] score ≥4 within 4 h after operation) after abdominal surgery with general anesthesia between February 24 and April 1, 2025, aged 18-70 yr, with a body mass index of 18-40 kg/m 2, were assigned to anrikefon group ( n=50) and tegileridine group ( n=51) in a 1∶1 ratio using stratified blocked randomization. Double-dummy design was employed to maintain blinding. Each group received an initial intravenous injection of anrikefon 1 μg/kg or tegileridine 1 mg, followed by connection to a patient-controlled intravenous analgesia (PCIA) pump (the PCIA solution contained normal saline in anrikefon group; the PCIA solution contained tegileridine 5 mg in tegileridine pump) within 10 min. If the patient′s NRS score ≥4 at 8 and 16 h after the initial injection, anrikefon 1 μg/kg was intravenously injected in anrikefon group, and tegileridine group received the equal volume of normal saline. The primary efficacy endpoint was the sum of pain intensity difference (SPID) over the first 24 h after the initial dose (SPID 0-24h). The secondary efficacy endpoints included the incidence and severity of vomiting and nausea, incidence of postoperative nausea and vomiting(PONV), the proportion of patients who received antiemetic treatment, and total consumption of antiemetics within 0-24 h after the initial dose, NRS score at rest ≤ 1 at 24 h after the initial dose, and NRS score at rest ≤ 3 over the first 24 h after the initial dose. Safety indicators included adverse events, vital signs, physical examination findings, 12-lead ECG and laboratory test indicators, and adverse events of special interest. Results:Compared with tegileridine group, no significant change was found in the SPID 0-24h ( P>0.05), and the incidence of vomiting, PONV, proportion of patients requiring antiemetic medication, and total consumption of antiemetics were significantly decreased within the first 24 h after the initial dose in tegileridine group ( P<0.05). One treatment-emergent adverse event of Common Terminology Criteria for Adverse Events grade 3 or higher occurred in tegileridine group, while no treatment-emergent adverse events of Common Terminology Criteria for Adverse Events grade 3 or higher were found in anrikefon group. Among the adverse events of special interest, one case of respiratory depression and one case of cough occurred in tegileridine group, while one case of cough occurred in anrikefon group, with no respiratory depression. Conclusions:Anrikefon and tegileridine provide comparable analgesic efficacy for moderate-to-severe pain after abdominal surgery with general anesthesia. However, anrikefon exhibits an advantage in reducing the risk of PONV, with a superior safety profile.
8.Effect analysis of minimally invasive modified Bristow-Latarjet and Bankart prosthesis in the treatment of recurrent dislocation of shoulder joint
Gao HUANG ; Jun XU ; Wenge CHEN ; Zhengdong XIA
Chinese Journal of Postgraduates of Medicine 2025;48(2):154-159
Objective:To analyze and compare the clinical efficacy of minimally invasive modified Bristow-Latarjet and Bankart prosthesis in the treatment of recurrent shoulder dislocation.Methods:A total of 58 patients with recurrent shoulder dislocation treated in the Hubei Minzu University Affiliated Minda Hospital from October 2019 to December 2022 were retrospectively selected, and 29 patients underwent minimally invasive modified Bristow-Latarjet (Latarjet group) and 29 patients underwent Bankart prosthesis (Bankart group). The basic conditions of surgery, postoperative shoulder joint activity, and postoperative Rowe Shoulder Function Score (Rowe score) of the two groups were compared. The shoulder function recovery of the patients was evaluated using the American Shoulder and Elbow Association Scoring (ASES) system and the University of California at Los Angeles (UCLA) shoulder joint scoring system, and the incidence of complications between the two groups were compared.Results:The operation time, incision length, intraoperative bleeding volume, recovery working time, and reset loss in the Latarjet group were shorter than those in the Bankart group: (45.56 ± 12.18) min vs. (70.35 ± 18.14) min, (4.04 ± 0.82) cm vs. (6.75 ± 0.95) cm, (61.54 ± 8.88) ml vs. (86.07 ± 7.53) ml, (13.31 ± 3.21) weeks vs.(16.24 ± 3.33) weeks, (0.97 ± 0.19) mm vs.(1.24 ± 0.35)mm, there were statistical differences ( P<0.01). The outreach and forward angles in the Latarjet group were higher than those in the Bankart group: (138.25 ± 10.36)° vs. (98.15 ± 9.64)°, (140.14 ± 10.43)° vs. (93.79 ± 8.84)°, there were statistical differences ( P<0.01). The total effective rate in the Latarjet group was higher than that in the Bankart group: 96.55%(28/29) vs. 75.86%(22/29), there was a statistical difference ( χ2 = 5.22, P = 0.022). The Rowe scores after operation in the Latarjet group were higher than that in the Bankart group: (82.52 ± 15.89) scores vs. (66.78 ± 15.34) scores, there was a statistical difference ( P<0.01). The scores of self-assessment and physician assessment of ASES system and UCLA shoulder scores after operation in the Latarjet group were higher than those in the Bankart group: (36.13 ± 8.45) scores vs. (30.17 ± 8.48) scores, (40.03 ± 9.54) scores vs. (34.25 ± 8.74) scores, (32.08 ± 2.44) scores vs. (27.49 ± 2.56) scores, there were statistical differences ( P<0.05). The complication rate in the Latarjet group was lower than that in the Bankart group: 6.90%(2/29) vs. 31.03%(9/29), there was a statistical difference ( χ2 = 5.50, P = 0.019). Conclusions:The minimally invasive modified Bristow-Latarjet in the treatment of recurrent dislocation of the shoulder joint is superior to Bankart prosthesis in terms of shoulder functional recovery and postoperative rehabilitation.
9.Mechanism of electroacupuncture-induced macrophage polarization in promoting acute skeletal muscle injury repair in rats.
Yuting HUANG ; Yuye LIN ; Guojun ZHANG ; Chufan ZENG ; Xia ZHANG ; Jingyu ZHANG ; Yu KAN ; Yanping FANG ; Xianghong JING ; Jun LIAO
Chinese Acupuncture & Moxibustion 2025;45(6):791-800
OBJECTIVE:
To investigate the potential mechanism by which electroacupuncture (EA) induces macrophage polarization to promote muscle satellite cell proliferation and differentiation, accelerating the repair of acute skeletal muscle injury.
METHODS:
Forty-two SPF-grade SD rats were randomly divided into three groups: a blank group (n=6), a model group (n=18), and an EA group (n=18). The model and EA groups established acute blunt contusion model of the right gastrocnemius muscle using a self-made striking device. From day 1 after modeling, rats in the EA group received EA at "Chengshan" (BL57) and "Yanglingquan" (GB34) on the right side, using disperse-dense wave with a frequency of 2 Hz/100 Hz and a current of approximately 2 mA. The EA treatment was administered once daily for 30 minutes for 3, 7, or 14 days based on the designated sampling time points. Gait analysis was performed using the Cat Walk XTTM system. Hematoxylin-eosin (HE) staining was used to observe the morphological changes in the gastrocnemius muscle. Masson staining was applied to evaluate collagen fiber content. Immunofluorescence was used to detect the expression of proliferating cell nuclear antigen (PCNA) in muscle satellite cells. Immunohistochemistry was used to assess the expression levels of CD68 and CD206, markers of macrophages. Serum levels of pro-inflammatory cytokines (TNF-α, IL-1β) and anti-inflammatory cytokines (IL-10, IL-13) were detected using ELISA.
RESULTS:
Compared with the blank group, the model group showed a significant reduction in average movement speed on days 3 and 7 after modeling (P<0.05), and a decrease in the right hind limb stride length on day 3 (P<0.05). Compared with the model group, the EA group showed increased average movement speed and right hind limb stride length on day 7 (P<0.05). In the blank group, the gastrocnemius muscle on the right side showed uniform and consistent inter-fiber spacing, with neatly and regularly arranged muscle cells. In contrast, the model group exhibited enlarged inter-fiber spacing, edema, and significant infiltration of red blood cells and inflammatory cells, with progressively increasing fibrosis over time. By day 14 after modeling, the EA group showed a return to baseline levels of inflammatory cell infiltration, and the degree of fibrosis was significantly lower than that observed in the model group. Compared with the blank group, the ratio of collagen fibers in the gastrocnemius muscle of the model group increased significantly on days 3, 7, and 14 after modeling (P<0.05). Compared with the model group, the EA group exhibited a lower collagen fiber ratio on days 3, 7, and 14 (P<0.05). Compared with the blank group, PCNA positive expression in the gastrocnemius muscle of the model group was significantly increased on days 3, 7, and 14 after modeling (P<0.05). Compared with the model group, the EA group exhibited significantly higher PCNA positive expression on days 3 and 7 (P<0.05). Compared with the blank group, the model group showed a significant increase in CD68-positive macrophage expression in the gastrocnemius muscle on day 3 after modeling (P<0.05), while CD206-positive macrophage expression increased on days 3, 7, and 14 (P<0.05). Compared with the model group, CD68 expression was significantly lower in the EA group on day 3 (P<0.05), whereas CD206 expression was significantly higher on days 3 and 7 (P<0.05), peaking on day 7 with CD206 expression. Compared with the blank group, serum TNF-α levels were significantly elevated in the model group on days 3 and 7 after modeling (P<0.05), while serum IL-1β levels were increased on days 3, 7, and 14 (P<0.05). Serum IL-10 and IL-13 levels were significantly higher on day 7 after modeling (P<0.05). Compared with the model group, the EA group exhibited lower serum TNF-α level on day 3 (P<0.05) and reduced serum IL-1β levels on days 3 and 7 (P<0.05), while serum IL-10 and IL-13 levels were significantly increased on day 7 (P<0.05).
CONCLUSION
EA could promote the repair of acute blunt contusion-induced gastrocnemius muscle injury by regulating the proliferation and differentiation of muscle satellite cells. This process is closely related to macrophage polarization.
Animals
;
Electroacupuncture
;
Rats, Sprague-Dawley
;
Rats
;
Macrophages/immunology*
;
Muscle, Skeletal/immunology*
;
Male
;
Humans
;
Female
;
Tumor Necrosis Factor-alpha/immunology*
;
Cell Proliferation
10.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

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