1.Efficacy of C3F8 versus silicone oil tamponade in highly myopic macular hole retinal detachment
Xiaoying WEN ; Na YANG ; Yueling ZHANG ; Weina MA ; Yan FU ; Renfei GENG
International Eye Science 2024;24(5):805-809
AIM: To compare the outcome of C3F8 versus silicone oil tamponade after pars plana vitrectomy(PPV)and inverted internal limiting membrane(ILM)for the treatment of highly myopic macular hole retinal detachment(MHRD).METHODS: Retrospective clinical study. Totally 45 patients(45 eyes)with highly myopic MHRD who visited our hospital between January 2019 and August 2022 were selected as the research subjects. The patients were divided into two groups according to different intraocular tamponade agents: C3F8(22 eyes)and silicone oil(23 eyes)groups. All patients underwent conventional three-incision PPV, ILM was tamped, a venous blood clot was placed on the tamped ILM, and 15% C3F8 and silicone oil were used as tamponade, respectively. The best corrected visual acuity(BCVA), multifocal electroretinogram(mfERG), the closure of the macular hole, retinal reattachment and the complications were observed.RESULTS: The macular hole closure rate was 77% in the C3F8 group and 83% in the silicone oil group, respectively(P>0.05), and retinal reattachment rates were 95% and 96%, respectively(P>0.05). The visual acuity of the two groups significantly improved, which was 0.99±0.34 and 1.22±0.37, respectively, and the C3F8 group was better than that of the silicone oil group(t=-2.156, P=0.037). After operation, the response density of the first ring of P1 wave in the first order kernel in mfERG was 114.27±26.37 nV/deg2 for the C3F8 group and 98.08±24.36 nV/deg2 for the silicone oil group, and the response density of the second ring of P1 wave was 80.45±14.94 nV/deg2 for the C3F8 group and 67.73±15.33 nV/deg2 for the silicone oil group, all of which were significantly higher compared to pre-operation [the response density of the first ring of P1 wave: 58.13±13.96 nV/deg2 for the C3F8 group and 55.30±10.48 nV/deg2 for the silicone oil group, the response density of the second ring of P1 wave: 51.18±8.19 nV/deg2 for the C3F8 group and 47.43±11.97 nV/deg2 for the silicone oil group](all P<0.05). It was found that the response density of the first ring of P1 wave was lower in the silicone oil group than in the C3F8 group(P<0.05). There was no statistically significant difference in the incidence of complications between the two groups(P>0.05).CONCLUSION: Silicone oil tamponade or C3F8 tamponade after PPV combined with ILM can both promote retinal reattachment and macular hole closure in patients with MHRD, and the C3F8 tamponade was superior to silicone oil in visual function recovery.
2.Ultrasound Diagnosis and Misdiagnosis Analysis of Accessory Cavitated Uterine Malformation
Fei JI ; Na SU ; Huazhen LIU ; Zijing FU ; Zhenhong QI ; Meng YANG
Medical Journal of Peking Union Medical College Hospital 2024;15(5):1100-1106
To explore the ultrasound characteristics of accessory cavitated uterine malformation (ACUM) and the causes of misdiagnosis, in order to better understand the disease and improve the diagnostic ability of radiologists. We retrospectively collected clinical, pathological and imaging data of ACUM patients who were diagnosed after surgery at Peking Union Medical College Hospital from December2013 to December 2023. Besides, we analyzed ultrasound features of ACUM and the cause of misdiagnosis. A total of 11 patients were included, with a mean age of diagnosis of (27.1±7.4)years. There was a mean interval of (6.1±5.5)years between menarche and onset of dysmenorrhea. Preoperative ultrasound showed that ACUM appeared as solitary lesions located under the insertion of the round ligament, with a mean maximum diameter of (3.0±1.0)cm. Lesions were round or oval (90.9%, 10/11) and not connected to the uterine cavity (100%, 11/11) with clear boundaries (72.7%, 8/11), presenting as regular thick-walled cystic-solid structures. Cystic areas were observed within the mass (100%, 11/11) with a ground-glass-like appearance (90.9%, 10/11) and hyperechoic endometrial lining (90.9%, 10/11). The surrounding of the mass showed hypoechoic or isoechoic areas resembling the muscular layer and circular or semi-circular vascularity was detected around the mass. No adenomyosis or other uterine lesions were found. Analysis of misdiagnosis: Among the 11 ACUM cases, 6 were correctly diagnosed by the initial ultrasound examination, and 5 cases by MRI. Two patients were misdiagnosed as residual horn uterus by MRI, despite ultrasound suggesting ACUM with visible bilateral uterine horns. ACUM exhibits distinct ultrasound characteristics. When young women present with progressive dysmenorrhea and ultrasound reveals a regular cystic-solid mass with thick wall located under the round ligament insertion, ACUM should be considered.
3.Clinical observation of kidney-tonifying and mind-calming acupuncture therapy in the treatment of perimenopausal insomnia
Wenjia YANG ; Xintong YU ; Na ZHAO ; Chen XIE ; Jinjin LI ; Xiaolin GAO ; Cong FU ; Yunfei CHEN ; Xiehe KONG
Journal of Acupuncture and Tuina Science 2024;22(1):48-57
Objective:To observe the effects of kidney-tonifying and mind-calming acupuncture therapy on sleep,mood,sex hormone levels,and traditional Chinese medicine(TCM)symptoms in patients with perimenopausal insomnia(PMI). Methods:A total of 90 patients with PMI were randomly divided into a treatment group and a control group,with 45 cases in each group.Patients in the treatment group were treated with acupuncture at Shenshu(BL23),Taixi(KI3),Baihui(GV20),and Anmian(Extra).The control group was treated with sham acupuncture.Both groups were treated 3 times a week for 4 weeks.Pittsburgh sleep quality index(PSQI)and insomnia severity index(ISI)were used to evaluate the sleep quality of the subjects before treatment,after treatment,and 1 month after treatment(follow-up).Beck depression inventory(BDI)and Beck anxiety inventory(BAI)were used to evaluate the depression and anxiety of the subjects before treatment,after treatment,and at 1-month follow-up.The TCM symptom scale was used to evaluate the TCM symptoms of the subjects before treatment,after treatment,and 1 month after treatment.Serum levels of estradiol(E2),follicle-stimulating hormone(FSH),and luteinizing hormone(LH)were measured before and after treatment. Results:During the study,2 cases dropped out of the treatment group,and no cases dropped out of the control group.The PSQI scores of the treatment group were significantly lower after treatment and at 1-month follow-up compared with those before treatment(P<0.05),and the difference was statistically significant compared with that of the control group(P<0.05).In the control group,the PSQI score was significantly lower after treatment compared with before treatment(P<0.05),and the difference was not statistically significant at 1-month follow-up compared with before treatment(P>0.05).Compared with the pre-treatment,the ISI,BDI,BAI,and TCM symptom scale scores of the treatment group were lower after treatment and at 1-month follow-up(P<0.05),and the differences with the control group at the same time point were statistically significant(P<0.05).The differences in ISI,BDI,BAI,and TCM symptom scale scores of the control group before treatment,after treatment,and at 1-month follow-up were not statistically significant(P>0.05).After treatment,the serum E2 level in the treatment group was significantly higher than that before treatment(P<0.05),and the difference with the control group was statistically significant(P<0.05).The difference in the serum E2 level before and after treatment in the control group was not statistically significant(P>0.05).The differences in the serum FSH and LH levels between before and after treatment were not statistically significant in either group of subjects(P>0.05). Conclusion:Kidney-tonifying and mind-calming acupuncture therapy can improve sleep quality,relieve anxiety and depression,delay the decrease of serum E2 level,and improve related TCM symptoms in patients with PMI.
4.Construction of evaluation index system for teaching ability of dental specialist nurses
Ke SHAO ; Jianan HAN ; Lan FU ; Na LI ; Xue YANG
Chinese Journal of Modern Nursing 2024;30(3):315-321
Objective:To establish a scientific and comprehensive evaluation index system for teaching ability of dental specialist nurses, providing a basis for the selection and assessment of dental nursing instructors.Methods:Based on literature analysis, semi-structured interviews, and group discussions, preliminary evaluation indicators were formulated. From February to April 2023, the Delphi method was used to conduct two rounds of consultation with 27 experts from six provinces and municipalities in China to determine the evaluation index system for the teaching ability of dental specialist nurses. The analytic hierarchy process (AHP) was used to determine the weights of each indicator and its combined weight.Results:The authority coefficient of 27 experts was 0.895. In the two rounds of expert consultation, the effective response rates of the questionnaire were all 100% (27/27), and the Kendall harmony coefficients of various indicators were 0.179 to 0.240 and 0.200 to 0.261 respectively ( P<0.01). The final evaluation index system for teaching ability of dental specialist nurses was formed, including six primary indicators, 15 secondary indicators, and 71 tertiary indicators. Conclusions:The evaluation index system for teaching ability of dental specialist nurses has a reasonable structure, a scientific and rigorous construction process, and can provide a theoretical basis for the allocation of dental specialist human resources and nursing training assessment.
5.Changing resistance profiles of Enterococcus in hospitals across China:results from the CHINET Antimicrobial Resistance Surveillance Program,2015-2021
Na CHEN ; Ping JI ; Yang YANG ; Fupin HU ; Demei ZHU ; Yingchun XU ; Xiaojiang ZHANG ; Yi XIE ; Mei KANG ; Chuanqing WANG ; Pan FU ; Yuanhong XU ; Ying HUANG ; Ziyong SUN ; Zhongju CHEN ; Yuxing NI ; Jingyong SUN ; Yunzhuo CHU ; Sufei TIAN ; Zhidong HU ; Jin LI ; Yunsong YU ; Jie LIN ; Bin SHAN ; Yan DU ; Sufang GUO ; Lianhua WEI ; Fengmei ZOU ; Hong ZHANG ; Chun WANG ; Yunjian HU ; Xiaoman AI ; Chao ZHUO ; Danhong SU ; Dawen GUO ; Jinying ZHAO ; Hua YU ; Xiangning HUANG ; Wen'en LIU ; Yanming LI ; Yan JIN ; Chunhong SHAO ; Xuesong XU ; Chao YAN ; Shanmei WANG ; Yafei CHU ; Lixia ZHANG ; Juan MA ; Shuping ZHOU ; Yan ZHOU ; Lei ZHU ; Jinhua MENG ; Fang DONG ; Zhiyong LÜ ; Fangfang HU ; Han SHEN ; Wanqing ZHOU ; Wei JIA ; Gang LI ; Jinsong WU ; Yuemei LU ; Jihong LI ; Jinju DUAN ; Jianbang KANG ; Xiaobo MA ; Yanping ZHENG ; Ruyi GUO ; Yan ZHU ; Yunsheng CHEN ; Qing MENG ; Shifu WANG ; Xuefei HU ; Jilu SHEN ; Ruizhong WANG ; Hua FANG ; Bixia YU ; Yong ZHAO ; Ping GONG ; Kaizhen WEN ; Yirong ZHANG ; Jiangshan LIU ; Longfeng LIAO ; Hongqin GU ; Lin JIANG ; Wen HE ; Shunhong XUE ; Jiao FENG ; Chunlei YUE
Chinese Journal of Infection and Chemotherapy 2024;24(3):300-308
Objective To understand the distribution and changing resistance profiles of clinical isolates of Enterococcus in hospitals across China from 2015 to 2021.Methods Antimicrobial susceptibility testing was conducted for the clinical isolates of Enterococcus according to the unified protocol of CHINET program by automated systems,Kirby-Bauer method,or E-test strip.The results were interpreted according to the Clinical & Laboratory Standards Institute(CLSI)breakpoints in 2021.WHONET 5.6 software was used for statistical analysis.Results A total of 124 565 strains of Enterococcus were isolated during the 7-year period,mainly including Enterococcus faecalis(50.7%)and Enterococcus faecalis(41.5%).The strains were mainly isolated from urinary tract specimens(46.9%±2.6%),and primarily from the patients in the department of internal medicine,surgery and ICU.E.faecium and E.faecalis strains showed low level resistance rate to vancomycin,teicoplanin and linezolid(≤3.6%).The prevalence of vancomycin-resistant E.faecalis and E.faecium was 0.1%and 1.3%,respectively.The prevalence of linezolid-resistant E.faecalis increased from 0.7%in 2015 to 3.4%in 2021,while the prevalence of linezolid-resistant E.faecium was 0.3%.Conclusions The clinical isolates of Enterococcus were still highly susceptible to vancomycin,teicoplanin,and linezolid,evidenced by a low resistance rate.However,the prevalence of linezolid-resistant E.faecalis was increasing during the 7-year period.It is necessary to strengthen antimicrobial resistance surveillance to effectively identify the emergence of antibiotic-resistant bacteria and curb the spread of resistant pathogens.
6.Effect of Bushen Tiaojing Decoction Combined with Human Recombinant Interferon-α-2b on Humoral Immunity,CA125 and EMAb Levels in Patients with Endometriosis
Xiao-Bei KANG ; Hong-Jie YANG ; Xiu-Fang DU ; Chang-Na FU ; Tao YANG ; Wei-Wei SUN
Journal of Guangzhou University of Traditional Chinese Medicine 2024;41(6):1472-1478
Objective To investigate the efficacy of Bushen Tiaojing Decoction,a prescription derived from Daying Decoction which recorded in Jing Yue Quan Shu(The Complete Works of ZHANG Jing-Yue)and with herbs added,combined with human recombinant interferon-α-2b for the treatment of endometriosis(EMs)and to observe its effects on humoral immunity,serum carbohydrate antigen 125(CA125)and anti-endometrial antibody(EMAb)levels of EMs patients.Methods A total of 86 cases of EMs patients with kidney deficiency and blood stasis syndrome were randomly divided into observation group and control group,with 43 cases in each group.The control group was treated with human recombinant interferon-α-2b alone,and the observation group was treated with Bushen Tiaojing Decoction combined with human recombinant interferon-α-2b.The course of treatment covered one month.The changes of humoral immune factors of immunoglobulin A(IgA),complement 3(C3)and complement 4(C4),endometrial ectopic mass diameter,and the levels of pathology-relevant factors of CA1 25 and EMAb in the two groups before and after treatment were observed.Moreover,the clinical efficacy and safety of the two groups were evaluated.Results(1)After one month of treatment,the total effective rate of the observation group was 97.67%(42/43),and that of the control group was 81.40%(35/43).The intergroup comparison(tested by chi-square test)showed that the efficacy of the observation group was significantly superior to that of the control group(P<0.05).(2)After treatment,the serum IgA level of the two groups was significantly higher than that before treatment(P<0.05),and the serum C3 and C4 levels were significantly lower than those before treatment(P<0.05).The increase of serum IgA level and the decrease of serum C3 and C4 levels in the observation group were significantly superior to those in the control group(P<0.01).(3)After treatment,the serum CA125 and EMAb levels and the diameter of endometrial ectopic mass in the two groups were significantly lower than those before treatment(P<0.05),and the decrease of serum CA125 and EMAb levels and the diameter of endometriosis mass in the observation group was significantly superior to that in the control group(P<0.01).(4)The total incidence of adverse reactions in the observation group was 6.98%(3/43)and that in the control group was 11.63%(5/43),and the difference between the two groups was not significant(P>0.05).Conclusion Bushen Tiaojing Decoction combined with human recombinant interferon-α-2b has a significant clinical effect for the treatment of EMs patients with kidney deficiency and blood stasis syndrome.The combined therapy is effective on improving the levels of humoral immune factors,serum CA125 and EMAb,and reducing endometrial ectopic mass,with high safety.
7.Research status on the mechanism of traditional Chinese medicine regulating TGF-β1/Smads signaling pathway to intervene liver fibrosis
Yong-Biao TAO ; Shi-Rui YANG ; Long-De WANG ; Ya-Na WU ; Zhi-Ming ZHANG ; Fu LI
The Chinese Journal of Clinical Pharmacology 2024;40(6):918-922
Hepatic fibrosis(HF)is a pathophysiological outcome of chronic liver injury and is characterized by excessive accumulation of extracellular matrix protein.A number of studies have confirmed that the signaling pathways formed by transforming growth factor-β1(TGF-β1)and its downstream Smad family play an important role in the occurrence and development of HF,and the traditional Chinese medicine(TCM)research targeting this pathway is currently a hot spot in the reversal of HF.Therefore,taking TGF-β1/Smads signaling pathway as the entry point,this paper reviewed the mechanism of action of TCM compound formula and single drug extract in intervening TGF-β1/Smad pathway and related factors upstream and downstream of the pathway to reverse HF in recent years,revealed the targeted therapeutic effect of TCM,and provided new strategies for clarifying the mechanism of TCM.
8.Influence of Tongfu Xiefei Guanchang Solution on intestinal barrier and intestinal flora of rats with acute lung injury based on p38 MAPK/MLCK signaling pathway.
Ming MA ; Kun WANG ; Yan-Hua YANG ; Meng-Ru YUE ; Quan-Na REN ; Yu-Han CHEN ; Yong-Zhen SONG ; Zi-Fu XU ; Xu ZHAO
China Journal of Chinese Materia Medica 2024;49(21):5919-5931
The study is designed to observe the mechanism of Tongfu Xiefei Guanchang Solution(TFXF) in the treatment of acute lung injury(ALI) in rats by improving intestinal barrier and intestinal flora structure via p38 mitogen-activated protein kinase(p38 MAPK)/myosin light chain kinase(MLCK) signaling pathway. Sixty SPF-grade Wistar rats were randomly divided into the control(CON) group, lipopolysaccharide(LPS) group(7.5 mg·kg~(-1)), LPS + dexamethasone(DEX) group(3.5 mg·kg~(-1)), LPS + high-dose(HD)-TFXF group(14.74 g·kg~(-1)), LPS + middle-dose(MD)-TFXF group(7.37 g·kg~(-1)), and LPS + low-dose(LD)-TFXF group(3.69 g·kg~(-1)). ALI model of the rat was established by intraperitoneal injection of LPS. The lactate dehydrogenase(LDH) activity and total protein concentration in the bronchoalveolar lavage fluid(BALF) were measured; tumor necrosis factor-α(TNF-α) and interleukin-1β(IL-1β) levels in lung and colon tissue of rats were detected by enzyme linked immunosorbent assay(ELISA). Hematoxylin-eosin(HE) staining was used to observe the pathological expression in the lung and colon tissue of rats. The mRNA expression of p38 MAPK, TNF-α, and IL-1β in rat lung tissue was determined by real-time fluorescence quantitative polymerase chain reaction(real-time PCR). Western blot was used to detect the protein expression related to the p38 MAPK/MLCK signaling pathway in the colon tissue of rats. 16S rRNA sequencing was used to detect changes in the composition and content of intestinal flora in rats, and correlation analyses were performed to explore the regulatory role of intestinal flora in improving ALI in rats. The results showed that compared with those in the LPS group, the histopathological scores of lung and colon tissue, LDH activity, and total protein concentration in BALF were significantly reduced in rats in all groups after drug administration. Except for the LPS + LD-TFXF group, the remaining groups significantly reduced the levels of TNF-α and IL-1β in the lung and colon tissue of rats. The protein expressions of phosphorylated p38 mitogen-activated protein kinase(p-p38 MAPK)/p38, phosphorylated myosin light chain(p-MLC)/myosin light chain 2(MLC2), and MLCK in colon tissue of rats in each drug administration group were significantly decreased. The mRNA expression levels of p38 MAPK, TNF-α, and IL-1β were significantly reduced in the LPS + HD-TFXF group. 16S rRNA sequencing results showed that the abundance of intestinal flora was significantly higher in the LPS + HD-TFXF group, and intestinal floras including Sobs, Shannon, and Npshannon were significantly higher. The β-diversity distribution of intestinal flora tends toward the CON group, and the abundance of Firmicutes was significantly higher. The abundance of Proteobacteria was significantly reduced; the abundance of Bacteroides was significantly reduced, and the abundance of Ruminococcus was significantly higher. The main species differences were Blautia, Roseburia_sp_499, and Butyricicoccus. TNF-α and IL-1β of lung tissue were negatively correlated with Muribaculaceae, unclassified norank_f_Eubacterium_coprostanoligenes, and Ruminococcus and positively correlated with Bacteroides. Meanwhile, TNF-α and IL-1β of colon tissue were negatively correlated with unclassified norank_f_Eubacterium_coprostanoligenes and Ruminococcus and positively correlated with Bacteroides. The predicted biological function of the flora was related to the biosynthesis of secondary metabolites, amino acid biosynthesis, sugar metabolism, and oxidative phosphorylation. The above studies show that TFXF can repair lung and colon tissue structure and regulate inflammatory factor levels by modulating the abundance and diversity of intestinal flora species in ALI rats. Its mechanism of action in ameliorating ALI in rats may be related to the inhibition of inflammation, improvement of intestinal mucosal permeability, and maintenance of intestinal flora homeostasis and barrier through the p38 MAPK/MLCK signaling pathway.
Animals
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Acute Lung Injury/genetics*
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Rats
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p38 Mitogen-Activated Protein Kinases/genetics*
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Drugs, Chinese Herbal/pharmacology*
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Myosin-Light-Chain Kinase/genetics*
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Male
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Gastrointestinal Microbiome/drug effects*
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Rats, Wistar
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Signal Transduction/drug effects*
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Interleukin-1beta/metabolism*
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Tumor Necrosis Factor-alpha/metabolism*
;
Lung/metabolism*
;
Intestinal Mucosa/metabolism*
;
Humans
9.Localized light-triggered release macrophage cytopharmaceuticals containing O-nitrobenzyl group for enhanced solid tumor cell-chemotherapy.
Jinhu LIU ; Han YANG ; Xiao SANG ; Tong GAO ; Zipeng ZHANG ; Shunli FU ; Huizhen YANG ; Lili CHANG ; Xiaoqing LIU ; Shuang LIANG ; Shijun YUAN ; Suyun WEI ; Yuxin YANG ; Xiaoxin YAN ; Xinke ZHANG ; Weiwei MU ; Yongjun LIU ; Na ZHANG
Acta Pharmaceutica Sinica B 2024;14(11):5053-5068
Cytopharmaceutical based on macrophages is a breakthrough in the field of targeted drug delivery. However, it remains a challenge to localize and control drug release while retaining macrophage activity and exerting its immunotherapeutic effect. Herein, a localized light-triggered release macrophage cytopharmaceutical (USIP@M) was proposed, which could utilize the tumor targeting and immunotherapy effects of macrophages to reverse the immune suppression of tumor microenvironment (TME). Amphiphilic block copolymers with ultraviolet (UV)-responsive o-nitrobenzyl groups were synthesized and co-loaded with sorafenib (SF), IMD-0354 (IMD), and upconverting nanoparticles (UCNPs), which were then taken up by macrophages, and the targeted delivery of drugs was realized by using the tumor tropism of macrophages. UCNPs converted near-infrared light with strong penetrability and high safety into UV light, which promoted the photoresponsive depolymerization of block copolymers and production of exosomes from USIP@M, accelerated drug efflux and maintained the activity of macrophages. IMD simultaneously polarized carrier macrophages and tumor-associated macrophages to exert the antitumor effect of macrophages, enhance T cell immunity, and alleviate the immunosuppressive state of TME. Synergistically with the chemotherapeutic effect of SF, it could effectively kill tumors. In conclusion, based on the localized light-triggered release strategy, this study constructed a novel macrophage cytopharmaceutical that could localize and control drug release while retaining the activity of macrophages and exerting its immunotherapeutic effect, which could effectively treat solid tumors.
10.Prognosis of childhood adrenoleukodystrophy with cognitive disorder after haploidentical allogenic hemato-poietic stem cell transplantation
Yu′ang AN ; Ying ZHANG ; Yijia ZHANG ; Ya′nan CUI ; Jingjing LIANG ; Qinrui LI ; Jie FU ; Na FU ; Yao CHEN ; Yanling YANG ; Jiong QIN
Chinese Journal of Applied Clinical Pediatrics 2023;38(8):566-570
Objective:To investigate the prognosis of childhood adrenoleukodystrophy (ALD) with cognitive disorder after haploidentical allogenic hematopoietic stem cell transplantation (haplo-HSCT), and to identify risk factors affecting the prognosis.Methods:It was a single-center retrospective study involving 31 ALD children receiving haplo-HSCT in Peking University People′s Hospital from January 2014 to October 2022.Survival analysis was performed by Kaplan-Meier method. Cox regression analysis was performed to identify risk factors for the prognosis of childhood ALD following haplo-HSCT. Results:Among the 31 children with ALD, 1 case died of cardiogenic shock during the transplantation, and the remaining had a successful haplo-HSCT.Ten children with ALD had cognitive disorder before haplo-HSCT, including 3 cases with the minimal LOES score ≥10 points and 8 cases with the Neurologic Function Score (NFS)>0 point before haplo-HSCT.Six children had major functional disability (MFD) and 2 cases died due to progression of ALD after haplo-HSCT.Twenty children did not have cognitive disorder before haplo-HSCT, of whom 3 cases had the LOES score≥10 points and 6 cases had NFS>0 before haplo-HSCT.Four children had MFD and 2 cases died due to progression of ALD after haplo-HSCT.For ALD patients without cognitive disorder after haplo-HSCT, the 3-year and 5-year survival rate were 100.0% and 72.9%, respectively, and the 5-year MFD-free survival was 61.6%.For ALD patients with cognitive disorder after haplo-HSCT, the 3-year survival rate was 83.3%.Compared with ALD patients with the LOES score<10 points before haplo-HSCT, those with the LOES score≥10 points had 9.243 times the risk of developing MFD after haplo-HSCT ( P=0.024, 95% CI: 1.332-64.127). Compared with ALD patients without cognitive disorder before haplo-HSCT, ALD patients with cognitive disorder had 9.749 times the risk of developing MFD after haplo-HSCT ( P=0.023, 95% CI: 1.358-66.148). Conclusions:Cognitive disorder and LOES score≥10 points before haplo-HSCT are risk factors for developing MFD in children with ALD following haplo-HSCT.

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