1.Multi-dimensional Holographic Characterization of Zhejiang Characteristic Atractylodis Macrocephalae Rhizoma with Nine-time Repeating Steaming and Processing
Xin WU ; Cuiwei CHEN ; Qiao YU ; Chao FENG ; Hongyan ZHANG ; Yan CHEN ; Caihua SUN ; Gang CAO
Chinese Journal of Experimental Traditional Medical Formulae 2026;32(6):197-205
ObjectiveHistorically documented Zhejiang Atractylodis Macrocephalae Rhizoma(Baizhu) possesses premium characteristics such as phoenix-like head and crane-like neck, pronounced sweetness, and fragrant aroma. However, its current market circulation is low, and the processed products with Zhejiang-style characteristics are at the risk of being lost. This study aims to preserve the ancient Zhejiang-style processing techniques and evaluate them using modern scientific methods. MethodsMultidimensional intelligent sensory evaluation was used to digitally characterize the "quality-structure" of the external appearance of nine-steamed and nine-processed Baizhu medicinal materials(intermediate processed products) and the "odor-taste" of the internal quality of its decoction pieces(slices), and the appearance parameters were digitally characterized by colorimeter, texture analyzer, electronic nose and electronic tongue, the chemical composition was analyzed via ultra-performance liquid chromatography-quadrupole-time-of-flight mass spectrometry(UPLC-Q-TOF-MS/MS). Then, cluster analysis on the differences in odor between the medicinal materials(intermediate processed products) and decoction pieces(slices) of nine-steamed and nine-processed Baizhu was conducted, as well as the differences in taste between water-soluble and alcohol-soluble extracts of the decoction pieces(slices), and the correlation analysis of chroma value-alcohol-soluble extract content-component response value. ResultsThe nine-steamed and nine-processed Baizhu had a dark brown to black epidermis, a brownish-yellow to brownish-gray cross-section, a slightly tough texture, a faint odor, and a slightly sweet, bitter and pungent taste. Texture analyzer measurements revealed minimal adhesion and maximum recovery in the middle section of the characteristic processed Baizhu, consistent with the processing endpoint of thorough steaming and cooking. The head section showed the highest internal hardness, elasticity and chewiness, indicating a denser texture in this area. The electronic nose sensor could clearly distinguish the difference between the medicinal materials and its decoction pieces, with a more significant clustering effect at 60 ℃ for 30 minutes compared to ambient temperature headspace for 2 hours, highlighting the significant impact of the baking degree before slicing on the quality. The electronic tongue taste signal map clearly distinguished the differences between water-soluble and alcohol-soluble extracts of nine-steamed and nine-processed Baizhu decoction pieces, and the addition of auxiliary materials during processing could enhance its alcohol-soluble extract content. A total of 82 chemical components were identified in the characteristic processed Baizhu. After processing, the contents of 58 components increased, while 24 components decreased. Correlation analysis revealed significant negative correlations(P<0.01) between ethanol-soluble extract content and colorimetric values of brightness(L*), yellow-bule value(b*), and total color difference(E*ab). E*ab showed marked negative correlations(P<0.05) with the response values of isochlorogenic acid A and C. ConclusionThis study establishes a modern intelligent sensory evaluation model for multidimensional holographic characterization of nine-steamed and nine-processed Baizhu, clarifying the correlation between increased isochlorogenic acid content and the visual color appearance after different steaming cycles, as well as its intrinsic alcohol-soluble extracts. This provides a reference for quality evaluation and processing standards of the Zhejiang-style characteristic processed products.
2.Study on the modeling method of general model of Yaobitong capsule intermediates quality analysis based on near infrared spectroscopy
Le-ting SI ; Xin ZHANG ; Yong-chao ZHANG ; Jiang-yan ZHANG ; Jun WANG ; Yong CHEN ; Xue-song LIU ; Yong-jiang WU
Acta Pharmaceutica Sinica 2025;60(2):471-478
The general models for intermediates quality analysis in the production process of Yaobitong capsule were established by near infrared spectroscopy (NIRS) combined with chemometrics, realizing the rapid determination of notoginsenoside R1, ginsenoside Rg1, ginsenoside Re, ginsenoside Rb1, ginsenoside Rd and moisture. The spray-dried fine powder and total mixed granule were selected as research objects. The contents of five saponins were determined by high performance liquid chromatography and the moisture content was determined by drying method. The measured contents were used as reference values. Meanwhile, NIR spectra were collected. After removing abnormal samples by Monte Carlo cross validation (MCCV), Monte Carlo uninformative variables elimination (MC-UVE) and competitive adaptive reweighted sampling (CARS) were used to select feature variables respectively. Based on the feature variables, quantitative models were established by partial least squares regression (PLSR), extreme learning machine (ELM) and ant lion optimization least squares support vector machine (ALO-LSSVM). The results showed that CARS-ALO-LSSVM model had the optimum effect. The correlation coefficients of the six index components were greater than 0.93, and the relative standard errors were controlled within 6%. ALO-LSSVM was more suitable for a large number of samples with rich information, and the prediction effect and stability of the model were significantly improved. The general models with good predicting effect can be used for the rapid quality determination of Yaobitong capsule intermediates.
3.High-sensitivity Ratio-type Surface-enhanced Raman Substrate for Rapid Quantitative Determination of 6-Thioguanine in Serum
Yan-Bin LIU ; Yi-Chao HAN ; Rong WANG ; Xiao-Mei WU ; Qin WANG ; Yuan-Yuan YAO ; Yue-Liang WANG ; Long-Hua GUO
Chinese Journal of Analytical Chemistry 2025;53(8):1300-1310
6-Thioguanine(6-TG)is an antineoplastic agent used in treatment of acute leukemia.However,significant interindividual variability in dosing regimens and frequent clinical manifestations of hepatotoxicity and myelosuppression as adverse effects have affected its therapeutic efficacy.Consequently,the development of rapid analytical methods for 6-TG in clinical samples,enabling continuous therapeutic drug monitoring of plasma concentrations,holds substantial significance in optimizing dosage regimens,mitigating adverse reactions,and investigating drug metabolism mechanisms.In this study,multi-tipped gold nanostars(AuNSs)were prepared.With bis-(p-sulfonylphenyl)phenylphosphine molecule as the protecting agent and internal standard molecule,the AuNSs were assembled onto a highly sensitive surface-enhanced Raman(SERS)substrate for developing a ratio-based SERS quantitative analysis method for 6-TG in serum.The AuNSs containing multiple tips and gaps exhibited strong local surface plasmon resonance effect and SERS activity,ensuring the sensitivity of the analytical method.Furthermore,the introduction of internal standard molecules could improve the reproducibility,which guaranteed this method suitable for rapid analysis of drug molecules in complex samples.Quantitative analysis of 6-TG was achieved with linear detetion range of 1.0×10?4-1.0 mmol/L.In the spiked recovery experiments of serum,the RSD was less than 5.32%,and the recoveries were 94%-104%,which proved that this method could be used for rapid quantitative determination of 6-TG in serum.This method provided a powerful tool for studying drug pharmacokinetics,which could promote the optimization of the usage methods of anti-cancer drugs,and it was expected to further enhance the clinical efficacy and safety of 6-TG,enabling it to achieve the best therapeutic effect.
4.Metabolomics and pharmacokinetics of Corni Fructus in ameliorating myocardial ischemic injury.
Xiang-Feng LIU ; Yu WU ; Chao-Yan YANG ; Hua-Wei LIAO ; Yan-Fen CHEN ; Xin HE ; Ying-Fang WANG ; Jin-Ru LIANG
China Journal of Chinese Materia Medica 2025;50(5):1363-1376
This study aims to investigate the ameliorating effect of Corni Fructus(CF) on the myocardial ischemic injury and the pharmacokinetic properties of characteristic components of CF. The mouse model of isoproterenol-induced myocardial ischemia was established and administrated with the aqueous extract of CF. The general efficacy of CF in ameliorating the myocardial ischemic injury was evaluated based on the cardiac histopathology and the levels of myocardial injury markers: creatine kinase isoenzyme(CK-MB) and cardiac troponin I(cTn-I). The metabolomics analysis was carried out for the heart and serum samples of mice to screen the biomarkers of CF in ameliorating the myocardial ischemic injury and then the predicted biomarkers were submitted to metabolic pathway enrichment. The pharmacokinetic analysis was performed for morroniside, loganin, and cornuside Ⅰ in mouse heart and serum samples to obtain the pharmacokinetic parameters of these components. The pharmacokinetic parameters were then integrated on the basis of self-defined weighting coefficients to simulate an integrated pharmacokinetic profile of CF iridoid glycosides in the heart and serum of the mouse model of myocardial ischemia. The results indicated that CF reduced the pathological damage to cardiac cells and tissue(hematoxylin-eosin staining) and lowered the levels of CK-MB and cTn-I in the serum of the mouse model of myocardial ischemia(P<0.01). Metabolomics analysis screed out 31 endogenous metabolites in the heart and 35 in the serum as biomarkers of CF in ameliorating the myocardial ischemic injury. These biomarkers were altered by modeling and restored by CF. Six metabolic pathways in the heart and 5 in the serum were enriched based on these metabolic markers. The main integrated pharmacokinetic parameters of CF iridoid glycosides were T_(max)=1 h, t_(1/2)=(1.52±0.05) h in the heart and T_(max)=1 h, t_(1/2)=(1.56±0.50) h in the serum. Both concentration-time curves showed a double-peak phenomenon. In conclusion, CF demonstrated the cardioprotective effect by regulating metabolic pathways such as taurine and hypotaurine metabolism, and pantothenic acid and coenzyme A biosynthesis. The integrated pharmacokinetics reflect the general pharmacokinetic properties of characteristic components in CF.
Animals
;
Cornus/chemistry*
;
Mice
;
Metabolomics
;
Drugs, Chinese Herbal/administration & dosage*
;
Male
;
Myocardial Ischemia/metabolism*
;
Humans
;
Troponin I/metabolism*
;
Myocardium/pathology*
;
Disease Models, Animal
;
Biomarkers/metabolism*
;
Creatine Kinase, MB Form/metabolism*
5.Effectiveness evaluation of three-dimensional printed customized hemi-pelvic prosthesis for pelvic reconstruction after resection of massive pelvic tumor.
Yuhui SU ; Chao GAO ; Huajian WU ; Zhengwang SUN ; Wangjun YAN
Chinese Journal of Reparative and Reconstructive Surgery 2025;39(11):1414-1420
OBJECTIVE:
To investigate the effectiveness of three-dimensional (3D) printed customized hemi-pelvic prosthesis for pelvic reconstruction after resection of massive pelvic tumors.
METHODS:
A retrospective analysis was conducted on 26 patients with massive pelvic tumors who met the selection criteria and were treated between November 2021 and May 2024. The cohort included 11 males and 15 females, with a mean age of 52.65 years (range, 17-73 years). Histopathological diagnoses were as follows: 9 cases of chondrosarcoma, 2 of undifferentiated pleomorphic sarcoma, 4 of spindle cell sarcoma, 2 of osteosarcoma, 1 of solitary fibrous tumor, 1 of myxoid chondroma, 1 of malignant peripheral nerve sheath tumor, 1 of chondromyxoid epithelioma, and 5 of metastatic malignant tumors. According to the Enneking classification, tumor involvement was distributed as 4 cases in zones Ⅰ+Ⅱ, 9 in zones Ⅱ+Ⅲ, 3 in zones Ⅰ+Ⅳ, 8 in zones Ⅰ+Ⅱ+Ⅲ, and 2 in zones Ⅰ+Ⅱ+Ⅳ. The disease duration ranged from 3 to 40 months, with a mean of 9.85 months. All patients underwent reconstruction with customized 3D-printed hemi-pelvic prostheses. The effectiveness was evaluated by Musculoskeletal Tumor Society (MSTS) score and Harris hip score before operation and at last follow-up, and pain levels were evaluated by visual analogue scale (VAS) score before operation, at 3 months after operation, and at last follow-up.
RESULTS:
The operation time ranged from 186 to 528 minutes, with a mean of 334.58 minutes. The intraoperative blood loss ranged from 1 400 to 4 000 mL, with a mean of 2173.08 mL, and the transfusion volume ranged from 750 to 3 500 mL, with a mean of 1 659.62 mL. All 26 patients were followed up 10-42 months (mean, 18.5 months). Postoperative complications included prosthetic dislocation in 2 cases, which were attributed to improper positioning during home care and an accidental fall, respectively. One patient developed a vesicocutaneous fistula and poor wound healing due to pre-existing tumor invasion into the bladder. One patient experienced failure and loosening of the internal fixation at 8 months after operation caused by local tumor recurrence, and subsequently died at 14 months postoperatively due to progression of brain metastases. Postoperative complications such as poor healing of incisions, prosthetic dislocation, or failure of internal fixation was not observed in the remaining patients. At last follow-up, the walking ability of most patients recovered to varying degrees. The VAS scores at 3 months and at last follow-up significantly improved when compared with those before operation, and the scores at last follow-up further improved when compared with 3 months after operation, all showing significant differences ( P<0.05). The MSTS scores and Harris scores at last follow-up were significantly higher than those before operation ( P<0.05).
CONCLUSION
3D printed customized hemi-pelvic prosthesis is effective for reconstruction of massive pelvic tumors after resection, but there are still some limitations, and soft tissue reconstruction should be paid attention to.
Humans
;
Printing, Three-Dimensional
;
Female
;
Male
;
Adult
;
Plastic Surgery Procedures/methods*
;
Retrospective Studies
;
Middle Aged
;
Aged
;
Pelvic Bones/surgery*
;
Bone Neoplasms/surgery*
;
Adolescent
;
Pelvic Neoplasms/surgery*
;
Prosthesis Design
;
Young Adult
;
Treatment Outcome
;
Prostheses and Implants
6.Clinical efficacy of minimally invasive tendon blade technique in the treatment of moderate and severe gluteal muscle contracture.
Jia-Kai GAO ; Tao-Ran WANG ; Long BI ; Xiao-Chao CHEN ; Yan-Wu LIU ; Yao-Ping WU ; Xiang HE ; Zhi-Xia NIU
China Journal of Orthopaedics and Traumatology 2025;38(4):420-423
OBJECTIVE:
To investigate the clinical effect of minimally invasive technique in the treatment of moderate and severe gluteal muscle contracture.
METHODS:
A retrospective study was conducted on 85 patients (170 sides) with bilateral gluteal muscle contracture admitted from January 2016 to December 2019. All patients were treated with minimally invasive release of tendon knife. There were 32 males and 53 females, ranging in age from 15 to 37 years old, with an average age of (22.3±6.3) years old. Operation time, intraoperative blood loss, incision length, first postoperative ambulation time, complication rate, recurrence rate, and Harris hip score (HHS) were analyzed and evaluated.
RESULTS:
The average follow-up time was (16.2±4.6) months, ranging from 12 to 30 months. The operation time ranged from 7 to 15 min, with an average of (10.2±3.1) min. Intraoperative blood loss ranged from 2 to 20 ml, with an average of (8.4±2.2) ml. The incision length ranged from 0.6 to 2.0 cm, with an average of (0.8±0.3) cm. The time to postoperative ambulation ranged from 12 to 28 h, with an average of (20.0±3.2) h. All patients achieved primary wound healing without sciatic nerve injury or recurrence. HHS hip function scores ranged from 90 to 98, with an average score of (96.2±1.4). Complications included intraoperative tendon blade tip fracture in two cases (removed under fluoroscopic guidance) and subcutaneous hematoma in three cases-two resolved with compression and one with open evacuation.. Twenty-nine patients exhibited transient swaying gait postoperatively, of which 24 patients returned to normal after 4 weeks and 5 patients returned to normal after 6 weeks.
CONCLUSION
Minimally invasive tendon blade release is a safe and effective technique for treating gluteal muscle contracture, offering minimal trauma, rapid recovery, and excellent cosmetic and functional outcomes. However, it exhibits a low risk of blade tip fracture and sciatic nerve injury, warranting experienced surgical handling.
Humans
;
Male
;
Female
;
Adult
;
Minimally Invasive Surgical Procedures/methods*
;
Adolescent
;
Retrospective Studies
;
Buttocks/surgery*
;
Young Adult
;
Contracture/surgery*
;
Tendons/surgery*
;
Muscle, Skeletal/surgery*
7.Acupuncture Combined with Periocular Injection for Treatment of Hydroxychloroquine Retinopathy with Cystoid Macular Edema: A Case Report.
Tian-Tian LI ; Yan WU ; Ying-Xin YANG ; Yu-Xin XUE ; Chao-Ting MA
Chinese journal of integrative medicine 2025;31(1):68-72
For HCQ retinopathy with CME, acupuncture combined with periocular injection can be used to improve the CME and protect the central vision. Subsequent research endeavors involving a more extensive cohort and extended observation periods are warranted to evaluate the effectiveness and safety profile of the intervention.
Humans
;
Macular Edema/drug therapy*
;
Acupuncture Therapy/methods*
;
Hydroxychloroquine/therapeutic use*
;
Female
;
Retinal Diseases/chemically induced*
;
Middle Aged
;
Combined Modality Therapy
;
Male
8.Primary intraosseous synovial sarcoma:a case report and literature review
Wen ZHAO ; Wei-Jun QIAN ; Li LI ; Yan-Min WANG ; Peng-Hui SU ; Chao-Xin ZHANG ; Liang XU ; Tie-Cheng WU ; Jun-Qi LIU ; Ya-Jun WANG
Medical Journal of Chinese People's Liberation Army 2025;50(11):1419-1425
Objective To report a case of tibial synovial sarcoma and review relevant literature to enhance understanding of this disease.Methods The clinical data of a patient with tibial synovial sarcoma treated at Kaifeng Central Hospital were retrospectively analyzed.A literature search was conducted in domestic and international databases,including China National Knowledge Infrastructure(CNKI),Wanfang Data,PubMed,Web of Science,and Embase,up to July 2024.Relevant literature was comprehensively reviewed to summarize the imaging and pathological characteristics,treatment,and prognosis of synovial sarcoma.Results A 29-year-old female patient was admitted with left lower extremity pain.X-ray examination revealed a proximal tibia space-occupying lesion suggestive of malignancy,and a mid-tibial space-occupying lesion considered benign.Contrast-enhanced computed tomography(CT)and plain magnetic resonance imaging(MRI)of the proximal tibial lesion also suggested malignancy.Ultrasound-guided biopsy of the proximal tibial tumor revealed a poorly differentiated malignant tumor.Immunohistochemistry results indicated monophasic synovial sarcoma,requiring genetic testing for definitive diagnosis.The patient underwent wide resection of the proximal left tibial malignancy with tumor-type artificial joint replacement,combined with curettage and bone cement filling for the left mid-tibial lesion under anesthesia.Postoperative pathology of space-occupying lesions in the proximal tibia confirmed monophasic synovial sarcoma,and fluorescence in situ hybridization(FISH)demonstrated a rupture of the synovial sarcoma translocation gene(SYT)(i.e.,SS18 positive).There was no recurrence or metastasis found in the patient during the reexamination 6 months after postoperative chemotherapy.As of July 2024,15 cases of genetically confirmed primary intraosseous synovial sarcoma have been reported internationally.Symptoms included pain and swelling,with a medical history of 1-2 years.The X-ray and CT findings showed osteolytic destruction with bone cortical discontinuity.In 13 cases,the intraosseous masses extended to the extraosseous area;in 2 cases,punctate calcifications were detected within the masses.Plain MRI scan showed iso-signal or hypo-signal on T1WI and hyper-signal,iso-signal,and hypo-signal on fat-suppressed T2WI,and enhanced MRI scan demonstrated heterogeneous enhancement.Pathological examination showed spindle-shaped cells under microscopy.Immunohistochemistry results showed positive epithelial membrane antigen(EMA),broad-spectrum cytokeratin(AE1/AE3),Ewing's sarcoma marker(CD99),and transducin-like enhancer of Split 1(TLE1).Twelve patients underwent surgical treatment;6 patients received adjuvant chemotherapy after surgery,of whom 4 developed local recurrence or distant metastasis at initial diagnosis,and 3 died during follow-up.Among the 6 patients who did not receive adjuvant chemotherapy,3 suffered from recurrence or distant metastasis.Conclusions Primary intraosseous synovial sarcoma is a rare malignant tumor with non-specific clinical manifestations.Imaging features typically include osteolytic destruction and intraosseous masses extending extraosseously,suggesting an intraosseous origin.Pathology and immunohistochemistry aid diagnosis,but definitive confirmation relies on further genetic testing.At present,the main treatment regimens for synovial sarcoma involve comprehensive therapies such as surgery and adjuvant chemotherapy,and the prognosis of patients is poor.
9.Psychological Characteristics and Pain Tolerance of Adolescents with Depression Experiencing Self-injury or Suicidality
Linyuan XU ; Huajun ZHAO ; Chao LI ; Yan SU ; Shihua BAO ; Lei WU ; Qingwei LI ; Bingkui ZHANG
Journal of Kunming Medical University 2025;46(1):68-77
Objective To explore the influencing factors of self injury or suicide in adolescent patients with depression.Methods A total of 96 adolescents who were diagnosed with depressive disorder at Yunnan Psychiatirc Hospital and Kunming Psychiatirc Hospital from January 2023 to May 2024 were selected for the study.The case group consisted of 61 adolescents who had self-injury or suicidal behaviors within 30 days,while the control group consisted of 35 adolescents who had no self-injury or suicidal behaviors within at least 30 days.Assessments were performed using the Patient Health Questionnaire-9(PHQ-9),Millon Screening Inventory for Borderline Personality Disorder(MSI-BPD),House-Tree-Person Drawing Test(HTP),and the Columbia Suicide Severity Rating Scale(C-SSRS).Among these,72 subjects underwent skin pressure pain threshold testing using the Algometer HP-50,with 12 in the self-harm or suicide group within 2 days and 60 in the group beyond 2 days.Results(1)Analysis of gender and age between the two groups showed statistically significant differences(P<0.05),with a higher proportion of females and younger ages in the 30-day group;(2)The PHQ-9 score for the 30-day group was(19.59±5.99),with a score of(2.41±0.97)for item 9("Thoughts that it would be better to be dead or to hurt oneself in some way"),and an MSI-BPD score of(7.43±1.61).The scores for the group over 30 days were(10.89±7.99),(1.00±1.11)for item 9,and(5.40±3.13)for MSI-BPD.Independent samples t-test analysis showed significant differences(P<0.01);(3)In the 30-day group,five drawing characteristics from the 47 HTP characteristics(shift of center of gravity,hair not disheveled or no hair,no mountains,no sun,and no flowers or grass)were found to occur at a higher rate,with statistical significance(P<0.05);(4)In the binary logistic regression analysis of 96 patients with the 30-day suicide or self-injury behavior as the dependent variable,the absence of flowers and PHQ-9 question 9 score are the risk factors for adolescent depression patients to have self-injury or suicide behavior within 30 days(OR no flowers=7.934,OR score on PHQ-9 question 9=3.554).(5)Among the 72 subjects the skin pressure pain threshold for adolescents who exhibited self-injure or suicide behaviors within 2 days(8.83±4.27 N)was significantly higher than that for patients beyond 2 days(6.48±3.00 N),with statistical significance(t=-2.201,P<0.05).Additionally,the score for item 9 of the PHQ-9 was higher(2.50±0.67 vs 1.87±0.95),also showing statistical significance(t=-2.201,P<0.05).Conclusion The absence of flowers or grass in HTP painting,and stronger suicidal ideation or suicide attempt are risk factors for self-injury or suicidal behavior in adolescents with depression.Furthermore,those who self-injure or commit suicide within 2 days have a higher pain threshold compared to those beyond 2 days.
10.Molecular targeted therapy for progressive low-grade gliomas in children.
Yan-Ling SUN ; Miao LI ; Jing-Jing LIU ; Wen-Chao GAO ; Yue-Fang WU ; Lu-Lu WAN ; Si-Qi REN ; Shu-Xu DU ; Wan-Shui WU ; Li-Ming SUN
Chinese Journal of Contemporary Pediatrics 2025;27(6):682-689
OBJECTIVES:
To evaluate the efficacy of molecular targeted agents in children with progressive pediatric low-grade gliomas (pLGG).
METHODS:
A retrospective analysis was conducted on pLGG patients treated with oral targeted therapies at the Department of Pediatrics, Beijing Shijitan Hospital, Capital Medical University, from July 2021. Treatment responses and safety profiles were assessed.
RESULTS:
Among the 20 enrolled patients, the trametinib group (n=12, including 11 cases with BRAF fusions and 1 case with BRAF V600E mutation) demonstrated 4 partial responses (33%) and 2 minor responses (17%), with a median time to response of 3.0 months. In the vemurafenib group (n=6, all with BRAF V600E mutation), 5 patients achieved partial responses (83%), showing a median time to response of 1.0 month. Comparative analysis revealed no statistically significant difference in progression-free survival rates between the two treatment groups (P>0.05). The median duration of clinical benefit (defined as partial response + minor response + stable disease) was 11.0 months for vemurafenib and 18.0 months for trametinib. Two additional cases, one with ATM mutation treated with olaparib for 24 months and one with NF1 mutation receiving everolimus for 21 months, discontinued treatment due to sustained disease stability. No severe adverse events were observed in any treatment group.
CONCLUSIONS
Molecular targeted therapy demonstrates clinical efficacy with favorable tolerability in pLGG. Vemurafenib achieves high response rates and induces early tumor shrinkage in patients with BRAF V600E mutations, supporting its utility as a first-line therapy.
Humans
;
Glioma/genetics*
;
Male
;
Female
;
Child
;
Child, Preschool
;
Retrospective Studies
;
Brain Neoplasms/genetics*
;
Molecular Targeted Therapy/adverse effects*
;
Adolescent
;
Infant
;
Proto-Oncogene Proteins B-raf/genetics*
;
Pyrimidinones/therapeutic use*
;
Mutation

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