1.Effect of tirofiban on prognosis in acute anterior circulation large vessel occlusion stroke patients with unsuccessful recanalization after endovascular treatment
Anyu LIAO ; Minxing ZHAO ; Kang YUAN ; Kangmo HUANG ; Zhiliang GUO ; Xianjun HUANG ; Zhenhui DUAN ; Rui LIU ; Guodong XIAO ; Wenhua LIU ; Zhiming ZHOU ; Wusheng ZHU
Chinese Journal of Cerebrovascular Diseases 2025;22(8):515-523,578
Objective To analyze the effects of intravenous tirofiban following endovascular treatment on the prognosis of patients with acute anterior circulation large vessel occlusion stroke who did not achieve successful recanalization.Methods This retrospective study included consecutive patients with acute anterior circulation ischemic stroke who underwent endovascular treatment but did not achieve successful recanalization.These patients were retrospectively enrolled from the Department of Neurology at Nanjing Jinling Hospital,Affiliated Hospital of Medical School,Nanjing University,the Second Affiliated Hospital of Soochow University,Wuhan No.1 Hospital,and Yijishan Hospital of Wannan Medical College(the First Affiliated Hospital of Wannan Medical College)between January 2015 and April 2023.Baseline and clinical data were collected including age,sex,medical history(hypertension,diabetes,atrial fibrillation,hyperlipidemia),personal history(smoking and drinking),National Institutes of Health stroke scale(NIHSS)score at admission,trial of Org 10172 in acute stroke treatment(TOAST)classification(large artery atherosclerosis,cardioembolism,or other types),Alberta stroke program early CT score(ASPECTS)on admission,intravenous thrombolysis,onset-to-puncture time(OTP),collateral circulation status(poor collaterals:American Society of Interventional and Therapeutic Neuroradiology/Society of Interventional Radiology[ASTIN/SIR]score 0-2;good collateral:ASTIN/SIR score 3-4),procedure duration(from femoral or radial artery puncture to device removal),occlusion site,number of passes for thrombus retrieval,and post-procedural modified thrombolysis in cerebral infarction(mTICI)grade.Patients were divided into a tirofiban group and a non-tirofiban group based on whether they received post-procedural intravenous tirofiban.The primary outcome is a favorable functional outcome at 90-day since onset(modified Rankin scale[mRS]score ≤ 3).Safety outcomes included mortality at 90-day since onset(mRS score 6),any intracranial hemorrhage within 24 h post-procedure,and symptomatic intracranial hemorrhage within 24 h post-procedure.1∶1 propensity score matchings using the nearest neighbor method was performed based on variables with P<0.05 in the comparison between the tirofiban and non-tirofiban groups.Differences in primary and safety outcomes between the matched groups were compared.Univariate and multivariate Logistic regression analyses were conducted with favorable outcomes at 90 days as the dependent variable to evaluate the effect of intravenous tirofiban administration after endovascular therapy on 90-day prognosis in patients without successful recanalization.Results A total of 356 patients without successful recanalization after endovascular treatment were included,comprising 195 males and 161 females,with a median age of 70(61,78)years(32-92 years).Among them,76 and 280patients were assigned to the tirofiban and non-tirofiban groups,respectively.At 90 days,114 patients had favorable outcomes,while 242 had unfavorable outcomes.(1)Before the 1∶1 propensity score matching,significant differences were observed between the tirofiban and non-tirofiban groups in terms of admission NIHSS score,ASPECTS,good collateral circulation,intravenous thrombolysis,and mTICI grade(all P<0.05).After 1∶1 propensity score matching,66 matched pairs(132 patients)were obtained.No significant differences in the baseline and clinical characteristics were found between the two groups after matching(all P>0.05).(2)After 1∶1 matching,a significant difference was observed in the rate of favorable outcomes at 90 days between the two groups(48.5%[32/66]vs.30.3%[20/66],P=0.033)after propensity score matching,while no significant differences were found in 90-day mortality,intracranial hemorrhage within 24h,or symptomatic intracranial hemorrhage within 24h(all P>0.05).(3)Univariate analysis after 1∶1 matching indicated that age,atrial fibrillation,smoking history,admission NIHSS score,M2 segment middle cerebral artery occlusion,good collateral circulation,number of passes for thrombus retrieval,and tirofiban treatment were factors influencing favorable outcomes at 90 days.Multivariate Logistic regression analysis showed that younger age(aOR,0.942,95%CI 0.906-0.978,P=0.002),lower admission NIHSS score(aOR,0.855,95%CI 0.777-0.941,P=0.001),good collateral circulation(aOR,5.534,95%CI 2.141-14.301,P<0.01),tirofiban treatment(aOR,2.774,95%CI 1.092-7.046,P=0.032),and M2 segment MCA occlusion(reference:internal carotid artery occlusion;aOR,4.874,95%CI 1.428-16.632,P=0.011)were independent predictors of favorable outcomes at 90 days.Conclusions Intravenous tirofiban administration after endovascular therapy may improve 90-day neurological outcomes in patients with acute anterior circulation large vessel occlusion stroke who did not achieve successful recanalization without increasing the risk of hemorrhage.Further large-scale randomized controlled trials are warranted to validate these findings.
2.Relationship between thyroid function abnormality and sindilizumab treatment effic-acy in patients with advanced gastric cancer
Gao WENHUA ; Mou KUN ; Fu YUAN ; Dai HUAN
Chinese Journal of Clinical Oncology 2025;52(17):877-883
Objective:To investigate the factors in fluencing thyroid function abnormality(TFA)and its relationship with the clinical efficacy of sindilizumab treatment in patients with advanced gastric cancer.Methods:The clinical data of 180 patients with advanced gastric cancer treated with sintilimab at Jinan People's Hospital from January 2021 to June 2024 were retrospectively reviewed.Patients were assigned in-to the TFA group and the normal control group according to thyroid function.Factors influencing the occurrence of TFA were investigated using the χ2 test and multifactorial Logistic regression.Kaplan-Meier analysis and the Log-rank test were used for comparison between groups.Univariate and multivariate Cox regression were used to assess the correlation between TFA and therapeutic efficacy.Results:A total of 52 patients developed TFA.Univariate analysis showed that history of thyroid nodule,treatment regimen,liver metastasis,and car-cinoembryonic antigen(CEA)were associated with the development of TFA.Multifactorial Logistic regression analysis showed that history of thyroid nodule,treatment regimen,and CEA were independent risk factors for the development of TFA(P<0.05).The objective response rate(ORR;34.6%vs.14.1%,P=0.002),disease control rate(DCR;69.2%vs.59.8%,P=0.024),and 1-year survival rate(59.6%vs.28.9%,P=0.012)were all better in the normal control group than those in the TFA group.Cox regression analysis showed that occurrence of TFA,liver meta-stasis,and≥6 treatment cycles were all independently associated with progression-free survival and overall survival in patients with ad-vanced gastric cancer(P<0.05).Conclusions:History of thyroid nodule,treatment regimen,and CEA may influence the occurrence of TFA.TFA may be a potential predictor of the efficacy of sindilizumab therapy for advanced gastric cancer,with patients who develop TFA poten-tially having better treatment outcomes.
3.Clinical characteristics and prognostic analysis of pulmonary mucormycosis in pediatric leukemia
Xiao FANG ; Huaili WANG ; Jiechao NIU ; Peina JIN ; Wenhua YUAN ; Haiying LI ; Erhu WEI
Chinese Journal of Applied Clinical Pediatrics 2025;40(5):350-356
Objective:To analyze the clinical characteristics of pulmonary mucormycosis (PM) in children with leukemia, and explore its diagnosis and treatment strategies and prognosis.Methods:In this case-series study, the clinical data of 19 children who were diagnosed with leukemia complicated by PM at the First Affiliated Hospital of Zhengzhou University from January 2020 to June 2024 were retrospectively analyzed.Their gender, age, type of leukemia, clinical manifestations, chest CT findings, bronchoscopy results, etiology, treatment regimens, and prognosis were summarized.The patients were divided into a survival group and a death group.The clinical data were compared between the 2 groups.Categorical variables were compared using the Fisher′s exact test, and continuous variables were compared using the Wilcoxon rank-sum test.Results:A total of 19 children aged 2 to 17 years with leukemia complicated by PM were involved, including 9 boys and 10 girls, with most cases (15 cases) occurring in the autumn and winter.The median time from the first leukemia chemotherapy to infection with mucor was 1 month.Clinical manifestations mainly included fever (100%), cough (94.7%), hemoptysis (42.1%), and pneumothorax (21.1%).Chest CT findings primarily showed extensive lung consolidation (84.2%), pleural effusion (84.2%), pulmonary nodules (78.9%), halo signs (73.7%), cavitation (36.8%), crescent signs (26.3%), and reverse halo signs (21.1%).Fourteen children underwent bronchoscopy, which primarily revealed tracheal obstruction, pale or congested mucosa, mucosal edema, mucosal necrosis, bronchial stenosis, and bronchial cavitary fistulas.Mucor was detected using various methods including metagenomic next-generation sequencing (mNGS), cultures, and lung tissue microscopy.Patients were mainly treated with intravenous administration of Amphotericin B in different formulations combined with oral Posaconazole.Three patients also underwent surgical resection of the affected lung lobe in addition to medical treatment.After treatment, 14 patients had a good prognosis, while 5 patients died.The causes of death were massive hemoptysis in 2 cases, severe respiratory failure in 1 case, and treatment withdrawn due to critical condition in 2 cases.There were no statistically significant differences in age, gender, type of leukemia, whether to undergo bronchoscopy and surgery, time from the first chemotherapy to onset of infection, presence of comorbid infection, chest CT characteristics, and time to start treatment between survival and death groups(all P>0.05). Conclusions:There is no specificity in clinical manifestations of leukemia complicated by PM, which, however, still exhibits some characteristics.mNGS plays a crucial role in early diagnosis.Intravenous administration of Amphotericin B combined with oral Posaconazole and surgical intervention are an effective treatment regimen.
4.Clinical characteristics and prognostic analysis of pulmonary mucormycosis in pediatric leukemia
Xiao FANG ; Huaili WANG ; Jiechao NIU ; Peina JIN ; Wenhua YUAN ; Haiying LI ; Erhu WEI
Chinese Journal of Applied Clinical Pediatrics 2025;40(5):350-356
Objective:To analyze the clinical characteristics of pulmonary mucormycosis (PM) in children with leukemia, and explore its diagnosis and treatment strategies and prognosis.Methods:In this case-series study, the clinical data of 19 children who were diagnosed with leukemia complicated by PM at the First Affiliated Hospital of Zhengzhou University from January 2020 to June 2024 were retrospectively analyzed.Their gender, age, type of leukemia, clinical manifestations, chest CT findings, bronchoscopy results, etiology, treatment regimens, and prognosis were summarized.The patients were divided into a survival group and a death group.The clinical data were compared between the 2 groups.Categorical variables were compared using the Fisher′s exact test, and continuous variables were compared using the Wilcoxon rank-sum test.Results:A total of 19 children aged 2 to 17 years with leukemia complicated by PM were involved, including 9 boys and 10 girls, with most cases (15 cases) occurring in the autumn and winter.The median time from the first leukemia chemotherapy to infection with mucor was 1 month.Clinical manifestations mainly included fever (100%), cough (94.7%), hemoptysis (42.1%), and pneumothorax (21.1%).Chest CT findings primarily showed extensive lung consolidation (84.2%), pleural effusion (84.2%), pulmonary nodules (78.9%), halo signs (73.7%), cavitation (36.8%), crescent signs (26.3%), and reverse halo signs (21.1%).Fourteen children underwent bronchoscopy, which primarily revealed tracheal obstruction, pale or congested mucosa, mucosal edema, mucosal necrosis, bronchial stenosis, and bronchial cavitary fistulas.Mucor was detected using various methods including metagenomic next-generation sequencing (mNGS), cultures, and lung tissue microscopy.Patients were mainly treated with intravenous administration of Amphotericin B in different formulations combined with oral Posaconazole.Three patients also underwent surgical resection of the affected lung lobe in addition to medical treatment.After treatment, 14 patients had a good prognosis, while 5 patients died.The causes of death were massive hemoptysis in 2 cases, severe respiratory failure in 1 case, and treatment withdrawn due to critical condition in 2 cases.There were no statistically significant differences in age, gender, type of leukemia, whether to undergo bronchoscopy and surgery, time from the first chemotherapy to onset of infection, presence of comorbid infection, chest CT characteristics, and time to start treatment between survival and death groups(all P>0.05). Conclusions:There is no specificity in clinical manifestations of leukemia complicated by PM, which, however, still exhibits some characteristics.mNGS plays a crucial role in early diagnosis.Intravenous administration of Amphotericin B combined with oral Posaconazole and surgical intervention are an effective treatment regimen.
5.Relationship between thyroid function abnormality and sindilizumab treatment effic-acy in patients with advanced gastric cancer
Gao WENHUA ; Mou KUN ; Fu YUAN ; Dai HUAN
Chinese Journal of Clinical Oncology 2025;52(17):877-883
Objective:To investigate the factors in fluencing thyroid function abnormality(TFA)and its relationship with the clinical efficacy of sindilizumab treatment in patients with advanced gastric cancer.Methods:The clinical data of 180 patients with advanced gastric cancer treated with sintilimab at Jinan People's Hospital from January 2021 to June 2024 were retrospectively reviewed.Patients were assigned in-to the TFA group and the normal control group according to thyroid function.Factors influencing the occurrence of TFA were investigated using the χ2 test and multifactorial Logistic regression.Kaplan-Meier analysis and the Log-rank test were used for comparison between groups.Univariate and multivariate Cox regression were used to assess the correlation between TFA and therapeutic efficacy.Results:A total of 52 patients developed TFA.Univariate analysis showed that history of thyroid nodule,treatment regimen,liver metastasis,and car-cinoembryonic antigen(CEA)were associated with the development of TFA.Multifactorial Logistic regression analysis showed that history of thyroid nodule,treatment regimen,and CEA were independent risk factors for the development of TFA(P<0.05).The objective response rate(ORR;34.6%vs.14.1%,P=0.002),disease control rate(DCR;69.2%vs.59.8%,P=0.024),and 1-year survival rate(59.6%vs.28.9%,P=0.012)were all better in the normal control group than those in the TFA group.Cox regression analysis showed that occurrence of TFA,liver meta-stasis,and≥6 treatment cycles were all independently associated with progression-free survival and overall survival in patients with ad-vanced gastric cancer(P<0.05).Conclusions:History of thyroid nodule,treatment regimen,and CEA may influence the occurrence of TFA.TFA may be a potential predictor of the efficacy of sindilizumab therapy for advanced gastric cancer,with patients who develop TFA poten-tially having better treatment outcomes.
6.Effect of tirofiban on prognosis in acute anterior circulation large vessel occlusion stroke patients with unsuccessful recanalization after endovascular treatment
Anyu LIAO ; Minxing ZHAO ; Kang YUAN ; Kangmo HUANG ; Zhiliang GUO ; Xianjun HUANG ; Zhenhui DUAN ; Rui LIU ; Guodong XIAO ; Wenhua LIU ; Zhiming ZHOU ; Wusheng ZHU
Chinese Journal of Cerebrovascular Diseases 2025;22(8):515-523,578
Objective To analyze the effects of intravenous tirofiban following endovascular treatment on the prognosis of patients with acute anterior circulation large vessel occlusion stroke who did not achieve successful recanalization.Methods This retrospective study included consecutive patients with acute anterior circulation ischemic stroke who underwent endovascular treatment but did not achieve successful recanalization.These patients were retrospectively enrolled from the Department of Neurology at Nanjing Jinling Hospital,Affiliated Hospital of Medical School,Nanjing University,the Second Affiliated Hospital of Soochow University,Wuhan No.1 Hospital,and Yijishan Hospital of Wannan Medical College(the First Affiliated Hospital of Wannan Medical College)between January 2015 and April 2023.Baseline and clinical data were collected including age,sex,medical history(hypertension,diabetes,atrial fibrillation,hyperlipidemia),personal history(smoking and drinking),National Institutes of Health stroke scale(NIHSS)score at admission,trial of Org 10172 in acute stroke treatment(TOAST)classification(large artery atherosclerosis,cardioembolism,or other types),Alberta stroke program early CT score(ASPECTS)on admission,intravenous thrombolysis,onset-to-puncture time(OTP),collateral circulation status(poor collaterals:American Society of Interventional and Therapeutic Neuroradiology/Society of Interventional Radiology[ASTIN/SIR]score 0-2;good collateral:ASTIN/SIR score 3-4),procedure duration(from femoral or radial artery puncture to device removal),occlusion site,number of passes for thrombus retrieval,and post-procedural modified thrombolysis in cerebral infarction(mTICI)grade.Patients were divided into a tirofiban group and a non-tirofiban group based on whether they received post-procedural intravenous tirofiban.The primary outcome is a favorable functional outcome at 90-day since onset(modified Rankin scale[mRS]score ≤ 3).Safety outcomes included mortality at 90-day since onset(mRS score 6),any intracranial hemorrhage within 24 h post-procedure,and symptomatic intracranial hemorrhage within 24 h post-procedure.1∶1 propensity score matchings using the nearest neighbor method was performed based on variables with P<0.05 in the comparison between the tirofiban and non-tirofiban groups.Differences in primary and safety outcomes between the matched groups were compared.Univariate and multivariate Logistic regression analyses were conducted with favorable outcomes at 90 days as the dependent variable to evaluate the effect of intravenous tirofiban administration after endovascular therapy on 90-day prognosis in patients without successful recanalization.Results A total of 356 patients without successful recanalization after endovascular treatment were included,comprising 195 males and 161 females,with a median age of 70(61,78)years(32-92 years).Among them,76 and 280patients were assigned to the tirofiban and non-tirofiban groups,respectively.At 90 days,114 patients had favorable outcomes,while 242 had unfavorable outcomes.(1)Before the 1∶1 propensity score matching,significant differences were observed between the tirofiban and non-tirofiban groups in terms of admission NIHSS score,ASPECTS,good collateral circulation,intravenous thrombolysis,and mTICI grade(all P<0.05).After 1∶1 propensity score matching,66 matched pairs(132 patients)were obtained.No significant differences in the baseline and clinical characteristics were found between the two groups after matching(all P>0.05).(2)After 1∶1 matching,a significant difference was observed in the rate of favorable outcomes at 90 days between the two groups(48.5%[32/66]vs.30.3%[20/66],P=0.033)after propensity score matching,while no significant differences were found in 90-day mortality,intracranial hemorrhage within 24h,or symptomatic intracranial hemorrhage within 24h(all P>0.05).(3)Univariate analysis after 1∶1 matching indicated that age,atrial fibrillation,smoking history,admission NIHSS score,M2 segment middle cerebral artery occlusion,good collateral circulation,number of passes for thrombus retrieval,and tirofiban treatment were factors influencing favorable outcomes at 90 days.Multivariate Logistic regression analysis showed that younger age(aOR,0.942,95%CI 0.906-0.978,P=0.002),lower admission NIHSS score(aOR,0.855,95%CI 0.777-0.941,P=0.001),good collateral circulation(aOR,5.534,95%CI 2.141-14.301,P<0.01),tirofiban treatment(aOR,2.774,95%CI 1.092-7.046,P=0.032),and M2 segment MCA occlusion(reference:internal carotid artery occlusion;aOR,4.874,95%CI 1.428-16.632,P=0.011)were independent predictors of favorable outcomes at 90 days.Conclusions Intravenous tirofiban administration after endovascular therapy may improve 90-day neurological outcomes in patients with acute anterior circulation large vessel occlusion stroke who did not achieve successful recanalization without increasing the risk of hemorrhage.Further large-scale randomized controlled trials are warranted to validate these findings.
7.A phase Ⅱ clinical study of the efficacy and safety of antaitasvir phosphate combined with yiqibuvir for the treatment of chronic hepatitis C in adults
Lai WEI ; Hongxin PIAO ; Jinglan JIN ; Shufen YUAN ; Xuan AN ; Jia SHANG ; Wenhua ZHANG ; Jiabao CHANG ; Tong SUN ; Yujuan GUAN ; Bo NING ; Jing ZHU ; Wentao GUO ; Qingwei HE ; Lin LUO ; Yulei ZHUANG ; Hongming XIE ; Yingjun ZHANG
Chinese Journal of Hepatology 2024;32(7):637-642
Objective:To evaluate the efficacy and safety of antaitasvir phosphate 100 mg or 200 mg combined with yiqibuvir for 12 weeks in patients with various genotypes of chronic hepatitis C, without cirrhosis or compensated stage cirrhosis.Methods:Patients with chronic hepatitis C (without cirrhosis or compensated stage cirrhosis) were randomly assigned to the antaitasvir phosphate 100 mg+yiqibuvir 600 mg group (100 mg group) or the antaitasvir phosphate 200 mg+yiqibuvir 600 mg group (200 mg group) in a 1∶1 ratio. The drugs were continuously administered once a day for 12 weeks and observed for 24 weeks after drug withdrawal. The drug safety profile was assessed concurrently with the observation of the sustained virological response (SVR12) in the two patient groups 12 weeks following the drug cessation. The intention-to-treat concept was used to define as closely as possible a full analysis set, including all randomized cases who received the experimental drug at least once. The safety set was collected from all subjects who received the experimental drug at least once (regardless of whether they participated in the randomization group) in this study. All efficacy endpoints and safety profile data were summarized using descriptive statistics. The primary efficacy endpoint was SVR12. The primary analysis was performed on a full analysis set. The frequency and proportion of cases were calculated in the experimental drug group (antaitasvir phosphate capsules combined with yiqibuvir tablets) that achieved "HCV RNA
8.Expert consensus on pediatric orthodontic therapies of malocclusions in children
Zhou CHENCHEN ; Duan PEIPEI ; He HONG ; Song JINLIN ; Hu MIN ; Liu YUEHUA ; Liu YAN ; Guo JIE ; Jin FANG ; Cao YANG ; Jiang LINGYONG ; Ye QINGSONG ; Zhu MIN ; Jiang BEIZHAN ; Ruan WENHUA ; Yuan XIAO ; Li HUANG ; Zou RUI ; Tian YULOU ; Gao LI ; Shu RUI ; Chen JIANWEI ; Liu RENKAI ; Zou SHUJUAN ; Li XIAOBING
International Journal of Oral Science 2024;16(2):186-196
Malocclusion,identified by the World Health Organization(WHO)as one of three major oral diseases,profoundly impacts the dental-maxillofacial functions,facial esthetics,and long-term development of~260 million children in China.Beyond its physical manifestations,malocclusion also significantly influences the psycho-social well-being of these children.Timely intervention in malocclusion can foster an environment conducive to dental-maxillofacial development and substantially decrease the incidence of malocclusion or reduce the severity and complexity of malocclusion in the permanent dentition,by mitigating the negative impact of abnormal environmental influences on the growth.Early orthodontic treatment encompasses accurate identification and treatment of dental and maxillofacial morphological and functional abnormalities during various stages of dental-maxillofacial development,ranging from fetal stages to the early permanent dentition phase.From an economic and societal standpoint,the urgency for effective early orthodontic treatments for malocclusions in childhood cannot be overstated,underlining its profound practical and social importance.This consensus paper discusses the characteristics and the detrimental effects of malocclusion in children,emphasizing critical need for early treatment.It elaborates on corresponding core principles and fundamental approaches in early orthodontics,proposing comprehensive guidance for preventive and interceptive orthodontic treatment,serving as a reference for clinicians engaged in early orthodontic treatment.
9.Construction of a core competency indicator system for oncology advanced practice nurses
Wenhua YU ; Yiyuan ZHAO ; Xiaoju ZHANG ; Zhihuan ZHOU ; Jinhua LI ; Liuliu ZHANG ; Li YIN ; Wanmin QIANG ; Huiyu LUO ; Guichun JIANG ; Yuan YU ; Yuhan LU
Chinese Journal of Modern Nursing 2024;30(10):1268-1275
Objective:To construct a core competency indicator system for oncology advanced practice nurses.Methods:This study is a cross-sectional study. A preliminary draft of the core competency indicator system for oncology advanced practice nurses was developed through literature review and expert group coordination from June to November 2022. The core competency indicator system for oncology advanced practice nurses was established using the Delphi method for expert consultation and the analytic hierarchy process.Results:A total of 54 experts from 11 hospitals and four medical schools in 10 provinces and municipalities directly under the central government across the country were included in two rounds of expert consultation. The effective response rates of the questionnaire were all 100%, with an expert authority coefficient of 0.90, Kendall coordination coefficients of 0.089 to 0.179 and 0.101 to 0.176 ( P<0.01). The final established core competency indicator system for oncology advanced practice nurses included seven primary indicators and 69 secondary indicators. Conclusions:The core competency indicator system for oncology advanced practice nurses is comprehensive and has the characteristics of specialized oncology nursing, and the construction process is scientific and reliable, laying the foundation for future training of oncology advanced practice nurses.
10.Long-term hypomethylating agents in patients with myelodysplastic syndromes: a multi-center retrospective study
Xiaozhen LIU ; Shujuan ZHOU ; Jian HUANG ; Caifang ZHAO ; Lingxu JIANG ; Yudi ZHANG ; Chen MEI ; Liya MA ; Xinping ZHOU ; Yanping SHAO ; Gongqiang WU ; Xibin XIAO ; Rongxin YAO ; Xiaohong DU ; Tonglin HU ; Shenxian QIAN ; Yuan LI ; Xuefen YAN ; Li HUANG ; Manling WANG ; Jiaping FU ; Lihong SHOU ; Wenhua JIANG ; Weimei JIN ; Linjie LI ; Jing LE ; Wenji LUO ; Yun ZHANG ; Xiujie ZHOU ; Hao ZHANG ; Xianghua LANG ; Mei ZHOU ; Jie JIN ; Huifang JIANG ; Jin ZHANG ; Guifang OUYANG ; Hongyan TONG
Chinese Journal of Hematology 2024;45(8):738-747
Objective:To evaluate the efficacy and safety of hypomethylating agents (HMA) in patients with myelodysplastic syndromes (MDS) .Methods:A total of 409 MDS patients from 45 hospitals in Zhejiang province who received at least four consecutive cycles of HMA monotherapy as initial therapy were enrolled to evaluate the efficacy and safety of HMA. Mann-Whitney U or Chi-square tests were used to compare the differences in the clinical data. Logistic regression and Cox regression were used to analyze the factors affecting efficacy and survival. Kaplan-Meier was used for survival analysis. Results:Patients received HMA treatment for a median of 6 cycles (range, 4-25 cycles) . The complete remission (CR) rate was 33.98% and the overall response rate (ORR) was 77.02%. Multivariate analysis revealed that complex karyotype ( P=0.02, OR=0.39, 95% CI 0.18-0.84) was an independent favorable factor for CR rate. TP53 mutation ( P=0.02, OR=0.22, 95% CI 0.06-0.77) was a predictive factor for a higher ORR. The median OS for the HMA-treated patients was 25.67 (95% CI 21.14-30.19) months. HMA response ( P=0.036, HR=0.47, 95% CI 0.23-0.95) was an independent favorable prognostic factor, whereas complex karyotype ( P=0.024, HR=2.14, 95% CI 1.10-4.15) , leukemia transformation ( P<0.001, HR=2.839, 95% CI 1.64-4.92) , and TP53 mutation ( P=0.012, HR=2.19, 95% CI 1.19-4.07) were independent adverse prognostic factors. There was no significant difference in efficacy and survival between the reduced and standard doses of HMA. The CR rate and ORR of MDS patients treated with decitabine and azacitidine were not significantly different. The median OS of patients treated with decitabine was longer compared with that of patients treated with azacitidine (29.53 months vs 20.17 months, P=0.007) . The incidence of bone marrow suppression and pneumonia in the decitabine group was higher compared with that in the azacitidine group. Conclusion:Continuous and regular use of appropriate doses of hypomethylating agents may benefit MDS patients to the greatest extent if it is tolerated.

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