1.Influence of Gene Mutation on the Effectiveness of Arsenic-Containing Herbal Compound Formula in Treatment of Myelodysplastic Syndromes of Different TCM Patterns
Zichun WANG ; Zhuo CHEN ; Dexiu WANG ; Haiyan XIAO ; Weiyi LIU ; Ruibai LI ; Chi LIU ; Fengmei WANG ; Shanshan ZHANG ; Mingjing WANG ; Liu LI ; Xiaoqing GUO ; Hongzhi WANG ; Xudong TANG
Journal of Traditional Chinese Medicine 2025;66(14):1463-1472
ObjectiveTo observe the effect of gene mutation on the effectiveness of arsenic-containing Chinese herbal compound formulas in the treatment of myelodysplastic syndromes (MDS) of different traditional Chinese medicine (TCM) patterns, so as to provide the basis for the clinical application. MethodsClinical data of 442 MDS patients who were treated with arsenic-containing herbal compound formulas were retrospectively collected, including the baseline demographic and clinical characteristics of the patients. Based on the TCM four examinations, the patients were divided into the spleen-kidney deficiency group as well as the qi-yin deficiency group, and according to the results of the next-generation sequencing (NGS) test, they were divided into the group with and without gene mutation respectively. The influence of gene mutation on the clinical effectiveness of patients with different TCM patterns was analyzed, the baseline demographic and clinical characteristics of the patients with different outcomes of the two TCM patterns were compared, and multivariate Logistic regression analysis was conducted on the influencing factors of the effective rate of MDS patients with gene mutation. ResultsA total of 190 cases were included in the spleen-kidney deficiency group (119 cases with gene mutation) and 43 cases in the qi-yin deficiency group (23 cases with gene mutation). No statistically significant differences were noted in effectiveness assessment, total effective rate, and total response rate between the spleen-kidney deficiency group and the qi-yin deficiency group (P>0.05). In the spleen-kidney deficiency group, the total effective rate of MDS with gene mutation was 65.55% (78/119), which was lower than 80.28% (57/71) of MDS without gene mutation, with statistical significance (P = 0.033), while no statistical differences in effectiveness assessment and total response rate were noted (P>0.05). In the qi-yin deficiency group, no statistical differences were observed in effectiveness assessment, total effective rate, and total response rate of the patients in with or without gene mutation (P>0.05). In the spleen-kidney deficiency group with gene mutation, the rate of complex karyotype (P = 0.031) and the mutation rate of CBL gene (P = 0.032) in the ineffective population were higher than those in the effective population, while the mutation rate of DDX41 gene in the effective population was higher than that in the ineffective population (P = 0.033). No statistically significant differences were found in other gene mutations, age, gender distribution, number of gene mutations, bone marrow hyperplasia degree, blast cell range, reticular fiber tissue proliferation or not, and prognosis of chromosomal abnormalities between the effective and ineffective populations (P>0.05). In the qi-yin deficiency group with gene mutation, no statistically significant differences were found in various items between populations with different outcomes (P>0.05). Multivariate Logistic regression analysis showed that complex karyotype, CBL mutation, and DDX41 mutation were independently associated with the effective rate of MDS with spleen-kidney deficiency and gene mutation (P<0.05). DDX41 mutation was an independent protective factor in the spleen-kidney deficiency group (OR>1), while complex karyotype and CBL mutation were independent risk factors (OR<1). ConclusionThe arsenic-containing TCM compound formulas exhibited better effectiveness in MDS with spleen-kidney deficiency pattern without mutation; and in MDS with spleen-kidney deficiency pattern without complex karyotypes, CBL mutation, and with DDX41 mutations. Furthermore, DDX41 mutation was an independent protective factor in the spleen-kidney deficiency group, while complex karyotype and CBL mutation were independent risk factors. In MDS with qi-yin deficiency pattern, gene mutation-related factors showed no significant impact on the effectiveness of arsenic-containing TCM compound formulas.
2.Clinical Study on Arsenic-containing TCM Compounds for the Treatment of High-risk Myelodysplastic Syndrome with Spleen-kidney Deficiency and Toxic Stagnation and Blood Stasis Syndrome
Nan JIN ; Yue MAO ; Yan LYU ; Zhuo CHEN ; Dexiu WANG ; Weiyi LIU ; Chi LIU ; Xudong TANG
Chinese Journal of Information on Traditional Chinese Medicine 2025;32(6):160-166
Objective To analyze the efficacy of arsenic-containing TCM compounds in treating high-risk myelodysplastic syndromes(HR-MDS)with spleen-kidney deficiency and toxic stagnation and blood stasis syndrome;To provide references for the clinical treatment of HR-MDS.Methods The medical records of HR-MDS with spleen-kidney deficiency and toxic stagnation and blood stasis syndrome treated with arsenic-containing TCM compounds in Xiyuan Hospital of China Academy of Chinese Medical Sciences from January 2016 to September 2022 were retrospectively analyzed.The patients were divided into arsenic-containing TCM compounds combined with demethylation drugs(HMAs)treatment group and arsenic-containing TCM compounds combined with androgen treatment group.The clinical efficacy of both groups were evaluated,including overall response rate(ORR),median overall survival(OS),1-,2-and 3-year survial rates,median progression free survival(PFS),and 1-,2-and 3-year PFS rates.Results Among 200 cases of HR-MDS,68 cases were treated with arsenic-containing TCM compounds combined with HMAs,and 132 cases were treated with arsenic-containing TCM compounds combined with androgen.The ORR was 30%,and the OS of HR-MDS after treatment was 42 months.The 1-,2-and 3-year survival rates were 78.6%,60.4%and 50.2%respectively.The median PFS was 15 months,and the 1-year,2-year and 3-year PFS rates were 57.8%,28.8%and 18.2%respectively.The ORR of 68 cases HR-MDS treated with arsenic-containing TCM compounds combined with HMAs was 44.1%.After treatment,the median OS of HR-MDS was 42 months,and the survival rates of 1-,2-and 3-years were 84%,75.6%and 61.9%respectively,and the median PFS was 24 months,and the PFS rates of 1-,2-and 3-years were 74.3%,48.5%and 35.2%,respectively.The ORR of 132 cases of HR-MDS treatmented with arsenic-containing TCM compounds combined with androgen was 26.5%,including 33.3%in the high-risk group of HR-MDS and 13.3%in the extremely high-risk group of HR-MDS.There was statistical significance between the two groups(P=0.014).After treatment,the median OS of HR-MDS was 29 months,and the survival rates of 1-,2-and 3 years were 78.6%,54.1%and 45.1%respectively.The median PFS was 13 months,and the PFS rates of 1-,2-and 3 years were 54.1%,28.2%and 12.9%respectively.Conclusion The arsenic-containing TCM compounds combined with HMAs can significantly prolong survival and delay disease progression in HR-MDS.Although the arsenic-based TCM compounds combined with androgen therapy demonstrate inferior efficacy compared to HMAs-based regimens,it has achieved significantly higher OS and PFS than historical controls,serving as an effective alternative for patients intolerant to HMAs.
3.Survival characteristics of myelodysplastic syndrome with U2AF1-mutation and the clinical efficacy of arsenic-containing traditional Chinese medicine compound prescription
Chaochang ZHANG ; Chi LIU ; Dexiu WANG ; Zhuo CHEN ; Yan LYU ; Haiyan XIAO ; Weiyi LIU ; Yonggang XU ; Xiupeng YANG ; Rou MA ; Xudong TANG
Journal of Beijing University of Traditional Chinese Medicine 2025;48(9):1211-1221
Objective To explore the clinical characteristics of myelodysplastic syndrome(MDS)with U2 small nuclear RNA cofactor 1(U2AF1)mutation in different age groups,as well as the efficacy and prognosis of an arsenic-containing traditional Chinese medicine(TCM)compound prescription(Qinghuang Capsules combined with Bushen Yijing Formula).Methods A retrospective analysis was conducted on the clinical data of patients with MDS who were hospitalized in the Hematology Department Ward of Xiyuan Hospital,China Academy of Chinese Medical Sciences,and received arsenic-containing TCM compound treatment from November 30,2020,to September 30,2023.Stratified by age,the U2AF1 mutation and wild-type groups aged<65 years and≥65 years were compared in terms of sex,TCM syndrome,World Health Organization classification,MDS Revised International Prognostic Score System(IPSS-R)score,blood routine indicators,serum lactate dehydrogenase content,nephroblastoma 1(WT1)expression level,bone marrow puncture and biopsy indicators,and chromosomal prognostic grades,et al.Furthermore,the efficacy of arsenic-containing TCM compound were compared in the U2AF1 mutation and wild-type groups among different age groups,as well as the influence of age on the survival prognosis of MDS patients with U2AF1 mutation.Results A total of 201 patients with MDS were included.104 patients were under 65 years old,among whom 20 had U2AF1 mutation,and 84 had wild-type.Ninety-seven patients were aged 65 years or older,among whom 19 patients had the U2AF1 mutation and 78 had the wild-type.Among patients aged<65 years,the U2AF1 mutation group had a higher proportion of male patients and very low-risk/low-risk patients with an IPSS-R score≤3(P<0.05),a lower mean corpuscular volume(MCV)(P<0.05),and a relatively higher proportion of peripheral blood cell line 1 reduction than the wild-type group(P<0.05).Among patients aged≥65 years,the MCV in the U2AF1 mutation group was lower(P<0.05),and the expression level of the bone marrow WT1 gene and the proportion of patients with reticular fiber grade 4 were relatively higher than in the wild-type group(P<0.05).The total effective rate of the arsenic-containing TCM compound for patients with U2AF1 mutation was 61.5%(24/39),and the total response rate was 30.8%(12/39).The total effective rate for the wild-type patients was 67.9%(110/162),and the total response rate was 29.6%(48/162).No significant difference was observed in the total effective and response rates.Kaplan-Meier survival analysis of 39 patients with U2AF1 mutation revealed that the median overall survival(mOS)of patients older than 65 years had not been reached.The 1-,2-,and 3-year survival rates were 93.8%,84.4%,and 84.4%,respectively.The mOS of the patients aged≥65 years was 35 months(95%confidence interval[CI]:7.559-62.441),and the 1-,2-,and 3-year survival rates were 66.2%,58.9%,and 29.4%,respectively.The mOS of patients in the aged≥65 years group was significantly lower than that in the aged<65 years group(P<0.05),and no significant difference was observed in median progression-free survival between the two groups.Conclusion The U2AF1 mutation is closely associated with the clinical characteristics of MDS.However,age and the presence of U2AF1 mutation have no significant effect on the total effective and response rates of arsenic-containing TCM compound.Age is a significant factor influencing the prognosis of patients with MDS with U2AF1 mutation.Patients aged 65 years or older have a shorter survival time than those younger than 65 years.
4.Survival characteristics of myelodysplastic syndrome with U2AF1-mutation and the clinical efficacy of arsenic-containing traditional Chinese medicine compound prescription
Chaochang ZHANG ; Chi LIU ; Dexiu WANG ; Zhuo CHEN ; Yan LYU ; Haiyan XIAO ; Weiyi LIU ; Yonggang XU ; Xiupeng YANG ; Rou MA ; Xudong TANG
Journal of Beijing University of Traditional Chinese Medicine 2025;48(9):1211-1221
Objective To explore the clinical characteristics of myelodysplastic syndrome(MDS)with U2 small nuclear RNA cofactor 1(U2AF1)mutation in different age groups,as well as the efficacy and prognosis of an arsenic-containing traditional Chinese medicine(TCM)compound prescription(Qinghuang Capsules combined with Bushen Yijing Formula).Methods A retrospective analysis was conducted on the clinical data of patients with MDS who were hospitalized in the Hematology Department Ward of Xiyuan Hospital,China Academy of Chinese Medical Sciences,and received arsenic-containing TCM compound treatment from November 30,2020,to September 30,2023.Stratified by age,the U2AF1 mutation and wild-type groups aged<65 years and≥65 years were compared in terms of sex,TCM syndrome,World Health Organization classification,MDS Revised International Prognostic Score System(IPSS-R)score,blood routine indicators,serum lactate dehydrogenase content,nephroblastoma 1(WT1)expression level,bone marrow puncture and biopsy indicators,and chromosomal prognostic grades,et al.Furthermore,the efficacy of arsenic-containing TCM compound were compared in the U2AF1 mutation and wild-type groups among different age groups,as well as the influence of age on the survival prognosis of MDS patients with U2AF1 mutation.Results A total of 201 patients with MDS were included.104 patients were under 65 years old,among whom 20 had U2AF1 mutation,and 84 had wild-type.Ninety-seven patients were aged 65 years or older,among whom 19 patients had the U2AF1 mutation and 78 had the wild-type.Among patients aged<65 years,the U2AF1 mutation group had a higher proportion of male patients and very low-risk/low-risk patients with an IPSS-R score≤3(P<0.05),a lower mean corpuscular volume(MCV)(P<0.05),and a relatively higher proportion of peripheral blood cell line 1 reduction than the wild-type group(P<0.05).Among patients aged≥65 years,the MCV in the U2AF1 mutation group was lower(P<0.05),and the expression level of the bone marrow WT1 gene and the proportion of patients with reticular fiber grade 4 were relatively higher than in the wild-type group(P<0.05).The total effective rate of the arsenic-containing TCM compound for patients with U2AF1 mutation was 61.5%(24/39),and the total response rate was 30.8%(12/39).The total effective rate for the wild-type patients was 67.9%(110/162),and the total response rate was 29.6%(48/162).No significant difference was observed in the total effective and response rates.Kaplan-Meier survival analysis of 39 patients with U2AF1 mutation revealed that the median overall survival(mOS)of patients older than 65 years had not been reached.The 1-,2-,and 3-year survival rates were 93.8%,84.4%,and 84.4%,respectively.The mOS of the patients aged≥65 years was 35 months(95%confidence interval[CI]:7.559-62.441),and the 1-,2-,and 3-year survival rates were 66.2%,58.9%,and 29.4%,respectively.The mOS of patients in the aged≥65 years group was significantly lower than that in the aged<65 years group(P<0.05),and no significant difference was observed in median progression-free survival between the two groups.Conclusion The U2AF1 mutation is closely associated with the clinical characteristics of MDS.However,age and the presence of U2AF1 mutation have no significant effect on the total effective and response rates of arsenic-containing TCM compound.Age is a significant factor influencing the prognosis of patients with MDS with U2AF1 mutation.Patients aged 65 years or older have a shorter survival time than those younger than 65 years.
5.Clinical Study on Arsenic-containing TCM Compounds for the Treatment of High-risk Myelodysplastic Syndrome with Spleen-kidney Deficiency and Toxic Stagnation and Blood Stasis Syndrome
Nan JIN ; Yue MAO ; Yan LYU ; Zhuo CHEN ; Dexiu WANG ; Weiyi LIU ; Chi LIU ; Xudong TANG
Chinese Journal of Information on Traditional Chinese Medicine 2025;32(6):160-166
Objective To analyze the efficacy of arsenic-containing TCM compounds in treating high-risk myelodysplastic syndromes(HR-MDS)with spleen-kidney deficiency and toxic stagnation and blood stasis syndrome;To provide references for the clinical treatment of HR-MDS.Methods The medical records of HR-MDS with spleen-kidney deficiency and toxic stagnation and blood stasis syndrome treated with arsenic-containing TCM compounds in Xiyuan Hospital of China Academy of Chinese Medical Sciences from January 2016 to September 2022 were retrospectively analyzed.The patients were divided into arsenic-containing TCM compounds combined with demethylation drugs(HMAs)treatment group and arsenic-containing TCM compounds combined with androgen treatment group.The clinical efficacy of both groups were evaluated,including overall response rate(ORR),median overall survival(OS),1-,2-and 3-year survial rates,median progression free survival(PFS),and 1-,2-and 3-year PFS rates.Results Among 200 cases of HR-MDS,68 cases were treated with arsenic-containing TCM compounds combined with HMAs,and 132 cases were treated with arsenic-containing TCM compounds combined with androgen.The ORR was 30%,and the OS of HR-MDS after treatment was 42 months.The 1-,2-and 3-year survival rates were 78.6%,60.4%and 50.2%respectively.The median PFS was 15 months,and the 1-year,2-year and 3-year PFS rates were 57.8%,28.8%and 18.2%respectively.The ORR of 68 cases HR-MDS treated with arsenic-containing TCM compounds combined with HMAs was 44.1%.After treatment,the median OS of HR-MDS was 42 months,and the survival rates of 1-,2-and 3-years were 84%,75.6%and 61.9%respectively,and the median PFS was 24 months,and the PFS rates of 1-,2-and 3-years were 74.3%,48.5%and 35.2%,respectively.The ORR of 132 cases of HR-MDS treatmented with arsenic-containing TCM compounds combined with androgen was 26.5%,including 33.3%in the high-risk group of HR-MDS and 13.3%in the extremely high-risk group of HR-MDS.There was statistical significance between the two groups(P=0.014).After treatment,the median OS of HR-MDS was 29 months,and the survival rates of 1-,2-and 3 years were 78.6%,54.1%and 45.1%respectively.The median PFS was 13 months,and the PFS rates of 1-,2-and 3 years were 54.1%,28.2%and 12.9%respectively.Conclusion The arsenic-containing TCM compounds combined with HMAs can significantly prolong survival and delay disease progression in HR-MDS.Although the arsenic-based TCM compounds combined with androgen therapy demonstrate inferior efficacy compared to HMAs-based regimens,it has achieved significantly higher OS and PFS than historical controls,serving as an effective alternative for patients intolerant to HMAs.
6.A Real-world Study on Bushen Jiedu Huayu Method in the Treatment of Higher-risk Myelodysplastic Syndromes
Jian LIU ; Rui LI ; Xiupeng YANG ; Hongzhi WANG ; Yonggang XU ; Zhuo CHEN ; Dexiu WANG ; Haiyan XIAO ; Xudong TANG
Chinese Journal of Information on Traditional Chinese Medicine 2024;31(9):145-151
Objective To explore the performance of routine blood test parameters,bone marrow parameters and the risk factors of leukemia conversion in higher-risk patients with myelodysplastic syndrome(MDS)treated with Bushen Jiedu Huayu Method in the real world.Methods The clinical data of 162 patients with higher-risk MDS who were admitted to the Department of Hematology,Xiyuan Hospital,China Academy of Chinese Medical Sciences from September 2017 to September 2022 were collected,and their clinical data,blood routine parameters,and bone marrow parameters were analyzed.Results A total of 162 higher-risk MDS patients were included,and the overall effective rate of the combination of traditional Chinese and Western medicine treatment,mainly using Bushen Jiedu Huayu Method being 48.8%.Patients with higher-risk MDS who were younger than 70 years old were more likely to obtain curative effect when treated with Bushen Jiedu Huayu Method combined with chemotherapy(P<0.05).After treatment with Bushen Jiedu Huayu Method,PLT levels in higher-risk MDS patients were significantly higher than those before treatment(P<0.05),and PLT levels in the ineffective group increased more significantly(P<0.05).After treatment,the HGB level in the effective group significantly increased(P<0.05).After treatment,the proportion of bone marrow granulocytes,megakaryocytes and lymphocytes in higher-risk MDS patients were significantly higher than those before treatment(P<0.05).Conclusion Bushen Jiedu Huayu Method,mainly using arsenic containing TCM compound,can treat higher-risk MDS.It can increase the HGB content and PLT level of patients,increase the proportion of bone marrow granulocytes,megakaryocytes and lymphocytes,and also play a certain role in reducing the proportion of bone marrow primitive cells,namely demethylation.
7.The clinical characteristics of chronic pancreatitis in children
Yongli FANG ; Jie WU ; Feihong YU ; Jin ZHOU ; Guoli WANG ; Dexiu GUAN ; Xiumin QIN
Chinese Pediatric Emergency Medicine 2024;31(10):756-760
Objective:To analyze the etiology of chronic pancreatitis(CP) and evaluate the impact of different intervention methods on the prognosis.Methods:This is a retrospective analysis conducted on clinical data of pediatric patients with CP admitted to Beijing Children's Hospital between January 2010 and December 2023,including etiology,clinical manifestations,imaging data and treatments.Follow-up assessments included height,weight,complications occurrence,and long-term nutritional status evaluated by using Z scores.Results:A total of 98 patients with CP were included in the study,containing 51 males and 47 females,with an age range of 1.95 to 15.96 years (median 8.49 years).The etiological contained the gene mutation (39.8%,39/98) (involving PRSS1, SPINK1, CFTR),the anatomical abnormality (26/98,26.5%),idiopathic pancreatitis (33.7%,33/98).Predominant clinical manifestations included abdominal pain (79/98,79.6%),nausea/vomit(48/98,49.0%),chest tightness/chest pain (10/98,10.2%),with malnutrition (44/98,44.9%) and the serum amylase increased in some patients.Imaging findings revealed heterogeneous pancreatic echoes,dilated pancreatic ducts and pancreatic stones via abdominal ultrasound,and a full or atrophic pancreas with irregular margins,tortuous or dilated pancreatic ducts through abdominal magnetic resonance imaging.Compared with CP caused by other reasons,hereditdry CP had a higher rate of pancreatic morphological changes(100.0% vs.88.1%, P<0.05).By March 2024,follow-up showed all 98 patients underwent initial medical treatment,followed by surgical intervention in 13 cases,endoscopic retrograde cholangiopancreatography intervention in 51 cases,and no surgical or endoscopic intervention in 34 cases.Six children developed diabetes,six had reduced fecal pancreatic elastase-1 but without fat diarrhea.Long-term follow-up indicated improved nutritional status among children who underwent endoscopic retrograde cholangiopancreatography intervention(Z score,-1.22 vs.0.74, P<0.001). Conclusion:Gene mutations and anatomical abnormalities is the main etiological factors in pediatric patients with CP.Early endoscopic intervention can significantly improve the long-term prognosis of the children.
8.Correlation Between Expression of HIF-1α and Prognosis of Arsenic-containing Chinese Herbal Compound in Treatment of Myelodysplastic Syndrome
Peizhen JIANG ; Xiupeng YANG ; Dexiu WANG ; Wenru WANG ; Yan LYU ; Weiyi LIU ; Zhuo CHEN ; Hongzhi WANG ; Xiaoqing GUO ; Shanshan ZHANG ; Yonggang XU ; Xudong TANG
Chinese Journal of Experimental Traditional Medical Formulae 2024;30(9):101-107
ObjectiveMyelodysplastic syndromes (MDS) is a group of clonal hematopoietic stem cell disorders,and this study aims to investigate the expression of hypoxia-inducible factor-1α(HIF-1α) in the bone marrow cells of patients with MDS and its correlation with the clinical features of MDS,the therapeutic efficacy of arsenic-containing Chineseherbal compound,and the survival prognosis. MethodAccording to the inclusion and exclusion criteria,27 MDS patients treated with arsenic-containing Chinese herbal compound in the Department of Hematology,Xiyuan Hospital,China Academy of Chinese Medical Sciences from January 2022 to September 2022 were included,and their bone marrow samples were collected by myelotomy. HIF-1α expression level in bone marrow cells was detected by real-time polymerase chain reaction (PCR) to analyze its correlation with clinical features,and logistic and Cox regression was used to analyze the risk factors affecting the efficacy and prognostic survival of MDS patients. ResultThe HIF-1α mRNA expression level was lower in bone marrow cells of MDS patients than in healthy subjects. HIF-1α was positively correlated with the degree of myelodysplasia(r=0.384,P<0.05) and bone marrow granulocytic system%(G%)(r=0.560,P<0.01). Logistic regression showed that HIF-1α was a risk factor for the prognosis in the follow-up of the efficacy of treatment(P<0.05)and Cox regression showed that HIF-1α was an independent factor affecting the survival prognosis of MDS patients [odds ratio(OR)=398.968,95% confidence interval(CI)(1.281,116 858.743),P<0.05]. ConclusionThe level of HIF-1α expression in bone marrow cells of MDS patients was closely related to the degree of clinical myelodysplasia and G%,and HIF-1α was a risk factor for the efficacy for and survival prognosis of MDS patients.
9.Survival characteristics of patients with high-risk myelodysplastic syndromes treated with an arsenic-containing herbal combination
Nan JIN ; Yue MAO ; Yan LYU ; Zhuo CHEN ; Dexiu WANG ; Weiyi LIU ; Chi LIU ; Xudong TANG
Journal of Beijing University of Traditional Chinese Medicine 2024;47(5):709-720
Objective To investigate the clinical characteristics of survival in patients with high-risk myelodysplastic syndromes(HR-MDS)and provide a reference for the clinical prognosis of patients with HR-MDS.Methods General data,blood routine test,bone marrow smear with histopathology,cytogenetics,and other clinical data of 200 patients diagnosed with HR-MDS at Xiyuan Hospital of China Academy of Chinese Medical Sciences,during the period of January 2016-September 2022,were retrospectively analyzed.The included patients were categorized into the arsenic-containing herbal combination combined with demethylating agents(HMAs)treatment group and the arsenic-containing Chinese medicine compound combined with androgen treatment group.The influence of clinical indices on the survival characteristics of each group was analyzed.Results Comparison of the impact of clinical indicators on survival in 200 patients with HR-MDS who were treated with arsenic-containing herbal compounds in combination with HMAs or androgens showed that high-risk vs.very high-risk(P=0.018),hemoglobin(Hb)<80 g/L vs.Hb≥80 g/L(P=0.035),platelet(PLT)counts<50×109 L-1 vs.PLT counts≥50×109 L-1(P<0.001),and the difference in median progression-free survival(PFS)time between myelodysplastic syndromes converted to leukemia(MDS-AML)and non-MDS-AML(P=0.003)were statistically significant.Comparison of survival effects of clinical indicators in 68 patients with HR-MDS who were treated with arsenic-containing Chinese medicine compound combined with HMAs showed that the difference in median PFS between PLT count<50×109 L-1 and PLT count≥50×109 L-1(P<0.001)and the difference in median PFS between<5 and≥5 courses of chemotherapy(P=0.018)were statistically significant.Comparison of survival effects of clinical indicators in 132 patients with HR-MDS who were treated with arsenic-containing Chinese medicine compound combined with androgens showed that Hb<80 g/L and Hg≥80 g/L(P=0.028),PLT count<50×109 L-1 and PLT count≥50×109 L-1(P=0.002),and the mean differences in PFS between MDS-AML and non-MDS-AML(P=0.024)were statistically significant.Conclusion The clinical characteristics of long-surviving patients treated with arsenic-containing herbal combination in combination with HMAs included PLT counts≥50×109 L-1 and≥5 courses of chemotherapy.The clinical characteristics of long-surviving patients treated with arsenic-containing herbal combination in combination with androgens included Hg≥80 g/L,PLT count≥50×109 L-1,and non-MDS-AML.
10.Analysis of the factors influencing the efficacy of glucocorticoids in the treatment of moderate to severe pediatric ulcerative colitis
Dexiu GUAN ; Jie WU ; Jing ZHANG ; Shu GUO ; Feihong YU ; Jin ZHOU ; Guoli WANG ; Xiwei XU
Chinese Journal of Inflammatory Bowel Diseases 2023;07(2):157-163
Objective:To explore the influencing factors of efficacy of glucocorticoids in the treatment of moderate to severe ulcerative colitis (UC) in children.Methods:A retrospective case-control study was conducted. The clinical data of 38 children with moderate to severe UC treated with glucocorticoids in Beijing Children's Hospital of Capital Medical University from January 2016 to December 2021 were analyzed. According to the response to glucocorticoids therapy, the patients were divided into steroid-intractable group and steroid-effective group. Kaplan-Meier method was used to calculate the cumulative recurrence rate. Univariate analysis was performed to analyze the differences of clinical data between the two groups, and Logistic regression was used to analyze the risk factors of steroid-intractable UC in children.Results:A total of 38 children with moderate to severe UC were enrolled, including 22 males and 16 females. The median onset age was 10.7 (8.5, 12.7) years old, and the median disease duration was 4.3 (1.1, 13.6) months. The cumulative recurrence rates of 38 UC patients at 3 months, 6 months, 1 year and 2 years after glucocorticoids treatment were 26.3%, 52.6%, 63.7% and 69.5%, respectively. There were 17 patients (44.7%) in steroid-effective group. There were 21 patients (55.3%) in steroid-intractable group, including 16 (42.1%) of steroid dependence and 5 (13.2%) of steroid resistance. In the steroid-intractable group, the PUCAI scores at baseline, on the 3rd day and 5th day of glucocorticoids treatment [65.0 (50.0, 70.0) points vs. 50.0 (37.5, 60.0) points, 25.0 (10.0, 37.5) points vs. 10.0 (10.0, 20.0) points, 25.0 (10.0, 37.5) points vs. 10.0 (5.0, 12.5) points] and early recurrence rate (85.7% vs. 35.3%) were higher than those in steroid-effective group, the recurrence time [4.0 (2.0, 5.0) months vs. 19.0 (7.5, 46.5) months] was shorter than that in steroid-effective group, albumin level at baseline [ (33.3 ± 5.5) g/L vs. (37.6 ± 5.9) g/L] was lower than that in steroid-effective group, and the differences were statistically significant (all P<0.05) . Multivariate Logistic regression analysis showed that the PUCAI score at baseline was an independent risk factor for steroid-intractable UC ( OR = 1.070, 95% CI: 1.011-1.132, P = 0.020) . Conclusions:The rates of steroid dependence and the steroid resistance are high in moderate to severe UC children. Children in the steroid-intractable group have an earlier recurrence time, a higher rate of early recurrence, and a lower albumin level at baseline. The steroid dependence and resistance are more likely to occur in children with moderate to severe UC when PUCAI score at baseline is high.

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