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Severe aplastic anemia (SAA) is a group of bone marrow hematopoietic failure syndromes caused by multiple causes.
The main treatments are hematopoietic stem cell transplantation and immunosuppressive therapy (IST).
Eltroipopag is an oral small molecule thrombopoietin receptor agonist.
The effectiveness and safety of combined with standard IST as the first-line treatment of SAA have been verified in phase II and phase III clinical trials.
Recently, a single-center, retrospective cohort study conducted by the Children's Hospital of Zhejiang University School of Medicine explored the combined use of Eltrombopag in IST in children with SAA.
In this regard, Yimaitong was fortunate to invite Professor Tang Yongmin and Professor Song Hua from the Children's Hospital of Zhejiang University School of Medicine to share the characteristics, purpose and results of the study.
Professor Tang Yongmin, Chief Physician, Doctoral Supervisor, Director of Hematology Oncology Center, Children's Hospital Affiliated to Zhejiang University School of Medicine, U.
S.
ECFMG Practicing Physician Certificate, Chairman, Pediatric Oncology Committee of China Anti-Cancer Association, Member of National Pediatric Leukemia Research Expert Committee Deputy Director, Pediatric Hematology Group, Pediatric Branch of the Chinese Medical Association Deputy Director, Pediatric Hematology Group, Zhejiang Pediatric Hematology Group, Zhejiang Pediatric Hematology Group, Deputy Director, Zhejiang Children’s Leukemia Diagnosis and Treatment Technology Research Center, Deputy Director, Hematology Society, Zhejiang Medical Association Vice Chairman of the Pediatric Oncology Committee of Zhejiang Anti-Cancer Association Vice Chairman of the Hematology Branch of Zhejiang Medical Doctor Association Member and Secretary-General of the Pediatric Hematology Committee of the Pediatrics Branch of the Chinese Medical Doctor Association Third of the Monoclonal Antibody Committee of the Chinese Medical Biotechnology Association Member of the Standing Committee of the Zhejiang Anti-Cancer Association, Member of the Seventh Council of Zhejiang Anti-Cancer Association, Vice Chairman of the Seventh Hematological and Lymphatic Tumor Professional Committee of Zhejiang Anti-Cancer Association, "Pediatric Blood & Cancer" Editorial Board "World Journal of Pediatrics" "American Journal" "of blood research", "Chinese Journal of Pediatrics", "Chinese Journal of Experimental Hematology", "Chinese Journal of Experimental Hematology", "Chinese Journal of Pediatric Blood and Tumor" deputy editor-in-chief won 19 national, provincial and ministerial scientific research projects, published more than 210 Chinese and English Articles, SC160 articles, 4 provincial and departmental achievements, 4 invention patents in both Chinese and English and 9 editors of higher medical education pediatric textbooks.
Professor Song Hua, chief physician, deputy director of the Department of Hematology and Oncology, is good at treating pediatric hematological diseases such as childhood leukemia , Treatment and comprehensive management of childhood hemophilia such as aplastic anemia, hemophagocytic syndrome, hemolytic anemia and thrombocytopenic purpura.
On the basis of routine treatment in the department of hematology, effective umbilical cord blood stem cell transplantation, related allogeneic hematopoietic stem cell transplantation, unrelated allogeneic hematopoietic stem cell transplantation and autologous treatment for children with refractory malignant tumors, aplastic anemia, and immunodeficiency have been effectively carried out.
Hematopoietic stem cell transplantation.
Undertook one project each from the Provincial Department of Health and the Provincial Education Commission, and participated in more than 10 related national and provincial-level projects.
More than 20 papers have been published in national primary and secondary medical journals.
Yimaitong: First of all, could you please introduce the current status of the treatment of severe aplastic anemia (SAA), and what are the unmet needs for the treatment of SAA? Professor Tang Yongmin’s changes in the blood picture caused by SAA, especially the reduction of white blood cell levels and platelet levels, can cause serious secondary infections and bleeding diseases.
Therefore, SAA is a clinical problem at present, and it still takes more time to explore the treatment of the disease.
.
At present, SAA treatment mainly adopts two methods, namely IST and hematopoietic stem cell transplantation.
For children with SAA, IST is the first choice of treatment, mainly using the standard IST regimen of antithymocyte globulin (ATG) combined with cyclosporin A (CsA).
The remission rate of this program in SAA children is about 50%, and only about 30% of children can be cured by IST treatment.
Most of the children still need hematopoietic stem cell transplantation.
The curative effect of SAA children receiving hematopoietic stem cell transplantation depends on the technical level of the transplantation and the matching degree of matching.
About 70%-90% of SAA children can be cured by hematopoietic stem cell transplantation.
Although hematopoietic stem cell transplantation has a good effect, hematopoietic stem cell transplantation also carries a greater risk.
Children with SAA may experience transplant failure, disease recurrence, and even serious complications leading to death.
How to improve the treatment of SAA children with poor efficacy of the standard IST regimen is a challenge facing SAA treatment.
Yimaitong: A study conducted by the Children's Hospital of Zhejiang University School of Medicine explored the efficacy of the standard IST regimen combined with the thrombopoietin receptor agonist etrobopar in children with SAA.
Could you please introduce the characteristics of this research? Professor Tang Yongmin's platelet level is an important indicator in the SAA treatment process.
The continuous decrease of platelet level will produce toxic and side effects and cause complications such as severe bleeding.
In severe cases, intracranial hemorrhage may lead to the death of children.
During SAA treatment, it is necessary to actively treat the reduction of platelet levels.
Although platelet transfusion will be performed during SAA treatment, the problem of platelet level reduction cannot be completely solved. In recent years, thrombopoietin receptor agonists such as Eltrombopag have been published one after another.
Many researchers have begun to explore whether adding Eltrombopag and other drugs to the treatment of IST regimens in SAA children can improve the efficacy.
The Children's Hospital of Zhejiang University School of Medicine has carried out a retrospective controlled study in recent years to compare the efficacy of the standard IST regimen in combination with Eltrobopar and standard IST in children with SAA.
After a long-term follow-up, the results of the study showed that the standard IST regimen combined with Eltroipopag showed a good effect, but the combined regimen requires a certain period of use to show the effectiveness of the treatment.
Children with SAA received the standard IST regimen combined with Eltrobopar after 3 months of treatment and did not show a significant efficacy advantage, but the combined regimen showed a better efficacy than the standard IST regimen after 6 months of treatment.
The study has been published in foreign SCI journals, initially confirming the efficacy of the standard IST regimen combined with Eltrobopar in SAA.
Yimaitong: A study conducted by the Children's Hospital of Zhejiang University School of Medicine explored the efficacy of the standard IST regimen combined with Eltroipopag in children with SAA.
Could you please introduce the purpose of the research and the expected results? Professor Song Hua SAA is a disease that may threaten the lives of children.
Current treatment methods include hematopoietic stem cell transplantation and IST.
However, some children with SAA have poor efficacy on standard IST regimens, with slower onset time and prolonged treatment time.
Eltrodippa is a thrombopoietin receptor agonist, which can increase the platelet level of SAA children and promote the growth and differentiation of stem cells.
The purpose of this study carried out by the Children’s Hospital of Zhejiang University School of Medicine is to compare the efficacy of the standard IST program and the program combined with Eltroipopag in children with SAA.
It is hoped that the combined program will improve the efficacy of SAA in children.
SAA children with poor efficacy of IST provide new treatment options.
Yimaitong: In the actual research process, how is the efficacy and safety of the combination of Eltrobopar and standard IST? Professor Song Hua's retrospective study included 57 children who received standard IST treatment (39 cases) or IST combined with Eltroipopag treatment (18 cases).
The results of the study showed that after 3 months of treatment, the ORR of the combined regimen group was 77.
7% and the CR rate was 22.
2%, while the ORR of the IST group was 56.
4%, and the CR rate was 12.
8%.
After 6 months of treatment, the 94.
4% ORR and 50% CR rates of the combined regimen group were significantly higher than the ORR (69.
2%) and CR rates (17.
9%) of the IST group (P<0.
05).
Common adverse events in the combined regimen group included mild to moderate increase in bilirubin levels, mild nausea, vomiting, and dizziness.
These adverse reactions were improved by symptomatic treatment.
No children discontinued the drug due to adverse reactions.
The addition of Eltrodopag to the standard IST can improve the hematological response of children with SSA, and the safety is good.
The research results support the use of standard IST combined with Eltrodopag in the treatment of children with SSA.
Yimaitong: What clinical benefits can the standard IST combined with Eltroipopag bring to children with SAA? Professor Song Hua's blood transfusion and infection are important indicators for the efficacy of SAA.
The results of this retrospective study show that the combined program can help children with SAA to get rid of blood transfusion earlier.
In the study, the median time for children in the IST group to escape from red blood cell transfusion was 51 days, and 35 days for the combined regimen group (p<0.
05); the median time for children in the IST group to escape from platelet transfusion was 53 days and the combined regimen group was 38 days (P<0.
05).
The 13 children in the combined regimen group were able to get rid of blood transfusion dependence within 1 month of treatment.
In addition, the median reticulocyte count (ARC) of children in the IST+EPAG group at the 8th week of treatment was significantly increased compared with that before treatment (1.
36×109/L vs 0.
38×109/L), the risk of infection was lowered, and the resistance during hospitalization was reduced.
The cost of infection treatment is reduced.
The treatment of Eltropapax also reduces the burden of follow-up treatment for children with SAA.
Twelve children in the IST group did not achieve remission (NR) after treatment, and 7 of them needed transplantation treatment; while the children in the Eltropapax group had better curative effects, and no children needed transplantation treatment.
Yimaitong: Based on the results of this research by your team, what other directions are worth exploring for the future use of IST combined with Eltrobopar in SAA? This study conducted by Professor Tang Yongmin at the Children's Hospital of Zhejiang University School of Medicine initially confirmed the efficacy of the IST combined with Eltroipopag regimen in SAA, and the results are encouraging.
However, the efficacy of the combined program in related foreign studies is still controversial.
Considering that this retrospective study conducted by the Children's Hospital of Zhejiang University School of Medicine has a relatively large time span and a relatively limited number of cases (only about 57 children were included), it is hoped that a prospective multi-center study can be carried out in the future to further verify The efficacy of IST combined with Eltroipopag regimen in children with SAA.
Yimaitong: In view of the excellent efficacy of Eltrombopag in children with SAA, what prospects do you have for the application of Eltrombopag in the future? Professor Tang Yongmin First of all, it is necessary to expand more cases in children with SAA in the future to further confirm the improvement of the efficacy of Atrobopar on SAA.
Secondly, due to oral administration of Eltrodopag, it has better compliance in children.
For children with immune thrombocytopenic purpura (ITP) and other blood diseases, the application prospects of Eltrodopax are also worth exploring.
.
In addition, childhood bone marrow failure syndrome is also a difficult problem in clinical practice, and the efficacy of Eltropapax in other bone marrow failure syndromes is also worthy of further exploration.
The MCC number REV21042096 is valid for 2022-04-21, and the information is expired and deemed invalid.
Poke "read the original text" and we will make progress together
The main treatments are hematopoietic stem cell transplantation and immunosuppressive therapy (IST).
Eltroipopag is an oral small molecule thrombopoietin receptor agonist.
The effectiveness and safety of combined with standard IST as the first-line treatment of SAA have been verified in phase II and phase III clinical trials.
Recently, a single-center, retrospective cohort study conducted by the Children's Hospital of Zhejiang University School of Medicine explored the combined use of Eltrombopag in IST in children with SAA.
In this regard, Yimaitong was fortunate to invite Professor Tang Yongmin and Professor Song Hua from the Children's Hospital of Zhejiang University School of Medicine to share the characteristics, purpose and results of the study.
Professor Tang Yongmin, Chief Physician, Doctoral Supervisor, Director of Hematology Oncology Center, Children's Hospital Affiliated to Zhejiang University School of Medicine, U.
S.
ECFMG Practicing Physician Certificate, Chairman, Pediatric Oncology Committee of China Anti-Cancer Association, Member of National Pediatric Leukemia Research Expert Committee Deputy Director, Pediatric Hematology Group, Pediatric Branch of the Chinese Medical Association Deputy Director, Pediatric Hematology Group, Zhejiang Pediatric Hematology Group, Zhejiang Pediatric Hematology Group, Deputy Director, Zhejiang Children’s Leukemia Diagnosis and Treatment Technology Research Center, Deputy Director, Hematology Society, Zhejiang Medical Association Vice Chairman of the Pediatric Oncology Committee of Zhejiang Anti-Cancer Association Vice Chairman of the Hematology Branch of Zhejiang Medical Doctor Association Member and Secretary-General of the Pediatric Hematology Committee of the Pediatrics Branch of the Chinese Medical Doctor Association Third of the Monoclonal Antibody Committee of the Chinese Medical Biotechnology Association Member of the Standing Committee of the Zhejiang Anti-Cancer Association, Member of the Seventh Council of Zhejiang Anti-Cancer Association, Vice Chairman of the Seventh Hematological and Lymphatic Tumor Professional Committee of Zhejiang Anti-Cancer Association, "Pediatric Blood & Cancer" Editorial Board "World Journal of Pediatrics" "American Journal" "of blood research", "Chinese Journal of Pediatrics", "Chinese Journal of Experimental Hematology", "Chinese Journal of Experimental Hematology", "Chinese Journal of Pediatric Blood and Tumor" deputy editor-in-chief won 19 national, provincial and ministerial scientific research projects, published more than 210 Chinese and English Articles, SC160 articles, 4 provincial and departmental achievements, 4 invention patents in both Chinese and English and 9 editors of higher medical education pediatric textbooks.
Professor Song Hua, chief physician, deputy director of the Department of Hematology and Oncology, is good at treating pediatric hematological diseases such as childhood leukemia , Treatment and comprehensive management of childhood hemophilia such as aplastic anemia, hemophagocytic syndrome, hemolytic anemia and thrombocytopenic purpura.
On the basis of routine treatment in the department of hematology, effective umbilical cord blood stem cell transplantation, related allogeneic hematopoietic stem cell transplantation, unrelated allogeneic hematopoietic stem cell transplantation and autologous treatment for children with refractory malignant tumors, aplastic anemia, and immunodeficiency have been effectively carried out.
Hematopoietic stem cell transplantation.
Undertook one project each from the Provincial Department of Health and the Provincial Education Commission, and participated in more than 10 related national and provincial-level projects.
More than 20 papers have been published in national primary and secondary medical journals.
Yimaitong: First of all, could you please introduce the current status of the treatment of severe aplastic anemia (SAA), and what are the unmet needs for the treatment of SAA? Professor Tang Yongmin’s changes in the blood picture caused by SAA, especially the reduction of white blood cell levels and platelet levels, can cause serious secondary infections and bleeding diseases.
Therefore, SAA is a clinical problem at present, and it still takes more time to explore the treatment of the disease.
.
At present, SAA treatment mainly adopts two methods, namely IST and hematopoietic stem cell transplantation.
For children with SAA, IST is the first choice of treatment, mainly using the standard IST regimen of antithymocyte globulin (ATG) combined with cyclosporin A (CsA).
The remission rate of this program in SAA children is about 50%, and only about 30% of children can be cured by IST treatment.
Most of the children still need hematopoietic stem cell transplantation.
The curative effect of SAA children receiving hematopoietic stem cell transplantation depends on the technical level of the transplantation and the matching degree of matching.
About 70%-90% of SAA children can be cured by hematopoietic stem cell transplantation.
Although hematopoietic stem cell transplantation has a good effect, hematopoietic stem cell transplantation also carries a greater risk.
Children with SAA may experience transplant failure, disease recurrence, and even serious complications leading to death.
How to improve the treatment of SAA children with poor efficacy of the standard IST regimen is a challenge facing SAA treatment.
Yimaitong: A study conducted by the Children's Hospital of Zhejiang University School of Medicine explored the efficacy of the standard IST regimen combined with the thrombopoietin receptor agonist etrobopar in children with SAA.
Could you please introduce the characteristics of this research? Professor Tang Yongmin's platelet level is an important indicator in the SAA treatment process.
The continuous decrease of platelet level will produce toxic and side effects and cause complications such as severe bleeding.
In severe cases, intracranial hemorrhage may lead to the death of children.
During SAA treatment, it is necessary to actively treat the reduction of platelet levels.
Although platelet transfusion will be performed during SAA treatment, the problem of platelet level reduction cannot be completely solved. In recent years, thrombopoietin receptor agonists such as Eltrombopag have been published one after another.
Many researchers have begun to explore whether adding Eltrombopag and other drugs to the treatment of IST regimens in SAA children can improve the efficacy.
The Children's Hospital of Zhejiang University School of Medicine has carried out a retrospective controlled study in recent years to compare the efficacy of the standard IST regimen in combination with Eltrobopar and standard IST in children with SAA.
After a long-term follow-up, the results of the study showed that the standard IST regimen combined with Eltroipopag showed a good effect, but the combined regimen requires a certain period of use to show the effectiveness of the treatment.
Children with SAA received the standard IST regimen combined with Eltrobopar after 3 months of treatment and did not show a significant efficacy advantage, but the combined regimen showed a better efficacy than the standard IST regimen after 6 months of treatment.
The study has been published in foreign SCI journals, initially confirming the efficacy of the standard IST regimen combined with Eltrobopar in SAA.
Yimaitong: A study conducted by the Children's Hospital of Zhejiang University School of Medicine explored the efficacy of the standard IST regimen combined with Eltroipopag in children with SAA.
Could you please introduce the purpose of the research and the expected results? Professor Song Hua SAA is a disease that may threaten the lives of children.
Current treatment methods include hematopoietic stem cell transplantation and IST.
However, some children with SAA have poor efficacy on standard IST regimens, with slower onset time and prolonged treatment time.
Eltrodippa is a thrombopoietin receptor agonist, which can increase the platelet level of SAA children and promote the growth and differentiation of stem cells.
The purpose of this study carried out by the Children’s Hospital of Zhejiang University School of Medicine is to compare the efficacy of the standard IST program and the program combined with Eltroipopag in children with SAA.
It is hoped that the combined program will improve the efficacy of SAA in children.
SAA children with poor efficacy of IST provide new treatment options.
Yimaitong: In the actual research process, how is the efficacy and safety of the combination of Eltrobopar and standard IST? Professor Song Hua's retrospective study included 57 children who received standard IST treatment (39 cases) or IST combined with Eltroipopag treatment (18 cases).
The results of the study showed that after 3 months of treatment, the ORR of the combined regimen group was 77.
7% and the CR rate was 22.
2%, while the ORR of the IST group was 56.
4%, and the CR rate was 12.
8%.
After 6 months of treatment, the 94.
4% ORR and 50% CR rates of the combined regimen group were significantly higher than the ORR (69.
2%) and CR rates (17.
9%) of the IST group (P<0.
05).
Common adverse events in the combined regimen group included mild to moderate increase in bilirubin levels, mild nausea, vomiting, and dizziness.
These adverse reactions were improved by symptomatic treatment.
No children discontinued the drug due to adverse reactions.
The addition of Eltrodopag to the standard IST can improve the hematological response of children with SSA, and the safety is good.
The research results support the use of standard IST combined with Eltrodopag in the treatment of children with SSA.
Yimaitong: What clinical benefits can the standard IST combined with Eltroipopag bring to children with SAA? Professor Song Hua's blood transfusion and infection are important indicators for the efficacy of SAA.
The results of this retrospective study show that the combined program can help children with SAA to get rid of blood transfusion earlier.
In the study, the median time for children in the IST group to escape from red blood cell transfusion was 51 days, and 35 days for the combined regimen group (p<0.
05); the median time for children in the IST group to escape from platelet transfusion was 53 days and the combined regimen group was 38 days (P<0.
05).
The 13 children in the combined regimen group were able to get rid of blood transfusion dependence within 1 month of treatment.
In addition, the median reticulocyte count (ARC) of children in the IST+EPAG group at the 8th week of treatment was significantly increased compared with that before treatment (1.
36×109/L vs 0.
38×109/L), the risk of infection was lowered, and the resistance during hospitalization was reduced.
The cost of infection treatment is reduced.
The treatment of Eltropapax also reduces the burden of follow-up treatment for children with SAA.
Twelve children in the IST group did not achieve remission (NR) after treatment, and 7 of them needed transplantation treatment; while the children in the Eltropapax group had better curative effects, and no children needed transplantation treatment.
Yimaitong: Based on the results of this research by your team, what other directions are worth exploring for the future use of IST combined with Eltrobopar in SAA? This study conducted by Professor Tang Yongmin at the Children's Hospital of Zhejiang University School of Medicine initially confirmed the efficacy of the IST combined with Eltroipopag regimen in SAA, and the results are encouraging.
However, the efficacy of the combined program in related foreign studies is still controversial.
Considering that this retrospective study conducted by the Children's Hospital of Zhejiang University School of Medicine has a relatively large time span and a relatively limited number of cases (only about 57 children were included), it is hoped that a prospective multi-center study can be carried out in the future to further verify The efficacy of IST combined with Eltroipopag regimen in children with SAA.
Yimaitong: In view of the excellent efficacy of Eltrombopag in children with SAA, what prospects do you have for the application of Eltrombopag in the future? Professor Tang Yongmin First of all, it is necessary to expand more cases in children with SAA in the future to further confirm the improvement of the efficacy of Atrobopar on SAA.
Secondly, due to oral administration of Eltrodopag, it has better compliance in children.
For children with immune thrombocytopenic purpura (ITP) and other blood diseases, the application prospects of Eltrodopax are also worth exploring.
.
In addition, childhood bone marrow failure syndrome is also a difficult problem in clinical practice, and the efficacy of Eltropapax in other bone marrow failure syndromes is also worthy of further exploration.
The MCC number REV21042096 is valid for 2022-04-21, and the information is expired and deemed invalid.
Poke "read the original text" and we will make progress together
