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Severe aplastic anemia (SAA) is a bone marrow hematopoietic failure syndrome with dysplasia of bone marrow and lack of hematopoietic stem cells.
At present, the main treatments are allogeneic hematopoietic stem cell transplantation and immunosuppressive therapy (IST)
.
With the emergence of transplantation techniques and IST combined treatment options such as Eltrobopar combined with IST, the survival status of SAA patients has been significantly improved
.
The 63rd American Hematology Annual Meeting (ASH) will be held online and online at the same time from December 11th to 14th, 2021.
At this ASH meeting, two studies by Prof.
He Guangsheng’s team "Intensive Immunosuppressive Therapy Combined with Eltrobopar "Chinese Adult SAA"1 and "Etrobopar combined with intensive immunosuppressive regimen in the treatment of Chinese SAA patients with efficacy predictors"2 were included in the ASH poster
.
On this occasion, Yimaitong invited Professor He Guangsheng from Jiangsu Provincial People's Hospital to accept an interview to share the status of SAA diagnosis and treatment and the clinical research progress of the treatment plan containing Eltrombopag
.
Yimaitong: Could you please introduce the current status of SAA diagnosis and treatment in my country, and what are the unmet needs for the treatment of SAA patients? Professor He Guangsheng’s "disease" of severe aplastic anemia is as its name suggests.
The patient has severe bone marrow hyperplasia, hematopoietic failure, and severe reduction of white blood cells, red blood cells and platelets, and it will cause a series of complications such as infection, bleeding and anemia, which is a serious threat Human life and health
.
The current SAA treatments mainly include allogeneic hematopoietic stem cell transplantation and IST
.
Due to the limitation of transplantation conditions, IST is usually selected clinically
.
Although the effective rate of IST treatment is relatively high, and most patients can escape from blood transfusion, the overall complete response rate (CR) is still low
.
In addition, many patients are at long-term risk of recurrence
.
Therefore, how to make patients obtain effective treatment, increase the CR rate, and reduce the long-term recurrence rate is still very difficult
.
Yimaitong: At this ASH annual meeting, your team’s article on the treatment of SAA in Chinese adults with Eltrobopar combined with IST was selected in the ASH poster.
Can you please discuss the treatment plan in SAA patients based on the results of the study? How effective is it in? Prof.
He Guangsheng Eltroipopag is an oral small-molecule thrombopoietin receptor agonist, which can promote the proliferation and differentiation of bone marrow hematopoietic stem cells and increase the level of three-line blood cells
.
At present, the data on the treatment of SAA patients with Eltrombopag combined with IST are mostly concentrated in European and American populations, and the results of real-world studies in China are still unclear
.
In addition, IST therapy at home and abroad differs greatly in the selection of anti-thymocyte/lymphocyte immunoglobulin (ATG/ALG) species and the dosage of Eltroipopag
.
It is urgent to explore the effect of the combined regimen on SAA patients in China .
After years of clinical accumulation and data analysis, our team found that at 1, 3, 6, and 12 months after IST treatment, the overall response rate (ORR) of the IST alone or IST combined with Eltroipopag treatment group was 12% vs 35%, respectively (P=0.
002), 44%vs64%(P=0.
028), 61%vs85%(P=0.
006) and 73%vs 91%(P=0.
031)
.
The CR rates at the 3rd, 6th, and 12th months after receiving IST or IST combined with Eltrombopag were 7% vs 17% (P=0.
069), 14% vs 27% (P=0.
11), and 33% vs 32% (P= 0.
92)
.
Analysis of factors related to the efficacy of patients treated for 6 months showed that ORR was significantly related to the use of Eltrodopag (P=0.
011, OR=3.
600, 95%CI 1.
345-9.
638), and the safety was good
.
Yimaitong: In this study, there was no significant difference in the CR rate of patients receiving IST and the combined IST regimen of Eltrodopax.
It is different from the result that the CR rate of the combined group of Eltrodopax in the EBMT RACE study was significantly increased.
What do you think is the reason? What? Prof.
He Guangsheng First of all, from a pathophysiological point of view, Eltrobomide is a blood-promoting drug.
Combining it with IST has the advantage of allowing patients to recover faster and get remission in the early stage
.
At the end of treatment, perhaps because IST has sufficient time and space to "play", the CR rate of patients with IST alone has gradually increased, resulting in no significant difference in the CR rate of patients with the two regimens in this study
.
In addition, the RACE study 3 of EBMT is different from the ATG species we selected, and considering the differences in metabolism between East Asian populations and European and American populations, the domestic dose of etrobopar is only 1/2 (75mg/d) of foreign countries
.
There are reports that different species of ATG may cause differences in the CR rate of patients
.
Therefore, the results of domestic and foreign research may be inconsistent
.
In the follow-up, we will further explore the effect of IST combined with different doses of Eltrodipam on SAA patients, in order to further improve the efficacy while maintaining patient tolerance; in addition, if conditions are mature, consider using porcine ALG-based IST combined Clinical research is carried out on Eltrombopag
.
It is hoped that workers in the field of aplastic anemia (AA) across the country will work together to accumulate clinical experience and data to jointly benefit patients
.
Yimaitong: Another study published by your team on the efficacy predictors of Eltrombopag combined with IST in the treatment of SAA patients in China was also included in this year’s ASH poster.
Could you please discuss the impact of Eltrobopar with the results of the research? Correlative predictors of the efficacy of Pa in the treatment of SAA patients? Professor He Guangsheng lacked research on predictors of the efficacy of this combination scheme in the past, so this study is of great significance
.
By comparing the clinical conditions of patients with effective and ineffective use of Eltrodopa, the ROC curve was used to predict the efficacy factors of Eltrodopa
.
The results showed that lymphocyte count (P=0.
042, AUC=0.
721, 95%CI 0.
537-0.
904), percentage of reticulocytes (P=0.
007, AUC=0.
793, 95%CI 0.
631-0.
955), red blood cell distribution width-coefficient of variation (RDW-CV) (P=0.
042, AUC=0.
721, 95%CI 0.
493-0.
948) was positively correlated with the efficacy of etrobopar
.
A higher lymphocyte count, percentage of reticulocytes, and RDW-CV may indicate better function of the remaining bone marrow
.
If the patient has better residual hematopoietic function, and at the same time, after IST “turns out the chaos” and adjusts the abnormal immunity, the hematopoietic drugs will be able to play a better role and advantage
.
Yimaitong: In recent years, with the advancement of medical technology and the continuous emergence of new drugs, the survival and prognosis of SAA patients have been significantly improved.
What are your prospects for the future treatment of SAA? Professor He Guangsheng firstly for allogeneic hematopoietic stem cell transplantation technology, through further optimization and combination of pretreatment plan and anti-rejection drug plan, expand the transplant applicable population, benefit patients
.
Taking the new pretreatment program as an example, it can further reduce the damage to the patient's reproductive function and reduce the risk of the second tumor in the later stage
.
In addition, the improvement of the combination of drugs such as Eltrombopag and IST is particularly worthy of attention
.
Taking Eltrobopar as an example, how to choose the dosage, time, nodes, structure, etc.
to further increase the patient's CR rate, reduce the recurrence rate, reduce the risk of transformation, and improve the prognosis and survival of patients still has a long way to go
.
Summary The above studies have not only verified and affirmed the effectiveness and safety of the combined IST regimen in Chinese adult SAA treatment, but also filled the gaps in the efficacy predictors of the combined regimen, which is of great significance
.
However, SAA treatment still has a long way to go.
How to further optimize the medication regimen and improve the prognosis and survival of patients requires the joint efforts of clinical and scientific researchers across the country
.
Prof.
He Guangsheng Deputy Director, Department of Hematology, First Affiliated Hospital of Nanjing Medical University, Jiangsu Provincial People's Hospital Chief Expert of Department of Hematology, Nanjing Hospital Affiliated to Nanjing University of Traditional Chinese Medicine Chief Expert of Department of Hematology, Second Affiliated Hospital of Xuzhou Medical University Chairman of Anemia Branch of Jiangsu Research Hospital Association Jiangsu Deputy Leader of the Red Cell Disease Group of the Hematology Branch of the Provincial Medical Association Member of the 7th and 8th Anemia Group of the Chinese Society of Hematology and Academic Secretary "Chinese Journal of Practical Internal Medicine" Standing Editor, National Health Commission "14th Five-Year Plan" textbook editor The editor in charge of Hematology of the Journal of the Journal of Transitional International Medicine of the Committee won the second prize of the Chinese Medical Award and the "Chen Jinrong Life Science Excellence Award" of Soochow University.
Baidu Academic (until 2017) shows: H index 26, G index 55 References: 1.
Ruixin Li, et al.
ASH 2021 abstract 2190.
2.
Ruixin Li, et al.
ASH 2021 abstract 1115.
3.
De Latour RP, et al.
EBMT 2020 abstract 0018.
Stamp "read the original text" and we will make progress together
At present, the main treatments are allogeneic hematopoietic stem cell transplantation and immunosuppressive therapy (IST)
.
With the emergence of transplantation techniques and IST combined treatment options such as Eltrobopar combined with IST, the survival status of SAA patients has been significantly improved
.
The 63rd American Hematology Annual Meeting (ASH) will be held online and online at the same time from December 11th to 14th, 2021.
At this ASH meeting, two studies by Prof.
He Guangsheng’s team "Intensive Immunosuppressive Therapy Combined with Eltrobopar "Chinese Adult SAA"1 and "Etrobopar combined with intensive immunosuppressive regimen in the treatment of Chinese SAA patients with efficacy predictors"2 were included in the ASH poster
.
On this occasion, Yimaitong invited Professor He Guangsheng from Jiangsu Provincial People's Hospital to accept an interview to share the status of SAA diagnosis and treatment and the clinical research progress of the treatment plan containing Eltrombopag
.
Yimaitong: Could you please introduce the current status of SAA diagnosis and treatment in my country, and what are the unmet needs for the treatment of SAA patients? Professor He Guangsheng’s "disease" of severe aplastic anemia is as its name suggests.
The patient has severe bone marrow hyperplasia, hematopoietic failure, and severe reduction of white blood cells, red blood cells and platelets, and it will cause a series of complications such as infection, bleeding and anemia, which is a serious threat Human life and health
.
The current SAA treatments mainly include allogeneic hematopoietic stem cell transplantation and IST
.
Due to the limitation of transplantation conditions, IST is usually selected clinically
.
Although the effective rate of IST treatment is relatively high, and most patients can escape from blood transfusion, the overall complete response rate (CR) is still low
.
In addition, many patients are at long-term risk of recurrence
.
Therefore, how to make patients obtain effective treatment, increase the CR rate, and reduce the long-term recurrence rate is still very difficult
.
Yimaitong: At this ASH annual meeting, your team’s article on the treatment of SAA in Chinese adults with Eltrobopar combined with IST was selected in the ASH poster.
Can you please discuss the treatment plan in SAA patients based on the results of the study? How effective is it in? Prof.
He Guangsheng Eltroipopag is an oral small-molecule thrombopoietin receptor agonist, which can promote the proliferation and differentiation of bone marrow hematopoietic stem cells and increase the level of three-line blood cells
.
At present, the data on the treatment of SAA patients with Eltrombopag combined with IST are mostly concentrated in European and American populations, and the results of real-world studies in China are still unclear
.
In addition, IST therapy at home and abroad differs greatly in the selection of anti-thymocyte/lymphocyte immunoglobulin (ATG/ALG) species and the dosage of Eltroipopag
.
It is urgent to explore the effect of the combined regimen on SAA patients in China .
After years of clinical accumulation and data analysis, our team found that at 1, 3, 6, and 12 months after IST treatment, the overall response rate (ORR) of the IST alone or IST combined with Eltroipopag treatment group was 12% vs 35%, respectively (P=0.
002), 44%vs64%(P=0.
028), 61%vs85%(P=0.
006) and 73%vs 91%(P=0.
031)
.
The CR rates at the 3rd, 6th, and 12th months after receiving IST or IST combined with Eltrombopag were 7% vs 17% (P=0.
069), 14% vs 27% (P=0.
11), and 33% vs 32% (P= 0.
92)
.
Analysis of factors related to the efficacy of patients treated for 6 months showed that ORR was significantly related to the use of Eltrodopag (P=0.
011, OR=3.
600, 95%CI 1.
345-9.
638), and the safety was good
.
Yimaitong: In this study, there was no significant difference in the CR rate of patients receiving IST and the combined IST regimen of Eltrodopax.
It is different from the result that the CR rate of the combined group of Eltrodopax in the EBMT RACE study was significantly increased.
What do you think is the reason? What? Prof.
He Guangsheng First of all, from a pathophysiological point of view, Eltrobomide is a blood-promoting drug.
Combining it with IST has the advantage of allowing patients to recover faster and get remission in the early stage
.
At the end of treatment, perhaps because IST has sufficient time and space to "play", the CR rate of patients with IST alone has gradually increased, resulting in no significant difference in the CR rate of patients with the two regimens in this study
.
In addition, the RACE study 3 of EBMT is different from the ATG species we selected, and considering the differences in metabolism between East Asian populations and European and American populations, the domestic dose of etrobopar is only 1/2 (75mg/d) of foreign countries
.
There are reports that different species of ATG may cause differences in the CR rate of patients
.
Therefore, the results of domestic and foreign research may be inconsistent
.
In the follow-up, we will further explore the effect of IST combined with different doses of Eltrodipam on SAA patients, in order to further improve the efficacy while maintaining patient tolerance; in addition, if conditions are mature, consider using porcine ALG-based IST combined Clinical research is carried out on Eltrombopag
.
It is hoped that workers in the field of aplastic anemia (AA) across the country will work together to accumulate clinical experience and data to jointly benefit patients
.
Yimaitong: Another study published by your team on the efficacy predictors of Eltrombopag combined with IST in the treatment of SAA patients in China was also included in this year’s ASH poster.
Could you please discuss the impact of Eltrobopar with the results of the research? Correlative predictors of the efficacy of Pa in the treatment of SAA patients? Professor He Guangsheng lacked research on predictors of the efficacy of this combination scheme in the past, so this study is of great significance
.
By comparing the clinical conditions of patients with effective and ineffective use of Eltrodopa, the ROC curve was used to predict the efficacy factors of Eltrodopa
.
The results showed that lymphocyte count (P=0.
042, AUC=0.
721, 95%CI 0.
537-0.
904), percentage of reticulocytes (P=0.
007, AUC=0.
793, 95%CI 0.
631-0.
955), red blood cell distribution width-coefficient of variation (RDW-CV) (P=0.
042, AUC=0.
721, 95%CI 0.
493-0.
948) was positively correlated with the efficacy of etrobopar
.
A higher lymphocyte count, percentage of reticulocytes, and RDW-CV may indicate better function of the remaining bone marrow
.
If the patient has better residual hematopoietic function, and at the same time, after IST “turns out the chaos” and adjusts the abnormal immunity, the hematopoietic drugs will be able to play a better role and advantage
.
Yimaitong: In recent years, with the advancement of medical technology and the continuous emergence of new drugs, the survival and prognosis of SAA patients have been significantly improved.
What are your prospects for the future treatment of SAA? Professor He Guangsheng firstly for allogeneic hematopoietic stem cell transplantation technology, through further optimization and combination of pretreatment plan and anti-rejection drug plan, expand the transplant applicable population, benefit patients
.
Taking the new pretreatment program as an example, it can further reduce the damage to the patient's reproductive function and reduce the risk of the second tumor in the later stage
.
In addition, the improvement of the combination of drugs such as Eltrombopag and IST is particularly worthy of attention
.
Taking Eltrobopar as an example, how to choose the dosage, time, nodes, structure, etc.
to further increase the patient's CR rate, reduce the recurrence rate, reduce the risk of transformation, and improve the prognosis and survival of patients still has a long way to go
.
Summary The above studies have not only verified and affirmed the effectiveness and safety of the combined IST regimen in Chinese adult SAA treatment, but also filled the gaps in the efficacy predictors of the combined regimen, which is of great significance
.
However, SAA treatment still has a long way to go.
How to further optimize the medication regimen and improve the prognosis and survival of patients requires the joint efforts of clinical and scientific researchers across the country
.
Prof.
He Guangsheng Deputy Director, Department of Hematology, First Affiliated Hospital of Nanjing Medical University, Jiangsu Provincial People's Hospital Chief Expert of Department of Hematology, Nanjing Hospital Affiliated to Nanjing University of Traditional Chinese Medicine Chief Expert of Department of Hematology, Second Affiliated Hospital of Xuzhou Medical University Chairman of Anemia Branch of Jiangsu Research Hospital Association Jiangsu Deputy Leader of the Red Cell Disease Group of the Hematology Branch of the Provincial Medical Association Member of the 7th and 8th Anemia Group of the Chinese Society of Hematology and Academic Secretary "Chinese Journal of Practical Internal Medicine" Standing Editor, National Health Commission "14th Five-Year Plan" textbook editor The editor in charge of Hematology of the Journal of the Journal of Transitional International Medicine of the Committee won the second prize of the Chinese Medical Award and the "Chen Jinrong Life Science Excellence Award" of Soochow University.
Baidu Academic (until 2017) shows: H index 26, G index 55 References: 1.
Ruixin Li, et al.
ASH 2021 abstract 2190.
2.
Ruixin Li, et al.
ASH 2021 abstract 1115.
3.
De Latour RP, et al.
EBMT 2020 abstract 0018.
Stamp "read the original text" and we will make progress together
