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Aplastic anemia (referred to as aplastic anemia) is a bone marrow failure disease.
Severe aplastic anemia has life-threatening characteristics such as a significant decline in three lines, blood transfusion dependence, and susceptibility to infection
.
All-sib hematopoietic stem cell transplantation is the first-line treatment option for the treatment of severe aplastic anemia; for patients lacking all-sibs, haploidentical transplantation was once a restricted area due to low engraftment rates and high graft-versus-host disease
.
In recent years, haploidentical hematopoietic stem cell transplantation has made great progress in the treatment of severe aplastic anemia.
The engraftment rate is as high as 99%, and the short-term survival rate and blood picture recovery rate are consistent with that of sibling allograft transplantation
.
However, there is a lack of long-term follow-up data: whether the long-term survival patients achieve a good quality of life, and whether they return to society normally has not been reported
.
Recently, the team of Professor Xiaojun Huang from the Institute of Hematology of Peking University published a research paper entitled "Long-term follow-up of haploidentical transplantation in relapsed/refractory severe aplastic anemia: a multicenter prospective study" in Science Bulletin.
The long-term follow-up of the center's clinical research focuses on the long-term efficacy and quality of life of haploidentical hematopoietic stem cell transplantation as a second-line regimen in the treatment of severe aplastic anemia
.
This article longitudinally and prospectively assessed quality of life (SF-36 for adults and PedsQL4.
0 for children) before, 3 years, and 5 years after transplantation
.
A total of 287 patients were included in the study, and the median follow-up time for surviving patients was 4.
56 (3.
01–9.
05) years
.
During long-term follow-up, 97.
5% of patients achieved stable complete donor chimerism, and 93.
4% of patients achieved complete hematopoietic reconstruction
.
The estimated 9-year overall and failure-free survival rates were 85.
4% ± 2.
1% and 84.
0% ± 2.
2%
.
Age at transplant (≥18 years) and performance status assessment (ECOG ≥2 points) were prognostic factors affecting survival
.
The quality of life of patients with aplastic anemia was significantly improved at 3 years after haplotype hematopoietic stem cell transplantation compared with that before transplantation, and further improved at 5 years after transplantation
.
Moderate to severe chronic graft-versus-host disease is an adverse factor affecting quality of life in both children and adults
.
At the last follow-up, 74.
0% of children and 72.
9% of adults had resumed normal study or work
.
Long-term survival outcomes in children and adults This study suggests that haplotype donor hematopoietic stem cell transplantation can be routinely recommended as a second-line treatment for patients with severe aplastic anemia in the absence of a fully syngeneic sibling donor
.
Professor Xu Lanping and Dr.
Xu Zhengli of Peking University People's Hospital, Professor Wang Shunqing of Guangzhou First People's Hospital are the co-first authors of the article, and Professor Huang Xiaojun is the corresponding author
.
Professor Wu Depei of the First Affiliated Hospital of Soochow University, Professor Gao Sujun of the First Affiliated Hospital of Jilin University, Professor Yang Jianmin of the Changhai Hospital of the Second Military Medical University, Professor Xia Linghui of the Union Hospital Affiliated to Huazhong University of Science and Technology, Professor Jiang Ming of the First Affiliated Hospital of Xinjiang Medical University, General Manager of Lanzhou Military Region Professor Bai Hai of the Hospital, Professor Zhang Xi of Xinqiao Hospital and Professor Wang Xin of Shandong Provincial Hospital are the co-authors of the paper
.
The research was funded by the National Natural Science Foundation of China Innovation Group and Key Projects, National Key R&D Program Projects and other projects
.
【Related Reading】Wang Y, Liu QF, Lin R, Yang T, Xu YJ, Mo XD, Huang XJ.
Optimizing antithymocyte globulin dosing in haploidentical hematopoietic cell transplantation: long-term follow-up of a multicenter, randomized controlled trial.
Science Bulletin 2021; 66: 2498-2505.
doi:10.
1016/j.
scib.
2021.
06.
002.
Gorin NC.
How antithymocyte globulin, a polyclonal soup of the past century, when carefully dosed, has become crucial for hematopoietic stem cell transplantation with haplo -identical donors in the 21st century.
Science Bulletin 2021; 66: 2445-2447.
doi: 10.
1016/j.
scib.
2021.
08.
002.
For details of the research, please read the original text▼[Click the link below or read the original text] https://www.
sciencedirect.
com/science/article/abs/pii/S2095927322000391
Severe aplastic anemia has life-threatening characteristics such as a significant decline in three lines, blood transfusion dependence, and susceptibility to infection
.
All-sib hematopoietic stem cell transplantation is the first-line treatment option for the treatment of severe aplastic anemia; for patients lacking all-sibs, haploidentical transplantation was once a restricted area due to low engraftment rates and high graft-versus-host disease
.
In recent years, haploidentical hematopoietic stem cell transplantation has made great progress in the treatment of severe aplastic anemia.
The engraftment rate is as high as 99%, and the short-term survival rate and blood picture recovery rate are consistent with that of sibling allograft transplantation
.
However, there is a lack of long-term follow-up data: whether the long-term survival patients achieve a good quality of life, and whether they return to society normally has not been reported
.
Recently, the team of Professor Xiaojun Huang from the Institute of Hematology of Peking University published a research paper entitled "Long-term follow-up of haploidentical transplantation in relapsed/refractory severe aplastic anemia: a multicenter prospective study" in Science Bulletin.
The long-term follow-up of the center's clinical research focuses on the long-term efficacy and quality of life of haploidentical hematopoietic stem cell transplantation as a second-line regimen in the treatment of severe aplastic anemia
.
This article longitudinally and prospectively assessed quality of life (SF-36 for adults and PedsQL4.
0 for children) before, 3 years, and 5 years after transplantation
.
A total of 287 patients were included in the study, and the median follow-up time for surviving patients was 4.
56 (3.
01–9.
05) years
.
During long-term follow-up, 97.
5% of patients achieved stable complete donor chimerism, and 93.
4% of patients achieved complete hematopoietic reconstruction
.
The estimated 9-year overall and failure-free survival rates were 85.
4% ± 2.
1% and 84.
0% ± 2.
2%
.
Age at transplant (≥18 years) and performance status assessment (ECOG ≥2 points) were prognostic factors affecting survival
.
The quality of life of patients with aplastic anemia was significantly improved at 3 years after haplotype hematopoietic stem cell transplantation compared with that before transplantation, and further improved at 5 years after transplantation
.
Moderate to severe chronic graft-versus-host disease is an adverse factor affecting quality of life in both children and adults
.
At the last follow-up, 74.
0% of children and 72.
9% of adults had resumed normal study or work
.
Long-term survival outcomes in children and adults This study suggests that haplotype donor hematopoietic stem cell transplantation can be routinely recommended as a second-line treatment for patients with severe aplastic anemia in the absence of a fully syngeneic sibling donor
.
Professor Xu Lanping and Dr.
Xu Zhengli of Peking University People's Hospital, Professor Wang Shunqing of Guangzhou First People's Hospital are the co-first authors of the article, and Professor Huang Xiaojun is the corresponding author
.
Professor Wu Depei of the First Affiliated Hospital of Soochow University, Professor Gao Sujun of the First Affiliated Hospital of Jilin University, Professor Yang Jianmin of the Changhai Hospital of the Second Military Medical University, Professor Xia Linghui of the Union Hospital Affiliated to Huazhong University of Science and Technology, Professor Jiang Ming of the First Affiliated Hospital of Xinjiang Medical University, General Manager of Lanzhou Military Region Professor Bai Hai of the Hospital, Professor Zhang Xi of Xinqiao Hospital and Professor Wang Xin of Shandong Provincial Hospital are the co-authors of the paper
.
The research was funded by the National Natural Science Foundation of China Innovation Group and Key Projects, National Key R&D Program Projects and other projects
.
【Related Reading】Wang Y, Liu QF, Lin R, Yang T, Xu YJ, Mo XD, Huang XJ.
Optimizing antithymocyte globulin dosing in haploidentical hematopoietic cell transplantation: long-term follow-up of a multicenter, randomized controlled trial.
Science Bulletin 2021; 66: 2498-2505.
doi:10.
1016/j.
scib.
2021.
06.
002.
Gorin NC.
How antithymocyte globulin, a polyclonal soup of the past century, when carefully dosed, has become crucial for hematopoietic stem cell transplantation with haplo -identical donors in the 21st century.
Science Bulletin 2021; 66: 2445-2447.
doi: 10.
1016/j.
scib.
2021.
08.
002.
For details of the research, please read the original text▼[Click the link below or read the original text] https://www.
sciencedirect.
com/science/article/abs/pii/S2095927322000391
