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Editor’s note iNature is China’s largest academic official account.
It is jointly created by the doctoral team of Tsinghua University, Harvard University, Chinese Academy of Sciences and other units.
The iNature Talent Official Account is now launched, focusing on talent recruitment, academic progress, scientific research information, interested parties can Long press or scan the QR code below to follow us
.
iNatureβ-thalassemia (BTM) is one of the most common single-gene genetic diseases in the world.
It is caused by mutations in the gene encoding the β-chain of hemoglobin (Hb).
It is characterized by reduced or missing β-globin synthesis, ineffective erythropoiesis, and Hemolysis of mature red blood cells (RBC) caused by excess alpha chains
.
Thalidomide can induce the expression of γ-globin in erythrocyte progenitor cells, but its efficacy in patients with transfusion-dependent β-thalassemia (TDT) remains unclear
.
On November 18, 2021, Zhou Guangbiao of Chinese Academy of Medical Sciences/Peking Union Medical College, Chen Saijuan of Shanghai Jiaotong University and Chen Jiangming of Wuzhou Workers Hospital jointly published an online report entitled "Safety and efficacy of thalidomide in Signal Transduction and Targeted Therapy (IF=18.
19)" Patients with transfusion-dependent β-thalassemia: a randomized clinical trial" research paper, in this multi-center, randomized, double-blind phase 2 clinical trial, the study aims to determine the safety of thalidomide in TDT patients Sex and effectiveness
.
One hundred patients 14 years of age or older were randomly assigned to receive placebo or thalidomide for 12 weeks, followed by an extended period of at least 36 weeks
.
The primary endpoint is the change in the patient's hemoglobin (Hb) level
.
Secondary endpoints include red blood cell (RBC) units infused and adverse reactions
.
During the placebo-controlled period, the median Hb concentration of patients receiving thalidomide increased by 14.
0 (range, 2.
5 to 37.
5) g/L, while the Hb concentration of patients receiving placebo did not change significantly
.
In 12 weeks, the average RBC blood transfusion of patients treated with thalidomide and placebo was 5.
4 ± 5.
0 U and 10.
3 ± 6.
4 U, respectively (P <0.
001)
.
Compared with placebo, adverse events such as drowsiness, dizziness, fatigue, fever, sore throat and rash were more common in thalidomide
.
During the expansion phase, thalidomide treatment for 24 weeks led to a continuous increase in Hb concentration, reaching 104.
9 ± 19.
0 g/L, without blood transfusion
.
The significant increase in Hb concentration and the decrease in RBC transfusion are related to non-β0/β0 and HBS1L-MYB (rs9399137 C/T, C/C; rs4895441 A/G, G/G) genotypes
.
These results indicate that thalidomide is effective for TDT patients
.
Beta-thalassemia (BTM) is one of the most common single-gene genetic diseases in the world.
It is caused by mutations in the gene encoding the β-chain of hemoglobin (Hb).
It is characterized by reduced or missing β-globin synthesis, ineffective erythropoiesis, and Hemolysis of mature red blood cells (RBC) caused by excess alpha chains
.
BTM is divided into major subtypes, medium subtypes and minor subtypes.
It is also divided into transfusion-dependent thalassemia (TDT) and transfusion-independent thalassemia (NTDT)
.
Every year, more than 40,000 BTM babies are born worldwide, including 26,000 TDTs, most of which are born in countries with limited resources
.
Currently, approximately 100,000 patients worldwide receive regular blood transfusions
.
Other treatment options for BTM include iron chelation, hematopoietic stem cell transplantation (HSCT), emerging gene therapy and red blood cell maturation agents
.
It is estimated that only 12% of TDT children receive adequate blood transfusions, and <40% of transfusion recipients receive adequate iron chelation therapy
.
Although HSCT can cure transplant patients under 14 years of age, it is suitable for less than 30% of patients, and the recipient's treatment-related mortality rate is 5-10%
.
Gene therapy is a promising treatment method, but its long-term efficacy remains to be determined
.
Therefore, there is an urgent need for effective and affordable remedies to save TDT patients, especially those 14 years and older
.
Induction of compensatory fetal Hb (Hb F) is an emerging treatment option for BTM, and its efficacy is similar to the single nucleotide polymorphism (SNP) in the Gγ globin gene HBG27 (also known as Xmn I polymorphism, At position -158 of the gene), BCL11A8 and HBS1L-MYB are related to the intergenic region
.
Thalidomide has been proven to be a Hb F inducer, and in some case reports, it can significantly increase the Hb (mainly Hb F) concentration in patients with TDT
.
In 9 patients treated with thalidomide, the Hb concentration and Hb F ratio increased from 51.
3 ± 21.
5 g/L and 35.
7 ± 26.
8% before treatment to 103.
8 ± 7.
5 g/L and 75.
7 ± 14.
6% after treatment
.
In these patients, an increase in Hb is usually observed within 1 month after thalidomide treatment
.
However, these clinical data are case reports or small-scale single-center studies, and other Hb F inducers (including hydroxyurea and butyrate) are not satisfactory
.
In this multicenter, randomized, double-blind phase 2 clinical trial, the study aims to determine the safety and effectiveness of thalidomide in patients with TDT
.
One hundred patients 14 years of age or older were randomly assigned to receive placebo or thalidomide for 12 weeks, followed by an extended period of at least 36 weeks
.
The primary endpoint is the change in the patient's hemoglobin (Hb) level
.
Secondary endpoints include red blood cell (RBC) units infused and adverse reactions
.
During the placebo-controlled period, the median Hb concentration of patients receiving thalidomide increased by 14.
0 (range, 2.
5 to 37.
5) g/L, while the Hb concentration of patients receiving placebo did not change significantly
.
In 12 weeks, the average RBC blood transfusion volume of patients treated with thalidomide and placebo was 5.
4 ± 5.
0 U and 10.
3 ± 6.
4 U, respectively (P <0.
001)
.
Compared with placebo, adverse events such as drowsiness, dizziness, fatigue, fever, sore throat and rash were more common in thalidomide
.
During the expansion phase, thalidomide treatment for 24 weeks resulted in a continuous increase in Hb concentration, reaching 104.
9 ± 19.
0 g/L, without the need for blood transfusion
.
The significant increase in Hb concentration and the decrease in RBC transfusion are related to non-β0/β0 and HBS1L-MYB (rs9399137 C/T, C/C; rs4895441 A/G, G/G) genotypes
.
These results indicate that thalidomide is effective for TDT patients
.
Reference message: https://
It is jointly created by the doctoral team of Tsinghua University, Harvard University, Chinese Academy of Sciences and other units.
The iNature Talent Official Account is now launched, focusing on talent recruitment, academic progress, scientific research information, interested parties can Long press or scan the QR code below to follow us
.
iNatureβ-thalassemia (BTM) is one of the most common single-gene genetic diseases in the world.
It is caused by mutations in the gene encoding the β-chain of hemoglobin (Hb).
It is characterized by reduced or missing β-globin synthesis, ineffective erythropoiesis, and Hemolysis of mature red blood cells (RBC) caused by excess alpha chains
.
Thalidomide can induce the expression of γ-globin in erythrocyte progenitor cells, but its efficacy in patients with transfusion-dependent β-thalassemia (TDT) remains unclear
.
On November 18, 2021, Zhou Guangbiao of Chinese Academy of Medical Sciences/Peking Union Medical College, Chen Saijuan of Shanghai Jiaotong University and Chen Jiangming of Wuzhou Workers Hospital jointly published an online report entitled "Safety and efficacy of thalidomide in Signal Transduction and Targeted Therapy (IF=18.
19)" Patients with transfusion-dependent β-thalassemia: a randomized clinical trial" research paper, in this multi-center, randomized, double-blind phase 2 clinical trial, the study aims to determine the safety of thalidomide in TDT patients Sex and effectiveness
.
One hundred patients 14 years of age or older were randomly assigned to receive placebo or thalidomide for 12 weeks, followed by an extended period of at least 36 weeks
.
The primary endpoint is the change in the patient's hemoglobin (Hb) level
.
Secondary endpoints include red blood cell (RBC) units infused and adverse reactions
.
During the placebo-controlled period, the median Hb concentration of patients receiving thalidomide increased by 14.
0 (range, 2.
5 to 37.
5) g/L, while the Hb concentration of patients receiving placebo did not change significantly
.
In 12 weeks, the average RBC blood transfusion of patients treated with thalidomide and placebo was 5.
4 ± 5.
0 U and 10.
3 ± 6.
4 U, respectively (P <0.
001)
.
Compared with placebo, adverse events such as drowsiness, dizziness, fatigue, fever, sore throat and rash were more common in thalidomide
.
During the expansion phase, thalidomide treatment for 24 weeks led to a continuous increase in Hb concentration, reaching 104.
9 ± 19.
0 g/L, without blood transfusion
.
The significant increase in Hb concentration and the decrease in RBC transfusion are related to non-β0/β0 and HBS1L-MYB (rs9399137 C/T, C/C; rs4895441 A/G, G/G) genotypes
.
These results indicate that thalidomide is effective for TDT patients
.
Beta-thalassemia (BTM) is one of the most common single-gene genetic diseases in the world.
It is caused by mutations in the gene encoding the β-chain of hemoglobin (Hb).
It is characterized by reduced or missing β-globin synthesis, ineffective erythropoiesis, and Hemolysis of mature red blood cells (RBC) caused by excess alpha chains
.
BTM is divided into major subtypes, medium subtypes and minor subtypes.
It is also divided into transfusion-dependent thalassemia (TDT) and transfusion-independent thalassemia (NTDT)
.
Every year, more than 40,000 BTM babies are born worldwide, including 26,000 TDTs, most of which are born in countries with limited resources
.
Currently, approximately 100,000 patients worldwide receive regular blood transfusions
.
Other treatment options for BTM include iron chelation, hematopoietic stem cell transplantation (HSCT), emerging gene therapy and red blood cell maturation agents
.
It is estimated that only 12% of TDT children receive adequate blood transfusions, and <40% of transfusion recipients receive adequate iron chelation therapy
.
Although HSCT can cure transplant patients under 14 years of age, it is suitable for less than 30% of patients, and the recipient's treatment-related mortality rate is 5-10%
.
Gene therapy is a promising treatment method, but its long-term efficacy remains to be determined
.
Therefore, there is an urgent need for effective and affordable remedies to save TDT patients, especially those 14 years and older
.
Induction of compensatory fetal Hb (Hb F) is an emerging treatment option for BTM, and its efficacy is similar to the single nucleotide polymorphism (SNP) in the Gγ globin gene HBG27 (also known as Xmn I polymorphism, At position -158 of the gene), BCL11A8 and HBS1L-MYB are related to the intergenic region
.
Thalidomide has been proven to be a Hb F inducer, and in some case reports, it can significantly increase the Hb (mainly Hb F) concentration in patients with TDT
.
In 9 patients treated with thalidomide, the Hb concentration and Hb F ratio increased from 51.
3 ± 21.
5 g/L and 35.
7 ± 26.
8% before treatment to 103.
8 ± 7.
5 g/L and 75.
7 ± 14.
6% after treatment
.
In these patients, an increase in Hb is usually observed within 1 month after thalidomide treatment
.
However, these clinical data are case reports or small-scale single-center studies, and other Hb F inducers (including hydroxyurea and butyrate) are not satisfactory
.
In this multicenter, randomized, double-blind phase 2 clinical trial, the study aims to determine the safety and effectiveness of thalidomide in patients with TDT
.
One hundred patients 14 years of age or older were randomly assigned to receive placebo or thalidomide for 12 weeks, followed by an extended period of at least 36 weeks
.
The primary endpoint is the change in the patient's hemoglobin (Hb) level
.
Secondary endpoints include red blood cell (RBC) units infused and adverse reactions
.
During the placebo-controlled period, the median Hb concentration of patients receiving thalidomide increased by 14.
0 (range, 2.
5 to 37.
5) g/L, while the Hb concentration of patients receiving placebo did not change significantly
.
In 12 weeks, the average RBC blood transfusion volume of patients treated with thalidomide and placebo was 5.
4 ± 5.
0 U and 10.
3 ± 6.
4 U, respectively (P <0.
001)
.
Compared with placebo, adverse events such as drowsiness, dizziness, fatigue, fever, sore throat and rash were more common in thalidomide
.
During the expansion phase, thalidomide treatment for 24 weeks resulted in a continuous increase in Hb concentration, reaching 104.
9 ± 19.
0 g/L, without the need for blood transfusion
.
The significant increase in Hb concentration and the decrease in RBC transfusion are related to non-β0/β0 and HBS1L-MYB (rs9399137 C/T, C/C; rs4895441 A/G, G/G) genotypes
.
These results indicate that thalidomide is effective for TDT patients
.
Reference message: https://
