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Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem cell gene mutation caused by erythrocyte membrane defects.
The 10-year survival rate of domestic patients is about 70%.
The clinical manifestations are mainly intravascular hemolytic anemia, which may be accompanied by symptoms such as thrombosis, renal insufficiency, and pulmonary hypertension.
Typical patients have characteristic intermittent nocturnal hemoglobinuria.
There are more male patients than females in China, and the peak age of onset is 20-40 years old.
The patient suffers from pain and suffering for a long time, and the quality of life is low.
Many patients are in the golden age of their lives.
As the backbone of their families, they have lost their labor force due to illness, causing a great social burden.
However, the diagnosis, treatment and follow-up of PNH need to be further improved.
In this regard, Yimaitong specially planned the "PNH Famous Doctors Lecture" in 2021, aiming to improve the level of PNH diagnosis and treatment of Chinese hematologists.
This issue of "PNH Famous Doctors Lecture" Yimaitong invited Professor Fu Rong from the General Hospital of Tianjin Medical University to share on the pathogenesis and treatment mechanism of PNH! Professor Fu Rong, Doctor of Medicine, Chief Physician, Second-Class Professor, Doctoral Supervisor, Associate Dean of Tianjin Medical University General Hospital, Tianjin "Tianmen Medical Talents" First "Tianjin Famous Doctor" Standing Committee of Chinese Medical Association Hematology Branch Chinese Medical Association Hematology Branch Red Cell Diseases The deputy leader of the group, the member of the Chinese Medical Doctor Association Hematology Physician Branch, the chairman of the Red Blood Cell Disease Professional Committee of the Beijing Cancer Prevention and Treatment Society, the chairman of the Tianjin Medical Association Hematology Branch, wrote the "Aplastic Anemia Expert Consensus", "PNH Expert Consensus", "Pure Red Blood Cells" Aplastic Experts Consensus" The pathogenesis of PNH, the incidence of PNH in China, and the survival status of patients.
The pathogenesis of PNH is the mutation of PIG-A gene on hematopoietic stem cells, which leads to non-malignant cloning of hematopoietic stem cells, which is an acquired disease.PNH is a rare disease.
At present, there is no regional incidence statistics in China.
Studies have shown that the incidence in some parts of Northeast China is nearly three millionths.
The age of onset is between 20 and 40, with a median age of 30.
.
PNH is a stubborn disease, and so far, it is still an incurable disease.
The 10-year survival rate of PNH patients is about 70%, and the survival rate statistics of more than 200 patients in Tianjin General Hospital show that the 20-year survival rate of PNH is about 50%.
The connection and difference between PNH and AA.
Because PNH and aplastic anemia (AA) both have anemia, even many PNH patients will have pancytopenia like AA patients, and they can be mutually transformed or coexist.
According to statistics, about 40%~50% of PNH patients have a history of AA, 30%~40% of AA patients will be accompanied by PNH clones, and 10%~20% of patients with simple AA will be treated with immunosuppressive agents.
A clone of PNH appears.
Therefore, PNH and AA are often mentioned together.
But PNH and AA are two different diseases.
PNH is caused by genetic mutations.
PNH anemia is mainly hemolytic anemia.
AA is a bone marrow failure disease caused by autoimmune damage to hematopoietic cells.
AA anemia is anemia caused by decreased hematopoietic function in the bone marrow.
Moreover, other clinical manifestations of PNH and AA are also different, such as bleeding and severe infection in AA.
If PNH patients do not have a reduction in white blood cells and platelets, the probability of bleeding or serious infection will be relatively low.
PNH has its own unique manifestations, such as chronic persistent hemolytic anemia, renal failure, dysphagia, dyspnea, and thrombosis.
Men will have some erectile dysfunction, etc.
These manifestations do not usually occur in AA patients.
The mechanism of action of C5 complement inhibitors in the treatment of PNH PNH is caused by mutations in the PIG-A gene on hematopoietic stem cells.
The function of the PIG-A gene is to synthesize ankyrin.
Once the PIG-A gene is mutated, ankyrin cannot be synthesized, which will cause the ankyrin connected by ankyrin to become dysfunctional and unable to connect to red blood cells.
Once the anchoring protein on the red blood cells is lacking, the complement will attack the red blood cells, resulting in the destruction of red blood cells, intravascular hemolysis, and a series of clinical manifestations of PNH.
C5 is the last link in the complement cascade.
C5 does not resist red blood cells under normal conditions, nor does it exhibit abnormal activation.
Once the complement is abnormally activated, C5 is cleaved into C5b and C5a, and C5b eventually forms the C5b-9 membrane attack complex, which destroys red blood cells.
The mechanism of C5 complement inhibitors to treat PNH is to inhibit the formation of membrane attack complexes and prevent the destruction of red blood cells.
At present, all complement inhibitors that have been marketed are C5 complement inhibitors.
The first generation of C5 complement inhibitors has been on the market abroad for more than ten years.
Many foreign patients have also benefited from the treatment of C5 complement inhibitors.
After using the drug, the patient’s hemolysis is reduced.
At the same time, PNH patients are very serious and lethal.
The complications of PNH have also been greatly reduced, and the quality of life and survival rate of PNH patients have also been improved.
Therefore, this drug is a very valuable drug for PNH patients.
At present, the second-generation C5 complement inhibitor Crovalimab has carried out clinical trials in China.
It is hoped that the clinical trials will be approved as soon as possible to be approved for marketing and better improve the quality of life of PNH patients. References: 1.
Hill A et al.
Nat Rev Dis Primers.
2017; 3:17028.
2.
Journal of Mudanjiang Medical College, 1997, Volume 18, Issue 3 P-7.
3.
Report on the Availability of Drugs for Rare Diseases in China (2019).
4 .
Fu, et al.
J ClinLab Anal.
2020; 34:e23008.
5.
Tu J.
et al.
Acta Haematologica 2021 144:1 (34-43).
6.
Schrezenmeier, H.
et al.
Haematologica, 2014, 99 , 922–929.
7.
T.
Yoshizato, et al.
Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia.
N Engl J Med 2015;373:35-47.
8.
Röth A et al.
ASH 2018 [oral presentation].
9.
Kelly et al .
Blood; 2011，117(25).
10.
Jong Wook Lee, et al.
Blood.
2019;133(6):530-539.
11.
Kulasekararaj et al.
2020 EHA.
Abstract.
EP855.
12.
Risitano et al.
EBMT 2020 abstract 0019.
Stamp "read the original text", we make progress together
The 10-year survival rate of domestic patients is about 70%.
The clinical manifestations are mainly intravascular hemolytic anemia, which may be accompanied by symptoms such as thrombosis, renal insufficiency, and pulmonary hypertension.
Typical patients have characteristic intermittent nocturnal hemoglobinuria.
There are more male patients than females in China, and the peak age of onset is 20-40 years old.
The patient suffers from pain and suffering for a long time, and the quality of life is low.
Many patients are in the golden age of their lives.
As the backbone of their families, they have lost their labor force due to illness, causing a great social burden.
However, the diagnosis, treatment and follow-up of PNH need to be further improved.
In this regard, Yimaitong specially planned the "PNH Famous Doctors Lecture" in 2021, aiming to improve the level of PNH diagnosis and treatment of Chinese hematologists.
This issue of "PNH Famous Doctors Lecture" Yimaitong invited Professor Fu Rong from the General Hospital of Tianjin Medical University to share on the pathogenesis and treatment mechanism of PNH! Professor Fu Rong, Doctor of Medicine, Chief Physician, Second-Class Professor, Doctoral Supervisor, Associate Dean of Tianjin Medical University General Hospital, Tianjin "Tianmen Medical Talents" First "Tianjin Famous Doctor" Standing Committee of Chinese Medical Association Hematology Branch Chinese Medical Association Hematology Branch Red Cell Diseases The deputy leader of the group, the member of the Chinese Medical Doctor Association Hematology Physician Branch, the chairman of the Red Blood Cell Disease Professional Committee of the Beijing Cancer Prevention and Treatment Society, the chairman of the Tianjin Medical Association Hematology Branch, wrote the "Aplastic Anemia Expert Consensus", "PNH Expert Consensus", "Pure Red Blood Cells" Aplastic Experts Consensus" The pathogenesis of PNH, the incidence of PNH in China, and the survival status of patients.
The pathogenesis of PNH is the mutation of PIG-A gene on hematopoietic stem cells, which leads to non-malignant cloning of hematopoietic stem cells, which is an acquired disease.PNH is a rare disease.
At present, there is no regional incidence statistics in China.
Studies have shown that the incidence in some parts of Northeast China is nearly three millionths.
The age of onset is between 20 and 40, with a median age of 30.
.
PNH is a stubborn disease, and so far, it is still an incurable disease.
The 10-year survival rate of PNH patients is about 70%, and the survival rate statistics of more than 200 patients in Tianjin General Hospital show that the 20-year survival rate of PNH is about 50%.
The connection and difference between PNH and AA.
Because PNH and aplastic anemia (AA) both have anemia, even many PNH patients will have pancytopenia like AA patients, and they can be mutually transformed or coexist.
According to statistics, about 40%~50% of PNH patients have a history of AA, 30%~40% of AA patients will be accompanied by PNH clones, and 10%~20% of patients with simple AA will be treated with immunosuppressive agents.
A clone of PNH appears.
Therefore, PNH and AA are often mentioned together.
But PNH and AA are two different diseases.
PNH is caused by genetic mutations.
PNH anemia is mainly hemolytic anemia.
AA is a bone marrow failure disease caused by autoimmune damage to hematopoietic cells.
AA anemia is anemia caused by decreased hematopoietic function in the bone marrow.
Moreover, other clinical manifestations of PNH and AA are also different, such as bleeding and severe infection in AA.
If PNH patients do not have a reduction in white blood cells and platelets, the probability of bleeding or serious infection will be relatively low.
PNH has its own unique manifestations, such as chronic persistent hemolytic anemia, renal failure, dysphagia, dyspnea, and thrombosis.
Men will have some erectile dysfunction, etc.
These manifestations do not usually occur in AA patients.
The mechanism of action of C5 complement inhibitors in the treatment of PNH PNH is caused by mutations in the PIG-A gene on hematopoietic stem cells.
The function of the PIG-A gene is to synthesize ankyrin.
Once the PIG-A gene is mutated, ankyrin cannot be synthesized, which will cause the ankyrin connected by ankyrin to become dysfunctional and unable to connect to red blood cells.
Once the anchoring protein on the red blood cells is lacking, the complement will attack the red blood cells, resulting in the destruction of red blood cells, intravascular hemolysis, and a series of clinical manifestations of PNH.
C5 is the last link in the complement cascade.
C5 does not resist red blood cells under normal conditions, nor does it exhibit abnormal activation.
Once the complement is abnormally activated, C5 is cleaved into C5b and C5a, and C5b eventually forms the C5b-9 membrane attack complex, which destroys red blood cells.
The mechanism of C5 complement inhibitors to treat PNH is to inhibit the formation of membrane attack complexes and prevent the destruction of red blood cells.
At present, all complement inhibitors that have been marketed are C5 complement inhibitors.
The first generation of C5 complement inhibitors has been on the market abroad for more than ten years.
Many foreign patients have also benefited from the treatment of C5 complement inhibitors.
After using the drug, the patient’s hemolysis is reduced.
At the same time, PNH patients are very serious and lethal.
The complications of PNH have also been greatly reduced, and the quality of life and survival rate of PNH patients have also been improved.
Therefore, this drug is a very valuable drug for PNH patients.
At present, the second-generation C5 complement inhibitor Crovalimab has carried out clinical trials in China.
It is hoped that the clinical trials will be approved as soon as possible to be approved for marketing and better improve the quality of life of PNH patients. References: 1.
Hill A et al.
Nat Rev Dis Primers.
2017; 3:17028.
2.
Journal of Mudanjiang Medical College, 1997, Volume 18, Issue 3 P-7.
3.
Report on the Availability of Drugs for Rare Diseases in China (2019).
4 .
Fu, et al.
J ClinLab Anal.
2020; 34:e23008.
5.
Tu J.
et al.
Acta Haematologica 2021 144:1 (34-43).
6.
Schrezenmeier, H.
et al.
Haematologica, 2014, 99 , 922–929.
7.
T.
Yoshizato, et al.
Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia.
N Engl J Med 2015;373:35-47.
8.
Röth A et al.
ASH 2018 [oral presentation].
9.
Kelly et al .
Blood; 2011，117(25).
10.
Jong Wook Lee, et al.
Blood.
2019;133(6):530-539.
11.
Kulasekararaj et al.
2020 EHA.
Abstract.
EP855.
12.
Risitano et al.
EBMT 2020 abstract 0019.
Stamp "read the original text", we make progress together
