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*The professional part involved in this article is only for medical professionals to read for reference.
Focus on rare diseases in children According to World Health Organization statistics, in addition to accidental injuries, malignant tumors have become the number one cause of death among children in China
.
Among them, neuroblastoma (NB) is called "the king of childhood tumors" and is the most common extracranial solid tumor in infants and young children
.
According to the statistics of childhood cancer in the United States in 2014, new cases of neuroblastoma accounted for 8% to 10% of all malignant tumors in children
.
The incidence rate in China is about 10.
1/100 million (children between the ages of 0 and 14).
The number of new cases of children is about 3,000 each year.
About 50% of neuroblastomas occur in infants and young children under 2 years of age
.
It is reported that neuroblastoma has the characteristics of high degree of malignancy, rapid disease progression, and difficult treatment.
In particular, children with high risk and refractory relapse often face severe survival challenges
.
With the advancement of diagnosis and treatment, under timely and standardized active treatment, children with neuroblastoma have also obtained more and more opportunities for cure
.
On September 12, focusing on the opportunities and progress of neuroblastoma and precision treatment, at the inaugural meeting of the neuroblastoma community co-sponsored by the God Mother Sunshine Home and the Pain Challenge Foundation, Professor Wang Huanmin, Director of Oncology Surgery, Beijing Children’s Hospital, Professor Yuan Xiaojun, director of the Pediatric Hematology Oncology Department of Xinhua Hospital, Shanghai Jiaotong University School of Medicine, and other pediatric oncologists discussed
.
Signs and symptoms of neuroblastoma Neuroblastoma is derived from undifferentiated sympathetic ganglion cells.
Any part with embryonic sympathetic ganglion cells has the potential for malignancy.
The adrenal gland is the most common source of neuroblastoma.
Hair site
.
Yuan Xiaojun, director of the Department of Pediatric Hematology and Oncology, Xinhua Hospital, Shanghai Jiaotong University School of Medicine, introduced that the clinical manifestations of neuroblastoma are quite heterogeneous, and the clinical manifestations are different according to the location and scope of the primary tumor and metastatic tumor
.
Professor Yuan Xiaojun, director of the Department of Pediatric Hematology and Oncology, Xinhua Hospital, Shanghai Jiaotong University School of Medicine, "Because cases of abdominal tumors are the most common, the clinical manifestations of neuroblastoma are mostly abdominal masses
.
" It is reported that tumors in the abdomen or pelvis may cause abdominal distension.
, But usually no tenderness.
The child may complain of abdominal pain, do not want to eat, and have symptoms such as weight loss, constipation or diarrhea, and changes in bowel habits; in a few cases, high blood pressure may even occur
.
Tumors on the chest and neck may appear as hard lumps on the body surface and are usually non-tender.
If the tumor compresses the large veins, it will cause swelling of the face, neck, arms, and upper chest, and blue or red ecchymosis may appear on the skin
.
Professor Yuan Xiaojun pointed out that in clinical practice, neuroblastoma is often difficult to detect, especially some primary doctors who lack experience in diagnosing neuroblastoma.
When the tumor invades other parts, patients will also have other symptoms and signs
.
If the tumor compresses the sympathetic nerves of the neck, the child may develop Horner syndrome, which is manifested by ptosis of the eyelids and unilateral miosis
.
When the tumor squeezes into the throat or trachea, it may cause coughing, breathing, and swallowing difficulties; head tumors may compress nerves and blood vessels in the brain, causing headache, dizziness or changes in consciousness
.
In addition, tumors near the spine may compress nerves, affecting sensation and mobility, and children may be affected when standing, walking or crawling; tumors around the orbit may cause bruises (commonly known as "panda eyes"), swelling, and protruding eyeballs.
; Bone marrow metastasis can cause anemia, manifested as a pale complexion, and may also lead to thrombocytopenia, which is not easy to stop bleeding; bone metastasis can cause bone pain
.
If the tumor infiltrates the liver, the liver will swell and may cause respiratory distress, coagulation disorders, gastrointestinal and kidney function damage.
These conditions are more common in young infants; when the patient has skin or subcutaneous metastases, it can appear as subcutaneous nodules Or masses, which can be accompanied by pain or the skin turns blue.
This situation is also more common in infants
.
Professor Yuan Xiaojun reminded that parents should pay more attention to their children's physical changes as their children grow up.
If they find that their children have the above-mentioned common symptoms of neuroblastoma, they should consult a doctor in time to avoid missing the best time for treatment
.
Pathological examination is the "gold standard" for diagnosing neuroblastoma.
Professor Yuan Xiaojun told the "medical community" that patients with neuroblastoma need multiple examinations before treatment, not only for the diagnosis as soon as possible, but also to determine the stage and stage of neuroblastoma.
The risk level provides a basis for doctors to evaluate the difficulty of surgery and formulate treatment plans
.
Combined with clinical cases, she suggested that the clinical manifestations and therapeutic effects of neuroblastoma are very different, and it is easy to be misdiagnosed.
If the child has symptoms such as bone pain and repeated fever, it should be paid attention to
.
Specific diagnostic tests include: (1) Pathological examination: mass removal, incision biopsy or needle biopsy; (2) bone marrow smear or biopsy, based on GD2 immunocytology testing; (3) urine test: 24-hour urine catecholamines and their metabolism Product (vanillamandelic acid [VMA] or homovanillic acid [HVA]); (4) imaging examination (B-ultrasound, CT, MRI, etc.
); (5) blood test: serum neuron-specific enolase (NSE)
.
Among them, pathological examination is the most reliable method for diagnosing neuroblastoma.
It usually uses directly obtained lesion tissue, slices, and stains.
After necessary immunohistochemical processing, the pathologist observes the tumor cell morphology under a microscope and analyzes it.
The results are written into a report
.
Pathological examination is the most reliable method for diagnosing neuroblastoma, which is the so-called "gold standard"
.
Therefore, the diagnosis of neuroblastoma needs to meet one of the following conditions: 1) Obtain a positive pathological diagnosis of tumor tissue under light microscope; 2) Bone marrow aspiration smear and biopsy found characteristic neuroblasts (small round cells, nested) Or chrysanthemum pattern arrangement; anti-GD2 antibody staining is positive), and accompanied by elevated serum NSE or catecholamine metabolites in urine
.
In addition, imaging examinations for neuroblastoma include ultrasound, CT, MRI, isotope bone scan and MIBG scan, etc.
, which can help determine the stage and risk group of children, and provide a basis for accurately formulating treatment plans
.
She believes that promoting the standardized diagnosis and treatment of neuroblastoma, enhancing public awareness of neuroblastoma, and reducing misdiagnosis and misdiagnosis are particularly important for improving the prognosis of children with neuroblastoma
.
The staging and treatment of neuroblastoma, Professor Wang Huanmin, director of the Department of Oncology, Beijing Children's Hospital, introduced that before treating neuroblastoma, a comprehensive assessment of the patient is required, and the disease must be staged in detail
.
Professor Wang Huanmin, Director of Oncology Surgery, Beijing Children's Hospital, has reported that there are two staging systems for neuroblastoma, of which the International Neuroblastoma Staging System (INSS) is the most used staging system
.
According to the extent to which the tumor can be surgically removed, INSS divides neuroblastoma into the following stages: Stage 1: 21%; local tumor is completely removed, with or without minimal residual lesions, and ipsilateral lymph nodes are negative under the microscope
.
Stage 2A: 15%; Stage 2A: Complete resection of the local tumor; microscopically, the tumor is positive for non-adhesive lymph nodes on the same side
.
Stage 2B: The local tumor is completely or incompletely removed, and the ipsilateral non-adhesive lymph nodes of the tumor are positive, and the contralateral enlarged lymph nodes are negative under microscope
.
Stage 3: 17%; unresectable unilateral tumors exceed the midline, with/without local lymph node invasion; or localized unilateral tumors with contralateral regional lymph node involvement; or midline tumors with extended contralateral invasion (unresectable) or Lymph node involvement
.
Stage 4: 41%; metastasis to distant lymph nodes, bones, bone marrow, liver, skin or other organs (except stage 4S)
.
4S stage: 6%; localized tumors of stage I or II, with distant metastases such as liver, skin and (or) bone marrow, and the age is less than 12 months
.
Professor Wang Huanmin pointed out that according to the stage of the disease and the physical condition of the patient, an individualized comprehensive treatment plan is designed, supplemented with appropriate supportive treatment, and the treatment plan is revised in time during the treatment process in order to improve the treatment benefit of the patient
.
At present, children with high-risk neuroblastoma usually undergo many rounds of complex and intensive treatment, which require multidisciplinary joint diagnosis and treatment including surgery, internal medicine, radiotherapy, transplantation, imaging, pathology, nutrition, psychology, and pain.
.
High-risk children usually need chemotherapy, surgery, stem cell transplantation, and radiotherapy, which are divided into induction phase, consolidation phase and maintenance phase
.
However, before the introduction of anti-GD2 (an antigen present on the surface of neuroblastoma cells, which is the most ideal therapeutic target for neuroblastoma) immunotherapy, the options for the maintenance phase are limited
.
Among high-risk neuroblastoma patients who have completed induction and consolidation therapy, nearly 60% relapsed without immunotherapy, and more than 50% of high-risk children and more than 75% of relapsed children survive for less than 5 years
.
It is reported that dartuximab β is an immunotherapy targeted to GD2, and combined with the specific target GD2 over-expressed by neuroblastoma cells, it can activate adaptive and innate immune responses and make the immune system destroy neuroblastoma.
Of cancer cells, leading to neuroblastoma cell death
.
In 2017, dartuximab beta was approved for marketing by the European Commission.
This is the only European Medicines Agency (EMA) approved by the European Medicines Agency (EMA) as of November 2020 that can be used for the treatment of newly treated high-risk and relapsed refractory neuroblastoma at the same time.
Targeted immunotherapy
.
In August 2021, BeiGene's dartuximab β injection was approved for marketing by the National Medical Products Administration (NMPA) of China
.
Experts believe that with the continuous in-depth understanding of the contents of pre-treatment staging and risk grouping by doctors, as well as the strengthening of domestic awareness of early diagnosis and treatment of childhood tumors, the advancement of inspection methods, and the optimization of treatment methods, neuroblastoma will be formulated in the future.
"Combat strategy" will be clearer and more precise
.
Focus on rare diseases in children According to World Health Organization statistics, in addition to accidental injuries, malignant tumors have become the number one cause of death among children in China
.
Among them, neuroblastoma (NB) is called "the king of childhood tumors" and is the most common extracranial solid tumor in infants and young children
.
According to the statistics of childhood cancer in the United States in 2014, new cases of neuroblastoma accounted for 8% to 10% of all malignant tumors in children
.
The incidence rate in China is about 10.
1/100 million (children between the ages of 0 and 14).
The number of new cases of children is about 3,000 each year.
About 50% of neuroblastomas occur in infants and young children under 2 years of age
.
It is reported that neuroblastoma has the characteristics of high degree of malignancy, rapid disease progression, and difficult treatment.
In particular, children with high risk and refractory relapse often face severe survival challenges
.
With the advancement of diagnosis and treatment, under timely and standardized active treatment, children with neuroblastoma have also obtained more and more opportunities for cure
.
On September 12, focusing on the opportunities and progress of neuroblastoma and precision treatment, at the inaugural meeting of the neuroblastoma community co-sponsored by the God Mother Sunshine Home and the Pain Challenge Foundation, Professor Wang Huanmin, Director of Oncology Surgery, Beijing Children’s Hospital, Professor Yuan Xiaojun, director of the Pediatric Hematology Oncology Department of Xinhua Hospital, Shanghai Jiaotong University School of Medicine, and other pediatric oncologists discussed
.
Signs and symptoms of neuroblastoma Neuroblastoma is derived from undifferentiated sympathetic ganglion cells.
Any part with embryonic sympathetic ganglion cells has the potential for malignancy.
The adrenal gland is the most common source of neuroblastoma.
Hair site
.
Yuan Xiaojun, director of the Department of Pediatric Hematology and Oncology, Xinhua Hospital, Shanghai Jiaotong University School of Medicine, introduced that the clinical manifestations of neuroblastoma are quite heterogeneous, and the clinical manifestations are different according to the location and scope of the primary tumor and metastatic tumor
.
Professor Yuan Xiaojun, director of the Department of Pediatric Hematology and Oncology, Xinhua Hospital, Shanghai Jiaotong University School of Medicine, "Because cases of abdominal tumors are the most common, the clinical manifestations of neuroblastoma are mostly abdominal masses
.
" It is reported that tumors in the abdomen or pelvis may cause abdominal distension.
, But usually no tenderness.
The child may complain of abdominal pain, do not want to eat, and have symptoms such as weight loss, constipation or diarrhea, and changes in bowel habits; in a few cases, high blood pressure may even occur
.
Tumors on the chest and neck may appear as hard lumps on the body surface and are usually non-tender.
If the tumor compresses the large veins, it will cause swelling of the face, neck, arms, and upper chest, and blue or red ecchymosis may appear on the skin
.
Professor Yuan Xiaojun pointed out that in clinical practice, neuroblastoma is often difficult to detect, especially some primary doctors who lack experience in diagnosing neuroblastoma.
When the tumor invades other parts, patients will also have other symptoms and signs
.
If the tumor compresses the sympathetic nerves of the neck, the child may develop Horner syndrome, which is manifested by ptosis of the eyelids and unilateral miosis
.
When the tumor squeezes into the throat or trachea, it may cause coughing, breathing, and swallowing difficulties; head tumors may compress nerves and blood vessels in the brain, causing headache, dizziness or changes in consciousness
.
In addition, tumors near the spine may compress nerves, affecting sensation and mobility, and children may be affected when standing, walking or crawling; tumors around the orbit may cause bruises (commonly known as "panda eyes"), swelling, and protruding eyeballs.
; Bone marrow metastasis can cause anemia, manifested as a pale complexion, and may also lead to thrombocytopenia, which is not easy to stop bleeding; bone metastasis can cause bone pain
.
If the tumor infiltrates the liver, the liver will swell and may cause respiratory distress, coagulation disorders, gastrointestinal and kidney function damage.
These conditions are more common in young infants; when the patient has skin or subcutaneous metastases, it can appear as subcutaneous nodules Or masses, which can be accompanied by pain or the skin turns blue.
This situation is also more common in infants
.
Professor Yuan Xiaojun reminded that parents should pay more attention to their children's physical changes as their children grow up.
If they find that their children have the above-mentioned common symptoms of neuroblastoma, they should consult a doctor in time to avoid missing the best time for treatment
.
Pathological examination is the "gold standard" for diagnosing neuroblastoma.
Professor Yuan Xiaojun told the "medical community" that patients with neuroblastoma need multiple examinations before treatment, not only for the diagnosis as soon as possible, but also to determine the stage and stage of neuroblastoma.
The risk level provides a basis for doctors to evaluate the difficulty of surgery and formulate treatment plans
.
Combined with clinical cases, she suggested that the clinical manifestations and therapeutic effects of neuroblastoma are very different, and it is easy to be misdiagnosed.
If the child has symptoms such as bone pain and repeated fever, it should be paid attention to
.
Specific diagnostic tests include: (1) Pathological examination: mass removal, incision biopsy or needle biopsy; (2) bone marrow smear or biopsy, based on GD2 immunocytology testing; (3) urine test: 24-hour urine catecholamines and their metabolism Product (vanillamandelic acid [VMA] or homovanillic acid [HVA]); (4) imaging examination (B-ultrasound, CT, MRI, etc.
); (5) blood test: serum neuron-specific enolase (NSE)
.
Among them, pathological examination is the most reliable method for diagnosing neuroblastoma.
It usually uses directly obtained lesion tissue, slices, and stains.
After necessary immunohistochemical processing, the pathologist observes the tumor cell morphology under a microscope and analyzes it.
The results are written into a report
.
Pathological examination is the most reliable method for diagnosing neuroblastoma, which is the so-called "gold standard"
.
Therefore, the diagnosis of neuroblastoma needs to meet one of the following conditions: 1) Obtain a positive pathological diagnosis of tumor tissue under light microscope; 2) Bone marrow aspiration smear and biopsy found characteristic neuroblasts (small round cells, nested) Or chrysanthemum pattern arrangement; anti-GD2 antibody staining is positive), and accompanied by elevated serum NSE or catecholamine metabolites in urine
.
In addition, imaging examinations for neuroblastoma include ultrasound, CT, MRI, isotope bone scan and MIBG scan, etc.
, which can help determine the stage and risk group of children, and provide a basis for accurately formulating treatment plans
.
She believes that promoting the standardized diagnosis and treatment of neuroblastoma, enhancing public awareness of neuroblastoma, and reducing misdiagnosis and misdiagnosis are particularly important for improving the prognosis of children with neuroblastoma
.
The staging and treatment of neuroblastoma, Professor Wang Huanmin, director of the Department of Oncology, Beijing Children's Hospital, introduced that before treating neuroblastoma, a comprehensive assessment of the patient is required, and the disease must be staged in detail
.
Professor Wang Huanmin, Director of Oncology Surgery, Beijing Children's Hospital, has reported that there are two staging systems for neuroblastoma, of which the International Neuroblastoma Staging System (INSS) is the most used staging system
.
According to the extent to which the tumor can be surgically removed, INSS divides neuroblastoma into the following stages: Stage 1: 21%; local tumor is completely removed, with or without minimal residual lesions, and ipsilateral lymph nodes are negative under the microscope
.
Stage 2A: 15%; Stage 2A: Complete resection of the local tumor; microscopically, the tumor is positive for non-adhesive lymph nodes on the same side
.
Stage 2B: The local tumor is completely or incompletely removed, and the ipsilateral non-adhesive lymph nodes of the tumor are positive, and the contralateral enlarged lymph nodes are negative under microscope
.
Stage 3: 17%; unresectable unilateral tumors exceed the midline, with/without local lymph node invasion; or localized unilateral tumors with contralateral regional lymph node involvement; or midline tumors with extended contralateral invasion (unresectable) or Lymph node involvement
.
Stage 4: 41%; metastasis to distant lymph nodes, bones, bone marrow, liver, skin or other organs (except stage 4S)
.
4S stage: 6%; localized tumors of stage I or II, with distant metastases such as liver, skin and (or) bone marrow, and the age is less than 12 months
.
Professor Wang Huanmin pointed out that according to the stage of the disease and the physical condition of the patient, an individualized comprehensive treatment plan is designed, supplemented with appropriate supportive treatment, and the treatment plan is revised in time during the treatment process in order to improve the treatment benefit of the patient
.
At present, children with high-risk neuroblastoma usually undergo many rounds of complex and intensive treatment, which require multidisciplinary joint diagnosis and treatment including surgery, internal medicine, radiotherapy, transplantation, imaging, pathology, nutrition, psychology, and pain.
.
High-risk children usually need chemotherapy, surgery, stem cell transplantation, and radiotherapy, which are divided into induction phase, consolidation phase and maintenance phase
.
However, before the introduction of anti-GD2 (an antigen present on the surface of neuroblastoma cells, which is the most ideal therapeutic target for neuroblastoma) immunotherapy, the options for the maintenance phase are limited
.
Among high-risk neuroblastoma patients who have completed induction and consolidation therapy, nearly 60% relapsed without immunotherapy, and more than 50% of high-risk children and more than 75% of relapsed children survive for less than 5 years
.
It is reported that dartuximab β is an immunotherapy targeted to GD2, and combined with the specific target GD2 over-expressed by neuroblastoma cells, it can activate adaptive and innate immune responses and make the immune system destroy neuroblastoma.
Of cancer cells, leading to neuroblastoma cell death
.
In 2017, dartuximab beta was approved for marketing by the European Commission.
This is the only European Medicines Agency (EMA) approved by the European Medicines Agency (EMA) as of November 2020 that can be used for the treatment of newly treated high-risk and relapsed refractory neuroblastoma at the same time.
Targeted immunotherapy
.
In August 2021, BeiGene's dartuximab β injection was approved for marketing by the National Medical Products Administration (NMPA) of China
.
Experts believe that with the continuous in-depth understanding of the contents of pre-treatment staging and risk grouping by doctors, as well as the strengthening of domestic awareness of early diagnosis and treatment of childhood tumors, the advancement of inspection methods, and the optimization of treatment methods, neuroblastoma will be formulated in the future.
"Combat strategy" will be clearer and more precise
.
