If a child is diagnosed with acute myeloid leukemia (AML), as there are not many drugs available, chemotherapy and bone marrow transplantation are generally used at present. However, standard treatment may not be suitable for all, thus increasing the risk of relapse.
In order to provide more options for sick children, the Faculty of Medicine of the Chinese University of Hong Kong conducted drug screening for a total of 52 children with AML in Hong Kong, and then combined the drugs with genetic and clinical data analysis, and based on the situation of each sick child, to find out The most effective targeted drug for him.
Two of the sick children in the study tried targeted drugs after repeated relapses and successfully extended their survival by one year and two and a half cases. The results have been published in "Blood Cancer Discovery".
CUHK has also established a patient database based on the data of each sick child, which will help speed up the identification of suitable medicines for the sick child in the future.
CUHK conducted drug screening for a total of 52 children with AML in Hong Kong, and then analyzed the drugs combined with genetic and clinical data, and identified the most effective target drugs for each patient based on the condition of each patient.
(Photo by Lu Yingshan)
Every year, 10 children in Hong Kong are newly diagnosed with acute myeloid leukemia (AML). After their diagnosis, they will receive standard treatment, namely chemotherapy and bone marrow transplantation. However, some patients may be resistant to chemotherapy, which increases the risk of relapse.
At present, 30% to 40% of children with AML in Hong Kong still relapse after receiving treatment. Once the relapse rate is lower than 40%, the survival period is only two to three months.
Every year, 10 children in Hong Kong are newly diagnosed with acute myeloid leukemia (AML).
(provided by respondents)
The medical community has also discussed the use of targeted therapy in the past, but because AML in children is relatively rare, most of the targeted drugs are used in adults, and there are few targeted therapy for children, and the effect is not good. .
In order to provide one more choice for sick children, CUHK conducted a study earlier to screen the bone marrow samples of each sick child against 45 FDA-approved and highly safe drugs, and then combined the drugs with genetic and clinical data analysis to find Develop the most suitable medicines for sick children, and set up precise treatment plans tailored for sick children.
Study: Seven targeted drugs are effective in children with AML
The study found that seven targeted drugs are very effective for most children with AML. Six of them are sold in Hong Kong and are mainly used for the treatment of recurrent tumors in adults. They are not the first-line treatment drugs for children with AML.
The study also prescribed appropriate targeted drugs to two children with multiple relapses of AML, one of whom was a 14-month-old girl.
The girl relapsed for the third time after receiving chemotherapy. After drug screening, CUHK prescribed the most effective targeted drug for her.
The baby girl was able to successfully control her condition after taking the drug and prolong her life by up to a year.
Another 14-year-old boy took targeted drugs when he relapsed for the second time. His condition was successfully relieved and his survival was extended by two and a half years.
Li Zhiguang, a professor at the Department of Pediatrics, Faculty of Medicine, CUHK, said that baby girls and boys did not experience any side effects when taking the targeted drugs. Taking the drug and recommending to stop it, both of them eventually died due to relapse of the disease.
He emphasized that if targeted drugs can be used before they relapse, the effect will be even better. In the future, he may hope that after they receive chemotherapy treatment, their bone marrow samples will be checked for cancer cells. If there are still 0.01% of cancer cells, Treat it with medicine.
Li Zhiguang, a professor at the Department of Pediatrics, Faculty of Medicine, CUHK, said that the baby girl and boy had no side effects when taking the targeted drug.
(Photo by Lu Yingshan)
Has KMT2C gene variant standard treatment or not effective
In addition, the study also found that if children with AML have the KMT2C gene mutation, the current standard treatment, namely chemotherapy and bone marrow transplantation, cannot increase their survival rate, and "100% will die." He said that there are currently 10% of children with AML have the above variants, and CUHK found that Venetoclax's target drug may be able to treat related children after analyzing its gene variants.
CUHK's Excellence in Children's Health Research has been approved by the government for 6.5 million to conduct related research. Earlier, the data of each sick child has been established to establish a high-dimensional patient database of children with acute myeloid leukemia combined with genetic, drug and clinical data.
(Photo by Lu Yingshan)
At present, even if children with AML participate in the research, they still need to undergo chemotherapy before receiving the precise treatment plan.
Leung Kam-tang, Assistant Professor of the Department of Paediatrics, Faculty of Medicine, CUHK said that there are not many clinical data on the use of targeted drugs in children with AML, and they cannot completely replace the current standard treatment. Are there any relevant precedents in the world, ask the parents of sick children and remind them of the risks.
CUHK’s Excellence in Children’s Health Research has been approved by the government for 6.5 million to carry out related research. The data of each sick child has been established earlier to establish a high-dimensional patient database of children with acute myeloid leukemia combined with genetic, drug and clinical data, which will help speed up the Find the right medicines to give to sick children.
Liang Jintang also plans to expand the research scope to all children diagnosed with leukemia in the future.
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