Many parents fall into endless despair when they learn that their child’s bone marrow transplant match has failed, or that they cannot raise up to hundreds of thousands of dollars for a transplant.
However, experts in pediatric leukemia treatment say that for most children with acute lymphoblastic leukemia, bone marrow transplantation is not only not the only path, but also not the first option for treatment.
Compared with adult patients, standardized chemotherapy for children with leukemia is significantly more effective than bone marrow transplantation, with clinical cure rates of up to 80%. Conversely, children with bone marrow transplantation may face long-term complications such as inability to have children, secondary tumors, and does not mean that they will not relapse in the future.
Parents must be careful about the trade-offs.
After a bone marrow transplant, is it a one-time thing?
Many parents believe that chemotherapy is time-consuming, relapse-prone, and costly in terms of time and money, whereas a bone marrow transplant can be a once-and-for-all solution.
This is a misconception, and a bone marrow transplant is not a one-time event.
Many experts say that the cure rate for bone marrow transplantation is only 50% to 60% and is not optimal for pediatric patients.
Professor Luo Xuequn, director of the Department of Pediatric Hematology at the First Hospital of Sun Yat-sen University, pointed out that the international consensus is that children who have been critically evaluated by their doctors as having less than a 40% to 50% chance of cure with chemotherapy need a bone marrow transplant. In these high-risk patients, leukemia cells are difficult to remove and require increased intensity of chemotherapy to overcome drug-resistant cancer cells, but too intense chemotherapy can simultaneously destroy the normal hematopoietic cells of the bone marrow and destroy hematopoietic function. This situation requires transplantation of hematopoietic stem cells (or bone marrow) to allow the patient’s hematopoietic function to be reestablished, as well as immune function to be reestablished, further clearing the body of leukemia cells.
In the past, there were few children with bone marrow transplants and few study samples, but now it has been found that the problems after bone marrow transplantation are not small.
Long-term problems, which are often overlooked compared to immediate problems such as rejection, are:
Difficulty in having children as adults
Many patients who receive bone marrow transplants are unable to have children.
This is because the high-dose chemotherapy, including alkylating agents, during bone marrow transplantation kills sperm cells indiscriminately in addition to leukemia cells.
Some children who receive bone marrow transplants reach the relationship stage and are unaware that they cannot have children.
High risk of secondary tumors
Many doctors have found that children who receive bone marrow transplants have a higher risk of secondary tumors, such as lymphoma, than the average child.
The drugs used to treat leukemia all have side effects, including those used in chemotherapy and bone marrow transplants, and the choice of chemotherapy or bone marrow transplant is the lesser of two evils, given the priority of saving lives.
In recent years, treatment options for childhood leukemia have been improving and side effects have been greatly reduced.
In children with acute leukemia, about 90% don’t need a bone marrow transplant
Thirty to 40,000 new pediatric malignancies are diagnosed in China each year, and one-third of them are leukemias.
Today, some hospitals do not have a pediatric hematology ward and admit children to internal medicine hematology departments, where they are treated according to adult protocols or unregulated pediatric protocols.
In fact, pediatric leukemia is different from adult leukemia in many ways, and treatment according to adult protocols can do more harm than good.
There are two main types of acute leukemia in children, acute lymphoblastic leukemia (acute gonorrhea) and acute myeloid leukemia. Of these, acute gonorrhea accounts for about 80% and acute myeloid leukemia accounts for about 20%.
Leukemia is treated in a stratified manner according to risk, and can be classified as standard-risk, intermediate-risk, or high-risk based on leukemic cell genetic analysis, immunophenotype, blood picture, and response to therapy. In children with acute gonorrhea, for example, 35%, 50% and 15% of patients are standard-risk, intermediate-risk and high-risk, respectively, and usually only some high-risk patients require bone marrow transplantation. In other words, more than 85% or even 90% of pediatric patients with acute gonorrhea do not require bone marrow transplantation.
But most adult patients with acute gonorrhea need a bone marrow transplant to be cured. The first time I saw a child with a bone marrow transplant, I was able to see the difference between a child with a bone marrow transplant and an adult with a bone marrow transplant. Some parents feel that they have not raised enough money for a transplant and are afraid to bring their children in for treatment, leading to delays in treatment.
However, unlike pediatric acute myeloid leukemia, more than half of children have an indication for bone marrow transplantation because of their poor prognosis. Although this is true, bone marrow transplantation is not the only way forward, and studies have shown that many children can still be cured with chemotherapy.
Patients with “late adolescence” should be treated on a pediatric regimen
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Many people are very vague about the concept of “cured leukemia” and often mistake the medical term “5-year survival rate” or “10-year survival rate” for patients living only 5 or 10 years. The first time I saw a patient with leukemia, I was able to see that he or she would have a 5-year survival rate.
Professor Luo Xuequn explained that acute myeloid leukemia is in remission for 5 years after treatment, which means 5 years of disease-free survival, with little chance of relapse; remission for 8 to 10 years is considered a cure. If you are able to stay in remission for 5 years, you can be considered cured.
Clinical cure rates for children under 14 years of age with acute gonorrhea are 50%, 70%-80%, and more than 80% with standard pediatric regimen chemotherapy, respectively. In contrast, only 20% of adults with acute gonorrhea are clinically cured with chemotherapy, which is four times worse than in children.
Why is there such a big difference between adult and pediatric patients with the same leukemia? Professor Luo Xuequn pointed out that there are significant differences in the immunophenotype and leukemia genes between children and adults with acute gonorrhea, and that the proportion of adult patients with high-risk leukemia is high. Another important reason is that adults, especially those of advanced age, are poorly tolerant of chemotherapy, so they cannot use too strong a drug, while children have a strong regenerative capacity and recover easily from chemotherapy, so they can be “heavily medicated” and are naturally more effective.
For “late adolescent” leukemia patients between the ages of 16 and 20, because of their similar characteristics to children and their ability to tolerate strong chemotherapy, numerous clinical trials and studies have shown that these patients are better treated with pediatric regimens.
Regulated chemotherapy lasts at least 6-7 months
Parents are often reluctant to undergo chemotherapy for fear that “their child will not be able to tolerate the pain of chemotherapy.
Standardized chemotherapy is about staging and stratification, with appropriate treatment regimens chosen based on chromosomal and genetic characteristics, age, and leukocyte base at the time of initiation for standard, intermediate, and high-risk stratification. In children, for example, clear stratification is usually achieved by day 33 after the start of treatment. It is important to emphasize that chemotherapy must be administered in its entirety, and that intermittent treatment in some children can easily result in leukemia cells that are resistant and insensitive to chemotherapeutic agents.
The primary chemotherapy for children with acute gonorrhea standard and intermediate risk is completed in about six to seven months, after which they can take the drugs home for about a year and a half of maintenance therapy. In general, it costs about $220,000 for a high-risk child, $150,000 for an intermediate-risk child, and $100,000 to $120,000 for a standard-risk child.