Individual dosing of preparatory drug makes stem cell transplants safer for children
Individual dosing of a drug used to prepare children for stem cell transplantation makes this treatment safer; that's according to new research by scientists from the Princess Máxima Center and the Wilhelmina Children's Hospital.
A stem cell transplant can form an important part of treatment for some children with leukemia. Preventing the immune system from rejecting the new stem cells is key to the success of this treatment. To that end, children are pre-treated with a drug called anti-thymocyte globulin (ATG). This drug weakens the immune system so that the new blood stem cells can carry on making healthy blood cells.
In this new study published in the leading journal The Lancet Haematology, researchers studied the effect of personalized dosing of ATG. The research was funded by the pharmaceutical company Sanofi, a manufacturer of this drug, and ZonMw.
A pediatrician specialized in stem cell transplants and one of the study's co-leading authors, explains: “The better the immune system recovers after a transplant, the better a child's chances of survival. We suspected that too high a dose and wrong timing of giving a child ATG, could hinder the recovery of the donor immune cells.”
In the past, the dose of ATG was determined on the basis of body weight. But in the new study, the number of immune cells before the stem cell transplant is also included in choosing the right dose for each child.
It should be noted, Some 58 children who received a stem cell transplant between 2015 and 2018, took part in the study. One of the researchers on the team, compared the immune recovery and survival of these patients with that of children previously treated with the standard dose of ATG. In 4 out of 5 children, the immune system fully recovered within 100 days of the stem cell transplant. Also, a similar proportion survived for at least 3 years after the stem cell transplant (1).
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