This is a rare and specific group of patients, which Eline Bertrums, PhD student/clinical researcher, has studied. Eline explains: 'Previously, this syndrome, called infantile myeloproliferative disease (IMD), was mainly known in babies with Down syndrome, but it appears to occur in other newborns as well. It is important to recognize this disease because the picture is very similar to leukemia. Leukemia is usually treated with intensive chemotherapy, but in this case it is not necessary because the picture disappears on its own. Chemotherapy also damages healthy cells and has side effects, so you gain a lot if you don't have to give chemotherapy.
Guideline
Together with pediatric oncologists Prof. Marry van den Heuvel-Eibrink and Dr. Bianca Goemans and an international AML working group, Eline Bertrums collected cases for research into this rare disease. Also in the context of her PhD project. Eline: 'With a group of international experts we had several online discussions to reach consensus on the guideline for diagnostics and treatment of this rare group of patients. I am very happy that so many people internationally have contributed to this guideline, because due to the rarity of this disorder you really need this cooperation. The newly identified patients and the corresponding guideline have now been published online.' Bianca Goemans is enthusiastic: 'The guideline is a practical tool for all practitioners dealing with this disease. It indicates step by step to which category a patient belongs. It is a rare disease, but the flow chart gives a good overview and insight.
Watch & Wait
'Transient leukemia' in newborns is very rare. But if a child has this disease, a dilemma immediately arises: If it does turn out to be leukemia, treatment must be given quickly and if it could be a transient condition, you don't want to give chemotherapy unnecessarily. Eline says: 'The guidelines that we have drawn up help to make the right diagnosis and to weigh up all the options carefully. ‘Watch & wait' means that you will not treat a patient with intensive chemotherapy because you have a strong suspicion of a transient picture. Since neonates can develop symptoms that do require chemotherapy treatment, and it is difficult to make the diagnosis with 100% certainty, you continue to monitor this patient closely with physical examination and monitoring of blood values. I can imagine that this is difficult for parents to grasp because there is indeed something going on in the blood of their baby, and they have to wait and see. It can be an uncertain period with hospitalization or regular hospital visits. Therefore, I think good explanation and communication about this is very important. I hope that with the guideline we will help doctors better distinguish between acute leukemia and this transient condition and thus choose the right treatment (or no treatment).’