John Libbey Eurotext



Juvenile myelomonocytic leukemia Volume 23, issue 2, Mars-Avril 2017


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1 Inserm UMR_S1131,
Institut universitaire d’hématologie,
hôpital Saint Louis,
Paris, France
2 Assistance Publique des Hôpitaux de Paris (AP-HP),
hôpital Robert Debré,
service d’hématologie pédiatrique,
Paris, France
3 Assistance Publique des Hôpitaux de Paris (AP-HP),
hôpital Robert Debré,
département de génétique,
Paris, France
* Tirés à part

Juvenile myelomonocytic leukemia is an agressive myeloproliferative/myelodysplastic syndrome of childhood due to the hyperactivation of the RAS pathway. Clinical heterogeneity and lack of specific biologic test make this rare malignancy difficult to diagnose. In approximately one third of patients, JMML occurs in a context of underlying constitutive condition, the RASopathies that require specific patient care. Thus, it is now strongly recommended to analyse germline DNA and to investigate this issue with the help of an onco-geneticist. Recent deciphering of the molecular basis of JMML has provided valuable help in the therapeutic stratification and it is now admitted that, for some patients, the watch-and-wait strategy is the best therapeutic option. Nevertheless, most patients still require hematopoietic stem cell transplantation that cures only half of the patients. Demethylating agents and new targeted molecules are currently on development to improve JMML prognosis.