Guideline for management of non-Down syndrome neonates with a myeloproliferative disease on behalf of the I-BFM AML Study Group and EWOG-MDS
Abstrakt
In neonates with myeloid hyperproliferation, apart from benign causes, Down syndrome (DS) related transient abnormal myelopoiesis (TAM), acute myeloid leukemia (AML) and juvenile myelomonocytic leukemia (JMML) are considered. Besides TAM, rarely, non-DS related transient myeloproliferative diseases occur, making clinical decisions challenging.4 TAM, according to World Health Organization (WHO) classification, only applies to children with (mosaic) Down syndrome.5 In the past, different terminology has been used in non-DS patients, such as transient myeloproliferative disease (TMD) and transient leukemia.
Since distinction from TAM is important, and it is challenging to determine whether this disease will be transient, the consensus group introduced the novel term 'infantile myeloproliferative disease' (IMD), in order to distinguish it from TAM. Both TAM and IMD can usually be managed with a 'watch and wait' strategy, while most fullblown AML or JMML cases require intensive treatment.
We collected rare IMD cases from study groups collaborating in the International Berlin-Frankfurt-Münster AML Study Group (I-BFM AML SG). In addition, we reviewed the literature for neonatal cases of malignant myeloid hyperproliferation without DS.
Based on these data, we developed, together with I-BFM AML SG and the European Working Group of Myelodysplastic syndromes in Childhood (EWOG-MDS) members, by consensus, clinical recommendations for the diagnostic approach and current adequate classification of malignant myeloid hyperproliferation in infancy. This is meant guiding clinicians in choosing the right strategy, i.e., whether to 'watch and wait' or start highly intensive treatment in individual cases.