This child underwent an allogenic stem cell transplant. He had to be treated for his HLH initially. Part of it involved chemotherapy (FLA) as JMML was the underlying cause driving the HLH.
The choice between ‘watch and wait ‘ vs HSCT depends on the underlying genetic subgroup along with presence or absence of somatic and germline mutations.
Increasingly DNA methylation status is being used determine prognosis and probability of relapse.
The use of azacytidine prior to HSCT in one subgroup is also being explored. Other targeted therapy trials for relapsed refractory patients are in the pipeline.
Most patients as of now, eventually require allogenic HSCT for cure of their disease.