CONCLUSION:
Relapsed/refractory BPCDN has limited therapeutic options and an overall
dismal outcome. Conventional therapies have shown to be beneficial but
transient remission forced to undergo allogeneic stem cell
transplantation. Targeted therapy in combination with bcl-2 inhibitor
molecule or demethylating agents are promising but rarity of the disease
is an obstacle for prospective studies. Data for BPDCN treatment is
extremely limited in pediatric age group. This is the first case report
for combination molecular targeted therapies which can be safe and well
tolerated options for difficult to treat, chemoresistant patients.