APHON Pediatric Chemotherapy-Biotherapy Renewal

Subependymal giant cell astrocytoma in pediatric patients with tuberous sclerosis complex Relapsed or refractory solid/ or CNS tumors Relapsed or refractory solid tumors High-risk hepatoblastoma Lymphangiomatosis

Everolimus (2012)

Temsirolimus

Bortezomib

T-cell leukemia T-cell lymphoma

NF-kB

Fever Nausea Diarrhea Neuropathies

Alisertib

Neuroblastoma

AURKA

Cytopenia

BCL-2 inhibitors

Venetoclax

Relapsed or refractory acute leukemia

BCL-2

Cytopenia Electrolyte disturbances Diarrhea Nausea

These agents target proteins that regulate apoptosis

Relapsed or refractory acute lymphoblastic leukemia or lymphoma Multiple myeloma Diffuse intrinsic pontine glioma Cutaneous T-cell lymphoma Relapsed or refractory solid tumors and CNS malignancies Relapsed or refractory acute leukemia High-grade gliomas Neuroblastoma

Navitoclax

Epigenic modulators Histone deacetylase (HDAC) inhibitors

Panobinostat

Vorinostat

Thrombocyto penia Neutropenia

These agents target enzymes within cells (HDACs) that are responsible for regulating gene expression

CNS = central nervous system; FDA = U.S. Food and Drug Administration.