APHON Pediatric Chemotherapy-Biotherapy Renewal

Nonreceptor tyrosine kinase inhibitors

Chronic myeloid leukemia (CML) Philadelphia chromosome– positive acute lymphoblastic leukemia (Ph+ ALL)

BCR-ABL, cKIT, stem cell factor

Cytopenia T prolongation Cardiomyopat hies

Imatinib (2006, 2013)

These agents target signaling proteins on the inside surface of a cell membrane.

CML Ph+ ALL

BCR-ABL, cKIT, PDGFR

Dasatinib (2018, 2019)

CML

BCR-ABL

Nilotinib (2018)

Relapsed and refractory acute leukemia

BTK

Ibrutinib

Relapsed or refractory leukemia T-cell leukemia

JAK

Cytopenia Fatigue Diarrhea

Ruxolitinib (2017)

Cytoplasmic Inhibitors (also known as serine/threonine kinase inhibitors)

Sorafenib Dabrafenib Vemurafenib

Relapsed or refractory solid tumors or leukemias

BRAF

Skin rashes Arthralgias Fatigue Elevated liver enzymes

Langerhans cell histiocytosis Melanoma High-grade gliomas

These agents target signaling proteins along pathways inside of a cell.

Trametinib

High-grade gliomas

MEK

Skin rashes Diarrhea Fatigue

Relapsed or refractory solid tumors Complex vascular anomalies

Sirolimus

mTOR, Pi3 Diarrhea Mucositis

Elevated liver enzymes