MEDICATION INDICATION DIAGNOSES INITIAL DOSE/ REGIMEN LENGTH OF THERAPY POTENTIAL COMPLICATIONS† POTENTIAL COMPLICATIONS†
Sirolimus
Bone lytic lesions Effusions LM engorgement Pain GLA GSD KLA CCLA†† 0.8mg/m2/dose PO every 12 hours* Trough target level: 8-12ng/mL
Indefinite OR transition to other targeted therapy
Oral mucositis High triglycerides Immunosuppression (consider PJP prophylaxis) Oral mucositis High triglycerides Immunosuppression (consider PJP prophylaxis)
Interferon alpha 2b
Bone lytic lesions Effusions GLA GSD KLA
Pegylated form: 1.5mcg/kg/dose subQ weekly
Indefinite (but may be limited due to complications)
Flu-like symptoms with administration Hypothyroidism
Vincristine
Coagulopathy Effusions
KLA
>10kg: 1.5mg/m2/dose IV weekly initially, max 2mg/dose ≦10 kg: 0.05mg/kg/dose
Taper off as coagulopathy improves
Peripheral neuropathy Constipation Bone pain SIADH Extravasation risk Hair loss Peripheral neuropathy Constipation Bone pain SIADH Extravasation risk Hair loss
Steroids
Coagulopathy Effusions
KLA
Methylprednisone IV 1.6mg/kg/day OR Prednisolone PO 2mg/kg/day, initially
Taper off over 2-3 months if possible
Hyperglycemia Increased appetite Stomach/duodenal ulcers Weight change Mood/sleeping disturbances Hyperglycemia Increased appetite Stomach/duodenal ulcers Weight change Mood/sleeping disturbances
Zoledronic acid
Bone lytic lesions Pathogenic fractures GLA GSD KLA
VAC: 0.05 mg/kg/dose IV every 6 months, max 4mg/dose
2 years max
Infusion reactions (including anaphylaxis) Osteonecrosis of jaw Electrolyte disturbances Atypical femur fracture Infusion reactions (including anaphylaxis) Osteonecrosis of jaw Electrolyte disturbances Atypical femur fracture
HVMC: 4mg/m2/dose IV monthly x 3 doses, then every 3 months, max 4mg/dose
Reassess need after 10 doses 5 years max
Trametinib
Bone lytic lesions Effusions Coagulopathy (KLA) KLA CCLA†† 0.025mg/kg/dose PO once daily, max 2mg/dose** 0.5mg, 1mg, 2mg tablets available only
Indefinite OR transition to other targeted therapy
Skin rash Edema Diarrhea Increased liver enzymes Hair lightening or loss Oral mucositis Skin rash Edema Diarrhea Increased liver enzymes Hair lightening or loss Oral mucositis