Table 3.
Off-label targeted therapies in children with leukemia or lymphoma.
| Targeted Therapy | Description | Off-Label Use | Grade ≥ 3 Infection Risk |
|---|---|---|---|
| ALL | |||
| Inotuzumab ozogamicin (Besponsa) [176] | ADC recombinant humanized IgG4κ mAb linked to N-acetyl-gamma-calicheamicin → CD22 |
r/r Ph-positive BCP-ALL after failure of ≥1 TKI; r/r CD22-positive B-ALL | 10.5% to 20% |
| Bortezomib (Velcade) | 26S proteasome inhibitor | r/r ALL | 16.2% to 45.5% |
| Also: bosutinib (Bosulif) and ponatinib (Iclusig) | |||
| AML | |||
| Venetoclax (Venclexta, Venclyxto) [177] | BCL2 inhibitor | r/r AML | 15.3% to 50% |
| FLT3 inhibitors midostaurin (Rydapt) and gilteritinib (Xospata), and IDH inhibitor enasidenib mesylate (Idhifa) | |||
| HL | |||
| Brentuximab vedotin or SGN-35 (Adcetris) [178] | ADC chimeric IgG1 mAb linked to MMAE → CD30 | r/r classical HL | 3.4% to 5.3% for monotherapy; 3.3% to 15.6% when combined with chemotherapy |
| Also: nivolumab (Opdivo) and atezolizumab (Tecentriq) | |||
| NHL | |||
| Brentuximab vedotin or SGN-35 (Adcetris) [178] | As in HL above | ||
| Also: ceritinib (Zykadia), atezolizumab (Tecentriq), nivolumab (Opdivo), ipilimumab (Yervoy), and ibritumomab (Zevalin) | |||
MMAE = monomethyl auristatin E.