Table 1.
Demographics of three children with newly diagnosed ACVR1-mutant DIPG treated with vandetanib and mTOR inhibitors upfront or at relapse
| Case | Age, y | Sex | Histology | Mutations | RT, Gy/n | Timing of combination | Cycles | Previous therapies | EFS months, Dx/combination | OS months, Dx/combination | Status |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 7 | Female | DMG | HIST2H3C p.K27M | 54/30 | Upfront | 7 | Everolimus | 11/6.5 | 20/9 | Deceased |
| ACVR1 p.G328V | |||||||||||
| BCOR p.Met1020fs | |||||||||||
| 2 | 4 | Female | DMG | HIST1H3B p.K27M | 54/30 | Upfront | 1 | Bevacizumab | 5/1 | 9/5 | Deceased |
| ACVR1 p.G328E | |||||||||||
| 3 | 4 | Female | DMG | HIST1H3B p.K27M | 54/30 | Relapse | 1 | Re-RT, bevacizumab, sirolimus | 8/0.7 | 16/0.7 | Deceased |
| PIK3CA p.E542K | |||||||||||
| PIK3CA p.E542K |
NOTE: Cases 1 and 3 were treated with vandetanib/everolimus; case 2 was treated with vandetanib/sirolimus.
Abbreviations: DMG, diffuse midline glioma; Dx, diagnosis; EFS, event-free survival; re-RT, reirradiation; RT, radiotherapy.