Table 1. Characteristics of Patients Included in the SACHA-France Study.
| Diagnosis | Patients, No. (N = 342) | No. of patients with ≥2 therapies | Targeted molecular alteration (No. of patients)a | Innovative therapies (No. of patients)a |
|---|---|---|---|---|
| CNS tumors (164 patients) | ||||
| Low-grade glioma | 95 | 3 | BRAF variant (35); BRAF fusion–positive (28); BRAF duplication (2); NF1 germ variant (15) | Trametinib (54); dabrafenib plus trametinib (36); selumetinib (2) |
| High-grade glioma | 46 | 3 | H3K27M variant (21); BRAF variant (7); ACV1 variant (2) | ONC201 (24); Dabrafenib plus trametinib (5); trametinib (3); vandetanib (2) |
| Atypical teratoid rhabdoid tumor | 7 | NA | SMARCA/B deletion/variant (7) | Tazemetostat (7) |
| Ependymoma | 2 | 1 | NA | NA |
| Medulloblastoma | 4 | NA | PTCH1 variant (3) | Vismodegib (3) |
| Other CNS tumors | 10 | 4 | MET fusion (3); ALK fusion (2) | Crizotinib (3) |
| Non-CNS tumors (122 patients) | ||||
| Osteosarcoma | 24 | 1 | NA | Regorafenib (14); cabozantinib (10) |
| Ewing sarcoma | 17 | NA | NA | Cabozantinib (9); regorafenib (5); pazopanib (2) |
| Plexiform neurofibroma | 13 | NA | NF1 germ variant (12) | Trametinib (11); selumetinib (2) |
| Neuroblastoma | 12 | NA | ALK variant (5) | Lorlatinib (5); Dinutuximab beta/chemotherapy (4) |
| Desmoid tumor | 6 | NA | NA | Pazopanib (6) |
| Rhabdomyosarcoma | 5 | NA | NA | Pazopanib (3) |
| Thyroid carcinoma | 5 | NA | RET variant (2); SMARCA/B deletion/variant (2) | Tazemetostat (2) |
| Inflamatory myofibroblastic tumor | 4 | NA | ALK fusion (2) | Crizotinib (3) |
| Hepatoblastoma | 3 | 1 | NA | NA |
| Infantile fibrosarcoma | 3 | NA | NTRK fusion (3) | Larotrectinib (3) |
| Melanoma | 3 | NA | NA | Nivolumab (2) |
| Other soft tissue sarcoma | 11 | 1 | NTRK fusion (4) | Larotrectinib (3); pazopanib (3) |
| Other non-CNS tumors | 15 | 1 | BRAF variant (3); SMARCA/B deletion/variant (2) | Dabrafenib plus trametinib (2); pazopanib (2); tazemetostat (2); atezolizumab (2) |
| Lymphoma (20 patients) | ||||
| Hodgkin lymphoma | 10 | NA | NA | Brentuximab/nivolumab (6); brentuximab (4) |
| Non-Hodgkin lymphoma | 10 | NA | ALK fusion (7) | Alectinib (6); anti-CD20/CD3 (2) |
| Leukemia (36 patients) | ||||
| Acute myeloblastic leukemia | 16 | 2 | ALK fusion (2) | Venetoclax/azacitidine (9); venetoclax (3) |
| B-cell acute lymphoblastic leukemia | 12 | 0 | BCL-ABL variant (2) | Inotuzumab (6); ponatinib (3) |
| T-cell acute lymphoblastic leukemia | 1 | NA | NA | NA |
| Other leukemia | 7 | 2 | NA | NA |
Abbreviations: CNS, central nervous system; NA, not applicable.
a
Minimum 2 patients.