Table 3. Patient Characteristics, Reported Outcomes, and Adverse Drug Reactions of the 5 Cohorts of ≥10 Patients Treated With the Same Medicine and Indication.
| Cohort No. | Innovative therapy | Tumor type | Biomarker | Patients, No. | Age at inclusion, median (range), y | Prior lines of therapy, median (range) | Time since diagnosis, median (range), y | ORR/PFS | PFS, median (range), d | Patients still on therapy at last follow-up | SACHA recommendation to SFCE centers | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Trametinib | Low grade glioma | BRAF fusion | 23 | 6.8 (0.7-16.5) | 3 (1-10) | 4.1 (0.4-14.8) | ORR, 5/23 (5 PR) | 275 (40-663) | 12/23 | Continue | |
| 2 | Trametinib | Low grade glioma | NF1 germline variant | 13 | 7.6 (3.3-13.7) | 2 (1-5) | 4.4 (2.0-10.4) | ORR, 4/13 (4 PR) | 334 (222-576) | 6/13 | Continue | |
| 3 | Trametinib | Plexiform neurofibroma | NF1 germline variant | 10 | 6.5 (0.3-13.5) | 0 (0-1) | 2.7 (0.1-7.1) | ORR, 0/10 | 455 (103-786) | 9/10 | No longer prescription | |
| 4 | Dabrafenib /trametinib | Low grade glioma | BRAF variant | 28 | 10.5 (0.8-19.2) | 2 (0-9) | 3.5 (0.2-16.5) | ORR, 19/27 (18 PR, 1 CR) | 372 (34-796) | 21/28 | Continue | |
| 5 | Regorafenib | Osteosarcoma | None | 13a | 17.3 (10.9-22.8) | 2 (1-6) | 1.8 (0.7-13.6) | 4-mo PFS, 37% | 125 (26-513) | 5/13 | Continue | |
Abbreviations: CR, complete response; ORR, objective tumor responses; PFS, progression-free survival; PR, partial response; SACHA, Securing Access to Innovative Therapies for Children, Adolescents, and Young Adults with Cancer Used Outside Clinical Trials; SFCE, French Society of Pediatric Oncology.
a
Two patients not evaluable (follow-up <4 months, and regorafenib ongoing at last follow-up).