Table 4.
Characteristics of patient with other advanced cancers characteristics.
| No | Age [Years], Sex | ECOG Status | Diagnosis | Treatment (Number of Cycles) | Previous Lines of Therapy | Effect of Treatment | Adverse Events (CTCAE Grade) |
|---|---|---|---|---|---|---|---|
| 1 | 12.1/M | 1 | Alveolar RMS of pancreas | NIVO (51) | 3 | CR (OS = 32.1 months) | Hyperthyroidism (3) |
| 2 | 17.8/M | 2 | Mediastinal gray zone lymphoma | NIVO (9) | 4 | CR (OS = 7.3 months) | None |
| 3 | 15.4//M | 2 | Osteosarcoma | NIVO (27) | 4 | PD | None |
| 4 | 16.8/M | 2 | Osteosarcoma | NIVO (6) | 2 | PD | Pseudoprogression |
| 5 | 16.4/F | 0 | Renal cell carcinoma | NIVO (17) | 2 | PD | Hypothyroidism (2) |
| 6 | 3.4/M | 0 | Hepatoblastoma | PEMBRO (10) | 9 | PD | Rash |
| 7 | 5.3/M | 2 | DIPG of brain | NIVO (2) | 1 | PD | Pneumonia (3) |
| 8 | 16.6/M | 3 | Medulloblastoma of cerebellum | NIVO (4) | 1 | PD | None |
| 9 | 17.9/F | 2 | Extrasceletal chondrosarcoma | NIVO (2) | 3 | PD | None |
| 10 | 0.7/F | 4 | Rhabdoid tumor of kidney | NIVO (2) | 1 | PD | None |
| 11 | 1.5/M | 4 | Rhabdoid tumor of liver | NIVO (1) | 1 | PD | None |
| 12 * | 18.0/M | 2 | Gastric cancer with lymphoid stroma | PEMBRO (3) | 1 | PD | None |
| 13 | 16.9/F | 0 | Adrenal cortex cancer | NIVO (3) | 3 | PD | None |
| 14 | 11.9/F | 0 | Ovarian germ cell tumor | PEMBRO (1) | 5 | PD | None |
| CUMULATIVE | Median age: 15.9 years (M = 9; F = 5) | Poor general condition (ECOG ≤ 2) in 9/14 patients | Most of the above diagnoses are ultra-rare cancers for the pediatric population | Median ICIs doses: 3.5 | Median: 3 | CR in two patients | Good tolerance of therapy in most patients; no patient required termination of therapy due to side effects |
* presence of a mutation in BRAF gene, F: female; M: male; RMS: rhabdomyosarcoma; DIPG: diffuse intrinsic pontine glioma; NIVO: nivolumab; PEMBRO: pembrolizumab; ICIs: immune checkpoint inhibitors; CR: complete remission; OS: overall survival; PD: progressive disease.