Table 1.
Features, outcome and long term morbidities of patients with neurofibromatosis type 1.
| Age at diagnosis of LGG (y) | Sex | Primary diagnosis and treatment details | LGG diagnosed by screening MRI | First mode of treatment | Treatment details and disease course | Number of progressions requiring Tx | Latest disease status | Duration of follow up from initial diagnosis (y) | Associated morbidities |
|---|---|---|---|---|---|---|---|---|---|
| 0.34 | F | Hypothalamic/optic pathway PA | NF1 diagnosed after LGG | Chemotherapy | Vincristine, at progression: cyclophosphamide → focal RT=49.5 Gy, biopsy at PD) – developed secondary GBM | 3 | Died of secondary GBM | 3.52 | VF defect, endocrinopathy, stroke, hemiparesis, seizure (after diagnosis) |
| 0.34 | F | Hypothalamic/optic pathway PA | NF1 diagnosed after LGG | Observation | At progression: carboplatin, topotecan (prolonged myelosuppression) → vincristine → focal RT | 2 | SD off therapy | 22.23 | VF defect, impaired VA, endocrinopathy, scoliosis, moyamoya, TIA, history of seizure, obesity, IQ = 74 |
| 0.68 | F | Optic pathway glioma (no biopsy) | Yes | Observation | At progression: carboplatin, vincristine → focal RT | 2 | SD off therapy | 14.21 | Endocrinopathy, scoliosis, moyamoya, stroke, hemiparesis, history of seizure, IQ = 77 |
| 0.98 | F | Optic pathway glioma (no biopsy) | Yes | Observation | At progression: carboplatin, vincristine, temozolomide → vinblastine | 2 | SD off therapy | 11.54 | Endocrinopathy, scoliosis, overweight, history of seizure, IQ = 76 |
| 0.01 | F | Tectal glioma (no biopsy) | NF1 diagnosed after LGG | Observation | Observation only | 0 | SD without therapy | 9.74 | Impaired VA, strabismus, seizure, IQ = 89 |
| 0.61 | F | Optic Pathway Glioma (no biopsy) | NF1 diagnosed after LGG | Observation | At progression: carboplatin, vincristine, temozolomide → vinblastine → selumetinib | 3 | SD on therapy | 5.33 | Impaired VA, scoliosis, IQ = 65 |
| 0.21 | F | Optic Pathway Glioma (no biopsy) | NF1 diagnosed after LGG | Chemotherapy | Carboplatin/vincristine → at progression: vinblastine → bevacizumab/irinotecan | 2 | SD off therapy | 6.78 | Impaired VA, endocrinopathy, obesity, scoliosis, hemiparesis, history of seizure |
Abbreviations: GBM, glioblastoma multiforme; IQ, intelligent quotient; LGG, low-grade glioma; PA, pilocytic astrocytoma; PD, progressive disease; RT, radiotherapy; SD, stable disease; TIA, transient ischemic attack; Tx, treatment; VA, visual acuity; VF, visual field; y, years