Outcome of neurofibromatosis type 1 patients treated with first line vinblastine for optic pathway gliomas: A Canadian multicenter study.

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2019 Background: To date, the first line chemotherapy treatment in the majority of countries for children with NF-1 and OPG is vincristine + carboplatin. Toxicity of this regimen consists mostly in neuropathy, allergic reactions, and hearing loss. Vinblastine has shown promising activity in a phase II study in children with recurrent/refractory low grade glioma (LGG). The aim of this study was to assess the activity of vinblastine in chemotherapy naïve children, and to assess the toxicity profile. Methods: Patients < 18 years old with unresectable or progressive LGG were eligible if they had not received any previous treatment with chemotherapy or radiation. Vinblastine was administered weekly at a dose of 6 mg/m2 over a period of 70 weeks. Patients who showed progression on 2 consecutive imaging studies or evidence of clinical progression were removed from treatment. Results: Overall, the study enrolled 54 patients with LGG. A total of 13 patients (24.1%) had NF-1. Patients with NF-1were younger at diagnosis: median age 3.84 y (range, 1.74-16.36 y) vs. 7 years in non-NF-1. Tumor location in all NF-1 patients was the optic pathway. Treatment was very well tolerated, however, 5 patients (38%) needed dose reductions. Most common toxicity was hematological: only 1 patient who experienced grade 3+ neutropenia (vs. 10 patients non-NF1). There were only 2 episodes of febrile neutropenia, no RBC transfusions and no toxic death. Best response to chemotherapy was assessed centrally by an independent radiologist: 2 PR, 1 MR, 8 SD, and 2 PD, for a response rate of 23.1%. At a median follow-up of 5.37 years (3.45 – 6.57 years): Only two NF-1patients had progression. Five year progression free survival (PFS) was 85.1± 9.7% (vs. 42±7,9% for all non-NF1, p = 0.01; and 41.7±14% for non-NF1 with OPG, p = 0.01). None of the NF1 patients received radiation (0 vs. 6 patients non-NF1). No patients died of progression (0 vs. 3 patients non-NF1). Conclusions: Weekly vinblastine is well tolerated and can be used in NF-1 children with OPG as first line chemotherapy with good results. The toxicity profile is lower than with other chemotherapies, offering a better quality of life to these patients. Clinical trial information: NCT00575796.

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