The Efficacy and Safety of Bevacizumab/Irinotecan/Temozolomide (BIT) for Relapsed/Refractory Neuroblastoma: The UK Children's Cancer and Leukaemia Group Experience.
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BACKGROUND: Patients with high-risk neuroblastoma who either are refractory to induction chemotherapy or relapse following multi-modal treatment have a dismal prognosis. Based on data from the BEACON trial, since 2021 the UK national guidelines recommend bevacizumab, irinotecan, and temozolomide (BIT) for patients with relapsed/refractory disease. METHODS: A retrospective UK national study of patients under 25 years with relapsed/refractory neuroblastoma treated with BIT 1/3/2021-28/02/25. RESULTS: Sixty-six patients were included from 15 UK centres; 40 (61%) had relapsed disease, 23 at first relapse. Overall objective response rate was 40.6% [95% CI: 28.5%-53.6%] and was higher in patients with refractory versus relapsed disease (56% vs. 31%, p = 0.068). Objective responses were achieved within six cycles for over 90%. Seventeen of 26 (65%) patients with primary refractory disease received high-dose chemotherapy with busulfan/melphalan and autologous stem cell rescue. Nine required additional therapies after BIT before they could proceed to high-dose treatment, and eight required BIT alone. Progression-free survival (PFS) and overall survival (OS) were significantly longer in refractory patients (median PFS 13.2 vs. 5.1 months, p = 0.012, median OS not reached by 3 years vs. 12.5 months, p = 0.0003). The most common CTCAE5.0 Grade 3-4 toxicities were anaemia (32%), neutropenic fever (23%) and diarrhoea (13.6%). One patient discontinued treatment due to toxicity (diarrhoea). CONCLUSIONS: The promising efficacy and tolerability of BIT reported by the BEACON trial are reproduced in real-world data with a larger cohort. Further randomised studies are needed to separately identify optimal treatment strategies for relapsed and refractory disease.