Pediatric blood & cancer · 2026
The Efficacy and Safety of Bevacizumab/Irinotecan/Temozolomide (BIT) for Relapsed/Refractory Neuroblastoma: The UK Children's Cancer and Leukaemia Group Experience.
СтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод пока не подготовлен
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Jackson Thomas J, Shamma Menna, Senanayake Upeka, Sahmoud Shaimaa, Duffin Kathleen, Howard Victoria, King David, Fathani Ayesha, Lumb Blanche, Pobischan Andrei, Memon Irum Latif, Ahmed Monzir, Thomas Rhian, Millen Gerard, Alomari Yasmeen, Vaidya Sucheta, Marshall Lynley V, George Sally L, Ramanujachar Ramya, Gray Juliet, Angelini Paola
Аннотация
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.
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.