The efficacy and safety of bevacizumab/irinotecan/temozolomide (BIT) for relapsed/refractory neuroblastoma: the UK Children’s Cancer and Leukaemia Group experience
The efficacy and safety of bevacizumab/irinotecan/temozolomide (BIT) for relapsed/refractory neuroblastoma: the UK Children’s Cancer and Leukaemia Group experience
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.
Jackson, Thomas J.
e731cecb-5b71-4506-875e-c2449f323d28
Gray, Juliet
12d5e17c-97bb-4d6d-8fc4-3914b730ed42
Jackson, Thomas J.
e731cecb-5b71-4506-875e-c2449f323d28
Gray, Juliet
12d5e17c-97bb-4d6d-8fc4-3914b730ed42
Jackson, Thomas J., Shamma, Menna and Senanayake, Upeka
,
et al.
(2026)
The efficacy and safety of bevacizumab/irinotecan/temozolomide (BIT) for relapsed/refractory neuroblastoma: the UK Children’s Cancer and Leukaemia Group experience.
Pediatric Blood and Cancer.
(doi:10.1002/1545-5017.70226).
Abstract
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.
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Pediatric Blood Cancer - 2026 - Jackson - The Efficacy and Safety of Bevacizumab Irinotecan Temozolomide BIT for
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Accepted/In Press date: 16 February 2026
e-pub ahead of print date: 16 March 2026
Identifiers
Local EPrints ID: 510420
URI: http://eprints.soton.ac.uk/id/eprint/510420
ISSN: 1545-5017
PURE UUID: c827b29a-2060-433c-a7ba-9bb9e3590cf6
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Date deposited: 30 Mar 2026 17:00
Last modified: 31 Mar 2026 01:39
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Author:
Thomas J. Jackson
Author:
Menna Shamma
Author:
Upeka Senanayake
Corporate Author: et al.
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