Results of the Childhood Cancer and Leukaemia Group's United Kingdom Relapsed Wilms Tumour Trial.

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All Authors

Vaidya, SJ.
Moroz, V.
Hale, J.
Pavasovic, V.
Hobson, R.
Sartori, P.
Saunders, D.
Powis, M.
Vujanic, GM.
Baker, S.

LTHT Author

Powis, Mark

LTHT Department

Leeds Children's Hospital
Paediatric Surgery

Non Medic

Publication Date

2026

Item Type

Journal Article Clinical Trial Multicenter Study

Language

Subject

CLINICAL TRIALS AS TOPIC , CHEMOTHERAPY , RECURRENCE , KIDNEY NEOPLASMS , PAEDIATRICS , ANTINEOPLASTIC AGENTS , ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS , CHILD

Subject Headings

Abstract

BACKGROUND: The United Kingdom relapsed Wilms tumour (UKW-R) trial aimed to improve the historically low survival rates after relapse of Wilms tumour (WT) through a prospective national risk-stratified protocol. The trial also evaluated efficacy and toxicity of high-dose melphalan. METHODS: Patients with relapsed/refractory WT were allocated to one of three treatment groups based on time to relapse and initial therapy. Group A: Patients with Stage 1 non-anaplastic WT treated only with vincristine (VCR) or up to two doses of VCR + actinomycin D (ACT) relapsing > 6 months from diagnosis received 'intensive' AVD (ACT + VCR + doxorubicin [DOX]). Group B: Patients with Stage 2 non-anaplastic WT treated with VCR + ACT relapsing > 6 months from diagnosis received eight alternating courses of DOX/cyclophosphamide (CPM) and CPM/etoposide (ETOP). Group C: All other patients whose initial therapy included additional DOX, other drugs and/or radiotherapy received six alternating courses of carboplatin (CARBO)/ETOP and CPM/ETOP followed by melphalan + autologous stem cell rescue (M + ASCT). All patients were recommended radiotherapy +/- surgery for sites of relapse. RESULTS: From 2002 to 2008, 78 children were enrolled (Group A: 13, Group B: 10 and Group C: 55). Median age was 5.3 years. 38 children received M + ASCT with no transplant related mortality. 25 children died (5: Group A + B and 20: Group C), all from tumour related causes. The 4-year event-free and overall survival for the whole group were 63% and 68%, and 77%/81% (Group A + B) and 57%/63% (Group C), respectively. Median follow-up for alive patients is 65.4 months (8.1-155.4). DISCUSSION: Survival rates following risk stratification including M + ASCT are higher than historical observations for similar high-risk groups that did not include high-dose chemotherapy.

Journal

Pediatric Blood & Cancer