Severe Hepatopathy in National Wilms Tumor Studies 3-5: Prevalence, Clinical Features, and Outcomes After Reintroduction of Chemotherapy

Oosterom N, Gooskens SLM, Renfro LA, Perlman EJ, van den Heuvel-Eibrink MM, Hamilton TE, Green DM, Grundy PE, Daw NC, Geller JI, Dome JS, Fernandez CV, Mullen EA. Severe Hepatopathy in National Wilms Tumor Studies 3-5: Prevalence, Clinical Features, and Outcomes After Reintroduction of Chemotherapy. J Clin Oncol. 2023 Sep 10;41(26):4247-4256. doi: 10.1200/JCO.22.02555. Epub 2023 Jun 21. PubMed PMID: 37343199; PubMed Central PMCID: PMC10852371.

The safety of reintroducing chemotherapy in the pediatric renal tumor setting after severe hepatopathy (SH), including sinusoidal obstruction syndrome (SOS), is uncertain. We describe the incidence, severity, outcomes, and impact on subsequent treatment for patients with SH from National Wilms Tumor Study (NWTS) protocols 3-5. Archived charts for patients enrolled on NWTS 3-5 who met study inclusion criteria for SH by using established hepatopathy grading scales and clinical criteria were reviewed for demographics, tumor characteristics, radio- and chemotherapy details, SH-related dose modifications, and oncologic outcomes. Genomic analysis for candidate polymorphisms associated with SH was performed in 14 patients.