Zahra Hudda, Aaron Webster, Eric Anderson, Priscila Badia, Rochelle Bagatell, Saleh Bhar, Alan Bidgoli, Karla Boyd, Sonali Chaudhury, Gabriel Salinas Cisneros, John Craddock, Stella M Davies, Ami V. Desai, Lianna Drobatz, Christopher C. Dvorak, Hannah Elkus, Vanessa A. Fabrizio, Jennifer H Foster, Ellen Fraint, Jason L. Freedman, Jorge Galvez, Mark Garcia, Ann Haight, Christine Higham, Sonata Jodele, Aarti Kamat, Saara Kaviany, Leslie Lehmann, Kathryn Leung, Katherine T. Lind, Gabriela Llaurador, Malika Kapadia, Sajad Khazal, Kayla Massi, Meaghan Mormann, Luke Pater, Hemalatha G. Rangarajan, Seth Rotz, Anthony Sabulski, Sarah Sartain, Michelle L. Schoettler, Elizabeth Sokol, Keri A. Streby, Matthew Stein, Michael R. Verneris, Brian D. Weiss, Sahr Yazdani, Daniel Zheng, Christopher E Dandoy
Cincinnati Children’s Hospital Medical Center and University of Cincinnati College of Medicine. Rady Children’s Hospital and University of California San Diego. University of Pennsylvania and Children’s Hospital of Philadelphia. Baylor College of Medicine and Texas Children’s Hospital. Children’s Healthcare of Atlanta and Emory University. Ann and Robert H. Lurie Children’s Hospital of Chicago and Northwestern University Feinberg School of Medicine. University of California, San Francisco. University of Chicago. Children’s Hospital of Colorado and University of Colorado School of Medicine. Nemours Children’s Hospital. Nicklaus Children’s Hospital. Methodist Children’s Hospital. Boston Children’s and Harvard University Medical School. Loma Linda School of Medicine. Nationwide Children’s Hospital and Ohio State University. Cleveland Clinic Children’s Hospital and Cleveland Clinic Lerner College of Medicine of Case Western Reserve University. Indiana University School of Medicine and Riley Hospital for Children.
United States
Transplantation and Cellular Therapy
Transplant Cell Ther 2025;
DOI: 10.1016/j.jtct.2025.10.022
Abstract
Background: High-dose chemotherapy with tandem autologous stem cell transplantation (ASCT) is a standard approach for pediatric high-risk neuroblastoma (HR-NB). However, data on acute regimen-related toxicities are limited.
Objective: This study evaluates treatment-related toxicities and day-180 outcomes following tandem ASCT in children with HR-NB.
Study design: We conducted a multi-institutional (n=13) retrospective review including pediatric patients with HR-NB scheduled for tandem ASCT between January 2014 and June 2021. Descriptive analyses were performed to evaluate organ toxicities and transplant-related outcomes, focusing on endothelial injury.
Results: 255 patients received ASCT #1 with Thiotepa/Cyclophosphamide (TT/Cy). Fourteen patients were unable to proceed to ASCT #2, due to death (n=9), with underlying disease relapse in 7 of the 9 (78%). Severe endothelial toxicities, including veno-occlusive disease (VOD) and transplant-associated thrombotic microangiopathy (TA-TMA) in 3 patients, prevented progression to tandem ASCT. The remaining 241 (94.5%) completed tandem ASCT with TT/Cy and Carboplatin/Etoposide/Melphalan (CEM). Post-ASCT #2 complications included bloodstream infections (17%), ICU admission (20%), respiratory failure requiring intubation (12%), pulmonary hypertension (4%), and acute kidney injury (18%), with 5% requiring dialysis. VOD occurred in 9% of patients, and TA-TMA occurred in 16% of patients completing tandem ASCT. Six-month survival was 94% for tandem ASCT recipients.
Conclusion: This study highlights the impact of regimen-related toxicities in pediatric HR-NB patients undergoing tandem ASCT. Despite a modest six-month mortality rate, the burden of endotheliopathies contributed to morbidity and mortality post-ASCT #2. Improved screening and early intervention strategies may help mitigate transplant-related morbidity.
Category
Class V. Pulmonary Hypertension Associated with Hematological, Systemic, Metabolic, Nutritional and Other Disorders
Age Focus: Pediatric Pulmonary Vascular Disease
Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication
Article Access
Free PDF File or Full Text Article Available Through PubMed or DOI: No