Title

Outcomes for Children With Type II and Type III Pleuropulmonary Blastoma Following Chemotherapy: A Report From the International PPB/ Registry

Authors

Kris Ann Schultz, International Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, MN.
Anne K. Harris, International Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, MN.
Alexander T. Nelson, International Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, MN.
Dave Watson, Research Institute, Children's Minnesota, Minneapolis, MN.
John T. Lucas, Department of Radiation Oncology, St Jude Children's Research Hospital, Memphis, TN.
Doug Miniati, Division of Pediatric Surgery, Kaiser Permanente Northern California, Roseville, CA.
Douglas R. Stewart, Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD.
Kelly N. Hagedorn, Department of Radiology, Children's Minnesota, Minneapolis, MN.
William Mize, Department of Radiology, Children's Minnesota, Minneapolis, MN.
Junne Kamihara, Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.
Sarah G. Mitchell, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA.
David B. Wilson, Department of Pediatrics, Washington University School of Medicine, St Louis Children's Hospital, St Louis, MO.
Katie Gettinger, Department of Pediatrics, Washington University School of Medicine, St Louis Children's Hospital, St Louis, MO.
Arun A. Rangaswami, Division of Pediatric Hematology and Oncology, University of California San Francisco, San Francisco, CA.
Laura A. Harney, Westat, Rockville, MD.
Carlos Rodriguez Galindo, Department of Global Pediatric Medicine, St Jude Children's Research Hospital, Memphis, TN.
Gianni Bisogno, Hematology, Oncology and Stem Cell Transplant Division, Department of Women's and Children's Health, University of Padova, Padova, Italy.
Louis P. Dehner, Lauren V. Ackerman Laboratory of Surgical Pathology, Department of Pathology and Immunology, Washington University School of Medicine, St Louis, MO.
D Ashley Hill, Division of Pathology, Children's National Medical Center, George Washington University School of Medicine and Health Sciences, Washington, DC.
Yoav H. Messinger, International Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, MN.

Document Type

Journal Article

Publication Date

9-22-2022

Journal

Journal of clinical oncology : official journal of the American Society of Clinical Oncology

DOI

10.1200/JCO.21.02925

Abstract

PURPOSE: Pleuropulmonary blastoma (PPB) is the most common primary lung neoplasm of infancy and early childhood. Type II and type III PPB have historically been associated with a poor prognosis. METHODS: Patients with known or suspected PPB were enrolled in the International PPB/ Registry. Medical records were abstracted with follow-up ascertained annually. All PPB diagnoses were confirmed by central pathology review. Beginning in 2007, the IVADo regimen (ifosfamide, vincristine, actinomycin-D, and doxorubicin) was recommended as a potential treatment regimen for children with type II and type III PPB. This regimen was compared with a historical control cohort. RESULTS: From 1987 to 2021, 314 children with centrally confirmed type II and type III PPB who received upfront chemotherapy were enrolled; 132 children (75 with type II and 57 with type III) received IVADo chemotherapy. Adjusted analyses suggest improved overall survival for children treated with IVADo in comparison with historical controls with an estimated hazard ratio of 0.65 (95% CI, 0.39 to 1.08). Compared with localized disease, distant metastasis at diagnosis was associated with worse PPB event-free survival and overall survival with hazard ratio of 4.23 (95% CI, 2.42 to 7.38) and 4.69 (95% CI, 2.50 to 8.80), respectively. CONCLUSION: The use of IVADo in children with type II and type III PPB resulted in similar-to-improved outcomes compared with historical controls. Inferior outcomes with metastatic disease suggest the need for novel therapies. This large cohort of uniformly treated children with advanced PPB serves as a benchmark for future multicenter therapeutic studies for this rare pediatric tumor.

Department

Pathology

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