Chemotherapy for Children With Type II or III Pleuropulmonary Blastoma

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In an analysis from the International PPB/DICER1 Registry reported in the Journal of Clinical Oncology, Schultz et al found that chemotherapy with IVADo (ifosfamide, vincristine, actinomycin-D, and doxorubicin) appeared to be associated with similar or improved outcomes vs historical controls among children with type II or III pleuropulmonary blastoma.

Study Details

The study population consisted of 314 patients (173 with type II and 141 with type III pleuropulmonary blastomas) who received upfront chemotherapy between 1987 to 2021. IVADo became a recommended treatment in this setting in 2007. Of the 314 patients, 132 (75 with type II and 57 with type III) received this combination regimen; the remaining 182 patients who did not receive this combination regimen constituted the historical control group.

Key Findings

In adjusted analyses, the adjusted hazard ratios (HRs) for the IVADo group vs historical controls were 0.77 (95% confidence interval [CI] = 0.51–1.18) for pleuropulmonary blastoma event-free survival and 0.65 (95% CI = 0.39–1.08) for overall survival. Adjusted hazard ratios were 1.16 (95% CI = 0.64–2.09) for pleuropulmonary blastoma event-free survival and 0.93 (95% CI = 0.43–2.01) for overall survival among those with type II disease and 0.52 (95% CI = 0.28–0.97) and 0.51 (95% CI = 0.26–1.01), respectively, among those with type III disease.

Among all patients, the presence of distant metastasis vs localized disease at diagnosis was associated with significantly poorer pleuropulmonary blastoma event-free survival (HR = 4.23, 95% CI = 2.42–7.38) and overall survival (HR = 4.69, 95% CI = 2.50–8.80).

The investigators concluded: “The use of IVADo in children with type II and type III pleuropulmonary blastoma 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 pleuropulmonary blastoma serves as a benchmark for future multicenter therapeutic studies for this rare pediatric tumor.”

Kris Ann P. Schultz, MD, of the Department of Cancer and Blood Disorders, Children’s Minnesota, Minneapolis, is the corresponding author of the Journal of Clinical Oncology article.

Disclosure: The study was supported by grants from the National Institutes of Health, National Cancer Institute, and others. For full disclosures of the study authors, visit

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