Outcomes for children with type II and type III pleuropulmonary blastoma (PPB) was similar to improved compared with an historical control group when treated with a regimen of ifosfamide, vincristine, actinomycin-D, and doxorubicin (IVADo). These findings were published in the Journal of Clinical Oncology.

Pleuropulmonary blastoma, a rare but often aggressive pediatric lung neoplasm, is diagnosed primarily in children younger than 7 years. It includes 4 subtypes: type I is a cystic lesion, type II is mixed cystic and solid, type III is a solid multipatterned sarcoma, and type Ir is a cystic lesion lacking a primitive malignant component.

Historically, prognosis is poor for children with type II and type III PPB. The International PPB/DICER1 Registry (ClinicalTrials.gov Identifier: NCT03382158) was founded to advance research for this rare tumor. In 2007, the International PPB/DICER1 introduced a roadmap for IVADo.

Continue Reading

This analysis sought to evaluate outcomes for children with type II and type III PPB who were treated with IVADo. Data was analyzed to determine if those treated with IVADo fared better than an historical control group.

The researchers obtained data from 314 children with type II (173 patients) and type III (141 patients) PPB who were enrolled in the registry. The IVADo cohort included 132 children from 108 centers in 24 countries treated with the regimen.

Their results showed improved overall survival in children treated with IVADo compared to children in the historical cohort (estimated hazard ratio, 0.65). Distant metastasis at the time of diagnosis was associated with worse PPB event-free survival and overall survival compared with patients with localized disease.

More than one-quarter of children with type II PPB and nearly half of children with type III PPB will die as a result of their cancer, noted the researchers. All the children with type III PPB with distant metastatic disease included in this analysis experienced an event within 15 months of diagnosis.

“Novel strategies to prevent on-treatment and post-treatment recurrence are needed,” stated the researchers in their discussion, adding that they encourage future international collaboration to work toward better treatments for children with advanced PPB.

Limitations for this study include the rarity of PPB, which limits study population and the retrospective nature of the registry research.

Disclosures: Some authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.


Schultz KAP, Harris AK, Nelson AT, et al. Outcomes for children with type II and type III pleuropulmonary blastoma following chemotherapy: a report from the International PPB/DICER1 Registry. J Clin Oncol. Published online September 22, 2022. doi:10.1200/JCO.21.02925