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Outcomes for Children With Type II and Type III Pleuropulmonary Blastoma Following Chemotherapy: A Report From the
Kris Ann P Schultz1,2,3, Anne K Harris1,2,3, Alexander T Nelson1,2,3,4
1International Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, MN.
Insights
The IVADo chemotherapy regimen shows similar-to-improved outcomes for children with advanced pleuropulmonary blastoma (PPB). Metastatic disease indicates a need for new therapies in this rare pediatric tumor.
Area of Science:
- Pediatric Oncology
- Thoracic Surgery
- Rare Diseases
Background:
- Pleuropulmonary blastoma (PPB) is the most common primary lung neoplasm in infants and young children.
- Types II and III PPB historically have a poor prognosis, necessitating improved treatment strategies.
Purpose of the Study:
- To evaluate the efficacy of the IVADo chemotherapy regimen for treating type II and type III pleuropulmonary blastoma in children.
- To compare outcomes of IVADo treatment with historical control data.
Main Methods:
- Patients with confirmed type II or III PPB were enrolled in the International PPB/DICER1 Registry.
- The IVADo regimen (ifosfamide, vincristine, actinomycin-D, doxorubicin) was recommended from 2007 onwards and compared to a historical cohort.
- Outcomes were analyzed using adjusted survival analyses.
Main Results:
- A total of 314 children with type II/III PPB received upfront chemotherapy; 132 received IVADo.
- IVADo treatment was associated with improved overall survival compared to historical controls (HR 0.65).
- Distant metastasis at diagnosis significantly worsened event-free and overall survival (HRs 4.23 and 4.69, respectively).
Conclusions:
- The IVADo regimen demonstrates comparable or improved outcomes for children with advanced PPB.
- The poor outcomes in metastatic cases highlight the urgent need for novel therapeutic approaches.
- This study establishes a benchmark for future research in advanced pediatric PPB.
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/DICER1 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.
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