1. Type I and Ir pleuropulmonary blastoma (PPB): A report from the International PPB/DICER1 Registry
- Author
-
Nelson, Alexander T, Harris, Anne K, Watson, Dave, Miniati, Doug, Finch, Mike, Kamihara, Junne, Mitchell, Sarah G, Wilson, David B, Gettinger, Katie, Rangaswami, Arun A, Campos, José M, Lederman, Sara, Feltis, Brad A, Vasta, Lauren M, Harney, Laura A, Stewart, Douglas R, Dehner, Louis P, Messinger, Yoav H, Hill, D Ashley, and Schultz, Kris Ann P
- Subjects
Pediatric ,Cancer ,Pediatric Research Initiative ,Pediatric Cancer ,Lung ,Rare Diseases ,Lung Cancer ,Child ,Humans ,Child ,Preschool ,Pulmonary Blastoma ,Lung Neoplasms ,Registries ,Ribonuclease III ,Drug-Related Side Effects and Adverse Reactions ,DEAD-box RNA Helicases ,DICER1 ,DICER1 tumor predisposition ,lung cyst ,pleuropulmonary blastoma ,type I PPB ,type Ir PPB ,Oncology and Carcinogenesis ,Public Health and Health Services ,Oncology & Carcinogenesis - Abstract
BackgroundPleuropulmonary blastoma (PPB) is the most common lung cancer of infancy and early childhood. Type I PPB is a purely cystic lesion that has a microscopic population of primitive small cells with or without rhabdomyoblastic features and may progress to type II or III PPB, whereas type Ir lacks primitive small cells.MethodsChildren with suspected PPB were enrolled in the International PPB/DICER1 Registry. Pathology was centrally reviewed, and follow-up was ascertained annually.ResultsBetween 2006 and 2022, 205 children had centrally reviewed type I or Ir PPB; 39% of children with type I and 5% of children with type Ir PPB received chemotherapy. Outcomes were favorable, although 11 children (nine with type I and two with type Ir PPB) experienced progression to type II/III (n = 8) or regrowth of type I PPB at the surgical site (n = 3), none of whom received chemotherapy before progression. Age and cyst size in combination were more suitable than either factor alone in predicting whether a particular lesion was type I or Ir PPB.ConclusionsFor young children with type I PPB, outcomes are favorable, but complete resection is indicated because of the risk for progression. Chemotherapy may be useful in a subset of children at increased risk for recurrence/progression. Efforts to risk stratify children with type I PPB to optimize outcomes while reducing treatment-related side effects are underway.
- Published
- 2023