Type I and Ir pleuropulmonary blastoma (PPB): A report from the International PPB/DICER1 Registry

Abstract

Background: Pleuropulmonary 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. Methods: Children with suspected PPB were enrolled in the International PPB/ DICER1 Registry. Pathology was centrally reviewed, and follow-up was ascertained annually. Results: Between 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 600 -Cancer.

Anaplastic sarcoma of the kidney (DICER1‐sarcoma of the kidney): A report from the International Pleuropulmonary Blastoma/DICER1 Registry

“…8 This apparent progression has been documented clinically in a cyst identified in a young child with type I PPB that was followed by ultrasound as it progressed over the span of 8 years to a growing multiloculated cyst and then ASK (Case 28). 10 Like cystic PPB (type I), sarcomatous progression of CN appears later than CN alone, as the median age of CN at diagnosis is 1.5 years 33 (near the median age of cystic PPB at 0.6 years 34 ). We found that the median age at diagnosis of ASK was 9 years, whereas the median ages at diagnosis for cystic-solid (PPB type II) and solid PPB (type III) are 35 and 39 months, respectively.…”

“…Like cystic PPB (type I), sarcomatous progression of CN appears later than CN alone, as the median age of CN at diagnosis is 1.5 years 33 (near the median age of cystic PPB at 0.6 years 34 ). We found that the median age at diagnosis of ASK was 9 years, whereas the median ages at diagnosis for cystic‐solid (PPB type II) and solid PPB (type III) are 35 and 39 months, respectively 35 .…”

Children's Oncology Group's 2023 blueprint for research: Rare tumors

“…Clinical outcomes in children with type I, Ir, II, and III PPB have recently been described 48,49 . A newly proposed PPB trial, ARAR2331, aims to test the addition of camptothecins and maintenance chemotherapy to the treatment of types II and III PPB with the goal of optimizing tumor shrinkage and reducing the risk for local and CNS recurrence and metastatic disease while standardizing the approach to types I and Ir PPB.…”

Assessing the role of positron emission tomography and bone scintigraphy in imaging of pleuropulmonary blastoma (PPB): A report from the International PPB/DICER1 Registry

“…As type II and III PPB have a propensity to metastasize to bone, 1 technetium 99 m‐methylenedisphosphonate skeletal scintigraphy scan (bone scan) has been utilized as part of the staging of advanced (type II and III) PPB. In contrast, type I PPB has not been shown to present with metastatic disease and given its cystic nature, 2 bone scans have not been recommended.…”