@article{e4541fe4f73d4626a61907220b18d4e1,
title = "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 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. Conclusions: For 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.",
keywords = "DICER1, DICER1 tumor predisposition, lung cyst, pleuropulmonary blastoma, type I PPB, type Ir PPB",
author = "Nelson, {Alexander T.} and Harris, {Anne K.} and Dave Watson and Doug Miniati and Mike Finch and Junne Kamihara and Mitchell, {Sarah G.} and Wilson, {David B.} and Katie Gettinger and Rangaswami, {Arun A.} and Campos, {Jos{\'e} M.} and Sara Lederman and Feltis, {Brad A.} and Vasta, {Lauren M.} and Harney, {Laura A.} and Stewart, {Douglas R.} and Dehner, {Louis P.} and Messinger, {Yoav H.} and Hill, {D. Ashley} and Schultz, {Kris Ann P.}",
note = "Funding Information: The authors thank the many treating physicians, genetic counselors, patients, and families who collaboratively support the International PPB/ Registry and the Pine Tree Apple Classic Fund, whose volunteers, tennis players, and donors have provided more than 30 years of continuous support for pleuropulmonary blastoma (PPB) research. The authors also thank Jason Albrecht, whose many contributions continue to inspire PPB research. The International PPB/ Registry is generously supported by the philanthropic efforts of the Pine Tree Apple Classic Fund. The International PPB/ Registry is also supported by the Children's Minnesota Foundation and Rein in Sarcoma. This analysis was supported by a grant from the Children's Minnesota Internal Research Grant Program. Research reported in this publication was supported by the National Cancer Institute of the National Institutes of Health under Award Numbers R44CA228850 and 1R37CA244940. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. This work was also supported by the Intramural Research Program of the Division of Cancer Epidemiology and Genetics of the National Cancer Institute, Rockville, Maryland. DICER1 DICER1 DICER1 Funding Information: Douglas R. Stewart provides telegenetics services for Genome Medical, Inc., in accordance with relevant National Cancer Institute policies. Dave Watson reports personal fees from Allina Health outside the submitted work. D. Ashley Hill is owner of ResourcePath LLC, a company that does research and development of laboratory tests, including for cancers; that work is unrelated to the information presented in this article. Junne Kamihara reports that her spouse receives research funding from ACD‐Biotechne; her spouse is an owner and founder of Rome Therapeutics, PanTher Therapeutics, and TellBio, Inc.; and her spouse receives personal fees from Tekla Capital, Ikena Oncology, and Rome Therapeutics outside the submitted work. The remaining authors made no disclosures. DICER1 Publisher Copyright: {\textcopyright} 2022 American Cancer Society.",
year = "2023",
month = feb,
day = "15",
doi = "10.1002/cncr.34593",
language = "English",
volume = "129",
pages = "600--613",
journal = "Cancer",
issn = "0008-543X",
number = "4",
}