Health-related quality of life in children and adolescents with pleuropulmonary blastoma: A report from the International PPB/DICER1 Registry

Alexander T. Nelson, Anna Dybvik, Paige Mallinger, Shelly Weiss McQuaid, Dave Watson, Laura A. Harney, Douglas R. Stewart, Louis P. Dehner, Yoav H. Messinger, Alicia S. Kunin-Batson, Kris Ann P. Schultz

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Purpose: Pleuropulmonary blastoma (PPB) is the most common lung cancer of infancy and early childhood and is associated with germline DICER1 variants. Type I and Ir PPB are cystic lesions treated surgically, with a subset of children with type I receiving chemotherapy. Type II and III are more aggressive lesions, treated with surgery, intensive chemotherapy and potentially radiation. We sought to assess health-related quality of life (HRQoL) in children with PPB and known germline DICER1 variants. Methods: Children with a diagnosis of PPB or germline DICER1 pathogenic variant without history of PPB or other DICER1-related neoplasm (DICER1+ only) were enrolled in the International PPB/DICER1 Registry. Parent reports for participants aged 2–17 years for the PedsQL v.4 and PedsQL Multidimensional Fatigue Scale v.3 were collected. Fatigue, physical, and psychosocial function scores were compared. Results: Analysis included 84 participants (PPB type Ir = 20, type I = 15, type II/III = 27, DICER1+ only = 22). Total fatigue scores of participants with type I and II/III PPB were lower compared to DICER1+ only, with effect size larger in type II/III (−0.82 vs. −0.40). Total psychosocial and physical functioning scores were lower in participants with type I and type II/III PPB compared to DICER1+ only, with larger effects noted in type II/III. Female sex was suggestive of worse HRQoL for both type I/Ir and type II/III cohorts. Conclusions: These data demonstrate the importance of regular HRQoL assessment in patients with a history of PPB as well as the importance and feasibility of studying HRQoL in children with rare tumors.

Original languageEnglish (US)
Article numbere30077
JournalPediatric Blood and Cancer
Issue number4
StatePublished - Apr 2023

Bibliographical note

Funding Information:
The authors wish to thank the many treating physicians, genetic counselors, patients, and families who collaboratively support the International PPB/ Registry, as well as the Pine Tree Apple Classic Fund whose volunteers, tennis players, and donors have provided more than 35 years of continuous support for PPB Research. The authors gratefully acknowledge the contributions of Jason Albrecht to ongoing PPB research initiatives. The International PPB/ Registry is also supported by the Children's Minnesota Foundation, Mendon F. Schutt Foundation, and Rein in Sarcoma. This analysis was supported by a grant from the Children's Minnesota Internal Research Grant Program and supported by funding from National Institute of Health National Cancer Institute grants 1R37CA244940‐01 and 2R01CA143167‐06A1, and the Intramural Research Program of the Division of Cancer Epidemiology and Genetics of the National Cancer Institute, Rockville, MD, USA. DICER1 DICER1

Publisher Copyright:
© 2022 Wiley Periodicals LLC.


  • DICER1
  • DICER1 tumor predisposition
  • Health-Related Quality of Life
  • PedsQL
  • Pleuropulmonary Blastoma

PubMed: MeSH publication types

  • Journal Article
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't


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