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

Abstract

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.

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

“…After initial staging, ongoing monitoring for pulmonary metastatic disease is recommended, as pulmonary metastases were detected in at least eight of 47 cases. Based on the improved sensitivity with 18 F‐fluorodeoxyglucose‐positron emission tomography (FDG‐PET) compared to bone scintigraphy in Ewing sarcoma 46 and the high sensitivity seen with FDG‐PET in PPB, 47 FDG‐PET should be considered for metastatic evaluation in all patients.…”

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

“…A systematic analysis of health-related quality of life in children with PPB is ongoing (Accessed December 14, 2022. www.PPBregistry.org). 27 Based on the incidence of surgery in children with cystic PPB This analysis suggests an increased risk for recurrence of type I PPB associated with less than R0 as the best resection; therefore, reresection or chemotherapy should be considered for children who achieve less than an R0 resection with initial surgery. The analysis also indicated an increased risk associated with known multifocality; however, we caution that multifocality was incompletely assessed in this retrospective analysis.…”