International Pleuropulmonary Blastoma/DICER1 Registry for PPB, DICER1 and Associated Conditions

What this Trial Is About

Pleuropulmonary blastoma (PPB) is a rare lung cancer that typically occurs in early childhood, often from birth to about 72 months. The disease has three main types: Type I (cystic), Type II (partially cystic and solid), and Type III (completely solid). This research also includes individuals with DICER1 gene mutations or related conditions to better understand how these tumors develop, their clinical course, and the best treatments. The study builds upon previous registry data and collaborates internationally. Treatment varies by PPB type and is determined by the treating institution. Children with Type I PPB may have surgery and sometimes chemotherapy, with options including a 22-week chemotherapy regimen. Types II and III PPB generally require surgery, chemotherapy over 36 weeks, and sometimes radiation therapy. The study also enrolls participants with Type Ir PPB and those with DICER1 mutations or associated conditions, regardless of age. Participants are followed through the International PPB/DICER1 Registry, which collects data on their health outcomes, treatment responses, and quality of life over time. The study tracks event-free survival over seven years and monitors overall survival, response to chemotherapy, heart and lung function, and the occurrence of tumors related to DICER1 conditions. Consent is required from participants or guardians, and treatment decisions remain with the treating doctors.

International PPB/DICER1 Registry