Testing a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma

This study is for children and young people aged 21 or younger who have recently been diagnosed with Pleuropulmonary Blastoma (PPB). You must have a new diagnosis of this specific lung cancer to be considered for the study.

Even if a diagnosis of PPB isn't fully confirmed by a biopsy (a sample of tissue), you might still be able to join if your doctors strongly suspect Type II or III PPB, especially if taking a biopsy isn't safe. For children with Type I PPB, you won't start chemotherapy until the diagnosis is fully confirmed by central experts. For Types II and III PPB, if you're very unwell, you might start treatment while waiting for the full pathology review.

You'll also need to meet certain health requirements, especially for your kidney function, which will be checked with blood and/or urine tests before you start. The medical team will review all these details with you to see if the study is a good fit.

If you join this study, your child will undergo various medical assessments. These include regular imaging scans such as CT, MRI, PET, and ultrasound scans, as well as bone scans and special heart tests like echocardiograms and Multigated Acquisition Scans to check heart function. Blood and tissue samples will also be collected at different stages of the treatment. For all types of PPB, your child's tumour tissue will be carefully examined by expert doctors.

For Type I PPB, depending on whether the tumour was fully removed by surgery, your child may either be closely observed without chemotherapy, or they may receive a specific chemotherapy regimen involving drugs like vincristine, dactinomycin, and cyclophosphamide. For Types II and III PPB, the standard chemotherapy involves a combination of drugs (Ifosfamide, Vincristine, Dactinomycin, Doxorubicin). In this study, some children with Types II and III PPB will also receive an additional drug called Topotecan. The entire course of treatment and observation varies, but for chemotherapy it can last many weeks or months, followed by ongoing monitoring. The medical team will explain the specific schedule and duration for your child.