About this study
Pleuropulmonary blastoma (PPB) is a rare cancer that starts in the lungs or the area around them. PPB is the most common cancerous lung tumor in infants and children. There are 4 types of PPB:
- Type 1: Made up of air pockets (cysts) that show early signs of cancer
- Type 1r: Similar to Type 1 but does not contain cancer cells. The "r" stands for "regressed," meaning the cysts may be shrinking or not growing into cancer.
- Type 2: More serious, with both cysts and solid cancer parts
- Type 3: Fully solid and more likely to spread to other parts of the body
This study is looking for the best way to treat PPB safely and effectively. Doctors want to find out if some children can avoid chemotherapy (chemo) and still have good outcomes. Chemo kills cancer cells but can cause strong side effects.
In this study, children with Type 1 or 1r PPB whose tumors were completely removed by surgery will be watched closely without chemo. If the tumor cannot be fully removed and the child is under age 5, they may get chemo. For Type 1r, which does not have cancer cells, children will be observed after surgery with no chemo.
Children with Type 2 or 3 PPB will usually have surgery to remove the tumor, followed by chemo with vincristine, topotecan, and cyclophosphamide; and ifosfamide, vincristine, and doxorubicin. Some of these children may also need radiation therapy. This part of the study will help doctors learn if current treatments work well or if they can be improved.
Doctors hope this study will show which children really need chemo and which ones may be able to avoid it. This could reduce side effects and help each child get the right treatment for their specific type of PPB. Right now, there is no single standard treatment plan, especially for Type 1 PPB.
Eligibility overview
- Newly diagnosed pleuropulmonary blastoma
- Up to 21 years old
- The tumor has been completely removed by surgery, or the patient is a candidate for surgical removal.
- No prior chemotherapy or radiation therapy for PPB