Surgery + Chemotherapy for Pleuropulmonary Blastoma

This trial aims to determine the effectiveness of combining surgery with chemotherapy compared to surgery alone in treating pleuropulmonary blastoma (PPB), a rare childhood lung cancer. It will also assess whether adding topotecan, a chemotherapy drug, to the standard chemotherapy enhances treatment effectiveness for more severe types of PPB. Children with newly diagnosed PPB, especially those with a large, solid mass in the chest, may be suitable for this trial if they have not received prior chemotherapy for PPB. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

The trial information does not specify whether you need to stop taking your current medications. However, if you are HIV-infected, you must not be on anti-retroviral agents that strongly affect the enzyme CYP3A4. It's best to discuss your current medications with the trial team.

Research has shown that the chemotherapy treatment for pleuropulmonary blastoma (PPB) in children, which includes vincristine, dactinomycin, and cyclophosphamide, has achieved some success. Although the exact safety details remain unclear, these drugs are commonly used in similar contexts. Another treatment option, IVADo, which includes ifosfamide, vincristine, dactinomycin, and doxorubicin, has demonstrated results as good as or better than previous treatments for types II and III PPB, suggesting these treatments are generally well-tolerated.

Studies indicate that adding topotecan to the treatment plan may help shrink the cancer and reduce the chance of it spreading. While topotecan is used in other cancer treatments, its safety in this specific context is still under investigation, though it is believed to have manageable side effects.

Most treatments for pleuropulmonary blastoma involve surgery and chemotherapy with drugs like vincristine, dactinomycin, and cyclophosphamide. But researchers are exploring something new and exciting with the addition of topotecan. Unlike the standard chemotherapy, topotecan works by blocking a specific enzyme needed for cancer cells to replicate their DNA, potentially leading to more effective cancer cell death. This unique mechanism could make it a powerful addition to current treatments, offering hope for better outcomes in patients.

This trial will compare different treatment approaches for pleuropulmonary blastoma (PPB). Studies have shown that surgery, either alone or with chemotherapy, can effectively treat PPB. For more advanced types of PPB, known as type II and III, a specific chemotherapy plan called the IVADo regimen—which includes ifosfamide, vincristine, dactinomycin, and doxorubicin—has delivered similar or better results compared to older treatments. In this trial, some participants will receive the IVADo regimen, and adding topotecan might enhance its effectiveness by targeting the cancer cell's DNA, potentially shrinking the tumor more effectively. For type I PPB, the situation is less clear, as some patients do well with just surgery and no chemotherapy. Overall, combining surgery and chemotherapy has shown promise, especially for the more severe types of PPB.⁵⁶⁷⁸⁹