International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol
This Registry will collect family medical history and establishes a collection of specimens
from PPB patients and their parents for future research. Primary goals of the biology
research are (1) to identify new scientific ways to learn about PPB and the best way to
treat it in the future and (2) to establish a collection of PPB biologic specimens for
Background: PPB is a rare cancer of the lung presenting in early childhood, mostly from
birth to age ~72 months. PPB occurs within the lung or between the lung and the chest wall.
There are three forms of PPB called Types I, II, and III PPB. Children with Type I PPB
require surgery and sometimes chemotherapy. Children with Types II and III PPB, require
surgery, chemotherapy and sometimes radiation therapy. Because there has never been a
consensus as to the best treatment for PPB, the Registry will provide treatment guidelines.
It is the goal of the Treatment and Biology Registry that by offering specific regimens the
Registry will be able to prospectively review and analyze the regimen for the first time.
- Diagnostic central pathology review will be required for patients who will be
- This Treatment Registry will offer surgery and chemotherapy regimen guidelines, and
optional radiation therapy guidelines for prospectively enrolled patients.
- The Treatment and Biology Registry will create a PPB biologic specimens reference bank
- For Type I PPB, surgery is necessary. Some doctors use chemotherapy after surgery. If
chemotherapy is used the Registry will suggest that it be combination chemotherapy with
VAC (vincristine, dactinomycin, cyclophosphamide).
- For Types II and III PPB patients surgery is necessary, followed by chemotherapy. The
Registry will suggest that subjects receive combination chemotherapy with Ifosphamide,
Vincristine, Actinomycin-D and Doxorubicin ("IVADo"). Second look and possible 3rd look
surgery may be required. Radiation therapy is recommended only for residual disease
after maximum surgery.
Analysis: Statistical data analysis will include only centrally confirmed cases.
Additionally only patients with PPB Type II or Type III who were enrolled and treated with
IVADo as per the regimen will be compared to historical controls. No statistical analysis is
planned for patients with Type I PPB. Future treatment will be compared to the IVADo regimen
in order to measure improvements.
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
The primary endpoint for statistical analysis will be time from start treatment to an event, defined as the occurrence of progression or recurrence of PPB, occurrence of a second malignant neoplasm, or death from any cause that is at least possibly related to the original disease or treatment.
Yoav H Messinger, MD
Children's Hospitals and Clinics of Minensota
United States: Food and Drug Administration
PPB Reg Tx-Biol-001
|Children's Hospitals and Clinics of Minnesota||Saint Paul, Minnesota 55102|