International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry

• Status: Active, not recruiting
• Trial end date: 2024-12-01
• Target enrollment: 0
• Participant gender: All

Summary

Pleuropulmonary Blastoma (PPB) is very rare and there is no established "standard" or "best" therapy. For many years, children with PPB around the world have been treated according to decisions made case-by-case in many different hospitals by many different physicians. No treatment has been tested in a large group of PPB patients.

The goal is to treat many children with one treatment program and to learn the results of the treatment.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospitals and Clinics of Minnesota
• Treatments: Cyclophosphamide; Dactinomycin; Doxorubicin; Ifosfamide; Isophosphamide mustard; Liposomal doxorubicin; Vincristine

Criteria

Inclusion Criteria:

1. Age : Patients from birth to < 21 years of age at the time of diagnosis will be included in the Treatment and Biology Registry.

Patients of any age will be included in the Associated Diseases arm of this study.

2. Pathology Diagnosis: Patients with newly-diagnosed PPB Types I, II or III. Diagnosis is made by the local pathologist. Real-time central pathology review is encouraged but is not required. All cases must be submitted for central pathology review. Only centrally-reviewed cases confirmed as PPB will be analyzed prospectively.

Cases in which the initial diagnosis is "suggestive" or "supportive" of PPB, but not diagnostic, and in which later resection specimens, including resections following chemotherapy, confirm a PPB diagnosis will be included. Patients diagnosed by fine needle aspiration biopsy will be included only if a later resection specimen, including resections following chemotherapy, is diagnostic of PPB.

Diagnostic pathology for cases of diseases associated with PPB will also require registry central pathology review.

3. Prior Therapy: PPB Type I: All patients are eligible and will be followed in the study.

PPB Types II or III: Newly-diagnosed Types II and III PPB patients will be included in the Treatment and Biology Registry.

DICER1-related condition and DICER1 gene mutation: all patients are eligible and will be followed in the study.

4. Prior corticosteroid therapy is allowed.

5. Patients who have received other chemotherapy regimens or radiation therapy will not be statistical analysis.

6. Types II and III PPB patients with PRIOR Type I PPB diagnosis: Types II and III PPB cases which are recurrences of an earlier Type I PPB are included.

7. Informed consent by patient or parent/guardian. (also, where appropriate: assent and HIPPA consent)

Exclusion Criteria:

1. Inability of patient, or parent/guardian to obtain informed consent.

2. Patients who have their PPB diagnosed ruled out by Registry central pathology review.