International PPB/DICER1 Registry
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- 100
Summary
- Conditions
- Nasal Chondromesenchymal Hamartoma
- Ciliary Body Medulloepithelioma
- Cystic Nephroma
- DICER1 Syndrome
- Embryonal Rhabdomyosarcoma
- Sertoli-Leydig Cell Tumor
- Neuroblastoma
- Nodular Hyperplasia of Thyroid
- Pineoblastoma
- Wilm's Tumor
- Pituitary Cancer
- Pleuropulmonary Blastoma
- Renal Sarcoma
- Type
- Observational
- Design
- Observational Model: CohortTime Perspective: Other
Participation Requirements
- Age
- Younger than 125 years
- Gender
- Both males and females
Description
PPB is a rare cancer of the lung presenting in early childhood, mostly commonly from birth to age ~72 months. PPB occurs within the lung or between the lung and the chest wall. There are three primary forms of PPB called Types I, II, and III PPB. PPB is related to an underlying change/mutation in a ...
PPB is a rare cancer of the lung presenting in early childhood, mostly commonly from birth to age ~72 months. PPB occurs within the lung or between the lung and the chest wall. There are three primary forms of PPB called Types I, II, and III PPB. PPB is related to an underlying change/mutation in a gene called DICER1 which impacts gene expression and cell growth. DICER1 mutations may also lead to the development of other tumors in children and adults. The International PPB/DICER1 Registry offers information based on previous data from Registry participants and the medical literature and collaborative efforts with international rare tumor groups. Retrospective and real-time central pathology review is encouraged. Therapy decisions remain at the discretion of the treating institution. Children with Type I PPB require surgery and sometimes chemotherapy. Therapy decisions are the responsibility of the treating institution. Surgical guidelines are presented. It is unknown whether adjuvant chemotherapy improves cure rates for Type I PPB patients. Chemotherapy options include a 22-week regimen: 4 courses of vincristine, actinomycin D and cyclophosphamide (VAC) followed by 3 courses of vincristine and actinomycin D (VA). Children with Types II and III PPB, require surgery, chemotherapy and sometimes radiation therapy. Many children with Types II or III PPB receive a single-arm multi-agent chemotherapy neo-adjuvant/adjuvant regimen of IVADo (ifosfamide, vincristine, actinomycin, doxorubicin) for 36 weeks. Second and possible 3rd look surgery may be considered for local control. Radiation therapy may be considered.
Tracking Information
- NCT #
- NCT03382158
- Collaborators
- Children's National Research Institute
- Washington University School of Medicine
- ResourcePath, LLC
- Beijing Children's Hospital
- University of Cambridge
- Emory University
- Dana-Farber Cancer Institute
- Investigators
- Principal Investigator: Kris Ann P Schultz, MD Children's Minnesota