Children's Hospitals and Clinics of Minnesota
Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a partially cystic, partially solid tumor, Type III is a completely solid tumor. Treatment of children with PPB is at the discretion of the treating institution. This study builds off of the 2009 study and will also seek to enroll individuals with DICER1-associated conditions, some of whom may present only with the DICER1 gene mutation, which will help the Registry understand how these tumors and conditions develop, their clinical course and the most effective treatments.
Pleuropulmonary Blastoma
Sertoli-Leydig Cell Tumor
DICER1 Syndrome
Cystic Nephroma
Wilms Tumor
Pineoblastoma
Renal Sarcoma
Nodular Hyperplasia of Thyroid
Nasal Chondromesenchymal Hamartoma
Ciliary Body Medulloepithelioma
Neuroblastoma
Pituitary Cancer
Embryonal Rhabdomyosarcoma
Ovarian Sarcoma
Gynandroblastoma
Thyroid Carcinoma
Embryonal Rhabdomyosarcoma of Vagina (Diagnosis)
Embryonal Rhabdomyosarcoma of Uterus (Diagnosis)
Embryonal Rhabdomyosarcoma of Cervix
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.
| Study Type : | OBSERVATIONAL |
| Estimated Enrollment : | 3400 participants |
| Official Title : | International Pleuropulmonary Blastoma/DICER1 Registry (for PPB, DICER1 and Associated Conditions) |
| Actual Study Start Date : | 2016-12-06 |
| Estimated Primary Completion Date : | 2030-12-06 |
| Estimated Study Completion Date : | 2035-12-06 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | 0 Minutes to 100 Years |
| Sexes Eligible for Study: | ALL |
| Accepts Healthy Volunteers: | 1 |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
RECRUITING
Children's Minnesota
Minneapolis, Minnesota, United States, 55404