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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.

Official Title

International Pleuropulmonary Blastoma Registry for PPB, DICER1 and Associated Conditions


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 Registry offers information based on previous data from Registry participants and the medical literature and collaborative efforts with international rare tumor groups.

Retrospective central pathology review is required. 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.


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 PPB DICER1 Neoplasms Hyperplasia Rhabdomyosarcoma Pinealoma Pulmonary Blastoma Neuroectodermal Tumors, Primitive Hamartoma Rhabdomyosarcoma, Embryonal Leydig Cell Tumor Goiter, Nodular Pituitary Neoplasms Type I PPB Types II and III PPB Type Ir PPB


You can join if…

  1. Pathology Diagnosis: Individuals with newly-diagnosed PPB Types I, II or III. Diagnosis is made by the local pathologist.

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 resection(s) following chemotherapy, is diagnostic of PPB.

  1. Prior Therapy: PPB Type I: All patients are eligible and will be followed in the study.
  2. PPB Types II or III: Newly-diagnosed Types II and III PPB patients will be included in the Treatment and Biology Registry.
  3. Prior corticosteroid therapy is allowed.
  4. Types II and III PPB patients with PRIOR Type I PPB diagnosis
  5. Individuals with DICER1-related conditions including PPB, Sertoli-Leydig cell tumor, gynandroblastoma, cystic nephroma, renal sarcoma and others.
  6. Individuals with the DICER1 gene mutation regardless of whether they have a known DICER1-associated condition
  7. Informed consent by patient/ or parent/guardian (also, where appropriate: assent and HIPAA consent)

You CAN'T join if...


  • Children;s Minnesota accepting new patients
    Minneapolis Minnesota 55404 United States


accepting new patients
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Children's Hospitals and Clinics of Minnesota
International Pleuropulmonary Blastoma (PPB) Registry web site
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