International PPB/DICER1 Registry

  • STATUS
    Recruiting
  • End date
    Dec 6, 2026
  • participants needed
    3400
  • sponsor
    Children's Hospitals and Clinics of Minnesota
Updated on 2 May 2021
cancer
initial diagnosis
solid tumor
sarcoma

Summary

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.

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 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.

Details
Condition Embryonal rhabdomyosarcoma, Pinealoma, Sertoli-Leydig Cell Tumor, Pituitary Disease, Malignant neoplasm of kidney, Ovarian Cancer, Hereditary Neoplastic Syndrome, Nephroblastoma, Malignant neoplasm of brain, Pituitary Adenoma, Neuroblastoma, Malignant neoplasm of testis, Brain Tumor (Pediatric), Pleuropulmonary Blastoma, Cystic Nephroma, Nasal Chondromesenchymal Hamartoma, DICER1 Syndrome, Recurrent Ovarian Cancer, Renal Sarcoma, Nodular Hyperplasia of Thyroid, Ciliary Body Medulloepithelioma, Kidney Cancer, Brain Cancer, Testicular Cancer, Pituitary Disorders, Wilms' Tumor, Brain Tumor, Hereditary Cancer Syndromes, Renal Cancer, neuroblastomas, pineoblastoma, pinealoblastoma
Clinical Study IdentifierNCT03382158
SponsorChildren's Hospitals and Clinics of Minnesota
Last Modified on2 May 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Gender: Male or Female
Do you have any of these conditions: Sertoli-Leydig Cell Tumor or Ciliary Body Medulloepithelioma or DICER1 Syndrome or Nephroblastoma or Pleuropulmonary Blastoma or Nasal Chondromesenchy...?
Do you have any of these conditions: Pinealoma or Embryonal rhabdomyosarcoma or Ciliary Body Medulloepithelioma or Brain Tumor or Brain Cancer or Cystic Nephroma or Malignant neoplasm of ...?
Do you have any of these conditions: Nodular Hyperplasia of Thyroid or Pituitary Adenoma or Malignant neoplasm of kidney or Pleuropulmonary Blastoma or Hereditary Neoplastic Syndrome or H...?
Do you have any of these conditions: Pituitary Adenoma or Nodular Hyperplasia of Thyroid or Ovarian Cancer or Hereditary Neoplastic Syndrome or Malignant neoplasm of testis or Pinealoma o...?
Known or suspected PPB or related thoracic tumor
Known or suspected sex-cord stromal tumor including Sertoli-Leydig cell tumor and gynandroblastoma (males or females)
Other known or suspected DICER1-related condition including ovarian sarcoma, cystic nephroma, renal sarcoma, pineoblastoma, pituitary blastoma, nasal chondromesenchymal hamartoma, ciliary body medulloepithelioma and others
Individuals with known or suspected DICER1 pathogenic variation regardless of whether they have an established DICER1-associated condition
Informed consent by patient/ or parent/guardian (also, where appropriate: assent and HIPAA consent)

Exclusion Criteria

Absence of appropriate consent for Registry participation
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