Symptoms and General Pathology

All Conditions

Respiratory Tract (Lung and Bronchial) Diseases

Neoplasms

Diseases and Abnormalities at or Before Birth

Rare Diseases

Syndrome

Pneumothorax

Cysts

Birt-Hogg-Dube Syndrome

Pleural Diseases

Respiratory Tract Diseases

Neoplastic Syndromes, Hereditary

Genetic Diseases, Inborn

Primary Spontaneous Pneumothorax

Patients who were treated for spontaneous pneumothorax in Rijnstate hospital. "Interventions": visit to out-patient clinic,CT scan of pulmones.

Patients who were treated for spontaneous pneumothorax in Rijnstate hospital. "Interventions": withdrawal of blood sample for DNA analyses,

A low dose CT scan of the thorax (2mSv) wil be performed once.

CT scan of the thorax

A venous punction in order to withdraw 16ml blood is undertaken once.

blood sample for FLCN mutation analyses

Jincey D. Sriram, MD, MSc

Hans JM Smit, MD,PhD

Hans JM Smit, MD. PhD

Jincey Sriram, MD

Pieter E. Postmus, MD, PhD

Hans JM Smit, MD, PhD

To assess the prevalence of BHD (Birt-Hogg-Dubé syndrome) among patients with spontaneous pneumothorax. Patients who were treated for primary spontaneous pneumothorax in Rijnstate hospital are to be included. Patients will receive a questionnaire. When given consent, the investigators will invite them for a one-time visit to the out-patient clinic. Patients will be asked for a blood sample to determine pathogenic FLCN (folliculin) mutations and a pulmonary CT scan for evaluation of presence of lung cysts.

Based on the results of the pilot study in VUmc \*Free University Medical Center), in which 3 out of 40 tested patients had a pathological FLCN mutation, the investigators decided to extend the study to a second center; Rijnstate Hospital Arnhem. In this hospital a retrospective search was performed; patients who were treated for primary spontaneous pneumothorax were included. In the dossiers the investigators searched for medical history, pneumothorax side and recurrence, diagnostic imaging, treatment, co-morbidity, complications of treatment, skin abnormalities, kidney disease, smoking behavior, medication, and familial incidence of pneumothorax and other diseases. Patients will receive a letter with explanation of the research and a questionnaire in which the investigators ask them their about the medical status, co-morbidity, pneumothorax (number and side), smoking behavior, use of drugs, familial incidence of pneumothorax and other diseases.

The population will be formed out of patients who have returned the fully filled in questionnaire and who have given permission to receive information for further research. This further information will consist of an information letter on BHD syndrome and a consent form for a one-time visit to the out-patient clinic of Rijnstate hospital. Investigators expect that about 200 patients will return the fully filled in questionnaire and give their consent for further research. In a one-time visit in out-patient clinic, there will be given personal information on BHD syndrome and there will be performed physical examination for finding fibrofolliculomas. A pulmonary CT scan for evaluation of presence of lung cysts will be performed. Two samples of venous blood will be collected to access information on DNA diagnostics for pathogenic FLCN mutations. These are associated with the BHD syndrome. This last diagnostic testing will be performed in VUmc (Vrije Universiteit medisch centrum or Free University Medical Centre).

Prevalence of Spontaneous Pneumothorax in BHD

Prevalence of Birt-Hogg-Dubé Syndrome Among Patients With (Hereditary) Spontaneous Pneumothorax

Prof. Dr. Jaap Swieringa stichting

Mr. Willem Bakhuys Roozeboomstichting

Rijnstate Hospital

Hans Smit

MD, PhD

Pulmonary Blastoma

Neoplasms by Histologic Type

Lung Neoplasms

Respiratory Tract Neoplasms

Thoracic Neoplasms

Pleuropulmonary Blastoma

Antineoplastic Agents

Antirheumatic Agents

All Drugs and Chemicals

Anti-Infective Agents

Cyclophosphamide

Doxorubicin

Liposomal doxorubicin

Vincristine

Ifosfamide

Dactinomycin

Isophosphamide mustard

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

Anti-Bacterial Agents

Antibiotics, Antitubercular

Enzyme Inhibitors

Tubulin Modulators

Antimitotic Agents

PPB Type I therapy: All patients will be treated with surgery. Chemotherapy after surgery is per the treating physician(s) discretion. If chemotherapy is used the Registry will suggest that it be combination chemotherapy with Vincristine, Dactinomycin, Cyclophosphamide (VAC).

Combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin and Doxorubicin ("IVADo"). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.

≥ 3 years: 1.5 mg/m2 IV x 1 (maximum dose 2 mg)

≥ 3 years: 0.045 mg/kg (maximum dose 2.5 mg) IV X 1

≥ 3 year: 1.2 gm/m2/dose IV as 1 hr infusion with IV fluids

≥ 3 years: 3 g/m2/dose IV over 3 hours on Days 1, 2, (6 g/m2/cycle)

≥ 3 years: 30 mg/m2/dose IV over 30 min, Days 1, 2 (60 mg/m2/cycle)

Kris Ann P Schultz, MD

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.

This Registry will collect family medical history and establishes a collection of specimens from PPB patients and their parents for future research. Primary goals of the biology research are (1) to identify new scientific ways to learn about PPB and the best way to treat it in the future and (2) to establish a collection of PPB biologic specimens for future research.

Background: PPB is a rare cancer of the lung presenting in early childhood, mostly from birth to age \~72 months. PPB occurs within the lung or between the lung and the chest wall. There are three forms of PPB called Types I, II, and III PPB. Children with Type I PPB require surgery and sometimes chemotherapy. Children with Types II and III PPB, require surgery, chemotherapy and sometimes radiation therapy. Because there has never been a consensus as to the best treatment for PPB, the Registry will provide treatment guidelines. It is the goal of the Treatment and Biology Registry that by offering specific regimens the Registry will be able to prospectively review and analyze the regimen for the first time.

Design:

* Diagnostic central pathology review will be required for patients who will be prospectively evaluated.
* This Treatment Registry will offer surgery and chemotherapy regimen guidelines, and optional radiation therapy guidelines for prospectively enrolled patients.
* The Treatment and Biology Registry will create a PPB biologic specimens reference bank for tumor.

Methods:

* For Type I PPB, surgery is necessary. Some doctors use chemotherapy after surgery. If chemotherapy is used the Registry will suggest that it be combination chemotherapy with vincristine, dactinomycin, cyclophosphamide (VAC ).
* For Types II and III PPB patients surgery is necessary, followed by chemotherapy. The Registry will suggest that subjects receive combination chemotherapy with Ifosphamide, Vincristine, Actinomycin-D and Doxorubicin (IVADo). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.

Analysis: Statistical data analysis will include only centrally confirmed cases. Additionally only patients with PPB Type II or Type III who were enrolled and treated with IVADo as per the regimen will be compared to historical controls. No statistical analysis is planned for patients with Type I PPB. Future treatment will be compared to the IVADo regimen in order to measure improvements.

International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry

International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol

The primary endpoint for statistical analysis will be time from start treatment to an event, defined as the occurrence of progression or recurrence of PPB, occurrence of a second malignant neoplasm, or death from any cause that is at least possibly related to the original disease or treatment.

Secondary endpoints will the best overall response to chemotherapy among patients with radiographically measurable tumor following initial surgery or biopsy, and time to death from any cause.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Prevalence of Spontaneous Pneumothorax in BHD	NCT02916992	Spontaneous Pne...	CT scan of the ... blood sample fo...	18 Years -	Free University Medical Center
International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry	NCT01464606	Pleuropulmonary...	Vincristine Dactinomycin Cyclophosphamid... Ifosfamide Doxorubicin	- 21 Years	Children's Hospitals and Clinics of Minnesota
International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry	NCT01464606	Pleuropulmonary...	Vincristine Dactinomycin Cyclophosphamid... Ifosfamide Doxorubicin	- 21 Years	Children's Hospitals and Clinics of Minnesota

Spots Global Cancer Trial Database for lung cysts

Every month we try and update this database with for lung cysts cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for lung cysts

Every month we try and update this database with for lung cysts cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for lung cysts cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.