Screening for Asymptomatic Portal Vein Thrombosis and Portal Hypertension in Patients With Philadelphia Negative Myeloproliferative Neoplasms

NCT01816256 | Completed

• 1 other identifier: MPN 12-01
• Study Type: interventional
• Target: 102
• Locations: 1 country
• Sites: 2

Brief Summary

This study involves screening for portal vein thrombosis and portal hypertension in patients with Philadelphia negative myeloproliferative neoplasms (MPNs). These include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis. Portal vein thrombosis and portal hypertension are serious complications that are often seen in myeloproliferative patients. These complications are usually diagnosed when patients become symptomatic, and are often already at an advanced stage. They can further progress to cause non-reversible damage to the liver, also called cirrhosis of the liver. As a result of this, patients often accumulate fluid in the abdomen which is ascites; and can develop swelling of veins in the lining of the esophagus known as varices. If untreated, varices have the risk of rupturing resulting in life-threatening bleeding. When diagnosed at an advanced stage, the treatment is usually supportive therapy and there are no treatments available at present which can reverse these conditions. This study is looking at screening for these two conditions using Doppler ultrasound and upper gastrointestinal endoscopy.

Conditions

Myeloproliferative Neoplasms (MPN); Polycythemia Vera (PV); Essential Thrombocythemia (ET); Myelofibrosis (MF)

Outcome Measures

Primary Outcomes (1)

• Incidence of asymptomatic portal vein thrombosis (PVT) and/or portal hypertension (PHTN) in patients with Philadelphia negative myeloproliferative neoplasms (MPNs)

  6 years

Secondary Outcomes (2)

• Identify the patient, disease and treatment related risk factors associated with PVT and PHTN

  6 years

• Comparison of survival and number of patients with leukemic transformation in patients with or without asymptomatic PVT/PHTN

  8 years

Other Outcomes (1)

• Identify any genetic markers or biomarkers associated with asymptomatic PVT and PHTN

  6 years

Study Arms (1)

Screening tests

OTHER

This is a one arm study. All patients will receive two screening tests (Doppler ultrasound, upper gastrointestinal endoscopy).

Procedure: Upper gastrointestinal endoscopy and Doppler ultrasound

Interventions

Upper gastrointestinal endoscopy and Doppler ultrasound PROCEDURE

This is a one arm study. All patients will receive two screening tests (Doppler ultrasound, upper gastrointestinal endoscopy).

Screening tests

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• One of the three classical Philadelphia negative myeloproliferative neoplasms (polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF)) diagnosed according to WHO or International working group-Myelofibrosis research and treatment (IWG-MRT) criteria
• Palpable spleen length greater than 5 cm below the costal margin in MF (including primary MF or post-polycythemia vera MF (PPV-MF) post-polycythemia vera ET (PPV-ET)) or palpable spleen of any size in patients with PV or ET.
• Ability to understand and willing to sign a written consent form.
• Age 18 years or older at time of consent.

You may not qualify if:

• Known history of portal vein thrombosis
• Known history of Budd-chairi syndrome
• Known history of oesophageal varices
• Known history of cirrhosis from any cause
• Known history of active bleeding

Sponsors & Collaborators

• University Health Network, Toronto: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (2)

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

Location

MeSH Terms

Conditions

Myeloproliferative Disorders; Polycythemia Vera; Thrombocythemia, Essential; Primary Myelofibrosis

Interventions

Endoscopy, Digestive System

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Blood Coagulation Disorders; Thrombocytosis; Blood Platelet Disorders; Hemorrhagic Disorders

Intervention Hierarchy (Ancestors)

Diagnostic Techniques, Digestive System; Diagnostic Techniques and Procedures; Diagnosis; Endoscopy; Diagnostic Techniques, Surgical; Digestive System Surgical Procedures; Surgical Procedures, Operative; Minimally Invasive Surgical Procedures

Study Officials

• Vikas Gupta, MD, FRCP

  The Princess Margaret Cancer Foundation

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SCREENING
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 14, 2013
• First Posted: March 22, 2013
• Study Start: May 1, 2013
• Primary Completion: March 11, 2019
• Study Completion: March 11, 2019
• Last Updated: July 21, 2023

Record last verified: 2023-07

Locations

CA (2)