Registry of Philadelphia-Negative Myeloproliferative Neoplasms (MPN)

This study was developed to document current diagnosis and treatment patterns, clinical outcomes, and health care resource use associated with Philadelphia-Negative Myeloproliferative Neoplasms, in the different risk classifications for each disease.

Study Overview

• Status: Recruiting
• Conditions: Hematologic Diseases

Detailed Description

Philadelphia-negative (Ph-negative) Myeloproliferative Neoplasms (MPN) include polycythemia vera (PV), essential thrombocythemia (TE), and primary myelofibrosis (MF). Ph-negative MPN are chronic myeloid neoplasms that appear after a malignant transformation of a hematopoietic stem cell. There are many clinical manifestations of these diseases, which include polyglobulia, thrombocytosis, leukocytosis, cytopenias, extramedullary hematopoiesis (e.g.: splenomegaly), increased thrombotic risk and the risk of transformation to acute myeloid leukemia (AML). Life expectancy varies from 5-6 years for patients with MF to more than 15 years for patients with PV and ET.

There are few therapeutic options for patients with Ph-negative MPN. In general, the treatment of these diseases is symptomatic and targeted to the relief of symptoms and control of hematological setting using oral chemotherapy agents such as hydroxyurea. Patients with MF receive palliative treatment, targeted to the relief of splenomegaly and cytopenias. Currently, there are no approved drugs for the treatment of MF.

In Latin America, there are no epidemiological studies with a great number of Ph-negative MPN patients describing the natural history of the disease, patient progress, clinical characteristics, standards of care and disease burden. The development of an up-to-date data registry of Brazilian patients with Ph-negative MPN allows the comprehension of the epidemiology of this disease, with determination of survival, thrombosis incidence, transformation to AML, use of disease treatment-related resources, as well as the impact of different therapeutic strategies in these parameters. Therefore, the objective of this study is to establish a registry of patients with Ph-negative MPN in order to document the disease diagnosis and current standards of care, clinical endpoints, and the use of treatment-related resources, according to risk classification in each MPN.

Data will be retrospectively and prospectively collected through an electronic form available at an Internet safe website. Each center participating in the study will have a principal investigator who will be in charge of the accuracy and quality of the data collected. Each principal investigator and staff will receive a login and a password to access the database and enroll patients in the registry. An employee from each site will be in charge of collecting the data and inserting them in the electronic registry. Patients shall be contacted in order to sign an informed consent form. Patient data will be updated every six months. There is no limit for the number of patients to be enrolled in the registry, either retrospectively or prospectively. The duration of this study is five years. Data collection will continue until all sites decide to interrupt the enrollment of new patients.

• Study Type: Observational
• Enrollment (Anticipated): 500

Contacts and Locations

• Study Contact: Name: Fabio P Santos; Email: fabiopss@gmail.com
• Study Contact Backup: Name: Tarcila S Datoguia; Email: tarcila.datoguia@einstein.br

Study Locations

• Brazil
  • SP
    • Sao Paulo, SP, Brazil, 05651901
      • Recruiting
      • Hospital Israelita Albert Einstein
      • Contact: Fabio P S Santos, MD; Phone Number: +551121511128; Email: santos.fabio2@einstein.br
      • Contact: Tarcila Datoguia, MD; Phone Number: +551121511128; Email: tarcila.datoguia@einstein.br

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients who are followed in hematological centers with confirmed diagnosis of Myeloproliferative Neoplasms since 2000.

Description

Inclusion Criteria:

• diagnosis of (according to WHO 2008 criteria) Polycythemia Vera, Essential Thrombocythemia, Primary Myelofibrosis or Post-Polycythemia Vera/Post-Essential Thrombocythemia Myelofibrosis, Unclassifiable Myeloproliferative Neoplasm, Chronic Neutrophilic Leukemia, Chronic Eosinophilic Leukemia, Systemic Mastocytosis
• diagnosis made since 2000
• patients who are being followed-up or not
• signed informed consent form (for alive patients included in the prospective step of the study).

Exclusion Criteria:

• diagnosis of Chronic Myelogenous Leukemia
• refusal to sign the informed consent form (for patients who are alive and will de included in the prospective step of the study.

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Myeloproliferative Neoplasms; Patients diagnosed with Myeloproliferative Neoplasms based on WHO 2008 criteria.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Registry of Philadelphia-Negative Myeloproliferative Neoplasms	5 years

Collaborators and Investigators

• Sponsor: Hospital Israelita Albert Einstein
• Collaborators: Federal University of Minas Gerais; Federal University of São Paulo; Hospital Santa Marcelina; Instituto Nacional de Cancer, Brazil; Irmandade da Santa Casa de Misericordia de Sao Paulo
• Investigators: Principal Investigator: Fabio P Santos, Instituto Israelita de Ensino e Pesquisa Albert Einstein´s (IIEP)

Study record dates

Study Major Dates

• Study Start: March 1, 2015
• Primary Completion (Anticipated): December 1, 2022
• Study Completion (Anticipated): April 1, 2025

Study Registration Dates

• First Submitted: March 2, 2015
• First Submitted That Met QC Criteria: March 2, 2015
• First Posted (Estimate): March 5, 2015

Study Record Updates

• Last Update Posted (Actual): April 2, 2019
• Last Update Submitted That Met QC Criteria: April 1, 2019
• Last Verified: April 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Neoplasms; Hematologic Diseases; Myeloproliferative Disorders
• Other Study ID Numbers: RMPN2015