Clinical trial NCT02380378: Registry of Philadelphia-Negative Myeloproliferative Neoplasms
Official title: Registry of Philadelphia-Negative Myeloproliferative Neoplasms
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Hematologic Diseases
Lead sponsor: Hospital Israelita Albert Einstein
Sponsor class: OTHER
Collaborators: name: Hospital Santa Marcelina
name: Instituto Nacional de Cancer, Brazil
name: Federal University of Minas Gerais
name: Irmandade da Santa Casa de Misericordia de Sao Paulo
name: Federal University of São Paulo
First posted: 2015-03-05
Enrollment: 500
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients who are followed in hematological centers with confirmed diagnosis of Myeloproliferative Neoplasms since 2000.

Brief summary: 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Registry of Philadelphia-Negative Myeloproliferative Neoplasms
timeFrame: 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Sao Paulo
country: Brazil
facility: Hospital Israelita Albert Einstein
state: SP
Study officials:
affiliation: Instituto Israelita de Ensino e Pesquisa Albert Einstein´s (IIEP)
name: Fabio P Santos
role: PRINCIPAL_INVESTIGATOR
References: