This is a prospective observational study that will enroll patients with high-risk
Polycythemia Vera (PV) with at least one Thromboembolic Event (TE) after diagnosis or up
to 2 years prior to diagnosis.

This is a non-randomized study, and to ensure a sufficient number of patients in both
cohorts, enrollment in each cohort will be terminated once the target of 150 patients has
been reached.

Study to Identify and Describe Predictive Factors for Thromboembolic Events in Patients With High-risk Polycythemia Vera

Polycythemia Vera

All patients are already on treatment with hydroxyurea or ruxolitinib at enrollment as
per clinical practice and independently of their participation in this study. In
addition, the follow-up visits and the evaluation procedures required in the study
protocol correspond to current clinical practice. According to local regulations related
to observational studies, assessments such as blood tests are justified by the purpose
and rationale of the study (i.e., the identification of possible predictive factors of
TEs) and are considered current clinical practice. Data related to other procedures will
be collected only if such procedures are performed as per clinical practice but are not
required otherwise.

Patients in both cohorts will be followed for 3 years after enrollment and will have
visits at Months 6, 12, 18, 24, 30 and 36. A time window of ± 1 month is permitted for
all visits.

Hydroxyurea

Ruxolitinib

Prospective Observational Study to Identify and Describe Predictive Factors for Thromboembolic Events in Patients With High-risk Polycythemia Vera

Demographics, blood pressure, laboratory blood tests, and use of antiplatelets and/or
anticoagulants are predictors of TE occurrence, a univariate Cox regression model for
repeated events will be applied for each predictor considering all the TEs occurring
within 12 months and the time from baseline to each occurrence. Predictors found
statistically significant at the 5% level will then be considered in a multivariate Cox
regression model for repeated events.

Number of patients with abnormal Body Mass Index (BMI) will be collected

Number of patients with abnormal weight will be collected

Number of patients with abnormal Neutrophil (NEP) count will be collected

Number of patients with abnormal White blood Count (WBC) will be collected

Number of patients with abnormal Lymphocytes (LYP) count will be collected

Number of participants using antiplatelets and/or anticoagulants on the incidence of
Thromboembolic Events (TEs) will be collected

Synergistic combinations of predictive factors (Red cell Distribution Width, neutrophils,
lymphocytes, neutrophil/ lymphocyte ratio and platelets).

Synergy score is defined as the product of the individual significances of variable 1 and
2 (expected) divided by the significance of the two-variable model (observed) To
investigate cases of extreme synergy, instances in which two variables split the given
cohort into high-risk and low-risk patients are far better than either variable alone. A
simple synergy scoring metric to rank variable in terms of synergy (S):S12=(P1*P2)/P12
where P1 and P2 are the maximum possible (Cox regression derived) p-values for variable 1
and variable 2 and P12 was the max. p-value possible from the combination of variables 1
and 1. This synergy is intended to capture a variable that may provide exclusive
non-redundant information when attempting to split a cohort based on risk and may provide
an added insight into the functional/clinical rationale of a model

Yearly incidence of Thromboembolic Events (TEs) will be collected

Incidence of arterial and venous TEs in the hydroxyurea and ruxolitinib cohorts will be
collected

Incidence and severity of adverse events according to National Cancer Institute Common
Terminology Criteria for Adverse Events (NCI CTCAE) v5.0.

Patients being treated with hydroxyurea at enrollment and for at least 18 months prior to
enrollment. Patients may switch to ruxolitinib treatment during the study in case of
inadequate response or intolerance.

Patients on treatment with ruxolitinib who started treatment up to 18 months prior to
enrollment.

Study to Identify and Describe Predictive Factors for Thromboembolic Events in Patients With High-risk Polycythemia Vera

About this trial

Study ID

Status

Type

Placebo

Accepting

Not accepting

Trial Timing

What is this trial about?

What are the participation requirements?

Locations

Location

Status