Clinical trial NCT05421104: Ruxolitinib for Polycythemia Vera in Patients Resistant to or Intolerant of Hydroxyurea.
Official title: Ruxolitinib for the Treatment of Polycythemia Vera in Patients Who Are Resistant to or Intolerant of Hydroxyurea: a Retrospective Non-interventional Study Using the US Optum Electronic Health Record Data Source.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Polycythemia Vera (PV)
Keywords: Polycythemia vera,
Ruxolitinib,
JAK protein inhibitor,
Hydroxyurea,
Thrombotic events
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2022-06-16
Enrollment: 1576
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: PV patients who were resistant to or intolerant of HU in the Optum EHR database between April 2007 and June 2019

Brief summary: This was an analytical and descriptive, non-interventional, retrospective cohort study of PV patients aged ≥ 18 years in the US using a secondary data source, Optum EHR database.

Detailed description: The Optum EHR database was current up to 30-Jun-2020.

Identification period: From 01-Apr-2007 to 30-Jun-2019

Study period: From 01-Jan-2007 to 30-Jun-2020

Index date:

First evidence of resistance to or intolerance of HU treatment in patients with PV according to modified European Leukemia Net (ELN) criteria and defined as:

1. HCT ≥ 45% with phlebotomy (last phlebotomy within last 3 months) Or
2. Platelet count \> 400 x 109/L and presence of palpable splenomegaly (palpable spleen up to 3 months after platelet count)

Pre-index period:

Patients had a minimum of 3 months pre-index data available. Pre-index data availability was determined using the reported 'first month active' field.

Post-index period:

There was no minimum post-index period required. Each patient had a 'first month active' and 'last month active' reported within the database. As the 'last month active' was based on any activity in the database, including encounters such as letters and emails which occurred several months after the 'death_date' of the patient, using the 'last month active' can overestimate the follow-up for a given patient. For this reason, the end of follow-up for each patient was defined as the date of the last activity within the diagnosis, observations, prescriptions, laboratories, procedures tables or discharge date from the last visit within the visit table (whichever of these activities occurs latest). This underestimated the follow-up for some patients where they were not actively using healthcare resources.

Eligibility criteria: Inclusion Criteria:

Included patients:

* With at least one International Classification of Diseases, 9th Revision, Clinical Modification/International Classification of Diseases,10th Revision, Clinical Modification code for PV in the identification period (01-Apr-2007 until 30-Jun-2019) that had non-missing sex and year of birth data and who were treated as part of the Integrated Delivery Network
* That were ≥ 18 years old at PV diagnosis
* With ≥ 2 prescriptions of HU
* That were classified as resistant to or intolerant of HU after a minimum of 3 months HU treatment (index date), defined as:

HCT ≥ 45% with phlebotomy (last phlebotomy within last 3 months) or Platelet count \> 400 x 109/L and presence of palpable splenomegaly (palpable spleen up to 3 months after platelet count).

To identify patients in the RUX group:

- With ≥ 2 prescriptions of RUX in the post-index period.

Exclusion Criteria:

Excluded patients:

- With a MF or AML diagnosis prior to a PV diagnosis.

Primary outcomes:
measure: Number of Thromboembolic events between the RUX and BAT group
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Number of Thromboembolic events between the high and low risk subgroups of BAT group
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Secondary outcomes:
measure: Incidence rate of thromboembolic event
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Time to first thromboembolic event
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Incidence rate of phlebotomy procedures
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Time to first phlebotomy procedure
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Incidence rate of neoplasm transformations
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Time to first neoplasm transformation
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Treatment patterns: Proportion of patients using different PV-related treatments
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Healthcare resource utilization (HCRU): Number of inpatient hospitalizations
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Healthcare resource utilization (HCRU): Number of outpatient visits
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
measure: Healthcare resource utilization (HCRU): Number of emergency room visits
timeFrame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Other outcomes:
Locations:
city: East Hanover
country: United States
facility: Novartis Investigative Site
state: New Jersey
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: