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Completed

NCT Number: NCT05421104

Ruxolitinib for Polycythemia Vera in Patients Resistant to or Intolerant of Hydroxyurea.

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.

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Key information

Conditions

Polycythemia Vera (PV) Bone Marrow Diseases Bone Marrow Neoplasms Hematologic Diseases Hematologic Neoplasms Hemic and Lymphatic Diseases Myeloproliferative Disorders Neoplasms Neoplasms by Site Polycythemia Vera

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis Investigative Site

East Hanover, New Jersey, 07936-1080, United States

About this study

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:

  • 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)

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Ruxolitinib

Other

PV patients who were resistant to or intolerant of HU (as defined on the index date) and switched to RUX in the post-index period.

Other names: Jakavi

Primary outcomes

  1. Number of Thromboembolic events between the RUX and BAT group

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Thromboembolic events in overall Polycythemia Vera cohort and in the BAT and RUX groups were reported. A TE was defined using International Classification of Diseases 9th Revision (ICD-9- CM) and International Classification of Diseases 10th Revision (ICD-10-CM) codes previously curated as restrictive (RESPONSE RCT) and extensive (GEMFIN) definitions of TE's within the Diagnosis table in Optum EHR database.

  2. Number of Thromboembolic events between the high and low risk subgroups of BAT group

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Within the BAT group, high risk (≥ 1 TE on average per year ) and low risk (< 1 TE on average per year) subgroups were identified based on the frequency of TEs and characterized according to patient sociodemographics, comorbidities, symptoms, clinical, and medication variables.

Secondary outcomes

  1. Incidence rate of thromboembolic event

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Difference in the incidence rate of TEs in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX were reported. A TE was defined using International Classification of Diseases 9th Revision (ICD-9- CM) and International Classification of Diseases 10th Revision (ICD-10-CM) codes previously curated as restrictive (RESPONSE RCT) and extensive (GEMFIN) definitions of TE's within the Diagnosis table in Optum EHR database.

  2. Time to first thromboembolic event

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Time to first TE in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported. A TE was defined using International Classification of Diseases 9th Revision (ICD-9- CM) and International Classification of Diseases 10th Revision (ICD-10-CM) codes previously curated as restrictive (RESPONSE RCT) and extensive (GEMFIN) definitions of TE's within the Diagnosis table in Optum EHR database.

  3. Incidence rate of phlebotomy procedures

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Difference in the incidence rate of phlebotomies in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported.

    Phlebotomies were defined using Current Procedural Terminology, Fourth Edition (CPT4) codes within the Procedure table in Optum EHR.

  4. Time to first phlebotomy procedure

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Time to first phlebotomy in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported. Phlebotomies were defined using Current Procedural Terminology, Fourth Edition (CPT4) codes within the Procedure table in Optum EHR.

  5. Incidence rate of neoplasm transformations

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Difference in the incidence rate of neoplasm transformations in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported.

    A neoplasm transformation was defined as:

    • PV to MF (Myelofibrosis)
    • MF to AML (Acute Myeloid Leukemia)
    • PV to AML Neoplasm transformations were detected using ICD-9-CM and ICD-10-CM codes within the Diagnosis table in Optum EHR.

  6. Time to first neoplasm transformation

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Time to first neoplasm transformation in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported.

    A neoplasm transformation was defined as:

    • PV to MF
    • MF to AML
    • PV to AML Neoplasm transformations were detected using ICD-9-CM and ICD-10-CM codes within the Diagnosis table in Optum EHR.

  7. Treatment patterns: Proportion of patients using different PV-related treatments

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Differences in treatment patterns in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported.

    BAT comprised of multiple therapies for PV including HU, IFN, pegylated IFN (PEG-IFN) and others. These therapies were reported as subcategories under BAT.

  8. Healthcare resource utilization (HCRU): Number of inpatient hospitalizations

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Hospitalization was defined as an inpatient stay with a valid visit_ID within the Visit table in Optum EHR. Inpatient hospitalizations were reported as allcause and as PV-specific respectively

  9. Healthcare resource utilization (HCRU): Number of outpatient visits

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Visits with the following visit type: "observation patient" with a valid visit_ID was included as an outpatient visit. Outpatient visits that resulted in an inpatient hospitalization were not included. Outpatient visits were reported as all-cause and as PV-specific respectively.

  10. Healthcare resource utilization (HCRU): Number of emergency room visits

    Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)

    Visits with the following visit type: "emergency patient" with a valid visit_ID was included as an emergency room visit. Emergency room visits that resulted in an inpatient hospitalization were not included. Emergency room visits were reported as all-cause and as PV-specific respectively.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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.

Important dates

Study start
2020
Primary completion
2021
Study completion
2021
First posted
Jun 16, 2022
Registry last updated
Jul 5, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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