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

NCT05421104 | Completed

• 1 other identifier: CINC424B3001
• Study Type: observational
• Target: 1,576
• Locations: 1 country
• Sites: 1

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.

Conditions

Polycythemia Vera (PV)

Keywords

Polycythemia vera,; Ruxolitinib,; JAK protein inhibitor,; Hydroxyurea,; Thrombotic events

Outcome Measures

Primary Outcomes (2)

• Number of Thromboembolic events between the RUX and BAT group

  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.

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

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

  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.

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

Secondary Outcomes (10)

• Incidence rate of thromboembolic event

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

• Time to first thromboembolic event

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

• Incidence rate of phlebotomy procedures

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

• Time to first phlebotomy procedure

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

• Incidence rate of neoplasm transformations

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

Study Arms (2)

Ruxolitinib (RUX)

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: Ruxolitinib

Best available therapy (BAT)

PV patients who were resistant to or intolerant of Hydroxyurea (HU) (as defined on the index date) and continued HU treatment or switched to other available therapies other than RUX in the post-index period.

Interventions

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.

Also known as: Jakavi

Ruxolitinib (RUX)

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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.

You may not qualify if:

• Excluded patients:
• \- With a MF or AML diagnosis prior to a PV diagnosis.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis Investigative Site

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

Location

Related Links

• Results for CINC424B3001 from Novartis Clinical Trials Results Database

MeSH Terms

Conditions

Polycythemia Vera

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 13, 2022
• First Posted: June 16, 2022
• Study Start: November 27, 2020
• Primary Completion: June 29, 2021
• Study Completion: June 29, 2021
• Last Updated: July 5, 2022

Record last verified: 2022-06

Locations

US (1)