Neoplasms

Blood and Lymph Conditions

All Conditions

Rare Diseases

Polycythemia Vera

Polycythemia

Hematologic Diseases

Bone Marrow Neoplasms

Hematologic Neoplasms

Bone Marrow Diseases

Myeloproliferative Disorders

Chronic Myeloproliferative Disorders

Antineoplastic Agents

Antisickling Agents

All Drugs and Chemicals

Hydroxyurea

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.

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.

Ruxolitinib

Novartis Pharmaceuticals

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.

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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.

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.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Ruxolitinib for Polycythemia Vera in Patients Resistant to or Intolerant of Hydroxyurea.	NCT05421104	Polycythemia Ve...	Ruxolitinib	18 Years - 99 Years	Novartis
Ruxolitinib for Polycythemia Vera in Patients Resistant to or Intolerant of Hydroxyurea.	NCT05421104	Polycythemia Ve...	Ruxolitinib	18 Years - 99 Years	Novartis

Spots Global Cancer Trial Database for hydroxyurea,

Every month we try and update this database with for hydroxyurea, cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.