Abstract

Introduction

The prevalence of myelofibrosis (MF) is 3.6 to 5.6 cases per 100,000 in the United States. Overall median survival is 3 years and 1.3 years for patients with intermediate-2 and high-risk disease, respectively. The MF PracticE-Based Network ResearCH (ENRiCH) platform is an adaptive, dynamic, data hub designed to accumulate real-world data to identify MF patients, understand their treatment patterns and unmet needs, inform clinical trial design, and conduct research using clinically relevant outcomes. A descriptive analysis of the demographic and clinical characteristics of MF patients from the ENRiCH platform is reported in this study.

Methods

The ENRiCH platform was developed from patient-level data representing approximately 1 billion US private practitioner medical claims, >1.6 billion pharmacy claims, and electronic medical records from >500,000 cancer patients treated in community oncology practices at 344 locations in 37 states and encompassing approximately 550 treating providers. ENRiCH includes patients with a diagnosis of MF, polycythemia vera (PV), or essential thrombocytopenia (ET), and/or a ruxolitinib prescription between November 1, 2010 and October 31, 2012; patients who received ruxolitinib in a clinical trial were excluded.

Results

As of March 2013, 6362 MF and 299 ruxolitinib patients were identified. Mean (SD) age was 67.0 (12.6) years for MF patients and 65.8 (15.0) years for the ruxolitinib subgroup, with 45% females. Hematologic comorbidities included myeloid disorders (80% MF; 70% ruxolitinib) and lymphoid disorders (10% MF; 5% ruxolitinib). The most common myeloid disorders were primary MF (47% MF; 29% ruxolitinib), myelodysplastic syndrome (35% MF; 26% ruxolitinib), ET (25% MF; 17% ruxolitinib), and PV (25% MF; 17% ruxolitinib). Common comorbidities of interest that were observed included infectious diseases (35% MF; 35% ruxolitinib), thrombocytopenia (33% MF; 23% ruxolitinib), pulmonary hypertension (7% MF; 7% ruxolitinib), and vitamin D deficiency (7% MF; 8% ruxolitinib). The mean (SD) Charlson comorbidity index (CCI) was 4.36 (2.4) for MF patients and 4.28 (2.4) for the ruxolitinib subgroup. Clinical characteristics and laboratory results among patients with diagnostic or clinical data from the index date forward are shown in the table.

The 5 most common concomitant medication groups for ruxolitinib patients were loop diuretics (11%), gout agents (10%), hydrocodone combinations (9%), opioid agonists (9%), and proton pump inhibitors (9%). The top 5 most common medications taken within 180 days prior to new ruxolitinib starts were hydrocodone combinations (21%), antineoplastics (19%), gout agents (18%), glucocorticosteroids (15%), and fluoroquinolones (14%). The predominant [MF (ruxolitinib)] payer types observed were Medicare [48% (37%)], Medicaid [4% (1%)], and commercial insurance [37% (21%)].

Conclusions

The observed demographic and clinical characteristics of MF patients are similar to prior MF epidemiological studies (Mehta J et al. Leuk Lymphoma. 2013;Early Online: 1-6), suggesting that the sample of MF ENRiCH patients is reflective of the broader US MF population and will allow for broad-based quantification of unmet needs and treatment outcomes. The addition of more MF patients and longer follow-up data will further enhance the value of this large US real-world MF patient data hub including those on current MF therapies.

This study was sponsored by Sanofi.

Disclosures:

Fonseca:IMS Health - a company that received funds from Sanofi to conduct the study: Employment. Lee:Sanofi: Employment. Wang:Sanofi: Employment, Equity Ownership. Dhawan:Sanofi: Employment. Thompson:Sanofi: Employment. Iqbal:Sanofi: Employment, Equity Ownership. Redmond:IMS Health: Consultancy, Employment.

 Myelofibrosis Patients Ruxolitinib Patients 
Clinical Characteristics   
6236 181 
Splenomegaly 22% 15% 
Myeloid metaplasia 45% 14% 
Thrombocytopenia 22% 9% 
Laboratory Test Results   
583 90 
Hemoglobin <10 g/dL 63% 72% 
Platelet count <50,000/microlitera 22% 18% 
Platelet count <100,000/microliterb 38% 42% 
Platelet count 100,000-200,000/microliterb 48% 57% 
Platelet count >200,000/microliterb 65% 62% 
 Myelofibrosis Patients Ruxolitinib Patients 
Clinical Characteristics   
6236 181 
Splenomegaly 22% 15% 
Myeloid metaplasia 45% 14% 
Thrombocytopenia 22% 9% 
Laboratory Test Results   
583 90 
Hemoglobin <10 g/dL 63% 72% 
Platelet count <50,000/microlitera 22% 18% 
Platelet count <100,000/microliterb 38% 42% 
Platelet count 100,000-200,000/microliterb 48% 57% 
Platelet count >200,000/microliterb 65% 62% 

aPossible thrombocytopenia

bAssociated with different dosing levels for ruxolitinib per FDA label.

Author notes

*

Asterisk with author names denotes non-ASH members.