Identifying Treatment Patterns for Essential Thrombocythemia Using Real-world Data
Author(s)
Andrew M. Howe, PharmD, BA1, Hung Lun Chien, MPH2, Craig Zimmerman, PhD3, Ru-Yun Chiang, MSc4, Paul Walden, PhD5, Robert Geller, MD5;
1Pharmaessentia, Woodstock, GA, USA, 2Pharmaessentia, Global HEOR, Burlington, MA, USA, 3Pharmaessentia, Regulatory, Burlington, MA, USA, 4Pharmaessentia, Commercial Analytics & Insights, Burlington, MA, USA, 5Pharmaessentia, Medical, Burlington, MA, USA
1Pharmaessentia, Woodstock, GA, USA, 2Pharmaessentia, Global HEOR, Burlington, MA, USA, 3Pharmaessentia, Regulatory, Burlington, MA, USA, 4Pharmaessentia, Commercial Analytics & Insights, Burlington, MA, USA, 5Pharmaessentia, Medical, Burlington, MA, USA
Presentation Documents
OBJECTIVES: Essential thrombocythemia (ET) is a rare myeloproliferative neoplasm (MPN) that may result in thromboembolic and hemorrhagic complications and potential transformation into aggressive myeloid neoplasms. Current ET treatment landscape information is limited despite ET patient medical expenditures being 2.3 times greater versus matched comparisons. Thus, the study objective was to characterize the treatment landscape among patients diagnosed with ET in a US healthcare population.
METHODS: Veeva Compass Claims were used to identify patients with an ET diagnosis claims (DC) using two (ICD-10 D47.3) claims ≥30 days apart between 2020-2023 and ≥1 DC and ≥1 pharmacy claim (PC) in reporting year for continuous enrollment criteria. Patients were excluded if they had DC for AML or MF any time prior to diagnosis or secondary thrombocytosis, or hereditary thrombocytosis any time before or after the initial ET diagnosis.
RESULTS: Among prevalent 2023 patients (n=41,400), 72% were female and 90% were age >40 years. The risk distribution (high, intermediate, low) was 20%, 54% and 26% respectively. The prescription treatment distribution was hydroxyurea 30%, anagrelide 3%, ruxolitinib 1%, interferon alfa 1% with 67% receiving no treatment observed. Amongst newly treated patients in 2021 (n=3,100), 90% were treated with only first-line therapy, 6% transitioned to second-line therapy and 3% of patients transitioned to third-line therapy. The mean and median annual switch frequency was 1.1 and 1.0 respectively. Hydroxyurea was used in 94%, 15% and 51% of patients in 1L, 2L and 3L with subsequent anagrelide use in 45% and 22% in 2L and 3L respectively. Patients experienced trigger events (leukocytosis, thrombosis and major bleeding) 23%, 15% and 12% respectively occurring within 3 months of a treatment switch.
CONCLUSIONS: Data suggests amongst treated patients, therapy switching involved cycling of hydroxyurea to anagrelide to hydroxyurea and limited use of other therapies, thus suggesting a lack of available treatment options for ET patients.
METHODS: Veeva Compass Claims were used to identify patients with an ET diagnosis claims (DC) using two (ICD-10 D47.3) claims ≥30 days apart between 2020-2023 and ≥1 DC and ≥1 pharmacy claim (PC) in reporting year for continuous enrollment criteria. Patients were excluded if they had DC for AML or MF any time prior to diagnosis or secondary thrombocytosis, or hereditary thrombocytosis any time before or after the initial ET diagnosis.
RESULTS: Among prevalent 2023 patients (n=41,400), 72% were female and 90% were age >40 years. The risk distribution (high, intermediate, low) was 20%, 54% and 26% respectively. The prescription treatment distribution was hydroxyurea 30%, anagrelide 3%, ruxolitinib 1%, interferon alfa 1% with 67% receiving no treatment observed. Amongst newly treated patients in 2021 (n=3,100), 90% were treated with only first-line therapy, 6% transitioned to second-line therapy and 3% of patients transitioned to third-line therapy. The mean and median annual switch frequency was 1.1 and 1.0 respectively. Hydroxyurea was used in 94%, 15% and 51% of patients in 1L, 2L and 3L with subsequent anagrelide use in 45% and 22% in 2L and 3L respectively. Patients experienced trigger events (leukocytosis, thrombosis and major bleeding) 23%, 15% and 12% respectively occurring within 3 months of a treatment switch.
CONCLUSIONS: Data suggests amongst treated patients, therapy switching involved cycling of hydroxyurea to anagrelide to hydroxyurea and limited use of other therapies, thus suggesting a lack of available treatment options for ET patients.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
RWD37
Topic
Real World Data & Information Systems
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases