Utility Estimates for Health States Associated With Immune Thrombocytopenia: A Systematic Literature Review
Author(s)
Isabelle Lundqvist, MSc1, Kalitsa Filioussi, MD, MPhil2, Shaun Walsh, MSc3, Aditi Kataria, M.Pharm4, vilas Belekar, M.Pharm4.
1Novartis Sverige AB, Kista, Sweden, 2Novartis Farma, Milan, Italy, 3Novartis Ireland Ltd, Dublin, Ireland, 4Novartis Healthcare Pvt. Ltd., Hyderabad, India.
1Novartis Sverige AB, Kista, Sweden, 2Novartis Farma, Milan, Italy, 3Novartis Ireland Ltd, Dublin, Ireland, 4Novartis Healthcare Pvt. Ltd., Hyderabad, India.
OBJECTIVES: The systematic review provided an overview of studies evaluating health-state utility values in immune thrombocytopenia (ITP) patients which are the most important data input in economic models.
METHODS: A systematic search was conducted in scientific databases (EMBASE®, PubMed® and Cochrane Central, inception-February 2025), along with HTA websites and conference proceedings to identify studies reporting utilities/disutilities in adults with ITP published in English.
RESULTS: Twenty-four studies met the inclusion criteria. EuroQol 5-Dimensions (EQ-5D) was the most frequently used tool for estimating utilities (n=12), followed by Time-Trade-Off (TTO; n=9) and Short-form-6 Dimension (SF-6D; n=3). Majority of studies focussed on second-line therapy (2L) (n=8), followed by ≥2L (n=6). Patients who received first-line corticosteroids demonstrated an increase in utilities over 12-months (0.70-0.76 using SF-6D). Among ≥2L eltrombopag responders, the utility value was 0.74 using SF-6D. For patients treated with ≥2L eltrombopag, utility values were 0.73 for those with controlled platelet counts (≥50×10⁹/L) and 0.69 for those with uncontrolled platelet counts (<30×10⁹/L), using SF-6D. The utility values for ≥2L romiplostim responders were 0.83 using EQ-5D and 0.63 using TTO. EQ-5D utilities remained stable across platelet counts (11-≥150×10⁹/L: 0.79-0.83), with a slight decline in ≤10×10⁹/L (0.75), suggesting limited sensitivity of EQ-5D to detect health-related quality-of-life (HRQoL) differences associated with platelet counts in ITP patients. Additionally, fatigue a symptom of ITP was not accounted for in EQ-5D. Disutilities were associated with serious adverse-events (AEs) (-0.4 to -0.08) and other AEs (-0.1 to -0.02).
CONCLUSIONS: Health-state utility values varied with response to different treatments in ITP patients. Additionally, differences in utilities were observed with different scales of measurement. The primarily used EQ-5D and SF-6D are generic scales for measurement of HRQoL and not sensitive enough to detect impairment associated with fatigue and changes in platelet count. These findings emphasize the need for more sensitive, disease-specific utility assessment tools in ITP.
METHODS: A systematic search was conducted in scientific databases (EMBASE®, PubMed® and Cochrane Central, inception-February 2025), along with HTA websites and conference proceedings to identify studies reporting utilities/disutilities in adults with ITP published in English.
RESULTS: Twenty-four studies met the inclusion criteria. EuroQol 5-Dimensions (EQ-5D) was the most frequently used tool for estimating utilities (n=12), followed by Time-Trade-Off (TTO; n=9) and Short-form-6 Dimension (SF-6D; n=3). Majority of studies focussed on second-line therapy (2L) (n=8), followed by ≥2L (n=6). Patients who received first-line corticosteroids demonstrated an increase in utilities over 12-months (0.70-0.76 using SF-6D). Among ≥2L eltrombopag responders, the utility value was 0.74 using SF-6D. For patients treated with ≥2L eltrombopag, utility values were 0.73 for those with controlled platelet counts (≥50×10⁹/L) and 0.69 for those with uncontrolled platelet counts (<30×10⁹/L), using SF-6D. The utility values for ≥2L romiplostim responders were 0.83 using EQ-5D and 0.63 using TTO. EQ-5D utilities remained stable across platelet counts (11-≥150×10⁹/L: 0.79-0.83), with a slight decline in ≤10×10⁹/L (0.75), suggesting limited sensitivity of EQ-5D to detect health-related quality-of-life (HRQoL) differences associated with platelet counts in ITP patients. Additionally, fatigue a symptom of ITP was not accounted for in EQ-5D. Disutilities were associated with serious adverse-events (AEs) (-0.4 to -0.08) and other AEs (-0.1 to -0.02).
CONCLUSIONS: Health-state utility values varied with response to different treatments in ITP patients. Additionally, differences in utilities were observed with different scales of measurement. The primarily used EQ-5D and SF-6D are generic scales for measurement of HRQoL and not sensitive enough to detect impairment associated with fatigue and changes in platelet count. These findings emphasize the need for more sensitive, disease-specific utility assessment tools in ITP.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR260
Topic
Economic Evaluation, Patient-Centered Research, Study Approaches
Topic Subcategory
Health State Utilities
Disease
Oncology, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)