Clinical Effectiveness and Safety of Second-Line Therapies for Immune Thrombocytopenia (ITP): A Systematic Literature Review
Author(s)
Isabelle Lundqvist, MSc1, Kalitsa Filioussi, MD2, Meritxell Vendranas, MD3, Shaun Walsh, MSc4, Aditi Kataria, M Pharm5, Lovneet Saini, M Pharm5.
1Novartis, Kista, Sweden, 2Novartis Farma, Origgio, Italy, 3Novartis Farmacéutica, S.A., Spain, 4Novartis Ireland Ltd, Dublin, Ireland, 5Novartis Healthcare Pvt. Ltd, Hyderabad, India.
1Novartis, Kista, Sweden, 2Novartis Farma, Origgio, Italy, 3Novartis Farmacéutica, S.A., Spain, 4Novartis Ireland Ltd, Dublin, Ireland, 5Novartis Healthcare Pvt. Ltd, Hyderabad, India.
OBJECTIVES: Immune Thrombocytopenia (ITP) is an autoimmune disorder characterized by low platelet counts and bleeding symptoms. This systematic literature review summarizes clinical evidence on the effectiveness and safety of second-line treatments for primary ITP.
METHODS: The review included studies from Embase®, PubMed®, and Cochrane database from 2007 to June 2024 following PRISMA and Cochrane guidelines. Also, grey literature searches, including congress abstracts, trial registries, HTA reports, and bibliographic searches were conducted from 2020 to June 2024.
RESULTS: A total of 45 unique studies were included based on predefined criteria. Most studies were conducted in Asia (19), Europe (14), and the Middle East (6), with observational studies (30) being the most common design. The most frequently evaluated clinical efficacy outcomes were complete response (CR) and platelet count, reported in 33 and 31 studies, respectively. TPO-RAs and splenectomy demonstrated highest rates of CR ranging between 61.9%-84.8% and 75%-86.4% at 48 to 52 weeks. Rituximab reported CR in 31.2%-35.9% at 48 weeks. A multi-country study reported that TPO-RAs achieved sustained response off treatment in 30.5% patients at 12 months after tapering and discontinuation following stable platelet counts. Adverse events (AEs) were reported across 33 studies, and ranged from mild to severe, with grade ≥3 AEs observed in 3%-31% patients. Treatment emergent AEs were predominantly linked to Eltrombopag and Romiplostim, with AEs reported in 2%-37% and serious AEs in 1%-4.8%. Mortality rates were in range of 8%-18.6% for rituximab and 1.4%-18% for splenectomy. Quality of life outcomes were reported in only three studies.
CONCLUSIONS: The review highlights that there remains ongoing need for treatment options in ITP that demonstrate higher efficacy while minimizing AEs. Addressing this gap is essential for advancing the overall management and quality of life for patients with ITP.
METHODS: The review included studies from Embase®, PubMed®, and Cochrane database from 2007 to June 2024 following PRISMA and Cochrane guidelines. Also, grey literature searches, including congress abstracts, trial registries, HTA reports, and bibliographic searches were conducted from 2020 to June 2024.
RESULTS: A total of 45 unique studies were included based on predefined criteria. Most studies were conducted in Asia (19), Europe (14), and the Middle East (6), with observational studies (30) being the most common design. The most frequently evaluated clinical efficacy outcomes were complete response (CR) and platelet count, reported in 33 and 31 studies, respectively. TPO-RAs and splenectomy demonstrated highest rates of CR ranging between 61.9%-84.8% and 75%-86.4% at 48 to 52 weeks. Rituximab reported CR in 31.2%-35.9% at 48 weeks. A multi-country study reported that TPO-RAs achieved sustained response off treatment in 30.5% patients at 12 months after tapering and discontinuation following stable platelet counts. Adverse events (AEs) were reported across 33 studies, and ranged from mild to severe, with grade ≥3 AEs observed in 3%-31% patients. Treatment emergent AEs were predominantly linked to Eltrombopag and Romiplostim, with AEs reported in 2%-37% and serious AEs in 1%-4.8%. Mortality rates were in range of 8%-18.6% for rituximab and 1.4%-18% for splenectomy. Quality of life outcomes were reported in only three studies.
CONCLUSIONS: The review highlights that there remains ongoing need for treatment options in ITP that demonstrate higher efficacy while minimizing AEs. Addressing this gap is essential for advancing the overall management and quality of life for patients with ITP.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO44
Topic
Clinical Outcomes, Study Approaches
Topic Subcategory
Comparative Effectiveness or Efficacy
Disease
Oncology, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)