Authors' objectives: CADTH undertook a Health Technology Assessment (HTA) to review the effectiveness and cost-effectiveness of treatments for ITP in adults after failure of first-line treatments. A secondary objective was to determine the place of splenectomy in therapy.

Authors' results and conclusions: Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by low platelets and an increase in bleeding risk. It has an incidence rate of 3.3 per 100,000 adults per year. After first-line corticosteroid treatment, there is limited evidence on the best subsequent-line therapies based on evidence of low certainty. Subsequent therapies after failure of corticosteroid use include several treatments, such as rituximab, eltrombopag, romiplostim, fostamatinib, avatrombopag, and splenectomy. The goal of treatment is to reduce bleeding and prolong life. Increases in platelet count are considered a surrogate to these treatment goals. We conducted a narrative review of 15 publications (10 randomized controlled trials) found through a systematic literature search. The review addressed the following policy questions: What treatment(s) should be used in adult patients with ITP who have failed first-line treatments? What is the place in therapy of splenectomy in adult patients with ITP? We found that eltrombopag and romiplostim increased platelet count response and improved ITP symptoms and health-related quality of life compared to placebo. Interpretation of the effectiveness of avatrombopag, rituximab, and fostamatinib was inconclusive due to the limited availability of evidence and varying risks of bias in the trials. There was a lack of evidence in specific subgroups of patients and on the role of splenectomy in the sequency of subsequent-line therapies. Further research is needed to compensate for clinical data gaps to inform an appropriate and relevant economic evaluation. CADTH suggests that jurisdictions may consider requesting that an implementation advice panel be conducted to bring together clinical experts to suggest measures (eg, discontinuation criteria) to manage the clinical and economic uncertainty identified in this report.

Project Status: Completed

URL for project: https://www.cadth.ca/treatment-adult-patients-immune-thrombocytopenia-after-failure-first-line-therapies

URL for protocol: https://www.cadth.ca/sites/default/files/hta-he/HT0035%20ITP%20Protocol%20Final.pdf

Year Published: 2023

URL for published report: https://www.cadth.ca/sites/default/files/hta-he/HT0035-ITP.pdf

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Canada

Organisation Name: Canadian Agency for Drugs and Technologies in Health

Contact Address: 600-865 Carling Avenue, Ottawa, ON K1S 5S8 Canada. Tel: +1 613 226 2553; Fax: +1 613 226 5392;

Contact Name: requests@cadth.ca

Contact Email: requests@cadth.ca

Copyright: <p>Canadian Agency for Drugs and Technologies in Health (CADTH)</p>