A EUROPEAN RETROSPECTIVE STUDY to DESCRIBE REAL-WORLD OUTCOMES, TREATMENT PATTERNS and RESOURCE USE in PATIENTS with PRIMARY IMMUNE THROMBOCYTOPENIA (ITP) TREATED with AVATROMBOPAG: INTERIM RESULTS.
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All Authors
Zaja, F.
Hill, Q.A.
Glen, F.
Hosnijeh, F.S.
Treust, L.L.
Groves, L.
Putnik, M.
Rich, C.
LTHT Author
Hill, Quentin
LTHT Department
Oncology
Haematology
Haematology
Non Medic
Publication Date
2024
Item Type
Conference Abstract
Language
Subject
ADULT , AGED , THROMBOCYTOPOENIA , TREATMENT OUTCOME , CLINICAL PROTOCOLS , COHORT STUDIES , DIAGNOSIS , DRUG THERAPY , WOMEN , FOLLOW-UP-STUDIES , STATISTICS AS TOPIC , MEN , MULTICENTRE STUDIES AS TOPIC , BLOOD PLATELETS , RETROSPECTIVE STUDIES
Subject Headings
Abstract
Background: Clinical trials show that avatrombopag (AVA), a thrombopoietin-receptor agonist (TPO-RA), is a safe and efficacious treatment option in patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments. However, there is limited real-world evidence on AVA's clinical effectiveness in the broader patient population treated in routine clinical practice. Aim(s): This study aims to report real-world evidence on the characteristics, treatment patterns, clinical outcomes, and ITP-related healthcare resource use (HCRU) of patients with primary ITP treated with AVA in Europe. Method(s): A non-interventional, retrospective, multi-center study based on a review of hospital medical records of adult patients with primary ITP treated with AVA as part of routine clinical care is currently being conducted in six European countries: Spain, Italy, Germany, Czech Republic, the United Kingdom (UK), and Belgium. Inclusion criteria are confirmed diagnosis of ITP, receiving AVA for the treatment of primary ITP within routine clinical practice, medical records available for >=12 weeks prior to and from index event (AVA initiation), aged >=18 years at the start of first TPO-RA treatment, and providing consent to access patient's medical records in line with country regulations. The baseline period is defined as 26 weeks prior to AVA initiation, and the post-index period (follow-up) will continue to date of most recent follow-up visit, platelet count (PC) assessment, or date of death (whichever was earliest). The primary endpoints include: proportion of patients achieving meaningful PC thresholds (i.e., PC >=50x109/L or >=30x109/L) and complete response (>=100x109/L) by week 12, 26, 52 and at any time while on AVA post-index; time from index date to an initial platelet response; proportion of patients with a >=50%, >=75%, and doubled increase in PC from baseline by week 12, 26, and 52 among patients who respond to AVA; and duration of response. The secondary endpoints include information related to demographic and clinical characteristics, treatment patterns, adverse events, and HCRU in ITP patients whilst on treatment with AVA. Data related to clinical outcomes, adverse events, and HCRU are being collected for the follow-up period. Treatment data including use of AVA, concomitant and prior treatments and those initiated post discontinuation of AVA are collected from index to the end of the follow-up. The key observation periods are summarised in Figure 1. The study analyses will be descriptive in nature and will be presented for the whole cohort and stratified by TPO-RA naive and TPO-RA exposed sub-groups. Time-to-event variables will be calculated using the Kaplan-Meier methodology or cumulative incidence functions in case of competing risks. Result(s): As of first data cut-off (2 Feb 2024), data relating to 17 patients have been collected: 7 TPO-RA naive and 10 TPO-RA exposed (70% eltrombopag, 20% romiplostim, 10% both) with a mean age of 57.4 (standard deviation: 23.4) years. 53% of patients are female. Median time from ITP diagnosis until AVA initiation was 7.1 years (range: 0-37.7) and patients were followed for a median time of 47.9 (8-142.1) weeks post AVA initiation. Data are being collected, and the results of the complete interim analysis will be presented in the close future. (Table present) Summary/Conclusion: This study will provide detailed insights into the real-world clinical effectiveness of AVA in patients with primary ITP in Europe as well a complete picture of the characteristics, treatment patterns, and HCRU.
Journal
HemaSphere