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Hamostaseologie
DOI: 10.1055/a-2404-0306
Original Article

Management of Adult Patients with Newly Diagnosed or Relapsed Primary Immune Thrombocytopenia in Eastern Austria

Jasmin Rast‡   *
1   Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
,
Theresa Schramm‡   *
1   Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
,
Dino Mehic
1   Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
,
Michael Fillitz
2   Department of Internal Medicine, Hanusch Hospital, Vienna, Austria
,
Tanja Drexel
3   Novartis Pharma GmbH, Vienna, Austria
,
Veronika Neusiedler-Nicolas
3   Novartis Pharma GmbH, Vienna, Austria
,
Cihan Ay
1   Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
,
Ingrid Pabinger
1   Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
,
Johanna Gebhart
1   Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
› Institutsangaben Funding The ITP Biobank was sponsored by research collaboration with Novartis (C0381) and research grants from Sobi (C0380, C1031).
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Abstract

Background Treatment sequence in primary immune thrombocytopenia (ITP) is based on national and international recommendations, treatment availability, and physician expertise.

Aim This article aimed to provide real-world data on treatment sequence and responses to first- and second-line treatments in newly diagnosed and relapsed adult ITP patients.

Methods We analyzed a cohort of 46 adult ITP patients from the Vienna ITP Biobank, who started first-line therapy within 1 week before their first study visit between February 2016 and March 2023. We investigated clinical patient characteristics and patient management in our specialized center and examined the impact of the international ASH guidelines on ITP treatment.

Results Forty-six primary ITP patients, 27 (58.7%) with newly diagnosed ITP and 19 (41.3%) with relapsed ITP, were investigated. Most patients were female (65.2%) with a median platelet count of 9 × 109/L, and 31 patients (67.4%) had bleeding symptoms. All patients received first-line treatment with oral prednisolone; 15 patients received oral prednisolone combined with intravenous immunoglobulins (IVIGs), which were more commonly administered in newly diagnosed than in relapsed ITP patients. First-line therapy resulted an overall response in 82.6% of patients after a median (interquartile range [IQR]) time of 10 (5–25) days. There was no difference in treatment responses between newly diagnosed and relapsed ITP patients, but newly diagnosed patients had a shorter time to response (median [IQR]: 8 [5–14] and 14 [8–27], p = 0.02). Twenty-three (50%) of the patients (11/27 newly diagnosed [40.7%], 12/19 relapsed [63.2%]) required second-line ITP therapy. Thrombopoietin-receptor agonists (TPO-RAs) were the most commonly used second-line therapy with a response rate of 73.7%, and a median (IQR) time to treatment response of 15 (12–20) days. Overall response rates to TPO-RA treatment did not differ between newly diagnosed and relapsed ITP. Following the publication of novel guidelines in 2019, the median (IQR) duration of corticosteroid treatment shortened (100–52 days, p = 0.01), as did the time to second-line treatment (160–47 days, p = 0.01), and the median number of first-line therapies decreased from 2 (1–3) to 1 (1–2).

Conclusion Initial treatment with corticosteroids was effective in the majority of newly diagnosed and relapsed ITP. Response rates to initial corticosteroid treatment in ITP patients are consistent with previous data, but only 50% achieve sustained remission. TPO-RAs, which are well tolerated and effective, are the most commonly used second-line therapy in our study population. International guidelines have led to faster treatment transitions and reduced splenectomy rates. Integration of real-life experience, expert consensus, and guidelines optimizes ITP patient management.

Keywords

primary immune thrombocytopenia - platelet function - bleeding severity - first-line therapy

Authors' Contributions

All authors contributed to the study's conception and design. Data collection was performed by J.R., T.S., D.M., and M.F. Data analysis was performed by J.R. and T.S. The first draft of the manuscript was written by J.R.; I.P. and J.G. commented on previous versions of the manuscript. All authors read and approved the final manuscript.


* These authors have contributed equally.




Publikationsverlauf

Eingereicht: 16. März 2024

Angenommen: 22. August 2024

Artikel online veröffentlicht:
05. November 2024

© 2024. Thieme. All rights reserved.

Georg Thieme Verlag KG
Stuttgart · New York

 

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