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
