Practical considerations for the management of immune thrombocytopenic purpura

Abstract

Immune thrombocytopenic purpura (ITP) is a rare hematological disorder with an autoimmune-mediated, often dramatic reduction of platelets in peripheral blood. Thrombocytopenia results from a reduced life span of thrombocytes and an additionally decreased production in bone marrow. For decades, the first-line therapy for ITP has been corticosteroids. As significant thrombocytopenic bleedings occur, the use of additional medication may be needed. Recent updates on therapy guidelines recommend the shortest possible use of corticosteroids. Thrombopoietin-receptor agonists are often used second line. Today splenectomy, which was previously recommended after unsuccessful first-line therapy, is usually considered much later. Patients who do not respond even after multiple lines of therapy continue to pose a major challenge. New drugs for ITP treatment are now available after steroid failure and will be discussed. This review gives a short summary on actual therapy guidelines taking into account newly available therapy options. In addition, comparisons between selected published data and experience at our department are made.

Keywords: Corticosteroids; Fostamatinib; Immunoglobulines; Rituximab; Splenectomy; Thrombopoietin agonists.

Fig. 1

Ratio of primary to secondary immune thrombocytopenic purpura (ITP) in 93 evaluable patients diagnosed at the 3rd Medical Department, Hanusch Krankenhaus, Vienna, between 2010 and 2020

Fig. 2

Documented infection at time of diagnosis in newly diagnosed immune thrombocytopenic purpura (ITP) patients (n = 93) at the 3rd Medical Department, Hanusch Krankenhaus, Vienna, between 2010 and 2020

Fig. 3

In 93 patients, 87.1% show initial response to first-line immune thrombocytopenic purpura (ITP) treatment, i.e., corticosteroids ± intravenous immunoglobulines (IvIG)

Fig. 4

Breakdown of best response to first-line treatment. 100% of the responding patients (n = 81) were able to reach at least a partial remission (PR; platelet count ≥50 G/L) and 71.6% were able to achieve a complete remission (CR; platelet count ≥100 G/L). ORR overall response rate

Fig. 5

Percentage of immune thrombocytopenic purpura (ITP) patients able to maintain the best response (partial remission defined as platelet count ≥50 G/L, complete remission defined as platelet count ≥100 G/L) for at least 3 months regardless of the dose change of corticosteroids