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How Do Patients Themselves Perceive Today's Treatment Options for ITP and What Are Their Biggest Fears?

21. 11. 2023

What is the perception and view of the treatment options for immune thrombocytopenia (ITP) from the patients' perspective, what do they fear, what approaches do they prefer, and why? Lozano et al. included a discussion on this topic in their review article for Expert Review of Hematology, which can be helpful in clinical practice for better understanding patients' needs and adjusting treatment algorithms.

Introduction

Immune thrombocytopenia (ITP) is a rare autoimmune disease characterized by increased destruction of platelets and impaired platelet production. The primary manifestation of ITP is an increased tendency to bleed, especially skin purpura and mucosal bleeding. For ITP patients, bleeding symptoms result in a reduced quality of life, both physically and mentally, as they may also suffer from anxiety, depression, or fatigue.

Currently, ITP is categorized into different phases – acute (symptoms lasting up to 3 months), persistent (symptoms lasting up to 12 months), and chronic (symptoms lasting more than 1 year). This classification impacts treatment options. Recently, there has been a greater emphasis on incorporating modern drugs (notably from the TPO-RA group, i.e., thrombopoietin receptor agonists) into earlier phases of treatment, not just in the chronic ITP phase or after the failure of other modalities.

What Do Patients Say?

For some patients, the watch & wait strategy is fully acceptable; however, for others, it increases anxiety because “nothing is happening.” The majority of patients are initially treated with corticosteroids (CS), which are well-known for their numerous side effects, potentially worsening the quality of life.

Some patients refuse CS use due to concerns about side effects. Current recommendations support shortening the duration of corticosteroid administration (no longer than 6–8 weeks), but even a short treatment cycle can be burdensome for some patients. Before beginning therapy, it is also important to assess any comorbidities (diabetes, depression, etc.) that may influence the decision to avoid corticosteroids if possible.

One survey study revealed that the perspectives of doctors and patients on this issue can differ significantly—patients expressed more concern about treatment side effects, particularly CS, whereas doctors were more concerned about bleeding symptoms if this therapy was not initiated. Another study highlighted that many patients negatively perceive treatment-related fatigue.

Conclusion

Practical experience shows that earlier use of second-line drugs reduces the burden associated with corticosteroid administration and offers improved quality of life for patients. From this perspective, the rapid initiation of TPO-RA even in the



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Authors: prof. MUDr. Tomáš Kozák, Ph.D., MBA

Authors: prof. MUDr. Tomáš Kozák, Ph.D., MBA

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