Does Immune Thrombocytopenic Purpura (ITP) require lifelong treatment or can it go into remission?

From the Guidelines

Lifelong treatment is not always required for ITP, as some patients may achieve long-term remissions that are sustained off treatment, with a remission rate of nearly 30% in TPO-RA studies. The management of immune thrombocytopenia (ITP) is complex and depends on various factors, including platelet count, bleeding symptoms, and individual risk factors. According to a study published in the American Journal of Hematology in 2018 1, thrombopoietin receptor agonists (TPO-RAs) are approved for the treatment of patients with chronic ITP who had an insufficient response to either a first-line therapy or splenectomy.

Treatment Options

• First-line treatments typically include corticosteroids or intravenous immunoglobulin (IVIG)
• Second-line options include TPO-RAs (eltrombopag, romiplostim), rituximab, immunosuppressants (azathioprine, mycophenolate), or splenectomy
• TPO-RAs have been shown to increase platelet counts in 70%-80% of patients, and 85%-95% of these patients responded at least once in the long-term extension studies 1

Remission Rates

• The overall rate of long-term (6 months) remission in TPO-RA studies is nearly 30% 1
• Spontaneous remissions in adult patients are only about 9% 1
• The remission rate in TPO-RA studies is higher than the spontaneous remission rate, suggesting that TPO-RAs may have a disease-modifying activity

Monitoring and Treatment Plans

• Regular monitoring of platelet counts is necessary to adjust treatment plans
• Treatment decisions should be individualized based on platelet count, bleeding symptoms, and individual risk factors
• Patients should be monitored weekly for platelet counts until they are stabilized, and then as needed 1

From the Research

Treatment Overview

• Immune thrombocytopenia (ITP) is an autoimmune disease that requires treatment to manage symptoms and prevent bleeding complications 2, 3, 4, 5, 6.
• The goal of treatment is to increase platelet count and reduce the risk of bleeding, with the choice of treatment depending on the severity of symptoms and the patient's overall health 3, 5, 6.

Remission and Treatment Duration

• Some patients with ITP may experience spontaneous remission, with one study finding that approximately 9% of untreated patients achieved remission 2.
• Treatment with corticosteroids, intravenous immunoglobulin, or splenectomy can also induce remission in some patients, with sustained complete remission lasting more than 6 months achieved in 18.7% of patients in one study 2.
• However, many patients with ITP require ongoing treatment to manage their condition, with some patients experiencing refractory or relapsed disease despite treatment 4, 5, 6.

Treatment Options

• First-line treatment options for ITP include corticosteroids, intravenous immunoglobulin, and intravenous anti-RhD immune globulin, with the choice of treatment depending on the severity of symptoms and the need for rapid response 3, 5, 6.
• Second-line treatment options include thrombopoietin receptor agonists, rituximab, and splenectomy, with the choice of treatment depending on the patient's response to first-line treatment and their overall health 4, 5, 6.
• Third-line treatment options are available for patients with refractory or relapsed disease, including fostamatinib and other immunosuppressive agents 6.