What is the evaluation and treatment of Immune Thrombocytopenic Purpura (ITP) in adults?

From the Guidelines

Immune thrombocytopenia (ITP) in adults should be evaluated with a complete blood count, peripheral blood smear, and exclusion of other causes of thrombocytopenia before diagnosis, and treatment should be initiated with corticosteroids, such as prednisone 0.5-2.0 mg/kg per day, for 2-4 weeks followed by tapering, as recommended by the American Society of Hematology 2019 guidelines 1. The goal of treatment is to achieve a safe platelet count, rather than normalizing it completely, and to minimize bleeding risk.

• First-line therapy options include:
  • Corticosteroids, such as prednisone or dexamethasone, to rapidly increase platelet count and reduce bleeding risk 1
  • Intravenous immunoglobulin (IVIG) for emergency situations requiring rapid platelet increase 1
• If patients fail to respond or relapse after first-line treatment, second-line options include:
  • Thrombopoietin receptor agonists (TPO-RAs), such as eltrombopag or romiplostim, which have a safer profile compared to corticosteroids and can be used for long-term management 1
  • Rituximab, which can be effective for patients who are corticosteroid-dependent or unresponsive to corticosteroids 1
  • Splenectomy, which can be considered for patients who are refractory to medical therapy or have a high bleeding risk 1 Treatment decisions should be individualized based on bleeding risk, comorbidities, and patient preferences, and patients should be monitored regularly for treatment response.
• Lifestyle modifications, such as avoiding activities with high bleeding risk and medications that affect platelet function, like NSAIDs and aspirin, are also important for managing ITP. The pathophysiology of ITP involves autoantibodies targeting platelets, leading to their premature destruction in the spleen and impaired platelet production, and understanding this mechanism can help guide treatment decisions 1. Overall, the management of ITP in adults requires a comprehensive approach that takes into account the patient's individual needs and preferences, and aims to achieve a safe platelet count and minimize bleeding risk.

From the FDA Drug Label

Nplate is indicated for the treatment of thrombocytopenia in: Adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy The effectiveness of ALVAIZ has been established based on adequate and well-controlled studies of eltrombopag olamine in adult and pediatric patients 6 years and older with persistent or chronic ITP Use the lowest dose of Nplate to achieve and maintain a platelet count ≥ 50 × 10^9/L as necessary to reduce the risk for bleeding. The initial dose of Nplate is 1 mcg/kg Actual body weight at initiation of treatment should always be used when calculating the initial dose. Adjust the weekly dose of Nplate by increments of 1 mcg/kg until the patient achieves a platelet count ≥ 50 × 10^9/L as necessary to reduce the risk for bleeding; do not exceed a maximum weekly dose of 10 mcg/kg

Evaluation and Treatment of ITP in Adults:

• Indications: Romiplostim (Nplate) and eltrombopag (ALVAIZ) are indicated for the treatment of thrombocytopenia in adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
• Dosing: The initial dose of Nplate is 1 mcg/kg, and the dose should be adjusted by increments of 1 mcg/kg until the patient achieves a platelet count ≥ 50 × 10^9/L.
• Treatment Goals: The goal of treatment is to achieve and maintain a platelet count ≥ 50 × 10^9/L to reduce the risk of bleeding.
• Key Considerations:
  • Nplate should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increases the risk for bleeding.
  • Nplate should not be used in an attempt to normalize platelet counts.
  • Eltrombopag has been shown to be effective in increasing platelet counts in adult patients with persistent or chronic ITP. 2 3 2

From the Research

Evaluation of ITP in Adults

• The diagnosis of ITP remains one of exclusion, despite the ability to detect anti-platelet antibodies 4
• Bleeding, not platelet count, should be the rationale for treatment 4
• The standard initial treatment for ITP is oral corticosteroids to increase platelet counts 4, 5

Treatment Options for ITP in Adults

• First-line therapies include corticosteroids, intravenous immunoglobulin, and anti-RhD immune globulin 4, 5, 6
• Second-line therapies include thrombopoietin receptor agonists (TPO-RAs), rituximab, and splenectomy 4, 5, 6
• Combination therapy with eltrombopag and rituximab may be effective for steroid-refractory chronic ITP 7
• Other treatments, such as danazol, azathioprine, cyclophosphamide, vinca alkaloids, and cyclosporin A, have limited evidence of efficacy and should be reserved for symptomatic thrombocytopenia after refractoriness to first-line therapies has been established 5

Considerations for Treatment

• Treatment should be individualized based on the extent of thrombocytopenia and symptoms of disease 8
• Minimizing toxicity associated with treatment while achieving hemostatic platelet counts is essential 8
• The occurrence of bleeding manifestations, severe thrombocytopenia, and requirement for invasive procedures are among the reasons necessitating initiation of therapy 6