Treatment Options for Immune Thrombocytopenic Purpura (ITP)

Corticosteroids are the standard first-line therapy for ITP, with the addition of IVIG when rapid platelet count increase is required. 1, 2

First-Line Treatment Options

Prednisone (1 mg/kg orally for 21 days followed by tapering) is the primary first-line therapy for ITP, with initial response rates of 70-80%, though sustained responses occur in only 20-40% of cases 3
Dexamethasone shows high initial response rates up to 90% with sustained responses in 50-80% of patients when given in 1-4 cycles 1
High-dose methylprednisolone has shown response rates as high as 95% with faster response times 1
Intravenous immunoglobulin (IVIg) at 0.4 g/kg/day for 5 days or 1 g/kg/day for 1-2 days is indicated when rapid platelet increase is needed, producing responses in up to 80% of patients, many within 24 hours 1, 2
IV anti-D (50-75 μg/kg) can be used for Rh(D) positive, non-splenectomized patients as an alternative first-line option 3
Corticosteroids should be rapidly tapered and stopped in responders, and especially in non-responders after 4 weeks to avoid corticosteroid-related complications 1

Thrombopoietin Receptor Agonists (TPO-RAs) such as romiplostim are now recommended as the mainstay of long-term treatment for chronic ITP due to their high response rates, sustained efficacy, and favorable safety profile 3
Romiplostim has shown durable platelet responses in patients with ITP duration >1 year, with overall response rates of 88% in non-splenectomized and 79% in splenectomized patients 3, 4
Rituximab (anti-CD20 monoclonal antibody) achieves responses in 60% of patients with complete responses in 40% of patients, though long-term responses occur in only 20-30% of cases 3, 5
Splenectomy provides long-term responses in 60-70% of patients and has historically been considered the gold standard second-line therapy 1, 2, 3

Azathioprine (1-2 mg/kg daily) achieves responses in up to two-thirds of patients but may take 3-6 months for effect 3
Cyclosporin A (5 mg/kg/day initially, then 2.5-3 mg/kg/day) shows response rates of 50-80% with onset within 3-4 weeks 3
Cyclophosphamide (1-2 mg/kg orally daily or 0.3-1 g/m² IV every 2-4 weeks) produces responses in 24-85% of patients 3
Danazol (200 mg 2-4 times daily) achieves responses in up to 67% of patients but requires 3-6 months of treatment 3
Mycophenolate mofetil (1000 mg twice daily) achieves responses in up to 75% of patients within 4-6 weeks 3

High-dose corticosteroids combined with IVIg are recommended for emergency treatment of severe bleeding 1
Platelet transfusions at larger-than-usual doses may be considered in life-threatening situations 1

Treatment should be based primarily on bleeding symptoms rather than platelet count alone 3
Treatment is generally indicated for patients with platelet counts <20-30 × 10^9/L due to significantly greater bleeding risks 6
The goal is to maintain a hemostatic platelet count (>30-50 × 10^9/L) rather than normalizing platelet counts 3

Testing for HCV and HIV is recommended for all patients with suspected ITP 2
For HCV-associated ITP, antiviral therapy should be considered in the absence of contraindications 2
For HIV-associated ITP, treatment with antiviral therapy should be considered before other treatment options unless clinically significant bleeding is present 2
Eradication therapy is recommended for patients who test positive for H. pylori 1, 2

In pregnancy, corticosteroids or IVIg are recommended first-line treatments 1, 2
Mode of delivery should be based on obstetric indications rather than platelet count 2

Patients on TPO-RAs may be at higher risk of blood clots if platelet counts become too high during treatment 4
Romiplostim is used to maintain platelet counts around 50,000 per microliter to lower bleeding risk, not to normalize platelet counts 4
Abrupt interruptions or excessive dose adjustments of TPO-RAs may cause platelet fluctuations and should be avoided 3
Up to 30% of patients on TPO-RAs may achieve remission after tapering and discontinuation 3
Patients who do not respond to one TPO-RA may respond to an alternate TPO-RA 3

Prolonged corticosteroid use (>6-8 weeks) should be avoided due to significant side effects including weight gain, mood alterations, hypertension, diabetes, osteoporosis, and increased infection risk 3
Bone marrow examination is not necessary for patients presenting with typical ITP 2
TPO-RAs were previously thought to require lifelong administration, but evidence now shows up to 30% of patients may achieve remission after discontinuation 3
Patients requiring on-demand administration of corticosteroids after completing first-line treatment should be considered non-responders and promptly switched to second-line therapy 3