How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment

• Published in: Blood Vol. 120; no. 5; pp. 960 - 969
• Main Authors: Ghanima, Waleed, Godeau, Bertrand, Cines, Douglas B., Bussel, James B.
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 02.08.2012; Americain Society of Hematology
• Subjects: Adult; Algorithms; Biological and medical sciences; Chemotherapy, Adjuvant - adverse effects; Chemotherapy, Adjuvant - statistics & numerical data; Choice Behavior - physiology; Comorbidity; Contraindications; Decision Support Techniques; Hematologic and hematopoietic diseases; Humans; Immunosuppressive Agents - adverse effects; Immunosuppressive Agents - therapeutic use; Medical sciences; Platelet diseases and coagulopathies; Purpura, Thrombocytopenic, Idiopathic - diagnosis; Purpura, Thrombocytopenic, Idiopathic - epidemiology; Purpura, Thrombocytopenic, Idiopathic - mortality; Purpura, Thrombocytopenic, Idiopathic - therapy; Splenectomy - adverse effects; Splenectomy - statistics & numerical data; Medicine; Splenectomy; Thrombocytopenia; Platelet; Choice; Hematology; Surgery; Second line treatment; Hemopathy
• ISSN: 0006-4971; 1528-0020; 1528-0020
• DOI: 10.1182/blood-2011-12-309153

The paradigm for managing primary immune thrombocytopenia (ITP) in adults has changed with the advent of rituximab and thrombopoietin receptor agonists (TPO-RAs) as options for second-line therapy. Splenectomy continues to provide the highest cure rate (60%-70% at 5+ years). Nonetheless, splenectomy is invasive, irreversible, associated with postoperative complications, and its outcome is currently unpredictable, leading some physicians and patients toward postponement and use of alternative approaches. An important predicament is the lack of studies comparing second-line options to splenectomy and to each other. Furthermore, some adults will improve spontaneously within 1-2 years. Rituximab has been given to more than 1 million patients worldwide, is generally well tolerated, and its short-term toxicity is acceptable. In adults with ITP, 40% of patients are complete responders at one year and 20% remain responders at 3-5 years. Newer approaches to using rituximab are under study. TPO-RAs induce platelet counts > 50 000/μL in 60%-90% of adults with ITP, are well-tolerated, and show relatively little short-term toxicity. The fraction of TPO-RA–treated patients who will be treatment-free after 12-24 months of therapy is unknown but likely to be low. As each approach has advantages and disadvantages, treatment needs to be individualized, and patient participation in decision-making is paramount.