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

Abstract

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 secondline 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/L 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. (Blood. 2012;120(5):960-969)

Fostamatinib for Persistent/Chronic Adult Immune Thrombocytopenia

“…Furthermore, many patients experience unsustained responses regardless of treatment choice. Current guidelines for ITP are clearer for frontline treatment, but very uncertain for second line and beyond [75]. Thus, too great a proportion of patients have a significant unmet medical need because they cannot easily and safely manage their disease.…”

Splenectomy as a curative treatment for immune thrombocytopenia: a retrospective analysis of 233 patients with a minimum follow up of 10 years

“…For this reason, both patients and physicians are increasingly reluctant to proceed to splenec-haematologica | 2013; 98(6) Relapse-free survival tomy as second-line therapy, and there is still lively international debate on the therapeutic algorithm of patients failing first-line corticosteroid treatment. 6,8,21 In this study, we report the largest cohort of ITP patients that was observed for such a long period of time (minimum 10 years) after splenectomy, with a particular focus on stable responders. Unlike previously published experiences, in the present paper, response to treatments was rated and classified according to current guidelines and terminology; 1 this might explain some possible differences in patients' outcome.…”

“…Indeed, after rituximab administration, only 20-30% of patients maintain the response at five years, 12 with no conclusive data concerning the best timing of its use. 9,21,22 TPO-ra treatment is also effective but needs continuous administration. Moreover, no definitive safety data are available in the use of these agents over 5-6 years.…”

A retrospective pilot evaluation of switching thrombopoietic receptor-agonists in immune thrombocytopenia

“…[15][16][17][18][19] In view of these robust data, both drugs have been approved for adult chronic ITP in more than 80 countries and some groups consider them second-line treatment for chronic ITP. 5,20 However, in Europe, they are only authorized for use after splenectomy failure or when splenectomy is contraindicated.…”