Chronic primary immune thrombocytopenia (ITP) can today benefit from multiple therapeutic approaches with proven clinical efficacy, including rituximab, thrombopoietin receptor agonists (TPO-RA), and splenectomy. However, some ITP patients are unresponsive to multiple lines of therapy with prolonged and severe thrombocytopenia. The diagnosis of refractory ITP is mainly performed by exclusion of other disorders and is based on the clinician's expertise. However, it significantly increases the risk of drug-related toxicity and of bleedings, including life-threatening events. The management of refractory ITP remains a major clinical challenge. Here, we provide an overview of the currently available treatment options, and we discuss the emerging rationale of new therapeutic approaches and their strategic combination. Particularly, combination strategies may target multiple pathogenetic mechanisms and trigger additive or synergistic effects. A series of best practices arising both from published studies and from real-life clinical experience is also included, aiming to optimize the management of refractory ITP.

Refractory primary immune thrombocytopenia (ITP): current clinical challenges and therapeutic perspectives / N. Vianelli, G. Auteri, F. Buccisano, V. Carrai, E. Baldacci, C. Clissa, D. Bartoletti, G. Giuffrida, D. Magro, E. Rivolti, D. Esposito, G.M. Podda, F. Palandri. - In: ANNALS OF HEMATOLOGY. - ISSN 0939-5555. - 101:5(2022 May), pp. 963-978. [Epub ahead of print] [10.1007/s00277-022-04786-y]

Refractory primary immune thrombocytopenia (ITP): current clinical challenges and therapeutic perspectives

G.M. Podda;
2022

Abstract

Chronic primary immune thrombocytopenia (ITP) can today benefit from multiple therapeutic approaches with proven clinical efficacy, including rituximab, thrombopoietin receptor agonists (TPO-RA), and splenectomy. However, some ITP patients are unresponsive to multiple lines of therapy with prolonged and severe thrombocytopenia. The diagnosis of refractory ITP is mainly performed by exclusion of other disorders and is based on the clinician's expertise. However, it significantly increases the risk of drug-related toxicity and of bleedings, including life-threatening events. The management of refractory ITP remains a major clinical challenge. Here, we provide an overview of the currently available treatment options, and we discuss the emerging rationale of new therapeutic approaches and their strategic combination. Particularly, combination strategies may target multiple pathogenetic mechanisms and trigger additive or synergistic effects. A series of best practices arising both from published studies and from real-life clinical experience is also included, aiming to optimize the management of refractory ITP.
Combination therapy; Immune thrombocytopenia (ITP); Real-life clinical practice; Refractory ITP; Rituximab; Thrombopoietin receptor agonists (TPO-RA); Humans; Receptors, Fc; Receptors, Thrombopoietin; Recombinant Fusion Proteins; Rituximab; Splenectomy; Thrombopoietin; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
Settore MED/09 - Medicina Interna
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/936153
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