2026-04-17T06:35:57Zhttps://recercat.cat/oai/request

oai:recercat.cat:11351/109702024-06-06T08:39:23Zcom_2072_378070com_2072_378040col_2072_378092

00925njm 22002777a 4500 dc Cooper, Nichola author Ghanima, Waleed author Vianelli, Nicola author yavasoglu, İrfan author Melikyan, Anait author VALCARCEL, DAVID author 2024-02-05T08:07:02Z 2024-02-05T08:07:02Z 2024-01 Immune thrombocytopenia; Relapsed; Steroids Trombocitopenia inmunitaria; Recaídas; Esteroides Trombocitopènia immune; Recaiguda; Esteroides Immune thrombocytopenia (ITP) is characterized by reduced platelet count due to increased destruction and is categorized according to the time following diagnosis (newly diagnosed, persistent, chronic). First-line corticosteroid therapy is associated with transient response, high relapse rates, and considerable toxicity. TAPER (NCT03524612) is a Phase II, prospective, single-arm trial investigating whether eltrombopag can induce a sustained response off-treatment (SRoT) in adult patients with ITP after first-line corticosteroid failure. This study defines SRoT as an off-treatment period wherein platelet count remains above 30 × 109/L in the absence of bleeding or rescue therapy. The primary endpoint was the proportion of patients who achieved SRoT until Month 12, which was 30.5% (n = 32/105; p < .0001 testing hypothesis H1: proportion >15%) following eltrombopag tapering and discontinuation, and median SRoT duration was ~8 months until Month 12. Median platelet count increased within 1 month of treatment and remained elevated until Month 12. Quality of life improved within 3 months and was maintained. Headache (21%) was the most common adverse event. None of the 4 deaths reported were considered treatment-related. In summary, ~one-third of patients achieved SRoT until Month 12 following eltrombopag tapering and discontinuation. An ad-hoc early-use analysis, stratified by ITP duration at baseline, assessed initial hematologic responses and safety. Results suggest that eltrombopag has similar efficacy in newly diagnosed and later stages of ITP. In follow-up until Month 24, a median SRoT duration of ~22 months was observed (n = 20). The safety profile was comparable across analyses and ITP duration groups and aligned with its well-established safety profile. Novartis Pharmaceuticals Corporation. http://hdl.handle.net/11351/10970 Trombocitopènia - Tractament Avaluació de resultats (Assistència sanitària) DISEASES::Hemic and Lymphatic Diseases::Hematologic Diseases::Blood Platelet Disorders::Thrombocytopenia CHEMICALS AND DRUGS::Amino Acids, Peptides, and Proteins::Proteins::Membrane Proteins::Receptors, Cell Surface::Receptors, Immunologic::Receptors, Cytokine::Receptors, Thrombopoietin Other subheadings::Other subheadings::Other subheadings::/agonists ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Diagnosis::Prognosis::Treatment Outcome ENFERMEDADES::enfermedades hematológicas y linfáticas::enfermedades hematológicas::trastornos de las plaquetas sanguíneas::trombocitopenia COMPUESTOS QUÍMICOS Y DROGAS::aminoácidos, péptidos y proteínas::proteínas::proteínas de membranas::receptores de superficie celular::receptores inmunológicos::receptores de citocinas::receptores de trombopoyetina Otros calificadores::Otros calificadores::Otros calificadores::/agonistas TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::diagnóstico::pronóstico::resultado del tratamiento Sustained response off-treatment in eltrombopag-treated adult patients with ITP who are refractory or relapsed after first-line steroids: Primary, final, and ad-hoc analyses of the Phase II TAPER trial