Eltrombopag in adult patients with immune thrombocytopenia in the real‐world in France, including off‐label use before 6 months of disease duration: The multicenter, prospective <scp>ELEXTRA</scp> study

Moulis, Guillaume; Germain, Johanne; Rueter, Manuela; Lafaurie, Margaux; Aroichane, Myriam; Mahévas, Matthieu; Viallard, Jean‐François; Chèze, Stéphane; Ebbo, Mikaël; Audia, Sylvain; Leclerc-Teffahi, S.; Sommet, Agnès; Michel, Marc; Godeau, Bertrand; Adoue, D.; Alric, Laurent; ANDRE, Baptiste; Arista, S.; Balardy, L.; Bonnet, Delphine; Bonnotte, Bernard; Beyne‐Rauzy, Odile; Borel, Cécile; Bréchemier, Delphine; Briantais, Antoine; Brun, Natacha; Carreiro, M.; Castel, Brice; Comont, T.; Cougoul, P.; COURTAULT, Carine; Danu, Alina; Delavigne, K.; Delbrel, X.; Deshayes, Samuel; Dingremont, C.; DION, Jérémie; Duffau, Pierre; Durand, J.M.; ESTELLE, Jean; Faucher, B.; Gaches, F.; Gaudin, C.; Giraud, P; GODEL‐LABOURET, Aurélie; Graveleau, J.; Hadj‐Khelifa, S.; Harlé, Jean‐Robert; Hébraud, Benjamin; HENNIQUE, Hélène; Khatibi, S; Lapeyre‐Mestre, Maryse; Laribi, Kamel; LEPLAY, Lorraine; Leveneur, Y.; Lifermann, François; Limal, Nicolas; Ollier, S.; Machelart, Irène; Madaule, S.; Maigné, G.; Martel, Clothilde; Martin‐Blondel, Guillaume; Michaud, Martin; Moeglin, J.; MOIGNET‐AUTREL, Aline; MOURGUET, Morgane; Roque, P. Montane De La; NUCCIO, Fanny; Orvain, Corentin; Prudhomme, Laurent; Pugnet, G.; Récher, Christian; Remy, Véronique; Rispal, P; Roy-Péaud, F.; Sailler, L.; SAINT‐LEZER, Arnaud; MARIE, Benjamin SAINTE; SAUNIER, Aurélie; Sauvètre, G.; Schleinitz, N.; Séguier, J.; Sire, Stéphane; Soubrier, C.; Swiader, L.; Tavitian, Suzanne; Urbanski, G.; Vaillant, Willy; Veit, V.

doi:10.1002/ajh.26404

Cited by 10 publications

(13 citation statements)

References 7 publications

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“…9 Previously published data from real-world clinical practice also suggest that eltrombopag is as effective and well tolerated in the early stages of ITP as it is in cITP. 12,13 We aimed to determine whether eltrombopag can induce SRoT in adult patients with ITP following CS failure. Here, we report the results of the 12-month TAPER primary analysis, follow-up analysis, and the results of an ad-hoc early-use analysis.…”

Section: Introductionmentioning

confidence: 99%

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

Cooper,

Ghanima,

Vianelli

et al. 2023

American J Hematol

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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.

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Section: Introductionmentioning

confidence: 99%

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

Cooper,

Ghanima,

Vianelli

et al. 2023

American J Hematol

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“…In the French ELEXTRA study, 95 of the 156 ITP patients (60.9%) were treated with eltrombopag before 6 months of disease duration. The overall response and complete response rates were similar between the two groups, with a slightly higher complete response rate in the <6 months group (72.9%) than in the ≥6 months group (66.7%) 26 . In the ELDERLY‐ITP‐TRA study, univariable analysis did not show that chronic phase of ITP at TPO‐RA start was associated with increased failure rate 25 .…”

Section: Thrombopoietin Receptor Agonistsmentioning

confidence: 86%

“…25 Likewise, the response rate to eltrombopag was found to be similar in terms of age and sex in the prospective ELEXTRA study that included 156 adult ITP patients from the CARMEN-France registry. 26 In children with ITP treated with TPO-RA, no difference in age was observed between the responders and non-responders in a retrospective multicentre study. 27…”

Section: Age and Sexmentioning

confidence: 93%

“…The overall response and complete response rates were similar between the two groups, with a slightly higher complete response rate in the <6 months group (72.9%) than in the ≥6 months group (66.7%). 26 In the ELDERLY-ITP-TRA study, univariable analysis did not show that chronic phase of ITP at TPO-RA start was associated with increased failure rate. 25 In the prospective phase II ESTIT study (eltrombopag second-line therapy in adult patients with primary ITP) that evaluated the efficacy of eltrombopag to achieve sustain remission off-treatment in 51 adults ITP non-responsive to or in relapse after first-line therapy, no difference was observed in terms of response or complete response at week 24 on treatment between patients with newly diagnosed and those with persistent ITP.…”

Section: Disease Durationmentioning

confidence: 93%

“…In the real‐world, observational retrospective study assessing the use of TPO‐RA in 384 patients with ITP aged ≥60 years (ELDERLY‐ITP‐TRA), older age (≥75 years) or male sex were not associated with increased failure rate to TPO‐RA treatment 25 . Likewise, the response rate to eltrombopag was found to be similar in terms of age and sex in the prospective ELEXTRA study that included 156 adult ITP patients from the CARMEN‐France registry 26 . In children with ITP treated with TPO‐RA, no difference in age was observed between the responders and non‐responders in a retrospective multicentre study 27 …”

Section: Thrombopoietin Receptor Agonistsmentioning

confidence: 94%

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Markers of refractory primary immune thrombocytopenia

Moulis,

Garabet

2023

Br J Haematol

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SummaryRefractory immune thrombocytopenia (ITP) is a challenging disease that can be defined by refractoriness to second‐line treatments. In this review, we list and comment available evidence about clinical and biological factors associated with refractoriness to splenectomy, thrombopoietin receptor agonists (TPO‐RAs), rituximab and fostamatinib, as well as those associated with multirefractory ITP (active disease with failure of rituximab, TPO‐RAs and splenectomy).

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Use of thrombopoietin receptor agonists in adults with immune thrombocytopenia: a systematic review and Central European expert consensus

et al. 2023

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There are currently three thrombopoietin receptor agonists (TPO-RAs) approved in Europe for treating patients with immune thrombocytopenia (ITP): romiplostim (Nplate®), eltrombopag (Revolade®), and avatrombopag (Doptelet®). However, comparative clinical data between these TPO-RAs are limited. Therefore, the purpose of this study was to perform a literature review and seek expert opinion on the relevance and strength of the evidence concerning the use of TPO-RAs in adults with ITP. A systematic search was conducted in PubMed and Embase within the last 10 years and until June 20, 2022. A total of 478 unique articles were retrieved and reviewed for relevance. The expert consensus panel comprised ITP senior hematologists from eight countries across Central Europe. The modified Delphi method, consisting of two survey rounds, a teleconference and email correspondence, was used to reach consensus. Forty articles met the relevancy criteria and are included as supporting evidence, including five meta-analyses analyzing all three European-licensed TPO-RAs and comprising a total of 31 unique randomized controlled trials (RCTs). Consensus was reached on seven statements for the second-line use of TPO-RAs in the management of adult ITP patients. In addition, the expert panel discussed TPO-RA treatment in chronic ITP patients with mild/moderate COVID-19 and ITP patients in the first-line setting but failed to reach consensus. This work will facilitate informed decision-making for healthcare providers treating adult ITP patients with TPO-RAs. However, further studies are needed on the use of TPO-RAs in the first-line setting and specific patient populations.

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Eltrombopag in adult patients with immune thrombocytopenia in the real‐world in France, including off‐label use before 6 months of disease duration: The multicenter, prospective ELEXTRA study

Cited by 10 publications

References 7 publications

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

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

Markers of refractory primary immune thrombocytopenia

Use of thrombopoietin receptor agonists in adults with immune thrombocytopenia: a systematic review and Central European expert consensus

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