<scp>TPO</scp>‐<scp>RA</scp>s in <scp>pITP</scp>: description of a case series and analysis of predictive factors for response

Mazzucconi, Maria Gabriella; Santoro, Cristina; Baldacci, Erminia; Angelis, Federico De; Chisini, Marta; Ferrara, Grazia; Volpicelli, Paola; Foà, Roberto

doi:10.1111/ejh.12822

Cited by 16 publications

(19 citation statements)

References 41 publications

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“…In a single-center study, unlike the others, response (R) was defined as a platelet count C 30 9 10 3 / dL and at least a two fold increase in the baseline count and complete response (CR) as a platelet count C 100 9 10 3 / dL, in the absence of bleeding. In other studies performed, the most accepted response was a platelet count C 50 9 10 3 /dL [4,10,[13][14][15][16][17][18]. The results of our patients revealed that 11/19 (58%) of them reached the platelet count above 30,000/mm 3 while 6/19 (31%) patients were above 50,000/mm 3 .…”

Section: Discussionsupporting

confidence: 51%

Eltrombopag For Immune Thrombocytopenic Children in a Single Region

Leblebisatan

Kılınç

Çil

et al. 2018

Indian J Hematol Blood Transfus

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Objective Child patients of chronic thrombocytopenic purpura with severe and resistant thrombocytopenia were evaluated to observe whether their clinical or laboratory states improve by one of the thrombomimetic therapeutic agent called Eltrombopag as in adults in a single center of different country from previous studies. Materials and Methods Nineteen patients with chronic immune thrombocytopenia were treated with Eltrombopag to dose in international guidelines. Results Approximately half (11/19:58%) of the patients benefitted from the treatment with Eltrombopag either by an increase of platelet levels at safe levels with a decrease in the frequency of bleedings which needed rescue treatment. Conclusion Thrombomimetic treatment options have strengthened the clinician's hand where the regular treatment options became insufficient.

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Section: Discussionsupporting

confidence: 51%

Eltrombopag For Immune Thrombocytopenic Children in a Single Region

Leblebisatan

Kılınç

Çil

et al. 2018

Indian J Hematol Blood Transfus

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“…These TFR, which have been observed in up to 10–30% of ITP patients receiving these drugs 10 , raise the question of additional potential immunomodulatory properties by increasing the regulatory T and B cell compartment and decreasing platelet destruction 12,13 . To date, there are no predictors to identify in which patients this approach is likely to be successful, other than earlier start of TPO-RA (which may relate to the pathophysiology of the disease), and robust platelet responses 14–16 .…”

Section: Introductionmentioning

confidence: 99%

Deciphering predictive factors for choice of thrombopoietin receptor agonist, treatment free responses, and thrombotic events in immune thrombocytopenia

Lozano

Mingot‐Castellano

Perera

et al. 2019

Sci Rep

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Very few data exist on when a particular thrombopoietin-receptor agonist (TPO-RA) is favored in clinical practice for the treatment of patients with immune thrombocytopenia (ITP), about novel risk factors for vascular events (VE) with these drugs, nor about predictive factors for therapy free responses (TFR). We conducted an observational, retrospective, long-term follow-up multicenter study from November 2016 to January 2018 of 121 adult ITP patients initiating TPO-RA between January 2012 to December 2014. Data reflected that a platelet count ≤25 × 109/l at the time when the TPO-RA was initiated was associated with a 2.8 higher probability of receiving romiplostim vs. eltrombopag (P = 0.010). VE on TPO-RA was related to previous neoplasia in patients over 65 years (50% vs. 2.2%, P < 0.001), and to previous splenectomy in younger patients (100% vs. 33%, P = 0.001). Receiving romiplostim as first TPO-RA with no subsequent TPO-RA switching was associated with a 50% likelihood of TFR after 2.9 years of therapy (3.3 years in chronic ITP patients). These real-world data help deciphering some areas of uncertainty, and offer insight into some of the most relevant challenges of ITP which may help clinicians make appropriate treatment decisions in the management of adult ITP patients with TPO-RA.

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“…On the one hand, a recent study of 39 patients with ITP treated with TPO-RAs (eltrombopag was used in 11 of them), demonstrated that, after treatment discontinuation, 7 of them (18%) reached a sustained response, defined as the first assessed platelet count ⩾30 × 10 3 /µl available at more than 4 weeks after discontinuation. 61 Notably, this response was achieved in the absence of concomitant or rescue therapies. Among these patients, 71% of them had previously reached a durable response, which was defined as a response (platelet count ⩾30 × 10 3 / µl and at least a twofold increase as compared with the baseline count) or complete response (platelet count ⩾100 × 10 3 /µl with no bleeding) persisting for at least 4 weeks during the treatment period.…”

Section: Eltrombopag Discontinuationmentioning

confidence: 99%

Eltrombopag in immune thrombocytopenia: efficacy review and update on drug safety

González‐Porras

Bastida

2018

Therapeutic Advances in Drug Safety

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Immune thrombocytopenia (ITP) is an autoimmune disorder that induces a decrease in the number of circulating platelets due to spleen destruction and inability of megakaryocytes to restore normal counts. Immunosuppressive therapy with glucocorticoid drugs constitutes the first line of treatment. However, lack of response to these agents is not uncommon, and the management of refractory patients is a matter of controversy. In fact, day-to-day clinical practice shows that, in spite of the current guidelines, splenectomy, which is currently considered a suitable second-choice therapy, is being replaced by treatment with thrombopoietin receptor agonists. These boost platelet production by megakaryocytes. The use of one of these, namely eltrombopag, has been permitted for ITP patients refractory to first-line drugs or splenectomy, for the last 10 years. This review summarizes the experience reported using eltrombopag in ITP, paying attention to efficacy and safety. Results from clinical trials will be discussed, and studies performed in the course of daily clinical practice will also be reviewed, as these are useful to assess the potential of the drug in real-world settings. The management of adverse events and the use of eltrombopag in particular situations will also be covered. The experience reported so far permits us to suggest that eltrombopag efficiently induces recovery of platelet counts. Furthermore, recent papers have demonstrated that a sustained response after discontinuation, initially thought to be problematic, may be possible in a nonnegligible number of cases. The safety profile is satisfactory, although patients presenting with thromboembolism risk factors should be treated with caution until the eltrombopag-associated prothrombotic risk is fully established. In summary, although larger studies are still needed to clarify some issues, eltrombopag may be a useful alternative tool for ITP patients refractory to conventional medical management or splenectomy.

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TPO‐RAs in pITP: description of a case series and analysis of predictive factors for response

Abstract: CR was a significant prognostic factor for the achievement of a DR. Moreover, we observed a trend for DR patients to obtain a subsequent SR.

Cited by 16 publications

References 41 publications

Eltrombopag For Immune Thrombocytopenic Children in a Single Region

Eltrombopag For Immune Thrombocytopenic Children in a Single Region

Deciphering predictive factors for choice of thrombopoietin receptor agonist, treatment free responses, and thrombotic events in immune thrombocytopenia

Eltrombopag in immune thrombocytopenia: efficacy review and update on drug safety

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