Total Remission of Severe Immune Thrombocytopenia after Short Term Treatment with Romiplostim

Vlachaki, Efthymia; Papageorgiou, Vaia; Klonizakis, Filippos; Spandonidou, Maria; Chisan, Sofia; Vetsiou, Evaggelia; Ioannidou, Elissavet

doi:10.4081/hr.2011.e20

Cited by 14 publications

(22 citation statements)

References 16 publications

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“…A post hoc analysis [32] indicated that ITP duration ≤ 1 year could be a potential predictor for remission, although additional studies are needed to confirm this possibility. Similar results of sustained remissions after TPO-ra treatment and discontinuation have also been reported in several small case series of ITP patients [18–20, 22, 27–29, 33]. …”

Section: Discussionsupporting

confidence: 86%

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Sustained Remission in Patients with Primary Immune Thrombocytopenia after Romiplostim Tapering and Discontinuation: A Case Series in Real Life Management in Spain

Mingot‐Castellano

Grande-García

Valcárcel-Ferreiras

et al. 2017

Case Reports in Hematology

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Romiplostim, a thrombopoietin-receptor agonist (TPO-ra), is a highly effective option in primary immune thrombocytopenia (ITP), with 80–90% of patients achieving platelet responses after few weeks of treatment. The evidence showing remissions, that is, sustained platelet counts after romiplostim discontinuation, in patients with ITP refractory to immunosuppressive therapy is steadily increasing. However, there is a lack of guidelines or recommendations addressing how and when to taper romiplostim in clinical practice in patients maintaining elevated and stable platelet counts. Furthermore, given the high heterogeneity of ITP patients, no associated predictive factors have been currently identified. Here, we present 4 representative clinical cases of the daily clinical practice in Spain comprising newly diagnosed, persistent, and both splenectomized and nonsplenectomized chronic ITP patients treated with romiplostim, achieving and maintaining clinical remission (platelet count ≥ 50 × 109/L for 24 consecutive weeks in the absence of any treatment for ITP) after treatment tapering and discontinuation, without observed safety concerns. Prospective studies identifying clinical and biological predictive factors of sustained response are warranted.

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Section: Discussionsupporting

confidence: 86%

“…Several other reports had observed similar low doses in long-term remitters [18–20, 22, 24, 30], being comparatively higher in patients not achieving stable platelets counts and subsequent discontinuation [24]. However, a direct causality between administered doses and remission status was not established.…”

Section: Discussionmentioning

confidence: 95%

Sustained Remission in Patients with Primary Immune Thrombocytopenia after Romiplostim Tapering and Discontinuation: A Case Series in Real Life Management in Spain

Mingot‐Castellano

Grande-García

Valcárcel-Ferreiras

et al. 2017

Case Reports in Hematology

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“…The effect of eltrombopag is transient, and platelet counts usually return to baseline within 2 weeks unless treatment is continued [8][9][10], but anecdotal evidence suggests that a number of patients have discontinued eltrombopag and been able to maintain platelet counts above baseline values without additional treatment [11,12]. This effect has been also reported with romiplostim [13][14][15][16][17][18]. Short-and medium-term treatment with TPO-RA may avoid side effects and reduce the financial burden this costly treatment places upon healthcare systems.…”

Section: Introductionmentioning

confidence: 94%

Successful discontinuation of eltrombopag after complete remission in patients with primary immune thrombocytopenia

et al. 2015

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Eltrombopag is effective and safe in immune thrombocytopenia (ITP). Some patients may sustain their platelet response when treatment is withdrawn but the frequency of this phenomenon is unknown. We retrospectively evaluated 260 adult primary ITP patients (165 women and 95 men; median age, 62 years) treated with eltrombopag after a median time from diagnosis of 24 months. Among the 201 patients who achieved a complete remission (platelet count >100 3 10 9 /l), eltrombopag was discontinued in 80 patients. Reasons for eltrombopag discontinuation were: persistent response despite a reduction in dose over time (n = 33), platelet count >400 3 10 9 /l (n = 29), patient's request (n = 5), elevated aspartate aminotransferase (n = 3), diarrhea (n = 3), thrombosis (n = 3), and other reasons (n = 4). Of the 49 evaluable patients, 26 patients showed sustained response after discontinuing eltrombopag without additional ITP therapy, with a median follow-up of 9 (range, 6-25) months. These patients were characterized by a median time since ITP diagnosis of 46.5 months, with 4/26 having ITP < 1 year. Eleven patients were male and their median age was 59 years. They received a median of 4 previous treatment lines and 42% were splenectomized. No predictive factors of sustained response after eltrombopag withdrawal were identified. Platelet response following eltrombopag cessation may be sustained in an important percentage of adult primary ITP patients who achieved CR with eltrombopag. However, reliable markers for predicting which patients will have this response are needed.Am. J. Hematol. 90:E40-E43,

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“…Despite the plethora of therapeutic strategies in ITP, there is no evidence to guide an algorithm of treatment for recurrent or persistent ITP cases [4]. Recently, thrombopoietin receptor agonists, namely romiplostim [5,24,25] and eltrombopag [6,10,11,12], have been established as novel second-line therapies in the treatment of chronic ITP.…”

Section: Discussionmentioning

confidence: 99%

“…In recent years, an improved understanding of the pathophysiology of ITP has been achieved, consequently leading to new therapeutic options and strategies. It is now accepted that the disorder is due to both increased immune-mediated platelet destruction and decreased platelet production from megakaryocytes associated with endogenous thrombopoietin levels [3,4]. Therefore, thrombopoietin receptor agonists (romiplostim and eltrombopag) are new therapeutic modalities that have recently been licensed for the treatment of chronic ITP [5,6].…”

Section: Introductionmentioning

confidence: 99%

Is There a Role for Low-Dose Eltrombopag as Maintenance Therapy in the Treatment of Immune Thrombocytopenia?

et al. 2014

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Background/Aim: Thrombopoietin receptor agonists (romiplostim and eltrombopag) have recently been licensed for the treatment of thrombocytopenia in patients with chronic immune thrombocytopenia (ITP) with an insufficient response to corticosteroids, immunoglobulins or splenectomy. In the present case series, we present 4 nonresponding patients with chronic ITP who achieved maintenance of complete response (CR) for a period of at least 6 months on eltrombopag treatment administered in a modified regimen of 25 mg for 2, 3 or 5 days a week. Methods: The present study is a retrospective, nonconsecutive case series of 4 eltrombopag-treated patients with chronic ITP. Secondary ITP had been excluded in each patient, first-line therapy had failed and splenectomy had been refused. Furthermore, each patient was treated with eltrombopag, which resulted in a CR for a mean of 2 months. Consequently, decreased eltrombopag dosages have been able to maintain long-term CR. Results/Conclusion: Despite the low quality of evidence, our study results support the use of reduced-dose eltrombopag as a maintenance therapy after achieving CR. It seems a very promising strategy for the effective maintenance of response, improving health-related quality of life, lowering costs and possibly improving the safety in the treatment of ITP.

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Total Remission of Severe Immune Thrombocytopenia after Short Term Treatment with Romiplostim

Cited by 14 publications

References 16 publications

Sustained Remission in Patients with Primary Immune Thrombocytopenia after Romiplostim Tapering and Discontinuation: A Case Series in Real Life Management in Spain

Sustained Remission in Patients with Primary Immune Thrombocytopenia after Romiplostim Tapering and Discontinuation: A Case Series in Real Life Management in Spain

Successful discontinuation of eltrombopag after complete remission in patients with primary immune thrombocytopenia

Is There a Role for Low-Dose Eltrombopag as Maintenance Therapy in the Treatment of Immune Thrombocytopenia?

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