Spontaneous remission after a year of romiplostim in an adult patient with refractory primary immune thrombocytopenia

Perera, María; Suarez, Arturo; Luzardo, Hugo; Lopez, J Martínez; Molero, Teresa

doi:10.1007/s00277-012-1437-y

Cited by 6 publications

(7 citation statements)

References 3 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%

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%

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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“…While it was previously thought that patients with ITP would have to remain on TPO-RAs indefinitely to maintain adequate platelet counts, recent case reports and cohort studies have shown that selected patients with ITP can safely discontinue treatment. [7][8][9][10][11][12][13][14][15][16][17][26][27][28][29][30][31][32][33][34][35][36][37][38][39] However, to date, no formal guidelines identify which patients can successfully discontinue TPO-RAs. 4,20 In response to this need, we developed consensus statements on when it is appropriate to consider tapering TPO-RAs; broad guidance on how to taper patients off therapy, how to monitor patients after discontinuation, and how to restart therapy in the event of relapse is also provided.…”

Section: Discussionmentioning

confidence: 99%

“…No human subjects were involved in this research; thus, ethics committee approval was not required. [26][27][28][29][30][31][32][33][34][35][36][37] 11 cohort studies, [7][8][9][10][11][12][13][14][15][16][17] and two analyses of pooled clinical trial data 38,39 on sustained remission in patients with ITP after discontinuation of TPO-RAs. We did not formally appraise the quality of evidence using a standard tool.…”

Section: Study Design and Participantsmentioning

confidence: 99%

Tapering thrombopoietin receptor agonists in primary immune thrombocytopenia: Expert consensus based on the RAND/UCLA modified Delphi panel method

Cuker

Despotovic

Grace

et al. 2021

Research and Practice in Thrombosis and Haemostasis

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Background Thrombopoietin receptor agonists (TPO‐RAs) are used to treat primary immune thrombocytopenia (ITP). Some patients have discontinued treatment while maintaining a hemostatic platelet count. Objectives To develop expert consensus on when it is appropriate to consider tapering TPO‐RAs in ITP, how to taper patients off therapy, how to monitor patients after discontinuation, and how to restart therapy. Methods We used a RAND/UCLA modified Delphi panel method. Ratings were completed independently by each expert before and after a meeting. Second‐round ratings were used to develop the panel’s guidance. The panel was double‐blinded: The sponsor and nonchair experts did not know each other’s identities. Results Guidance on when it is appropriate to taper TPO‐RAs in children and adults was developed based on patient platelet count, history of bleeding, intensification of treatment, trauma risk, and use of anticoagulants/platelet inhibitors. For example, it is appropriate to taper TPO‐RAs in patients who have normal/above‐normal platelet counts, have no history of major bleeding, and have not required an intensification of treatment in the past 6 months; it is inappropriate to taper TPO‐RAs in patients with low platelet counts. Duration of ITP, months on TPO‐RA, or timing of platelet response to TPO‐RA did not have an impact on the panel’s guidance on appropriateness to taper. Guidance on how to taper patients off therapy, how to monitor patients after discontinuation, and how to restart therapy is also provided. Conclusion This guidance could support clinical decision making and the development of clinical trials that prospectively test the safety of tapering TPO‐RAs.

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“…In the last few years, some reports have suggested the possibility of maintaining the response after discontinuation of either romiplostim or eltrombopag, but the suspension criteria adopted by each single center have not been homogeneous (11,18,(26)(27)(28)(29)(30)(31)(32)(33)(34)(35)(36)(37)(38)(39)(40)(41)(42). Seven retrospective series of persisting response have been published by different authors (35,(37)(38)(39)(40)(41)(42).…”

Section: Discussionmentioning

confidence: 99%

“…In the last few years, some reports have suggested the possibility of maintaining the response, without other concomitant or rescue therapies, after discontinuation of either romiplostim or eltrombopag in 3-39% and 4-33% of patients, respectively (11,18,(26)(27)(28)(29)(30)(31)(32)(33)(34)(35)(36)(37)(38)(39)(40)(41)(42). These observations generated hypotheses about the mechanisms of action of the TPO-RAs.…”

mentioning

confidence: 99%

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

Mazzucconi

Santoro

Baldacci

et al. 2016

European J of Haematology

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Spontaneous remission after a year of romiplostim in an adult patient with refractory primary immune thrombocytopenia

Cited by 6 publications

References 3 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

Tapering thrombopoietin receptor agonists in primary immune thrombocytopenia: Expert consensus based on the RAND/UCLA modified Delphi panel method

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

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