Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: A retrospective collaborative survey from Italian hematology centers

Background / Aim. The availability of thrombopoietin receptor agonists (TPO-RA) for the treatment of primary immune thrombocytopenia (ITP) over the last decade has transformed its management. The aim of this study was to assess the efficacy of TPO-RA in adults with chronic ITP treated in the University Clinical Centre of Serbia. Methods. A total of 28 adult ITP patients (10 males; 18 females) given eltrombopag and/or romiplostim were enrolled in the study. Data on demographic characteristics, ITP duration, previous therapeutic modalities, comorbidities, concomitant therapy, indications for TPO-RA, bleeding episodes, TPO-RA doses, adverse events and response rates were collected from the patients? medical records. TPO-RAs were administered: (a) in patients with chronic refractory ITP; (b) when splenectomy was contraindicated/unfeasible; (c) as preparation for splenectomy. A favourable treatment response was defined as a stable platelet count ? 50x109/L. Results. Twenty two (78.57%) and 14 (50.0%) subjects were treated with eltrombopag and romiplostim, respectively. A good treatment response (GTR) was achieved in 81.8% of the patients receiving eltrombopag and in 71.4% of those treated with romiplostim. The non-responders to eltrombopag (4 patients) and those who had lost their response to eltrombopag (4 patients) were switched to romiplostim. Six of them achieved a GTR. At the time of TPO-RA initiation, 46.4% of the patients used concomitant ITP therapy which was ceased in all those with a GTR. The following adverse effects of TPO-RA were registered: transaminitis and transient ischemic attack for eltrombopag - one patient each, and pulmonary embolism in one romiplostim treated patient. Conclusion. Our study showed that TPO-RAs are an effective and safe treatment option, since the majority of patients achieved stable remission without bleeding episodes.

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Exposure to non-corticosteroid treatments in adult primary immune thrombocytopenia before the chronic phase in the era of thrombopoietin receptor agonists in France. A nationwide population-based study

Autoimmunity Reviews ◽

10.1016/j.autrev.2014.10.017 ◽

2015 ◽

Vol 14 (2) ◽

pp. 168-173 ◽

Cited By ~ 13

Author(s):

Guillaume Moulis ◽

Maryse Lapeyre-Mestre ◽

Jean-Louis Montastruc ◽

Laurent Sailler

Keyword(s):

Immune Thrombocytopenia ◽

Chronic Phase ◽

Population Based ◽

Population Based Study ◽

Thrombopoietin Receptor Agonists ◽

Receptor Agonists ◽

Thrombopoietin Receptor ◽

Primary Immune Thrombocytopenia

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Second-line administration of thrombopoietin receptor agonists in immune thrombocytopenia: Italian Delphi-based consensus recommendations

Therapeutic Advances in Hematology ◽

10.1177/20406207211048361 ◽

2021 ◽

Vol 12 ◽

pp. 204062072110483

Author(s):

Monica Carpenedo ◽

Erminia Baldacci ◽

Claudia Baratè ◽

Alessandra Borchiellini ◽

Francesco Buccisano ◽

...

Keyword(s):

Platelet Count ◽

Delphi Technique ◽

Immune Thrombocytopenia ◽

Daily Practice ◽

Second Line ◽

Thrombopoietin Receptor Agonists ◽

Receptor Agonists ◽

Line Therapy ◽

Thrombopoietin Receptor ◽

Primary Immune Thrombocytopenia

Introduction: In patients with primary immune thrombocytopenia (ITP), a short course of steroids is routinely given as first-line therapy. However, the response is often transient and additional therapy is usually needed. Thrombopoietin receptor agonists (TPO-RAs) are frequently used as second-line therapy, although there is little clinical guidance on the timing of their administration and on tapering/discontinuation of the drug. To provide clinical recommendations, we used the Delphi technique to obtain consensus for statements regarding administration and on tapering/discontinuation of second-line TPO-RAs among a group of Italian clinicians with expertise in management of ITP. Methods: The Delphi process was used to obtain agreement on five statements regarding initiation and on tapering/discontinuation of second-line TPO-RAs. Agreement was considered when 75% of participants approved the statement. Eleven experts participated in the voting. Results: Full consensus was reached for three of the five statements. The experts held that an early switch from corticosteroids to a TPO-RA has the dual advantage of sparing patients from corticosteroid abuse and improve long-term clinical outcomes. All felt that dose reduction of TPO-RAs can be considered in patients with a stable response and platelet count >100 × 109/L that is maintained for at least 6 months in the absence of concomitant treatments, although there was less agreement in patients with a platelet count >50 × 109/L. Near consensus was reached regarding the statement that early treatment with a TPO-RA is associated with an increase in clinically significant partial or complete response. The experts also agreed that optimization of tapering and discontinuation of TPO-RA therapy in selected patients can improve the quality of life. Conclusion: The present consensus can help to provide guidance on use of TPO-RAs in daily practice in patients with ITP. Plain language summary Second-line administration of thrombopoietin receptor agonists in immune thrombocytopenia There is little guidance on the timing of administration and tapering/discontinuation of thrombopoietin receptor agonists (TPO-RAs) in patients with primary immune thrombocytopenia (ITP). The Delphi technique was used to obtain consensus for five statements. The present consensus among Italian clinicians aims to provide guidance on second-line use of TPO-RAs for patients with ITP in daily practice.

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