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

Abstract

Sequential use of the TPO-RAs romiplostim and eltrombopag in ITP patients failing either agent was retrospectively evaluated to assess efficacy and impact of clinical characteristics on outcome. Patients were grouped into 5 categories: efficacy issues: 1 TPO-RA failure; loss of response; non-efficacy issues: platelet fluctuations; patient's preference; adverse event development. Either one TPO-RA sequence was analyzed at 3 month and at last follow-up. 106/546 patients on TPO-RA underwent switch and 65% achieved, regained or maintained a short- term response independent of switch sequence, gender or age; lower response rates were associated with lines of previous therapy; disease duration lowers probability to respond. Clinically, patients switched for efficacy issue did not differ from those switched for non-efficacy issues. Response was achieved/regained in 57.8% of patients switched for efficacy issues, the lowest response rates were observed in non-responders to 1 TPO-RA; 80% of patients switched for non-efficacy issues maintained a response. Platelet fluctuation resolved in 44.4%. Of the 49 patients evaluable for long-term outcome, 27 were in response on therapy; 16 discontinued the TPO-RA for reasons other than efficacy, while only 6 were non responders. We confirm the efficacy of TPO-RA switch; once achieved, response to the 2 TPO-RA seems durable.

A critical appraisal of the evidence for the role of splenectomy in adults and children with ITP

“…Whereas patients aged >65 years tend to obtain a better response in terms of platelet count, they appear to be burdened by a higher risk of severe adverse events, fatal adverse events and thromboembolic events compared to younger patients (Michel et al , ). Up to 10–30% of cases may experience a durable response off‐treatment for 6 months or more (Gonzalez‐Lopez et al , ; Newland et al , ; Cantoni et al , ). Furthermore, subjects not responsive or intolerant to one agent may switch to the second with substantial benefit (Cantoni et al , ).…”

“…Up to 10–30% of cases may experience a durable response off‐treatment for 6 months or more (Gonzalez‐Lopez et al , ; Newland et al , ; Cantoni et al , ). Furthermore, subjects not responsive or intolerant to one agent may switch to the second with substantial benefit (Cantoni et al , ).…”

Transitioning patients with immune thrombocytopenia to second‐line therapy: Challenges and best practices

“…Patients who do not respond to or do not tolerate a TPO‐RA may switch to the alternate TPO‐RA. The majority of patients who switch TPO‐RA treatment respond to the alternate TPO‐RA, even if the reason for switching was lack of efficacy with the initial TPO‐RA …”

“…Abrupt interruptions of TPO‐RAs or excessive dose adjustments may cause platelet fluctuations and should be avoided. Platelet fluctuations are more common with romiplostim and in that case could be resolved by switching to eltrombopag . Platelet fluctuations may also be more common in splenectomized patients, potentially due to the absence of normal splenic functions, including platelet sequestration …”

Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP)

“…Data on eltrombopag use in children are limited to single center experiences or studies conducted outside EMA approved indications (28)(29)(30). Recently, two "real life" retrospective Italian multicenter experiences explored the benefits of eltrombopag use in adults with chronic ITP underlying its potential role in delaying splenectomy (33,34).…”