Use of eltrombopag for secondary immune thrombocytopenia in clinical practice

Abstract

Eltrombopag is a second-line treatment in primary immune thrombocytopenia (ITP). However, its role in secondary ITP is unknown. We evaluated the efficacy and safety of eltrombopag in secondary ITP in daily clinical practice. Eighty-seven secondary ITP patients (46 with ITP secondary to autoimmune syndromes, 23 with ITP secondary to a neoplastic disease subtype: lymphoproliferative disorders [LPDs] and 18 with ITP secondary to viral infections) who had been treated with eltrombopag were retrospectively evaluated. Forty-four patients (38%) had a platelet response, including 40 (35%) with complete responses. Median time to platelet response was 15 days (95% confidence interval, 7-28 days), and was longer in the LPD-ITP group. Platelet response rate was significantly lower in the LPD-ITP than in other groups. However, having achieved response, there were no significant differences between the durable response of the groups. Forty-three patients (49·4%) experienced adverse events (mainly grade 1-2), the commonest being hepatobiliary laboratory abnormalities. There were 10 deaths in this case series, all of which were related to pre-existing medical conditions. In routine clinical practice, eltrombopag is effective and well-tolerated in unselected patients with ITP secondary to both immune and infectious disorders. However, the response rate in LPD-ITP is low.

Use of eltrombopag for patients 65 years old or older with immune thrombocytopenia

“…Probably, number of deaths of our case series is relatively high. However, given the elevated age of our population and its high number of comorbidities, we consider safety profile of eltrombopag in this scenario is good and comparable to our previous observations with younger cohorts . Similarly, prior publications confirm low overall survival in the elderly compared to younger patients …”

“…Our group was the first to publish a large clinical practice study of 87 secondary ITP patients treated with eltrombopag. We reported great evidence in favor of eltrombopag use in ITP secondary to both infectious and immune diseases . Nevertheless, we consider additional trials in this context are mandatory.…”

“…Our efficacy results in secondary ITP show high response rates in immune and infectious ITP with poor results in LPD‐ITP. Six deaths occurred in this cohort but only 2 were directly related to treatment failure (2 brain hemorrhages in an Evans syndrome and in a CLL patient, respectively, both very elderly patients). Therefore, here we demonstrate eltrombopag can also be effective in secondary ITP in elderly situations.…”

“…Nevertheless, in last years good few studies regarding eltrombopag use in primary ITP beyond clinical trials were published and increasing number of papers is available for elderly situations . Regarding secondary ITP, lack of clinical trials and shortage of publications outside of clinical trials avoid a clear demonstration of eltrombopag usefulness for this type of patients …”

Eltrombopag in long‐term management of pediatric thrombocytopenia

“…It is indicated in patients with chronic ITP and in adult patients with severe aplastic anemia, in whom it may help restore bone marrow cellularity and improve multilineage responses . In addition, eltrombopag has shown activity in a broad range of primary and secondary thrombocytopenias; studies in transplant, chemotherapy, hepatitis C virus‐associated thrombocytopenia, and certain hereditary thrombocytopenias have provided preliminary evidence for the efficacy of eltrombopag in these conditions …”