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

Abstract

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 Some patients may sustain their platelet response when treatment is withdrawn but the frequency of this phenomenon is unknown.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,

Eltrombopag for use in children with immune thrombocytopenia

“…One study of adult patients with chronic ITP showed that 53% of patients had a sustained remission after stopping eltrombopag at a median follow-up of 9 months. 39 Other studies of AA have also showed durable remissions after a course of treatment with eltrombopag. 46 The ITP Consortium of North America reported on the experience of children with chronic ITP who were receiving TPO-RAs, romiplostim, and eltrombopag.…”

“…At this time, it is not possible to predict which patients might experience relapse or have a sustained response after discontinuing eltrombopag treatment. 39 …”

Splenectomy for immune thrombocytopenia: down but not out

“…on May 9, 2018. by guest www.bloodjournal.org From able to discontinue these agents and maintain stable platelet counts. 88,89 Similar to rituximab, some of these remissions may reflect the natural history of ITP.…”

Is there still a place for “old therapies” in the management of immune thrombocytopenia?

“…Indeed, they represent 15% of treatments given during the first year following diagnosis of ITP [9]. Furthermore, this percentage will probably increase following the recent publications reporting prolonged remission after temporary use of TPO-RA in 10 to 30% of patients [10,11].…”