Front-Line Treatment in Immune Thrombocytopenia Using Eltrombopag and High-Doses of Dexamethasone, Long-Term Follow-up

Introduction

Primary immune thrombocytopenia (ITP) is an autoimmune disease that results in platelet destruction as well as suppression in its production. There are a large number of therapeutic options, however, achieving a long-term and safe response has been the subject of study. As front-line therapies, gammaglobulin and corticosteroids are the most common treatments, however our group reported the use of eltrombopag and high doses of dexamethasone in the first line of treatment. In this study update we propose to inform the combined effectiveness of eltrombopag and dexamethasone at high doses as first line with a median follow-up of 4 years.

Material and methods

A prospective longitudinal single arm study was performed in patients with ITP at the University Hospital "Dr. José Eleuterio González "in Monterrey Mexico and of the Clinic Ruiz in Puebla, Mexico. Patients older than 18 years, with bleeding manifestations and platelet count <30 x 109 / L without previous treatment were included. Treatment consisted of eltrombopag (50 mg daily, day 1-28) in addition to dexamethasone (40 mg PO, day 1-4) on an outpatient basis. A platelet count> 30 x 10⁹ / L and complete response (CR)> 100 x 10⁹ / L was considered partial response (PR). Relapse-free survival was considered from the initial response day until the presence of relapse <30 x 10⁹ / L and the duration of the response as the maintenance period of the PR or CR.

Results

We included 9 patients from June 2012 to July 2013. The median follow-up was 41 months (10-51). At diagnosis, the median platelet count was 23.4 x 10⁹ (2-30) and at the end of follow-up was 89 x 10⁹ (65-282). Currently 5 patients (55.6%) maintained CR and 4 (44.4%) PR. Three patients relapsed during follow-up at a median of 7 months (1-14), of whom 2 achieved PR and 1 CR subsequently. The relapse free survival (RFS) was 64% at 2 years. There were no manifestations of bleeding, no events of myelofibrosis or venous thrombosis were recorded and treatment was well tolerated.

CONCLUSIONS

Combined eltrombopag and high-dose dexamethasone therapy is a viable first-line option in ITP that can generate sustained complete long-term response in adult patients. Clinical trials with larger numbers of samples are required, as well as randomized comparative studies.