preheader BJH 1

header website

Eltrombopag use in adult ITP patients from the UK Adult ITP Registry

The data presented by Provan and colleagues at EHA 2017 aimed to describe the adult patients with immune thrombocytopenic purpura (ITP) receiving eltrombopag. For this analysis, all patients receiving eltrombopag from the UK Adult ITP Registry were used. This registry involved more than 70 UK collaborating centres, coordinated by The Royal London Hospital. In particular, Provan and colleagues were interested in understanding the mean dose used, number of prior therapies, median length of treatment with eltrombopag, median counts at baseline before treatment and at six months following treatment, and sustained response in patients who had received eltrombopag.

Primary ITP is an autoimmune disorder associated with a reduced peripheral blood platelet count. Although many patients are relatively asymptomatic, many suffer with bruising, mucosal bleeding and quality of life issues. The standard initial treatment for ITP is oral corticosteroids and has remained unchanged for decades. Until recently, second-line therapy has been unsatisfactory, using empirical treatments. The recently approved thrombopoietin receptor agonists eltrombopag and romiplostim have transformed patient care and these agents are licensed second-line therapies in adults.

The total number of patients evaluated in this study was 129, among which 74 males (57.4%) and 55 females (42.6%). The median age at diagnosis was 49.4 years (26.9-66.4). Most patients had received prior ITP therapies; 10 patients (7.8%) had received one prior ITP therapy and 99 patients (77%) had received three or more prior therapies before starting eltrombopag. The most common prior therapies were corticosteroids in 110 patients (87%); IVIg in 91 patients (72%); rituximab in 68 patients (54%); romiplostim in 47 patients (37%); and immunosuppressants in 71 patients (56%). 29 patients (22.4%) had undergone prior splenectomy.

At baseline, prior to starting eltrombopag, the median platelet count was 21×109/L (and the majority of patients (64.5%) had platelets less than 30×109/L). The mean platelet count at 6 months was 206.2×109/L and at 1 year the level increased to 288×109/L. The median dose of eltrombopag used was 50mg/day and the median length of eltrombopag therapy was 14.7 weeks.

After initiation, 53 patients (41%) remained on eltrombopag as a monotherapy, whereas 27 patients (21%) had other ITP treatment concurrently with eltrombopag. Forty nine patients (38%) changed treatment after eltrombopag, of which prednisolone (47%), IVIg (33%), mycophenolate (18%), and rituximab (14%) were the most common ones, and 10% underwent a splenectomy.

The response to eltrombopag was assessed for 106 patients who had an adequate follow-up time and platelet counts. 81 patients (76%) had a response, of which 54 patients ( 51%) had platelet counts above 100x109/L and 27 patients (25%) had a partial response (defined as platelet counts between 30 to 100 x 109/L). Among those that had a response, 15 patients (14%) became unresponsive after some time, whereas 2 patients (2%) were unresponsive soon after a brief episode of response. In short, 64 patients (60%) had a sustained response to eltrombopag (among patients who remained or came off eltrombopag).

In summary, only 10 patients (7.8%) received eltrombopag as a second line therapy, whereas over 75% of the patients had received 3 or more prior therapies before starting eltrombopag despite its license as a second line therapy. Since 60% of the patients have a sustain response to eltrombopag, Provan and colleagues concluded their poster with the statement that as clinicians become more familiar with the use of eltrombopag, a greater proportion of patients are likely to receive eltrombopag as a second line therapy.


Provan D, Doobaree U, Newland A, et al. Primary ITP in adults treated with eltrombopag: a retrospective study using data from the United Kingdom adult immune thrombocytopenia registry. EHA2017, poster presentation, E1453


Speaker Drew Provan


Drew Provan, MD, Barts and The London School of Medicine and Dentistry, London, UK


See: Keyslides


Back to Top