Health‐related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care

Abstract

Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by platelet destruction and insufficient platelet production. The resulting thrombocytopenia reduces patient health-related quality of life (HRQOL). In a randomized, open-label, 52-week study of nonsplenectomized ITP patients treated with romiplostim or medical standard of care (SOC), patients completed the 10-scale ITP-patient assessment questionnaire (PAQ) at the start of the study and after 12, 24, 36, 48, and 52 weeks of treatment. HRQOL changes were examined for all patients in both treatment groups and by responder status, splenectomy status, and after the use of rituximab. Patients in both groups showed marked increases in all HRQOL scales over 52 weeks of treatment. These change scores exceeded the minimally important difference values (a measure of clinical relevance) for most of these scales, especially in responders to treatment. Compared with baseline, patients receiving romiplostim showed statistically significant improvements compared to SOC over 52 weeks for the ITP-PAQ scales of Symptoms, Bother, Activity, Psychological Health, Fear, Overall QOL, and Social QOL. Overall, treatment of ITP was associated with improvement in HRQOL. Patients receiving romiplostim had greater HRQOL improvements than those receiving SOC, but the magnitude of the difference is of uncertain clinical benefit.Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by persistent thrombocytopenia due to antibody binding to platelet antigen(s) causing their premature destruction by the reticuloendothelial system, particularly in the spleen [1]. In addition, recent evidence suggests that there is also insufficient platelet production, possibly immune-mediated [2]. The resulting thrombocytopenia places patients at risk for bruising and bleeding.Recent international practice guidelines recommend that initial ITP treatment include glucocorticoids, intravenous immunoglobulin (IVIg), or intravenous anti-D. Second-line treatment includes splenectomy or medical therapies such as azathioprine, danazol, rituximab, or the thrombopoietin (TPO) receptor agonists [3,4]. Many of these second-line treatments are associated with significant toxicities or cost [3,4].Romiplostim is a novel TPO receptor agonist that binds to and stimulates the TPO receptor. It has been shown to increase platelet counts with few adverse events [5]. In two randomized studies, an overall platelet response (weekly platelet counts 50 3 10 9 /L during 4 or more weeks of the 24-week study) was achieved by 83% of patients receiving romiplostim versus 7% of patients receiving placebo (P < 0.0001) [6]. In addition, 87% of romiplostim versus 38% of placebo patients were able to reduce or discontinue their concurrent ITP medication, including corticosteroids and IVIg [6]. Continuous long-term (up to 156 weeks) treatment with romiplostim produced a platelet response (platelet counts 50 3 10 9 /L) in 87% of 142 ITP patients [7]. Finally, romiplostim treatment led to fewer bleeding events, tran...

The efficacy and safety of romiplostim in adult patients with chronic immune thrombocytopenia

“…The overall results were better, with fewer experiencing treatment failure or proceeding to splenectomy with associated clinically important improvements in their ITP-PAQ scores. Statistically significant improvements in symptoms, bother, activity, psychological health, fear, social activity and overall HR-QOL were seen (p < 0.05) [Kuter et al 2010a[Kuter et al , 2012b, confirming the impact on quality of life in patients with thrombocytopenia and the importance to the patient in its reversal.…”

Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults

“…After dexamethasone treatment, 5 patients responded and 5 achieved CR. All patients achieved either R or CR by the time treatment with eltrombopag was complete (median 5 days, range [5][6][7][8][9][10][11][12][13][14][15][16][17][18][19]. The CR rate was 83.3%, whereas the R rate was 16.7%.…”

“…4 Corticosteroids remain the standard of care, but high failure/relapse rates and considerable adverse effects from long-term use continue to stimulate the search for better treatments. 3,5,6 We and others have reported sustained response rates ranging from 58% to 76% using rituximab plus dexamethasone as a frontline therapy. [7][8][9][10] Eltrombopag is a thrombopoietin (TPO) nonpeptide mimetic that has been shown to raise the Plt in both continued long-term administration and in a repeated short-term administration in chronic ITP.…”

A multicenter randomized open-label study of rituximab plus rhTPO vs rituximab in corticosteroid-resistant or relapsed ITP

“…32,33 The significantly shortened TTR in the combination group was likely to be attributed to the fast onset of rhTPO. 20,34 This result suggested that rhTPO could be used as a salvage treatment and combined with RTX in the treatment of ITP.…”