Comment on: Use of thrombopoietin receptor agonist (romiplostim) in neonatal autoimmune thrombocytopenia due to maternal immune thrombocytopenia

2019 ◽  
Vol 66 (6) ◽  
pp. e27706 ◽  
Author(s):  
Upendra Mahat ◽  
Ravi Talati ◽  
Eric Kodish
Keyword(s):  
Receptor Agonist ◽  
Immune Thrombocytopenia ◽  
Autoimmune Thrombocytopenia ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist

Quality of Life Improvements Following Eltrombopag Treatment in Children with Chronic Immune Thrombocytopenia: Case Report

2021 ◽  
Vol 104 (4) ◽  
pp. 672-675
Keyword(s):  
Quality Of Life ◽  
Platelet Count ◽  
Receptor Agonist ◽  
Immune Thrombocytopenia ◽  
Case Series ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist ◽  
Low Platelet Count ◽  
Chronic Immune Thrombocytopenia

The present case series described six chronic immune thrombocytopenia patients (cITP), with a median age of 7.7 (7.0 to 13.0) years and low platelet count at 15,500 (7,000 to 20,000)/uL. They were suffering from bleeding symptoms and side effects of treatment. After enrollment, they were treated with thrombopoietin receptor agonist (eltrombopag). Five patients responded positively, showing a median platelet count of 115,000 (39,000 to 433,000)/uL. The median dose of eltrombopag used was 1.3 (0.8 to 2.2) mg/kg/day. The quality of life (QoL) improved for all patients, with their median overall score using a Pediatric QoL questionnaire showing 25.0% improvement. Median scores also showed improvements in each sphere of life functioning as physical (30.8%), emotional (26.4%), social (16.4%), and school (21.4%). The present report demonstrated that a select group of cITP patients, with low platelet count and bleeding symptoms, benefitted from treatment with eltrombopag, as shown by increased platelet counts and improved QoL. Keywords: Chronic ITP, Thrombopoietin receptor agonist, Children

scholarly journals Treatment of Immune Thrombocytopenia (ITP) with Eltrombopag - Results of the 4 th Interim Analysis of the German Non-Interventional Trial RISA

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3150-3150
Author(s):  
Oliver Meyer ◽  
Rudolf Schlag ◽  
Thomas Stauch ◽  
Bastian Fleischmann ◽  
Marcel Reiser ◽  
...  
Keyword(s):  
Platelet Count ◽  
Interim Analysis ◽  
Receptor Agonist ◽  
Immune Thrombocytopenia ◽  
Bleeding Complications ◽  
Bleeding Events ◽  
Interventional Trial ◽  
Platelet Counts ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist

Abstract Background: Immune thrombocytopenia (ITP) is an acquired autoimmune disorder with increased platelet destruction and impaired platelet production. Patients present with bleeding complications of various severity. Another common symptom of ITP is fatigue, which can severely affect patient's quality of life. Eltrombopag (EPAG) is an oral thrombopoietin receptor agonist, which is proved to be effective and safe in the treatment of ITP. In Europe, it is approved for the therapy of patients who were diagnosed with ITP at least 6 months ago and who have not responded to other treatments. Here we present data from the 4 th interim analysis of the RISA study. Methods: RISA is a prospective multicenter non-interventional trial in Germany. It was launched in December 2015, and it will be continued until December 2023. In accordance with the inclusion criteria, adults with persisting or chronic pITP (primary ITP) have been enrolled. Patients with pre-treatment could only be included if it was terminated 4 weeks prior to the patient's consent to participate in the study. Exclusion criteria comprised pregnancy, hepatitis C infection and severe aplastic anaemia. Dosage of EPAG and treatment of patients follows the SmPC and the routine of treating physicians. According to the study protocol, patient questionnaires must be completed at 0,1,3,6,9,12,18 and 24 months. Fatigue is assessed using the FACIT-F score, which includes a score range from 0 to 52, with score values <30 indicating severe fatigue. Statistical elaboration is predominantly descriptive. Calculations of confidence intervals and significance values are performed only for explorative purposes. Results: Data cutoff for this 4 th interim analysis was 23.02.2021. 275 patients were enrolled. 261 of them received at least one dose of EPAG and completed one post baseline assessment. Mean duration of participation was 5.2 years. Mean±SD age was 62.7±17.6 years. 54.8% of the patients were female. Median (range) duration of ITP at baseline was 5.3 (0.0-44.9) years. Comorbidity was present in 80.5% of all patients. 79 (28.7%) patients completed all scheduled visits before data cutoff. Median treatment duration was 395.0 days. Treatment with EPAG was carried out at a median dosage of 50 mg daily. In 255 patients, baseline platelet counts were available. The proportion of patients with a platelet count ≥50x10 9/L was 30.6% at baseline. With EPAG treatment, it increased to 75.4% within the first month (N=224) and to 89.0% within 24 months (N=73) from baseline. 12.6% of the patients who completed at least one assessment visit after baseline were pre-treated with the thrombopoietin receptor agonist romiplostim. Within this subgroup as well, platelet counts responded well to EPAG treatment. In 35.6% of patients, at least one bleeding event had occurred in the 12 months prior to baseline. During EPAG therapy, the incidence of bleeding events per patient year was reduced from 1.40 before baseline to 0.60 and 0.13 within the first and second treatment year respectively. This corresponds to a relative reduction in bleeding events of 57% and 91% respectively. Over the entire two years treatment period, the average incidence of bleeding events per patient year accounted for 0.44, which is 69% below the incidence at baseline. Bleeding events were mostly of low severity. (Tab.) Median FACIT-F score was 37.0 at baseline (N=202; mean 36.0±11.0) and 42.5 after 24 months (N=48; mean 38.1±12.1). This difference was not statistically significant. According to exploratory calculations, severity of fatigue was not correlated to platelet count, hemoglobin concentration or incidence of bleeding events. Discussion: In line with previously published randomized controlled trials (Birocchi et al. Platelets 2021), this non-interventional study confirmed the effectiveness of EPAG in adults with persistent or chronic ITP in a routine care setting. During treatment with EPAG, the prevalence and severity of thrombocytopenia, as well as the incidence of bleeding events, decreased. We could also confirm that fatigue is a significant issue in patients with ITP. A FACIT-F score of 37.0 is comparable to average score values in cancer patients (Montan et al. Value Health 2018). Under treatment with EPAG, we observed a decrease in fatigue that was clinically relevant but not statistically significant. Further research is needed to explore possible additional effects of EPAG, for example on fatigue. Figure 1 Figure 1. Disclosures Meyer: Swedish Orphan Biovitrum: Consultancy, Honoraria; Grifols: Consultancy, Honoraria; Amgen: Consultancy, Honoraria; Novartis: Consultancy, Honoraria. Stauch: Novartis: Honoraria, Research Funding; Amgen: Honoraria. Willy: Novartis Pharma: Current Employment.

scholarly journals Deciphering predictive factors for choice of thrombopoietin receptor agonist, treatment free responses, and thrombotic events in immune thrombocytopenia

2019 ◽  
Vol 9 (1) ◽  
Author(s):  
Maria L. Lozano ◽  
Maria E. Mingot-Castellano ◽  
María M. Perera ◽  
Isidro Jarque ◽  
Rosa M. Campos-Alvarez ◽  
...  
Keyword(s):  
Predictive Factors ◽  
Receptor Agonist ◽  
Immune Thrombocytopenia ◽  
Real World Data ◽  
Vascular Events ◽  
Appropriate Treatment ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist ◽  
Long Term Follow Up ◽  
Few Data

Abstract Very few data exist on when a particular thrombopoietin-receptor agonist (TPO-RA) is favored in clinical practice for the treatment of patients with immune thrombocytopenia (ITP), about novel risk factors for vascular events (VE) with these drugs, nor about predictive factors for therapy free responses (TFR). We conducted an observational, retrospective, long-term follow-up multicenter study from November 2016 to January 2018 of 121 adult ITP patients initiating TPO-RA between January 2012 to December 2014. Data reflected that a platelet count ≤25 × 109/l at the time when the TPO-RA was initiated was associated with a 2.8 higher probability of receiving romiplostim vs. eltrombopag (P = 0.010). VE on TPO-RA was related to previous neoplasia in patients over 65 years (50% vs. 2.2%, P < 0.001), and to previous splenectomy in younger patients (100% vs. 33%, P = 0.001). Receiving romiplostim as first TPO-RA with no subsequent TPO-RA switching was associated with a 50% likelihood of TFR after 2.9 years of therapy (3.3 years in chronic ITP patients). These real-world data help deciphering some areas of uncertainty, and offer insight into some of the most relevant challenges of ITP which may help clinicians make appropriate treatment decisions in the management of adult ITP patients with TPO-RA.

Safety and Efficacy of Eltrombopag in Children and Adults with Immune Thrombocytopenia: a Systematic Review and Meta-analysis.

2020 ◽  
Vol 18 ◽  
Author(s):  
Savvas Kolanis ◽  
Eleni Vasileiou ◽  
Emmanuel Hatzipantelis ◽  
Marina Economou ◽  
Athanasios Tragiannidis
Keyword(s):  
Receptor Agonist ◽  
Immune Thrombocytopenia ◽  
Meta Analysis ◽  
Efficacy And Safety ◽  
Primary Target ◽  
Rescue Treatment ◽  
Thrombopoietin Receptor ◽  
Number Of Patients ◽  
Thrombopoietin Receptor Agonist ◽  
Adults And Children

: Immune thrombocytopenia is an immune condition where antibodies are produced against platelets. Eltrombopag is a thrombopoietin receptor agonist that stimulates and promotes platelet production approved for treating thrombocytopenia in patients with chronic immune thrombocytopenia, where other treatments as corticosteroids, splenectomy or immunoglobulins are inadequate. The aim of this meta-analysis was to evaluate the efficacy and safety of the eltrombopag in adults and children with immune thrombocytopenia. We included 7 studies with a total of 765 patients (606 adults and 159 children). We evaluated the number of patients that achieved a post treatment platelet count equal or above 50x109 /L (primary result-target) without the need of rescue treatment for at least 4 weeks. Our data showed that patients who received eltrombopag were almost 4 times more probable in achieving the primary target when compared to patients that received placebo (RR 3.84, 95% CI 2.39 to 6.14; I2 = 46%). The number of patients that needed rescue treatment and the number of bleeding incidents were reduced in the group that received eltrombopag when compared to those who received placebo (RR 0.40, 95% CI 0.25 to 0.62; I2 = 40%) (RR 0.74, 95% CI 0.62 to 0.89; I2 = 68%). The total number of side effects did not statistically differ between the two groups (RR 0.99, 95% CI 0.90 to 1.08; I2 = 14%). Our findings were similar to previously published studies and confirm that eltrombopag is safe and efficient in immune thrombocytopenia. However more clinical trials are needed in order to enhance our findings.

scholarly journals Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia

2018 ◽  
Vol 183 (3) ◽  
pp. 479-490 ◽  
Author(s):  
Wojciech Jurczak ◽  
Krzysztof Chojnowski ◽  
Jiří Mayer ◽  
Katarzyna Krawczyk ◽  
Brian D. Jamieson ◽  
...  
Keyword(s):  
Receptor Agonist ◽  
Immune Thrombocytopenia ◽  
Phase 3 ◽  
Randomised Study ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist ◽  
Chronic Immune Thrombocytopenia

scholarly journals A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Heng Mei ◽  
Xiaofan Liu ◽  
Yan Li ◽  
Hu Zhou ◽  
Ying Feng ◽  
...  
Keyword(s):  
Tract Infection ◽  
Primary Endpoint ◽  
Receptor Agonist ◽  
Immune Thrombocytopenia ◽  
Phase Iii ◽  
Upper Respiratory Tract ◽  
Platelet Response ◽  
Double Blind ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist

Abstract Background Hetrombopag, a novel thrombopoietin receptor agonist, has been found in phase I studies to increase platelet counts and reduce bleeding risks in adults with immune thrombocytopenia (ITP). This phase III study aimed to evaluate the efficacy and safety of hetrombopag in ITP patients. Methods Patients who had not responded to or had relapsed after previous treatment were treated with an initial dosage of once-daily 2.5 or 5 mg hetrombopag (defined as the HETROM-2.5 or HETROM-5 group) or with matching placebo in a randomized, double-blind, 10-week treatment period. Patients who received placebo and completed 10 weeks of treatment switched to receive eltrombopag, and patients treated with hetrombopag in the double-blind period continued hetrombopag during the following open-label 14-week treatment. The primary endpoint was the proportion of responders (defined as those achieving a platelet count of ≥ 50 × 109/L) after 8 weeks of treatment. Results The primary endpoint was achieved by significantly more patients in the HETROM-2.5 (58.9%; odds ratio [OR] 25.97, 95% confidence interval [CI] 9.83–68.63; p < 0.0001) and HETROM-5 (64.3%; OR 32.81, 95% CI 12.39–86.87; p < 0.0001) group than in the Placebo group (5.9%). Hetrombopag was also superior to placebo in achieving a platelet response and in reducing the bleeding risk and use of rescue therapy throughout 8 weeks of treatment. The durable platelet response to hetrombopag was maintained throughout 24 weeks. The most common adverse events were upper respiratory tract infection (42.2%), urinary tract infection (17.1%), immune thrombocytopenic purpura (17.1%) and hematuria (15%) with 24-week hetrombopag treatment. Conclusions In ITP patients, hetrombopag is efficacious and well tolerated with a manageable safety profile. Trial registration Clinical trials.gov NCT03222843, registered July 19, 2017, retrospectively registered.

The burden of immune thrombocytopenia in adults: Evaluation of the thrombopoietin receptor agonist romiplostim

2012 ◽  
Vol 15 (5) ◽  
pp. 956-976 ◽  
Author(s):  
Robert Deuson ◽  
Mark Danese ◽  
Susan D. Mathias ◽  
Marieke Schoonen ◽  
Jon Fryzek
Keyword(s):  
Receptor Agonist ◽  
Immune Thrombocytopenia ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist
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