Retrospective study on the treatment of patients with primary immune thrombocytopenia with atrepopa / 中国现代医生

@#Objective To investigate the retrospective study of the treatment of primary immune thrombocytopenia(ITP)with Aitracopol.Methods Medical records of 90 patients with ITP who received treatment in our hospital from October 2018 to February 2022 were retrospectively analyzed and grouped according to different treatment methods,with an average n=45.Dexamethasone group was treated with dexamethasone group,and itrepopopa group was treated with dexamethasone group and itrepopopa group combined with dexamethasone group,all n=45.The immune function,therapeutic effect and adverse reactions were compared.Results At 4 weeks after treatment,the levels of interleukin(IL)-2 and interferon(INF)-γ in the Altropopar group and dexamethasone group were lower than before treatment,and the levels of IL-4 in the Altropopar group and dexamethasone group were higher than before treatment.Aitripopal group was higher than dexamethasone group(P<0.05);At 4 weeks after treatment,the proportions of platelet(PLT)≥30×109/L were 68.89%and 40.00%in the Altropopar group and dexamethasone group,and the proportions of PLT≥50×109/L were 55.55%and 20.00%,respectively.The proportions in the Altropopar group were higher than those in the dexamethasone group(P<0.05).The proportions of PLT≥50×109/L in the half time and dexamethasone groups were 66.67%and 33.33%,respectively,and the proportions of PLT≥50×109/L for at least one time were 93.33%and 44.44%,respectively.The proportions of PLT≥50×109/L at least once after 9 months of treatment were 60.00%and 26.67%,respectively,and the attripopal group was higher than dexamethasone group(P<0.05).A comparison of the adverse reaction rate between aitopopal group and dexamethasone group(P>0.05).Conclusion Aitracopal can significantly reduce the risk of bleeding in patients with ITP,improve immune function,improve therapeutic effect,and high safety.

Treatment of severe aplastic anemia and hematopoietic stem cell transplantation / 器官移植

Severe aplastic anemia (SAA) is a severe bone marrow failure syndrome caused by multiple causes, which is clinically manifested with severe anemia, infection and bleeding. The complex pathogenesis of SAA has not been fully understood. SAA is characterized with acute onset, severe disease condition and rapid progression. At present, with the in-depth study of SAA and the improvement of diagnosis and treatment, the therapeutic strategy for SAA has been evolved from classical immunosuppressive therapy based on antithymocyte globulin and cyclosporine to the application of thrombopoietin receptor agonist and combined treatment based on allogeneic hematopoietic stem cell transplantation, which may promote the reconstruction of hematopoietic function of SAA patients to varying degree and significantly improve survival and clinical prognosis, becoming the research hotspot of SAA treatment. In this article, new advances in the treatment of SAA at home and abroad were reviewed.

Maintenance of efficacy and its predictors after discontinuation of eltrombopag in adults with primary immune thrombocytopenia / 中华血液学杂志

Objective: To determine the efficacy of eltrombopag for primary immune thrombocytopenia (ITP) in adults and the predictive factors for treatment-free response (TFR) . Methods: Clinical data of adults with ITP who received eltrombopag from June 14, 2013 to May 31, 2021 in the Hematology Department of Ruijin Hospital affiliated with Shanghai Jiao Tong University Medical College were retrospectively analyzed. The initial dose of eltrombopag was 25 mg/d, and the maximum dose was 75 mg/d; the dose was adjusted to maintain the platelet count to within 50-150×10(9)/L. Treatment was discontinued according either to the protocol, on the patient's wishes or doctor's judgment (prescription medication), or based on clinical trials. The efficacy of eltrombopag and factors for TFR among patients who achieved complete response and those who discontinued treatment were analyzed. Results: Overall, 106 patients with ITP (33 men and 73 women) were included in the study. The median age of patients was 50 (18-89) years. There were 2, 10, and 94 cases of newly diagnosed, persistent, and chronic ITP, respectively. The complete response rate was 44.3% (47/106), the response rate was 34.0% (36/106), and the overall response rate was 78.3% (83/106). Meanwhile, 83 patients who responded to treatment discontinued eltrombopag; of these, 81 patients were evaluated. Additionally, 17 patients (21.0%) achieved TFR. The median follow-up duration of patients who achieved TFR was 126 (30-170) weeks. The recurrence rate was 17.6% (3/17), and the relapse-free survival rate was 76.5%. The results of univariate analysis revealed that non-recurrence after discontinuation of other treatments for ITP (P=0.001), and platelet count and eltrombopag dose of ≥100×10(9)/L (P=0.007) and ≤25 mg/d (P=0.031), respectively, upon discontinuation of eltrombopag were predictors of TFR; these effects were attributed to prolonged effective duration of eltrombopag. Multivariate analysis showed that there was a correlation between non-recurrence and prolonged effective duration after discontinuation of other treatments for ITP (P=0.002) . Conclusion: Eltrombopag is effective for patients with ITP as it can result in TFR. Predictors for TFR include non-recurrence after discontinuation of concomitant ITP treatment, and platelet count and eltrombopag dose of ≥100 × 10(9)/L and ≤25 mg/d upon discontinuation of treatment, respectively.

Effect of Eltrombopag on Response to Immunosuppressive Therapy in Patients with Transfusion-Dependent Non-Severe Aplastic Anemia / 中国实验血液学杂志

OBJECTIVE@#To compare the efficacy of eltrombopag combined with cyclosporine A (CsA) and CsA alone in patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA).@*METHODS@#The clinical data of 76 patients with treatment-naive TD-NSAA in Ningde Municipal Hospital of Ningde Normal University and Affiliated Hospital of Nantong University from December 2017 to June 2021 were retrospectively analyzed. Among them, 45 cases were treated with eltrombopag combined with CsA, and 31 patients with compatible baseline characters were treated with CsA alone. The efficacy of patients between the two groups was compared, and the factors affecting the curative effects were also analyzed.@*RESULTS@#There were significant differences in hematological response (HR) and complete response(CR) rates between the two groups at 3, 6, 12 months, and follow-up endpoint of treatment (P<0.05). With the prolongation of eltrombopag treatment time, the curative effect increased gradually, and the patients achieved more CR and HR rates by the end of the follow-up period. Simultaneously, with the increase in the maximum stable dose of eltrombopag, the HR rate increased gradually. The megakaryocyte count in eltrombopag group was higher than that in control at 6 and 12 months (P<0.05). Compared with the control group, the median time of platelet transfusion independence in eltrombopag group was more shorter (P=0.018), and the median platelets transfusion volume was lower (P=0.009). At 3, 6, 12 months after eltrombopag, the change of platelet in eltrombopag group was higher than that in the control group (P<0.05). Analysis of related factors affecting the efficacy showed that sex, age, iron overload, platelet count before treatment had no effect on the efficacy, and the median maximum stable dosage and the administration period for eltrombopag were related to the curative effect. The patients of eltrombopag group experienced adverse events of varying degrees, but the reactions were mild and mostly tolerated.@*CONCLUSION@#Eltrombopag can effectively improve the hematopoietic response and promote platelet recovery for TD-NSAA patients with relatively more residual hematopoietic cells, and it is safe and well tolerated.

Efficacy and safety of eltrombopag combined with immunosuppressive therapy in the treatment of severe aplastic anemia：a meta-analysis / 中国药房

OBJECTIVE To systematically evaluate the efficacy and safety of eltrombopag combined with immunosuppressive therapy （IST） for severe aplastic anemia （SAA）， and to provide evidence-based basis for clinical treatment of SAA. METHODS Retrieved from PubMed， Embase， Cochrane Library， ClinicalTrials.gov， VIP， CNKI and Wanfang data， randomized controlled trials （RCTs） and cohort studies about eltrombopag combined with IST （trial group） versus IST alone （control group） were collected from the inception to May 2023. After data extraction and quality evaluation （Cochrane manual 5.1.0） of included studies， meta-analysis， subgroup analysis， sensitivity analysis and publication bias analysis were performed by using RevMan 5.4 software. RESULTS A total of 12 studies were screened， including 1 344 patients. Compared with control group， objective remission rate （ORR） （RR＝1.34， 95%CI was 1.06-1.69， P＝0.01） and complete response rate （CRR） （RR＝1.88， 95%CI was 1.31-2.71， P＝ 0.000 6） at 3 months， ORR （RR＝1.33，95%CI was 1.23-1.43， P＜0.000 01） and CRR （RR＝1.88，95%CI was 1.57-2.25，P＜0.000 01） at 6 months were significantly increased in trial group. There was no statistically significant difference between the two groups in ORR （RR＝0.99， 95%CI was 0.82-1.18， P＝0.88） and CRR （RR＝1.02， 95%CI was 0.78-1.34， P＝0.87） at 12 months， two-year overall survival （OS） rate （HR＝0.61， 95%CI was 0.31-1.22， P＝0.17）， two-year event-free survival （EFS） rate （HR＝0.81， 95%CI was 0.61-1.07， P＝0.14）， clone evolution rate（RR＝1.01， 95%CI was 0.51-2.00， P＝ 0.98） or the incidence of adverse drug reactions such as liver/renal insufficiency， rash （P＞0.05）. Results of subgroup analysis showed that ORR and CRR of trial group at 6 months were higher than those of the control group in RCT and the cohort study subgroups （P＜0.05）. There was no statistically significant difference in the two-year OS rate， two-year EFS rate or clone evolution rate between trial group and control group in the two subgroups （P＞0.05）. The results of sensitivity analysis and publication bias analysis showed that the results of this study were robust and the possibility of publication bias was small. CONCLUSIONS The addition of eltrombopag in the IST regimen of SAA can improve the early hematological remission rate of patients， has no significant impact on short-term survival， and will not increase the occurrence of adverse drug reactions and clonal evolution.

Efficacy of eltrombopag on recurrent connective tissue disease related thrombocytopenia patients who failed to respond to rhTPO or recombinant human thrombopoietin: a report of 4 cases and literature review / 中华风湿病学杂志

Objective:To evaluate the efficacy and safety of eltrombopag standard therapy in the treatment of patients with connective tissue disease (CTD) associated with thrombocytopenia who were non-responder to recombinant human thrombopoietin (rhTPO) or relapsed after discontinuation of rhTPO.Methods:Retrospectively analysis of clinical data of 4 cases presented with CTD associated with thrombocytopenia who were non-responder to rhTPO or relapsed after discontinuation of rhTPO, and then treated with eltrombopag from November 2017 to June 2022. The literature were reviewed on eltrombopag in the treatment of CTD associated with thrombocytopenia.Results:The platelet count of 4 patients increased to more than 30×10 9/L after 2 weeks administration of eltrombopag. The median time was 32 (14, 41) days for platelet counts returning to normal level. The dosage of eltrombopag was gradually reduced to maintain normal platelet count, which was helpful for tapering of glucocorticoid and other immunosuppressant. No adverse event was observed during the treatment. Conclusion:Eltrombopag is safe and effective for CTD associated with thrombocytopenia, for patients who are non-responders to rhTPO or relapsed after discontinuation of rhTPO treatment in particular.

Mining and analysis of ADE signals of romiplostim and eltrombopag based on FAERS database / 中国药房

OBJECTIVE To provide a reference for the safe use of thrombopoietin receptor agonists romiplostim and eltrombopag in clinic. METHODS FDA adverse event reporting system （FAERS） in the United States was adopted to collect adverse drug event （ADE） reports of romiplostim and eltrombopag from their launch in the United States to September 30， 2022； the ADE signals of the two drugs were analyzed using the reporting odds ratio （ROR） method and the comprehensive standard method of the UK Medicines and Healthcare Products Regulatory Agency. RESULTS A total of 14 021 and 4 431 ADE reports were collected about romiplostim and eltrombopag， respectively， with a gender composition of more females than males. After signal screening， 563 ADE signals were obtained about romiplostim， involving 25 system organ classes （SOC）； eltrombopag had 433 ADE signals， involving 26 SOC. The most frequently reported ADE for both drugs was platelet count decreased （2 060， 1 585 cases）， which was mentioned in their drug instructions. In terms of signal intensity， romiplostim exhibited the highest signal for abnormal thrombopoietin levels （ROR of 2 268.85）， while eltrombopag had the highest signal for positive dengue virus test （ROR of 954.50）， with neither of these signals mentioned in their respective drug instructions. CONCLUSIONS The ADE of romiplostim and eltrombopag mainly affects the blood and lymphatic system， and there are many new suspicious high-risk signals.

Efficacy and Safety of Low Dose, Short Course Eltrombopag in Management of DF and DHF

Background: Dengue is the most widespread aedes mosquito borne viral disease which infects more than 50 million people every year. The clinical symptoms of dengue may vary from mild fever to life-threatening incidents. Eltrombopag, a non-peptide, oral TPO-R agonist, small molecular weight is quandaries with the transmembrane domain of a TPO receptor and persuades the Janus Kinase/Signal transducer and activator of transcription pathway, with a significant rise in platelet production.Material & Methods:This study was a cross-sectional observational study which was conducted at Tairunnesa Memorial Medical College and Hospital (TMMCH), Gazipur and Shin Shin Japan Hospital, Uttara, Dhaka. The study was conducted in between July 2021- December 2021. The sample size for this study was 100.Results:The mean age in group 1 was 25� in group 2 29�and in control group 29� The mean Baseline PLT * 109 /L for group 1 was 57�, for group 2, 51� and for control group 54�. Mean of systolic baseline BP (mmHg) for group 1 was 103.55� 5.04 for group 2 was 105.38�.34 and for control group was 101.97�28 and followed by the mean of diastolic baseline BP (mmHg) was 72.83�56, 72.84�.93 and 71.97�75. AEs was found in 4(12.1%) cases of group 1 where in group 2 it was 2(5.9%) and in control group was 3(9.1%). In day 7, the recovery rate of group 1 was 93.9% and in group 2 the recovery rate was 94.1%.Conclusions:Dengue is a vector-borne viral disease which needs medical assistance because it may lead to life-threatening outcome. Eltrombopag can be considered as a therapeutic option to increase the PLT counts in DF and DHF patients in the management of thrombocytopenia.

The efficacy of immunosuppression combined with Eltrombopag in the treatment of severe aplastic anemia in children / 中华实用儿科临床杂志

Objective:To explore the efficacy and clinical significance of Eltrombopag combined with immunosuppression therapy(IST) in the treatment of severe aplastic anemia (SAA) in children.Methods:Clinical data of 63 children with initially diagnosed SAA in the Department of Hematology of Xinxiang Central Hospital from May 2017 to June 2019 were retrospectively analyzed.All of them were all donors without siblings and they were classified into observation group (31 cases) and control group (32 cases). Patients in the observation group received IST combined with Eltrombopag treatment, and those in the control group received IST treatment.The Chi- square test was used to compare the overall remission (OR) rate and complete remission (CR) rate at 3 months, 6 months and 12 months, and incidence of infection and significant bleeding between groups.The t-test was used to compare the application of gra-nulocyte colony stimulating factor, the mean red blood cell transfusion, and the mean platelet infusion volume between groups.Kaplan-Meier method was adopted to analyze the 2-year overall survival (OS) rate and failure-free survival (FFS) rate, followed by the Log- rank test. Results:The 3-month and 6-month OR rate of the observation group were 61.29%(19/31 cases) and 80.64% (25/31 cases), respectively, which was significantly higher than that of the control group [37.50%(12/32 cases) and 59.38%(19/32 cases), χ2=45.27, 43.81, respectively, all P<0.05]. The 3-month and 6-month CR rate of the observation group were 32.26%(10/31 cases) and 45.16%(14 /31 cases), respectively, which was significantly higher than that of the control group [15.62%(5/32 cases) and 28.13%(9/32 cases), χ2=47.02, 48.35, respectively, all P<0.05]. The 12-month OR rate and CR rate in the observation group were 83.87%(26/31 cases), and 64.52%(20/31 cases), respectively, which were 81.25%(26/32 cases), and 59.38%(19/32 cases), respectively in the control group, and no significant differences in them were detected between the two groups (all P>0.05). The total amount of granulocyte colony stimulating factors [(13.58±4.28) doses vs.(23.24±6.68) doses, t=2.591], and the mean infusion volume of red blood cells [(5.48±1.67) U vs.(10.58±3.67) U, t=2.040] and platelets (4.15±2.47) bags vs.(9.15±3.87) bags, t=2.744) used in observation group within 6 months of treatment were significantly lower than those of the control group (all P<0.05). The rate of infection (16.13% vs.43.75%, χ2=47.12) and significant bleeding (16.13% vs.37.50%, χ2=44.52) in the observation group were significantly lower than those of the control group (all P<0.05). The 2-year OS rate of the observation group and control group were 93.55% (29/31 cases), and 87.50% (28/32 cases), respectively.No significant difference in the OS rate was found between groups ( P=0.407 3), nor as the 2-year FFS rate(87.10% vs.78.13%, P=0.326 6). Conclusions:IST combined with Eltrombopag can significantly improve the early treatment response rate of SAA children without a sibling identical donor, which can reduce red blood cell and platelet transfusion, and the incidence of infection and bleeding.

The efficacy of eltrombopag plus cyclosporine A in patients with transfusion-dependent non-severe aplastic anemia: a retrospective study from single center / 中华内科杂志

The main purpose of our study was to evaluate the efficacy and safety of eltrombopag plus cyclosporine A (CsA) in transfusion-dependent non-severe aplastic anemia(TD-NSAA). The clinical characteristics of 13 TD-NSAA patients who received initial treatment of eltrombopag plus CsA from 2019 to 2021 were retrospectively analyzed. The 3-month overall hematological response (OR) rate was 12/13. Until the end of follow-up, 12 patients responded, among whom 2 patients reached complete response (CR) and 9 patients reached partial response (PR) and 1 with HR. Paroxysmal nocturnal hemoglobinuria (PNH) developed in one patient at 6 months after treatment. Five of thirteen patients reported mild adverse reactions, which were all manageable. Compared with historical data, the combination of eltrombopag with CsA is an effective regimen in patients with TD-NSAA.

The efficacy and safety of eltrombopag in 13 children with chronic immune thrombocytopenia / 中国医师进修杂志

Objective:To explore the efficacy and safety of eltrombopag in children with chronic primary immune thrombocytopenia (cITP).Methods:The clinical data of 13 children with cITP in Children′s Hospital of Soochow University from July 2017 to December 2021 were retrospectively analyzed.Results:Among 13 children with cITP, 7 males and 6 females, the median age was 5 years old, the median duration of illness was 2.2 years. The median therapy duration of eltrombopag was 5 months, the median maintenance dose was 25.0 mg/d, the median time to response was 4 weeks; complete response was in 5 cases, partial response was in 2 cases; 2 cases had skin disease, 1 case had arthralgia, 1 case had liver function abnormal, all complications did not cause serious impact.Conclusions:Eltrombopag was an effective and safe therapeutic option for children with cITP.

Progress in the treatment of acquired aplastic anemia in children / 国际儿科学杂志

Aplastic anemia(AA) is a bone marrow failure disease in which peripheral blood is reduced in two or three lines.Clinically, patients are at high risk of bleeding and infection, which may endanger their lives in serious cases.Due to the great differences in economic conditions, medical conditions and AA epidemiology in various regions around the world, there are many treatment methods for newly diagnosed, refractory / relapsed AA patients and their curative effects are different.Haploidentical hematopoietic stem cell transplantation, eltrombopag and tacrolimus are the research focuses in recent years.To facilitate clinicians to choose more appropriate treatments, this paper reviews the progress of hematopoietic stem cell transplantation therapy and non-transplantation therapy in patients with AA.

Diagnoses, outcomes, and chronicity predictors of patients with secondary immune thrombocytopenia: ten-year data from a hematology referral center

ABSTRACT Background: Secondary immune thrombocytopenia (ITP) is a heterogeneous and unpredictable disease associated with various underlying conditions. Objective: The objective of the study was to investigate clinical evolution and chronicity predictors in secondary ITP. Methods: Patients treated at an academic medical center during 2008-2019 were stratified by age as children <16 years and adults >16 years. Responses to steroids, intravenous immunoglobulin G (IVIG), rituximab, and eltrombopag were classified as response (R) and complete response (CR). Risk factors for chronic ITP were determined by multiple regression with uni- and multi-variate analysis. Results: Eighty-three patients were included, 37 children and 46 adults. The most frequent associated conditions were infections 53%, systemic lupus erythematosus (SLE) 24%, thyroid disease 9.6%, and Evans syndrome 3.6%. Response to first-line treatment in the whole cohort was 94%; CR 45.7%; and R 50.6%. Initial response to steroids alone was 91.3% (n = 21/23), rituximab plus high-dose dexamethasone (HDD) 93.3% (n = 14/15); children receiving IVIG alone 100% (n=12/12); and eltrombopag in adults 100% (n = 3/3). Relapse was documented in 19.4% of children and 34% of adults, at a median time of 15 and 2 months, respectively; 30.4% of adults (15.2% from the miscellaneous group, 10.9% SLE-associated, and 4.3% infection-associated) and 18.9% of children followed a chronic course; age ≥10 years and platelets ≥20 × 109/L were risk factors for chronic ITP in children. Conclusion: Evolution was heterogeneous: a better and more sustained response was documented in the infections group compared to SLE or the miscellaneous group. (REV INVEST CLIN. 2021;73(1):31-8)

Anemia aplásica / Aplastic anemia

Resumen La pandemia de enfermedad por coronavirus 2019 (COVID-19) ha cambiado la perspectiva médica para el tratamiento de no solo de enfermedades hematológicas, sino en general de la medicina. Respecto a la anemia aplásica (AA), principalmente la muy severa, en la que el paciente se presenta con menos de 200 neutrófilos absolutos, el riesgo de infección potencialmente mortal es alta y el inicio de terapia inmunosupresora también representa un riesgo, al menos temporal, para COVID-19. Se ha recomendado incluso aplazar el trasplante de células progenitoras hematopoyéticas en muchos pacientes para evitar un contagio. Una inmunosupresión moderada preferentemente ambulatoria que incluya agentes trombomiméticos es la opción terapéutica en tiempos de la pandemia actual. En esta revisión se enlistan las recomendaciones internacionales y nacionales respecto al tratamiento y seguimiento de pacientes con AA con base en experiencias de países que ya han pasado por esta emergencia sanitaria.

Abstract Medical practice in general has changed due to coronavirus disease 2019 (COVID-19) pandemic. Some hematologic diseases require immunosuppresive therapy placing patients at high risk of infection, including severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Aplastic anemia (AA) especially the very severe type in which the count of absolute neutrophils is less than 200/ml is a life-threatening condition. Although bone marrow transplant is a potential curative treatment, it should be delayed temporally in order to prevent a contagion. Hospitalization may expose patients to infection, thus an ambulatory immunosuppression with oral cyclosporine and thrombopoietin agonist should be an adequate option. This work reviews international and national treatment recommendations and follow-up of patients with AA based on experiences from countries that have already faced this health emergency.

Discovery of the anti-angiogenesis effect of eltrombopag in breast cancer through targeting of HuR protein

HuR (human antigen R), an mRNA-binding protein responsible for poor prognosis in nearly all kinds of malignancies, is a potential anti-tumor target for drug development. While screening HuR inhibitors with a fluorescence polarization (FP) based high-throughput screening (HTS) system, the clinically used drug eltrombopag was identified. Activity of eltrombopag on molecular level was verified with FP, electrophoretic mobility shift assay (EMSA), simulation docking and surface plasmon resonance (SPR). Further, we showed that eltrombopag inhibited cell proliferation of multiple cancer cell lines and macrophages, and the anti-tumor activity was also demonstrated in a 4T1 tumor-bearing mouse model. The data showed that eltrombopag was efficient in reducing microvessels in tumor tissues. We then confirmed the HuR-dependent anti-angiogenesis effect of eltrombopag in 4T1 cells and RAW264.7 macrophages with qRT-PCR, HuR-overexpression and HuR-silencing assays, RNA stability assays, RNA immunoprecipitation and luciferase assays. Finally, we analyzed the anti-angiogenesis effect of eltrombopag on human umbilical vein endothelial cells (HUVECs) mediated by macrophages with cell scratch assay and Matrigel angiogenesis assay. With these data, we revealed the HuR-dependent anti-angiogenesis effect of eltrombopag in breast tumor, suggesting that the existing drug eltrombopag may be used as an anti-cancer drug.

Eltrombopag for the treatment of primary immune thrombocytopenia in 23 pediatric patients / 中华血液学杂志

Objective@#To evaluate the efficacy and safety of eltrombopag in the treatment of pediatric primary immune thrombocytopenia (ITP) .@*Methods@#The clinical characteristics of 23 pediatric ITP patients who received eltrombopag from May 2015 to March 2019 were retrospectively analyzed. Eltrombopag started with an initial dose of 12.5-50.0 mg/d and the maximum dose was 75.0 mg/d.@*Results@#Among 23 children, there were 11 boys and 12 girls with median age 11.0 (2.0-17.0) years. Four cases were newly diagnosed ITP, the other 8 of persistent ITP and 11 of chronic ITP. The duration of eltrombopag application ranged from 4.5 to 95 weeks (8/23 still ongoing) . The median platelet (PLT) counts at 2 weeks, 4 weeks, 3 months and the 6 months after treatment were 40 (4-170) ×109/L, 20 (4-130) ×109/L, 60 (4-110) ×109/L, and 70 (18-160) ×109/L, which were all significantly higher than that before treatment 14 (2-82) ×109/L (z=-3.440, P=0.001; z=-1.964, P=0.049; z=-4.339, P<0.001;z=-5.794, P<0.001 respectively) . The overall response rate was 60.87% (14/23 cases) . The median time to PLT count ≥30×109/L was 10.5 (3-42) days. Seven patients (30.43%) responded within the first week, and 10 cases (43.48%) achieved PLT counts ≥30×109/L within 2 weeks. All patients were divided into three groups according to the age (<6 years old, 6-12 years old, 13-17 years old) . The response rates were similar in three groups, as 33.33%, 60.00%, 85.71%, respectively. WHO bleeding scores as 0, 1, 2 were corresponded to 4, 12 and 7 patients before treatment. Patient numbers changed to 13, 7, 3 with bleeding scores 0, 1, 2 respectively after treatment (χ2=7.558, P=0.006) . Eltrombopag was well tolerated, the common adverse events included elevated transaminase (4 cases) and serum bilirubin (4 cases) ; mild nausea (1 case) , vomiting (1 case) and dizziness (1 case) . No drug withdrawal occurred due to adverse events.@*Conclusion@#Eltrombopag is safe and effective in pediatric patients with primary ITP.

Eltrombopag for the treatment of refractory acquired pure red cell aplasia / 中国实用内科杂志

OBJECTIVE: To investigate the efficacy of eltrombopag in the treatment of refractory acquired pure red cell aplasia(PRCA). METHODS: Three patients with refractory acquired PRCA treated in the First Affiliated Hospital of Nanjing Medical University/Jiangsu Province Hospital from March 2018 to May 2019 were treated with eltrombopag(75 mg/d). The clinical data were collected for evaluating efficacy and tolerance. RESULTS: The erythrocyte count(P=0.039), hemoglobin concentration(P=0.018) and reticulocyte percentage(P=0.046) in 3 patients were significantly higher than those before treatment. The platelet count was higher than that before treatment(P=0.024). The leukocyte count and absolute neutrophil count increased in 2 patients, and decreased in 1 patient, but still remained in the normal range(P=0.924; P =0.565). Total bilirubin(TBIL) and direct bilirubin(DBIL) increased in 1 case; alanine aminotransferase(ALT), aspartate aminotransferase(AST) and serum creatinine(Scr) increased in 1 case; palpitation occurred in 1 case. All the side effects were alleviated after symptomatic treatment. CONCLUSION: Eltrombopag has certain efficacy and good tolerance in the treatment of refractory acquired PRCA, which is worthy of further exploration.

Eltrombopag for the treatment of primary immune thrombocytopenia in 23 pediatric patients / 中华血液学杂志

Objective: To evaluate the efficacy and safety of eltrombopag in the treatment of pediatric primary immune thrombocytopenia (ITP) . Methods: The clinical characteristics of 23 pediatric ITP patients who received eltrombopag from May 2015 to March 2019 were retrospectively analyzed. Eltrombopag started with an initial dose of 12.5-50.0 mg/d and the maximum dose was 75.0 mg/d. Results: Among 23 children, there were 11 boys and 12 girls with median age 11.0 (2.0-17.0) years. Four cases were newly diagnosed ITP, the other 8 of persistent ITP and 11 of chronic ITP. The duration of eltrombopag application ranged from 4.5 to 95 weeks (8/23 still ongoing) . The median platelet (PLT) counts at 2 weeks, 4 weeks, 3 months and the 6 months after treatment were 40 (4-170) ×10(9)/L, 20 (4-130) ×10(9)/L, 60 (4-110) ×10(9)/L, and 70 (18-160) ×10(9)/L, which were all significantly higher than that before treatment 14 (2-82) ×10(9)/L (z=-3.440, P=0.001; z=-1.964, P=0.049; z=-4.339, P<0.001;z=-5.794, P<0.001 respectively) . The overall response rate was 60.87% (14/23 cases) . The median time to PLT count ≥30×10(9)/L was 10.5 (3-42) days. Seven patients (30.43%) responded within the first week, and 10 cases (43.48%) achieved PLT counts ≥30×10(9)/L within 2 weeks. All patients were divided into three groups according to the age (<6 years old, 6-12 years old, 13-17 years old) . The response rates were similar in three groups, as 33.33%, 60.00%, 85.71%, respectively. WHO bleeding scores as 0, 1, 2 were corresponded to 4, 12 and 7 patients before treatment. Patient numbers changed to 13, 7, 3 with bleeding scores 0, 1, 2 respectively after treatment (χ(2)=7.558, P=0.006) . Eltrombopag was well tolerated, the common adverse events included elevated transaminase (4 cases) and serum bilirubin (4 cases) ; mild nausea (1 case) , vomiting (1 case) and dizziness (1 case) . No drug withdrawal occurred due to adverse events. Conclusion: Eltrombopag is safe and effective in pediatric patients with primary ITP.

The efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia / 中华血液学杂志

Objective@#To investigate the safety and efficacy of eltrombopag for adult patients with chronic immune thrombocytopenia (cITP).@*Methods@#It was a randomised, single-centre, 6 weeks, placebo-controlled study. Beginning in January 29th, 2013, 35 patients were enrolled, and the trial was completed on May 16th, 2014. 17 patients were assigned to receive eltrombopag (starting dose 25 mg/d) and 18 were assigned to receive placebo.@*Results@#A total of 35 cases of adult cITP, 6 males and 29 females with a median age of 42(22-66) years were enrolled. One patient withdrew from eltrombopag treatment group for the adverse event (AE) and discontinued treatment. In first two weeks, 27.78% (5/18) of placebo-treated compared with 64.71%(11/17) of eltrombopag-treated patients achieved platelet counts ≥ 30×109/L(P=0.031); Treatment 6 weeks, the proportion of platelet counts reached ≥50×109/L and ≥ 30×109/L in eltrombopag-treated were higher than placebo-treated ones with statistically significant differences in both groups [64.71%(11/17) vs 11.11% (2/18), P=0.001; 76.47% (13/17) vs 38.89% (7/18), P=0.028]; The study also indicated a statistically significant difference in favour of eltrombopag compared with placebo in the odds of achieving the outcome of a platelet count ≥ 50×109/L at least once during 6-week treatment (94.11% vs 33.33%, P<0.001), and 70.59%(12/17) of patients with the platelet count continuously ≥ 50×109/L in 50% of treatment time in eltrombopag-treated group was more than placebo-treated one [11.11%(2/18), P<0.001]. Proportions of patients who required rescue treatment were 44.44% in placebo group and none in eltrombopag-treated one, respectively (P=0.002); The odds of bleeding symptoms with the WHO bleeding scale had no difference in both groups after 6 weeks (P=0.066). Adverse events that occurred more frequently due to eltrombopag than placebo included increased transaminase (3/17) and blood bilirubin (5/17), cerebral infarction(1/17).@*Conclusions@#The thrombopoietin receptor agonist eltrombopag was a suitable therapeutic option for Chinese adults with cITP.

The efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia / 中华血液学杂志

Objective: To investigate the safety and efficacy of eltrombopag for adult patients with chronic immune thrombocytopenia (cITP). Methods: It was a randomised, single-centre, 6 weeks, placebo-controlled study. Beginning in January 29(th), 2013, 35 patients were enrolled, and the trial was completed on May 16(th), 2014. 17 patients were assigned to receive eltrombopag (starting dose 25 mg/d) and 18 were assigned to receive placebo. Results: A total of 35 cases of adult cITP, 6 males and 29 females with a median age of 42(22-66) years were enrolled. One patient withdrew from eltrombopag treatment group for the adverse event (AE) and discontinued treatment. In first two weeks, 27.78% (5/18) of placebo-treated compared with 64.71%(11/17) of eltrombopag-treated patients achieved platelet counts ≥ 30×10(9)/L(P=0.031); Treatment 6 weeks, the proportion of platelet counts reached ≥50×10(9)/L and ≥ 30×10(9)/L in eltrombopag-treated were higher than placebo-treated ones with statistically significant differences in both groups [64.71%(11/17) vs 11.11% (2/18), P=0.001; 76.47% (13/17) vs 38.89% (7/18), P=0.028]; The study also indicated a statistically significant difference in favour of eltrombopag compared with placebo in the odds of achieving the outcome of a platelet count ≥ 50×10(9)/L at least once during 6-week treatment (94.11% vs 33.33%, P<0.001), and 70.59%(12/17) of patients with the platelet count continuously ≥ 50×10(9)/L in 50% of treatment time in eltrombopag-treated group was more than placebo-treated one [11.11%(2/18), P<0.001]. Proportions of patients who required rescue treatment were 44.44% in placebo group and none in eltrombopag-treated one, respectively (P=0.002); The odds of bleeding symptoms with the WHO bleeding scale had no difference in both groups after 6 weeks (P=0.066). Adverse events that occurred more frequently due to eltrombopag than placebo included increased transaminase (3/17) and blood bilirubin (5/17), cerebral infarction(1/17). Conclusions: The thrombopoietin receptor agonist eltrombopag was a suitable therapeutic option for Chinese adults with cITP.