Multicentre, randomised phase <scp>III</scp> study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia

Yang, Renchi; Jm, Li; Jin, Jie; Huang, Meijuan; Yu, Ziqiang; Xu, Xiaojun; Zhang, Xiaohui; Hou, Ming

doi:10.1111/bjh.14380

Cited by 55 publications

(67 citation statements)

References 17 publications

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“…[13][14][15] TPO-RAs, romiplostim 16 and eltrombopag, 17 are approved for use in chronic ITP in adults in whom ITP is refractory to other treatments. As shown in randomised double-blind trials, [16][17][18][19][20][21][22] TPO-RAs stimulate megakaryopoiesis and increase platelet counts, resulting in fewer bleeding episodes and reduction in rescue medication use. Longterm responses in patients who are no longer receiving TPO-RAs (ie sustained remission) have been reported in the range of 10%-32% of patients in clinical trials 22 and observational studies.…”

Section: Introductionmentioning

confidence: 92%

Primary immune thrombocytopenia (ITP) treated with romiplostim in routine clinical practice: retrospective study from the United Kingdom ITP Registry

Doobaree

Newland

McDonald

et al. 2019

European J of Haematology

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BackgroundRomiplostim is a thrombopoietin‐mimetic peptibody for adult refractory chronic immune thrombocytopenia (ITP). We aimed to describe ITP patients receiving romiplostim, platelet counts and romiplostim usage in UK clinical practice.MethodsThis was a retrospective cohort study of patients in the UKITP Registry who received romiplostim between October 2009 and January 2015, including data up to 6 months before romiplostim initiation through follow‐up.ResultsOf 1440 patients in the UKITP Registry, 118 adults with primary ITP were eligible. Before romiplostim, 22% had splenectomy, 12% received platelet transfusion, 97% received ≥ 1 different ITP medication and 77% received ≥ 3. Most patients (73%) initiated romiplostim ≥ 1 year after ITP diagnosis (chronic phase). The mean duration of romiplostim treatment was 5.7 (SE 0.9) months, and the median was 1.4 months (IQR: 0.2, 6.5). Mean platelet count before romiplostim was 38 × 109/L, rising to 103 × 109/L within 1 month, and remaining 50‐150 × 109/L through up to 3 years of follow‐up. After romiplostim, 4% of patients had splenectomy, 6% received platelet transfusion, and 57% received just one ITP medication other than romiplostim.ConclusionThe study provides valuable insights into the real‐world use of romiplostim in primary ITP in routine practice and highlighted the timing of romiplostim initiation at different ITP disease phases.

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Section: Introductionmentioning

confidence: 92%

Primary immune thrombocytopenia (ITP) treated with romiplostim in routine clinical practice: retrospective study from the United Kingdom ITP Registry

Doobaree

Newland

McDonald

et al. 2019

European J of Haematology

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“…All studies were RCTs and mainly multi‐centre, except for one (Shirasugi et al , ), with sample sizes ranging from 21 to 234. All studies were two‐arm comparisons, including 5 studies (Bussel et al , ; Bussel et al , ; Cheng et al , ; Tomiyama et al , ; Yang et al , ) for eltrombopag versus placebo, 4 (Bussel et al , ; Kuter et al , ; Kuter et al , ; Shirasugi et al , ) for romiplostim versus placebo, 2 (Arnold et al , ; Ghanima et al , ) for rituximab versus placebo, 1 (Cui et al , ) for rhTPO+ciclosporin versus rhTPO, 1 (Zhou et al , ) for rhTPO+rituximab versus rituximab and 1 (Wang et al , ) for rhTPO+danazol versus danazol. Nine RCTs (Bussel et al , ; Bussel et al , ; Bussel et al , ; Cheng et al , ; Shirasugi et al , ; Tomiyama et al , ; Cui et al , ; Zhou et al , ; Yang et al , ) included exclusively patients with persistent ITP, while 4 (Kuter et al , ; Kuter et al , ; Arnold et al , ; Ghanima et al , ) included mixed newly diagnosed and persistent ITP patients and 1 (Wang et al , ) did not mention ITP phase.…”

Section: Resultsmentioning

confidence: 99%

“…Median platelet count at baseline ranged from 10 × 10 9 /l to 29 × 10 9 /l. Platelet response was defined as platelet ≥50 × 10 9 /l in 11 studies (Bussel et al , ; Bussel et al , ; Kuter et al , ; Bussel et al , ; Kuter et al , ; Cheng et al , ; Shirasugi et al , ; Arnold et al , ; Tomiyama et al , ; Wang et al , ; Yang et al , ), and ≥30 × 10 9 /l in 3 studies (Cui et al , ; Ghanima et al , ; Zhou et al , ). The treatment duration ranged from 2 to 52 weeks (median = 6 weeks), while the follow‐up period ranged from 4 to 78 weeks (median = 24 weeks).…”

Section: Resultsmentioning

confidence: 99%

“…The treatment duration ranged from 2 to 52 weeks (median = 6 weeks), while the follow‐up period ranged from 4 to 78 weeks (median = 24 weeks). One (Wang et al , ) and 11 studies (Bussel et al , ; Bussel et al , ; Kuter et al , ; Bussel et al , ; Kuter et al , ; Cheng et al , ; Shirasugi et al , ; Arnold et al , ; Tomiyama et al , ; Ghanima et al , ; Yang et al , ) reported platelet response at 4 and 6 weeks, respectively. Only 2 studies (Bussel et al , ; Kuter et al , ) of romiplostim versus placebo reported baseline thrombopoietin level.…”

Section: Resultsmentioning

confidence: 99%

“…Corticosteroids were the most common previous treatment followed by intravenous immunoglobulin. Percentage of splenectomy was reported in 10 studies (Bussel et al , ; Bussel et al , ; Kuter et al , ; Bussel et al , ; Cheng et al , ; Shirasugi et al , ; Tomiyama et al , ; Wang et al , ; Zhou et al , ; Yang et al , ) ranging from 10·4% to 69·6%, with a median time after splenectomy of 6·6–8·1 years.…”

Section: Resultsmentioning

confidence: 99%

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Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta‐analysis

et al. 2019

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Persistent immune thrombocytopenia (ITP) patients require second-line treatments, for which information on clinical outcomes are lacking. A systematic review and network meta-analysis (NMA) were conducted. Only randomised controlled trials (RCT) of second-line drugs in adult persistent ITP patients with platelet response, platelet count, any bleeding or serious adverse events (SAE) outcome were eligible. Twelve RCTs (n = 1313) were included in NMA. For platelet response outcome, eltrombopag and romiplostin were the best relative to placebo; the former had a non-significant advantage [risk ratio (RR) = 1Á10 (95% confidence interval: 0Á46, 2Á67)]. Both treatments were superior to rituximab and recombinant human thrombopoietin (rhTPO)+rituximab, with corresponding RRs of 4Á56 (1Á89, 10Á96) and 4Á18 (1Á21, 14Á49) for eltrombopag; 4Á13 (1Á56, 10Á94) and 3Á79 (1Á02, 14Á09) for romiplostim. For platelet count, romiplostim ranked highest, followed by eltrombopag, rhTPO+rituximab, and rituximab. For bleeding, rituximab had lowest risk, followed by eltrombopag and romiplostim. For SAEs, rhTPO+rituximab had highest risk, followed by rituximab, eltrombopag and romiplostim. From clustered ranking, romiplostim had the best balance between short-term efficacy and SAEs, followed by eltrombopag. In conclusion, romiplostim and eltrombopag may yield high efficacy and safety. Rituximab may not be beneficial due to lower efficacy and higher complications compared with the thrombopoietin receptor agonists. RCTs with long-term clinical outcomes are required.

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Efficacy and safety of eltrombopag in the treatment of immune thrombocytopenia patients with hepatitis B virus infection: A prospective, multicenter, single arm, phase II trial

Chen,

Xu,

Huang

et al. 2023

American J Hematol

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Hepatitis B virus (HBV) infection is the most common chronic viral infection worldwide. Epidemiological data showed that the hepatitis virus surface antigen carrier rate among those aged 1-59 was 7.2% in China, ranking at the intermediate prevalence level. 1 Although there is no epidemiological data report on immune thrombocytopenia patients with HBV infection (ITP-HBV), previous small sample studies showed that the detection rate of anti-hepatitis B core antigen (anti-HBc) in ITP patients was 30.3%. 2 For ITP-HBV, immunosuppressive agents increase the risk of HBV reactivation. Elevating platelet counts while controlling HBV replication is the key to a good prognosis for these patients.Thrombopoietin receptor agonists (TPO-RAs) can promote bone marrow megakaryocytes to produce mature platelets but have weak regulatory effects on the immune system. 3 Studies have shown that the overall response rate of TPO-RAs in the treatment of adult ITP is approximately 80%. 4 The advent of the era of TPO-RAs has changed the pattern of second-line treatment of ITP and provides new possibilities for disease management of ITP-HBV. Therefore, to evaluate the efficacy and safety of TPO-RAs in the treatment of ITP-HBV, we conducted a prospective, multicenter, single-arm clinical study on eltrombopag in the treatment of ITP-HBV (ClinicalTrials.gov Identifier: NCT03664518). Methods are detailed in the Supplementary Methods.Between December 2018 and December 2021, 48 patients from six sites in China were enrolled in the study and subsequently included in the safety analysis set (SS). In total, 46 patients completed the stage I study and were considered as the full analysis set (FAS) and perprotocol analysis set (PPS) (Figure S1). The demographics and baseline characteristics of patients were summarized in Tables S1 and S2. The median age of total patients was 49 years (interquartile range [IQR] 34-57), and 58.7% of patients were female. The median baseline platelet count was 13 Â 10 9 /L (IQR 7-23). Thirty-seven percentage (17/46) of patients were treated with glucocorticoids at baseline, 80.4% (37/46) received antiviral drugs, and 80.4% (37/46) had a history of steroid therapy. The difference of baseline characteristics was not statistically significant among the newly diagnosed, persistent, and chronic groups (all p > .05, Table S1). Additionally, we found that no baseline characteristic was correlated with treatment response (Table S3).

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Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia

Cited by 55 publications

References 17 publications

Primary immune thrombocytopenia (ITP) treated with romiplostim in routine clinical practice: retrospective study from the United Kingdom ITP Registry

Primary immune thrombocytopenia (ITP) treated with romiplostim in routine clinical practice: retrospective study from the United Kingdom ITP Registry

Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta‐analysis

Efficacy and safety of eltrombopag in the treatment of immune thrombocytopenia patients with hepatitis B virus infection: A prospective, multicenter, single arm, phase II trial

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