Adapted guideline for the diagnosis and treatment of primary immune thrombocytopenia for Chinese children (2021)

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doi:10.1002/ped4.12305

Cited by 12 publications

(6 citation statements)

References 43 publications

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“… 5 , 6 , 7 , 8 , 9 , 10 Many authorities recommend eltrombopag as a treatment for ITP. 1 , 11 , 12 With the widespread use of eltrombopag, the value of the drug concentration is becoming more apparent. Some studies indicate that insufficient concentration may impact the efficacy of eltrombopag.…”

Section: Discussionmentioning

confidence: 99%

“…Inclusion criteria: 1) Diagnosed with persistent or chronic ITP based on the Chinese adapted guidelines for pediatric ITP. 1 2) Younger than 18 years old. 3) Failed to respond to glucocorticoid and intravenous immunoglobulin.…”

Section: Methodsmentioning

confidence: 99%

“…2) Bleeding score reached grade 4 according to the guidelines for rescue therapy. 1 3) Children during the dose reduction period. 4) Abnormal liver function before the initial dose of eltrombopag.…”

Section: Methodsmentioning

confidence: 99%

“…Primary immune thrombocytopenia (ITP) is the most common hemorrhagic disorder in childhood, with an annual incidence of 1.6–5.3 per 100 000. 1 Although most children with ITP have a good prognosis, 20%–30% will develop refractory persistent and chronic ITP, and mostly presents with mucocutaneous bleeding. 2 These children will suffer from a lousy quality of life and require second‐line therapy.…”

Section: Introductionmentioning

confidence: 99%

“… 5 , 6 , 7 , 8 , 9 , 10 The updated international consensus report, along with the guidelines from the American Society of Hematology and adapted Chinese guidelines for children's ITP, suggest eltrombopag as a viable treatment for ITP. 1 , 11 , 12 …”

Section: Introductionmentioning

confidence: 99%

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Spotlight on eltrombopag concentration in pediatric immune thrombocytopenia: A single‐center observational study in China

Dong,

Wang,

Wang

et al. 2024

Pediatric Investigation

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ImportanceEltrombopag has been recommended for pediatric immune thrombocytopenia (ITP). Response and adverse drug reactions (ADRs) varied widely between individuals, even at the same dose of eltrombopag. The appropriate eltrombopag concentration in ITP has not been reported.ObjectiveThis study aims to explore the appropriate eltrombopag concentration in pediatric ITP.MethodsThis was a single‐center, prospective cohort study. Children diagnosed with refractory persistent/chronic ITP and platelet count < 30×109/L were treated with eltrombopag and followed up for at least 2 months. Concentration was detected by high‐performance liquid chromatography‐mass spectrometry at least 2 weeks after eltrombopag. The clinical characteristics‐concentration, concentration‐response, and concentration‐ADRs were analyzed.ResultsA total of 30 patients were enrolled, comprising 13 males and 17 females, with a median age of 72 (45‒94) months. The median dose and concentration were 1.39 (1.09‒1.56) mg/kg and 2.70 (2.25‒4.13) mg/L, respectively. Of the enrolled patients, 14 responded to treatment, whereas 16 did not. Additionally, five experienced adverse drug reactions. No linear correlation was observed between eltrombopag concentration and clinical characteristics. The concentration was lower in the response group than in the nonresponse group, but there was no significant difference (t = 0.755, P = 0.457). Patients who experienced ADRs had a higher concentration than those without ADRs (t = 2.538, P = 0.017). The area under the receiver operating characteristic curve of ADRs was 0.78 (95% confidence interval: 0.56‒1.00). Youden's index identified the cutoff point as 4.33 mg/L, with a sensitivity of 88% and a specificity of 60%. Logistic regression analysis demonstrated that a higher platelet count before eltrombopag predicted a favorable response.InterpretationEltrombopag proves efficacious and well‐tolerated for treating pediatric ITP. However, prolonged and high‐dose administration may increase the likelihood of ADRs. Thus, examining the appropriate eltrombopag concentration assists in directing individualized management of pediatric ITP.

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Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

Spotlight on eltrombopag concentration in pediatric immune thrombocytopenia: A single‐center observational study in China

Dong,

Wang,

Wang

et al. 2024

Pediatric Investigation

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Efficient adaptation of high‐quality international guidelines for Chinese children with primary immune thrombocytopenia

Liu

2022

Pediatric Investigation

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The Differences of Serum Thrombopoietin Levels Between Acquired Aplastic Anemia and Immune Thrombocytopenia in Pediatric Patients

Chen,

Ma,

Ouyang

et al. 2024

Journal of Pediatric Hematology/Oncology

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Thrombopoietin (TPO) is the critical regulator of platelet production. However, the role of TPO in pediatric patients with thrombocytopenic disorders has not been fully elucidated. In the present study, we attempted to investigate serum TPO levels in patients with acquired aplastic anemia (aAA) and immune thrombocytopenia (ITP). We analyzed the endogenous plasma concentration of TPO and platelet count at the time of TPO measurement in 166 patients with aAA and 280 patients with ITP retrospectively. We further observed a correlation between platelet counts and TPO. Serum TPO levels were significantly higher in aAA compared with ITP (1142 vs. 77.99 pg/mL, P<0.001). In patients with aAA, an elevation for TPO levels in very severe AA (VSAA) was seen when compared with non-severe AA (NSAA) (1360 vs. 984.4 pg/mL, P<0.05). In contrast, the circulating TPO levels with chronic ITP (CITP) showed a decrease than newly diagnosed ITP (NITP) and persistent ITP (PITP) (62.28 vs. 81.56 pg/mL, P<0.01, 62.28 vs. 87.82 pg/mL, P<0.05, respectively). There was a negative correlation between platelet counts and TPO levels in aAA (rs=−0.3325, P<0.001) as well as ITP (rs=−0.2570, P<0.001). Especially, TPO levels were inversely correlated with platelet counts in NSAA (rs=−0.3672, P<0.001) and NITP (rs=−0.3316, P<0.001). After grouping by age or sex, there were no statistical differences in aAA or ITP. Serum TPO levels were markedly elevated in pediatric patients with aAA compared with ITP. It was higher in VSAA and lower in CITP, suggesting that serum TPO level could play a role in classifying disease severity or clinical course in aAA and ITP.

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Adapted guideline for the diagnosis and treatment of primary immune thrombocytopenia for Chinese children (2021)

Cited by 12 publications

References 43 publications

Spotlight on eltrombopag concentration in pediatric immune thrombocytopenia: A single‐center observational study in China

Spotlight on eltrombopag concentration in pediatric immune thrombocytopenia: A single‐center observational study in China

Efficient adaptation of high‐quality international guidelines for Chinese children with primary immune thrombocytopenia

The Differences of Serum Thrombopoietin Levels Between Acquired Aplastic Anemia and Immune Thrombocytopenia in Pediatric Patients

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