Role of eltrombopag in severe aplastic anemia treatment in children

Su, Min-Yu; Chang, Hong-Yeh; Chou, Shu‐Wei; Lu, Meng‐Yao; Yang, Yung‐Li; Lin, Dong‐Tsamn; Lin, Kai‐Hsin; Jou, Shiann-Tarng

doi:10.1016/j.pedneo.2021.07.002

Cited by 4 publications

(4 citation statements)

References 6 publications

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“…One of the 5 patients had a relapse of the disease and underwent TCS. The mean duration of eltrombopag use was 12.9 months ( 20 ). The other case series considered 11 pediatric patients who received ELT in addition to standard IST.…”

Section: Resultsmentioning

confidence: 99%

The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review

et al. 2023

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BackgroundAcquired aplastic anemia (AAA) in pediatric patients is a rare disorder characterized by hypocellular bone marrow and pancytopenia. Eltrombopag, an oral thrombopoietin receptor agonist, provides a hematologic improvement in adults with severe aplastic anemia (SAA) refractory to immunosuppressive therapy (IST). The association of ELT and IST was approved by the US Food and Drug Administration (FDA) for adults and children ≥2 years of age as a first-line treatment for SAA. However, the effects of ELT on pediatric patients with SAA remain controversial and limited.Methods and findingsWe conducted a systematic review of the most recent literature from Pubmed, Web of Science, and Embase, published up to 20th December 2022, in order to evaluate the available evidence on the efficacy and safety of ELT added to IST for the treatment of SAA in the pediatric population.ConclusionEltrombopag added to the IST has shown a good safety profile, without manifestations of excessive toxic effects, although not all the results obtained from our studies support the addition of ELT to the IST in the first-line treatment of children with SAA.Systematic Review Registrationhttps://www.crd.york.ac.uk/prospero/, identifier: CRD42022325859.

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Section: Resultsmentioning

confidence: 99%

The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review

et al. 2023

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“…14,15 However, data on the efficacy of eltrombopag plus IST in children with different types of AA, especially NSAA, are limited. [16][17][18][19][20][21][22][23][24] Until now, only one case study reported the upfront monotherapy of eltrombopag in two boys with NSAA. 17 Therefore, acquiring more data on the efficacy and safety of eltrombopag in children with different types of AA is needed.…”

Section: Introductionmentioning

confidence: 99%

“…Based on the effective clinical response and tolerable safety of using eltrombopag in children with SAA, 10–13 the Chinese guidelines and expert opinions have added eltrombopag to the treatment regimens of children with AA, including SAA, VSAA, and NSAA 14,15 . However, data on the efficacy of eltrombopag plus IST in children with different types of AA, especially NSAA, are limited 16–24 . Until now, only one case study reported the upfront monotherapy of eltrombopag in two boys with NSAA 17 .…”

Section: Introductionmentioning

confidence: 99%

Efficacy, Safety, and Population Pharmacokinetics of Eltrombopag in Children with Different Severities of Aplastic Anemia

Zhang,

Chang,

Zhao

et al. 2024

The Journal of Clinical Pharma

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Eltrombopag was approved as a first‐line treatment for patients older than 2 years old with severe aplastic anemia (SAA). However, data on eltrombopag in children with different types of aplastic anemia (AA), especially non‐severe AA (NSAA), are limited. We performed a prospective, single‐arm, and observational study to investigate eltrombopag's efficacy, safety, and pharmacokinetics in children with NSAA, SAA, and very severe AA (VSAA). The efficacy and safety were assessed every 3 months. The population pharmacokinetic (PPK) model was used to depict the pharmacokinetic profile of eltrombopag. Twenty‐three AA children with an average age of 7.9 (range of 3.0–14.0) years were enrolled. The response (complete and partial response) rate was 12.5%, 50.0%, and 100.0% after 3, 6, and 12 months in patients with NSAA. For patients with SAA and VSAA, these response rates were 46.7%, 61.5%, and 87.5%. Hepatotoxicity occurred in one patient. Fifty‐three blood samples were used to build the PPK model. Body weight was the only covariate for apparent clearance (CL/F) and volume of distribution. The allele‐T carrier of adenosine triphosphate‐binding cassette transporter G2 was found to increase eltrombopag's clearance. However, when normalized by weight, the clearance between the wild‐type and variant showed no statistical difference. In patients with response, children with NSAA exhibited lower area under the curve from time zero to infinity, higher CL/F, and higher weight‐adjusted CL/F than those with SAA or VSAA. However, the differences were not statistically significant. The results may support further individualized treatment of eltrombopag in children with AA.

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“…Studies of EPAG with IST as a first-line therapy have been conducted primarily with adults in the past decade, while clinical evidence for the benefit of adding EPAG to standard IST in children remains limited and conflicting (Supplementary Table S1) (12)(13)(14)(15)(16)(17)(18)(19)(20). According to a retrospective study performed by the NIH, it seems that the addition of EPAG to IST did not provide the same benefit in children as that observed in adults with SAA (16).…”

Section: Introductionmentioning

confidence: 99%

Efficacy of eltrombopag with immunosuppressive therapy for children with acquired aplastic anemia

et al. 2023

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BackgroundEltrombopag (EPAG), an oral thrombopoietin receptor agonist (TPO-RA), has been proven to improve the hematologic response without increasing toxic effects as a first-line therapy combined with standard immunosuppressive treatment (IST) in adults with severe aplastic anemia (SAA). Nevertheless, the clinical evidence on the efficacy of EPAG in children with acquired aplastic anemia is limited and controversial.MethodsWe performed a single-center, retrospective study to analyze the clinical outcomes of fifteen patients aged ≤18 years with newly diagnosed acquired SAA who received first-line IST and EPAG (EPAG group) compared with those of forty-five patients who received IST alone (IST group) by propensity score matching (PSM).ResultsThere was no difference in the overall response (OR) rate between the EPAG group and IST group (53.3% vs. 46.7% at 3 months, P = 0.655; 66.7% vs. 57.8% at 6 months, P = 0.543), but the complete response (CR) rate was statistically significant (20.0% vs. 4.4% at 3 months, P = 0.094; 46.7% vs. 13.3% at 6 months, P = 0.012). The median time to achieve a hematological response in the EPAG and IST groups was 105 days and 184 days, respectively. No difference was observed in the event-free survival (EFS) or overall survival (OS) rates.ConclusionAdding EPAG to standard IST as the first-line treatment for children with acquired SAA improved the rapidity of hematological response and the CR rate but did not improve the OR or EFS rates.

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Role of eltrombopag in severe aplastic anemia treatment in children

Cited by 4 publications

References 6 publications

The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review

The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review

Efficacy, Safety, and Population Pharmacokinetics of Eltrombopag in Children with Different Severities of Aplastic Anemia

Efficacy of eltrombopag with immunosuppressive therapy for children with acquired aplastic anemia

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