Efficacy and quality of life of Romiplostim in adults and children with immune thrombocytopenia: A review

He, Xin; Ran, Ningyuan; Wang, Ting; Shao, Zonghong

doi:10.1097/md.0000000000032345

Cited by 2 publications

(2 citation statements)

References 22 publications

(27 reference statements)

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“…30 In a meta-analysis including nine randomized controlled trials assessing the efficacy and QOL of Romiplostim in adults and children with ITP, no statistically significant difference in HRQoL was found in the study population compared to the control group. 31 Although TPO-RAs increased platelet counts, they did not seem to improve HRQoL.…”

Section: Th E I N Flu E Nce Of Tr E Atm E N Ts On R Efr Ac Tory Itp-c...mentioning

confidence: 96%

Burden of immune thrombocytopenia (ITP): Special considerations for refractory ITP

Rüfer,

Terrell

2023

Br J Haematol

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SummaryIt is known that patients with immune thrombocytopenia (ITP) have fatigue and impairment of health‐related quality of life (HRQoL). However, it is hypothesized that patients with refractory ITP have additional burdens that should be considered. Specifically, fatigue is more pronounced in patients with refractory disease, there are additional side effects from second‐ and third‐line treatments, additional anxiety about the long‐term course of the disease, impairment in HRQoL resulting from heavy menstrual bleeding and concerns related to family planning. The burden of disease, therefore, should be carefully assessed and considered in these patients. However, researchers have utilized numerous tools for evaluating HRQoL and fatigue, making comparison of data across studies challenging. There is a need to standardize assessment using either disease‐specific or generic instruments that can be easily implemented in routine clinical practice. Additionally, whether treatment of low platelet count and bleeding symptoms will have a positive influence on HRQoL remains to be seen and published evidence is conflicting. Nevertheless, improvement of HRQoL is a major treatment goal for both patients and physicians and should be especially considered when treating patients with refractory ITP.

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Section: Th E I N Flu E Nce Of Tr E Atm E N Ts On R Efr Ac Tory Itp-c...mentioning

confidence: 96%

Burden of immune thrombocytopenia (ITP): Special considerations for refractory ITP

Rüfer,

Terrell

2023

Br J Haematol

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“…25 Systematic reviews indicated a beneficial effect as second-line treatment in children older than 1 year with persistent/chronic ITP. [26][27][28] These reviews, however, did not include most recent data of the subsequent international phase 3b RCT on the long-term use of romiplostim in children with ITP, following the same protocol over a 36-month period (NCT02279173). This RCT showed a median remission rate of 50.0% (interquartile range (IQR) 16.7-83.3%) during the first 6 months, increasing to 78.2% (IQR 26.7-90.4%) during the overall 36-month treatment period.…”

Section: Schlüsselwörtermentioning

confidence: 99%

Update on the Use of Thrombopoietin-Receptor Agonists in Pediatrics

Gebetsberger,

Streif,

Dame

2024

Hamostaseologie

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This review summarizes the rationale and current data on the use of thrombopoietin receptor agonists (TPO-RAs) for treating severe thrombocytopenia in infants, children, and adolescents. It focuses on substances that have been approved by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for pediatric patients. Romiplostim and eltrombopag are already established as second-line treatment for persistent or chronic immune thrombocytopenia (ITP). As in adults, TPO-RAs are currently also evaluated in severe aplastic anemia (SAA), chemotherapy-induced thrombocytopenia (CIT), myelodysplastic syndromes (MDS), and poor engraftment after hematopoietic stem cell transplantation in pediatric and adolescent patients. Moreover, studies on the implication of TPO-RA in treating rare inherited thrombocytopenias, such as Wiskott-Aldrich syndrome (WAS), congenital amegakaryocytic thrombocytopenia (CAMT), or MYH9-associated thrombocytopenia, deserve future attention. Current developments include testing of avatrombopag and lusutrombopag that are approved for the treatment of thrombocytopenia associated with chronic liver disease (CLD) in adult patients. In pediatric and adolescent medicine, we expect in the near future a broader use of TPO-RAs as first-line treatment in primary ITP, thereby considering immunomodulatory effects that increase the rate of sustained remission off-treatment, and a selective use in rare inherited thrombocytopenias based on current clinical trials.

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Efficacy and quality of life of Romiplostim in adults and children with immune thrombocytopenia: A review

Cited by 2 publications

References 22 publications

Burden of immune thrombocytopenia (ITP): Special considerations for refractory ITP

Burden of immune thrombocytopenia (ITP): Special considerations for refractory ITP

Update on the Use of Thrombopoietin-Receptor Agonists in Pediatrics

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