The treatment for primary immune thrombocytopenia with romiplostim in adult and paediatric patients: use experience at a Spanish university hospital

Marquínez-Alonso, I; Escudero‐Vilaplana, Vicente; Pernía, S; Bieler, Cristina Beléndez; Fernández-Llamazares, Cecilia M.; Sanjurjo‐Sáez, María

doi:10.1111/jcpt.12156

Cited by 5 publications

(4 citation statements)

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“…Supplementary Table SI outlines the lack of uniformity in applying a standardize response criteria across both pediatric and adult trials. [5][6][7][8][9][10][11][19][20][21][22][23][24][25][26][27][28][29][30][31][32][33][34][35][36] We therefore applied definitions common to the literature to allow for the best comparison between our data and the existing literature.…”

Section: Discussionmentioning

confidence: 99%

“…In the open‐label phase of these trials, 80% of patients were able to achieve a platelet count ≥50 × 10 9 /l at least once . Additional data about the use of these agents in pediatric ITP is limited to small single center retrospective reviews and case series focused on children with persistent and chronic ITP …”

Section: Introductionmentioning

confidence: 99%

“…[6,7] Additional data about the use of these agents in pediatric ITP is limited to small single center retrospective reviews and case series focused on children with persistent and chronic ITP. [9][10][11] Despite a paucity of literature regarding TPO-RA use in children, physicians are prescribing TPO-RAs to pediatric patients off-label and outside of clinical trials. Furthermore, physicians may not be reserving these agents exclusively for chronic primary ITP, the only group in whom these agents have been investigated and are indicated.…”

Section: Introductionmentioning

confidence: 99%

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Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study

Neunert

Despotovic

Haley

et al. 2016

Pediatr Blood Cancer

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Background Data on second-line treatment options for pediatric patients with immune thrombocytopenia (ITP) are limited. Thrombopoietin receptor agonists (TPO-RA) provide a non-immunosuppressive option for children who require an increased platelet count. Procedure We performed a multi-center retrospective study of pediatric ITP patients followed at ITP Consortium of North America (ICON) sites to characterize TPO-RA use. Results Seventy-nine children had a total of 87 treatments (28 eltrombopag, 43 romiplostim, and 8 trialed on both). The majority had primary ITP (82%) and most (60.8%) had chronic ITP. However, 22% had persistent ITP and 18% had newly diagnosed ITP. During the first three months of treatment, 89% achieved a platelet count ≥ 50 × 109/l (86% romiplostim, 81% eltrombopag, p=0.26) at least once in the absence of rescue therapy. The average time to a response was 6.4 weeks for romiplostim and 7.0 weeks for eltrombopag (p=0.83). Only 40% of patients demonstrated a stable response with consistent dosing over time. An intermittent response with constant dose titration was seen in 15%, and an initial response that waned to no response was seen in 13%. Significant adverse events were minimal with the exception of two patients with thrombotic events and one who developed a neutralizing antibody. Conclusions Our results demonstrate that TPO-RA agents are being used in children with ITP of varying duration and severity. The response was similar to clinical trials, but the sustainability of response varied. Future studies need to focus on the ideal timing and rationale for these medications in pediatric patients.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study

Neunert

Despotovic

Haley

et al. 2016

Pediatr Blood Cancer

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“…The patient also required concomitant corticosteroids and intravenous immunoglobulin. Marquinez-Alonso et al32 reported on four children with a mean age of 10.3 years treated with romiplostim for chronic ITP. The median treatment duration was 50.5 weeks.…”

Section: Introductionmentioning

confidence: 99%

Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy

Buchbinder¹,

Nugent²,

Hsieh³

2017

DDDT

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Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia. In approximately one-third of cases, the duration of thrombocytopenia will extend beyond 12 months consistent with a diagnosis of chronic ITP. Minor bleeding manifestations are common in chronic ITP while severe or life-threatening bleeding complications are uncommon. Moreover, spontaneous resolution occurs in the majority of children with chronic ITP necessitating treatment in only those children with ongoing bleeding manifestations or impairment in health-related quality of life (HRQOL). The characterization of thrombopoietin (TPO) and remarkable advancements in our understanding of the pathophysiology of ITP has led to the development of a new class of agents, the TPO-receptor agonists that have documented efficacy in the amelioration of thrombocytopenia and bleeding manifestations in chronic ITP. Romiplostim is a second-generation TPO-receptor agonist that has undergone limited evaluation in the treatment of chronic ITP in children. Evolving data suggest that romiplostim may be a safe and effective agent in the treatment of chronic ITP in children. Additional data are needed to confirm its ability to increase platelet counts, decrease bleeding manifestation, and improve the HRQOL of children and caregivers impacted by chronic ITP.

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Romiplostim

2014

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The treatment for primary immune thrombocytopenia with romiplostim in adult and paediatric patients: use experience at a Spanish university hospital

Cited by 5 publications

References 29 publications

Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study

Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study

Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy

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