Thrombopoietin Receptor Agonist Use in Children

Data From the Pediatric ITP Consortium of North America ICON2 Study

on behalf of the Pediatric ITP Consortium of North America (ICON)

Research output: Contribution to journalArticle

25 Citations (Scopus)

Abstract

Background: Data on second-line treatment options for pediatric patients with immune thrombocytopenia (ITP) are limited. Thrombopoietin receptor agonists (TPO-RA) provide a nonimmunosuppressive option for children who require an increased platelet count. Procedure: We performed a multicenter 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 eight 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 3 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.

Original languageEnglish (US)
Pages (from-to)1407-1413
Number of pages7
JournalPediatric Blood and Cancer
Volume63
Issue number8
DOIs
StatePublished - Aug 1 2016

Fingerprint

Thrombopoietin Receptors
Idiopathic Thrombocytopenic Purpura
North America
Pediatrics
Platelet Count
Therapeutics
Neutralizing Antibodies
Multicenter Studies
Retrospective Studies
Clinical Trials

Keywords

  • ICON
  • immune thrombocytopenia
  • pediatrics
  • thrombopoietin receptor agonists
  • treatment

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

Thrombopoietin Receptor Agonist Use in Children : Data From the Pediatric ITP Consortium of North America ICON2 Study. / on behalf of the Pediatric ITP Consortium of North America (ICON).

In: Pediatric Blood and Cancer, Vol. 63, No. 8, 01.08.2016, p. 1407-1413.

Research output: Contribution to journalArticle

on behalf of the Pediatric ITP Consortium of North America (ICON). / Thrombopoietin Receptor Agonist Use in Children : Data From the Pediatric ITP Consortium of North America ICON2 Study. In: Pediatric Blood and Cancer. 2016 ; Vol. 63, No. 8. pp. 1407-1413.
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title = "Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study",
abstract = "Background: Data on second-line treatment options for pediatric patients with immune thrombocytopenia (ITP) are limited. Thrombopoietin receptor agonists (TPO-RA) provide a nonimmunosuppressive option for children who require an increased platelet count. Procedure: We performed a multicenter 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 eight 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 3 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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