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Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered

Rachael F Grace1, Kristin A Shimano2, Rukhmi Bhat3

  • 1Division of Hematology/Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center, Boston, Massachusetts.

American Journal of Hematology
|April 5, 2019
PubMed

Insights

Second-line treatments for immune thrombocytopenia (ITP) in children show varied effects on platelet counts, bleeding, and quality of life. Romiplostim and rituximab effectively reduced bleeding, while eltrombopag improved quality of life.

Area of Science:

  • Pediatric Hematology
  • Autoimmune Diseases
  • Clinical Outcomes Research

Background:

  • Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder in children.
  • Second-line treatments are necessary when observation is insufficient or to improve quality of life (HRQoL).
  • Comparative data on second-line treatment outcomes for pediatric ITP is limited.

Purpose of the Study:

  • To compare outcomes of second-line treatments for pediatric ITP.
  • To evaluate effects on platelet count, bleeding symptoms, and HRQoL.

Main Methods:

  • ICON1: A prospective, multi-center, observational study.
  • 120 children initiating second-line ITP treatment.
  • Outcomes assessed using platelet counts, bleeding assessments, and the Kids ITP Tool (KIT) for HRQoL.

Main Results:

  • All treatments increased platelet counts; romiplostim showed the most significant effect at 6 months.
  • Romilostim and rituximab significantly reduced both skin and non-skin bleeding symptoms within 1 month.
  • HRQoL improved with all treatments; eltrombopag met the minimal important difference (MID) for KIT scores at 1 month.

Conclusions:

  • Second-line treatments for pediatric ITP vary in their efficacy and timing of effect on platelet count, bleeding, and HRQoL.
  • Romilostim and rituximab demonstrated notable reductions in bleeding, while eltrombopag showed significant HRQoL improvements.
  • Findings are hypothesis-generating for optimizing individual treatment selection in pediatric ITP, pending further randomized trials.

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