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Delayed treatment-free response after romiplostim discontinuation in pediatric chronic immune thrombocytopenia

Hyun Ji Lim1, Young Tae Lim1, Jeong Ok Hah2

  • 1Department of Pediatrics, Yeungnam University Hospital, Daegu, Korea.

Yeungnam University Journal of Medicine
|September 6, 2020
PubMed

Insights

A pediatric patient with chronic immune thrombocytopenia (ITP) achieved a delayed treatment-free response (TFR) after romiplostim discontinuation. This case highlights a potential long-term remission possibility in children with ITP.

Area of Science:

  • Pediatric Hematology
  • Immunology
  • Pharmacology

Background:

  • Chronic immune thrombocytopenia (ITP) in children often requires long-term management.
  • Standard therapies include corticosteroids, intravenous immunoglobulin, and immunosuppressants.
  • Thrombopoietin receptor agonists like romiplostim are used for refractory ITP.

Observation:

  • A 16-month-old with chronic ITP, unresponsive to rituximab and cyclosporine, received romiplostim.
  • Platelet counts normalized within 4 weeks of romiplostim initiation.
  • After 7 months, romiplostim was discontinued, leading to a decrease in platelet count.

Findings:

  • The patient achieved a sustained platelet count above 50,000/μL without bleeding, 2 years post-romiplostim discontinuation.
  • This represents a delayed treatment-free response (TFR) to romiplostim in pediatric chronic ITP.
  • This is the first reported case of TFR following romiplostim in this population.

Implications:

  • Romilostim may induce long-term remission or TFR in pediatric chronic ITP.
  • This finding could alter treatment strategies for refractory pediatric ITP.
  • Further research is warranted to explore the mechanisms and long-term efficacy of romiplostim-induced TFR.

Keywords:
ChildImmune thrombocytopenia, Idiopathic thrombocytopenic purpuraRomiplostimTreatment-free response

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