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Managing Immune Thrombocytopenic Purpura in infants: is it time to think of Eltrombopag?
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  • Giulia Ceglie,
  • Giulia Nocentini,
  • Francesca de Gennaro,
  • Vitangelo Clemente,
  • Margherita Di Mauro,
  • Giuseppe Palumbo
Giulia Ceglie
Bambino Gesù Pediatric Hospital Oncohematology Department of Cell Therapy Gene Therapies and Hemopoietic Transplant
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Giulia Nocentini
Ospedale Pediatrico Bambino Gesù
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Francesca de Gennaro
Ospedale Pediatrico Bambino Gesù
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Vitangelo Clemente
Ospedale Pediatrico Bambino Gesù
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Margherita Di Mauro
Ospedale Pediatrico Bambino Gesù
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Giuseppe Palumbo
Bambino Gesù Pediatric Hospital Oncohematology Department of Cell Therapy Gene Therapies and Hemopoietic Transplant
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Abstract

We report 2 cases of infants with acute and persistent Immune Thrombocytopenic Purpura (ITP) treated with Eltrombopag. Since ITP is rare in infants, robust evidence about how to treat these patients is not available. Both children underwent multiple lines of treatment without success and were successfully managed with off-label use of Eltrombopag. We did not observe any of the reported adverse effects of the drug and complete remission was achieved in both cases. In one child, we were able to discontinue treatment without any ITP relapse. This is the first report of an off-label use of Eltrombopag in infants.