Successful treatment of post-transplant thrombocytopenia with romiplostim in a pediatric patient with X-linked chronic granulomatous disease

Pediatr Transplant. 2014 Nov;18(7):E252-4. doi: 10.1111/petr.12325. Epub 2014 Aug 13.

Abstract

Thrombocytopenia is a frequent complication following HSCT in pediatric patients. Romiplostim is a TPO receptor agonist that has been utilized successfully in the treatment of pediatric patients with immune thrombocytopenia. We describe a three-yr-old male with X-linked CGD treated with an unrelated donor bone marrow transplant. His course was complicated by the development of symptomatic thrombocytopenia. He was started on romiplostim with prompt improvement in his thrombocytopenia. We found the use of romiplostim to be an effective and safe alternative to the potential complications as well as morbidity and mortality associated with the use of immunosuppressive agents such as corticosteroids.

Keywords: chronic granulomatous disease; hematopoietic cell transplantation; immune deficiency; romiplostim; thrombocytopenia.

Publication types

  • Case Reports

MeSH terms

  • Adrenal Cortex Hormones / therapeutic use
  • Bone Marrow Transplantation / adverse effects*
  • Child, Preschool
  • Granulomatous Disease, Chronic / complications*
  • Granulomatous Disease, Chronic / therapy*
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Male
  • Postoperative Complications
  • Receptors, Fc / therapeutic use*
  • Recombinant Fusion Proteins / therapeutic use*
  • Thrombocytopenia / etiology*
  • Thrombopoietin / therapeutic use*
  • Transplantation, Homologous

Substances

  • Adrenal Cortex Hormones
  • Immunosuppressive Agents
  • Receptors, Fc
  • Recombinant Fusion Proteins
  • Thrombopoietin
  • romiplostim