title: Successful treatment of post-transplant thrombocytopenia with romiplostim in a pediatric patient with X-linked chronic granulomatous disease.
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.
authors.affiliation: Division of Hematology, CHOC Children's Hospital, Orange, CA, USA.
authors.forename: David
authors.initials: D
authors.lastname: Buchbinder
authors.forename: Loan
authors.initials: L
authors.lastname: Hsieh
authors.forename: Robert
authors.initials: R
authors.lastname: Krance
authors.forename: Diane J
authors.initials: DJ
authors.lastname: Nugent
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000009:adverse effects*; D002675:Child, Preschool; D006105:Granulomatous Disease, Chronic / Q000150:complications* / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D011183:Postoperative Complications; D011961:Receptors, Fc / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use*; D013921:Thrombocytopenia / Q000209:etiology*; D013926:Thrombopoietin / Q000627:therapeutic use*; D014184:Transplantation, Homologous
chemical_list: D000305:Adrenal Cortex Hormones; D007166:Immunosuppressive Agents; D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim
keywords: chronic granulomatous disease; hematopoietic cell transplantation; immune deficiency; romiplostim; thrombocytopenia