Treatment of dyskeratosis congenita with granulocyte-macrophage colony-stimulating factor and erythropoietin


Erduran E. , HACISALIHOGLU S., OZORAN Y.

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY, cilt.25, ss.333-335, 2003 (SCI İndekslerine Giren Dergi) identifier identifier identifier

  • Cilt numarası: 25 Konu: 4
  • Basım Tarihi: 2003
  • Doi Numarası: 10.1097/00043426-200304000-00015
  • Dergi Adı: JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY
  • Sayfa Sayıları: ss.333-335

Özet

Dyskeratosis congenita (DC) is a rare inherited disorder characterized by reticulate skin pigmentation, nail dystrophy, mucosal leucoplakia, and bone marrow failure, Pancytopenia is difficult to manage in patients,with this disorder. We describe a 13-month-old-boy who presented with reticulate skin lesions. paleness, and hepatosplenomegaly. Anemia and leukopenia developed by the age of 43 months. The patient was treated with granulocyte-macrophage colony-stimulating factor (GM-CSF) (5 mug/kg/d, subcutaneously) for 19 months and erythropoietin (150 U/k 3 days in a week, subcutaneously) for 8 months. with excellent neutrophil and hemoglobin response. Recurrent infections were not developed after starting GM-CSF. and packed red blood cell transfusion was not given to the patient after starting erythropoietin. GM-CSF combined with erythropoietin may be used in the treatment of bone marrow failure in patients with DC without an HLA-identical donor.