Treatment of dyskeratosis congenita with granulocyte-macrophage colony-stimulating factor and erythropoietin.
J Pediatr Hematol Oncol
; 25(4): 333-5, 2003 Apr.
Article
en En
| MEDLINE
| ID: mdl-12679652
ABSTRACT
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 microg/kg/d, subcutaneously) for 19 months and erythropoietin (150 U/kg 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.
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Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Pancitopenia
/
Factor Estimulante de Colonias de Granulocitos y Macrófagos
/
Eritropoyetina
/
Disqueratosis Congénita
Tipo de estudio:
Etiology_studies
Límite:
Child, preschool
/
Humans
/
Male
Idioma:
En
Revista:
J Pediatr Hematol Oncol
Asunto de la revista:
HEMATOLOGIA
/
NEOPLASIAS
/
PEDIATRIA
Año:
2003
Tipo del documento:
Article
País de afiliación:
Turquía