Use of hydroxyurea and recombinant erythropoietin in management of homozygous beta0 thalassemia.
This report describes the sustained response of an Iranian girl with homozygous beta(0) thalassemia (IVS-II-1G-->A) to hydroxyurea (HU) and recombinant erythropoietin (rEPO). Since the start of this regimen 7 years ago, she has been transfusion-independent and her hemoglobin is maintained between 9.5-11.0 gm/dL. She is maintaining consistent growth around the 10th percentile for age and enjoys a good quality of life. She has not had any therapy-related adverse effects. This experience suggests that therapy with HU and rEPO may be useful long-term in some patients with beta thalassemia.
Kohli-Kumar
Mudra
M
Pediatrics, University of South Florida, 17th Davis Boulevard 2nd floor, Tampa, FL 33606-3475, USA. mukumar@hsc.usf.edu
Marandi
Hossain
H
Keller
Margaret A
MA
Guertin
Kathy
K
Hvizdala
Eva
E
D000986:Antisickling Agents / Q000627:therapeutic use; D002648:Child; D004921:Erythropoietin / Q000627:therapeutic use*; D005260:Female; D006529:Hepatomegaly / Q000209:etiology; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use*; D011994:Recombinant Proteins; D013163:Splenomegaly / Q000209:etiology; D033581:Stem Cell Transplantation; D017086:beta-Thalassemia / Q000097:blood / Q000188:drug therapy*
D000986:Antisickling Agents; D011994:Recombinant Proteins; D004921:Erythropoietin; D006918:Hydroxyurea