title: Low and fixed dose of hydroxyurea is effective and safe in patients with HbSβ(+) thalassemia with IVS1-5(G→C) mutation.
abstract: BACKGROUND
  Despite compelling evidence that hydroxyurea is safe and effective in sickle cell disease, it is prescribed sparingly due to several barriers like knowledge gaps in certain genotypes, apprehension about its safety and toxicity, and limited resources. We undertook this study to find out the efficacy and safety of HU in patients with HbSβ(+) -thalassemia with IVS1-5(G→C) mutation.
  
  
  METHODS
  We registered 318 patients with HbSβ(+) -thalassemia with IVS1-5(G→C) mutation. Of these, 203 were enrolled for hydroxyurea treatment at a low and fixed dose of 10 mg/kg/day. One hundred four patients (Group-I: 37 children and Group-II: 67 adults) with ≥2 years of hydroxyurea treatment were studied.
  
  
  RESULTS
  The rate of vaso-occlusive crises, requirement of blood transfusion and rate of hospitalization reduced from 3 to 0.5, 1 to 0 and 1 to 0 in Group-I and 3 to 0, 1 to 0 and 0.5 to 0 in Group-II respectively after HU therapy (P < 0.0001). %HbF level, hemoglobin, MCV and MCH increased significantly, whereas HbS, WBC, platelet count, serum-bilirubin and LDH levels decreased significantly after HU therapy. It has been observed that along with fairly subtle hematological changes following HU therapy, there was a substantial clinical improvement occurred in these patients. Transient myelotoxicity was observed in 4.8%. There was minimal gonadal toxicity without affecting reproductive function.
  
  
  CONCLUSIONS
  In view of easy affordability, better acceptability, minimal toxicity, the need of infrequent monitoring and its potential effectiveness, low and fixed dose of hydroxyurea is suitable for treatment of patients with HbSβ(+) -thalassemia in resource poor setting.
authors.affiliation: Sickle Cell Clinic and Molecular Biology Laboratory, Veer Surendra Sai Medical College, Burla, Sambalpur, Odisha, India.
authors.forename: Snehadhini
authors.initials: S
authors.lastname: Dehury
authors.forename: Prasanta
authors.initials: P
authors.lastname: Purohit
authors.forename: Siris
authors.initials: S
authors.lastname: Patel
authors.forename: Satyabrata
authors.initials: S
authors.lastname: Meher
authors.forename: Bipin Kishore
authors.initials: BK
authors.lastname: Kullu
authors.forename: Lulup Kumar
authors.initials: LK
authors.lastname: Sahoo
authors.forename: Nayan Kumar
authors.initials: NK
authors.lastname: Patel
authors.forename: Alok Kumar
authors.initials: AK
authors.lastname: Mohapatra
authors.forename: Kishalaya
authors.initials: K
authors.lastname: Das
authors.forename: Dilip Kumar
authors.initials: DK
authors.lastname: Patel
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005260:Female; D005298:Fertility; D006451:Hemoglobin, Sickle / Q000032:analysis / Q000235:genetics*; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage* / Q000009:adverse effects; D008297:Male; D009154:Mutation; D011446:Prospective Studies; D013789:Thalassemia / Q000097:blood / Q000188:drug therapy* / Q000235:genetics
chemical_list: D006451:Hemoglobin, Sickle; D006918:Hydroxyurea
keywords: HbF; HbSβ+ thalassemia; fertility; hydroxyurea; low dose; vaso occlusive crisis (VOC)