Fouzia Ishaq, Jovaria Mannan, Talat Seyal, Hasnain Abid, Sadia Hassan.
Efficacy and side effects of hydroxyurea in patient with thalassemia intermedia.
Pak Paed J Jan ;35(1):8-12.

Objective: Purpose of this study was to determine the efficacy and possible side effects of Hydroxyurea (HU) in patient with Thalassemia Intermedia and its relation to I-globin gene mutations. Study Design: Non Randomized Experimental Study. Study Place & Duration: The study was undertaken at Thalassemia Center, Sir Ganga Ram Hospital Lahore over 3 years period from February, 2007 Jan, 2010. Patients & Methods: Fifty five patients of thalassemia Intermedia with mean age of 8.6 years, comprising of 28 males and 27 females were given Hydroxyurea in a single daily dose of 15 mg/kg orally with oral folic acid. Twenty nine (53%) were blood transfusion dependent, seventeen (31%) had received 1-2 blood transfusion and nine (16%) were never transfused prior to starting HU. Response of drug was evaluated in terms of rise in hemoglobin (g/dl) or a reduction in transfusion requirement. Median follow up of patients was 12 months (range 6 -36 months). Response criterion was classified into good, partial and no response as below: Good Response: Transfusion Independence or Hb rise of > 2g/dl. Partial Response:Rise in Hb of 1-2 g/dl or reduction in transfusion frequency by 50%. No Response: No rise in Hb or remained at the same level of transfusion dependency. Blood samples were also examined by Amplification Refractory Mutation Specific (ARMS) PCR for common beta-thalassemia mutations and results were correlated with response to HU. Mutations in eighteen patients could not be studied due to economic constraint. Results: In our study 80% patients showed response to HU. Thirty eight (69%) were good responders and six (11%) showed partial response. Among good responders, 66% had a rise in hemoglobin > 2 g/dl and reduction in spleen size was found in 60.7%. All 38 patients became transfusion independent (100%). Only 2 (3.6%) patients complained at nausea, mild abdominal pain and diarrhea. On analysis of beta globin gene mutations in 37 TI patients, five common mutations identified were IVS1-5, CD-30, Codon 8/9 (+G), Fr 8-9 (+G) and CD 41/42 these constituted 86.4% of total mutations. In our study three patients with good response and one patient with partial response had Codon 8/9 (+G) mutation, but none of seven non-responder patients had this mutation. Conclusion: Hydroxyurea is a safe, well tolerated and affordable drug with minimal side effects for patients with β thalassemia intermedia. Patients clinically diagnosed as TI and receiving regular blood transfusions can also be given a trial of HU to minimize or even eliminate the need for regular blood transfusion and concomitant iron over load.

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