Indian Pediatrics

, Volume 50, Issue 10, pp 929–933 | Cite as

Efficacy of fixed low dose hydroxyurea in Indian children with sickle cell anemia: A single centre experience

  • Dipti L. JainEmail author
  • Mohini Apte
  • Roshan Colah
  • Vijaya Sarathi
  • Saumil Desai
  • Amruta Gokhale
  • Amol Bhandarwar
  • Harshwardhan L. Jain
  • Kanjaksha Ghosh
Research Paper



Data on the efficacy of hydroxyurea (HU) in Indian children with sickle cell anaemia (SCA) is limited. Hence, we have evaluated the efficacy of fixed low dose HU in Indian children.


The study cohort consisted of 144 children (<18 years of age) with SCA having severe manifestations (≥3 episodes of vasocclusive crisis or blood transfusions, or having ≥1 episode of acute chest syndrome or cerebrovascular stroke or sequestration crisis) who were started on fixed low dose HU (10 mg/kg/day). They were followed up for two years and monitored for the hematological and clinical efficacy and safety.


There was significant increase in the fetal hemoglobin level (HbF%), total hemoglobin and mean corpuscular volume. Vasoocclusive crises, blood transfusions, acute chest syndrome, sequestration crises and hospitalizations decreased significantly. Baseline HbF% had significant positive correlation with HbF% at 24 months. There was significant negative correlation between baseline HbF% and change in HbF% from baseline to 24 months. No significant correlation was found between HbF% at baseline and clinical event rates per year after HU. No major adverse events occurred during the study period.


Fixed low dose HU is effective and safe in Indian children with SCA.


Child Hydroxyurea India Low dose Sickle Cell disease 


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  1. 1.
    Weatherall D. The inherited disorders of haemoglobin: an increasingly neglected global health burden. Indian J Med Res. 2011;134:493–497.PubMedGoogle Scholar
  2. 2.
    Mukherjee MB, Colah RB, Ghosh K, Mohanty D, Krishnamoorthy R. Milder clinical course of sickle cell disease in patients with alpha thalassemia in the Indian subcontinent. Blood. 1997;89:732.PubMedGoogle Scholar
  3. 3.
    Mohanty D, Mukherjee MB. Sickle cell disease in India. Curr Opin Hematol. 2002;9:117–122.PubMedCrossRefGoogle Scholar
  4. 4.
    Mashon RS, Dash PM, Khalko J, Dash L, Mohanty PK, Patel S, et al. Higher fetal haemoglobin concentration in patients with sickle cell disease in Eastern India reduces the frequency of painful crisis. Eur J Haematol. 2009;83:383–384.PubMedCrossRefGoogle Scholar
  5. 5.
    Kar BC, Devi S. Clinical profile of sickle cell disease in Orissa. Indian J Pediatr. 1997;64:73–77.PubMedCrossRefGoogle Scholar
  6. 6.
    Kinney TR, Helms RW, O’Branski EE, Ohene-Frempong K, Wang W, Daeschner C, et al. Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group. Blood. 1999;94:1550–1554.Google Scholar
  7. 7.
    Steinberg MH, McCarthy WF, Castro O, Ballas SK, Armstrong FD, Smith W, et al. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia and MSH Patients’ Follow-Up. The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up. Am J Hematol. 2010;85:403–408.PubMedGoogle Scholar
  8. 8.
    Italia K, Jain D, Gattani S, Jijina F, Nadkarni A, Sawant P, et al. Hydroxyurea in sickle cell disease-a study of clinicopharmacological efficacy in the Indian haplotype. Blood Cells, Molecules and Diseases. 2009;42:25–31.PubMedCrossRefGoogle Scholar
  9. 9.
    Singh H, Dulhani N, Kumar BN, Singh P, Tiwari P. Effective control of sickle cell disease with hydroxyurea therapy. Indian J Pharmacol. 2010;42:32–35.PubMedCrossRefGoogle Scholar
  10. 10.
    Jain DL, Sarathi V, Desai S, Singh P, Tiwari P. Low fixed dose Hydroxyurea in severely affected Indian children with sickle cell disease. Hemoglobin. 2012;36:323–332.PubMedCrossRefGoogle Scholar
  11. 11.
    Coleman E, Inusa B. Sickle cell anaemia: targeting the role of fetal haemoglobin in therapy. Clinical Pediatrics (Phila). 2007;46:386–391.CrossRefGoogle Scholar
  12. 12.
    Zimmerman SA, Schultz WH, Davis JS, Pickens CV, Mortier NA, Howard TA, et al. Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease. Blood. 2004;103:2039–2045.PubMedCrossRefGoogle Scholar
  13. 13.
    Hankins JS, Ware RE, Rogers ZR, Wynn LW, Lane PA, Scott JP, et al. Long-term hydroxyurea therapy for infants with sickle cell anemia: the HUSOFT extension study. Blood. 2005;106:2269–2275.PubMedCrossRefGoogle Scholar
  14. 14.
    Patel DK, Mashon RS, Patel S, Das BS, Purohit P, Bishwal SC. Low dose hydroxyurea is effective in reducing the incidence of painful crisis and frequency of blood transfusion in sickle cell anemia patients from eastern India. Hemoglobin. 2012;36:409–420.PubMedCrossRefGoogle Scholar
  15. 15.
    Svarch E, Machín S, Nieves RM, Mancia de Reyes AG, Navarrete M, Rodríguez H. Hydroxyurea treatment in children with sickle cell anemia in Central America and the Caribbean countries. Pediatr Blood Cancer. 2006;47: 111–112.PubMedCrossRefGoogle Scholar
  16. 16.
    Al-Nood HA, Al-Khawlani MM, Al-Akwa A. Fetal hemoglobin response to hydroxyurea in Yemeni sickle cell disease patients. Hemoglobin. 2011;35:13–21.PubMedCrossRefGoogle Scholar
  17. 17.
    Strouse JJ, Heeney MM. Hydroxyurea for the treatment of sickle cell disease: efficacy, barriers, toxicity, and management in children. Pediatr Blood Cancer. 2012;59:365–371.PubMedCrossRefGoogle Scholar
  18. 18.
    Wang WC, Ware RE, Miller ST, Iyer RV, Casella JF, Minniti CP, et al; BABY HUG investigators. Hydroxycarbamide in very young children with sicklecell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet. 2011;377:1663–1672.PubMedCrossRefGoogle Scholar
  19. 19.
    Green NS, Barral S. Genetic modifiers of HbF and response to hydroxyurea in sickle cell disease. Pediatric Blood Cancer. 2011;56:177–181.PubMedCrossRefGoogle Scholar
  20. 20.
    Flanagan JM, Steward S, Howard TA, Mortier NA, Kimble AC, Aygun B, et al. Hydroxycarbamide alters erythroid gene expression in children with sickle cell anaemia. British J Haematol. 2012;157:240–248.CrossRefGoogle Scholar
  21. 21.
    Ware RE, Eggleston B, Redding-Lallinger R, Wang WC, Smith-Whitley K, Daeschner C, et al. Predictors of fetal hemoglobin response in children with sickle cell anemia receiving hydroxyurea therapy. Blood. 2002;99:10–14.PubMedCrossRefGoogle Scholar
  22. 22.
    Kattamis A, Lagona E, Orfanou I, Psichou F, Ladis V, Kanavakis E, et al. Clinical response and adverse events in young patients with sickle cell disease treated with hydroxyurea. Pediatric Hematology Oncology. 2004;21: 335–3342.PubMedCrossRefGoogle Scholar
  23. 23.
    Ware RE. How I use hydroxyurea to treat young patients with sickle cell anemia. Blood. 2010;115:5300–5311.PubMedCrossRefGoogle Scholar

Copyright information

© Indian Academy of Pediatrics 2013

Authors and Affiliations

  • Dipti L. Jain
    • 1
    • 3
    Email author
  • Mohini Apte
    • 1
  • Roshan Colah
    • 2
  • Vijaya Sarathi
    • 1
  • Saumil Desai
    • 1
  • Amruta Gokhale
    • 1
  • Amol Bhandarwar
    • 1
  • Harshwardhan L. Jain
    • 1
  • Kanjaksha Ghosh
    • 2
  1. 1.Department of PediatricsGovernment Medical CollegeNagpurIndia
  2. 2.National Institute of ImmunohaematologyMumbaiIndia
  3. 3.Department of PediatricsGovernement Medical CollegeNagpurIndia

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