HYDROXYCARBAMINE: FROM AN OLD DRUG USED IN MALIGNANT HEMOPATHIES TO A CURRENT STANDARD IN SICKLE CELL DISEASE
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Keywords
hydroxyurea, treatment, sickle cell anemia, clinical management, hemoglobinopathies, prognosis.
Abstract
While hydroxycarbamine (hydroxyurea, HU) has less and less indications in malignant hemopathies, it represents the only widely used drug which modifies sickle cell disease pathogenesis. Clinical experience with HU for patients with sickle cell disease has been accumulated over the past 25 years in Western countries. The review of the literature provides increasing support of safety and efficacy in both children and adults for reducing acute vaso-occlusive events including pain episodes and acute chest syndrome. HU has become the standard-of-care for sickle cell anemia, but remains underused. Barriers to its use should be identified and overcome.
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References
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3. Stearns B, Losee KA, Bernstein J. Hydroxyurea: a new type of potential antitumor agent. J Med Chem 1963; 6:201.
4. Fishbein WN, Carbone PP, Freireich EJ, et al. Clinical trials of hydroxyurea in patients with cancer and leukemia. Clin Pharmacol Ther 1964; 5:574-80.
5. Becloff GL. Pharmacological, metabolic and clinical experience with hydroxyurea. Clin Trials J 1967; 4:873-83.
6. Kennedy BJ. Hydroxyurea therapy in chronic myelogenous leukemia.Cancer 1972; 29:1052-6.
7. Tefferi A, Pardanani A. Myeloproliferative neoplasms. A contemporary review. JAMA Oncol 2015; 1:97-105.
8. Cortelazzo S, Finazzi G, Specchia G, et al. Hydroxyurea for patients with essential thrombocythemia and a high risk of thrombosis. N Engl J Med 1995; 332:1132-6.
9. Fruchtman SM, Mack K, Kaplan ME, et al. From efficacy to safety: a Polycytemia Vera Study Group report on hydroxyurea in patients with polycytemia vera. Semin Hematol 1997; 34:17-23.
10. Tefferi A. Primary myelofibrosis: 2014 update on diagnosis, risk-stratification, and management. Am J Hematol 2014; 89:915-25.
11. Leavell UW, Yarbro JW. Hydroxyurea. A new treatment for psoriasis. Arch Dermatol 1970; 102:144-50.
12. Herrick JB. Peculiar elongated and sickle-shaped red blood corpuscules in a case of severe anemia. Arch Intern Med 1910; 6:517-21.
13. Pauling L, ItanoHA.Sickle cell anemia, a molecular disease. Science 1949; 109:443.
14. Allison AC.The distribution of the sickle-cell trait in East Africa and elsewhere, and its apparent relationship to the incidence of subtertianmalaria.Trans R SocTrop Med Hyg 1954; 48:312-8.
15. Taylor SM, Parobeck CM, Fairhurst RM. Haemoglobinopathies and the clinical epidemiology of malaria: a systematic review and meta-analysis. Lancet Infect Dis 2012; 12:457-68.
16. Ingram VM. A specific chemical difference between the globins of normal human and sickle-cell anaemiahaemoglobin.Nature 1956; 178:792-4.
17. Schmidt RM, Brosious EM. Evaluation of proficiency in the performance of tests for abnormal hemoglobins.Am J ClinPathol 1974; 62:664-9.
18. Scott RB. Health care priority and sickle cell anemia. JAMA 1970; 214:731-4.
19. Benson JM, Therrell BL Jr. History and current status of newborn screening for hemoglobinopathies. SeminPerinatol 2010; 34: 134-44.
20. Quinn CT, Rogers ZR, McCavit TL, Buchanan GR. Improved survival of children and adolescents with sickle cell disease. Blood 2010; 115:3447-52.
21. Naik RP, Derebail VK, Grams ME, et al. Association of sickle cell trait with chronic kidney disease and albuminuria in African Americans. JAMA 2014; 312:2115-25.
22. Auer PL, Johnsen JM, Johnson AD, et al. Imputation of exome sequence variants into population-based samples and blood-cell-trait associated loci in African Americans: NHLBI GO Exome Sequencing Project. Am J Hum Genet 2012; 91:794-808.
23. Platt OS, Orkin SH, Dover G, et al. Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia. J Clin Invest 1984 ; 74 :652-6.
24. Platt OS, Thorington BD, Brambilla DJ, et al. Pain in sickle cell disease: rates and risk factors. N Engl J Med 1991; 325:11-6.
25. Castro O, Brambilla DJ, Thorington BD, et al. The acute chest syndrome in sickle cell disease: incidence and risk factors.The Cooperative Study of Sickle Cell Disease.Blood 1994; 84:643-9.
26. Gladwin MT, Vichinsky E. Pulmonary complications of sickle cell disease. N Engl J Med 2008; 359:2254-65.
27. Platt OS, Brambilla DJ, Rosse WF, et al. Mortality in sickle cell disease: life expectancy and risk factors for early death. N Engl J Med 1994; 330:1639-44.
28. Charache S, Terrin ML, Moore RD, et al. Effects of hydroxyurea on the frequency of painful crises in sickle cell anemia. N Engl J Med 1995; 332:1317-22.
29. Segal JB, Strouse JJ, Beach MC, et al. Hydroxyurea for the treatment of sickle cell disease.Evid Rep Technol Assess 2008; 165:1-95.
30. Brawley OW, Cornelius LJ, Edwards LR, et al. National Institutes of Health Consensus Development Conference statement: hydroxyurea treatment for sickle cell disease. Ann Intern Med 2008; 148:932-8.
31. Franco RS, Yasin Z, Palascak MB, et al. The effect of fetal hemoglobin on the survival characteristics of sickle cells.Blood 2006; 108:1073-6.
32. Platt OS.Hydroxyurea for the treatmet of sickle cell anemia. N Engl J Med 2008; 358:1362-9.
33. Cokic VP, Smith RD, Beleslin-Cokic BB, et al. Hydroxyurea induces fetal hemoglobin by the nitric oxide-dependent activation of soluble guanylylcyclase. J Clin Invest 2003; 111:231-9.
34. Letvin NL, Linch DC, Beardsley GP, McIntyre KW, Nathan DG. Augmentation of fetal-hemoglobin production in anemic monkeys by hydroxyurea. N Engl J Med 1984; 310:869-73.
35. Wong TE, Brandow AM, Lim W, Lottenberg R. Update on the use of hydroxyurea therapy in sickle cell disease. Blood 2014; 124:3850-7.
36. Buckner TW, Ataga KI. Does hydroxyurea prevent pulmonary complications of sickle cell disease? Hematology (Educational Sessions of the American Society of Hematology) 2014; 432-7.
37. Wang WC, Ware RE, Miller ST, et al. Hydroxycarbamine in very young children with sickle-cell anaemia: a multicentre, randomized, controlled trial (BABY HUG). Lancet 2011; 377:1663-72.
38. Ware RE, Helms RW. Stroke with transfusions changing to hydroxyurea (SWiTCH). Blood 2012; 119:3925-32.
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