The role of hydroxyurea in the management of sickle cell disease

Blood Reviews

Volumn 17, Issue 2, 2003, Pages 99-109

  Davies, Sally C ^a   Gilmore, Annette ^a  

^a CENTRAL MIDDLESEX HOSPITAL   (United Kingdom)

Author keywords

Crisis; HbF; Hydroxyurea; Sickle; Toxicity

Indexed keywords

BETA GLOBIN; HYDROXYUREA;

ARTICLE; AUTOSOMAL RECESSIVE INHERITANCE; BETA THALASSEMIA; DISEASE SEVERITY; DRUG EFFECT; GENETIC DISORDER; HETEROZYGOTE; HUMAN; LIFE EXPECTANCY; PREGNANCY; PRIORITY JOURNAL; SICKLE CELL ANEMIA; STEM CELL TRANSPLANTATION; VASCULAR ENDOTHELIUM;

EID: 0037409211     PISSN: 0268960X     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0268-960X(02)00074-7     Document Type: Article

References (125)

1. Hickman M, Modell B, Greengross P et al. Mapping the prevalence of sickle cell and β thalassaemia in England: recommended rates for local service planning. Br J Haem 1999; 104(4): 860-867.
2. Flint J, Harding RM, Boyce AJ, Clegg JB. The population genetics of the haemoglobinopathies. Baillieres Clin Haematol 1993; 6: 215-262.
3. 3 gene [Review] Baillieres Clin Haematol 1992; 5: 331-365.
4. Hood AT, Fabry ME, Costantini F, Nagel RL, Shear HL. Protection from lethal malaria in transgenic mice expressing sickle hemoglobin. Blood 1996; 87: 1600-1603.
5. Kaul DK, Fabry ME, Nagel RL. The pathophysiology of vascular obstruction in the sickle syndromes. Blood Rev 1996; 10: 29-44.
6. Brozovic M, Davies SC, Brownell AI. Acute admissions of patients with sickle cell disease who live in Britain. Br Med J 1987; 294: 1206-1208.
7. Fuggle P, Shand PAX, Gill LJ, Davies SC. Pain, quality of life, and coping in sickle cell disease. Arch Dis Child 1996; 75: 199-203.
8. Powers D, Wilson B, Imbus C, Pegelow C, Allen J. The natural history of stroke in sickle cell disease. Am J Med 1978; 65(3): 461-471.
9. Rogers DW, Clarke JM, Cupidore L, Ramlal A, Sparke BR, Serjeant GR. Early deaths in Jamaican children with sickle cell disease. Br Med J 1978; 1: 1515-1516.
10. Gray A, Anionwu EN, Davies SC, Brozovic M. Patterns of mortality in sickle cell disease the the United Kingdom. J Clin Pathol 1991; 44: 459-463.
11. Perronne V, Roberts-Harewood M, Bachir D et al. Patterns of mortality in sickle cell disease in adults in France and England. Haematol J 2002; 3: 56-60.
12. Leikin SL, Gallagher D, Kinney TR, Sloane D, Klug P, Rida W. Mortality in children and adolescents with sickle cell disease. Cooperative study of sickle cell disease. Pediatrics 1989; 84(3): 500-508.
13. 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: 1639-1644.
14. Steinberg MH, Ballas SK, Brunson CY, Bookchin R. Sickle cell anemia in septuagenarians. [Letter]. Blood 1995; 86: 3997-3998.
15. Wierenga KLL, Hambleton IR, Lewis NA. Survival estimates for patients with homozygous sickle-cell disease in Jamaica: a clinic-based population study. Lancet 2001; 357: 680-683.
16. Davies SC, Oni L. Management of patients with sickle cell disease. Br Med J 1997; 315: 656-660.
17. Davies SC, Roberts-Harewood M. Blood transfusion in sickle cell disease. Blood Rev 1997; 11: 57-71.
18. Bainbridge R, Higgs DR, Maude GH, Sarjeant GR. Clinical presentation of homezygous sickle cell disease. J Pediatr 1985; 106: 881-885.
19. Powars DR, Weiss JN, Chan LS, Schroeder WA. Is there a threshold level of fetal hemoglobin that ameliorates morbidity on sickle cell anaemia? Blood 1983; 63: 921-926.
20. Charache S. Fetal hemoglobin, sickling and sickle cell disease. Adv Pediatr 1990; 37: 1-31.
21. Gγ globin production. Blood 1985; 66: 786-787.
22. Nagel RL, Fabry ME, Pagnier J et al. Hematologically and genetically distinct forms of sickle cell anaemia in Africa. The Senegal type and the Benin type. N Engl J Med 1985; 312: 880-884.
23. Steinberg MH, Hsu H, Nagel RL et al. Gender and haplotype effects upon hematological manifestations of adult sickle cell anemia. Am J Hematol 1995; 48: 175-181.
24. Chang YC, Smith KD, Moore RD, Sarjeant GR, Dover GJ. An analysis of fetal hemoglobin variation in sickle cell disease: the' relative contributions of the X-linked factor, β-globin haplotypes, α-globin gene number, gender and age. Blood 1995; 85: 1111-1117.
25. DeSimone J, Heller P, Hall L, Zwiers D. 5-Azacytidine stimulates fetal hemoglobin synthesis in anemic baboons. Proc Natl Acad Sci USA 1982; 79: 4428-4431.
26. Platt OS, Orkin SH, Dover G, Beardsley GP, Miller B, Nathan DG. Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia. J Clin Invest 1984; 74: 652-656.
27. Papayannopoulou T, Torrealba-de Ron A, Veith R, Knitter G. Stamatoyannopoulos G:arabinosylcytosine induces fetal hemoglobin in baboons by perturbing erythroid cell differentiation kinetics. Science 1984; 224: 617-618.
28. Veith R, Galanello R, Papayannopoulou T, Stamatoyannopoulos G. Stimulation of F-cell production in patients with sickle-cell anemia treated with cytarabine or hydroxyurea. N Engl J Med 1985; 313: 1571-1575.
29. 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: 869-873.
30. Charache S, Dover GJ, Moore RD et al. Hydroxyurea: effects on hemoglobin F production in patients with sickle cell anemia. Blood 1992; 79: 2555-2565.
31. Rodgers GP, Dover GJ, Uyesaka N, Noguchi CT, Schechter AN, Nienhuis AW. Augmentation by erythropoietin of the fetal hemoglobin response to hydroxyurea in sickle cell disease. N Engl J Med 1993; 328: 73-80.
32. Charache S. Hydroxyurea as treatment for sickle cell anemia. [Review]. Hematol Oncol Clin North Am 1991; 5(3): 571-583.
33. Dover GJ, Humphries RK, Moore JG et al. Hydroxyurea induction of hemoglobin F production in sickle cell disease: relationship between cytotoxicity and F cell production. Blood 1986; 67(3): 735-738.
34. McDonagh KT, Dover GJ, Donahue RE et al. Hematopoietic growth factors and sodium butyrate. Exp Hemat 1992; 20: 1156-1164.
35. Charache S, Terrin M, Moore RD et al. Effect of hydroxyurea on the frequency of painful crisis in sickle cell anemia. Investigators of the Multicentre Study of Hydroxyurea in Sickle Cell Anemia. N Engl J Med 1995; 332: 1317-1322.
36. Charache S. Mechanism of action of hydroxyurea in the management of sickle cell anemia in adults. [Review]. Semin Hematol 1997; 34(3 Suppl. 3): 15-21.
37. Steinberg MH, Lu ZH, Barton FB, Terrin ML, Charache S, Dover GJ. Fetal hemoglobin in sickle cell anemia:determinants of response to hydroxyurea. Multicenter Study of Hydroxyurea. Blood 1997; 89(3): 1078-1088.
38. Charache S. Experimental therapy of sickle cell disease; use of hydroxyurea. Am J Pediatr Hematol Oncol 1994; 16: 62-66.
39. Orringer EP, Blythe DS, Johnson AE, Phillips GJ. Dover GJ, Parker JC. Effects of hydroxyurea on hemoglobin F and water content in the red blood cells of dogs and of patients with sickle cell anemia. Blood 1991; 78: 212-216.
40. Ballas SK, Dover GJ, Charache S. Effect of hydroxyurea on the rheological properties of sickle erythrocytes in vivo. Am J Hematol 1989; 32: 104-111.
41. Adragna NC, Fonseca P, Lauf PK. Hydroxyurea affects cell morphology, cation transport and red blood cell adhesion in cultured vascular endothelial cells. Blood 1994; 83: 553-560.
42. Charache S, Barton FB, Moore RD et al. Hydroxyurea and sickle cell anemia. Clinical utility of a myelosuppressive "switching" agent. The Multicenter Study of Hydroxyurea in Sickle Cell Anemia. Medicine 1996; 75(6): 300-326.
43. Zimmerman GA, Prescott SM, McIntyre TM. Endothelial cell interactions with granulocytes: tethering and signalling molecules. Immunol Today 1992; 13: 93-100.
44. Warren JS, Ward PA. The inflammatory response. In: Beutler E, Lichtman MA, Coller BS, Kipps TJ, Seligsohn U, eds. Williams Hematology, sixth ed. New York: Mc Graw-Hill; 2001: 67-75.
45. Weissmann G, Smolen JE, Hoffstein S. Polymorphonuclear leukocytes as secretory organs of inflammation. J Invest Dermatol 1978; 71: 95-99.
46. Hofstra TC, Kalra VK, Meiselman BJ, Coates TD. Sickle erythrocytes adhere to polymorphonuclear neutrophils and activate the neutrophil respiratory burst. Blood 1996; 87: 4440-4447.
47. Kim-Shapiro DB, King SB, Bonifant CL, Kolibash CP, Ballas SK. Time resolved absorption study of the reaction of hydroxyurea with sickle cell hemoglobin. Biochim Biophys Acta 1998; 1380(1): 64-74.
48. Pacelli R, Taira J, Cook JA, Wink DA, Krishna MC. Hydroxyurea reacts with heme proteins to generate nitric oxide [letter]. Lancet 1996; 347: 900.
49. Stolze K, Nohl H. EPR studies on the oxidation of hydroxyurea to paramagnetic compounds by oxyhemoglobin. Biochem Pharmacol 1990; 40: 799-802.
50. Jiang J, Jordan SJ, Bar DP, Gunther MR, Maeda H, Mason RP. In vivo production of nitric oxide in rats after administration of hydroxyurea. Mol Pharmacol 1997; 52: 1081-1086.
51. Glover RE, Ivy ED, Orringer EP, Maeda H, Mason RP. Detection of nitrosyl hemoglobin in venous blood in the treatment of sickle cell anaemia with hydroxyurea. Mol Pharmacol 1999; 55: 1006-1010.
52. Ikuta T, Ausenda S, Cappellini MD. Mechanism for fetal globin gene expression:role of the soluble guanylate cyclase-cGMP-dependent protein kinase pathway. Proc Natl Acad Sci USA 2001; 98: 1847-1852.
53. Gladwin MT, Schechter AN, Shelhamer JH et al. Inhaled nitric oxide augments transport of sickle cell hemoglobin without affecting oxygen affinity (see comments). J Clin Invest 1999; 104: 937-945.
54. Gladwin MT, Ognibene FP, Pannell LK et al. Relative role of heme nitrosylation and β-cysteine 93 nitrosation in the transport and metabolism of nitric oxide by hemoglobin in the human circulation. Proc Natl Acad Sci USA 2000; 97: 9943-9948.
55. Kaul DK, Hebbel RP. Hypoxia/reoxygenation causes inflammatory response in transgenic sickle mice but not in normal mice. J Clin Invest 2000; 106: 411-420.
56. Wolzt M, MacAllister RJ, Davis D et al. Biochemical characterization of S-nitrosohemoglobin. Mechanisms underlying synthesis, NO release, and biological activity In Process Citation. J Biol Chem 1999; 274: 28983-28990.
57. Garel MC, Domenget C, Caburi-Martin J, Prehu C, Galacteros F, Beuzard Y. Covalent binding of glutathione to hemoglobin I. Inhibition of hemoglobin S polymerization. J Biol Chem 1986; 261: 14704-14709.
58. Adams MR, Forsyth CJ, Jessup W, Robinson W, Robinson J, Celermajer DS. Oral L-arginine inhibits platlet aggregation but does not enhance endothelium-dependent dilation in healthy young men. J Am Coll Cardiol 1995; 26: 1054-1061.
59. Fox-Robichaud, Payne A, Hasan D et al. Inhaled NO as a viable antiadhesive therapy for ischemia/reperfusion injury of distal microvascular beds. J Clin Invest 1998; 101: 2497-2505.
60. Lerman A, Burnett JC, Higano ST, McKinley LJ, Holmes DR. Long-term L-arginnine supplementation improves small-vessel coronary endothelial function in humans. Circulation 1998; 97: 2123-2128.
61. + cell production to red cell survival in sickle cell disorder with and without hydroxyurea therapy. Blood 1999; 94(Suppl. 1): 421a.
62. Saleh AW, Hillen HF, Duits AJ. Levels of endothelial, neutrophil and platelet-specific factors in sickle cell anemia patients during hydroxyurea therapy. Acta Haematol 1999; 102: 31-37.
63. Styles LA, Lubin B, Vichinsky E et al. Decrease of very late activation antigen-4 and CD36 on reticulocytes in sickle cell patients treated with hydroxyurea. Blood 1997; 89: 2554-2559.
64. Saleh AW, Dults AJ, Gerbers A, de Vries C, Hillen HF. Cytokines and soluble adhesion molecules in sickle cell anemia patients during hydroxyurea therapy. Acta Haematol 1998; 100: 26-31.
65. Hillery CA, Du MC, Wang WC, Scott JP. Hydroxyurea therapy decreases the in vitro adhesion of sickle erythrocytes to thrombospondin and laminin. Br J Haematol 2000; 109: 322-327.
66. Eckman R, Casey S, Tomer A. [Abstract 1847] Sickle Cell Disease (SCD) patients treated with hydroxyurea have evidence of increased prothrombotic activity. Blood 1999; 94(10 Suppl. 1): 417a.
67. Nahavandi M, Perlin E, Kassim OO, Wyche MO, Castro O, Tavakkoli F. [Abstract 44] Upregulation of TNFα by hydroxyurea in patients with sickle cell anemia. Blood 2000; 96(11 Suppl. 1): 14a.
68. Papassotiriou I, Voskaridou E, Stamoulakatou A, Loukopoulos D. Increased erythropoietin level induced by hydroxyurea treatment of sickle cell patients. Hematol J 2000; 1: 295-300.
69. Davies SC, Olujohungbe A. Hydroxyurea for sickle cell disease. Cochrane Database Syst Rev 2001;(2): CD002202 (Review).
70. Schulz KF, Chalmers I, Hayes RJ, Altman DG. Empirical evidence of bias. Dimensions of methodological quality associated with estimates of treatment in controlled trials. JAMA 1995; 273: 408-412.
71. Ferster A, Vermylen C, Cornu G et al. Hydroxyurea for treatment of severe sickle cell anemia: a pediatric clinical trial. Blood 1996; 88(6): 1960-1964.
72. Ferster A, Tahriri, Vermylen P et al. Five years of experience with hydroxyurea in children and young adults with sickle cell disease. Blood 2001; 97(11): 3628-3632.
73. de Montalembert M, Belloy M, Bernaudin F, Gouraud F, Capdeville R, Mardini R et al. Three-year follow-up of hydroxyurea treatment in severely ill children with sickle cell disease. The French Study Group on Sickle Cell Disease. J Pediatr Hematol Oncol 1997; 19(4): 313-318.
74. El Alfy MS. Hydroxyurea in children with sickle cell anaemia, should it be a life long treatment? [Abstract]. Blood 2000; 96(11): 16b.
75. Jayabose S, Tugal O, Sandoval C et al. Clinical and hematologic effects of hydroxyurea in children with sickle cell anemia. J Pediatr 1996; 129: 559-565.
76. Kinney TR, Helms RW, O'Branski EE 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(5): 1550-1554.
77. Koren A, Segal-Kupershmit D, Zalman L et al. Effect of hydroxyurea in sickle cell anemia: a clinical trial in children and teenagers with severe sickle cell anemia and sickle cell β-thalassemia. Pediatr Hematol Oncol 1999; 16(3): 221-232.
78. Lima CS, Arruda VR, Costa FF, Saad ST. Minimal doses of hydroxyurea for sickle cell disease. Braz J Med Biol Res 1997; 30(8): 933-940.
79. Olivieri NF, Vichinsky EP. Hydroxyurea in children with sickle cell disease:impact on splenic function and compliance with therapy. J Pediatr Hematol Oncol 1998; 20: 26-31.
80. Vichinsky EP. Hydroxyurea in children: present and future [Review]. Semin Hematol 1997; 34(3 Suppl. 3): 22-29.
81. Oury AP, Hoyoux C, Dresse MF, Chantraine JM. Anemie falciforme chez l'enfant:interet de l'hydroxyuree dans les formes graves [Sickle cell anaemia in children:the value of hydroxyurea in severe forms]. Arch Pediatr 1997; 4: 839-844.
82. Scott JP, Hillery CA, Brown ER, Misiewicz V, Labotka RJ. Hydroxyurea therapy in children severely affected with sickle cell disease. J Pediatr 1996; 128(6): 820-828.
83. Ware RE, Zimmerman SA, Schultz WH. Hydroxyurea as an alternative to blood transfusions for the prevention of recurrent stroke in children with sickle cell disease. Blood 1999; 94: 3022-3026.
84. Hoppe C, Vichinsky E, Quirolo K, van Warmerdam J, Allen K, Styles L. Use of hydroxyurea in children ages 2 to 5 years with sickle cell disease. J Pediatr Hematol Oncol 2000; 22: 330-334.
85. Wang WC, Wynn LW, Rogers ZR, Scott JP, Lane PA, Ware RE. A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia. J Pediatr 2001; 139: 790-796.
86. Wang WC, Helms RW, Lynn HS et al. Effect of hydroxyurea on growth in children with sickle cell anemia: results of the HUG-KIDS study. J Pediatr 2002; 140: 225-229.
87. Claster S, Vichinsky E. First report of reversal of organ dysfunction in sickle cell anemia by the use of hydroxyurea: splenic regeneration. Blood 1996; 88: 1951-1953.
88. Rogers ZR. Hydroxyurea therapy for diverse pediatric populations with sickle cell disease. Semin Hematol 1997; 34(3 Suppl. 3): 42-47.
89. Horiuchi K, Golden JA, Das SK et al. Adverse effects of hydroxyurea on growth and development of young mice. Blood 1998; 92(10 Suppl. 1): 160a.
90. Bernaudin F, Freard E et al. [Abstract 1846] Hydroxyurea (HU) therapy improves cognitive functioning in sickle cell disease patients. Blood 1999; 94(10 Suppl. 1): 416a.
91. Vichinsky EP, Lubin BH. A cautionary note regarding hydroxyurea in sickle cell disease. Blood 1994; 83: 1124-1128.
92. Kutlar A, Woods KF, Clair B et al. Long term use of hydroxyurea in adults with sickle cell disease: a large single centre experience. Blood 2000; 96: 10a.
93. Daoud MS, Gibson LE, Pittelkow MR. Hydroxyurea dermopathy:a unique lichenoid eruption complicating long-term therapy with hydroxyurea. J Am Acad Dermatol 1998; 38: 781-782.
94. Najean Y, Rain JD. Treatment of polycythemia vera:the use of hydroxyurea and pipobroman in 292 patients under the age of 65 years. Blood 1997; 90: 3370-3377.
95. Velez A, Garcia-Aranda JM, Moreno JC. Hydroxyurea induced leg ulcers: is macroerythrocytosis a pathogenic factor? J Eur Acad Dermatol Venereol 1999; 12: 243-244.
96. DePass LR, Weaver EV. Comparison of teratogenic effects of aspirin and hydroxyurea in the Fischer 344 and Wistar strains. J Toxicol Environ Health 1982; 10(2): 297-305.
97. Ferm VH. Severe development malformations: malformations induced by urethane and hydroxyurea in the hamster. Arch Pathol 1966; 81: 174-177.
98. Diav-Citrin O, Hunnisett L, Sher GD, Koren G. Hydroxyurea use during pregnancy: case report in sickle cell disease and review of the literature. Am J Hematol 1999; 60: 148-150.
99. Evenson DP, Jost LK. Hydroxyurea exposure alters mouse testicular kinetics and sperm chromatin structure. Cell Prolif 1993; 26(2): 147-159.
100. Steinberg MH, Barton F, Castro O, Ramirez G, Bellevue R, Terrin M. [Abstract 2087] Hydroxyurea (HU) is associated with reduced mortality in adults with sickle cell anaemia. Blood 2000; 96(11 Suppl. 1): 485a.
101. de Montalembert M, Begue P, Bernaudin F, Thuret I, Bachir D, Micheau M. Preliminary report of a toxicity study of hydroxyurea in sickle cell disease. French Study Group on Sickle Cell Disease. Arch Dis Child 1999; 81(5): 437-439.
102. Moschovi M, Psychou F, Menegas D, Tsangaris GT, Tzortzatou-Stathopoulou F, Nikolaidou P. Hodgkin's disease in a child with sickle cell disease treated with hydroxyurea. Pediatr Hematol Oncol 2001; 18: 371-376.
103. Rauch A, Borromeo M, Ghafoor A, Khoyratty B, Maheshwari J. Leukemogenesis of hydroxyurea in the treatment of sickle cell anemia. Blood 1999; 94: 415a.
104. Wilson S. Acute leukemia in a patient with sickle-cell anemia treated with hydroxyurea. Ann Intern Med 2000; 133: 925-926.
105. Yardumian A. Personal communication, 2002.
106. Berk PD, Wasserman LR, Fruchtman SM, Goldberg JD. Treatment of polycythemia vera:a summary of clinical trails conducted by the Polycythemia Vera Study Group. In: Wassermain LR, Berlin NI, eds. Polycythemia vera and the myeloproliferation disorders. Philadelphia: WB Saunders; 1995: 166-194.
107. Fruchtman SM, Mack K, Kaplan ME, Peterson P, Berk PD, Wasserman LR. From efficacy to safety: a Polycythemia Vera Study group report on hydroxyurea in patients with polycythemia vera. Semin Hematol 1997; 34: 17-23.
108. Sterkers Y, Preudhomme C, Lai JL et al. Acute myeloid leukaemia and myelodysplastic syndromes following essential thrombocythemia treated with hydroxyurea: high proportion of cases with 17p deletion. Blood 1998; 91: 616-622.
109. Triadou P, Maier-Redelsperger M, Krishnamoorty R et al. Fetal haemoglobin variations following hydroxyurea treatment in patients with cyanotic congenital heart disease. Nouv Rev Fr Hematol 1994; 36: 367-372.
110. Hanft VN, Fruchtman SR, Pickens CV, Rosse WF, Howard TA, Ware RE. Acquired DNA mutations associated with in vivo hydroxyurea exposure. Blood 2000; 95: 3589-3593.
111. Voskaridou E, Kalotychou V, Loukopoulos D. Clinical and laboratory effects of long-term administration of hydroxyurea to patients with sickle-cell/beta-thalassaemia. Br J Haematol 1995; 89: 479-484.
112. Loukopoulos D, Voskaridou E, Kalotychou V, Schina M, Loutradi A, Theodoropoulos I. Reduction of the clinical severity of sickle cell/ beta-thalassemia with hydroxyurea: the experience of a single center in Greece. Blood Cells Mol Dis 2000; 26: 453-466.
113. Rigano P, Rodgers GP, Renda D, Renda MC, Aquino A, Maggio A. Clinical and hematological responses to hydroxyurea in Sicilian patients with Hb S/beta-thalassemia. Hemoglobin 2001; 25: 9-17.
114. Steinberg MH, Nagel RL, Brugnara C. Cellular effects of hydroxyurea in Hb SC disease. Br J Haematol 1997; 98: 838-844.
115. Miller MK, Zimmerman SA, Schultz WH, Ware RE. Hydroxyurea therapy for pediatric patients with hemoglobin SC disease. J Pediatr Hematol Oncol 2001; 23: 306-308.
116. Udden MM, Lo MN, Sears DA. Successful hydroxyurea treatment of a patient with SD hemoglobinopathy. Am J Hematol 1999; 60: 84-85.
117. Ware RE, Eggleston B, Redding-Lallinger R et al. Predictors of fetal hemoglobin response in children with sickle cell anaemia receiving hydroxyurea therapy. Blood 2002; 99: 10-14.
118. Mentzer WC, Smith FO, Sullivan KM. Impact of bone marrow transplantation for symptomatic SCD: an interim report. Multicenter investigation of bone marrow transplantation for SCD. Blood 2000; 95: 1918-1924.
119. Walters MC, Storb R, Patience M et al. Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicentre investigation of bone marrow transplantation for sickle cell disease. Blood 2000; 95: 1918-1924.
120. Bernaudin F, Souillet G, Vannie JP et al. Report of the French experience concerning 26 children transplanted for severe SCD. Bone Marrow Transplant 1997; 19(Suppl. 2): 112-115.
121. Vermylen C, Cornu G, Ferster A et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant 1998; 22: 1-6.
122. Ferster A, Corazza F, Vertongen F et al. Transplanted sickle-cell disease patients with autologous bone marrow recovery after graft failure develop increased levels of fetal haemoglobin which corrects disease severity. Br J Haematol 1995; 90: 804-808.
123. Locatelli F, Rocha V, Ertem M et al. Influence of preparative regimen and methotrexate GVHD prophylaxis on engraftment after related cord blood transplant for hemoglobinopathies. An Eurocord group analysis. Blood 2000; 96(Suppl. 1): 200a.
124. Yeager AM, Mehta PS, Adamkiewicz TV et al. Unrelated placental/ umbilical cord blood cell (UCBC) transplantation in children with high-risk sickle cell disease (SCD). Blood 2000; 96(Suppl. 2): 366b.
125. Rubinstein P, Carrier C, Scaradavou A et al. Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 1998; 339: 1565-1577.