Treatment of two infants with Cooley's anemia with sodium phenylbutyrate

Annals of the New York Academy of Sciences

Volumn 850, Issue , 1998, Pages 452-454

  Macmillan, M L ^b   Fouladi, M ^b   Nisbet Brown, E ^b   Waye, J S ^b   Olivieri, N F ^a  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BUTYRIC ACID DERIVATIVE; SODIUM PHENYLBUTYRATE; UNCLASSIFIED DRUG;

CASE REPORT; CONFERENCE PAPER; FEMALE; HUMAN; MALE; NEWBORN; ORAL DRUG ADMINISTRATION; THALASSEMIA MAJOR;

EID: 0031872839     PISSN: 00778923     EISSN: None     Source Type: Book Series    
DOI: 10.1111/j.1749-6632.1998.tb10518.x     Document Type: Conference Paper

References (10)

1. OLIVIERI, N. F. 1996. Reactivation of fetal hemoglobin in patients with β-thalassemia. Sem. Hematol. 33 (1): 24-42.
2. PERRINE, S. P., M. F. GREENE & D. V. FALLER. 1985. Delay in the fetal globin switch in infants of diabetic mothers. N. Engl. J. Med. 312: 334-338.
3. BARD, H., & J. PROSMANNE. 1985. Relative rates of fetal hemoglobin and adult hemoglobin synthesis in cord blood of infants of insulin-dependent diabetic mothers. Pediatrics 75: 1143-1147.
4. GINDER, G. D., M. J. WHITTERS & J. K. POHLMAN. 1984. Activation of a chicken embryonic globin gene in adult erythroid cells by 5-azacytidine and sodium butyrate. Proc Natl. Acad. Sci. USA 81: 3954-3958.
5. GLAUBER, J. G. & J. A. WANDERSEE. 1991. Little 5'-flanking sequences mediate butyrate stimulation of embryonic globin gene expression in adult erythroid cells. Mol. Cell. Biol. 11: 4690-4697.
6. PACE, B., Q. LI, G. PETERSON & G. STAMATOYANNOPOULOS. 1994. α-amino butyric acid cannot reactivate the silenced γ-gene of the β-locus YAC transgenic mouse. Blood 84(12): 4344-4353.
7. PERRINE, S. P., G. D. GINDER, D. V. FALLER, G. H. DOVER, T. IKUTA, H. E. WITKOWSKA, S-P CAI, E. P. VICHINSKY & N. F. OLIVIERI. 1993. A short-term trial of butyrate to stimulate fetal-globin-gene expression in the β-globin disorders. N. Engl. J. Med. 328: 81-86.
8. COLLINS, A. F., H. A. PEARSON, P. GIARDINA, K. T. MCDONAGH, S. W. BRUSILOW & G. J. DOVER. 1995. Oral sodium phenylbutyrate therapy in homozygous β-thalassemia: A clinical trial. Blood 84(91): 43-49.
9. SHER, G. D., G. D. GINDER, J. LITTLE, S. YANG, G. J. DOVER & N. F. OLIVIERI. 1995. Extended therapy with intravenous arginine butyrate in patients with β-hemoglobinopathies. N. Engl. J. Med. 332: 1606-1610.
10. OLIVIERI, N. F., D. C. REES, G. D. GINDER, S. W. THEIN, J. S. WAYE, L. CHANG, G. M. BRITTENHAM & D. J. WEATHERALL. 1996. First report of long-term elimination of red cell transfusions in thalassemia major through augmentation of fetal hemoglobin during with sodium phenylbutyrate and hydroxyurea. Blood 88(Suppl 1): 310a.