Efficacy of Rapamycin as Inducer of Hb F in Primary Erythroid Cultures from Sickle Cell Disease and β-Thalassemia Patients

References

• Weatherall DJ, Clegg JB. Inherited haemoglobin disorders: An increasing global health problem. Bull World Health Organ. 2001;79(8):704–712
   PubMed Web of Science ®Google Scholar
• Borgna-Pignatti C, Cappellini MD, De Stefano P, et al. Survival and complications in thalassemia. Ann N Y Acad Sci. 2005;1054:40–47
   PubMed Web of Science ®Google Scholar
• Bunn HF. Pathogenesis and treatment of sickle cell disease. N Engl J Med. 1997;337(11):762–769
   PubMed Web of Science ®Google Scholar
• Noguchi C, Schechter AN, Rodgers GP. Sickle cells disease pathophysiology. In: Higgs DR, Weatherall DJ, Eds. Bailliére’s Clinical Haematology: The Haemoglobinophathies, Vol. 6. London, UK: Bailliére Tindall. 1993:57–91
   Google Scholar
• Perrine SP. Fetal globin stimulant therapies in the β-hemoglobinopathies: Principles and current potential. Pediatr Ann. 2008;37(5):229–346
   Google Scholar
• Lal A, Vichinsky E. The role of fetal hemoglobin-enhancing agents in thalassemia. Semin Hematol. 2004;41(6):17–22
   PubMed Web of Science ®Google Scholar
• Steinberg MH, Rodgers GP. Pharmacologic modulation of fetal haemoglobin. Medicine. 2001;80(5):328–344
   PubMed Web of Science ®Google Scholar
• Atweh G, Fathallah H. Pharmacologic induction of fetal hemoglobin production. Hematol Oncol Clin North Am. 2010;24(6):1131–1144
   PubMed Web of Science ®Google Scholar
• Platt OS. Hydroxyurea for the treatment of sickle cell anemia. N Engl J Med. 2008;358(13):1362–1369
   PubMed Web of Science ®Google Scholar
• Rigano P, Manfrè L, La Galla R, et al. Clinical and hematological response to hydroxyurea in a patient with Hb Lepore/β-thalassemia. Hemoglobin. 1997;21(3):219–226
   PubMed Web of Science ®Google Scholar
• Pecoraro A, Rigano P, Troia A, et al. Quantification of HBG mRNA in primary erythroid cultures: Prediction of the response to hydroxyurea in sickle cell and β-thalassemia. Eur J Haematol. 2013;92(1):66–72
   PubMed Web of Science ®Google Scholar
• Fucharoen S, Siritanaratkul N, Winichagoon P, et al. Hydroxyurea increases Hemoglobin F levels and improves the effectiveness of erythropoiesis in β-thalassemia/Hemoglobin E disease. Blood. 1996;87(3):887–892
   PubMed Web of Science ®Google Scholar
• Olivieri NF. Reactivation of fetal hemoglobin in patients with β-thalassemia. Semin Hematol. 1996;33(1):24–42
   PubMed Web of Science ®Google Scholar
• Bradai M. Hydroxyurea can eliminate transfusion requirements in children with severe β-thalassemia. Blood. 2003;102(4):1529–1530
   PubMed Web of Science ®Google Scholar
• Mancuso A, Maggio A, Renda D, et al. Treatment with hydroxycarbamide for intermedia thalassaemia: Decrease of efficacy in some patients during long-term follow up. Br J Haematol. 2006;133(1):103–108
   PubMed Web of Science ®Google Scholar
• Rigano P, Pecoraro A, Calzolari R, et al. Desensitization to hydroxycarbamide following long-term treatment of thalassaemia intermedia as observed in vivo and in primary erythroid cultures from treated patients. Br J Haematol. 2010;151(5):509–515
   PubMed Web of Science ®Google Scholar
• Sehgal SN. Sirolimus: Its discovery, biological properties, and mechanism of action. Transplant Proc. 2003;35(3 Suppl):S7–S14
   PubMed Web of Science ®Google Scholar
• Saunders RN, Metcalfe MS, Nicholson ML. Rapamycin in transplantation: A review of the evidence. Kidney Int. 2001;59(1):3–16
   PubMed Web of Science ®Google Scholar
• Mischiati C, Sereni A, Lampronti I, et al. Rapamycin-mediated induction of γ-globin mRNA accumulation in human erythroid cells. Br J Haematol. 2004;126(4):612–621
   PubMed Web of Science ®Google Scholar
• Fibach E, Bianchi N, Borgatti M, et al. Effects of rapamycin on accumulation of α-, β- and γ-globin mRNAs in erythroid precursor cells from β-thalassaemia patients. Eur J Haematol. 2006;77(5):437–441
   PubMed Web of Science ®Google Scholar
• Fibach E, Manor D, Oppenheim A, Rachmilewitz EA. Proliferation and maturation of human erythroid progenitors in liquid culture. Blood. 1989;73(1):100–103
   PubMed Web of Science ®Google Scholar
• Di Marzo R, Acuto S, Calzolari R, Maggio A. Allele-specific transcription of fetal genes in primary erythroid cell cultures from Lepore and δβ0 thalassemia patients. Exp Hematol. 2005;33(11):1363–1370
   PubMed Web of Science ®Google Scholar
• Quentmeier H, Zaborski M, Drexler HG. The human bladder carcinoma cell line 5637 constitutively secretes functional cytokines. Leuk Res. 1997;21(4):343–350
   PubMed Web of Science ®Google Scholar
• Calzolari R, Pecoraro A, Borruso V, et al. Induction of γ-globin gene transcription by hydroxycarbamide in primary erythroid cell cultures from Lepore patients. Br J Haematol. 2008;141(5):720–727
   PubMed Web of Science ®Google Scholar
• Smith RD, Li J, Noguchi CT, Schechter AN. Quantitative PCR analysis of HbF inducers in primary human adult erythroid cells. Blood. 2000;95(3):863–868
   PubMed Web of Science ®Google Scholar
• Lampronti I, Bianchi N, Borgatti M, et al. Accumulation of γ-globin mRNA in human erythroid cells treated with angelicin. Eur J Haematol. 2003;71(3):189–195
   PubMed Web of Science ®Google Scholar
• Gummert JF, Ikonen T, Morris RE. Newer immunosuppressive drugs: A review. J Am Soc Nephrol. 1999;10(6):1366–1380
   PubMed Web of Science ®Google Scholar
• Mahalati K, Kahan BD. Clinical pharmacokinetics of sirolimus. Clin Pharmacokinet. 2001;40(8):573–585
   PubMed Web of Science ®Google Scholar
• Italia K, Jijina F, Merchant R, et al. Comparison of in-vitro and in-vivo response to fetal hemoglobin production and γ-mRNA expression by hydroxyurea in hemoglobinopathies. Indian J Hum Genet. 2013;19(2):251–258
   PubMedGoogle Scholar
• Fibach E, Burkle LP, Schechter AN, et al. Hydroxyurea increases fetal hemoglobin in cultured erythroid cells derived from normal individuals and patients with sickle cell anemia or β-thalassemia. Blood. 2003;81(6):1630–1635
   Google Scholar
• Saxon BR, Rees D, Olivieri NF. Regression of extramedullary haemopoiesis and augmentation of fetal haemoglobin concentration during hydroxyurea therapy in β thalassaemia. Br J Haematol. 1998;101(3):416–419
   PubMed Web of Science ®Google Scholar