Page 40 - 2019_09-HaematologicaMondo-web

M.C. Carden and J. Little et al.
silencing through the transcriptional repro- gramming of adult hematopoietic progeni- tors. Blood. 2016;127(11):1481-1492.
38. Kutlar A, Swerdlow PS, Meiler SE, et al. Pomalidomide in sickle cell disease: phase I study of a novel anti-switching agent. Blood. 2013;122(21):777.
39. Cui S, Lim KC, Shi L, et al. The LSD1 inhibitor RN-1 induces fetal hemoglobin synthesis and reduces disease pathology in sickle cell mice. Blood. 2015;126(3):386-396.
40. Zhang Y, Paikari A, Sumazin P, et al. Metformin induces FOXO3-dependent fetal hemoglobin production in human primary erythroid cells. Blood. 2018;132(3):321-333.
41. Lew VL, Bookchin RM. Ion transport pathology in the mechanism of sickle cell dehydration. Physiol Rev. 2005;85(1):179- 200.
42. Nash GB, Johnson CS, Meiselman HJ. Mechanical properties of oxygenated red blood cells in sickle cell (HbSS) disease. Blood. 1984;63(1):73-82.
43. Zhou GP, Anderson KP, Joiner CH, Gallagher PG. Modification of erythrocyte hydration in the treatment of sickle cell disease. Blood Cells Mol Dis. 2001;27(1):65-68.
44. Joiner CH, Jiang M, Claussen WJ, Roszell NJ, Yasin Z, Franco RS. Dipyridamole inhibits sickling-induced cation fluxes in sickle red blood cells. Blood. 2001;97(12):3976-3983.
45. Ataga KI, Reid M, Ballas SK, et al. Improvements in haemolysis and indicators of erythrocyte survival do not correlate with acute vaso-occlusive crises in patients with sickle cell disease: a phase III randomized, placebo-controlled, double-blind study of the Gardos channel blocker senicapoc (ICA- 17043). Br J Haematol. 2011;153(1):92-104.
46. De Franceschi L, Bachir D, Galacteros F, et al. Oral magnesium supplements reduce ery- throcyte dehydration in patients with sickle cell disease. J Clin Invest. 1997;100(7):1847- 1852.
47. Brousseau DC, Scott JP, Badaki-Makun O, et al. A multicenter randomized controlled trial of intravenous magnesium for sickle cell pain crisis in children. Blood. 2015;126(14): 1651-1657.
48. Lehrer-Graiwer J, Howard J, Hemmaway CJ, et al. GBT440, a potent anti-sickling hemoglobin modifier reduces hemolysis, improves anemia and nearly eliminates sick- le cells in peripheral blood of patients with sickle cell disease. Blood. 2015;126(23):542.
49. Howard J, Hemmaway CJ, Telfer P, et al. A phase 1/2 ascending dose study and open- label extension study of voxelotor in patients with sickle cell disease. Blood. 2019;133(17):1865-1875.
50. Vichinsky E, Hoppe C, Howard J, et al. Results from part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) trial (GBT440-031), a placebo-controlled randomized study eval- uating voxelotor (GBT440) in adults and adolescents with sickle cell disease. Blood. 2018;132(Suppl 1):505.
51. Kato G, Lawrence MP, Mendelsohn L, et al. Phase 1 clinical trial of the candidate anti- sickling agent Aes-103 in adults with sickle cell anemia. Blood. 2013;122(21):1009.
52. Abuchowski A. PEGylated Bovine carboxy- hemoglobin (SANGUINATE): results of clin- ical safety testing and use in patients. Adv Exp Med Biol. 2016;876:461-467.
53. Misra H, Bainbridge J, Berryman J, et al. A phase Ib open label, randomized, safety study of SANGUINATE in patients with sickle cell anemia. Rev Bras Hematol Hemoter. 2017;39(1):20-27.
54. Buontempo P, Jubin RG, Buontempo C, et al.
Pegylated carboxyhemoglobin bovine (SANGUINATETM) restores RBCs round- ness and reduces pain during a sickle cell vaso-occlusive crisis Blood. 2017;130(Suppl 1):969.
55. Swift R, Abdulmalik O, Chen Q, et al. SCD- 101: a new anti-sickling drug reduces pain and fatigue and improves red blood cell shape in peripheral blood of patients with sickle cell disease. Blood. 2016;128(22):121.
56. Jagadeeswaran R, Rivers A. Evolving treat- ment paradigms in sickle cell disease. Hematology Am Soc Hematol Educ Program. 2017;2017(1):440-446.
57. Quinn CT. l-Glutamine for sickle cell ane- mia: more questions than answers. Blood. 2018;132(7):689-693.
58. Niihara Y, Miller ST, Kanter J, et al. A phase 3 trial of l-glutamine in sickle cell disease. N Engl J Med. 2018;379(3):226-235.
59. Minniti CP. l-glutamine and the dawn of combination therapy for sickle cell disease. N Engl J Med. 2018;379(3):292-294.
60. Pace BS, Shartava A, Pack-Mabien A, Mulekar M, Ardia A, Goodman SR. Effects of N-acetylcysteine on dense cell formation in sickle cell disease. Am J Hematol. 2003;73(1):26-32.
61. Sins JWR, Fijnvandraat K, Rijneveld AW, et al. Effect of N-acetylcysteine on pain in daily life in patients with sickle cell disease: a ran- domised clinical trial. Br J Haematol. 2018;182(3):444-448.
62. Chen J, Reheman A, Gushiken FC, et al. N- acetylcysteine reduces the size and activity of von Willebrand factor in human plasma and mice. J Clin Invest. 2011;121(2):593-603.
63. Sins JWR, Schimmel M, Luken BM, et al. Dynamics of von Willebrand factor reactivi- ty in sickle cell disease during vaso-occlusive crisis and steady state. J Thromb Haemost. 2017;15(7):1392-1402.
64. Hebbel RP, Boogaerts MA, Eaton JW, Steinberg MH. Erythrocyte adherence to endothelium in sickle-cell anemia. A possi- ble determinant of disease severity. N Engl J Med. 1980;302(18):992-995.
65. Takehara K, Soma Y, Igarashi A, Kikuchi K, Moro A, Ishibashi Y. Response of scleroder- ma fibroblasts to various growth factors. Arch Dermatol Res. 1991;283(7):461-464.
66. Chen G, Chang J, Zhang D, Pinho S, Jang JE, Frenette PS. Targeting Mac-1-mediated leukocyte-RBC interactions uncouples the benefits for acute vaso-occlusion and chron- ic organ damage. Exp Hematol. 2016;44(10):940-946.
67. Telen MJ. Erythrocyte adhesion receptors: blood group antigens and related molecules. Transfus Med Rev. 2005;19(1):32-44.
68. Telen MJ. Cellular adhesion and the endothelium: E-selectin, L-selectin, and pan- selectin inhibitors. Hematol Oncol Clin North Am. 2014;28(2):341-354.
69. Okpala I. Investigational selectin-targeted therapy of sickle cell disease. Expert Opin Investig Drugs. 2015;24(2):229-238.
70. Kutlar A, Embury SH. Cellular adhesion and the endothelium: P-selectin. Hematol Oncol Clin North Am. 2014;28(2):323-339.
71. Koehl B, Nivoit P, El Nemer W, et al. The endothelin B receptor plays a crucial role in the adhesion of neutrophils to the endothe- lium in sickle cell disease. Haematologica. 2017;102(7):1161-1172.
72. Hines PC, Zen Q, Burney SN, et al. Novel epinephrine and cyclic AMP-mediated acti- vation of BCAM/Lu-dependent sickle (SS) RBC adhesion. Blood. 2003;101(8):3281- 3287.
73. De Castro LM, Zennadi R, Jonassaint JC, Batchvarova M, Telen MJ. Effect of propra-
nolol as antiadhesive therapy in sickle cell
disease. Clin Transl Sci. 2012;5(6):437-444. 74. Kutlar A, Ataga KI, McMahon L, et al. A potent oral P-selectin blocking agent improves microcirculatory blood flow and a marker of endothelial cell injury in patients with sickle cell disease. Am J Hematol.
2012;87(5):536-539.
75. Ataga KI, Kutlar A, Kanter J, et al.
Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017;376(5):429-439.
76. Telen MJ, Wun T, McCavit TL, et al. Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use. Blood. 2015;125(17):2656-2664.
77. Jang JE, Hidalgo A, Frenette PS. Intravenous immunoglobulins modulate neutrophil acti- vation and vascular injury through FcgammaRIII and SHP-1. Circ Res. 2012;110(8):1057-1066.
78. Manwani D, Chen G, Carullo V, et al. Single-dose intravenous gammaglobulin can stabilize neutrophil Mac-1 activation in sick- le cell pain crisis. Am J Hematol. 2015;90(5):381-385.
79. Kato GJ, Steinberg MH, Gladwin MT. Intravascular hemolysis and the pathophys- iology of sickle cell disease. J Clin Invest. 2017;127(3):750-760.
80. Maitre B, Djibre M, Katsahian S, et al. Inhaled nitric oxide for acute chest syn- drome in adult sickle cell patients: a random- ized controlled study. Intensive Care Med. 2015;41(12):2121-2129.
81. Gladwin MT, Kato GJ, Weiner D, et al. Nitric oxide for inhalation in the acute treat- ment of sickle cell pain crisis: a randomized controlled trial. JAMA. 2011;305(9):893-902.
82. Minniti CP, Gorbach AM, Xu D, et al. Topical sodium nitrite for chronic leg ulcers in patients with sickle cell anaemia: a phase 1 dose-finding safety and tolerability trial. Lancet Haematol. 2014;1(3):e95-e103.
83. Connor JL, Jr., Sclafani JA, Kato GJ, Hsieh MM, Minniti CP. Brief topical sodium nitrite and its impact on the quality of life in patients with sickle leg ulcers. Medicine (Baltimore). 2018;97(46):e12614.
84. Waugh WH, Daeschner CW, 3rd, Files BA, McConnell ME, Strandjord SE. Oral cit- rulline as arginine precursor may be benefi- cial in sickle cell disease: early phase two results. J Natl Med Assoc. 2001;93(10):363- 371.
85. Morris CR, Kuypers FA, Lavrisha L, et al. A randomized, placebo-controlled trial of argi- nine therapy for the treatment of children with sickle cell disease hospitalized with vaso-occlusive pain episodes. Haematologica. 2013;98(9):1375-1382.
86. Stasch JP, Evgenov OV. Soluble guanylate cyclase stimulators in pulmonary hyperten- sion. Handb Exp Pharmacol. 2013;218:279- 313.
87. Narang BK, Roy S, Sharma R, Singh V, Rawal RK. Riociguat as a treatment regime for pul- monary arterial hypertension: a review. Clin Exp Hypertens. 2015;37(4):323-331.
88. Bereal-Williams C, Machado RF, McGowan V 2nd, et al. Atorvastatin reduces serum cholesterol and triglycerides with limited improvement in vascular function in adults with sickle cell anemia. Haematologica. 2012;97(11):1768-1770.
89. Hoppe C, Kuypers F, Larkin S, Hagar W, Vichinsky E, Styles L. A pilot study of the short-term use of simvastatin in sickle cell disease: effects on markers of vascular dys- function. Br J Haematol. 2011;153(5):655- 663.
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