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Transfusion practices and complications in thalassemia.

Lal A, Wong TE, Andrews J, Balasa VV, Chung JH, Forester CM, Ikeda AK, Keel SB, Pagano MB, Puthenveetil G, Shah SJ, Yu JC, Vichinsky EP.

Transfusion. 2018 Dec;58(12):2826-2835. doi: 10.1111/trf.14875. Epub 2018 Sep 27.


A Phase 3 Trial of l-Glutamine in Sickle Cell Disease.

Niihara Y, Miller ST, Kanter J, Lanzkron S, Smith WR, Hsu LL, Gordeuk VR, Viswanathan K, Sarnaik S, Osunkwo I, Guillaume E, Sadanandan S, Sieger L, Lasky JL, Panosyan EH, Blake OA, New TN, Bellevue R, Tran LT, Razon RL, Stark CW, Neumayr LD, Vichinsky EP; Investigators of the Phase 3 Trial of l-Glutamine in Sickle Cell Disease.

N Engl J Med. 2018 Jul 19;379(3):226-235. doi: 10.1056/NEJMoa1715971.


Sickle cell disease.

Kato GJ, Piel FB, Reid CD, Gaston MH, Ohene-Frempong K, Krishnamurti L, Smith WR, Panepinto JA, Weatherall DJ, Costa FF, Vichinsky EP.

Nat Rev Dis Primers. 2018 Mar 15;4:18010. doi: 10.1038/nrdp.2018.10. Review.


Fertility and Pregnancy in Women with Transfusion-Dependent Thalassemia.

Carlberg KT, Singer ST, Vichinsky EP.

Hematol Oncol Clin North Am. 2018 Apr;32(2):297-315. doi: 10.1016/j.hoc.2017.11.004. Review.


Dietary nonheme iron is equally bioavailable from ferritin or ferrous sulfate in thalassemia intermedia.

Khurana M, Fung EB, Vichinsky EP, Theil EC.

Pediatr Hematol Oncol. 2017 Nov;34(8):455-467. doi: 10.1080/08880018.2017.1395935. Epub 2017 Dec 12.


NNKTT120, an anti-iNKT cell monoclonal antibody, produces rapid and sustained iNKT cell depletion in adults with sickle cell disease.

Field JJ, Majerus E, Ataga KI, Vichinsky EP, Schaub R, Mashal R, Nathan DG.

PLoS One. 2017 Feb 2;12(2):e0171067. doi: 10.1371/journal.pone.0171067. eCollection 2017.


Emergency department utilization by Californians with sickle cell disease, 2005-2014.

Paulukonis ST, Feuchtbaum LB, Coates TD, Neumayr LD, Treadwell MJ, Vichinsky EP, Hulihan MM.

Pediatr Blood Cancer. 2017 Jun;64(6). doi: 10.1002/pbc.26390. Epub 2016 Dec 21.


Dysregulated arginine metabolism and cardiopulmonary dysfunction in patients with thalassaemia.

Morris CR, Kim HY, Klings ES, Wood J, Porter JB, Trachtenberg F, Sweeters N, Olivieri NF, Kwiatkowski JL, Virzi L, Hassell K, Taher A, Neufeld EJ, Thompson AA, Larkin S, Suh JH, Vichinsky EP, Kuypers FA; Thalassemia Clinical Research Network.

Br J Haematol. 2015 Jun;169(6):887-98. doi: 10.1111/bjh.13452. Epub 2015 Apr 24.


Is the medical home for adult patients with sickle cell disease a reality or an illusion?

Ballas SK, Vichinsky EP.

Hemoglobin. 2015;39(2):130-3. doi: 10.3109/03630269.2015.1023312. Epub 2015 Mar 25.


Emerging therapy in hemoglobinopathies: lessons from the past and optimism for the future.

Vichinsky EP.

Hematol Oncol Clin North Am. 2014 Apr;28(2):xiii-xviii. doi: 10.1016/j.hoc.2014.01.001. Epub 2014 Jan 28. No abstract available.


Elevated tricuspid regurgitant jet velocity in subgroups of thalassemia patients: insight into pathophysiology and the effect of splenectomy.

Singer ST, Kuypers F, Fineman J, Gildengorin G, Larkin S, Sweeters N, Rosenfeld H, Kurio G, Higa A, Jeng M, Huang J, Vichinsky EP.

Ann Hematol. 2014 Jul;93(7):1139-48. doi: 10.1007/s00277-014-2037-9. Epub 2014 Feb 28.


Neuroimaging abnormalities in adults with sickle cell anemia: associations with cognition.

Mackin RS, Insel P, Truran D, Vichinsky EP, Neumayr LD, Armstrong FD, Gold JI, Kesler K, Brewer J, Weiner MW; Neuropsychological Dysfunction and Neuroimaging Adult Sickle Cell Anemia Study Group.

Neurology. 2014 Mar 11;82(10):835-41. doi: 10.1212/WNL.0000000000000188. Epub 2014 Feb 12.


Validation and reliability of a disease-specific quality of life measure (the TranQol) in adults and children with thalassaemia major.

Klaassen RJ, Barrowman N, Merelles-Pulcini M, Vichinsky EP, Sweeters N, Kirby-Allen M, Neufeld EJ, Kwiatkowski JL, Wu J, Vickars L, Blanchette VS, Forgie M, Yamashita R, Wong-Rieger D, Young NL.

Br J Haematol. 2014 Feb;164(3):431-7. doi: 10.1111/bjh.12631. Epub 2013 Nov 3.


Zinc supplementation improves bone density in patients with thalassemia: a double-blind, randomized, placebo-controlled trial.

Fung EB, Kwiatkowski JL, Huang JN, Gildengorin G, King JC, Vichinsky EP.

Am J Clin Nutr. 2013 Oct;98(4):960-71. doi: 10.3945/ajcn.112.049221. Epub 2013 Aug 14.


Treatment of heart failure in adults with thalassemia major: response in patients randomised to deferoxamine with or without deferiprone.

Porter JB, Wood J, Olivieri N, Vichinsky EP, Taher A, Neufeld E, Giardina P, Thompson A, Moore B, Evans P, Kim HY, Macklin EA, Trachtenberg F.

J Cardiovasc Magn Reson. 2013 May 20;15:38. doi: 10.1186/1532-429X-15-38.


A randomized, placebo-controlled trial of arginine therapy for the treatment of children with sickle cell disease hospitalized with vaso-occlusive pain episodes.

Morris CR, Kuypers FA, Lavrisha L, Ansari M, Sweeters N, Stewart M, Gildengorin G, Neumayr L, Vichinsky EP.

Haematologica. 2013 Sep;98(9):1375-82. doi: 10.3324/haematol.2013.086637. Epub 2013 May 3.


Sildenafil therapy in thalassemia patients with Doppler-defined risk of pulmonary hypertension.

Morris CR, Kim HY, Wood J, Porter JB, Klings ES, Trachtenberg FL, Sweeters N, Olivieri NF, Kwiatkowski JL, Virzi L, Singer ST, Taher A, Neufeld EJ, Thompson AA, Sachdev V, Larkin S, Suh JH, Kuypers FA, Vichinsky EP; Thalassemia Clinical Research Network.

Haematologica. 2013 Sep;98(9):1359-67. doi: 10.3324/haematol.2012.082065. Epub 2013 Apr 12.


Application of multiplex ligation-dependent probe amplification to screen for β-globin cluster deletions: detection of two novel deletions in a multi ethnic population.

Cui J, Azimi M, Baysdorfer C, Vichinsky EP, Hoppe CC.

Hemoglobin. 2013;37(3):241-56. doi: 10.3109/03630269.2013.782461. Epub 2013 Apr 11.


Clinical manifestations of α-thalassemia.

Vichinsky EP.

Cold Spring Harb Perspect Med. 2013 May 1;3(5):a011742. doi: 10.1101/cshperspect.a011742. Review.


A phase 1/2 trial of HQK-1001, an oral fetal globin inducer, in sickle cell disease.

Kutlar A, Ataga K, Reid M, Vichinsky EP, Neumayr L, Blair-Britt L, Labotka R, Glass J, Keefer JR, Wargin WA, Berenson R, Perrine SP.

Am J Hematol. 2012 Nov;87(11):1017-21. doi: 10.1002/ajh.23306. Epub 2012 Aug 7.


The effect of whole body vibration therapy on bone density in patients with thalassemia: a pilot study.

Fung EB, Gariepy CA, Sawyer AJ, Higa A, Vichinsky EP.

Am J Hematol. 2012 Oct;87(10):E76-9. doi: 10.1002/ajh.23305. Epub 2012 Aug 7. No abstract available.


Inadequate dietary intake in patients with thalassemia.

Fung EB, Xu Y, Trachtenberg F, Odame I, Kwiatkowski JL, Neufeld EJ, Thompson AA, Boudreaux J, Quinn CT, Vichinsky EP; Thalassemia Clinical Research Network.

J Acad Nutr Diet. 2012 Jul;112(7):980-90. doi: 10.1016/j.jand.2012.01.017. Epub 2012 May 1.


Identification of three novel Hb F variants: Hb F-Hayward [Gγ1(NA1)Gly→Asp, GGT>GAT], Hb F-Chori-I [AγT16(A13)Gly→Asp, GGC>GAC] and Hb F-Chori-II [AγI29(B11)Gly→Glu, GGA>GAA].

Cui J, Baysdorfer C, Azimi M, Vichinsky EP, Hoppe CC.

Hemoglobin. 2012;36(3):305-9. doi: 10.3109/03630269.2012.659304. Epub 2012 Mar 2.


Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort.

Kwiatkowski JL, Kim HY, Thompson AA, Quinn CT, Mueller BU, Odame I, Giardina PJ, Vichinsky EP, Boudreaux JM, Cohen AR, Porter JB, Coates T, Olivieri NF, Neufeld EJ; Thalassemia Clinical Research Network.

Blood. 2012 Mar 22;119(12):2746-53. doi: 10.1182/blood-2011-04-344507. Epub 2012 Jan 25.


Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report.

Morris CR, Kim HY, Trachtenberg F, Wood J, Quinn CT, Sweeters N, Kwiatkowski JL, Thompson AA, Giardina PJ, Boudreaux J, Olivieri NF, Porter JB, Neufeld EJ, Vichinsky EP; Thalassemia Clinical Research Network.

Blood. 2011 Oct 6;118(14):3794-802. doi: 10.1182/blood-2010-11-319152. Epub 2011 Jul 19.


Reproductive capacity in iron overloaded women with thalassemia major.

Singer ST, Vichinsky EP, Gildengorin G, van Disseldorp J, Rosen M, Cedars MI.

Blood. 2011 Sep 8;118(10):2878-81. doi: 10.1182/blood-2011-06-360271. Epub 2011 Jul 14.


A pilot study of subcutaneous decitabine in β-thalassemia intermedia.

Olivieri NF, Saunthararajah Y, Thayalasuthan V, Kwiatkowski J, Ware RE, Kuypers FA, Kim HY, Trachtenberg FL, Vichinsky EP; Thalassemia Clinical Research Network.

Blood. 2011 Sep 8;118(10):2708-11. doi: 10.1182/blood-2011-03-341909. Epub 2011 Jun 23.


Darbepoetin alfa for the treatment of anaemia in alpha- or beta- thalassaemia intermedia syndromes.

Singer ST, Vichinsky EP, Sweeters N, Rachmilewitz E.

Br J Haematol. 2011 Jul;154(2):281-4. doi: 10.1111/j.1365-2141.2011.08617.x. Epub 2011 Apr 18. No abstract available.


Characterization of low bone mass in young patients with thalassemia by DXA, pQCT and markers of bone turnover.

Fung EB, Vichinsky EP, Kwiatkowski JL, Huang J, Bachrach LK, Sawyer AJ, Zemel BS.

Bone. 2011 Jun 1;48(6):1305-12. doi: 10.1016/j.bone.2011.03.765. Epub 2011 Apr 5.


Heterogeneity of hemoglobin H disease in childhood.

Lal A, Goldrich ML, Haines DA, Azimi M, Singer ST, Vichinsky EP.

N Engl J Med. 2011 Feb 24;364(8):710-8. doi: 10.1056/NEJMoa1010174.


Chart Card: feasibility of a tool for improving emergency department care in sickle cell disease.

Neumayr L, Pringle S, Giles S, Quirolo KC, Paulukonis S, Vichinsky EP, Treadwell MJ.

J Natl Med Assoc. 2010 Nov;102(11):1017-23.


Pulmonary hypertension in thalassemia.

Morris CR, Vichinsky EP.

Ann N Y Acad Sci. 2010 Aug;1202:205-13. doi: 10.1111/j.1749-6632.2010.05580.x.


Preface to Cooley's Anemia: Ninth Symposium.

Vichinsky EP, Neufeld EJ.

Ann N Y Acad Sci. 2010 Aug;1202:ix-x. doi: 10.1111/j.1749-6632.2010.05672.x. No abstract available.


Pulmonary hypertension and NO in sickle cell.

Gladwin MT, Barst RJ, Castro OL, Gordeuk VR, Hillery CA, Kato GJ, Kim-Shapiro DB, Machado R, Morris CR, Steinberg MH, Vichinsky EP.

Blood. 2010 Aug 5;116(5):852-4. doi: 10.1182/blood-2010-04-282095. No abstract available.


Transfusion and chelation practices in sickle cell disease: a regional perspective.

Vichinsky EP, Ohene-Frempong K, Thein SL, Lobo CL, Inati A, Thompson AA, Smith-Whitley K, Kwiatkowski JL, Swerdlow PS, Porter JB, Marks PW.

Pediatr Hematol Oncol. 2011 Mar;28(2):124-33. doi: 10.3109/08880018.2010.505506. Epub 2010 Jul 30.


Approaches to transfusion therapy and iron overload in patients with sickle cell disease: results of an international survey.

Vichinsky EP, Ohene-Frempong K; Transfusion Committee.

Pediatr Hematol Oncol. 2011 Feb;28(1):37-42. doi: 10.3109/08880018.2010.505497. Epub 2010 Jul 30.


Relationship between chronic transfusion therapy and body composition in subjects with thalassemia.

Fung EB, Xu Y, Kwiatkowski JL, Vogiatzi MG, Neufeld E, Olivieri N, Vichinsky EP, Giardina PJ; Thalassemia Clinical Research Network.

J Pediatr. 2010 Oct;157(4):641-7, 647.e1-2. doi: 10.1016/j.jpeds.2010.04.064. Epub 2010 Jun 12.


Neuropsychological dysfunction and neuroimaging abnormalities in neurologically intact adults with sickle cell anemia.

Vichinsky EP, Neumayr LD, Gold JI, Weiner MW, Rule RR, Truran D, Kasten J, Eggleston B, Kesler K, McMahon L, Orringer EP, Harrington T, Kalinyak K, De Castro LM, Kutlar A, Rutherford CJ, Johnson C, Bessman JD, Jordan LB, Armstrong FD; Neuropsychological Dysfunction and Neuroimaging Adult Sickle Cell Anemia Study Group.

JAMA. 2010 May 12;303(18):1823-31. doi: 10.1001/jama.2010.562.


Alpha thalassemia major--new mutations, intrauterine management, and outcomes.

Vichinsky EP.

Hematology Am Soc Hematol Educ Program. 2009:35-41. doi: 10.1182/asheducation-2009.1.35. Review.


Hemoglobin Hakkari: an autosomal dominant form of beta thalassemia with inclusion bodies arising from de novo mutation in exon 2 of beta globin gene.

Kanathezhath B, Hazard FK, Guo H, Kidd J, Azimi M, Kuypers FA, Vichinsky EP, Lal A.

Pediatr Blood Cancer. 2010 Feb;54(2):332-5. doi: 10.1002/pbc.22167.


Hydroxycarbamide-induced changes in E/beta thalassemia red blood cells.

Singer ST, Vichinsky EP, Larkin S, Olivieri N, Sweeters N, Kuypers FA; E/beta Thalassemia Study Group.

Am J Hematol. 2008 Nov;83(11):842-5. doi: 10.1002/ajh.21266.


Disparity in the management of iron overload between patients with sickle cell disease and thalassemia who received transfusions.

Fung EB, Harmatz PR, Milet M, Balasa V, Ballas SK, Casella JF, Hilliard L, Kutlar A, McClain KL, Olivieri NF, Porter JB, Vichinsky EP; Multi-Center Iron Overload Research Group.

Transfusion. 2008 Sep;48(9):1971-80. doi: 10.1111/j.1537-2995.2008.01775.x. Epub 2008 May 29.


Fracture prevalence and relationship to endocrinopathy in iron overloaded patients with sickle cell disease and thalassemia.

Fung EB, Harmatz PR, Milet M, Coates TD, Thompson AA, Ranalli M, Mignaca R, Scher C, Giardina P, Robertson S, Neumayr L, Vichinsky EP; Multi-Center Iron Overload Study Group.

Bone. 2008 Jul;43(1):162-168. doi: 10.1016/j.bone.2008.03.003. Epub 2008 Mar 15.


Clinical differences between children and adults with pulmonary hypertension and sickle cell disease.

Hagar RW, Michlitsch JG, Gardner J, Vichinsky EP, Morris CR.

Br J Haematol. 2008 Jan;140(1):104-12. Epub 2007 Oct 3.


Erythrocyte glutamine depletion, altered redox environment, and pulmonary hypertension in sickle cell disease.

Morris CR, Suh JH, Hagar W, Larkin S, Bland DA, Steinberg MH, Vichinsky EP, Shigenaga M, Ames B, Kuypers FA, Klings ES.

Blood. 2008 Jan 1;111(1):402-10. Epub 2007 Sep 11.


Iron homeostasis during transfusional iron overload in beta-thalassemia and sickle cell disease: changes in iron regulatory protein, hepcidin, and ferritin expression.

Jenkins ZA, Hagar W, Bowlus CL, Johansson HE, Harmatz P, Vichinsky EP, Theil EC.

Pediatr Hematol Oncol. 2007 Jun;24(4):237-43.


Physical therapy alone compared with core decompression and physical therapy for femoral head osteonecrosis in sickle cell disease. Results of a multicenter study at a mean of three years after treatment.

Neumayr LD, Aguilar C, Earles AN, Jergesen HE, Haberkern CM, Kammen BF, Nancarrow PA, Padua E, Milet M, Stulberg BN, Williams RA, Orringer EP, Graber N, Robertson SM, Vichinsky EP; National Osteonecrosis Trial in Sickle Cell Anemia Study Group.

J Bone Joint Surg Am. 2006 Dec;88(12):2573-82.


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