Patricia J. Giardina, M.D.

Pediatric Hematology-Oncology

Publications

  1. Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S. Therapeutic Hemoglobin Levels after Gene Transfer in β-Thalassemia Mice and in Hematopoietic Cells of β-Thalassemia and Sickle Cells Disease Patients. PLoS One. 2012;7(3):e32345. Epub 2012 Mar 27. PubMed PMID: 22479321; PubMed Central PMCID: PMC3314006.
  2. 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. Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort. Blood. 2012 Mar 22;119(12):2746-2753. Epub 2012 Jan 25. PubMed PMID: 22279056.
  3. Sheth S, Soff G, Mitchell B, Green D, Kaicker S, Fireman F, Tugal O, Guarini L, Giardina P, Aledort L. Managing incidentally diagnosed isolated factor VII deficiency perioperatively: a brief expert consensus report. Expert Rev Hematol. 2012 Feb;5(1):47-50. PubMed PMID: 22272705.
  4. 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. Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report. Blood. 2011 Oct 6;118(14):3794-802. Epub 2011 Jul 19.
  5. Rachmilewitz ER, Giardina PJ. How I Treat Thalassemia. Blood. Sept 29; 118 (13): 3479-88. Epub 2011 Aug 2.
  6. Olivieri NF, Saunthararajah Y, Thayalasuthan V, Kwiatkowski J, Ware RE, Kuypers FA, Kim HY, Trachtenberg FL and Vichinsky EP for the Thalassemia Clinical Research Network. Blood. 2011 Sep 8: 118(10):2708-11. Epub 2011 Jun 23.
  7. Trachtenberg F, Vichinsky E, Haines D, Pakbaz Z, Mednick L, Sobota A, Kwiatkowski J, Thompson AA, Porter J, Coates T, Giardina PJ, Olivieri N, Yamashita R, Neufeld EJ for the Thalassemia Clinical Research Network. Iron chelation adherence to deferoxamine and deferasirox in thalassemia. Am J Hematol. 2011 May;86(5):433-6. doi: 10.1002/ajh.21993.
  8. Thompson AA, Cunningham MJ, Singer ST, Neufeld EJ, Vichinsky E, Yamashita R, Giardina P, Kim HY, Trachtenberg F, Kwiatkowski JL; for the Thalassemia Clinical Research Network Investigators. Red cell alloimmunization in a diverse population of transfused patients with thalassaemia. Br J Haematol. 2011 Apr;153(1):121-128. doi: 10.1111/j.1365-2141.2011.08576.x. Epub 2011 Feb 17. PMID: 21323889
  9. Quinn CT, Johnson VL, Kim HY, Trachtenberg F, Vogiatzi MG, Kwiatkowski JL, Neufeld EJ, Fung E, Oliveri N, Kirby M, Giardina PJ; for the Thalassemia Clinical Research Network. Renal dysfunction in patients with thalassaemia. Br J Haematol. 2011 Apr;153(1):111-117. doi: 10.1111/j.1365-2141.2010.08477.x. Epub 2011 Feb 21. PMID: 21332704
  10. Wood JC, Glynos T, Thompson A, Giardina PJ, Harmatz P, Kang B, Paley C, Coates TD. Relationship between LPI, LIC, and cardiac response in a deferasirox monotherapy trial. Haematologica. 2011 Mar 10. [Epub ahead of print] PMID:
  11. Papapetrou EP, Lee G, Malani N, Setty M, Riviere I, Tirunagari LM, Kadota K, Roth SL, Giardina P, Viale A, Leslie C, Bushman FD, Studer L, Sadelain M: Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol. 2011 Jan;29(1):73-8. Epub 2010 Dec 12. PMID: 21151124
  12. Mednick L, Yu S, Trachtenberg F, Xu Y, Kleinert DA, Giardina PJ, Kwiatkowski JL, Foote D, Thayalasuthan V, Porter JB, Thompson AA, Schilling L, Quinn CT, Neufeld EJ, Yamashita R; Thalassemia Clinical Research Network. Symptoms of depression and anxiety in patients with thalassemia: prevalence and correlates in the thalassemia longitudinal cohort. Am J Hematol. 2010 Oct;85(10):802-5.PMID: 20806230
  13. Sobota A, Yamashita R, Xu Y, Trachtenberg F, Kohlbry P, Kleinert DA, Giardina PJ, Kwiatkowski JL, Foote D, Thayalasuthan V, Porter JB, Thompson AA, Schilling L, Quinn CT, Neufeld EJ; for the Thalassemia Clinical Research Network: Quality of life in thalassemia: A comparison of SF-36 results from the thalassemia longitudinal cohort to reported literature and the US norms. Am J Hematol. 2011 Jan;86(1):92-5. [Epub ahead of print - 2010 Sep 30] PMID: 21061309
  14. Gardenghi S, Ramos P, Marongiu MF, Melchiori L, Breda L, Guy E, Muirhead K, Rao N, Roy CN, Andrews NC, Nemeth E, Follenzi A, An X, Mohandas N, Ginzburg Y, Rachmilewitz EA, Giardina PJ, Grady RW, Rivella S. Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice. J Clin Invest. 2010 Dec 1;120(12):4466-77. doi: 10.1172/JCI41717. Epub 2010 Nov 22.PMID: 21099112
  15. Trachtenberg F, Foote D, Martin M, Carson S, Coates T, Beams O, Vega O, Merrelles-Pulcini M, Giardina PJ, Kleinert DA, Kwiatkowski J, Thompson AA, Neufeld EJ, Schilling L, Thayalasuthan V, Pakbaz Z, Yamashita R for the Thalassemia Clinical Research Network. Pain as an emergent issue in thalassemia. Am J Hematol. 2010;85:367-370. PMID: 20232403
  16. Wood JC, Glynos T, Thompson A, Giardina P, Harmatz P, Kang BP, Paley C, Coates TD. Follow-up report on the 2-year cardiac data from a deferasirox monotherapy trial. Am J Hematol. 2010 Oct;85(10):818-9.PMID: 20799360
  17. Arinsburg SA, Skerrett DL, Kleinert D, Giardina PJ, Cushing MM: The significance of a positive DAT in thalassemia patients. Immunohematology. 2010 Sep;26(3):87-91. PMID: 21214294
  18. Gardenghi S, Ramos P, Follenzi A, Rao N, Rachmilewitz EA, Giardina PJ, Grady RW, Rivella S. Hepcidin and Hfe in iron overload in beta-thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:221-5. Review.PMID: 20712796
  19. Breda L, Kleinert DA, Casu C, Casula L, Cartegni L, Fibach E, Mancini I, Giardina PJ, Gambari R, Rivella S. A preclinical approach for gene therapy of beta-thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:134-40.PMID: 20712784
  20. Galanello R, Agus A, Campus S, Danjou F, Giardina PJ, Grady RW. Combined iron chelation therapy. Ann N Y Acad Sci. 2010 Aug;1202:79-86.PMID: 20712777
  21. Sadelain M, Rivière I, Wang X, Boulad F, Prockop S, Giardina P, Maggio A, Galanello R, Locatelli F, Yannaki E. Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in beta-thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:52-8.PMID: 20712772
  22. Fung EB, Xu Y, Kwiatkowski JL, Vogiatzi MG, Neufeld E, Olivieri N, Vichinsky EP, Giardina PJ; Thalassemia Clinical Research Network. Relationship between Chronic Transfusion Therapy and Body Composition in Subjects with Thalassemia. J Pediatr. 2010 Jun 12. [Epub ahead of print] PMID: 20547400
  23. Tsay J, Yang Z, Ross FP, Cunningham-Rundles S, Lin H, Coleman R, Mayer-Kuckuk P, Doty SB, Grady RW, Giardina PJ, Boskey AL and Vogiatzi MG. Bone loss caused by iron overload in a murine model: importance of oxidative stress. Blood. 2010 Jun 16 [Epub ahead of print]
  24. Wood JC, Kang BP, Thompson A, Giardina P, Harmatz P, Glynos T, Paley C, Coates TD. The effect of Deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood. 2010 Jul 29; 116(4):537-43. Epub 2010 Apr 26.
  25. Pakbaz Z, Treadwell M, Kim HY, Trachtenberg F, Parmar N, Kwiatkowski JL, Cunningham MJ, Martin M, Sweeters N, Neufeld EJ, Giardina PJ, Olivieri N, Yamashita RC, Vichinsky E. Education and employment status of children and adults with thalassemia in North America. Pediatr Blood Cancer. 2010 Jun 9. [Epub ahead of print] PMID: 20535817
  26. Vogiatzi MG, Tsay J, Verdelis K, Rivella S, Grady RW, Doty S, Giardina PJ and Boskey AL. Changes in bone microarchitecture and biomechanical properties in the th3 thalassemia mouse are associated with decreased bone turnover and occur during the period of bone accrual. Calcif Tissue Int. 2010:(86)484-494.
  27. Singer ST, Kim HY, Olivieri NF, Kwiatkowski JL, Coates TD, Carson S, Neufeld E, Cunningham MJ, Giardina PJ, Mueller BU, Quinn CT, Fung E, Vichinsky E; Thalassemia Clinical Research Network. Hemoglobin H-constant spring in North America: an alpha thalassemia with frequent complications. Am J Hematol. 2009 Nov;84(11):759-61. PMID: 19787795
  28. Vogiatzi MG, Macklin EA, Trachtenberg FL, Fung EB, Cheung AM, Vichinsky E, Olivieri N, Kirby M, Kwiatkowski JL, Cunningham M, Holm IA, Fleisher M, Grady RW, Peterson CM and Giardina PJ for the Thalassemia Clinical Research Network. Differences in the Prevalence of growth, endocrine and vitamin D abnormalities among the various thalassemia syndromes in North America. Br J Haematol. 2009 Sep;146(5):546-56. Epub 2009 Jul 13. PMID: 19604241
  29. Vogiatzi MG, Macklin EA, Fung EB, Cheung AM, Vichinsky E, Olivieri N, Kirby M, Kwiatkowski JL, Cunningham M, Holm I, Lane J, Schneider R, Fleisher M, Grady RW, Peterson C, Giardina PJ; for the Thalassemia Clinical Research Network. Bone Disease in Thalassemia: A Frequent and Still Unresolved Problem. J Bone Miner Res. 2009 Mar;24(3):543-57. PMID: 18505376
  30. 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. Disparity in the management of iron overload between patients with sickle cell disease and thalassemia who received transfusions. Transfusion. 2008 Sep;48(9):1971-80. Epub 2008 May 29 PMID: 18513257
  31. Harmatz P, Jonas MM, Kwiatkowski J, Wright EC, Fischer R, Vichinsky E, Giardina PJ, Neufeld EJ, Porter J, Olivieri NF and the Thalassemia Clinical Research Network. Safety and efficacy of pegylated interferon alfa-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia. Haematologica. 2008 Aug;93(8):1247-51. Epub 2008 Jun 12. PMID: 18556414
  32. Libani IV, Guy EC, Melchiori L, Schiro R, Ramos P, Breda L, Scholzen T, Chadburn A, Liu YF, Kernbach M, Baron-Luhr B, Porotto, de Sousa M, Rachmilewitz EA, Hood JD, Cappellini MD, Giardina PJ, et al. Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia. Blood. 2008 Aug 1;112(3):875-85. Epub 2008 May 14. PMID: 18480424
  33. 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. Fracture prevalence and relationship to endocrinopathy in iron overloaded patients with sickle cell disease and thalassemia.Bone. 2008 Jul;43(1):162-8. Epub 2008 Mar 15. PMID: 18430624
  34. Walter PB, Macklin EA, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Vichinsky E, Olivieri N, Alberti D, Holland J, Harmatz P; Thalassemia Clinical Research Network. Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial. Haematologica. 2008 Jun;93(6):817-25. Epub 2008 May 10. PMID: 18469351
  35. Porter, J, Galanello R, Saglio G, Neufeld EJ, Vichinsky E, Cappellini MD, Olivieri N, Piga A, Cunningham MJ, Soulieres D, Gattermann N, Tchernia G, Maertens J, Giardina P, et al. Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study. Eur J Haematol. 2008 Feb;80(2):168-76. PMID: 18028431
  36. Sadelain M, Boulad F, Galanello R, Giardina P, Locatelli F, Maggio A, Rivella S, Riviere I, Tisdale J. Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy. Hum Gene Ther. 2007 Jan;18(1):1-9
  37. Gardenghi S, Marongiu MF, Ramos P, Guy E, Breda L, Chadburn A, Liu Y, Amariglio N, Rechavi G, Rachmilewitz EA, Breuer W, Cabantchik ZI, Wrighting DM, Andrews NC, de Sousa M, Giardina PJ, Grady RW, Rivella S. Ineffective erythropoiesis in b-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin. Blood. 2007 Jun 1;109(11):5027-35. Epub 2007 Feb 13.
  38. Harmatz P, Grady RW, Dragsten P, Vichinsky E, Giardina P, et al. Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): a novel long-acting iron chelator. Br J Haematol. 2007 Aug;138(3):374-81.
  39. Vogiatzi MG, Macklin EA, Fung EB, Vichinsky E, Olivieri N, Kwiatkowski J, Cohen A, Neufeld E, Giardina PJ for the Thalassemia Clinical Research Network: Prevalence of Fractures Among the Thalassemia Syndromes in North America. Bone, 2006, 38(4):571-575.
  40. Cappellini MD, Cohen A, Piga A, et. al.: A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood, May 2006; 107: 3455-3462.
  41. Breda L, Gardenghi S, Guy E, Rachmilewitz EA, Weizer-Stern O, Adamsky K, Amariglio N, Rechavi G, Giardina PJ, Grady RW, Rivella S. Exploring the Role of Hepcidin, an Antimicrobial and Iron Regulator Peptide, in Increased Iron Absorption in Beta-Thalassemia. Annals New York Academy of Science, 2005 Nov;1054:417-22.
  42. Vogiatzi MG, Autio KA, Mait JE, Schneider R, Lesser M, and Giardina PJ: Low Bone Mineral Density in Adolescents with Thalassemia. Annals of the New York Academy of Science, 2005 Nov;1054:462-6.
  43. Cunningham MJ, Macklin EA, Neufeld EJ and Cohen AR. Complications of b-thalassemia major in North America. Blood. Jul 1;104(1):34-39, 2004
  44. Vogiatzi MG, Autio KA, Schneider R and Giardina PJ: Low Bone Mass in Prepubertal Children with Thalassemia Major: Insights into the Pathogenesis of Low Bone Mass in Thalassemia. Journal of Pediatric Endocrinology and Metabolism, 17(10):1415-21, 2004
  45. Nisbet-Brown, E., Olivieri, N.F., Giardina, P.J., Grady, R.W., Neufeld, E.J., Sechaud, R., Krebs-Brown, A.J., Anderson, J.R., Alberti, D., Sizer, K.C., Nathan, D.G.: Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomized, double-blind, placebo-controlled, dose-escalation trial. Lancet, 361(9369):1597-602, 2003.
  46. Vogiatzi, M.G., Autio, K., Schneider, R., Giardina, P.J.: Low Bone Mass in Prepubertal Children with Thalassemia Major: Insights in the Pathogenesis of Low Bone Mass in Thalassemia and Implications for Treatment. J Ped Endo and Metab, 2002
  47. Vogiatzi, M.G., Auto, K., Schneider, R., Giardina, P.J.: Low Bone Mass in Prepubertal Children with Thalassemia Major: Insights in the Pathogenesis of Low Bone Mass in Thalassemia and Implications for Treatment. J Peds, 2002.
  48. Giardina, P.J. and Grady, R.W.: Chelation Therapy in Beta Thalassemia: An Optimistic Update. Seminars in Hematology, 38(4):360-366, 2001.
  49. Heath, J.A., Boehm, C., Beaverson, K., Cutting, G., Giardina, P.J.: A novel Bet-thalassemia Intermedia Phenotype Containing Nt494 129T>C and Nt494 132C>A Mutation in cis A Nt168C>T (beta(0) 39 Point) Mutation in Trans. Am J Hematol, 67(1):57-8, 2001.
  50. Cunningham-Rundles, S., Giardina, P.J., Grady, R.W., Califano, C., McKenzie, P., DeSousa, M.: Effect of Transfusional Iron Overload on Immune Response. Journal of Infectious Disease, 182 Suppl. 1:S115-121, 2000.
  51. Grady, R.W. and Giardina, P.J.: Iron chelation with oral deferipone in patients with thalassemia. N Eng J Med. 339(23):1712-1713, 1998.
  52. Hayward, A., Ambruso, D., Battaglia, F., Donlon, T., Eddelman, K., Giller, R., Hobbins, J., Hsia, Y.E., Quinones, R., Shpall, E., Trachtenberg, E., Giardina, P.J.: Microchimerism and Tolerance following Intrauterine Transplantation and Transfusion for ***-Thalassemia-1. Fetal Diagn Ther 13:8-14, 1998.
  53. Bush, S., Mandel, F.S. and Giardina, P.J.: Future Orientation and Life Expectations of Young Adults with Thalassemia Major. Ann NY Acad. Sci. 850:361-369, 1998.
  54. Calleja, E., Shen, J.Y., Lesser, M., Grady, R.W., New, M.I. and Giardina, P.J.: Survival and Morbidity in Transfusion Dependent Thalassemia Patients on Subcutaneous Desferrioxamine Chelation: Nearly Two Decades of Experience. NY Acad. Sci. 850:469-470, 1998.
  55. Boulad, F., Giardina, P.J., Gillio, A., Kernan, N., Small, T., Brochstein, J., Van Syckle, K., George, D., Szabolcs, P., O'Reilly, R.: Bone Marrow Transplantation for Homozygous Beta-Thalassemia-The Sloan-Kettering Cancer Center Experience. NY Acad. Sci. 850:469-470, 1998.

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