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Clinical Trials and Research

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Thalassemia Research and Care: 2014 Update

VII. List of Published Articles About Thalassemia

  1. Angelucci E, Matthes-Martin S, Baronciani D, Bernaudin F, Bonanomi S, Cappellini MD, Dalle JH, Di Bartolomeo P, de Heredia CD, Dickerhoff R, Giardini C, Gluckman E, Hussein AA, Kamani N, Minkov M, Locatelli F, Rocha V, Sedlacek P, Smiers F, Thuret I, Yaniv I, Cavazzana M, Peters C; EBMT Inborn Error and EBMT Paediatric Working Parties. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.Haematologica 2014;May;99(5):811-20. abstract; PDF.
  2. Cappellini MD, Cohen A, Porter J, Taher A, Viprakasit V. Guidelines for the Management of Transfusion Dependent Thalassaemia, 3rd Edition. Thalaessemia International Federation 2014. PDF.
  3. Borgna-Pignatti C, Meloni A, Guerrini G, Gulino L, Filosa A, Ruffo GB, Casini T, Chiodi E, Lombardi M, Pepe A. Myocardial iron overload in thalassaemia major. How early to check? Br J Haematol 2014;Feb;164(4):579-85. abstract; PDF.
  4. Derderian SC, Jeanty C, Walters MC, Vichinsky E, MacKenzie TC. In utero hematopoietic cell transplantation for hemoglobinopathies. Front. Pharmacol., 12 January 2015. Abstract; PDF.
  5. Di Marco V, Capra M, Angelucci E, Borgna-Pignatti C, Telfer P, Harmatz P, Kattamis A, Prossamariti L, Filosa A, Rund D, Gamberini MR, Cianciulli P, De Montalembert M, Gagliardotto F, Foster G, Grangè JD, Cassarà F, Iacono A, Cappellini MD, Brittenham GM, Prati D, Pietrangelo A, Craxì A, Maggio A. Management of chronic viral hepatitis in patients with thalassemia: recommendations from an international panel. Blood 2010;116:2875-83. abstract; PDF.
  6. El-Beshlawy AM, El-Alfy MS, Sari TT, Chan LL, Tricta F. Continuation of deferiprone therapy in patients with mild neutropenia may not lead to a more severe drop in neutrophil count. Eur J Haematol 2014;92 4:337-40. abstract; PDF.
  7. Fung EB, Gariepy CA, Sawyer AJ, Higa A, Vichinsky EP. The effect of whole body vibration therapy on bone density in patients with thalassemia: a pilot study. Am J Hematol 2012;87 10:E76-9. abstract; PDF.
  8. Fung EB, Kwiatkowski JL, Huang JN, Gildengorin G, King JC, Vichinsky EP. Zinc supplementation improves bone density in patients with thalassemia: a double-blind, randomized, placebo-controlled trial. Am J Clin Nutr 2013;98 4:960-71. abstract; PDF.
  9. Fung EB. Nutritional deficiencies in patients with thalassemia. Ann N Y Acad Sci 2010;1202:188-96. abstract; PDF.
  10. Fung EB, Aguilar C, Micaily I, Haines D, Lal A. Treatment of vitamin D deficiency in transfusion-dependent thalassemia. Am J Hematol 2011;86:871-3. abstract; PDF.
  11. Fung EB, Vichinsky EP, Kwiatkowski JL, Huang J, Bachrach LK, Sawyer AJ, Zemel BS. Characterization of low bone mass in young patients with thalassemia by DXA, pQCT and markers of bone turnover. Bone 2011;48:1305-12. abstract; PDF.
  12. Fung EB, Xu Y, Kwiatkowski JL, Vogiatzi MG, Neufeld E, Olivieri N, Vichinsky EP, Giardina PJ, Network TCR. Relationship between chronic transfusion therapy and body composition in subjects with thalassemia. J Pediatr 2010;157:641-7, 7.e1-2. abstract; PDF.
  13. Fung EB, Xu Y, Trachtenberg F, Odame I, Kwiatkowski JL, Neufeld EJ, Thompson AA, Boudreaux J, Quinn CT, Vichinsky EP, Thalassemia Clinical Research N. Inadequate dietary intake in patients with thalassemia. J Acad Nutr Diet 2012;112:980-90. abstract. PDF.
  14. Gaziev J, Marziali M, Isgrò A, Sodani P, Paciaroni K, Gallucci C, Andreani M, Testi M, De Angelis G, Alfieri C, Cardarelli L, Ribersani M, Armiento D, Lucarelli G. Bone marrow transplantation for thalassemia from alternative related donors: improved outcomes with a new approach. Blood 2013;122 15:2751-6. abstract; PDF.
  15. Goss C, Giardina P, Degtyaryova D, Kleinert D, Sheth S, Cushing M. Red blood cell transfusions for thalassemia: results of a survey assessing current practice and proposal of evidence-based guidelines. Transfusion 2014. abstract; PDF.
  16. Haines D, Martin M, Carson S, Oliveros O, Green S, Coates T, Eile J, Schilling L, Dinu B, Mendoza T, Gerstenberger E, Trachtenberg F, Vichinsky E, Network TCR. Pain in thalassaemia: the effects of age on pain frequency and severity. Br J Haematol 2013;160 5:680-7. abstract; PDF.
  17. 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. Validation and reliability of a disease-specific quality of life measure (the TranQol) in adults and children with thalassaemia major. Br J Haematol 2014;164 3:431-7. abstract; PDF.
  18. Klings ES, Morris CR, Hsu L, Castro O, Gladwin MT, Mubarak KK. Letter to the Editor: Response to "Updated Clinical Classification of Pulmonary Hypertension". J Am Coll Cardiol 2014. abstract; PDF.
  19. 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, Network TCR. Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort. Blood 2012;119 12:2746-53. abstract; PDF.
  20. Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E. Combined chelation therapy with deferasirox and deferoxamine in thalassemia. Blood Cells Mol Dis 2013;50 2:99-104. abstract; PDF.
  21. Lal A, Goldrich ML, Haines DA, Azimi M, Singer ST, Vichinsky EP. Heterogeneity of hemoglobin H disease in childhood. N Engl J Med 2011;364:710-8. abstract; PDF.
  22. Mathews V, Savani BN. Conditioning regimens in allo-SCT for thalassemia major. Bone Marrow Transplant 2014;49 5:607-10. abstract; PDF.
  23. Mathews V, George B, Viswabandya A, Abraham A, Ahmed R, Ganapule A, Sindhuvi E, Lakshmi KM, Srivastava A. Improved clinical outcomes of high risk β thalassemia major patients undergoing a HLA matched related allogeneic stem cell transplant with a treosulfan based conditioning regimen and peripheral blood stem cell grafts. PLoS One 2013;8 4:e61637. abstract; PDF.
  24. 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 N. Symptoms of depression and anxiety in patients with thalassemia: prevalence and correlates in the thalassemia longitudinal cohort. Am J Hematol 2010;85:802-5. abstract; PDF.
  25. 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, Network TCR. Sildenafil therapy in thalassemia patients with Doppler-defined risk of pulmonary hypertension. Haematologica 2013;98 9:1359-67. abstract; PDF.
  26. 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 N. 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;118:3794-802. abstract; PDF.
  27. Musallam KM, Rivella S, Vichinsky E, Rachmilewitz EA. Non-transfusion-dependent thalassemias. Haematologica 2013;98 6:833-44. abstract; PDF.
  28. Oliveros O, Trachtenberg F, Haines D, Gerstenberger E, Martin M, Carson S, Green S, Calamaras D, Hess P, Yamashita R, Vichinsky E, Network TCR. Pain over time and its effects on life in thalassemia. Am J Hematol 2013;88 11:939-43. abstract; PDF.
  29. Olivieri NF, Pakbaz Z, Vichinsky E. Hb E/beta-thalassaemia: a common & clinically diverse disorder. Indian J Med Res 2011;134:522-31. abstract; PDF.
  30. Olivieri NF, Saunthararajah Y, Thayalasuthan V, Kwiatkowski J, Ware RE, Kuypers FA, Kim HY, Trachtenberg FL, Vichinsky EP, Thalassemia Clinical Research N. A pilot study of subcutaneous decitabine in beta-thalassemia intermedia. Blood 2011;118:2708-11. abstract; PDF.
  31. Patthamalai P, Fuchareon S, Chaneiam N, Ghalie RG, Chui DH, Boosalis MS, Perrine SP. A phase 2 trial of HQK-1001 in HbE-β thalassemia demonstrates HbF induction and reduced anemia. Blood 2014;123 12:1956-7. abstract; PDF.
  32. 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;55:678-83. abstract; PDF.
  33. Pennell DJ, Porter JB, Piga A, Lai Y, El-Beshlawy A, Belhoul KM, Elalfy M, Yesilipek A, Kilinç Y, Lawniczek T, Habr D, Weisskopf M, Zhang Y, Aydinok Y, investigators Cs. A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in β-thalassemia major (CORDELIA). Blood 2014;123 10:1447-54. abstract; PDF.
  34. Pennell DJ, Udelson JE, Arai AE, Bozkurt B, Cohen AR, Galanello R, Hoffman TM, Kiernan MS, Lerakis S, Piga A, Porter JB, Walker JM, Wood J, Imaging AHACoHFaTotCoCCaCoCRa. Cardiovascular function and treatment in β-thalassemia major: a consensus statement from the American Heart Association. Circulation 2013;128 3:281-308. abstract; PDF.
  35. Pepe A, Meloni A, Rossi G, Caruso V, Cuccia L, Spasiano A, Gerardi C, Zuccarelli A, D'Ascola DG, Grimaldi S, Santodirocco M, Campisi S, Lai ME, Piraino B, Chiodi E, Ascioti C, Gulino L, Positano V, Lombardi M, Gamberini MR. Cardiac complications and diabetes in thalassaemia major: a large historical multicentre study. Br J Haematol 2013;163 4:520-7. abstract; PDF.
  36. 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. Treatment of heart failure in adults with thalassemia major: response in patients randomised to deferoxamine with or without deferiprone. J Cardiovasc Magn Reson 2013;15 1:38. abstract; PDF.
  37. Porter J, Taher A, Mufarij A, Gavalas M. Emergency Management of Thalassaemia. Thalaessemia International Federation 2012. PDF.
  38. Ruggeri A, Eapen M, Scaravadou A, Cairo MS, Bhatia M, Kurtzberg J, Wingard JR, Fasth A, Lo Nigro L, Ayas M, Purtill D, Boudjedir K, Chaves W, Walters MC, Wagner J, Gluckman E, Rocha V, Eurocord R, Center for International B, Marrow Transplant R, New York Blood C. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant 2011;17:1375-82. abstract; PDF.
  39. Singer ST, Kuypers F, Fineman J, Gildengorin G, Larkin S, Sweeters N, Rosenfeld H, Kurio G, Higa A, Jeng M, Huang J, Vichinsky EP. Elevated tricuspid regurgitant jet velocity in subgroups of thalassemia patients: insight into pathophysiology and the effect of splenectomy. Ann Hematol 2014. abstract; PDF.
  40. Singer ST, Vichinsky EP, Gildengorin G, van Disseldorp J, Rosen M, Cedars MI. Reproductive capacity in iron overloaded women with thalassemia major. Blood 2011;118 10:2878-81. abstract; PDF.
  41. Singer ST, Vichinsky EP, Sweeters N, Rachmilewitz E. Darbepoetin alfa for the treatment of anaemia in alpha- or beta- thalassaemia intermedia syndromes. Br J Haematol 2011;154:281-4. abstract; PDF.
  42. 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 N. Hemoglobin H-constant spring in North America: an alpha thalassemia with frequent complications. Am J Hematol 2009;84:759-61. abstract; PDF.
  43. 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, Thalassemia Clinical Research N. 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;86:92-5. abstract; PDF.
  44. Sohn EY, Noetzli LJ, Gera A, Kato R, Coates TD, Harmatz P, Keens TG, Wood JC. Pulmonary function in thalassaemia major and its correlation with body iron stores. Br J Haematol 2011;155:102-5. abstract; PDF.
  45. Switzer WM, Shankar A, Trimble SR, Thompson AA, Giardina PJ, Cohen AR, Coates TD, Vichinsky E, Neufeld EJ, Boudreaux JM, Heneine W. Human T cell lymphotropic virus type 1 infection among U.S. thalassemia patients. AIDS Res Hum Retroviruses 2013;29 7:1006-9. abstract; PDF.
  46. Taher A, Vichinsky E, Musallam K, Cappellini MD, Viprakasit V. Guidelines for the Management of Non Tranfusion Dependent Thalassemia (NTDT). ed. Cyprus: Thalassemia International Federation; 2013. abstract; PDF.
  47. Thompson AA, Kim HY, Singer ST, Vichinsky E, Eile J, Yamashita R, Giardina PJ, Olivieri N, Parmar N, Trachtenberg F, Neufeld EJ, Kwiatkowski JL, Network TCR. Pregnancy outcomes in women with thalassemia in North America and the United Kingdom. Am J Hematol 2013;88 9:771-3. abstract; PDF.
  48. Trachtenberg F, Foote D, Martin M, Carson S, Coates T, Beams O, Vega O, Merelles-Pulcini M, Giardina PJ, Kleinert DA, Kwiatkowski J, Thompson AA, Neufeld EJ, Schilling L, Thayalasuthan V, Pakbaz Z, Yamashita R, Thalassemia Clinical Research N. Pain as an emergent issue in thalassemia. Am J Hematol 2010;85:367-70. abstract; PDF.
  49. 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, Thalassemia Clinical Research N. Iron chelation adherence to deferoxamine and deferasirox in thalassemia. Am J Hematol 2011;86:433-6. abstract; PDF.
  50. Vichinsky E, Neumayr L, Trimble S, Giardina PJ, Cohen AR, Coates T, Boudreaux J, Neufeld EJ, Kenney K, Grant A, Thompson AA, Investigators CT. Transfusion complications in thalassemia patients: a report from the Centers for Disease Control and Prevention (CME). Transfusion 2014;54 4:972-81. abstract; PDF.
  51. Vichinsky EP. Clinical manifestations of α-thalassemia. Cold Spring Harb Perspect Med 2013;3 5:a011742. abstract; PDF.
  52. Vichinsky E. Emerging 'A' therapies in hemoglobinopathies: agonists, antagonists, antioxidants, and arginine. Hematology Am Soc Hematol Educ Program 2012;2012:271-5. abstract; PDF.
  53. Vichinsky EP. Alpha thalassemia major--new mutations, intrauterine management, and outcomes. Hematology Am Soc Hematol Educ Program 2009:35-41. doi: 10.1182/asheducation-2009.1.35. abstract; PDF.
  54. Vogiatzi MG, Macklin EA, Fung EB, Cheung AM, Vichinsky E, Olivieri N, Kirby M, Kwiatkowski JL, Cunningham M, Holm IA, Lane J, Schneider R, Fleisher M, Grady RW, Peterson CC, Giardina PJ, Thalassemia Clinical Research N. Bone disease in thalassemia: a frequent and still unresolved problem. J Bone Miner Res 2009;24:543-57. abstract; PDF.
  55. 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, Giardina PJ, Thalassemia Clinical Research N. Differences in the prevalence of growth, endocrine and vitamin D abnormalities among the various thalassaemia syndromes in North America. Br J Haematol 2009;146:546-56. abstract; PDF.
  56. Voskaridou E, Komninaka V, Karavas A, Terpos E, Akianidis V, Christoulas D. Combination therapy of deferasirox and deferoxamine shows significant improvements in markers of iron overload in a patient with β-thalassemia major and severe iron burden. Transfusion 2014;54 3:646-9. abstract; PDF.
  57. Walter PB, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Grady RW, Vichinsky E, Olivieri N, Trachtenberg F, Alberti D, Fung E, Ames B, Higa A, Harmatz P, Network TCR. Increased leucocyte apoptosis in transfused β-thalassaemia patients. Br J Haematol 2013;160 3:399-403. abstract; PDF.
  58. Wood JC, Glynos T, Thompson A, Giardina P, Harmatz P, Kang BP, Paley C, Coates TD. Relationship between labile plasma iron, liver iron concentration and cardiac response in a deferasirox monotherapy trial. Haematologica 2011;96:1055-8. abstract; PDF.
  59. 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;116:537-43. abstract; PDF.

Updated 2/19/2015


Northern California Comprehensive Thalassemia Center
UCSF Benioff Children's Hospital Oakland
747 52nd Street, Oakland CA 94609   •   Phone: (510) 428-3651   •   Fax: (510) 450-5647
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