Page 668 - Hematology_ Basic Principles and Practice ( PDFDrive )

570 Part V Red Blood Cells

Haute) 499,500 arise from mutations that produce extremely labile Kattamis A, Ladis V, Berdousi H, et al: Iron chelation treatment with com-
globin chains. The chains fail to pair with the complementary chain, bined therapy with deferiprone and deferioxamine: a 12-month trial.
or they precipitate and are degraded so rapidly that they never form Blood Cells Mol Dis 36:21, 2006.
tetramers. These posttranslational lesions have the same pathophysi- Kolnagou A, Economides C, Eracleous E, et al: Low serum ferritin levels are
ologic effects on Hb biogenesis as reduction of globin mRNA produc- misleading for detecting cardiac iron overload and increase the risk of
tion or function. Another group of β-globin chain variants, usually cardiomyopathy in thalassemia patients: the importance of cardiac iron
caused by mutations in exon 3 of the β-globin gene, are associated overload monitoring using magnetic resonance imaging T2 and T2*.
with inclusion body formation and a phenotype of dominant Hemoglobin 30:219, 2006.
β-thalassemia intermedia. 15,16,501 Kolnagou A, Kontoghiorghes GJ: Effective combination therapy of deferi-
prone and deferoxamine for the rapid clearance of excess cardiac iron
and the prevention of heart disease in thalassemia. The Protocol of
SUGGESTED READINGS the International Committee on Oral Chelators. Hemoglobin 30:239,
2006.
Angelopoulos NG, Goula A, Rombopoulos G, et al: Hypoparathyroidism Neufeld EJ: Oral chelators deferasirox and deferiprone for transfusional iron
in transfusion-dependent patients with beta-thalassemia. J Bone Miner overload in thalassemia major: new data, new questions. Blood 107:3436,
Metab 24:138, 2006. 2006.
Borgna-Pignatti C, Cappellini MD, De Stefano P, et al: Cardiac morbidity Pennell DJ, Berdoukas V, Karagiorga M, et al: Randomized controlled trial
and mortality in deferoxamine- or deferiprone-treated patients with of deferiprone or deferoxamine in beta-thalassemia major patients with
thalassemia major. Blood 107:3733, 2006. asymptomatic myocardial siderosis. Blood 107:3738, 2006.
Boulad F: Hematopoietic stem cell transplantation for the treatment of Rachmilewitz EA, Giardina PJ: How I treat Thalassemia. Blood 118:3479,
beta thalassemia. In Kline R, editor: Pediatric hematopoietic stem cell 2011.
transplantation, New York, 2006, Informa Healthcare, p 383. Tanner M, Galanello R, Dessi C, et al: A randomized placebo controlled
Cappellini MD, Cohen A, Piga A, et al: A phase 3 study of deferasirox double blind trial of the effect of combination therapy with deferoxamine
(ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. and deferiprone on myocardial iron in thalassemia major using cardiovas-
Blood 107:3455, 2006. cular magnetic resonance. Blood 106:1017A, 2006.
Cohen A, Glimm E, Porter JB: Effect of transfusional iron intake on response Vogiatzi MG, Macklin EA, Fung EB, et al: Prevalence of fractures among the
to chelation therapy in β-thalassemia major. Blood 111:583, 2008. Thalassemia syndromes in North America. Bone 38:571, 2006.
Dodd RY: Current safety of the blood supply in the United States. Int J Voskaridou E, Anagnostopoulos A, Konstantopoulos K, et al: Zoledronic acid
Hematol 80:301, 2004. for the treatment of osteoporosis in patients with beta-thalassemia: results
Gilfillan CP, Strauss BJ, Rodda CP, et al: A randomized, double-blind, from a single-center, randomized, placebo-controlled trial. Haematologica
placebo-controlled trial of intravenous zoledronic acid in the treatment 91:1193, 2006.
of thalassemia-associated osteopenia. Calcif Tissue Int 79:138, 2006. Wood JC, Otto-Duessel M, Gonzalez I, et al: Deferasirox and deferiprone
Harmatz P, Grady R, Dragsten P, et al: Phase Ib clinical trial of starch- remove cardiac iron in the iron-overloaded gerbil. Transl Res 148:272,
conjugated deferoxamine (40SD02): a novel long-acting iron chelator. 2006.
Br J Haematol 138:374, 2007.
Harmatz P, Olivieri N, Kwiatkowski J, et al: Safety and efficacy of peginter-
feron alfa-2a and ribavirin for hepatitis C in thalassemia. Blood 108:558, REFERENCES
2006.
Hongeng S, Pakakasama S, Chuansumrit A, et al: Outcomes of transplanta- For the complete list of references, log on to www.expertconsult.com.
tion with related- and unrelated-donor stem cells in children with severe
thalassemia. Biol Blood Marrow Transplant 12:683, 2006.