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Expert Rev Hematol 2018 02 2;11(2):109-116. Epub 2018 Jan 2.

b Ludwig Boltzmann Cluster Oncology , Medical University of Vienna , Vienna , Austria.

Introduction: Despite the availability of effective iron chelators, transfusion-related morbidity is still a challenge in chronically transfused patients with myelodysplastic syndromes (MDS). In these patients, transfusion-induced iron overload may lead to organ dysfunction or even organ failure. In addition, iron overload is associated with reduced overall survival in MDS. Read More

Pediatr Blood Cancer 2017 Nov 19;64(11). Epub 2017 May 19.

Department of Pediatrics, Washington University School of Medicine, St. Louis, Missouri.

Background: Children and adolescents with sickle cell disease (SCD) are at high risk of strokes and are frequently treated with red blood cell (RBC) transfusions. The goal is to suppress hemoglobin (Hb) S while minimizing transfusion-induced iron overload. RBCs may be given via simple transfusion, manual exchange transfusion (MET), or erythrocytapheresis (aRBCX). Read More

Burns Trauma 2016 2;4:10. Epub 2016 May 2.

Glenrose Rehabilitation Hospital, #1126, 10230 111 Ave, Edmonton, AB T5G 0B7 Canada.

Background: Red blood cell transfusions are critical in burn management. The subsequent iron overload that can occur from this treatment can lead to secondary hemochromatosis with multi-organ damage.

Case Presentation: While well recognized in patients receiving chronic transfusions, we present a case outlining the acute development of hemochromatosis secondary to multiple transfusions in a burn patient. Read More

Paediatr Int Child Health 2016 Jan 29:1-5. Epub 2016 Jan 29.

a Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital , Mahidol University , Bangkok , Thailand.

Background: Data on the use of deferiprone in young children with iron overload are limited.

Objective: To study the safety profile of a liquid formulation of deferiprone in chelating young children with transfusion-induced iron overload.

Patients And Methods: A daily dose of 50-100 mg/kg BW in three divided doses of oral deferiprone was given to young patients who had received at least ten packed red cell transfusions and achieved a serum ferritin level >1000 μg/L during a 12-month period from 2011 to 2012. Read More

Surv Ophthalmol 2016 Jan-Feb;61(1):33-50. Epub 2015 Aug 29.

Department of Ophthalmology, F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Philadelphia, Pennsylvania, USA. Electronic address:

Patients with beta (β)-thalassemia (β-TM: β-thalassemia major, β-TI: β-thalassemia intermedia) have a variety of complications that may affect all organs, including the eye. Ocular abnormalities include retinal pigment epithelial degeneration, angioid streaks, venous tortuosity, night blindness, visual field defects, decreased visual acuity, color vision abnormalities, and acute visual loss. Patients with β-thalassemia major are transfusion dependent and require iron chelation therapy to survive. Read More

Paediatr Int Child Health 2016 Aug;36(3):209-13

a Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital , Mahidol University , Bangkok , Thailand.

Background: Data on the use of deferiprone in young children with iron overload are limited.

Objective: To study the safety profile of a liquid formulation of deferiprone in chelating young children with transfusion-induced iron overload.

Patients And Methods: A daily dose of 50-100 mg/kg BW in three divided doses of oral deferiprone was given to young patients who had received at least ten packed red cell transfusions and achieved a serum ferritin level >1000 μg/L during a 12-month period from 2011 to 2012. Read More

Pediatr Endocrinol Rev 2015 Mar;12(3):313-22

The VIII ICET-A International Symposium was held in Muscat (Sultanate of Oman) on the 20th of December, 2014. The symposium included four sessions on a wide range of topics covering growth disorders and endocrine complications in thalassaemia. Despite the fact that endocrine complications are very common in multi-transfused thalassaemia patients a recent survey conducted by the International Network of Clinicians for Endocrinopathies in Thalassemia and Adolescent Medicine (ICET-A) in 2014 in Acitrezza (Catania, Italy) showed that the major difficulties reported by hematologists or pediatricians experienced in thalassaemias or thalassaemia syndromes in following endocrine complications included: Lack of familiarity with medical treatment of endocrine complications, interpretation of endocrine tests, lack of collaboration and on-time consultation between thalassaemic centres supervised by haematologists and endocrinologists. Read More

Hematology 2015 Jun 9;20(5):304-10. Epub 2014 Sep 9.

Background/Purpose Retention of excess iron from transfused blood in organs in patients with renal anemia may lead to various systemic complications. Iron chelating agents such as deferasirox (DFX) decrease such iron overload. This study assessed the efficacy, safety, and tolerability of DFX in hemodialysis (HD) patients with iron overload. Read More

J Korean Med Sci 2013 Nov 31;28(11):1563-72. Epub 2013 Oct 31.

Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.

Many Korean patients with transfusion-induced iron overload experience serious clinical sequelae, including organ damage, and require lifelong chelation therapy. However, due to a lack of compliance and/or unavailability of an appropriate chelator, most patients have not been treated effectively. Deferasirox (DFX), a once-daily oral iron chelator for both adult and pediatric patients with transfusion-induced iron overload, is now available in Korea. Read More

PLoS One 2013 30;8(5):e64114. Epub 2013 May 30.

Department of Pediatrics, Seoul National University College of Medicine, Seoul, Republic of Korea.

Transfusion-associated iron overload induces systemic toxicity. Deferasirox, a convenient long acting oral agent, has recently been introduced in clinical practice with a promising efficacy. But there are some patients who experience drug-related toxicities and cannot tolerate it. Read More

Acta Haematol 2013 15;129(2):72-7. Epub 2012 Nov 15.

Department of Hematology, Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, Catholic University of Korea, Seoul, Korea.

Iron overload due to regular transfusions of packed red cells can cause multiple organ damage. Iron chelation therapy (ICT) is important in patients with aplastic anemia (AA) who require blood transfusions as supportive management. With the introduction of the oral iron chelator deferasirox, ICT has become more widely available and feasible. Read More

Intern Med 2010 1;49(5):453-6. Epub 2010 Mar 1.

Department of Hematology and Clinical Immunology, Kobe City Medical Center General Hospital.

A 21-year-old man who was diagnosed with Diamond-Blackfan anemia at 2 years of age came to our hospital with the hope of undergoing bone marrow transplantation (BMT). He had been red cell transfusion-dependent for about 8 years; at presentation he had transfusion-induced hemochromatosis, a subsequent low left ventricular ejection fraction (LVEF) of 43%, and diabetes mellitus requiring insulin therapy. He received unrelated BMT with reduced intensity conditioning and sufficient GVHD prophylaxis. Read More

Acta Haematol 2008 1;120(2):70-4. Epub 2008 Oct 1.

Division of Hematology and Internal Medicine, Department of Clinical and Biological Sciences, University of Turin, Orbassano, Italy.

Transfusion-induced iron overload is a frequent problem that clinicians have to face in the treatment of patients affected by both myelodysplastic syndrome (MDS) and primary myelofibrosis (PMF). Different options are currently available for chelation therapy, e.g. Read More

Int J Hematol 2008 Jul 27;88(1):30-35. Epub 2008 Jun 27.

Division of Hematology, Department of Medicine, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke-shi, Tochigi, 329-0498, Japan.

Many patients with bone marrow failure syndromes need frequent transfusions of red blood cells, and most of them eventually suffer from organ dysfunction induced by excessively accumulated iron. The only way to treat transfusion-induced iron overload is iron chelating therapy. However, most patients have not been treated effectively because daily/continuous administration of deferoxamine is difficult for outpatients. Read More

Eur J Clin Invest 2008 Mar 24;38(3):143-9. Epub 2008 Jan 24.

Department of Internal Medicine I, Division of Haematology & Haemostaseology, Medical University of Vienna, Austria.

Transfusion-related morbidity is an emerging challenge in chronically transfused patients with low-risk myelodysplastic syndromes (MDS). In these patients, transfusion-induced iron overload may represent a leading medical problem. However, although iron-chelating drugs are available, little is known about optimal diagnostic tools, predisposing factors, and the optimal management of these patients. Read More

Med Sci Monit 2007 May;13(5):CR235-9

Iranian Thalassemia Society, Iran.

Background: The aim was to determine whether reduced serum zinc level has a contributory effect on impairment of insulin secretion in beta-thalassemic patients with transfusion-induced iron overload.

Material/methods: Seventy thalassemia patients who received deferoxamine chelation therapy and 69 healthy individuals as the control group were evaluated. A standard oral glucose tolerance test (OGTT) was performed and blood samples for measurement of serum ferritin, zinc, and insulin concentrations were obtained. Read More

ScientificWorldJournal 2006 Oct 9;6:1278-97. Epub 2006 Oct 9.

TriStem U.K. Limited.

Beta-thalassemia is a genetic, red blood cell disorder affecting the beta-globin chain of the adult hemoglobin gene. This results in excess accumulation of unpaired alpha-chain gene products leading to reduced red blood cell life span and the development of severe anemia. Current treatment of this disease involves regular blood transfusion and adjunct chelation therapy to lower blood transfusion-induced iron overload. Read More

J Egypt Soc Parasitol 1999 ;29(3):791-803

Department of Parasitology, Faculty of Medicine, Ain Shams University, Cairo 11566, Egypt.

The standard regimen of treatment for toxoplasmosis is pyrimethamine with sulfadiazine. However, it is not suitable in some conditions, and nontolerable in AIDS patients. Deferoxamine (DFO), an iron chelator, is well tolerated clinically in transfusion induced - iron overload. Read More

Hematology Am Soc Hematol Educ Program 2001 :47-61

Department of Hematology, University College London, 98 Chenies Mews, London WC1 6HX.

Transfusion therapy for inherited anemias and acquired refractory anemias both improves the quality of life and prolongs survival. A consequence of chronic transfusion therapy is secondary iron overload, which adversely affects the function of the heart, the liver and other organs. This session will review the use of iron chelating agents in the management of transfusion-induced secondary iron overload. Read More

Klin Monbl Augenheilkd 1996 Jul;209(1):31-6

Universitäts-Augenklinik Ulm.

Background: Deferoxamine, an iron chelating agent, has been used for the treatment of hemochromatosis for more than 30 years. Ocular toxicity has begun to be reported only in the last few years. In most cases differentiation of the true etiology, i. Read More

J Lab Clin Med 1994 Aug;124(2):193-8

Department of Medicine, Veterans Administration Medical Center, Birmingham, AL.

We postulated that patients with hereditary hemochromatosis (HH) absorb increased quantities of lead, as do iron-deficient subjects. To test this hypothesis, whole blood lead concentration ([blood Pb]) was quantified by atomic absorption spectrometry in HH homozygotes (n = 44), obligate heterozygotes (n = 19), normal control subjects (n = 33), and abnormal controls, with transfusion-induced iron overload (n = 8). HH homozygotes had higher [blood Pb] than did normal control subjects (5. Read More

J Electrocardiol 1994 Jan;27(1):91-6

Department of Internal Medicine, National Taiwan University Hospital, Taipei, Republic of China.

The most important manifestations of heart disease in hemochromatosis are congestive heart failure and cardiac arrhythmia. The spectrum of disturbances in cardiac rhythm ranges from minor abnormalities on the electrocardiogram to supraventricular arrhythmia, atrioventricular conduction block, and ventricular tachyarrhythmia. Sinus node dysfunction is, however, rarely mentioned. Read More

J Lab Clin Med 1993 Jul;122(1):48-54

Colorado Sickle Cell Treatment and Research Center, Denver.

Transfusion-induced hemosiderosis is a serious and potentially life-threatening complication for some patients with sickle cell anemia. The use of high-dose intravenous deferoxamine (DFO) has become widespread in spite of a paucity of published data on safety and efficacy. We report a randomized double-blind study of the dose-response relationship of intravenous DFO in six subjects with sickle cell anemia and severe transfusion-induced hemosiderosis (serum ferritin 4100 to 14,176 ng/ml). Read More

Pediatrics 1993 Mar;91(3):537-9

Rockefeller University Hospital, New York, NY 10021.

The heme oxygenase inhibitor tin (Sn4+)-mesoporphyrin, administered to two 17-year-old Crigler-Najjar type I patients during a 400-day study to lower plasma bilirubin levels, also produced changes, beginning approximately 50 days after initiation of treatment, in hematological and iron metabolism indices consistent with the development of iron deficiency anemia. These indices were responsive to iron supplementation and reverted to normal after termination of inhibitor treatment. Tin-mesoporphyrin enhances biliary heme excretion and inhibits intestinal heme oxygenase when administered orally or parenterally; the changes in blood indices could thus reflect, in part, blockade of heme catabolism and therefore of uptake of heme-derived iron, by intestinal epithelium. Read More

Acta Paediatr Scand Suppl 1989 ;349:93-9; discussion 100

Department of Paediatric Endocrinology, Children's Hospital, University of Munich, West Germany.

The growth hormone-releasing hormone (GHRH) test was applied to more than 230 children. Twenty-five out of 61 patients with proven growth hormone (GH) deficiency responded to GHRH with a GH increase of greater than 10 ng/ml. In most of the patients with idiopathic GH deficiency, a priming procedure using daily injections of GHRH improved the secretory response to GHRH. Read More

Clin Exp Immunol 1987 Nov;70(2):345-53

Department of Pediatrics, Cornell University Medical Center, New York, NY 10021.

We previously observed that natural killer (NK) activity toward K562 cells is markedly depressed in patients with beta-thalassaemia major. Here we report that these patients also exhibit significantly decreased (P less than 0.005) NK cytotoxicity against human fibroblasts infected with herpes simplex virus-type 1 (HSV-1) and that the amount of alpha-interferon (alpha-IFN) generated during the latter assays is significantly less than normal (P less than 0. Read More

Am J Med 1987 Feb;82(2):367-70

Aluminum-associated osteomalacia and transfusion-induced hemosiderosis developed in an anephric patient receiving long-term maintenance treatment with continuous ambulatory peritoneal dialysis. Intravenous administration of 1.0 g of deferoxamine led to marked increases in the fecal elimination of aluminum and iron. Read More

Exp Hematol 1986 Nov;14(10):971-4

Previously reported animal models of hemosiderosis fall short of simulating the human disease state of transfusion-induced hemosiderosis. An explanation for this was found by reexamining heat-treated red blood cell (rbc) loading in the mouse. After 18 intraperitoneal transfusions, each equal to two-thirds of mouse rbc volume, liver Fe reaches a level of 0. Read More

Lancet 1983 Jul;2(8343):181-4

Desferrioxamine was given intravenously, at higher doses than previously reported, to counter the effects of transfusion-induced iron overload in four patients with beta thalassaemia major. In two of them retinal abnormalities developed, presenting with night blindness and field defects, which improved on withdrawal of the drug. Read More

Z Gesamte Inn Med 1983 Feb;38(3):81-3

Congestive cardiomyopathy and final stage of a liver cirrhosis due to massive overload of iron are often the cause of death in polytransfused patients with aplastic anaemia. With increasing frequency of transfusion the problem of overload of iron becomes more actual. The toxicity of abundant iron is based on various mechanisms of action, which are compiled with the help of literature. Read More

Am J Pediatr Hematol Oncol 1982 ;4(2):162-5

Transfusion-induced hemochromatosis is a potential therapeutic complication of the management of sickle cell anemia. Selective transfusion with young erythrocytes (neocytes), which theoretically have a longer life-span than do regular donor red cells, could decrease transfusion frequency and reduce iron deposition in the sickle cell patient. Using blood cell separators, a method has been devised to prepare neocyte units for transfusion. Read More

Blood 1981 Mar;57(3):599-606

Transfusion-induced hemochromatosis remains a major therapeutic complication in the management of thalassemia major patients. Using available blood cell component separators, a system has been devised to selectively harvest young red cells from transfusion support of these subjects. Red cell units isolated by this method have an average estimated mean cell age of 30 days, compared to 60 days for unfractionated blood, and contain 80% of the hemoglobin content of standard red cell units. Read More

J Pediatr Ophthalmol Strabismus 1978 May-Jun;15(3):154-6

This is the first case report of cataracts in patients with thalassemia major. Desferrioxamine, an iron-chelating agent is being used with increasing frequency in the treatment of transfusion-induced iron overload. There has been some concern in the literature about possible cataract formation with use of this drug. Read More

N Engl J Med 1976 Jun;294(26):1421-3

Treatment of transfusion-induced iron overload by daily intramuscular injection of the chelator desferrioxamine has not produced impressive urinary iron excretion. We attempted to augment net iron excretion by altering both the route and quantity of chelator administered. Two ascorbic acid-replete patients excreted a mean of 14. Read More

Proc Soc Exp Biol Med 1976 Jun;152(2):249-52

The influence of vitamin E on the mobilization and excretion of storage iron induced by DF was studied in normal and iron-overloaded rats. Vitamin E administration in pharmacologic doses resulted in complete inhibition of the effect of DF on storage iron in iron-overloaded rats while no such effect could be demonstrated in rats with normal iron stores. The mechanism of the observed inhibition of DF action by vitamin E is at present unknown. Read More

Am J Med 1976 Jun;60(7):1032-5

A 25 year old patient with congenital hypoplastic anemia (Black-fan-Diamond syndrome) is described. This patient was hepatitis-antigen negative, had not received androgens and had a hepatoma develop in a transfusional hemochromatotic liver. Since androgens have been associated with hepatocellular carcinoma, the use of androgenic steroids for other than life-threatening symptoms in this disease should be avoided. Read More

Birth Defects Orig Artic Ser 1976 ;12(8):53-61

Because of its high prevalence in Italy, thalassemia is officially considered a disease of major social importance. Several centers have been established for the specific purpose of population screening and treatment of patients with Cooley disease. Survival, in some cases up to 20 years, has been reached by the therapeutic methods adopted, consisting mainly of an intensive transfusion program combined with splenectomy. Read More

Osp Maggiore 1961 Aug;49:352-9