Oral chelators deferasirox and deferiprone for transfusional iron overload in thalassemia major: new data, new questions

Neufeld, Ellis J.

doi:10.1182/blood-2006-02-002394

Cited by 285 publications

(208 citation statements)

References 39 publications

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“…In addition, the pumps are not always available or need continuous maintenance and repair. These children and their parents are hoping to switch to oral chelation therapy, assuming it is more comfortable and painless [24,25].…”

Section: Discussionmentioning

confidence: 99%

Quality of Life Among Thalassemia Children Patients in the Gaza Strip

Aljeesh

2016

AJNS

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Abstract:This study aimed to assess the quality of life of children (3-12 years) diagnosed with Thalassemia in Gaza Strip.A descriptive, analytical, cross-sectional design was used. All children 3-12 years old who were diagnosed with thalassemia and live in GS were included in the study (75 cases) with response rate (81.3%). Participants were interviewed by the researcher and completed the questionnaire which focused on quality of life of chronic diseases and included five domains. These domains are physical health, psychological health, social relationships, environment health and personal safety. Validity and reliability of the instrument were tested and the total instrument reliability test (Cronbach's Alpha) was 0.767. The scores for the study domains ranged between 2.81 for personal security domain and 3.66 for environmental health domain. Moreover, the study domains did not show statistically significant difference when compared by sex, living places, monthly income the relationship between parents and recurrent hospitalization. The findings of this study suggest the need of children diagnosed with thalassemia for support in all study domains. Health professionals need to work to minimize the disease's burdens. The current study calls the attention of health policy makers for new policies and new roles for the community health nurses and social workers. The study also revealed that there is a bad need to pay more attention when caring and dealing with thalassemia patients. Special food supplementation should be available in order to improve their physical health, more psychological support from their families and the society is needed. In addition, there is a need to provide safe environment and to enhance personal safety of these children.

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Section: Discussionmentioning

confidence: 99%

Quality of Life Among Thalassemia Children Patients in the Gaza Strip

Aljeesh

2016

AJNS

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“…3 UCB has the advantages of rapid availability and low risk of severe GVHD despite donorrecipient HLA disparity. 5 The Chang Gung Paediatric HSCT Program has an active protocol for using unrelated cord blood for transplantation as an alternative source of stem cells when an HLA-compatible sibling donor is not available. The current study, using a rigid intent-to-treat statistical approach, suggested that the risks associated with HSCT would be increased by the iron overload and sensitization to HLA induced by hypertransfusion.…”

Section: Discussionmentioning

confidence: 99%

“…4 Strategies to reduce cardiac disease by improving chelation regimens have been of the highest priority. 5 Haematopoietic SCT (HSCT) remains the only definitive curative therapy for thalassaemia and other haemoglobinopathies. The development of oral chelators has not changed this situation.…”

Section: Introductionmentioning

confidence: 99%

Unrelated cord blood transplantation for thalassaemia: a single-institution experience of 35 patients

Jaing

Hung

Yang

et al. 2011

Bone Marrow Transplant

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Our study was designed to prospectively determine whether or not unrelated cord blood transplantation (CBT) can produce outcomes comparable to related donor transplantation for children with b-thalassaemia. In 35 patients, 40 transplants were performed between October 2003 and September 2009. HLA matching at enrolment was 6/6 (n ¼ 8), 5/6 (n ¼ 16), 4/6 (n ¼ 27), or 3/6 (n ¼ 1) by low-resolution HLA-A, -B, and high-resolution DRB1. These patients received non-manipulated grafts without ex vivo expansion or T-cell depletion. The median number of nucleated and CD34 þ cells infused was 7.8 Â 10 7 /kg (range, 2.8-14.7 Â 10 7 /kg) and 4.0 Â 10 5 /kg (range, 1.7-19.9 Â 10 5 /kg), respectively. The 5-year OS and thalassaemia-free survival after the first transplant were 88.3 and 73.9%, respectively. The cumulative incidence of TRM at 2 years was 11.7%. Fourteen patients developed chronic skin GVHD. Thirty patients were alive and transfusion-independent with a Lansky performance score X80% achieved between 6 and 76 months post transplant (median, 36 months). These data compare acceptably with the survival rates of related-donor BMT for thalassaemia and suggest that patients without an available HLAcompatible sibling but who have well-matched unrelated donors should also be considered for CBT.

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“…Deferasirox has been introduced relatively recently and appears to have a favorable toxicity profile, although skin rashes, gastrointestinal disturbances, and occasionally abnormal liver function tests have been encountered. It seems to be roughly equivalent in effectiveness to desferrioxamine, and may well become the treatment of choice for patients with secondary hemochromatosis [92].…”

Section: Treatment Of Iron Overloadmentioning

confidence: 99%

Iron storage disease: Facts, fiction and progress

Beutler

2007

Blood Cells, Molecules, and Diseases

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There are many forms of iron storage disease, some hereditary and some acquired. The most common of the hereditary forms is HFE-associated hemochromatosis, and it is this disorder that is the main focus of this presentation. The body iron content is regulated by controlling absorption, and studies in the past decade have clarified, in part, how this regulation functions. A 25 amino acid peptide hepcidin is upregulated by iron and by inflammation, and it inhibits iron absorption and traps iron in macrophages by binding to and causing degradation of the iron transport protein ferroportin. Most forms of hemochromatosis results from dysregulation of hepcidin or defects of hepcidin or ferroportin themselves.Hereditary hemochromatosis was once considered to be very rare, but in the 1970s and 1980s, with the introduction of better diagnostic tests, it became to be considered the most common disease of Europeans. Controlled epidemiologic studies carried out in the last decade have shown, however, the disease itself actually is rare, and it is only the genotype and associated biochemical changes that are common. We do not understand why only a few homozygotes develop severe disease. It now seems unlikely that there are important modifying genes, and although alcohol is known to have some effect, excess drinking probably plays only a modest role in determining the hemochromatosis phenotype.Hereditary hemochromatosis is readily treated by phlebotomy. Secondary forms of the disease require chelation therapy, and the recent introduction of effective oral chelating agents is an important step forward in treating patients with disorders in which iron overload often proves to be fatal, such as thalassemia, myelodysplastic anemias, and dyserythropoietic anemias.While much has been learned about the regulation of iron homeostasis in the past decade, many mysteries remain and represent challenges that will keep us occupied for years to come.It is a great honor for me to present this lecture in memory of my good friend Bracha Ramot. It is because this lecture is in honor of Bracha that I do not feel the least bit uncomfortable speaking about iron storage diseases in a symposium entitled "Genomics and Molecular Biology of Hematopoietic Malignancies". I know that had it been possible to ask her whether this might be appropriate Bracha would have said: "Of course, Ernie. Talk about whatever you want to". That was her way. And, of course, Bracha was no stranger to iron metabolism. She was scientifically broad, a Renaissance hematologist. A MEDLINE search reveals 261 publications by Bracha and eight of those deal with iron --iron in thalassemia, iron in sideroblastic anemia, and iron in iron deficiency anemia.Publisher's Disclaimer: This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final citable...

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Oral chelators deferasirox and deferiprone for transfusional iron overload in thalassemia major: new data, new questions

Cited by 285 publications

References 39 publications

Quality of Life Among Thalassemia Children Patients in the Gaza Strip

Quality of Life Among Thalassemia Children Patients in the Gaza Strip

Unrelated cord blood transplantation for thalassaemia: a single-institution experience of 35 patients

Iron storage disease: Facts, fiction and progress

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