The Treatment of Sickle Cell Anemia

Charache, Samuel

doi:10.1001/archinte.1974.00320160192018

Cited by 48 publications

(11 citation statements)

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“…Chelation guidelines for sickle-cell syndromes are similar to those for other iron overload situations as thalassemia major and multi-transfused patients [14,15]. Until recently, desferrioxamine and deferiprone were the only active iron-chelating agent approved for clinical use, mainly in the thalassemia setting [16,17].…”

Section: Discussionmentioning

confidence: 99%

“…We have clarified now in the "study design" section that "The starting deferasirox dose was 10 or 20 mg/kg/day depending on previous transfusion requirements: if the patient had received 20-30 red blood cell (RBC) units the dose of 10 mg/kg/day was used; if the patients had received more than 30 RBC units the dose of 20 mg/kg/day was used." [page 4, lines [15][16][17].…”

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confidence: 99%

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Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia

et al. 2010

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Iron overload is present in several cases of double heterozygous HbS/beta-thalassemia (HbS/beta-thal). Deferasirox is an orally administered iron chelator which is effective on iron overloaded patients with transfusion-dependent anemia. The aim of this study was to investigate the efficacy and safety of deferasirox in HbS/beta-thal patients with iron overload. We evaluated 31 adult patients with HbS/beta-thal (14M/17F; median age 41 years) who had serum ferritin levels >1000 ng/mL and who were sporadically transfused. Total iron burden was monitored by measuring serum ferritin levels before and monthly after starting deferasirox, while liver iron concentration and cardiac iron burden were measured by magnetic resonance imaging (MRI) T2 and T2* parameters at baseline and 12 months after deferasirox treatment. Deferasirox managed to reduce the mean serum ferritin levels after 12 months of treatment from 1989±923 to 1008±776 ng/mL (P<0.001). This reduction was accompanied by a significant improvement on MRI T2* of the liver (from 3.9±3.2 to 5.8±3.1 ms; P<0.01) and by a comparable improvement of biochemical parameters of liver function. Mild nausea and diarrhoea of grade 1/2 were reported in 25% of patients within the first month of treatment, but did not re-occur. These data indicate that deferasirox provided effective control of iron levels (mainly of the liver) in minimally transfused patients with HbS/beta-thal, without significant adverse events, at similar doses to those studied widely for the treatment of patients with thalassemia syndromes.Response to Reviewers: I would like to thank the reviewer for the valuable comments about our paper entitled "Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/beta-thalassemia". Please find below our answers to these comments. All changes have been bolded into the text.Reviewer #1: Comment 1. The manuscript was thoroughly revised and has remarkably improved. However, concerning two of the previous comments were incompletely answered. A more detailed answer will increase the comprehensibility of the corresponding parts of the paper. Certainly, it will be very easy for authors to add the missing information: As stated in the previous comment 5, for liver MRI, it would be helpful for the reader to know how the T2star values correspond to liver iron concentration given as mg/g or micromole/g dry or wet weight since at present the majority of institutions and colleagues are using these values and related cut-offs for the assessment of liver siderosis. Comment 2. (see previous comment 6): In "Methods" it is stated that: "The starting deferasirox dose was 10 or 20 mg/kg/day depending on baseline iron burden..." In "Results", it is written that "patients were treated with deferasirox at 10 and 20mg/kg/day? based on the number of transfusions?")? How was the "baseline iron burden defined" (according to "Results" by the number of transfusions) and what was the cut-off number of blood transfusions to decide for 20mg/kg/d? Answer: We tha...

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Section: Discussionmentioning

confidence: 99%

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confidence: 99%

Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia

et al. 2010

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“…One of the few forms of treatment available is eryth rocyte (RBC) replacement, either as simple or ex change transfusion. RBC transfusion has bee advo cated for the treatment or prevention of a variety of complications of sickle cell syndromes, including cer ebrovascular occlusion [1,2], recurrent painful crises [3,4], leg ulcers [5], priapism [6] and bone involvement [7], to reduce foetal and maternal risks in pregnancy [8][9][10] and SC disease [11], and preoperatively before general anaesthesia [12] or retinal surgery [13]. How ever the long-term risks and benefits of such interven tion are ill-defined and controversial at present.…”

Section: Introductionmentioning

confidence: 99%

“…In particular, the risks to sickle patients from iron over load resulting from multiple transfusions are unclear. There have been isolated reports of clear-cut transfu sion siderosis [14] but it has also been suggested that patients with sickle cell anaemia may be less suscepti ble to the effects of transfusion siderosis [15,16] than other multiply transfused patients, possibly because of increased urinary iron excretion [17,18]. It has fur ther been suggested that the use of exchange transfu sion rather than simple RBC transfusion should result in less transfusional siderosis [12].…”

Section: Introductionmentioning

confidence: 99%

Transfusion and Exchange Transfusion in Sickle Cell Anaemias, with Particular Reference to Iron Metabolism

Porter

Huehns

1987

Acta Haematol

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The relationship between the number of units of blood transfused and indicators of iron status in 37 patients with sickle cell anaemia (Hb SS), SC disease (Hb SC) or Sβ-thalassaemia has been studied. The correlation coefficient between serum ferritin and the number of units transfused was good (r = 0.86), provided that ferritin samples taken within one week following a crisis were excluded. The relationship of transfusion history to serum ferritin in the steady state showed a similar relationship to that previously observed for other multiply transfused patients. The serum ferritin taken within 7 days of a painful crisis was significantly greater than the serum ferritin from the same patients in the steady state (p < 0.025). The serum alanine transaminase did not rise as consistently as the serum ferritin during crises; it correlated with the serum ferritin but not the transfusion burden in the steady state. Transferrin iron saturation correlated less clearly with transfusion history than serum ferritin (r = 0.62). Patients who had received exchange transfusions were less likely to be iron-overloaded (ferritin increment per unit of blood = 9.9 ± 3.8 μg/l) than patients who had received an equivalent number of units by conventional transfusion (ferritin increment per unit of blood transfused = 25.1 ± 2.42 μg/l)

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“…Treatment options include rehydration and blood transfusion to correct anemia, vaccinations and prophylactic antibiotics to reduce and prevent infections, as well as pain management to reduce the burden of painful crisis and improve the functioning abilities of patients. (16)(17)(18) Hydroxy Urea (HU) is a urea analogue which inhibits DNA synthesis in the S-phase of cell cycle. (19) It is used to modulate the severity of SCA phenotype, and for the treatment of painful crisis.…”

Section: Int J Adv Res 5(2) 2345-2350mentioning

confidence: 99%

The Role of Hu in the Management of Vaso-Occlusive Crises in Sickle Cell Anemia.

Sulami¹,

Najjar.²

2017

IJAR

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The Treatment of Sickle Cell Anemia

Cited by 48 publications

References 100 publications

Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia

Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia

Transfusion and Exchange Transfusion in Sickle Cell Anaemias, with Particular Reference to Iron Metabolism

The Role of Hu in the Management of Vaso-Occlusive Crises in Sickle Cell Anemia.

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