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Prophylactic transfusion for pregnant women with sickle cell disease: a systematic review and meta-analysis.
Prophylactic transfusions may positively impact several adverse maternal and neonatal outcomes in women with sickle cell disease; however, the evidence stems from a relatively small number of studies with methodologic limitations. Expand
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia.
- M. Cappellini, V. Viprakasit, +37 authors A. Piga
- The New England journal of medicine
- 25 March 2020
The percentage of patients with transfusion-dependent β-thalassemia who had a reduction in transfusion burden was significantly greater in the luspatercept group than in the placebo group, and few adverse events led to the discontinuation of treatment. Expand
A Systematic Review and Meta-Analysis of Deferiprone Monotherapy and in Combination with Deferoxamine for Reduction of Iron Overload in Chronically Transfused Patients with β-Thalassemia
There is currently insufficient evidence to show that DFP is superior to DFO in the treatment of iron overload, and the use of DFP must be weighed against the potential side-effects, patient compliance and preference. Expand
Fondaparinux: A potential new therapy for HIT
Fondaparinux is a novel synthetic heparin pentasaccharide capable of inhibiting factor Xa via the action of antithrombin but devoid of anti-factor IIa (thrombin) activity, theoretically an excellent candidate agent for the treatment of HIT. Expand
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency.
The administration of mitapivat was associated with a rapid increase in the hemoglobin level in 50% of adults with pyruvate kinase deficiency, with a sustained response during a median follow-up of 29 months during the extension phase. Expand
A retrospective observational study of leucoreductive strategies to manage patients with acute myeloid leukaemia presenting with hyperleucocytosis
The results suggest that AML patients with hyperleucocytosis should be induced, if eligible, without pre‐induction leucoreduction. Expand
Successful treatment of heparin induced thrombocytopenia (HIT) with fondaparinux.
Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.
Although the risk of complications increases with severity of anemia and a genotype-phenotype relationship was observed, complications were common in all patients with PK deficiency and diagnostic testing for PK deficiency should be considered in patients with apparent congenital hemolytic anemia. Expand
A comparison of chronic manual and automated red blood cell exchange transfusion in sickle cell disease patients
This retrospective observational cohort study was conducted as part of a quality assurance audit between 1 May, 2011 and 30 April, 2012 at two SCD comprehensive care centres in London, United Kingdom to examine whether adult SCD patients on manual RBCX differ from those on automated R BCX in their ability to achieve pre-defined haematological targets, rate of complications, blood usage and clinical outcome over a 1-year period. Expand
The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions
Treatment with luspatercept resulted in significant reductions in RBC transfusion burden in adults with transfusion-dependent β-thalassemia, and was generally well tolerated in this patient population. Expand