Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey

Colombatti, Raffaella; Palazzi, Giovanni; Masera, Nicoletta; Notarangelo, Lucia Dora; Bonetti, Elisa; Samperi, Piera; Barone, Angelica; Perrotta, Silverio; Facchini, Elena; Miano, Maurizio; Vecchio, Giovanni Carlo Del; Guerzoni, Maria Elena; Cortí, Paola; Menzato, Federica; Cesaro, Simone; Casale, Maddalena; Rigano, Paolo; Forni, Gian Luca; Russo, Giovanna; Sainati, Laura

doi:10.1002/pbc.26774

Cited by 30 publications

(36 citation statements)

References 33 publications

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“…As expected, in contrast to SCA patients who did not receive HU, those who received this treatment presented increased HbF levels and improvements in hemolytic, hepatic and inflammatory profiles (hemoglobin, hematocrit, MCV, MCH, reticulocytes, MCHC, RDW, AST, total bilirubin, LDH, WBC, neutrophils, eosinophils, lymphocytes, monocytes, platelets, and plateletcrit). These findings corroborate previous studies, which also demonstrated improvements in hemolytic, hepatic and inflammatory profiles in patients with SCA undergoing HU therapy (de Souza Santos and Maia, 2010;Voskaridou et al, 2010;de Souza Torres et al, 2012;Pallis et al, 2014;Belini Junior et al, 2015;Shome et al, 2016;Quarmyne et al, 2017;Colombatti et al, 2018). We further observed an increase in HDL-C concentration, demonstrating the effect of HU on lipid metabolism, which is consistent with our previous results (Yahouedehou et al, 2018b;Yahouedehou et al, 2019).…”

Section: Discussionsupporting

confidence: 93%

Sickle Cell Anemia: Variants in the CYP2D6, CAT, and SLC14A1 Genes Are Associated With Improved Hydroxyurea Response

Yahouédéhou

Neres²,

Guarda

et al. 2020

Front. Pharmacol.

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Differences in hydroxyurea response in sickle cell anemia may arise due to a series of factors with genetic factors appearing to be predominant. This study aims to investigate the effects of single nucleotide polymorphisms in genes encoding drug-metabolizing enzymes and solute carriers on hydroxyurea response, in patients with sickle cell anemia. For that purpose, a total number of 90 patients with sickle cell anemia were recruited, 45 were undergoing hydroxyurea treatment, while 45 were not under the treatment. Association analyses were performed between CYP3A4 (rs2740574), CYP2D6 (rs3892097), CAT (rs7943316 and rs1001179), and SLC14A1 (rs2298720) variants and laboratory parameters. According to our findings, patients with hydroxyurea treatment demonstrated higher HbF levels and a significant improvement in hemolytic, hepatic, inflammatory, and lipid parameters in comparison to those without the treatment. We also found significant associations between the CYP2D6 (rs3892097), CAT (rs7943316 and rs1001179), and SLC14A1 (rs2298720) variants and an improvement of the therapeutic effects, specifically the hemolytic, hepatic, inflammatory, lipid, and renal parameters. In conclusion, our results highlight the importance of the investigated variants, and their strong association with hydroxyurea efficacy in patients with sickle cell anemia, which may be considered in the future as genetic markers.

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Section: Discussionsupporting

confidence: 93%

Sickle Cell Anemia: Variants in the CYP2D6, CAT, and SLC14A1 Genes Are Associated With Improved Hydroxyurea Response

Yahouédéhou

Neres²,

Guarda

et al. 2020

Front. Pharmacol.

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“…A large body of preclinical and clinical evidence has demonstrated that HU reduces the morbidity and mortality of both adults and children (including infants) with SCD, with a favorable tolerability profile and without significant short-term and long-term safety concerns [56–68]. A series of real-life reports documenting the use of HU in Italy have recently been published [20, 22, 69]. HU was shown to be beneficial as a treatment option in a retrospective, nationwide cohort study of 1,638 patients with SCD of whom 652 patients had received HU during their disease course [20].…”

Section: Introductionmentioning

confidence: 99%

Current challenges in the management of patients with sickle cell disease – A report of the Italian experience

Russo

Franceschi

Colombatti

et al. 2019

Orphanet J Rare Dis

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Sickle cell disease (SCD) is an inherited red blood cell disorder caused by a structural abnormality of hemoglobin called sickle hemoglobin (HbS). Clinical manifestations of SCD are mainly characterized by chronic hemolysis and acute vaso-occlusive crisis, which are responsible for severe acute and chronic organ damage. SCD is widespread in sub-Saharan Africa, in the Middle East, Indian subcontinent, and some Mediterranean regions. With voluntary population migrations, people harboring the HbS gene have spread globally. In 2006, the World Health Organization recognized hemoglobinopathies, including SCD, as a global public health problem and urged national health systems worldwide to design and establish programs for the prevention and management of SCD. Herein we describe the historical experience of the network of hemoglobinopathy centers and their approach to SCD in Italy, a country where hemoglobinopathies have a high prevalence and where SCD, associated with different genotypes including ß-thalassemia, is present in the native population.

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“…Follow‐up from the subjects enrolled in these trials showed salutary long‐term benefits in morbidity, mortality, laboratory tests and preservation of organ function (McGann & Ware, , ). Several studies conducted in Italy, Belgium and India have shown significant clinical benefits attributable to HC (Jain et al , ; Nottage et al , ; Le et al , ; Colombatti et al , ). However, these studies were limited with respect to the relatively small simple size, single institution design and shorter follow‐up periods in randomized clinical trials.…”

Section: Introductionmentioning

confidence: 99%

Hydroxycarbamide adherence and cumulative dose associated with hospital readmission in sickle cell disease: a 6‐year population‐based cohort study

et al. 2018

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Sickle cell disease (SCD) is a congenital haemoglobinopathy that causes frequent acute care/emergency room visits and hospital admissions for affected individuals. Evidence from population-based studies demonstrating the role of hydroxycarbamide (HC, also termed hydroxyurea) in reducing hospital readmission rates is limited. Our objective was to describe the use of HC and its association with acute care utilization and readmission rates using a large, nationally-representative US health insurance claims database over a 6-year period between 2009 and 2014. We identified 20 721 SCD-related inpatient and acute care encounters. Patients had been exposed to HC within 6 months prior to admission in 4263 (21%) of SCD-related admission events. HC use was more common among children aged 10-17 years and young adults aged 18-29 years. HC was associated with lower 30-day all-cause readmission rates in adults treated with average daily doses ≥1 g (odds ratio [OR], 0·72, 95% confidence interval [CI] 0·52-0·99) and doses of 0·5-1 g (OR, 0·73, 95% CI 0·57-0·93), compared to HC treatment with average daily doses of <0·5 g; adherence to HC with proportion of days covered of ≥0·80 was also associated with significantly lower 30-day all-cause readmission risks (OR, 0·59, 95% CI 0·41-0·84). Optimal therapeutic dosing and adherence to HC treatment significantly reduces 30-day readmissions among patients with SCD.

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Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey

Abstract: HU effectiveness is demonstrated in a national cohort of children with SCD living in Italy, even at a lower dose than recommended, revealing good adherence to a treatment program by a socially vulnerable group of patients such as immigrants.

Cited by 30 publications

References 33 publications

Sickle Cell Anemia: Variants in the CYP2D6, CAT, and SLC14A1 Genes Are Associated With Improved Hydroxyurea Response

Sickle Cell Anemia: Variants in the CYP2D6, CAT, and SLC14A1 Genes Are Associated With Improved Hydroxyurea Response

Current challenges in the management of patients with sickle cell disease – A report of the Italian experience

Hydroxycarbamide adherence and cumulative dose associated with hospital readmission in sickle cell disease: a 6‐year population‐based cohort study

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