Hydroxyurea for sickle cell disease

Jones, Ashley P; Davies, Sally C.; Olujohungbe, Adebayo

doi:10.1002/14651858.cd002202

Cited by 31 publications

(9 citation statements)

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“…Two systematic reviews were identified: one involving adults 16 and another children, 17 Additionally, there was a recent Cochrane Library review of “Hydroxyurea for sickle cell disease”. 18 Together, these reviews reported only two randomised controlled prospective trials of hydroxyurea in sickle cell disease. The Multi-Center Study of Hydroxyurea (MSH, see text) was a “high quality” double-blinded trial of hydroxyurea in 299 adults with severe sickle cell anaemia, that demonstrated significant reduction in pain, acute chest syndrome, hospitalisation, and transfusion in the treated group.…”

Section: Discussionmentioning

confidence: 99%

“…16–18 BABY HUG is the first randomised double-blinded trial of hydroxyurea in children with SCA (see Panel: Research in Context). It differs from all other paediatric trials (except for the pilot HUSOFT study) in the subjects’ much younger age at enrollment (mean age 13·6 months) and the absence of a prerequisite for a severe clinical course (e.g., ≥3 vaso-occlusive events in the previous year).…”

Section: Discussionmentioning

confidence: 99%

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Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG)

et al. 2011

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Background Sickle cell anaemia (SCA) is associated with significant morbidity from acute complications and organ dysfunction beginning in the first year of life. In the first multicenter randomised double-blinded trial in very young children with SCA, the impact of hydroxyurea (hydroxycarbamide) therapy on organ dysfunction, clinical complications, and laboratory findings, and its toxicity, were examined. Methods Eligible subjects had HbSS or Sβ0thalassaemia, were age 9–18 months at randomisation, and were not selected for clinical severity. Subjects received liquid hydroxyurea, 20 mg/kg/day, or placebo for two years. Primary study endpoints were splenic function (qualitative uptake on 99Tc spleen scan) and renal function (glomerular filtration rate by 99mTc-DTPA clearance). Additional evaluations included: blood counts, HbF, chemistry profiles, spleen function biomarkers, urine osmolality, neurodevelopment, transcranial Doppler ultrasonography, growth, and mutagenicity. Study visits occurred every two to four weeks. Findings Ninety-six subjects received hydroxyurea and 97 placebo; 86% completed the study. Significant differences were not seen for the primary endpoints, but suggestive benefit was noted in quantitative measures of spleen function. Hydroxyurea significantly decreased pain and dactylitis with trends for decreased acute chest syndrome, hospitalisation and transfusion. Hydroxyurea increased haemoglobin and HbF and decreased WBC count. Toxicity was limited to mild-moderate neutropaenia. Interpretation Although hydroxyurea treatment did not reduce splenic and renal dysfunction assessed by primary endpoint measures, it resulted in major clinical benefit because of diminished acute complications, favorable haematologic results, and a lack of unexpected toxicities. Based on the safety and efficacy data from this trial, hydroxyurea can now be considered for all very young children with SCA.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG)

et al. 2011

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“…Hydroxyurea is an oral chemotherapeutic drug used for individuals with SCD to ameliorate some of the clinical problems of sickle cell disease, especially pain (Jones, Davies, & Olujohungbe, 2001). One recent study examined the cognitive abilities of children with SCD on hydroxyurea as compared to children with SCD not receiving hydroxyurea, and found that children on hydroxyurea demonstrated better performance on measures of verbal comprehension, fluid reasoning, and general cognitive ability as compared to the control group (Puffer et al, 2007).…”

Section: Medical Interventionsmentioning

confidence: 99%

The Role of Neuropsychological Evaluation in Pediatric Sickle Cell Disease

Daly

Kral

Tarazi

2011

The Clinical Neuropsychologist

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Neuropsychological impairment occurs in children and adolescents with sickle cell disease. While the neuropsychological sequelae associated with cerebrovascular disease in these children have been identified, a well-informed clinical approach to neuropsychological evaluation for these vulnerable children has not been delineated. Moreover, issues related to standard of care for children and adolescents with sickle cell disease and transition to adulthood remain understudied despite the relevance to long-term medical, psychosocial, and neuropsychological outcomes. We provide recommendations for an informed approach to neuropsychological evaluation of youth with sickle cell disease that is determined by pertinent illness-related factors, consideration of general developmental level and timing of evaluation (i.e., baseline vs follow-up evaluation), relationship to medical treatment, and transition to adulthood. Although the neuropsychological methods presented in this manuscript are specific to sickle cell disease, this empirically informed approach may serve as a model for other pediatric populations.

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“…Hydroxyurea modifies the comAfrican Health Sciences Vol 17 Issue 1, March, 2017 255 plex pathophysiology of SCD through multiple mechanisms including induction of fetal haemoglobin production, improving cellular hydration, marrow suppression (reduction of leucocyte and platelet counts), anti-adhesive properties and production of nitric oxide (vasodilator) 19,22 . Clinically, this translates to reduced frequency and intensity of bone pain crisis, hospitalizations, blood transfusions, thus better quality of life 20,23,24 . This study evaluates the level of awareness, drug use pattern, clinic-laboratory benefits, level of compliance and safety/toxicity profile of hydroxyurea in a cross section of Nigerian SCD patients.…”

Section: Introductionmentioning

confidence: 99%

Hydroxyurea therapy in adult Nigerian sickle cell disease: a monocentric survey on pattern of use, clinical effects and patient’s compliance

2017

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Background: The clinical prospects of hydroxyurea therapy in the management of sickle cell disease (SCD) require evaluation in the Nigerian setting to develop indigenous guidelines. This survey examines the pattern of hydroxyurea therapy, its clinico-haematologic benefits and safety profile in Nigerian SCD subjects. Methods: A cross sectional pilot survey was carried out among 60 adult SCD subjects over 3 months. Data on clinical phenotypes, relevant haematological parameters and details of hydroxyurea therapy were obtained using a structured questionnaire through an interview process and case file review. Results: The median age was 30 years. Thirty-four (56.7%) of the subjects are aware of hydroxyurea therapy in SCD. Twenty-four (40%) SCD patients had previously used hydroxyurea. Only 4 subjects were fully compliant. Reasons for non-compliance included poor knowledge and lack of funds. In particular, hydroxyurea reduced leucocyte count and increased mean red cell volume (MCV) in compliant subjects. Conclusion: Hydroxyurea use is low among Nigerian SCD subjects despite its proven efficacy/clinical prospects in the developed nations. Large scale multicenter studies and clinical trials are needed to form a basis for developing standard local treatment protocol for its use.

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Hydroxyurea for sickle cell disease

Cited by 31 publications

References 61 publications

Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG)

Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG)

The Role of Neuropsychological Evaluation in Pediatric Sickle Cell Disease

Hydroxyurea therapy in adult Nigerian sickle cell disease: a monocentric survey on pattern of use, clinical effects and patient’s compliance

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