Hydroxyurea (hydroxycarbamide) for sickle cell disease

Nevitt, Sarah J; Jones, Ashley P; Howard, Jo

doi:10.1002/14651858.cd002202.pub2

Cited by 122 publications

(135 citation statements)

References 125 publications

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“…Parents’ factors are well‐known causes of lack of compliance with HU treatment as well as adherence to comprehensive care . Free health care, as the one provided in Europe, is not always sufficient to ensure compliance . We believe that the education and motivation of patients and families and the type of comprehensive care that ensures attention to all aspects of the children's life (linguistic, social, cultural) as the one developed in many pediatric hematology centers in Italy allowed the disease to be seen as a possibility of social integration in the hosting country.…”

Section: Discussionmentioning

confidence: 98%

“…Previous experiences in India with SS/Sβ° patients and also with SDPunjab patients have shown the efficacy of low‐dose regimens . A recent Cochrane review confirms there is still not sufficient evidence to determine which is the best dose and probably more data from different settings is needed. Very few short‐term toxicities were reported in our cohort, probably due to the low dose used.…”

Section: Discussionmentioning

confidence: 99%

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

Colombatti

Palazzi²,

Masera

et al. 2017

Pediatric Blood & Cancer

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Section: Discussionmentioning

confidence: 98%

Section: Discussionmentioning

confidence: 99%

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

Colombatti

Palazzi²,

Masera

et al. 2017

Pediatric Blood & Cancer

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“…Studies of HU were excluded from this review because these have been discussed extensively in previous reviews. [22][23][24]29 Both inactive control as well as active control interventions were considered.…”

Section: Study Selectionmentioning

confidence: 99%

“…HU has been shown to be highly efficacious in SCD. [22][23][24] However, patients receiving HU often do not experience complete relief from their symptoms, and it is currently the only disease-modifying therapy for SCD. Therefore, there remains an urgent need for new therapeutic approaches.…”

Section: Introductionmentioning

confidence: 99%

Pharmacotherapeutical strategies in the prevention of acute, vaso-occlusive pain in sickle cell disease: a systematic review

et al. 2017

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Sickle-cell disease (SCD) is characterized by frequent and painful vaso-occlusive crises (VOCs).Various treatments have been evaluated over the years. However, a clear overview is lacking.The objective of this study was to systematically review all pharmacotherapeutical strategies in the prevention of VOCs beyond hydroxyurea. We performed a systematic literature search (MEDLINE, Embase, CENTRAL). Eligible studies were controlled clinical trials evaluating pharmacotherapeutical interventions targeting the reduction of VOCs in patients with SCD.Primary outcomes were the number or duration of SCD-related pain days, VOCs, or hospital admissions for VOCs. Secondary outcomes included time to first VOC or hospital admission for a VOC. A standardized data extraction sheet was used. The methodological quality of studies was assessed using Cochrane's risk-of-bias tool. A total of 36 studies were included in this review, covering 26 different prophylactic interventions. The most promising interventions for reducing the frequency of either VOCs or hospitalizations were the oral antioxidants L-glutamine and v-3 fatty acids and the IV antiadhesive agent crizanlizumab. Twenty-three studies did not show any beneficial effect of the intervention under investigation, and 6 studies were either too small or methodologically inadequate to draw conclusions. Because of the heterogeneity of interventions, no meta-analysis was performed. In conclusion, this review identified 3 promising pharmacotherapeutical strategies in the prevention of VOCs in SCD. Importantly, this study highlights the discrepancy between the significant burden of SCD worldwide and the low number of adequate trials performed. This review was

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“…anticoagulant, anti-platelet therapy) [36]. Exchange blood transfusions are the main component of primary stroke prophylaxis, and recent evidence suggests that HU may also be effective in preventing stroke-related complications [37,57,58]…”

Section: Clinical Challengesmentioning

confidence: 99%

Sickle cell disease: a malady beyond a hemoglobin defect in cerebrovascular disease

Ansari

Moufarrej

Pawliński

et al. 2017

Expert Review of Hematology

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Introduction Sickle cell disease (SCD) is a devastating monogenic disorder that presents as a multisystem illness and affects approximately 100,000 individuals in the United States alone. SCD management largely focuses on primary prevention, symptomatic treatment and targeting of hemoglobin polymerization and red blood cell sickling. Areas covered This review will discuss the progress of SCD over the last few decades, highlighting some of the clinical (mainly cerebrovascular) and psychosocial challenges of SCD in the United States. In addition, focus will also be made on the evolving science and management of this inherited disease. Expert Commentary Until recently hydroxyurea (HU) has been the only FDA approved therapy for SCD. However, advancing understanding of SCD pathophysiology has led to multiple clinical trials targeting SCD related thrombo-inflammation, abnormal endothelial biology, increased oxidant stress and sickle cell mutation. Yet, despite advancing understanding, available therapies are limited. SCD also imposes great psychosocial challenges for the individual and the affected community, which has previously been under-recognized. This has created a pressing need for complementary adjuvant therapies with repurposed and novel drugs, in addition to the establishment of comprehensive clinics focusing on both the medical treatment and the psychosocial issues associated with SCD.

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Hydroxyurea (hydroxycarbamide) for sickle cell disease

Abstract: Analysis 1.3. Comparison 1 Hydroxyurea versus placebo for participants with sickle cell disease, Outcome 3 Proportion experiencing life threatening events during study.

Cited by 122 publications

References 125 publications

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

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

Pharmacotherapeutical strategies in the prevention of acute, vaso-occlusive pain in sickle cell disease: a systematic review

Sickle cell disease: a malady beyond a hemoglobin defect in cerebrovascular disease

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