Proportion of Adults With Sickle Cell Anemia and Pain Crises Receiving Hydroxyurea

Stettler, Nicolas; McKiernan, Colleen; Melin, Court Q.; Adejoro, Oluwakayode; Walczak, Nancy Barker

doi:10.1001/jama.2015.3075

Cited by 62 publications

(57 citation statements)

References 8 publications

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“…If hydroxyurea is so effective, why are not more patients taking it [70]? Part of the problem relates to an unexplained reluctance to consider SCA as a life-threatening disease warranting aggressive preventive therapy.…”

Section: Expert Opinionmentioning

confidence: 99%

Hydroxyurea therapy for sickle cell anemia

McGann

Ware

2015

Expert Opinion on Drug Safety

201

204

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Introduction Sickle cell anemia (SCA) is a severe, inherited hemoglobin disorder affecting 100,000 persons in the US and millions worldwide. Hydroxyurea, a once daily oral medication, has emerged as the primary disease-modifying therapy for SCA. The accumulated body of evidence over 30 years demonstrates that hydroxyurea is a safe and effective therapy for SCA, but hydroxyurea remains underutilized for a variety of reasons. Areas covered In this review, we summarize the available evidence regarding the pharmacology, clinical, and laboratory benefits, and safety of hydroxy-urea therapy for the treatment of SCA. The purpose of this review is to provide the reader a comprehensive understanding of hydroxyurea and to reinforce the fact that hydroxyurea is a safe and effective medication for the treatment of SCA. Expert opinion In our opinion, hydroxyurea therapy should be considered standard-of-care for SCA, representing an essential component of patient management. Early initiation and broader use of hydroxyurea will alter the natural history of SCA, so affected children can live longer and healthier lives. In addition, hydroxyurea use should be extended to low-resource settings such as sub-Saharan Africa, where the burden of SCA and the need for hydroxyurea is arguably the greatest.

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Section: Expert Opinionmentioning

confidence: 99%

Hydroxyurea therapy for sickle cell anemia

McGann

Ware

2015

Expert Opinion on Drug Safety

201

204

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“…Some of the common barriers to HU treatment are lack of knowledge, misperception about HU’s disease modifying efficacy and concerns about its ‘experimental’ nature, as well as short and long term effects of HU including risks for cancer, birth defects and infertility [42]. In consideration of these issues, healthcare experts now argue for the use of HU both in pediatric and adult population [43]. In a recent clinical trial (TWiTCH study) comparing HU with chronic transfusion, high-risk children with SCD maintained their TCD velocities with no new cerebral infarcts in either treatment group.…”

Section: Hydroxyurea (Hu)mentioning

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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“…With this supportive evidence, the most recent SCD management guidelines recommend the use of hydroxyurea in both adults with three or more VOCs in any 12-month period and, regardless of symptoms, in children greater than 9 months of age diagnosed with sickle cell anemia [2,29]. Despite the overwhelming evidence supporting the clinical benefits of hydroxyurea, only one out of four adult patients and possibly even fewer are treated with this drug [36]. Extending hydroxyurea therapy to all eligible patients should be a major target for all therapeutic interventions for SCD.…”

Section: Major Therapeutic Strategies For Sickle Cell Diseasementioning

confidence: 99%

“…Clinical trials with dietary magnesium supplementation (Mg-pidolate) in patients with SCD have shown increases in red cell magnesium content, inhibition of K-Cl cotransport, and improvements in erythrocyte hydration, but there have been no completed controlled trials testing clinical efficacy of this approach [63,64]. A phase 1 study of oral Mg-pidolate supplementation established the MTD for children with SCD concomitantly treated with hydroxyurea [36]. As discussed above, a study on oral Mg pidolate supplementation in patients with SC disease was terminated early due to poor enrollment, but the limited data collected showed no changes in erythrocyte Mg or cell dehydration [37].…”

Section: Major Therapeutic Strategies For Sickle Cell Diseasementioning

confidence: 99%

2015 Clinical trials update in sickle cell anemia

2015

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Polymerization of HbS and cell sickling are the prime pathophysiological events in sickle cell disease (SCD). Over the last 30 years, a substantial understanding at the molecular level has been acquired on how a single amino acid change in the structure of the beta chain of hemoglobin leads to the explosive growth of the HbS polymer and the associated changes in red cell morphology. O2 tension and intracellular HbS concentration are the primary molecular drivers of this process, and are obvious targets for developing new therapies. However, polymerization and sickling are driving a complex network of associated cellular changes inside and outside of the erythrocyte, which become essential components of the inflammatory vasculopathy and result in a large range of potential acute and chronic organ damages. In these areas, a multitude of new targets for therapeutic developments have emerged, with several ongoing or planned new therapeutic interventions. This review outlines the key points of SCD pathophysiology as they relate to the development of new therapies, both at the pre-clinical and clinical levels.

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Proportion of Adults With Sickle Cell Anemia and Pain Crises Receiving Hydroxyurea

Cited by 62 publications

References 8 publications

Hydroxyurea therapy for sickle cell anemia

Hydroxyurea therapy for sickle cell anemia

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

2015 Clinical trials update in sickle cell anemia

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