Comparison of two vitamin D supplementation strategies in children with sickle cell disease: a randomized controlled trial

Grégoire-Pelchat, Pascale; Pastore, Yves D.; Robitaille, Nancy; LeMay, Sylvie; Khamessan, Ali; Kleiber, Niina; Nyalendo, Carine; Gagné, Nancy; Alos, Nathalie; Mailhot, Geneviève

doi:10.1111/bjh.17119

Cited by 9 publications

(14 citation statements)

References 27 publications

(62 reference statements)

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“…Although Gregoire-Pelchat et al did not demonstrate a benefit with vitamin D in the study assessed in this review [ 8 ], this same research group had previously identified an interesting trend. In tracking SCD children through a large tertiary care center hospital in Montreal, Canada, a high vitamin D deficiency rate (<50 nmol/L) was observed [ 33 ].…”

Section: Reviewmentioning

confidence: 71%

“…There have been a variety of clinical treatment approaches to the management of these pain crisis episodes in patients, but with no clearly superior therapy emerging. Therapeutic agents deployed include naturally occurring organic compounds such as amino acids arginine [ 4 ], L-glutamine (GLN) [ 5 ], amino acid derivative N-acetylcysteine (NAC) [ 6 ], vitamin D [ 7 - 8 ], and even cannabis [ 9 ]. In addition, a number of biopharmaceuticals have been used, including pregabalin [ 10 ], montelukast [ 11 ], voxelotor [ 12 ], crizanlizumab [ 13 ], morphine [ 14 - 16 ], ketamine [ 14 - 15 ], and ticagrelor [ 17 ].…”

Section: Introductionmentioning

confidence: 99%

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Emerging Therapies for the Management of Pain and Vaso-Occlusive Crises in Patients With Sickle Cell Disease: A Systematic Review of Randomized Controlled Trials

Lowe¹,

Bambhroliya²,

Patel³

et al. 2023

Cureus

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Sickle cell disease (SCD) is an inherited disorder that impairs red blood cells (RBCs) and disrupts the delivery of oxygen to tissues. There is currently no cure. Symptoms can appear as early as six months of age and include anemia, acute episodes of pain, swelling, infections, delayed growth, and vision problems. A growing number of therapies are being investigated for reducing these episodes of pain, also known as vaso-occlusive crises (VOCs). The research literature evidence, however, currently includes far more approaches that have not shown superiority versus placebo than ones that have been proven effective. The purpose of this systematic review is to evaluate the body of randomized controlled trials (RCTs) to determine the quality of support for and against the use of a variety of current and emerging therapies for treading SCD VOCs. Several important new papers have emerged since previous systematic reviews with similar objectives were published. This review was conducted according to the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines and focused on PubMed exclusively. Only RCTs were sought, and no other filters, except for a five-year historical timeline cut-off, were used. Of the 46 publications that were returned in response to the query, 18 were ultimately accepted as meeting the pre-established inclusion criteria. The Cochrane risk-of-bias tool was utilized as a quality assessment measure, and the GRADE (Grading of Recommendations, Assessment, Development, and Evaluations) framework was used to assess the certainty of the evidence. Among the included publications, five out of 18 featured positive results with superiority and statistical significance versus placebo for either reduction in pain score or number/duration of VOCs. The approaches featured therapies ranging from de novo molecules to currently available drugs approved for other indications to naturally occurring metabolites such as amino acids and vitamins. A single therapy, arginine, was supported for both clinical endpoints: pain score reduction and shortened VOC duration. Currently, two therapies are approved by the United States Food and Drug Administration (FDA) and are commercially available (crizanlizumab, ADAKVEO and L-glutamine, Endari). All other therapies are investigational only in nature. Several studies included measurement of biomarker endpoints as well as clinical outcomes. Generally, beneficial outcomes related to improving biomarker levels did not also translate into statistically significant reduction of pain scores or number/duration of VOCs. While measuring biomarkers may contribute to the understanding of pathophysiology, it does not appear to directly offer predictive value toward treatment success clinically. It can be concluded that there exists a specific opportunity to design, fund, and execute investigations that both compare emerging and existing therapies versus one another and compare combinational therapies versus placebo.

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Section: Reviewmentioning

confidence: 71%

Section: Introductionmentioning

confidence: 99%

Emerging Therapies for the Management of Pain and Vaso-Occlusive Crises in Patients With Sickle Cell Disease: A Systematic Review of Randomized Controlled Trials

Lowe¹,

Bambhroliya²,

Patel³

et al. 2023

Cureus

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“…Daily oral supplementation with high doses of D3 between 4000 and 7000 IU for 6 to 12 weeks was well tolerated and significantly enhanced health-related quality of life and physical performance in African American SCD-patients (38). A recent randomised controlled trial found that a daily dose of 1000 IU vitamin D3 and a high-dose vitamin D bolus will help SCD patients to maintain 25(OH)D levels ≥ 75 nmol/L (39).…”

Section: Resultsmentioning

confidence: 99%

Defining vitamin D deficiency in patients with sickle cell disease; A meta-analysis

2022

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Introduction: Sickle cell disease (SCD) is one of the hereditary blood disorders that affects the red blood cells. Several lines of evidence indicated that the vitamin D deficiency (VDD) is quite common in children with SCD and vitamin D supplementation enhanced health-related quality of life in these patients. The present study is aimed to assess the exact prevalence of VDD in SCD patients using meta-analysis. Materials and Methods: A systematic search was conducted in PubMed and Google Scholar to extract the papers that contain prevalence data and numbers of patients with VDD in SCD patients and healthy people. Pooled prevalence was estimated using MAJOR module of Jamovi library. The overall risk ratio of having VDD in patients with SCD was calculated using the Review Manager (RevMan 5.4.1) program. Results: A total of 26 prevalence estimates from 25 papers were included in the meta-analysis. The pooled prevalence of VDD among SCD patients is 60% (95% CIs: 50%-70%). Further, VDD is not significantly different in both SCD patients and healthy controls (risk ratio of 1.28 and 95% CI of 0.81-2.04). Conclusion: Results of this meta-analysis indicate prevalence of VDD in SCD patients. Further, a well-designed, placebo-controlled RCTs have to be conducted to determine the effects and the safety of vitamin D supplementation in children and adults with SCD.

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“…History of fractures was documented in 30% patients, and osteopenia and osteoporosis in 40%. In a recent study by Gregoire-Pelchat et al [ 41 ], the use of a high-dose vitamin D bolus combined with daily 1000 IU vitamin D3 was more effective in raising 25-OH vitamin D levels than only daily supplementation in SCD children. Further studies are needed to determine if this approach is safe and improves outcomes in children with SCD.…”

Section: Resultsmentioning

confidence: 99%

Mechanisms of Bone Impairment in Sickle Bone Disease

Giordano

Urbano

Lassandro

et al. 2021

IJERPH

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Sickle bone disease (SBD) is a chronic and invalidating complication of Sickle cell disease (SCD), a multisystem autosomal recessive genetic disorder affecting millions of people worldwide. Mechanisms involved in SBD are not completely known, especially in pediatric age. Among the hypothesized pathogenetic mechanisms underlying SBD are bone marrow compensatory hyperplasia and bone ischemic damage, both secondary to vaso-occlusive crisis (VOC), which leads to cell sickling, thus worsening local hypoxia with a negative impact on osteoblast recruitment. Furthermore, the hypoxia is a strong activator of erythropoietin, which in turn stimulates osteoclast precursors and induces bone loss. Hemolysis and iron overload due to a chronic transfusion regimen could also contribute to the onset of bone complications. Vitamin D deficiency, which is frequently seen in SCD subjects, may worsen SBD by increasing the resorptive state that is responsible for low bone mineral density, acute/chronic bone pain, and high fracture risk. An imbalance between osteoblasts and osteoclasts, with a relative decrease of osteoblast recruitment and activity, is a further possible mechanism responsible for the impairment of bone health in SCD. Moreover, delayed pubertal growth spurt and low peak bone mass may explain the high incidence of fracture in SCD adolescents. The aim of this review was to focus on the pathogenesis of SBD, updating the studies on biochemical, instrumental, and biological markers of bone metabolism. We also evaluated the growth development and endocrine complications in subjects affected with SCD.

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Comparison of two vitamin D supplementation strategies in children with sickle cell disease: a randomized controlled trial

Cited by 9 publications

References 27 publications

Emerging Therapies for the Management of Pain and Vaso-Occlusive Crises in Patients With Sickle Cell Disease: A Systematic Review of Randomized Controlled Trials

Emerging Therapies for the Management of Pain and Vaso-Occlusive Crises in Patients With Sickle Cell Disease: A Systematic Review of Randomized Controlled Trials

Defining vitamin D deficiency in patients with sickle cell disease; A meta-analysis

Mechanisms of Bone Impairment in Sickle Bone Disease

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