How Yogurt Science Could Lead To A Cure For Sickle Cell Anemia
The discovery of CRISPR and gene editing was not made by a geneticist or a stem cell biologist. CRISPR was discovered by a bunch of microbiologists, scientists who study bacteria and viruses.
In fact, much of the foundational work in CRISPR was done by nutritional microbiologists who wanted to understand how the bacteria we use to make cheese and yogurt are able to fight off viral infections. Imagine that! The future of gene therapy began in a yogurt vat.
New Pain Protocol Cut Time to Treatment in Sickle Cell Kids
Using intranasal fentanyl instead of oral morphine cut delays
Switching from an oral morphine protocol to intranasal fentanyl for children with sickle cell disease and vaso-occlusive crisis significantly reduced the time to first analgesia — an indicator of improved care quality, researchers said here.
A chart review of 107 pediatric sickle cell patients treated at the Centre Hospitalier Universitaire Sainte-Justine here, covering two six-month periods before and after the protocol change, showed the time from patient registration to first opiate dose decreased by 46 minutes, reported Yves Pastore, MD, co-director of the hospital’s sickle cell disease program and assistant professor at the Université de Montréal here, at the American Society of Pediatric Hematology/Oncology’s annual meeting.
Inequities in funding and research on sickle cell disease
Remember a year ago when your friends were pouring buckets of ice cold water over the heads in the name of science? In total, a whopping $220 million was raised from the challenge to help from 12,000 to 15,000 people in United States who reportedly live with Amyotrophic Lateral Sclerosis.
But did you know that sickle cell anemia is the single most common life-threatening genetic disease in the United States? I sure didn’t.
Over 100,000 Americans suffer from sickle cell disease and it is a torturous, painful life to live. Yet, the funding and publicity of sickle cell disease lags drastically far behind that of virtually every other genetic illness.
Articles in the Medical Literature
- Transfusion. 2017 May 3. doi: 10.1111/trf.14154. [Epub ahead of print]
Erythrocytapheresis is an important procedure in the management of certain complications of sickle cell disease, including acute stroke, stroke prevention, acute chest syndrome, and multiorgan failure. Erythrocytapheresis in sickle cell disease simply entails the removal of the patient’s red blood cells containing the abnormal sickle hemoglobin and replacing them with normal red blood cells carrying normal hemoglobin. In these procedures, the patient’s plasma is not exchanged but is returned to the patient. Several studies have demonstrated that the plasma of patients with sickle cell disease contains several components that increase blood viscosity and initiate or promote vaso-occlusion. These factors include increased levels of globulins, especially immunoglobulin G, acute-phase reactants, fibrinogen, coagulation factors, inflammatory mediators, and heme in the steady state and increase further during painful crises. This may explain why, in certain complications of sickle cell disease, such as acute chest syndrome, hepatic crisis, and priapism, erythrocytapheresis by itself may not be effective despite repetitive cycles of red blood cell exchange. The use of therapeutic plasma exchange in addition to erythrocytapheresis in these situations seems to be useful in resolving them more efficiently. The role of therapeutic plasma exchange in the management of certain complications of sickle cell disease needs further evaluation. This commentary addresses the role of therapeutic plasma exchange in the management of complications of sickle cell disease.
© 2017 AABB.
- Br J Haematol. 2017 May 3. doi: 10.1111/bjh.14693. [Epub ahead of print]
Möckesch B1,2,3, Connes P4,2,3,5, Charlot K1,2,3,6, Skinner S4,3, Hardy-Dessources MD2,3, Romana M2,3, Jumet S1, Petras M7, Divialle-Doumdo L7, Martin C4,3, Tressières B8, Tarer V7, Hue O1, Etienne-Julan M7, Antoine S1, Pialoux V4,3,5.
Oxidative stress and haemolysis-associated nitric oxide (NO) depletion plays a crucial role in the development of vasculopathy in sickle cell anaemia (SS). However it remains unknown whether oxidative stress and haemolysis levels influence vascular function in patients with sickle haemoglobin C disease (SC). Microvascular response to heat (using Laser Doppler flowmetry on finger), oxidative stress biomarkers, NO metabolites, endothelin-1 and haematological parameters were compared between patients with SS and SC. Vascular function, oxidative and nitrosative markers were also measured in healthy (AA) children. SS and SC had increased plasma advanced oxidation protein products (AOPP), malondialdehyde, plasma antioxidant activities and NO end products, compared to AA. SC had lower catalase activity compared to AA and SS. Haemolytic rate, glutathione peroxidase and nitrotyrosine concentrations were significantly increased in children with SS compared to SC and AA. SS and SC had impaired microvascular reactivity compared to AA. In SS, the plateau phase of the response to local thermal heating was negatively associated with nitrotyrosine and AOPP. No association between vascular function parameters and oxidative stress markers was observed in SC. Mild haemolysis in SC, compared to SS, may limit oxidative and nitrosative stress and could explain the better preserved microvascular function in this group.
© 2017 John Wiley & Sons Ltd.
- Am J Med Qual. 2017 Apr 1:1062860617707262. doi: 10.1177/1062860617707262. [Epub ahead of print]
Sickle cell disease (SCD), an inherited red blood cell disorder, is characterized by anemia, end-organ damage, unpredictable episodes of pain, and early mortality. Emergency department (ED) visits and hospitalizations are frequent, leading to increased burden on patients and increased health care costs. This study assessed the effects of a multidisciplinary care team intervention on acute care utilization among adults with SCD. The multidisciplinary care team intervention included monthly team meetings and development of individualized care plans. Individualized care plans included targeted pain management plans for management of uncomplicated pain crisis. Following implementation of the multidisciplinary care team intervention, a significant decrease in ED utilization was identified among those individuals with a history of high ED utilization. Findings highlight the potential strength of multidisciplinary interventions and suggest that targeting interventions toward high-utilizing subpopulations may offer the greatest impact.
- Biomed Res Int. 2017;2017:4070547. doi: 10.1155/2017/4070547. Epub 2017 Mar 28.
Background. Patients with SCD now usually live well into adulthood. Whereas transitions into adulthood are now often studied, little is published about aging beyond the transition period. We therefore studied age-associated SCD differences in utilization, pain, and psychosocial variables. Methods. Subjects were 232 adults in the Pain in Sickle Cell Epidemiology Study (PiSCES). Data included demographics, comorbidity, and psychosocial measures. SCD-related pain and health care utilization were recorded in diaries. We compared 3 age groups: 16-25 (transition), 26-36 (younger adults), and 37-64 (older adults) years. Results. Compared to the 2 adult groups, the transition group reported fewer physical challenges via comorbidities, somatic complaints, and pain frequency, though pain intensity did not differ on crisis or noncrisis pain days. The transition group utilized opioids less often, made fewer ambulatory visits, and had better quality of life, but these differences disappeared after adjusting for pain and comorbidities. However, the transition group reported more use of behavioral coping strategies. Conclusion. We found fewer biological challenges, visits, and better quality of life, in transition-aged versus older adults with SCD, but more behavioral coping. Further study is required to determine whether age-appropriate health care, behavioral, or other interventions could improve age-specific life challenges of patients with SCD.
PMCID: PMC5387810 Free PMC Article
- J Thromb Haemost. 2017 Apr 29. doi: 10.1111/jth.13728. [Epub ahead of print]
Endothelial activation plays a central role in the pathophysiology of vaso-occlusion in sickle cell disease (SCD), facilitating adhesive interactions with circulating blood cells. Upon activation various adhesive molecules are expressed, including von Willebrand factor (VWF). Increased VWF levels have been observed in patients with SCD during steady state. However, the role of VWF in the pathogenesis of SCD vaso-occlusion is unclear.
To longitudinally assess quantity and reactivity of VWF and its regulating protease ADAMTS-13 during vaso-occlusive crisis (VOC).
In this observational study we obtained sequential blood samples in adult SCD patients during VOC.
VWF reactivity was significantly higher during VOC (active VWF, VWF GPIb-binding activity and high-molecular weight multimers), whereas platelet count and levels of ADAMTS-13 antigen and ADAMTS-13 activity were concomitantly lower when compared to steady state. Levels of VWF antigen, VWF propeptide and ADAMTS-13 specific activity did not change during VOC. VWF reactivity correlated strongly with markers of inflammation and neutrophil activation, and was inversely correlated with the platelet count. In patients that developed acute chest syndrome, levels of VWF, VWF:PP and aVWF were significantly higher, while the ADAMTS13:act was lower than in patients without this complication.
We provide first evidence that VOC in SCD is associated with increased reactivity of VWF, without a pronounced ADAMTS-13 deficiency. This hyper-reactivity may be explained by resistance of VWF to proteolysis, secondary to processes such as inflammation and oxidative stress. Hyper-adhesive VWF, scavenging blood cells in the microcirculation, may thereby amplify and sustain VOC in SCD. This article is protected by copyright. All rights reserved.
This article is protected by copyright. All rights reserved.
- Can J Anaesth. 2017 Apr 28. doi: 10.1007/s12630-017-0883-3. [Epub ahead of print]
Approximately 200,000 individuals worldwide are born annually with sickle cell disease (SCD). Regions with the highest rates of SCD include Africa, the Mediterranean, and Asia, where its prevalence is estimated to be 2-6% of the population. An estimated 70,000-100,000 people in the United States have SCD. Due to enhanced newborn screening, a better understanding of this disease, and more aggressive therapy, many sickle cell patients survive into their adult years and present more frequently for surgery.
The authors identified relevant medical literature by searching PubMed, MEDLINE®, EMBASE™, Scopus™, Web of Science, and Google Scholar databases for English language publications appearing from 1972-September 2016. Case reports, abstracts, review articles, and original research articles were reviewed-with particular focus on the pathophysiology and medical management of SCD and any anesthesia-related issues.
Perioperative physicians should be familiar with the triggers of a sickle cell crisis and vaso-occlusive disease. Sickle cell disease affects various organ systems, including the central nervous, cardiovascular, pulmonary, genitourinary, and musculoskeletal systems. Preoperative assessment should focus on end-organ dysfunction. Controversy continues regarding if and when sickle cell patients should receive transfusions and which anesthetic technique (regional or general) confers any benefits. Timely, appropriate, and sufficient analgesia is critical, especially when patients experience a vaso-occlusive crisis, acute chest syndrome, or acute postoperative pain.
Effective management of SCD patients in the perioperative setting requires familiarity with the epidemiology, pathophysiology, clinical manifestations, and treatment of SCD.
- Pediatr Blood Cancer. 2017 Apr 28. doi: 10.1002/pbc.26607. [Epub ahead of print]
Anemia, hemolysis-driven vasculopathy, and intrinsic myocardial injury have been proposed as predisposing factors to cardiac disease in sickle cell anemia (SCA). The individual impact of these mechanisms on the cardiac features of SCA and the way they influence complications such as sudden death and dysrhythmias have been unclear. Recent findings of an acquired restrictive SCA-related cardiomyopathy, driven by myocardial fibrosis, may explain some of these cardiac features. Given the complexity of cardiac pathology in SCA, using additional parameters to tricuspid regurgitant jet velocity (left atrial volume, diastolic parameters, NT-proBNP) may improve the accuracy of noninvasive screening for cardiopulmonary complications in SCA.
© 2017 Wiley Periodicals, Inc.
- Hum Gene Ther Clin Dev. 2017 Apr 27. doi: 10.1089/humc.2017.029. [Epub ahead of print]
Steady state bone marrow (BM) is the preferred hematopoietic stem cell (HSC) source for gene therapy in sickle cell disease (SCD) due to the recognized risk of vaso-occlusive crisis during granulocyte colony-stimulating factor mobilization. We previously established clinically relevant HSC gene transfer in the rhesus model following transplantation of mobilized peripheral blood (PB) CD34+ cells transduced with lentiviral vectors. In this study, we examined steady state bone marrow (BM) in the rhesus competitive repopulation model and demonstrate similar gene marking in vitro and in vivo, as compared to mobilized PB CD34+ cells. We then evaluated PB and steady state BM in subjects with SCD, and observed a higher frequency of CD34+ cells when compared to controls, likely due to enhanced hematopoiesis. However, CD34+ cell counts were reduced in both the PB and BM in patients treated with hydroxyurea, and hydroxyurea treatment strongly inhibited iPS cell generation from SCD subjects. Our data support that steady state BM is a useful HSC source for SCD gene therapy with similar transduction. The lower CD34+ percentages observed with hydroxyurea treatment warrants withholding hydroxyurea temporarily prior to harvesting HSCs. Our results are important for the design of gene targeting strategies for SCD.
- Am J Hematol. 2017 Apr 25. doi: 10.1002/ajh.24770. [Epub ahead of print]
Galadanci N1, Abdullahi SU2, Vance LD3, Tabari AM4, Ali S4, Belonwu R2, Salihu A5, Galadanci AA1, Jibir BW6, Bello-Manga H7, Neville K8, Kirkham FJ9, Shyr Y10, Phillips S10, Covert BV11, Kassim AA12, Jordan LC11, Aliyu MH13, DeBaun MR11.
The vast majority of children with sickle cell anemia (SCA) live in Africa, where evidence-based guidelines for primary stroke prevention are lacking. In Kano, Nigeria, we conducted a feasibility trial to determine the acceptability of hydroxyurea therapy for primary stroke prevention in children with abnormal transcranial Doppler (TCD) measurements. Children with SCA and abnormal non-imaging TCD measurements (≥ 200 cm/s) received moderate fixed-dose hydroxyurea therapy (∼20 mg/kg/day). A comparison group of children with TCD measurements < 200 cm/s was followed prospectively. Approximately 90% (337 of 375) of families agreed to be screened, while 92% (25 of 27) of those with abnormal TCD measurements, on two separate occasions, enrolled in the trial. No participant elected to withdraw from the trial. The average mean corpuscular volume increased from 85 fL at baseline to 101.3 fL at 24 months, demonstrating adherence to hydroxyurea. The comparison group consisted of 210 children, of which four developed abnormal TCD measurements, and were started on hydroxyurea. None of the monthly research visits were missed (n = total 603 visits). Two and ten deaths occurred in the treatment and comparison groups, with mortality rates of 2.69 and 1.81 per 100 patient-years, respectively (p = 0.67). Our results provide strong evidence, for high family recruitment, retention, and adherence rates, to undertake the first randomized controlled trial with hydroxyurea therapy for primary stroke prevention in children with SCA living in Africa. This article is protected by copyright. All rights reserved.
© 2017 Wiley Periodicals, Inc.
- West J Emerg Med. 2017 Apr;18(3):335-339. doi: 10.5811/westjem.2016.11.32273. Epub 2017 Feb 7.
A subpopulation of sickle-cell disease patients, termed super-utilizers, presents frequently to emergency departments (EDs) for vaso-occlusive events and may consume disproportionate resources without broader health benefit. To address the healthcare needs of this vulnerable patient population, we piloted a multidisciplinary intervention seeking to create and use individualized patient care plans that alter utilization through coordinated care. Our goals were to assess feasibility primarily, and to assess resource use secondarily.
We evaluated the effects of a single-site interventional study targeted at a population of adult sickle-cell disease super-utilizers using a pre- and post-implementation design. The pre-intervention period was 06/01/13 to 12/31/13 (seven months) and the post-intervention period was 01/01/14 to 02/28/15 (14 months). Our approach included patient-specific best practice advisories (BPA); an ED management protocol; and formation of a “medical home” for these patients.
For 10 subjects targeted initially we developed and implemented coordinated care plans; after deployment, we observed a tendency toward reduction in ED and inpatient utilization across all measured indices. Between the annualized pre- and post-implementation periods we found the following: ED visits decreased by 16.5 visits/pt-yr (95% confidence interval [CI] [-1.32-34.2]); ED length of state (LOS) decreased by 115.3 hours/pt-yr (95% CI [-82.9-313.5]); in-patient admissions decreased by 4.20 admissions/pt-yr (95% CI [-1.73-10.1]); in-patient LOS decreased by 35.8 hours/pt-yr (95% CI [-74.9-146.7]); and visits where the patient left before treatment were reduced by an annualized total of 13.7 visits. We observed no patient mortality in our 10 subjects, and no patient required admission to the intensive care unit 72 hours following discharge.
This effort suggests that a targeted approach is both feasible and potentially effective, laying a foundation for broader study.
PMCID: PMC5391880 Free PMC Article
Conflict of interest statement
Conflicts of Interest: By the WestJEM article submission agreement, all authors are required to disclose all affiliations, funding sources and financial or management relationships that could be perceived as potential sources of bias. Research reported in this publication was partly supported by the National Center for Advancing Translational Sciences of the National Institutes of Health under Award Number UL1TR001427. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
- J Pain Res. 2017 Apr 5;10:787-795. doi: 10.2147/JPR.S131156. eCollection 2017.
Subanesthetic doses of ketamine, an N-methyl-d-aspartate receptor antagonist used as an adjuvant to opioid for the treatment of pain in adults with acute and chronic pain, have been shown, in some instances, to improve pain intensity and to decrease opioid intake. However, less is known about the role of ketamine in pain management in children, adolescents, and young adults.
We examined the effects of subanesthetic ketamine on pain intensity and opioid intake in children, adolescents, and young adults with acute and chronic pain syndromes treated in an inpatient setting.
This is a longitudinal cohort study of patients treated with subanesthetic ketamine infusions in regular patient care units in a tertiary pediatric hospital. Primary outcomes included changes in pain scores and morphine-equivalent intake.
The study cohort included 230 different patients who during 360 separate hospital admissions received subanesthetic ketamine infusions for pain management. Overall, ketamine infusions were associated with significant reductions in mean pain scores from baseline (mean pain scores 6.64 [95% CI: 6.38-6.90]) to those recorded on the day after discontinuation of ketamine (mean pain scores 4.38 [95% CI: 4.06-4.69]), p<0.001. Importantly, the effect of ketamine on pain scores varied according to clinical diagnosis (p=0.011), infusion duration (p=0.004), and pain location (p=0.004). Interestingly, greater reductions in pain scores were observed in patients with cancer pain and patients with pain associated with pancreatitis and Crohn’s disease. There were no records of psychotomimetic side effects requiring therapy.
These data suggest that administration of subanesthetic ketamine for pain management is feasible and safe in regular inpatient care units and may benefit children, adolescents, and young adults with acute and chronic pain. This study is informative and can be helpful in determining sample and effect sizes when planning clinical trials to determine the role of subanesthetic ketamine infusions for pain management in pediatric patients.
PMCID: PMC5388303 Free PMC Article
Conflict of interest statement
Disclosure This article was prepared while Zenaide MN Quezado, MD was employed at the Children’s National Health System. The opinions expressed in this article are the author’s own and do not reflect the view of the National Institutes of Health, the Department of Health and Human Services, or the United States government. The authors report no other conflicts of interest in this work.
- Cochrane Database Syst Rev. 2017 Apr 20;4:CD002202. doi: 10.1002/14651858.CD002202.pub2.
Sickle cell disease (SCD) is one of the most common inherited diseases worldwide. It is associated with lifelong morbidity and a reduced life expectancy. Hydroxyurea (hydroxycarbamide), an oral chemotherapeutic drug, ameliorates some of the clinical problems of SCD, in particular that of pain, by raising fetal haemoglobin. This is an update of a previously published Cochrane Review.
To assess the effects of hydroxyurea therapy in people with SCD (all genotypes), of any age, regardless of setting.
We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Register, comprising of references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We also searched online trial registries.Date of the most recent search: 16 January 2017.
Randomised and quasi-randomised controlled trials, of one month or longer, comparing hydroxyurea with placebo, standard therapy or other interventions for people with SCD.
DATA COLLECTION AND ANALYSIS:
Authors independently assessed studies for inclusion, carried out data extraction and assessed the risk of bias.
Seventeen studies were identified in the searches; eight randomised controlled trials were included, recruiting 899 adults and children with SCD (haemoglobin SS (HbSS), haemoglobin SC (HbSC) or haemoglobin Sβºthalassaemia (HbSβºthal) genotypes). Studies lasted from six to 30 months.Four studies (577 adults and children with HbSS or HbSβºthal) compared hydroxyurea to placebo; three recruited individuals with only severe disease and one recruited individuals with all disease severities. There were statistically significant improvements in terms of pain alteration (using measures such as pain crisis frequency, duration, intensity, hospital admissions and opoid use), measures of fetal haemoglobin and neutrophil counts and fewer occurrences of acute chest syndrome and blood transfusions in the hydroxyurea groups. There were no consistent statistically significant differences in terms of quality of life and adverse events (including serious or life-threatening events). Seven deaths occurred during the studies, but the rates by treatment group were not statistically significantly different.Two studies (254 children with HbSS or HbSβºthal also with risk of primary or secondary stroke) compared hydroxyurea and phlebotomy to transfusion and chelation; there were statistically significant improvements in terms of measures of fetal haemoglobin and neutrophil counts, but more occurrences of acute chest syndrome and infections in the hydroxyurea and phlebotomy group. There were no consistent statistically significant differences in terms of pain alteration and adverse events (including serious or life-threatening events). Two deaths occurred during the studies (one in a the hydroxyurea treatment arm and one in the control arm), but the rates by treatment group were not statistically significantly different. In the primary prevention study, no strokes occurred in either treatment group but in the secondary prevention study, seven strokes occurred in the hydroxyurea and phlebotomy group (none in the transfusion and chelation group) and the study was terminated early.The quality of the evidence for the above two comparisons was judged as moderate to low as the studies contributing to these comparisons were mostly large and well designed (and at low risk of bias); however evidence was limited and imprecise for some outcomes such as quality of life, deaths during the studies and adverse events and results are applicable only to individuals with HbSS and HbSβºthal genotypes.Of the remaining two studies, one (22 children with HbSS or HbSβºthal also at risk of stoke) compared hydroxyurea to observation; there were statistically significant improvements in terms of measures of fetal haemoglobin and neutrophil counts but no statistically significant differences in terms of adverse events (including serious or life-threatening events).The final study (44 adults and children with HbSC) compared treatment regimens with and without hydroxyurea – there was statistically significant improvement in terms of measures of fetal haemoglobin, but no statistically significant differences in terms of adverse events (including serious or life-threatening events). No participants died in either of these studies and other outcomes relevant to the review were not reported.The quality of the evidence for the above two comparisons was judged to be very low due to the limited number of participants, the lack of statistical power (as both studies were terminated early with approximately only 20% of their target sample size recruited) and the lack of applicability to all age groups and genotypes.
There is evidence to suggest that hydroxyurea is effective in decreasing the frequency of pain episodes and other acute complications in adults and children with sickle cell anaemia of HbSS or HbSβºthal genotypes and in preventing life-threatening neurological events in those with sickle cell anaemia at risk of primary stroke by maintaining transcranial doppler velocities. However, there is still insufficient evidence on the long-term benefits of hydroxyurea, particularly in preventing chronic complications of SCD, recommending a standard dose or dose escalation to maximum tolerated dose. There is also insufficient evidence about the long-term risks of hydroxyurea, including its effects on fertility and reproduction. Evidence is also limited on the effects of hydroxyurea on individuals with HbSC genotype. Future studies should be designed to address such uncertainties.
- N Engl J Med. 2017 Apr 20;376(16):1561-1573. doi: 10.1056/NEJMra1510865.
- Cochrane Database Syst Rev. 2017 Apr 14;4:CD011358. doi: 10.1002/14651858.CD011358.pub2.
Sickle cell disease is an autosomal recessive inherited haemoglobinopathy which causes painful vaso-occlusive crises due to sickle red blood cell dehydration. Vaso-occlusive crises are common painful events responsible for a variety of clinical complications; overall mortality is increased and life expectancy decreased compared to the general population. Experimental studies suggest that intravenous magnesium has proven to be well-tolerated in individuals hospitalised for the immediate relief of acute (sudden onset) painful crisis and has the potential to decrease the length of hospital stay. Some in vitro studies and open studies of long-term oral magnesium showed promising effect on pain relief but failed to show its efficacy. The studies show that oral magnesium therapy may prevent sickle red blood cell dehydration and prevent recurrent painful episodes. There is a need to access evidence for the impact of oral and intravenous magnesium effect on frequency of pain, length of hospital stay and quality of life.
To evaluate the effects of short-term intravenous magnesium on the length of hospital stay and quality of life in children and adults with sickle cell disease. To determine the effects of long-term oral magnesium therapy on the frequency of painful crises and the quality of life in children and adults with sickle cell disease.
We searched the Cochrane Haemoglobinopathies Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books.Date of last search of the Cochrane Cystic Fibrosis and Genetic Disorders Group’s Haemoglobinopathies Trials Register: 01 December 2016.Date of last search of other resources (clinical trials registries): 29 March 2017.
We searched for published and unpublished randomized controlled studies of oral or intravenous magnesium compared to placebo or no magnesium.
DATA COLLECTION AND ANALYSIS:
Authors independently assessed the study quality and extracted the data using standard Cochrane methodologies.
We included five randomized placebo-controlled studies with a total of 386 participants (aged three to 53 years). Two shorter parallel studies (n = 306) compared intravenous magnesium sulphate to placebo (normal saline) for admission to hospital due to a vaso-occlusive crisis, for which we were able to analyse data. The quality of evidence was moderate for studies presenting this comparison mainly due to limitations due to risk of bias and imprecision. Two of the three longer-term studies comparing oral magnesium pidolate to placebo had a cross-over design. The third was a parallel factorial study which compared hydroxyurea and oral magnesium to each other and to placebo over a longer period of time; we only present the comparison of oral magnesium to placebo from this study. The quality of evidence was very low with uncertainty of the estimation.The eight-hourly dose levels in the two studies of intravenous magnesium were different; one used 100 mg/kg while the second used 40 mg/kg. Only one of these studies (n = 104) reported the mean daily pain score while hospitalised (a non-significant difference between groups, moderate quality evidence). The second study (n = 202) reported a number of child- and parent-reported quality of life scores. None of the scores showed any difference between treatment groups (low quality evidence). Data from one study (n = 106) showed no difference in length of stay in hospital between groups (low quality evidence). Both studies reported on adverse events, but not defined by severity as we had planned. One study showed significantly more participants receiving intravenous magnesium experienced warmth at infusion site compared to placebo; there were no differences between groups for other adverse events (low quality evidence).Three studies (n = 80) compared oral magnesium pidolate to placebo. None of them reported data which we were able to analyse. One study (n = 24) reported on the number of painful days and stated there was no difference between two groups (low quality evidence). None of the studies reported on quality of life or length of hospital stay. Two studies (n = 68) reported there were no differences in levels of magnesium in either plasma or red blood cells (moderate quality evidence). Two studies (n = 56) reported adverse events. One reported episodes of mild diarrhoea and headache, all of which resolved without stopping treatment. The second study reported adverse events as gastrointestinal disorders, headache or migraine, upper respiratory infections and rash; which were all evenly distributed across treatment groups (moderate quality evidence).
Moderate to low quality evidence showed neither intravenous magnesium and oral magnesium therapy has an effect on reducing painful crisis, length of hospital stay and changing quality of life in treating sickle cell disease. Therefore, no definitive conclusions can be made regarding its clinical benefit. Further randomized controlled studies, perhaps multicentre, are necessary to establish whether intravenous and oral magnesium therapies have any effect on improving the health of people with sickle cell disease.
Sickle Cell Conferences and Events
Pediatric Sickle Cell Mini Symposium: The School-Aged Child
Saturday, September 9, 2017 7:45 a.m. to 3:15 p.m. Atlanta,GA
Who Should Attend:The conference will benefit pediatricians, family practice physicians, advanced practice providers (NP’s, PA’s), nurses, fellows, and residents. Other healthcare professionals involved in the care of pediatric patients with sickle cell disease mayfind the information useful and are welcome to attend.
The purpose of this symposium is to update pediatricians and family practitioners
on the most current research and clinical guidelines related to pediatric sickle cell
disease, particularly in the school-aged child, and to discuss key considerations
when caring for these patients.
Location & Accommodations Emory Health Sciences Research Building
1760 Haygood Drive NE Atlanta, Georgia 30322
For more information, contact email@example.com.
SCDAA is pleased to announce the 45th Annual National Convention on Sickle Cell Disease.
With over 447 researchers, physicians, nurses, socials workers, individuals living with SCD & SCT and more we are excited to reunite with you again on October 23-28, 2017!
The SCDAA Annual Convention is a four-day conference designed to address the multi-factorial aspects of Sickle Cell Disease. This year the event will be held in Atlanta, Georgia, a city near and dear to the sickle cell community! https://www.sicklecelldisease.org/2017/03/07/45th-annual-national-convention/
SCDAA Announces National Abstract Competition for the 45th Annual Convention
Sickle Cell Disease Association of America, Inc.(SCDAA) seeks to highlight the work of Researchers, Community-based Member Organizations, Physicians, Nurses, Social Workers and others working on behalf of people with sickle cell disease and their families. Individuals or organizations interested in presenting reports on work completed or in progress should submit an abstract using the link below. All approved abstracts will be published in the final program to be distributed to registered conference attendees. During peer-review, abstracts judged to be the best in their categories will be selected as national finalists.
Abstract Categories Include:
- Community Based Research
- Clinical Research
- Public Health, Policy, and Psychosocial Research
- Basic Science and Translational Research
To be eligible, abstracts must meet guidelines and be submitted by June 15, 2017 (there will be no deadline extensions). Abstracts will be reviewed and ranked by the national abstract review committee. Abstract finalists will be judged during oral presentation at convention and the “Best Abstract” in each category will be announced at the conclusion of the 45th Annual National SCDAA Convention. Special awards for the best student and trainee abstracts will also be given. We look forward to seeing you in October!
*Upon submission you will also be prompted to submit a Disclosure form.
Click here to submit your abstract today!
Click here to download and complete the Disclosure form.
The 11th Sickle Cell in Focus Conference
26-27 October 2017 Kingston, Jamaica
We are pleased to announce that Sickle Cell in Focus (SCiF) will be held for the first time in Kingston, Jamaica on October 26-27, 2017. This year, SCiF will be co-hosted by the National Heart, Lung, and Blood Institute and the University of West Indies, Jamaica. SCiF is a two-day, intensive, educational update on sickle cell disease. This year’s conference will focus on the latest clinical trials, the science and mechanisms for new therapeutic targets, and curative therapies. This two-day intensive educational conferences includes both clinical and scientific lectures, aimed at clinicians, academics, and other healthcare professionals involved in sickle cell disease around the world. Contact Rusinel Amarante| firstname.lastname@example.org |