FDA Advisory Committee Gives Thumbs Up for L-glutamine for Sickle Cell Disease
The U.S. Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee voted 10-to-3 that the overall Benefit-Risk profile of Endari (L-glutamine) for treating patients with sickle cell disease is favorable.
The FDA is set to make its final decision on the drug by July 7, 2017.
If approved, Endari would be the first FDA-approved treatment for pediatric patients with sickle cell disease, and the first new treatment for adult patients in almost 2 decades.
Efficacy and Safety
The Committee focused on the phase 3 clinical trial in which patients with sickle cell or sickle ß0 -thalassemia aged 5 years and older were randomized (2:1) to receive oral L-glutamine 0.3 mg/kg/day or placebo for 48 weeks.
The primary outcome measure was the frequency of sickle cell crises and, after 48 weeks, the median number of sickle cell crisis events was 3 in the L-glutamine arm and 4 in the placebo arm (P = .0052).
The FDA documented that while the difference was statistically different, the efficacy data was complicated by differences in discontinuation rates observed during the course of the study. While that was a concern, the FDA documented that their own exploration of the data favored L-glutamine over placebo in reducing the rates of crises.
The safety review revealed that the most common adverse reactions (incidence ≥ 10% and greater than placebo) were constipation, nausea, headache, cough, pain in extremity, back pain, chest pain, and abdominal pain.
World Sickle Cell Awareness Day Celebration – June 19th
New York Times – Patient Voices: Sickle Cell Anemia
Here, six men and women speak about the impact sickle cell anemia has had on their lives and families.
Mixing Music and Medicine: Meet Grammy-Nominated Producer Nana Kwabena
Grammy-nominated songwriter and music producer, Nana Kwabena has helped create hits for many notable artists including John Legend, Rick Ross and fellow Wondaland label mates Janelle Monae and Jidenna. However, his musical genius and path to the entertainment industry began in one of the most unlikely places—a hospital.
The 31-year-old was diagnosed with sickle cell disease at a young age and says that he spent so much of his childhood in and out of medical facilities; it often felt like he was raised in the hospital.
As a teenager, complications from the disease often meant that Kwabena was in the hospital for up to two months at a time. The musician says he remembers feeling like the beeps of the machines in his room were creating ‘a soundtrack of death’ and he had to do something to change it. He taught himself how to use the music production program Fruity Loops and started to create his own songs. “I had to create my own music to drown out the noise of those beeps and blips,” he said.
After that experience Kwabena says he knew he wanted to pursue music and at the same time shed light on sickle cell disease.
Articles in the Medical Literature
- Biol Blood Marrow Transplant. 2017 May 31. pii: S1083-8791(17)30493-7. doi: 10.1016/j.bbmt.2017.05.027. [Epub ahead of print]
Unrelated Umbilical Cord Blood Transplantation for Sickle Cell Disease Following Reduced Intensity Conditioning: Results of a Phase I Trial.
Abraham A1, Cluster A2, Jacobsohn D1, Delgado D3, Hulbert ML2, Kukadiya D1, Murray L2, Shenoy S4.
Hematopoietic stem cell transplantation from HLA-matched sibling donors results in disease free survival of >90% in patients with sickle cell disease (SCD) but only about 18% have suitable donors. Unrelated cord blood transplantation (UCBT) is one way to expand donor options for patients with severe SCD but historically has been associated with high graft rejection rates (50-62%). We hypothesized that the addition of thiotepa to a previously tested reduced intensity conditioning (RIC) regimen would support engraftment after UCBT in SCD patients. Nine children (3-10 years) with cerebrovascular complications of SCD underwent 5-6/6 HLA-matched (A, B and DRB1 loci) UCBT after conditioning with hydroxyurea, alemtuzumab, fludarabine, thiotepa and melphalan. A calcineurin inhibitor and mycophenolate mofetil were used for graft-versus-host-disease (GVHD) prophylaxis. With median follow up of 2.1 (range 1-4.2) years, seven patients had sustained donor cell engraftment and are free of SCD, two had autologous recovery. Acute GVHD (grade 2-4), mild, and moderate chronic GVHD developed in three, two, and one patient respectively. Of five patients >2 years post-UCBT, four have discontinued systemic immunosuppression. Seven patients had viral infections (CMV, EBV, RSV or Adenovirus) and recovered. The 1-year overall and disease free survival rates were 100% and 78% respectively. Thus, this RIC regimen was able to achieve donor engraftment in the majority. Future efforts will focus on further reducing acute GVHD and viral infection rates.
Copyright © 2017. Published by Elsevier Inc.
- J Genet Couns. 2017 Jun 3. doi: 10.1007/s10897-017-0107-6. [Epub ahead of print]
Perspectives and Practices of Athletic Trainers and Team Physicians Implementing the 2010 NCAA Sickle Cell Trait Screening Policy.
McDonald MA1, Creary MS2, Powell J1, Daley LA1, Baker C3, Royal CD4,5.
Sickle cell trait (SCT) is usually benign. However, there are some conditions that may lead to SCT-related problems and put athletes with the trait at particular risk. In 2010 the National Collegiate Athletic Association (NCAA) issued a policy that required all Division I (DI) student-athletes to confirm their SCT status or sign a liability waiver to opt out of testing. Athletic trainers and team physicians play key roles in the policy implementation and we examined their perceptions and practices. Between December 2013 and March 2014 we interviewed 13 head athletic trainers and team physicians at NCAA Division I colleges and universities in North Carolina. We used an interview guide with open-ended questions covering knowledge of SCT, historical screening and education practices, current implementation, and policy benefits and challenges. Participants were knowledgeable about SCT and thought the policy was beneficial in providing SCT health information to and for student-athletes. Schools varied in provision of genetic counseling, offering the waiver, SCT tests administered, and other aspects. Challenges included: insufficient guidance from the NCAA; financial considerations; and misunderstanding of the relationships of race and ancestry to SCT risk. Athletic staff found the policy valuable, but felt it needs clarity and standardization.
- Health Qual Life Outcomes. 2017 Jun 2;15(1):117. doi: 10.1186/s12955-017-0661-5.
Sensitivity of alternative measures of functioning and wellbeing for adults with sickle cell disease: comparison of PROMIS® to ASCQ-Me℠.
Keller S1, Yang M2, Treadwell MJ3, Hassell KL4.
Sickle Cell Disease (SCD) causes profound suffering and decrements in daily functioning. Demand is growing for valid and reliable measures to systematically document these effects, particularly in adults. The Adult Sickle Cell Quality of Life Measurement System, ASCQ-Me℠, was developed for this purpose. ASCQ-Me℠ is one of four measurement systems housed within the Person-Centered Assessment Resource (PCAR), funded by the National Institutes of Health, to support clinical research. To help users select the best of these measures for adults with SCD, we evaluated and compared two PCAR systems: one designed to be “universally applicable” (the Patient-Reported Outcome Measurement Information System, PROMIS®) and one designed specifically for SCD (ASCQ-Me℠).
Respondents to PROMIS and ASCQ-Me questions were 490 adults with SCD from seven geographically-disbursed clinics within the US. Data were collected for six ASCQ-Me measures (Emotional Impact, Sleep Impact, Social Impact, Stiffness Impact, Pain Impact, SCD Pain Episode Frequency and Severity) and ten PROMIS measures (Pain Impact, Pain Behavior, Physical Functioning, Anxiety, Depression, Fatigue, Satisfaction with Discretionary Social Activities, Satisfaction with Social Roles, Sleep Disturbance, and Sleep-Related Impairment). Statistical analyses, including analysis of variance and multiple linear regression, were conducted to determine the sensitivity of measures to SCD severity. SCD severity was assessed via a checklist of associated treatments and conditions.
For those with the most severe SCD, PROMIS scores showed worse health compared to the general population for nine of ten health domains: the magnitude of the difference ranged 0.5 to 1.1 standard deviation units. The PROMIS domains most severely affected were Physical Functioning and Pain (Impact and Behavior). Significant differences by tertile of the SCD-MHC were shown for most PROMIS short forms and all ASCQ-Me short and fixed forms. In most models, ASCQ-Me measures explained statistically significant unique variance in SCD-MHC scores complementary to that explained by corresponding PROMIS measures.
Study results supported the validity of both PROMIS and ASCQ-Me measures for use in adults with SCD. Compared to comparable PROMIS scores, most ASCQ-Me scores were better predictors of SCD disease severity, as measured by a medical history checklist. The clinical implications of these results require further investigation.
- Pediatr Blood Cancer. 2017 May 19. doi: 10.1002/pbc.26635. [Epub ahead of print]
Increased complications of chronic erythrocytapheresis compared with manual exchange transfusions in children and adolescents with sickle cell disease.
Woods D1, Hayashi RJ1, Binkley MM2, Sparks GW3, Hulbert ML1.
Children and adolescents with sickle cell disease (SCD) are at high risk of strokes and are frequently treated with red blood cell (RBC) transfusions. The goal is to suppress hemoglobin (Hb) S while minimizing transfusion-induced iron overload. RBCs may be given via simple transfusion, manual exchange transfusion (MET), or erythrocytapheresis (aRBCX). Chronic transfusion practices vary among institutions.
This single-institution, retrospective cohort study compares Hb S control and therapy complication rates between MET and aRBCX in a cohort of children and adolescents with SCD and stroke during a 5-year period from 2008 through 2012. Duration and mode of transfusion therapy, achievement of Hb S suppression goal, iron burden by ferritin levels, and catheter complications were evaluated.
Thirty-seven children were included in analysis. The prevalence of catheter complications was 75% in aRBCX recipients compared with 0% in MET recipients (P < 0.001). There was no significant difference between modalities in achieving Hb S suppression or ferritin goals, but those receiving aRBCX had a greater likelihood of discontinuing chelation therapy. Among aRBCX recipients, adherence to >90% of transfusion appointments was associated with achieving Hb S suppression goals.
aRBCX may have increased complication risks compared with MET for chronic transfusion therapy in SCD. Risks and benefits of aRBCX and MET should be considered when selecting a chronic transfusion modality. Transfusion therapy modalities should be compared in prospective studies for stroke prevention in children with SCD.
© 2017 Wiley Periodicals, Inc.
- J Pediatr Hematol Oncol. 2017 May 22. doi: 10.1097/MPH.0000000000000847. [Epub ahead of print]
Attention Deficit Hyperactivity Disorder in Children With Sickle Cell Disease Referred for an Evaluation.
Acquazzino MA1, Miller M, Myrvik M, Newby R, Scott JP.
Neuropsychological deficits, including difficulties with attention, are well described in children with sickle cell disease (SCD). Very little is known about attention deficit hyperactivity disorder (ADHD) in children with SCD. The objective of this study was to determine the proportion of ADHD in children with SCD referred for neuropsychological evaluation. This prospective, cross-sectional study included patients (age, 4 to 18 y) with SCD and completion of a neuropsychological evaluation between December 2013 and March 2016. Patients were referred for neuropsychological evaluation because of concern regarding school performance, development, and/or behavior. The diagnosis of ADHD was made by a neuropsychologist on the basis of the diagnostic criteria in the Diagnostic Statistical Manual-Fourth or Fifth Editions. ADHD medication usage rate was obtained by medical record review. Of the 89 patients with SCD referred for neuropsychological evaluation, 25% (95% confidence interval, 16%-35%) met diagnostic criteria for ADHD. Only 21% of the patients with SCD and ADHD were prescribed an ADHD medication. Our study supports routine ADHD screening in children with SCD who have poor school performance or behavioral concerns. Despite the benefits of pharmacologic treatment, the majority of patients with SCD and ADHD did not receive a medication for management of their ADHD.
- J Pediatr Hematol Oncol. 2017 May 22. doi: 10.1097/MPH.0000000000000858. [Epub ahead of print]
Invasive Pneumococcal Disease in Patients With Sickle Cell Disease.
Navalkele P1, Özgönenel B, McGrath E, Lephart P, Sarnaik S.
Patients with sickle cell disease (SCD) are at risk of fatal sepsis with encapsulated bacteria, such as Streptococcus pneumoniae, because of the inherent autosplenectomy that occurs in SCD. This risk is thwarted with oral penicillin prophylaxis during the first 5 years of life, and with stringent vaccination against S. pneumoniae alongside routine childhood immunization. But compared with the general African American pediatric population, the rate of invasive pneumococcal disease (IPD) in patients with SCD still remains high, resulting in hospitalization and fatality.
Patients with SCD who developed IPD from 2004 up to 2013 were identified using microbiology records. Descriptive analysis of presence of risk factors for IPD, type of SCD, pneumococcal vaccination and prophylaxis status, clinical presentation, microbiological data, and the outcome of IPD was performed.
Eight patients with SCD developed IPD (7 bacteremia and 1 respiratory tract infection). Three of the 8 isolates underwent serotype analysis (15 C in 2 and 15A in 1), none covered with the current vaccination program. One patient had fatal outcome (15A).
Breakthrough cases of IPD may involve nonvaccine isolates, and seem to occur after 5 years of age when oral penicillin prophylaxis has been terminated.
- Appl Clin Inform. 2017 May 24;8(2):541-559. doi: 10.4338/ACI-2016-12-RA-0203.
Improving Medication Adherence with Two-way Short Message Service Reminders in Sickle Cell Disease and Asthma. A feasibility randomized controlled trial.
Pernell BM, DeBaun MR, Becker K, Rodeghier M, Bryant V, Cronin RM1.
Sickle cell disease (SCD) is a childhood and adult disease that primarily affects African Americans, characterized by life threatening sequelae mitigated by medications. One-way and two-way short message service (SMS) medication reminders have differing efficacy in chronic diseases. There is limited literature about SMS medication reminders in SCD.
The goal of this study was to test the feasibility, defined by recruitment/acceptance, retention/attrition, and technology utilization, of two-way SMS medication reminders in individuals with SCD with and without asthma.
MATERIALS AND METHODS:
Participants were randomly allocated to standard care or reminders. Two-way SMS reminders were automated using Research Electronic Data Capture (REDCap) for hydroxyurea, fluticasone, budesonide and montelukast. Adherence was measured using the Morisky Medication Adherence Scale-8 (MMAS-8). Asthma control was assessed using the Childhood and Adult-Asthma Control Tests (ACT). Participants were enrolled 28 to 60 days with a common termination date.
The recruitment rate was 95% (47/49) and 82.9% completed the study. Among the 47 study participants enrolled, 51.1% were male, 61.7% were adults, median age was 20 (range: 3 to 59), and 98% were African Americans. Of the 26 participants receiving messages, 20% responded on over 95% of the days and usage varied with an average response rate of 33%, ranging from 21% to 46%. Medication adherence scores improved significantly in the intervention group (3.42 before, 5.46 after; p=0.002), but not in the control group (3.90 before, 4.75 after; p=0.080). Childhood-ACT scores improved in the intervention group (19.20 before, 24.25 after). Adult-ACT scores within the intervention arm were unchanged (21.0 before, 22.0 after. ACT scores did not improve significantly.
This study demonstrated the feasibility for two-way SMS medication reminders to improve medication adherence in a high-risk population where daily medication adherence is critical to health outcomes and quality of life.
- J Emerg Nurs. 2017 May 17. pii: S0099-1767(16)30388-9. doi: 10.1016/j.jen.2017.04.009. [Epub ahead of print]
Identifying Social-Behavioral Health Needs of Adults with Sickle Cell Disease in the Emergency Department.
Smith SK1, Johnston J1, Rutherford C1, Hollowell R1, Tanabe P2.
Sickle cell disease (SCD) is a complex illness with many social-behavioral co-morbidities. The aim of this project was to describe unmet social-behavioral health needs for adults with SCD who presented to the emergency department for treatment of vaso-occlusive episodes (VOEs).
A descriptive study using 1:1 interviews during an ED visit for a VOE was conducted; a brief social behavioral health screening interview guide was used. A convenience sample of adults with SCD treated in the emergency department for a VOE were eligible for inclusion.
We conducted 147 interviews over 14 months. Patients reported transportation and/or scheduling difficulties with clinic appointments in one third of the interviews. Four major themes emerged: clinic appointment barriers, medication barriers, other care barriers, and social-behavioral issues. A majority of patients (53%) reported being brought to the emergency department by a family member at their current visit. Patients cited having insurance coverage issues in more than one quarter (27%) of the interviews. Difficulties in obtaining prescriptions were cited as a result of a financial copay (17%), transportation (11%), and pharmacy (9%) issues. Almost one third of patients (29%) reported feeling depressed, and 20% reported feeling anxious.
Many patients with SCD who are treated in the emergency department have social or behavioral health risk factors. Emergency departments have an opportunity to screen and refer patients for follow-up. Future research should investigate referral outcomes and their effect on ED and hospital use.
Copyright © 2017 Elsevier Inc. All rights reserved.
- Cochrane Database Syst Rev. 2017 May 13;5:CD012389. doi: 10.1002/14651858.CD012389.pub2.
Interventions for preventing silent cerebral infarcts in people with sickle cell disease.
Estcourt LJ1, Fortin PM2, Hopewell S3, Trivella M4, Doree C2, Abboud MR5.
Sickle cell disease (SCD) is one of the commonest severe monogenic disorders in the world, due to the inheritance of two abnormal haemoglobin (beta globin) genes. SCD can cause severe pain, significant end-organ damage, pulmonary complications, and premature death. Silent cerebral infarcts are the commonest neurological complication in children and probably adults with SCD. Silent cerebral infarcts also affect academic performance, increase cognitive deficits and may lower intelligence quotient.
To assess the effectiveness of interventions to reduce or prevent silent cerebral infarcts in people with SCD.
We searched for relevant trials in the Cochrane Library, MEDLINE (from 1946), Embase (from 1974), the Transfusion Evidence Library (from 1980), and ongoing trial databases; all searches current to 19 September 2016. We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register: 06 October 2016.
Randomised controlled trials comparing interventions to prevent silent cerebral infarcts in people with SCD. There were no restrictions by outcomes examined, language or publication status.
DATA COLLECTION AND ANALYSIS:
We used standard Cochrane methodological procedures.
We included five trials (660 children or adolescents) published between 1998 and 2016. Four of the five trials were terminated early. The vast majority of participants had the haemoglobin (Hb)SS form of SCD. One trial focused on preventing silent cerebral infarcts or stroke; three trials were for primary stroke prevention and one trial dealt with secondary stroke prevention.Three trials compared the use of regular long-term red blood cell transfusions to standard care. Two of these trials included children with no previous long-term transfusions: one in children with normal transcranial doppler (TCD) velocities; and one in children with abnormal TCD velocities. The third trial included children and adolescents on long-term transfusion.Two trials compared the drug hydroxyurea and phlebotomy to long-term transfusions and iron chelation therapy: one in primary prevention (children), and one in secondary prevention (children and adolescents).The quality of the evidence was moderate to very low across different outcomes according to GRADE methodology. This was due to trials being at high risk of bias because they were unblinded; indirectness (available evidence was only for children with HbSS); and imprecise outcome estimates. Long-term red blood cell transfusions versus standard care Children with no previous long-term transfusions and higher risk of stroke (abnormal TCD velocities or previous history of silent cerebral infarcts) Long-term red blood cell transfusions may reduce the incidence of silent cerebral infarcts in children with abnormal TCD velocities, risk ratio (RR) 0.11 (95% confidence interval (CI) 0.02 to 0.86) (one trial, 124 participants, low-quality evidence); but make little or no difference to the incidence of silent cerebral infarcts in children with previous silent cerebral infarcts on magnetic resonance imaging and normal or conditional TCDs, RR 0.70 (95% CI 0.23 to 2.13) (one trial, 196 participants, low-quality evidence).No deaths were reported in either trial.Long-term red blood cell transfusions may reduce the incidence of: acute chest syndrome, RR 0.24 (95% CI 0.12 to 0.49) (two trials, 326 participants, low-quality evidence); and painful crisis, RR 0.63 (95% CI 0.42 to 0.95) (two trials, 326 participants, low-quality evidence); and probably reduces the incidence of clinical stroke, RR 0.12 (95% CI 0.03 to 0.49) (two trials, 326 participants, moderate-quality evidence).Long-term red blood cell transfusions may improve quality of life in children with previous silent cerebral infarcts (difference estimate -0.54; 95% confidence interval -0.92 to -0.17; one trial; 166 participants), but may have no effect on cognitive function (least squares means: 1.7, 95% CI -1.1 to 4.4) (one trial, 166 participants, low-quality evidence). Transfusions continued versus transfusions halted: children and adolescents with normalised TCD velocities (79 participants; one trial)Continuing red blood cell transfusions may reduce the incidence of silent cerebral infarcts, RR 0.29 (95% CI 0.09 to 0.97 (low-quality evidence).We are very uncertain whether continuing red blood cell transfusions has any effect on all-cause mortality, Peto odds ratio (OR) 8.00 (95% CI 0.16 to 404.12); or clinical stroke, RR 0.22 (95% CI 0.01 to 4.35) (very low-quality evidence).The trial did not report: comparative numbers for SCD-related adverse events; quality of life; or cognitive function. Hydroxyurea and phlebotomy versus transfusions and chelation Primary prevention, children (121 participants; one trial)We are very uncertain whether switching to hydroxyurea and phlebotomy has any effect on: silent cerebral infarcts (no infarcts); all-cause mortality (no deaths); risk of stroke (no strokes); or SCD-related complications, RR 1.52 (95% CI 0.58 to 4.02) (very low-quality evidence). Secondary prevention, children and adolescents with a history of stroke (133 participants; one trial)We are very uncertain whether switching to hydroxyurea and phlebotomy has any effect on: silent cerebral infarcts, Peto OR 7.28 (95% CI 0.14 to 366.91); all-cause mortality, Peto OR 1.02 (95%CI 0.06 to 16.41); or clinical stroke, RR 14.78 (95% CI 0.86 to 253.66) (very low-quality evidence).Switching to hydroxyurea and phlebotomy may increase the risk of SCD-related complications, RR 3.10 (95% CI 1.42 to 6.75) (low-quality evidence).Neither trial reported on quality of life or cognitive function.
We identified no trials for preventing silent cerebral infarcts in adults, or in children who do not have HbSS SCD.Long-term red blood cell transfusions may reduce the incidence of silent cerebral infarcts in children with abnormal TCD velocities, but may have little or no effect on children with normal TCD velocities. In children who are at higher risk of stroke and have not had previous long-term transfusions, long-term red blood cell transfusions probably reduce the risk of stroke, and other SCD-related complications (acute chest syndrome and painful crises).In children and adolescents at high risk of stroke whose TCD velocities have normalised, continuing red blood cell transfusions may reduce the risk of silent cerebral infarcts. No treatment duration threshold has been established for stopping transfusions.Switching to hydroxyurea with phlebotomy may increase the risk of silent cerebral infarcts and SCD-related serious adverse events in secondary stroke prevention.All other evidence in this review is of very low-quality.
- Redox Biol. 2017 May 10;12:1026-1039. doi: 10.1016/j.redox.2017.05.006. [Epub ahead of print]
Potential therapeutic action of nitrite in sickle cell disease.
Wajih N1, Basu S1, Jailwala A2, Kim HW2, Ostrowski D2, Perlegas A2, Bolden CA2, Buechler NL3, Gladwin MT4, Caudell DL5, Rahbar E6, Alexander-Miller MA7, Vachharajani V8, Kim-Shapiro DB9.
Sickle cell disease is caused by a mutant form of hemoglobin that polymerizes under hypoxic conditions, increasing rigidity, fragility, calcium influx-mediated dehydration, and adhesivity of red blood cells. Increased red cell fragility results in hemolysis, which reduces nitric oxide (NO) bioavailability, and induces platelet activation and inflammation leading to adhesion of circulating blood cells. Nitric Oxide inhibits adhesion and platelet activation. Nitrite has emerged as an attractive therapeutic agent that targets delivery of NO activity to areas of hypoxia through bioactivation by deoxygenated red blood cell hemoglobin. In this study, we demonstrate anti-platelet activity of nitrite at doses achievable through dietary interventions with comparison to similar doses with other NO donating agents. Unlike other NO donating agents, nitrite activity is shown to be potentiated in the presence of red blood cells in hypoxic conditions. We also show that nitrite reduces calcium associated loss of phospholipid asymmetry that is associated with increased red cell adhesion, and that red cell deformability is also improved. We show that nitrite inhibits red cell adhesion in a microfluidic flow-channel assay after endothelial cell activation. In further investigations, we show that leukocyte and platelet adhesion is blunted in nitrite-fed wild type mice compared to control after either lipopolysaccharide- or hemolysis-induced inflammation. Moreover, we demonstrate that nitrite treatment results in a reduction in adhesion of circulating blood cells and reduced red blood cell hemolysis in humanized transgenic sickle cell mice subjected to local hypoxia. These data suggest that nitrite is an effective anti-platelet and anti-adhesion agent that is activated by red blood cells, with enhanced potency under physiological hypoxia and in venous blood that may be useful therapeutically.
Copyright © 2017 The Authors. Published by Elsevier B.V. All rights reserved.
PMCID: PMC5430577 Free PMC Article
Sickle Cell Conferences and Events
World Sickle Cell Awareness Day Celebration- June 19th
The Sickle Cell Awareness Group of Ontario is holding a World Sickle Cell Day celebration at the Tree of Life in front of the Toronto General Hospital on June 19th from 12PM- 1:30PM. The President/Executive Director of the Sickle Cell Disease Association of Canada, Mrs. Lanre Tunji-Ajayi will be joined by Senator Jane Cordy who introduced Bill S-211 in the Senate ( a bill to recognize June 19 in Canada as the National Sickle Cell Day). Event is free.
For more information – go to: www.sicklecellanemia.ca
Join Sickle Cell Partners of the Carolinas for the 4th annual conference, “Sickle Cell Disease…. Let’s Talk About It”
Saturday September 9, 2017 | 8 am to 2 pm | Charlotte, NC
Friendship Missionary Baptist Church Conference Center 3400 Beatties Ford Road, Charlotte NC 28216
This day conference will feature a myriad of topics designed to engage patients, families and the at-large community and to build broader awareness about the challenges of sickle cell disease and how patients and families may be able to get beyond those challenges. Keynote Luncheon Speaker: Howard University President, Wayne A.I. Frederick, M.D., MBA.
Friendship Missionary Baptist Church Conference Center Charlotte, North Carolina. Cost is $5 for participants.
For more information, visit our website www.sicklecellpartnersofthecarolinas.org and register for the event at http://scpocconference2017.eventbrite.com
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