Sickle Cell News | November 2015

Sickle Cell News for November 2015 Sickle cell disease cure in sights of UAB Stem Cell Institute - See more at: http://alabamanewscenter.com/2015/11/13/sickle-cell-disease-cure-in-sights-of-uab-stem-cell-institute/#sthash.UcB4ug9H.dpuf Chevron Boosts Its Support Of The Angola Sickle Cell Initiative https://www.polymerupdate.com/press-releases/press-release-details.aspx?id=10275 Chevron Corp. is continuing its support of the Angola Sickle Cell Initiative by committing an additional $5 million endowment over the course of five years. The oil and gas company, which initially contributed $4 million to support the program’s launch and pilot phase in 2011, made the official announcement of the additional funding in September at a signing ceremony in Luanda, Angola, where organization leaders and the nation’s officials joined together to celebrate the program’s success and outline future goals. “One of the things I try to emphasize is that health should be looked at from a global perspective. Health is no longer local or regional,” said Ali Moshiri, president of Chevron Africa and Latin America Exploration and Production. “Today we live in a globalized environment. We may still have political boundaries, but when it comes to health, that is a fungible issue. That means that a disease outbreak could spread beyond borders. Therefore, global health is an issue that we must all embrace.” The Angola Sickle Cell Initiative was born out of an initial meeting with Angola’s First Lady Ana Paula dos Santos and Moshiri at the African First Ladies Health Summit in Los Angeles in 2009. The meeting soon turned into a conversation about how Chevron one of the largest oil and gas producers in Angola could help tackle a serious health problem in her nation: sickle cell disease. Web article: Sickle cell trait and disease: raising awareness   Medical News Today http://www.medicalnewstoday.com/articles/302841.php New Book for Patients and Family members Living with Sickle Cell: The Inside Story by Judy Gray Johnson - http://www.knowledgepowerinc.com/Sickle_Cell.html Judy Gray Johnson has been living with Sickle Cell for more than 70 years. Other books by the author are Living with Sickle Cell Disease: the Struggle to Survive and Resilience: A Personal Story of Coping with the Ravages of Sickle Cell Disease gainst All Odds. Judy lives in Valencia, California and continues to advocate in the cause of finding real quality of life changes for those suffering with Sickle Cell. New  Web Resource from NICHQ National Institute For Children's Health Quality http://sicklecell.nichq.org/ Sickle cell in the Medical Literature 1. J Am Soc Nephrol. 2015 Nov 19. pii: ASN.2014111126. [Epub ahead of print] Six Months of Hydroxyurea Reduces Albuminuria in Patients with Sickle Cell Disease. Bartolucci P1, Habibi A2, Stehlé T3, Di Liberto G4, Rakotoson MG4, Gellen-Dautremer J2, Loric S5, Moutereau S5, Sahali D6, Wagner-Ballon O7, Remy P6, Lang P6, Grimbert P6, Audureau E8, Godeau B9, Galacteros F10, Audard V11. Abstract The earliest symptom of glomerular injury in patients with sickle cell disease (SCD) is microalbuminuria. The effect of hydroxyurea (HU) on urine albumin-to-creatinine ratio (ACR) is unclear and should be determined, because increasing numbers of patients with SCD take this drug to improve red blood cell function. In this cohort study of 58 SS-homozygous adults with SCD who initiated HU therapy, we evaluated ACR changes and relationships of these changes with demographic, clinical, and biologic parameters at HU initiation (baseline) and 6 months later (follow-up). Between baseline and follow-up, ACR declined significantly for the entire population (3.0-1.7 mg/mmol; P<0.01), but this was primarily driven by the ACR reduction in the microalbuminuria subgroup (8.1-2.3 mg/mmol; P=0.03; n=23). According to bivariate analyses on 39 patients who did not receive a blood transfusion during the study period, the baseline to follow-up ACR decline was strongly associated with decreases in levels of hemolysis markers, percentage of dense red blood cells, and systolic BP. Bivariate analysis also revealed a close association between the ACR decrease and high baseline levels of hemolysis markers and percentage of dense red blood cells. These results show that urine ACR decreased significantly after 6 months of HU and confirm a close relationship between ACR and hemolysis evolution in patients with SCD. Copyright © 2015 by the American Society of Nephrology. PMID: 26586692 [PubMed - as supplied by publisher] Similar articles 2. PLoS One. 2015 Nov 17;10(11):e0141706. doi: 10.1371/journal.pone.0141706. eCollection 2015. Hydroxyurea-Increased Fetal Hemoglobin Is Associated with Less Organ Damage and Longer Survival in Adults with Sickle Cell Anemia. Fitzhugh CD1,2, Hsieh MM1, Allen D2, Coles WA1, Seamon C2, Ring M3, Zhao X4, Minniti CP2, Rodgers GP1, Schechter AN5, Tisdale JF1, Taylor JG 6th2. Abstract BACKGROUND: Adults with sickle cell anemia (HbSS) are inconsistently treated with hydroxyurea. OBJECTIVES: We retrospectively evaluated the effects of elevating fetal hemoglobin with hydroxyurea on organ damage and survival in patients enrolled in our screening study between 2001 and 2010. METHODS: An electronic medical record facilitated development of a database for comparison of study parameters based on hydroxyurea exposure and dose. This study is registered with ClinicalTrials.gov, number NCT00011648. RESULTS: Three hundred eighty-three adults with homozygous sickle cell disease were analyzed with 59 deaths during study follow-up. Cox regression analysis revealed deceased subjects had more hepatic dysfunction (elevated alkaline phosphatase, Hazard Ratio = 1.005, 95% CI 1.003-1.006, p<0.0.0001), kidney dysfunction (elevated creatinine, Hazard Ratio = 1.13, 95% CI 1.00-1.27, p = 0.043), and cardiopulmonary dysfunction (elevated tricuspid jet velocity on echocardiogram, Hazard Ratio = 2.22, 1.23-4.02, p = 0.0082). Sixty-six percent of subjects were treated with hydroxyurea, although only 66% of those received a dose within the recommended therapeutic range. Hydroxyurea use was associated with improved survival (Hazard Ratio = 0.58, 95% CI 0.34-0.97, p = 0.040). This effect was most pronounced in those taking the recommended dose of 15-35 mg/kg/day (Hazard Ratio 0.36, 95% CI 0.17-0.73, p = 0.0050). Hydroxyurea use was not associated with changes in organ function over time. Further, subjects with higher fetal hemoglobin responses to hydroxyurea were more likely to survive (p = 0.0004). While alkaline phosphatase was lowest in patients with the best fetal hemoglobin response (95.4 versus 123.6, p = 0.0065 and 96.1 versus 113.6U/L, p = 0.041 at first and last visits, respectively), other markers of organ damage were not consistently improved over time in patients with the highest fetal hemoglobin levels. CONCLUSIONS: Our data suggest that adults should be treated with the maximum tolerated hydroxyurea dose, ideally before organ damage occurs. Prospective studies are indicated to validate these findings. Free Article PMID: 26576059 [PubMed - in process] Similar articles 3. Pediatr Blood Cancer. 2015 Nov 17. doi: 10.1002/pbc.25838. [Epub ahead of print] Prevalence of Developmental Delay and Contributing Factors Among Children With Sickle Cell Disease. Drazen CH1, Abel R1, Gabir M1, Farmer G2, King AA1,2,3. Abstract BACKGROUND: Children with sickle cell disease (SCD) are at higher risk for deficits in cognition compared to the general population, even at young ages. Disease severity has been co-assessed in earlier studies, but the home environment has not. The purpose of the current study was to investigate the development of young children with SCD and secondarily, the impact of environmental and family factors. METHODS: The current study is a baseline cross-sectional evaluation of a prospective, single-center cohort. Children with SCD between the ages of 1 and 42 months and their primary caregiver were included. Participants lived within 30 miles of the site and spoke English. Children underwent developmental evaluation using the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III). Home visits were completed and screened using the Home Observation for Measurement of the Environment (HOME). RESULTS: Over 3 years, 43 caregiver-child dyads consented and participated. Over 50% of children scored significantly below average on cognition and expressive language subscales. SCD severity was not associated with BSID-III scores. Socioeconomic status (SES) determined by the Diez-Roux method positively correlated (r = 0.401, P < 0.01) with the home environment. The HOME correlated (r = 0.360, P < 0.05) with the cognitive subscale on the BSID-III. CONCLUSIONS: Given the high prevalence of developmental delay in this population, identifying modifiable factors to maximize developmental progress is essential. The home environment would be a targeted method for intervention. Future research is needed to identify the benefits of home-based intervention for this population. © 2015 Wiley Periodicals, Inc. PMID: 26575319 [PubMed - as supplied by publisher] Similar articles 4. Health Qual Life Outcomes. 2015 Nov 16;13(1):183. doi: 10.1186/s12955-015-0380-8. Health-related quality of life in patients with sickle cell disease in Saudi Arabia. Ahmed AE1,2, Alaskar AS3,4,5, Al-Suliman AM6, Jazieh AR7, McClish DK8, Al Salamah M9,10, Ali YZ11, Malhan H12, Mendoza MA13, Gorashi AO14, El-Toum ME15, El-Toum WE16. Abstract BACKGROUND: There is a lack of research concerning health-related quality of life (HRQoL) in Saudi patients with sickle cell disease (SCD), particularly among adult populations. The aim of the current study was to describe the characteristics of SCD patients and their impact on their quality of life (QoL). METHODS: Six hundred twenty-nine adult SCD patients who attended King Fahad Hospital in Hofuf and King Fahad Central Hospital in Jazan were included in the analysis. Demographic/clinical data were collected and an Arabic version of the Medical Outcomes 36-Item Short-Form Health Survey (SF-36) questionnaire was used to assess QoL. RESULTS: SCD patients who hold a university degree reported positive impacts on the following domains of SF-36: physical role function, vitality, emotional well being, social function, pain reduction, and general health (P = .002, P = .001, P = .001, P = .003, P = .004, and P = .001, respectively). In general, patients with fever, skin redness, swelling, or history of blood transfusion tended to impair the health status of the SF-36. A multivariate analysis revealed that patients with a university degree tended to report high scores of physical role functions, emotional role function, and vitality. Patients with regular exercise tend to increase vitality, social function, general health, and reduce pain. Unemployment tends to lessen vitality and worsen pain. On average, pain, social function, and physical function scores tended to worsen in patients with swelling or history of blood transfusion. CONCLUSIONS: This study highlighted that poor education, fever, skin redness, and swelling were negatively associated with specific components of SF-36. SCD patients with a history of blood transfusion found their QoL poorer, whereas regular exercise tended to improve QoL. PMCID: PMC4647668 Free PMC Article  PMID: 26573908 [PubMed - in process] Similar articles 5. Child Neuropsychol. 2015 Nov 15:1-18. [Epub ahead of print] Association of social-environmental factors with cognitive function in children with sickle cell disease. Yarboi J1, Compas BE1, Brody GH2, White D3, Rees Patterson J3, Ziara K3, King A3. Abstract The aim of this study was to examine the relationship between cognitive function in pediatric sickle cell disease (SCD) patients and mothers' reports of social-environmental stress, depressive symptoms, and parenting. A total of 65 children with SCD completed comprehensive neuropsychological testing to assess several domains of cognitive functioning, including general intellectual ability, academic achievement, and executive function. Mothers reported on demographics, social-environmental stress, depressive symptoms, and parenting. As predicted, children with SCD significantly underperformed relative to normative data on measures of cognitive function. Associations between maternal social-environmental stress, maternal depressive symptoms, and parenting were mixed. The results show partial support for the hypothesis that greater stress and depressive symptoms and less positive parenting are associated with poorer cognitive function in children with SCD. Linear regression analyses showed that maternal financial stress was the strongest predictor across all domains of cognitive function. The findings replicate and extend past research, reaffirming that children with SCD are at risk for cognitive impairment across multiple domains. Additionally, social-environmental stress, particularly financial strain, is linked to mothers' depressive symptoms and parenting behaviors as well as children's cognitive function. Future studies using direct observations of parenting behaviors are needed. These findings, along with recent research on parenting interventions, may inform the development of concrete, teachable parenting and coping skills to improve cognitive functioning in children with SCD. PMID: 26568287 [PubMed - as supplied by publisher] Similar articles 6. Am J Hematol. 2015 Nov 6. doi: 10.1002/ajh.24235. [Epub ahead of print] Evolution of Sickle Cell Disease from a life-threatening disease of children to a chronic disease of adults: The last 40 years. Chaturvedi S1, DeBaun MR2. Author information: . Abstract Over the past 40 years, public health measures such as universal newborn screening, penicillin prophylaxis, vaccinations and hydroxyurea therapy have led to an impressive decline in sickle cell disease (SCD)-related childhood mortality and SCD-related morbidity in high-income countries. We remain cautiously optimistic that the next 40 years will be focus on meeting the challenges in SCD today: to address chronic complications of SCD to reduce mortality and improve quality of life in a growing population of adults with SCD in high-income countries, while simultaneously decreasing the disparity of medical care between high and low-income countries. This article is protected by copyright. All rights reserved. © 2015 Wiley Periodicals, Inc. PMID: 26547630 [PubMed - as supplied by publisher] Similar articles 7. Curr Opin Crit Care. 2015 Dec;21(6):569-75. doi: 10.1097/MCC.0000000000000258. Sickle cell disease in the ICU. Cecchini J1, Fartoukh M. Author information: Abstract PURPOSE OF REVIEW: The review focuses on severe acute vaso-occlusive manifestations of sickle cell disease leading adult patients to the ICU. RECENT FINDINGS: Careful consideration should be paid to look for pulmonary vascular dysfunction and acute kidney injury, because of their prognostic role during acute vaso-occlusive manifestations. Alloimmunization and delayed haemolytic transfusion reactions are emerging complications that should be thought to be diagnosed, as they may imply a conservative management. The life-threatening complication raises the question about the indications of blood transfusion therapy for acute sickle cell disease complications, no randomized controlled trials being available to assess the role of blood transfusion in the acute setting. SUMMARY: Acute vaso-occlusive episodes are characterized by an unpredictable course that needs for vigilance for everyone, and justifies ICU or intermediate care unit admission to allow close monitoring, and supportive treatment in a timely fashion. PMID: 26539931 [PubMed - in process] Similar articles 8. Int Wound J. 2015 Nov 4. doi: 10.1111/iwj.12522. [Epub ahead of print] A treatment algorithm to identify therapeutic approaches for leg ulcers in patients with sickle cell disease. Altman IA1, Kleinfelder RE2, Quigley JG3, Ennis WJ1, Minniti CP4. Abstract Sickle cell leg ulcers (SCLUs) are a common complication of sickle cell disease (SCD). Patients who develop ulcers appear to have a more severe haemolysis-associated vasculopathy than individuals who do not develop them, and manifest other complications such as priapism and pulmonary hypertension. SCLUs are slow to heal and often recur, affecting both the emotional and physical well-being of patients. Here we summarise what is known about the pathophysiology of SCLUs, describe available treatment options and propose a treatment algorithm. © 2015 Medicalhelplines.com Inc and John Wiley & Sons Ltd. PMID: 26537664 [PubMed - as supplied by publisher] Similar articles 9. Am J Hematol. 2015 Nov 4. doi: 10.1002/ajh.24232. [Epub ahead of print] Characteristics of a rapid, point-of-care lateral flow immunoassay for the diagnosis of sickle cell disease. McGann PT1, Schaefer BA1, Paniagua M1, Howard TA1, Ware RE1. : Abstract Sickle cell disease (SCD) is a common and life-threatening hematological disorder, affecting approximately 400,000 newborns annually worldwide. Most SCD births occur in low-resource countries, particularly in sub-Saharan Africa, where limited access to accurate diagnostics results in early mortality. We evaluated a prototype immunoassay as a novel, rapid, and low-cost point-of-care (POC) diagnostic device (Sickle SCANTM ) designed to identify HbA, HbS, and HbC. A total of 139 blood samples were scored by three masked observers and compared to results using capillary zone electrophoresis. The sensitivity (98.3-100%) and specificity (92.5-100%) to detect the presence of HbA, HbS, and HbS were excellent. The test demonstrated 98.4% sensitivity and 98.6% specificity for the diagnosis of HbSS disease and 100% sensitivity and specificity for the diagnosis of HbSC disease. Most variant hemoglobins, including samples with high concentrations of HbF, did not interfere with the ability to detect HbS or HbC. Additionally, HbS and HbC were accurately detected at concentrations as low as 1-2%. Dried blood spot samples yielded clear positive bands, without loss of sensitivity or specificity, and devices stored at 37C gave reliable results. These analyses indicate that the Sickle SCAN POC device is simple, rapid, and robust with high sensitivity and specificity for the detection of HbA, HbS, and HbC. The ability to obtain rapid and accurate results with both liquid blood and dried blood spots, including those with newborn high-HbF phenotypes, suggests that this POC device is suitable for large-scale screening and potentially for accurate diagnosis of SCD in limited resource settings. This article is protected by copyright. All rights reserved. © 2015 Wiley Periodicals, Inc. PMID: 26537622 [PubMed - as supplied by publisher] Similar articles 10. Circulation. 2015 Nov 3. pii: CIR.0000000000000329. [Epub ahead of print] Pediatric Pulmonary Hypertension: Guidelines From the American Heart Association and American Thoracic Society. Abman SH, Hansmann G, Archer SL, Ivy DD, Adatia I, Chung WK, Hanna BD, Rosenzweig EB, Raj JU, Cornfield D, Stenmark KR, Steinhorn R, Thébaud B, Fineman JR, Kuehne T, Feinstein JA, Friedberg MK, Earing M, Barst RJ, Keller RL, Kinsella JP, Mullen M, Deterding R, Kulik T, Mallory G, Humpl T, Wessel DL. Abstract Pulmonary hypertension is associated with diverse cardiac, pulmonary, and systemic diseases in neonates, infants, and older children and contributes to significant morbidity and mortality. However, current approaches to caring for pediatric patients with pulmonary hypertension have been limited by the lack of consensus guidelines from experts in the field. In a joint effort from the American Heart Association and American Thoracic Society, a panel of experienced clinicians and clinician-scientists was assembled to review the current literature and to make recommendations on the diagnosis, evaluation, and treatment of pediatric pulmonary hypertension. This publication presents the results of extensive literature reviews, discussions, and formal scoring of recommendations for the care of children with pulmonary hypertension. © 2015 by the American Heart Association, Inc., and the American Thoracic Society. PMID: 26534956 [PubMed - as supplied by publisher] Similar articles 11. J Pediatr Nurs. 2015 Oct 31. pii: S0882-5963(15)00295-X. doi: 10.1016/j.pedn.2015.09.011. [Epub ahead of print] Caregiver Perspectives of Stigma Associated With Sickle Cell Disease in Adolescents. Abstract Patients and families affected by various medical conditions report experiencing health-related stigma, which contributes to detrimental physical, psychological, and social outcomes. Sickle cell disease (SCD) is a genetic disorder that affects 89,000 individuals in the United States and is often associated with negative stereotypes and incorrect assumptions. The present study explored the perception of stigma as reported by caregivers of adolescents with SCD. DESIGN AND METHODS: Focus groups were conducted with 20 caregivers of patients with SCD. Focus groups were audio recorded and transcribed. The data were coded independently by two authors, and then reviewed conjointly until consensus was reached. RESULTS: Caregivers reported the perception of stigma in academic, medical, community, and family settings. They also reported internalized stigma including negative feelings toward having a child with SCD, feeling upset with others, and seeing negative emotions in their child due to SCD. Caregivers reported a general lack of knowledge about SCD across settings. CONCLUSION: These results demonstrated that stigma may affect individuals with SCD across multiple settings. These results also highlighted areas for intervention, with a focus on increasing communication and education toward medical providers, schools, and communities. PRACTICAL IMPLICATIONS: Interventions can utilize technology, social media, and advertisement campaigns. Additionally, support groups for patients with SCD may help decrease stigma and validate patients' experiences. Copyright © 2015 Elsevier Inc. All rights reserved. PMID: 26534838 [PubMed - as supplied by publisher] Similar articles 12. Circulation. 2015 Nov 2. pii: CIR.0000000000000250. [Epub ahead of print] Eligibility and Disqualification Recommendations for Competitive Athletes With Cardiovascular Abnormalities: Task Force 14: Sickle Cell Trait: A Scientific Statement From the American Heart Association and American College of Cardiology. Maron BJ, Harris KM, Thompson PD, Eichner ER, Steinberg MH; American Heart Association Electrocardiography and Arrhythmias Committee of the Council on Clinical Cardiology, Council on Cardiovascular Disease in the Young, Council on Cardiovascular and Stroke Nursing, Council on Functional Genomics and Translational Biology, and the American College of Cardiology. PMID: 26527715 [PubMed - as supplied by publisher] Similar articles 13. Eur Heart J. 2015 Oct 29. pii: ehv555. [Epub ahead of print] Haematological determinants of cardiac involvement in adults with sickle cell disease†. Abstract AIMS: Cardiac involvement is common in sickle cell disease (SCD). Studies are needed to establish haematological determinants of this involvement and prognostic markers. The aim of the study was to identify haematological factors associated with cardiac involvement in SCD and their impact on prognosis. METHODS AND RESULTS: This longitudinal observational study was performed on 1780 SCD patients with SS or S-β0-thalassemia referred to our centre. Six hundred fifty-six met our inclusion criteria (availability of a blood-workup and echocardiogram obtained <1 year apart, no heart valve surgery and no current pregnancy). Median age was 31 (interquartile range, 25-40) years, and median haemoglobin (Hb) was 87 (80-95)g/L. Left ventricular (LV) dilation, left atrial dilation, cardiac index (CI) >4 L/min/m2, LV ejection fraction <55%, and tricuspid regurgitant velocity (TRV) ≥2.5 m/s were found in 35, 78, 23, 8.5, and 17% of patients, respectively. Compared with other patients, those in the fourth quartiles (Q4) of LV end-diastolic dimension index (LVEDDind) and left atrial dimension index (LADind) and those with high CI had significantly lower Hb, % foetal Hb (HbF), and red blood cell (RBC) counts; and significantly higher lactate dehydrogenase, bilirubin, and %dense RBCs. Independent haematologic determinants of Q4 LVEDDind and LADind were low RBC count and %HbF; high %dense RBCs were associated with LADind. Low %HbF and RBC count were associated with high CI. High %dense RBCs or no α-thalassemia gene deletion was associated with greater severity of anaemia and cardiac dilation and with higher CI. During the median follow-up of 48 (32-59) months, 50 (7.6%) patients died. Tricuspid regurgitant velocity ≥ 2.5 m/s was a predictor of mortality. The risk of death increased four-fold when left ventricular ejection fraction <55% was present also (P = 0.0001). CONCLUSION: Cardiac dilation and CI elevation in patients with SCD are associated with haematologic variables reflecting haemolysis, RBC rigidity, and blood viscosity. Tricuspid regurgitant velocity ≥ 2.5 and LV dysfunction (even mild) predict mortality. Published on behalf of the European Society of Cardiology. All rights reserved. © The Author 2015. For permissions please email: journals.permissions@oup.com. PMID: 26516176 [PubMed - as supplied by publisher] Similar articles 14. Br J Haematol. 2015 Nov;171(4):638-46. doi: 10.1111/bjh.13641. Epub 2015 Sep 7. Coagulation activation in sickle cell trait: an exploratory study. Abstract Recent epidemiologic data suggest that sickle cell trait (HbAS; AS) is a risk factor for venous thromboembolism. We conducted an exploratory study of healthy subjects with AS under baseline conditions to determine whether a chronic basal hyperactivation of coagulation exists, and if so, what mechanism(s) contribute to this state. Eighteen healthy AS individuals were compared to 22 African-American controls with a normal haemoglobin profile (HbAA; AA) and 17 patients with sickle cell disease (HbSS; SS). Plasma thrombin-antithrombin complexes and D-dimer levels were elevated in AS relative to AA patients (P = 0·0385 and P = 0·017, respectively), and as expected, were much higher in SSversusAA (P < 0·0001 for both). Thrombin generation in platelet poor plasma was indistinguishable between AA and AS subjects, whereas a paradoxical decrease in endogenous thrombin potential was observed in SS (P ≤ 0·0001). Whole blood tissue factor was elevated in SS compared to AA (P = 0·005), but did not differ between AA and AS. Plasma microparticle tissue factor activity was non-significantly elevated in AS (P = 0·051), but was clearly elevated in SS patients (P = 0·004) when compared to AA controls. Further studies in larger cohorts of subjects with sickle cell trait are needed to confirm the results of this preliminary investigation. © 2015 John Wiley & Sons Ltd. PMID: 26511074 [PubMed - in process] Similar articles

Sickle Cell Conferences and Events

RUN, WALK, FEAST – University City Turkey Trot 5K Run/Walk Charlotte, North Carolina Thursday, November 26, 2015 – Sickle Cell Partners of the Carolinas is hosting a Turkey Trot 5k in Charlotte, NC on November 26th Thanksgiving Day. Join fellow runners and walkers at this 2^nd annual event in Charlotte, NC for all to enjoy. After the race, cool down and enjoy a post-race mini-feast featuring food, drinks and music. The race will feature a fun trot for kids and costumes are encouraged by all those young at heart. Prizes will be awarded for the top three costumes. Your participation in this event will support Sickle Cell Partners of The Carolinas. Volunteer: universitycityturkeytrot@gmail.com   Register: runsignup.com/Race/NC/Charlotte/UniversityCityTurkeyTrot5K       

4th Caribbean Conference on SCD to be held in Kingston, Jamaica during January 20-22, 2016.  For information:

http://www.carest-network.org/2/projects/article/iv-caribbean-conference-on-sickle-cell-disease-advances-in-clinical-care

Listserv  address to join or leave http://scinfo.org/world-wide-resources/sickle-cell-newsletter-listserv

http://listserv.cc.emory.edu/cgi-bin/wa?A0=sicklecell

www.SCInfo.org

Sickle Cell in the Medical Literature | July 2015

Sickle cell in the Medical Literature 1. BMC Med Genomics. 2015 Jul 29;8(1):44. doi: 10.1186/s12920-015-0120-2. Genomic approaches to identifying targets for treating β hemoglobinopathies. Ngo DA1, Steinberg MH2. Author information:   1Department of Medicine, Boston University School of Medicine, 820 Harrison Ave., FGH 1st Floor, Boston, MA, 02118, USA. duyen.ngo@bmc.org.   2Departments of Medicine, Pediatrics, Pathology and Laboratory Medicine, Boston University School of Medicine, 72 E. Concord Street, Boston, MA, 02118, USA. mhsteinb@bu.edu. Abstract Sickle cell disease and β thalassemia are common severe diseases with little effective pathophysiologically-based treatment. Their phenotypic heterogeneity prompted genomic approaches to identify modifiers that ultimately might be exploited therapeutically. Fetal hemoglobin (HbF) is the major modulator of the phenotype of the β hemoglobinopathies. HbF inhibits deoxyHbS polymerization and in β thalassemia compensates for the reduction of HbA. The major success of genomics has been a better understanding the genetic regulation of HbF by identifying the major quantitative trait loci for this trait. If the targets identified can lead to means of increasing HbF to therapeutic levels in sufficient numbers of sickle or β-thalassemia erythrocytes, the pathophysiology of these diseases would be reversed. The availability of new target loci, high-throughput drug screening, and recent advances in genome editing provide the opportunity for new approaches to therapeutically increasing HbF production. Free Article PMID: 26215470 [PubMed - in process] Similar articles 2. Transfus Med Rev. 2015 Jun 19. pii: S0887-7963(15)00071-1. doi: 10.1016/j.tmrv.2015.06.001. [Epub ahead of print] Hyperhemolysis in Patients With Hemoglobinopathies: A Single-Center Experience and Review of the Literature. Danaee A1, Inusa B2, Howard J3, Robinson S4. Author information:   1Department of Hematology, Guys Hospital, London, UK. Electronic address: anicee.danaee@gstt.nhs.uk.   2Evelina Children's Hospital, St Thomas' Hospital, London, UK. Electronic address: baba.inusa@gstt.nhs.uk.   3Department of Hematology, Guys Hospital, London, UK. Electronic address: jo.howard@gstt.nhs.uk.   4Department of Hematology, Guys Hospital, London, UK. Electronic address: susan.robinson@gstt.nhs.uk. Abstract Hyperhemolysis is a severe and potentially life-threatening complication of transfusion described in numerous case reports and gaining recognition since 2009 via the UK Serious Hazards of Transfusion scheme. Although it is predominantly seen in patients with sickle cell disease, there are several reports of this complication in patients with other hemoglobinopathies as well as patients with a range of other hematologic diagnoses who have blood transfusions as part of their management. Our understanding of the underlying pathophysiology of this subtype of delayed transfusion reaction has increased over the last few years; however, there are still questions, which remain unanswered. In our center alone, we have encountered 9 cases in the last 5 years both in the adult and pediatric population. Here we discuss our experience in the diagnosis and management of this complication, and review other cases reported in the literature and the various existing theories behind the pathophysiology of this process. We also discuss the role of genotyping and using DNA technology to aid selection of the most appropriate blood for this patient group. With an increased awareness of hyperhemolysis, it would be advantageous to finally develop international registries to determine the true incidence of hyperhemolysis, better understand the pathophysiology, identify markers to predict which patients are at risk, and inform management guidelines. Copyright © 2015 Elsevier Inc. All rights reserved. PMID: 26209603 [PubMed - as supplied by publisher] Similar articles 3. Health (London). 2015 Jul 24. pii: 1363459315595850. [Epub ahead of print] Resignifying the sickle cell gene: Narratives of genetic risk, impairment and repair. Berghs M1, Dyson SM2, Atkin K3. Author information:   1University of York, UK maria.berghs@york.ac.uk.   2De Montfort University, UK.   3University of York, UK. Abstract Connecting theoretical discussion with empirical qualitative work, this article examines how sickle cell became a site of public health intervention in terms of 'racialised' risks. Historically, sickle cell became socio-politically allied to ideas of repair, in terms of the state improving the health of a neglected ethnic minority population. Yet, we elucidate how partial improvements in care and education arose alongside preventative public health screening efforts. Using qualitative research based in the United Kingdom, we show how a focus on collective efforts of repair can lie in tension with how services and individuals understand and negotiate antenatal screening. We illustrate how screening for sickle cell disorder calls into question narrative identity, undoing paradigms in which ethnicity, disablement and genetic impairment become framed. Research participants noted that rather than 'choices', it is 'risks' and their negotiation that are a part of discourses of modernity and the new genetics. Furthermore, while biomedical paradigms are rationally and ethically (de)constructed by participants, this was never fully engaged with by professionals, contributing to overall perception of antenatal screening as disempowering and leading to disengagement. © The Author(s) 2015. PMID: 26208697 [PubMed - as supplied by publisher] Similar articles 4. Hemoglobin. 2015 Jul 24:1-4. [Epub ahead of print] Incidence and Predictors of Bacterial infection in Febrile Children with Sickle Cell Disease. Morrissey BJ1, Bycroft TP, Almossawi O, Wilkey OB, Daniels JG. Author information:   1Department of Paediatrics, North Middlesex University Hospital, Sterling Way , Edmonton, London , UK. Abstract Children with sickle cell disease are at increased risk of developing bacteremia and other serious bacterial infections. Fever is a common symptom in sickle cell disease and can also occur with sickle cell crises and viral infections. We aimed to evaluate the incidence and predictors of bacteremia and bacterial infection in children with sickle cell disease presenting with fever to a district hospital and sickle cell center in London. A retrospective analysis was performed on all attendances of children (aged under 16 years) with sickle cell disease presenting with a fever of 38.5 °C or higher over a 1-year period. Confirmed bacterial infection was defined as bacteremia, bacterial meningitis, urinary tract infection (UTI), pneumonia, osteomyelitis or other bacterial infection with positive identification of organism. Children were defined as having a suspected bacterial infection if a bacterial infection was suspected clinically, but no organism was identified. Over a 1-year period there were 88 episodes analyzed in 59 children. Bacteremia occurred in 3.4% of episodes and confirmed bacterial infection in 7.0%. Suspected bacterial infection occurred in 33.0%. One death occurred from Salmonella typhirium septicemia. C-reactive protein (CRP) level and white blood cell (WBC) count were both significantly associated with bacterial infection (p = 0.004 and 0.02, respectively.) In conclusion, bacterial infections continue to be a significant problem in children with sickle cell disease. C-reactive protein was significantly associated with bacterial infections, and could be included in clinical risk criteria for febrile children with sickle cell disease. PMID: 26207314 [PubMed - as supplied by publisher] Similar articles 5. J Blood Med. 2015 Jul 10;6:229-38. doi: 10.2147/JBM.S60515. eCollection 2015. Hematopoietic stem cell transplantation in sickle cell disease: patient selection and special considerations. Bhatia M1, Sheth S2. Author information:   1Division of Pediatric Hematology/Oncology/Stem Cell Transplantation, Columbia University Medical Center, New York, NY, USA.   2Division of Pediatric Hematology and Oncology, Weill Cornell Medical College, New York, NY, USA. Abstract Hematopoietic stem cell transplantation remains the only curative treatment currently in use for patients with sickle cell disease (SCD). The first successful hematopoietic stem cell transplantation was performed in 1984. To date, approximately 1,200 transplants have been reported. Given the high prevalence of this disorder in Africa, and its emergence in the developed world through immigration, this number is relatively small. There are many reasons for this; primary among them are the availability of a donor, the risks associated with this complex procedure, and the cost and availability of resources in the developing world. Of these, it is fair to say that the risks associated with the procedure have steadily decreased to the point where, if currently performed in a center with experience using a matched sibling donor, overall survival is close to 100% and event-free survival is over 90%. While there is little controversy around offering hematopoietic stem cell transplantation to symptomatic SCD patients with a matched sibling donor, there is much debate surrounding the use of this modality in "less severe" patients. An overview of the current state of our understanding of the pathology and treatment of SCD is important to show that our current strategy is not having the desired impact on survival of homozygous SCD patients, and should be changed to significantly impact the small proportion of these patients who have matched siblings and could be cured, especially those without overt clinical manifestations. Both patient families and providers must be made to understand the progressive nature of SCD, and should be encouraged to screen full siblings of patients with homozygous SCD for their potential to be donors. Matched siblings should be referred to an experienced transplant center for evaluation and counseling. In this review, we will discuss the rationale for these opinions and make recommendations for patient selection.  PMCID: PMC4506029 Free PMC Article  PMID: 26203293 [PubMed] Similar articles 6. J Clin Pharmacol. 2015 Jul 22. doi: 10.1002/jcph.598. [Epub ahead of print] Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia. Estepp JH1, Melloni C2, Thornburg CD3, Wiczling P4, Rogers Z5, Rothman JA6, Green NS7, Liem R8, Brandow AM9, Crary SE10, Howard TH11, Morris MH12, Lewandowski A13, Garg U14, Jusko WJ15, Neville KA16; Best Pharmaceuticals for Children Act - Pediatric Trials Network Administrative Core Committee. Author information:   1Departments of Hematology and Pathology, St. Jude Children's Research Hospital, Memphis, TN, USA.   2Division of Clinical Pharmacology, Department of Medicine, Duke University Medical Center, Durham, NC, USA.   3Department of Pediatrics, University of California-San Diego, La Jolla, CA, USA.   4Department of Biopharmaceutics and Pharmacodynamics, Medical University of Gdańsk, Gdańsk, Poland.   5Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.   6Division of Pediatric Hematology/Oncology, Duke University Medical Center, Durham, NC, USA.   7Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Columbia University Medical Center, New York, NY, USA.   8Division of Hematology, Oncology, and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.   9Section of Pediatric Hematology/Oncology, Medical College of Wisconsin, and Children's Research Institute of the Children's Hospital of Wisconsin, Milwaukee, WI, USA.   10Division of Pediatric Hematology-Oncology, University of Arkansas for Medical Sciences/Arkansas Children's Hospital, Little Rock, AR, USA.   11Division of Pediatric Hematology and Oncology, University of Alabama at Birmingham School of Medicine, Birmingham, AL, USA.   12Duke Clinical Research Institute, Durham, NC, USA.   13The Emmes Corporation, Rockville, MD, USA.   14Department of Pathology and Laboratory Medicine, Children's Mercy Hospitals and Clinics, Kansas City, MO, USA.   15Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, University at Buffalo, Buffalo, NY, USA.   16Section of Pharmacology and Toxicology, University of Arkansas for Medical Sciences and Arkansas Children's Hospital, Little Rock, AR, USA. Abstract Hydroxyurea (HU) is recommended for all children starting as young as 9 months of age with sickle cell anemia (SCA; HbSS and HbSβ0 thalassemia); however, a paucity of pediatric data exists regarding the pharmacokinetics (PK) or the exposure-response relationship of HU. This trial aimed to characterize the PK of HU in children and to evaluate and compare the bioavailability of a liquid versus capsule formulation. This multi-center, prospective, open-label trial enrolled 39 children with SCA who provided 682 plasma samples for PK analysis following administration of HU. Noncompartmental and population PK models are described. We report that liquid and capsule formulations of HU are bioequivalent, weight-based dosing schemes provide consistent drug exposure, and age-based dosing schemes are unnecessary. These data support the use of liquid HU in children unable to swallow capsules and in those whose weight precludes the use of fixed capsule formulations. Taken with existing safety and efficacy literature, these findings should encourage the use of HU across the spectrum of age and weight in children with SCA, and they should facilitate the expanded use of HU as recommended in the National Heart, Lung, and Blood Institute guidelines for individuals with SCA. This article is protected by copyright. All rights reserved. This article is protected by copyright. All rights reserved. PMID: 26201504 [PubMed - as supplied by publisher] Similar articles 7. Hemoglobin. 2015 Jul 16:1-4. [Epub ahead of print] Aging in Sickle Cell Disease: Co-morbidities and New Issues in Management. Sandhu MK1, Cohen A. Author information:   1Department of Hematology/Oncology, Newark Beth Israel Medical Center , Newark, New Jersey , USA. Abstract Availability of hydroxyurea (HU) coupled with early therapeutic interventions has increased the life expectancy of patients with sickle cell disease. Hence, the sickle cell community needs to be aware of common diseases of aging that survivors are predisposed to. We chose to investigate the sickle cell disease-related complications as well as non sickle cell disease-related medical problems of aging in 45 sickle cell patients over the age of 40 years. The most frequent chronic complications of sickle cell disease were elevated tricuspid regurgitant jet velocity on echocardiogram, chronic renal disease, iron overload and leg ulcers. Medical co-morbidities in this patient group included hypertension, diabetes mellitus (DM), hypercholesterolemia and symptomatic coronary artery disease (CAD). In our cohort, only 38.0% had a primary care doctor. Only 11.0% over age 50 had a screening colonoscopy, and of the women, 42.0% had a screening mammography. Medical co-morbidities and lack of health maintenance in older sickle cell patients are likely to impact overall health and mortality. Aging patients with sickle cell disease may benefit from a primary medical home for age appropriate comprehensive health care. PMID: 26182337 [PubMed - as supplied by publisher] Similar articles 8. Pediatr Blood Cancer. 2015 Jul 14. doi: 10.1002/pbc.25634. [Epub ahead of print] Adherence to prompt fever evaluation in children with sickle cell disease and the health belief model. Schultz CL1, Tchume-Johnson T1, Schapira MM2,3,4, Bellamy S5, Smith-Whitley K1,3, Ellison A1,3. Author information:   1The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.   2Abramson Cancer Center, Philadelphia, Pennsylvania.   3Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania.   4Philadelphia VA Hospital, Philadelphia, Pennsylvania.   5Department of Biostatistics and Epidemiology, Center for Clinical Epidemiology and Biostatistics at the University of Pennsylvania, Philadelphia, Pennsylvania. Abstract BACKGROUND: Children with sickle cell disease (SCD) are at increased risk of death from invasive bacterial infections. Emergent evaluation of fever allows early treatment of potentially fatal infections. Limited data exist regarding caregiver adherence to physician recommendations of prompt medical evaluation of fever in children with SCD. Better understanding of parental behavior around fever management may inform improved models for support in families of children with SCD. PROCEDURE: Cross-sectional survey based on health belief domains, Wake Forest trust scales, and self-reported adherence among 163 caregivers of children with SCD during routine hematology visit. RESULTS: Fifty-five percent of caregivers were adherent to fever evaluation recommendations as defined by "always" seeking medical evaluation of fever in their child with SCD. Perceived susceptibility to fever/infection, benefits of prompt evaluation, and cues to action were significantly different between those who adhere to recommendations versus those who do not. Twenty-five percent believe their child does not need antibiotics with every fever whereas 17% believe their child does not need evaluation of fever after immunizations. Fifty-seven percent report their employer understands missing work whereas 25% report concern regarding cost of evaluation. Trust in their child's hematologist and medical profession was high (composite scores 23.4/25 and 21/25, respectively). CONCLUSION: Despite a high degree of agreement in importance of fever evaluation and high levels of trust, many caregivers do not consistently seek care when their child has a fever. Future studies should address additional barriers to seeking emergency care in children with SCD and fever. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc. © 2015 Wiley Periodicals, Inc. PMID: 26179160 [PubMed - as supplied by publisher] Similar articles 9. Am J Hematol. 2015 Jul 15. doi: 10.1002/ajh.24116. [Epub ahead of print] 2015 Clinical Trials Update in Sickle Cell Anemia. Archer N1, Galacteros F2, Brugnara C3. Author information:   1Pediatric Hematology/Oncology Dana-Farber/Children's Hospital Blood Disorders and Cancer Center, Boston, MA, USA.   2Centre de Référence des syndromes drépanocytaires majeurs, Hôpital Henri-Mondor, APHP, UPEC, U955 Inserm, Creteil, France.   3Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School Boston, MA, USA. Abstract 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. This article is protected by copyright. All rights reserved. © 2015 Wiley Periodicals, Inc. PMID: 26178236 [PubMed - as supplied by publisher] Similar articles 10. Curr Opin Pulm Med. 2015 Sep;21(5):432-437. Pulmonary hypertension in sickle cell disease. Fonseca G1, Souza R. Author information:   1aDepartment of Hematology bDepartment of Pulmonary, Heart Institute, University of São Paulo Medical School, São Paulo, Brazil. Abstract PURPOSE OF REVIEW: This review highlights the prevalence of pulmonary hypertension as a chronic complication of sickle cell disease (SCD) and its importance in the prognosis. RECENT FINDINGS: In recent years, the limitations of echocardiogram for the appropriate diagnosis of SCD-associated pulmonary hypertension have been demonstrated, emphasizing the need of invasive hemodynamics assessment before any specific treatment for pulmonary hypertension is considered. The hemodynamic profile observed in this clinical situation is characterized by elevated cardiac output and low pulmonary vascular resistance that differs considerably from what is seen in pulmonary arterial hypertension. Furthermore, both hemodynamic profiles, precapillary and postcapillary, can be equally found in this setting stressing the need for a better understanding of the multiple pathophysiological mechanisms involved in the development of pulmonary hypertension before considering those patients for targeted therapies. Nevertheless, the presence of any form of pulmonary hypertension clearly denotes worse prognosis in SCD. SUMMARY: Pulmonary hypertension is an important and prevalent complication of SCD with multiple associated mechanisms. A more aggressive approach of the baseline condition might be necessary, although the data supporting this assumption and also the use of targeted pulmonary arterial hypertension therapy are still lacking. PMID: 26176970 [PubMed - as supplied by publisher] Similar articles 11. Semin Dial. 2015 Jul 14. doi: 10.1111/sdi.12403. [Epub ahead of print] Management of the Dialysis Patient with Sickle Cell Disease. Boyle SM1, Jacobs B2, Sayani FA2, Hoffman B3. Author information:   1Division Nephrology and Hypertension, Drexel University College of Medicine, Philadelphia, Pennsylvania.   2Hematology and Oncology Division, Hospital of the University of Pennsylvania, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania.   3Renal, Hypertension and Electrolyte Division, Penn Presbyterian Medical Center, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania. Abstract While patients with sickle cell disease currently constitute a very small minority of the US dialysis population (0.1%), there is anticipated growth of this group as the life expectancy of those with sickle cell disease (SCD) increases. SCD patients suffer a high burden of morbidity, which is enhanced by the presence of end-stage renal disease (ESRD). In this review, we discuss the pathophysiology of SCD and the basic tenets of its management with focus on the dialysis patient with SCD. Anemia in dialysis patients with SCD is a unique challenge. The hemoglobin target in SCD dialysis patients with ESRD should not exceed 10 g/dl. SCD patients, and particularly those on dialysis, are likely to be poorly responsive to erythropoietin-stimulating agent (ESA) therapy and might be at increased risk for vaso-occlusive crisis (VOC) with ESA. Iron chelation and hydroyxurea therapy require special considerations and modifications in dialysis patients with SCD. There are theoretical advantages to both hemodialysis (HD) and peritoneal dialysis (PD) in SCD patients. With HD, there is a secure vascular access available for both standard and exchange blood transfusion in patients who need them. With PD, the absence of an acute rise in hematocrit with ultrafiltration (UF) might offer lower risk of VOC. During VOC, reduction in UF goals should be considered but administration of intravenous fluids should be reserved only for clear cases of volume depletion. Finally, renal transplantation appears to confer a survival advantage to dialysis in SCD patients and should be pursued when possible. © 2015 Wiley Periodicals, Inc. PMID: 26174870 [PubMed - as supplied by publisher] Similar articles 12. Pediatr Blood Cancer. 2015 Jul 14. doi: 10.1002/pbc.25655. [Epub ahead of print] Contribution of sickle cell disease to the pediatric stroke burden among hospital discharges of African-Americans-United States, 1997-2012. Baker C1, Grant AM1, George MG2, Grosse SD1, Adamkiewicz TV3. Author information:   1Division of Blood Disorders, National Center for Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, Georgia.   2Division for Heart Disease and Stroke Prevention, National Center for Chronic Disease Prevention and Health Promotion, Centers for Disease Control and Prevention, Atlanta, Georgia.   3Department of Family Medicine, Morehouse School of Medicine, Atlanta, Georgia. Abstract BACKGROUND: Approximately 10-20% of children with sickle cell disease (SCD) develop stroke, but few consistent national estimates of the stroke burden for children with SCD exist. The purpose of this study is to determine the proportion of diagnosed stroke among African-American pediatric discharges with and without SCD. PROCEDURE: Records for African-Americans aged 1-18 years in the Kids' Inpatient Database (KID) 1997-2012 with ≥1 ICD-9-CM diagnosis code for stroke were included. Data were weighted to provide national estimates. A total of 2,994 stroke cases among African-American children were identified. Diagnoses co-existing with ischemic or hemorrhagic stroke were frequency ranked separately. RESULTS: From 1997 through 2012, SCD was present in 24% of stroke discharges, with 89% being ischemic stroke. For hospital discharges of African-American children, SCD is the highest co-existing risk factor for ischemic stroke (29%). Stroke in children with SCD occurred predominantly in children aged 5-9 years, older than previously reported. The trend of stroke discharges significantly decreased for children with SCD from 1997 to 2012 for children aged 10-14 years. CONCLUSIONS: SCD is a leading risk factor to pediatric stroke in African-American children. Reducing the number of strokes among children with SCD would have a significant impact on the rate of strokes among African-American children. Preventative intervention may be modifying initial age of presentation of stroke in children with SCD. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc. © 2015 Wiley Periodicals, Inc. PMID: 26174777 [PubMed - as supplied by publisher] Similar articles 13. J Pediatr. 2015 Jul 7. pii: S0022-3476(15)00611-3. doi: 10.1016/j.jpeds.2015.06.028. [Epub ahead of print] Impact of a Comprehensive Sickle Cell Center on Early Childhood Mortality in a Developing Country: The Jamaican Experience. King LG1, Bortolusso-Ali S2, Cunningham-Myrie CA3, Reid ME4. Author information:   1Sickle Cell Unit, Tropical Medicine Research Institute, University of the West Indies, Mona, Kingston, Jamaica, West Indies. Electronic address: lesley.king@uwimona.edu.jm.   2Sickle Cell Unit, Tropical Medicine Research Institute, University of the West Indies, Mona, Kingston, Jamaica, West Indies.   3Department of Community Health and Psychiatry, University of the West Indies, Mona, Kingston, Jamaica, West Indies.   4Tropical Metabolism Research Unit, Tropical Medicine Research Institute, University of the West Indies, Mona, Kingston, Jamaica, West Indies. Abstract OBJECTIVE: To compare mortality in children <5 years of age with sickle cell disease (SCD) in Jamaica, a resource-limited country, diagnosed by newborn screening and managed in a comprehensive care facility, to that of the general population. STUDY DESIGN: The study was carried out at the Sickle Cell Unit in Kingston, Jamaica. We determined the status (dead/alive) at age 5 years in a cohort of 548 children with SCD diagnosed by newborn screening and managed at the Sickle Cell Unit during the period November 1995 to December 2009. The standardized mortality ratio was calculated using World Health Organization life tables for reference mortality. RESULTS: Eight deaths (1.5%) occurred in children <5 years of age during the study period. The mean age at death was 2.0 ± 1.5 years. The overall mortality incidence in children <5 years of age was 3.1 (95% CI 1.6, 6.2) per 1000 person-years with a standardized mortality ratio of 0.52 (95% CI 0.3, 1.0). CONCLUSIONS: Mortality in children <5 years of age with SCD diagnosed at birth and managed at a comprehensive care clinic in Jamaica is equivalent to that of the general population. Children with SCD, a highly vulnerable population, can be effectively managed, even in resource-limited environments. Copyright © 2015 Elsevier Inc. All rights reserved. PMID: 26163082 [PubMed - as supplied by publisher] Similar articles 14. Clin Pediatr (Phila). 2015 Jul 6. pii: 0009922815594345. [Epub ahead of print] Outpatient Management of Febrile Children With Sickle Cell Disease. Sokol E1, Obringer E1, Palama B1, Hageman J1, Peddinti R2. Author information:   1University of Chicago, Chicago, IL, USA.   2University of Chicago, Chicago, IL, USA rpeddint@peds.bsd.uchicago.edu. Abstract The electronic medical records at 2 children's hospitals were reviewed from June 1, 2011 to May 31, 2013 for all patients with sickle cell disease who presented with fever. Of a total of 390 blood cultures drawn, 11 cultures (2.8%) turned positive with only 1 (0.3%) growing a true pathogen. This culture turned positive in 13 hours. There were 154 patients who received exclusive outpatient management of fever. Fourteen patients (9.1%) completed 1 acute care visit, 16 patients (10.4%) completed 2 acute care visits, and 124 patients (80.5%) completed 3 acute care visits. Of those treated exclusively as outpatients, there was 1 positive culture that was considered a contaminant. Although the overall rate of positivity was low, this study confirms previous findings that pediatric blood cultures become positive with pathogens within 48 hours. Given the high rate of compliance and early time to positivity of true pathogens, we suggest that follow-up for the febrile sickle cell disease patients can be treated on an outpatient basis. © The Author(s) 2015. PMID: 26149843 [PubMed - as supplied by publisher] Similar articles 15. Haematologica. 2015 Jul 2. pii: haematol.2015.130435. [Epub ahead of print] Hydroxyurea treatment does not increase blood viscosity and improves red blood cell rheology in sickle cell anemia. Lemonne N1, Charlot K2, Waltz X2, Ballas SK3, Lamarre Y2, Lee K1, Hierso R2, Connes C2, Etienne-Julan M1, Romana M2, Connes P4. Author information:   1UTD;   2Inserm;   3Jefferson University;   4Universite' Lyon 1 pconnes@yahoo.fr. Free Article PMID: 26137960 [PubMed - as supplied by publisher] Similar articles 16. Hosp Pediatr. 2015 Jul;5(7):377-84. doi: 10.1542/hpeds.2014-0171. Improving Clinician Attitudes of Respect and Trust for Persons With Sickle Cell Disease. Haywood C Jr1, Williams-Reade J2, Rushton C3, Beach MC3, Geller G3. Author information:   1The Berman Institute of Bioethics, Johns Hopkins University, Baltimore, Maryland; and chaywoodjr@jhu.edu.   2Loma Linda University, Loma Linda, California.   3The Berman Institute of Bioethics, Johns Hopkins University, Baltimore, Maryland; and. Abstract OBJECTIVE: To test the effect of 1 high-intensity, and 1 reduced-intensity, educational intervention designed to improve health care provider attitudes toward youth with sickle cell disease (SCD). METHODS: We exposed a regional sample of pediatric health care providers to a 2.5-day high-intensity educational and experiential intervention using videos about the SCD patient experience. Additionally, we traveled to a different set of regional health care institutions and offered pediatric providers a reduced-intensity intervention, consisting of a 90-minute lunchtime in-service centered on our same set of videos about the patient's experience. We assessed the impact of both interventions by taking pre/post measurements of the negative and positive attitudes expressed by participating providers toward patients with SCD. RESULTS: Both interventions tested elicited improvements in the SCD attitudes expressed by the pediatric providers as suggested through a reduction in measured negative attitude scores (20.0 vs 12.1, P < .001), and an improvement in positive attitude scores (67.1 vs 72.2, P < .001). Further testing suggested that the high-intensity intervention elicited a stronger effect than the reduced-intensity intervention across multiple attitudinal domains. CONCLUSIONS: Video-based interventions can be used to improve the attitudes of pediatric providers toward patients with SCD. The availability of interventions of varying intensities provides greater flexibility in designing efforts to advance the quality of SCD care through the improvement of provider attitudes. Copyright © 2015 by the American Academy of Pediatrics. PMID: 26136312 [PubMed - in process] Similar articles 17. Hematology. 2015 Jul 2. [Epub ahead of print] Splenic infarction associated with sickle cell trait at low altitude. Seegars MB, Brett AS. Abstract Objective and importance Sickle cell trait is widely known to be associated with splenic infarction at high altitudes. Although textbooks and reviews imply that this complication does not occur at low altitudes, we encountered such a case and identified several previous cases in the literature. Clinical presentation An 18-year-old woman with sickle cell trait who resided near sea level presented with left upper quadrant abdominal pain and was found to have multiple splenic infarcts. She was otherwise well, with no comorbidities that would predispose to hypoxemia or vascular injury. A review of the literature revealed 12 previously published cases of low-altitude splenic infarction in patients with sickle trait; 7 of those patients had comorbidities that likely predisposed to splenic infarction. Intervention None. Conclusion Spontaneous splenic infarction can occur in patients with sickle trait who live at low altitudes. It is unclear whether this complication is rare, or whether it is relatively common but under-recognized.
Sickle Cell Conferences and Events 2015 Sickle Cell Disease Conference: Improving the Health of Individuals and Families Living with Sickle Cell Date: September 11-12, 2015 Venue: Duke University School of Nursing For more information: events.duke.edu/scdconference2015