Sickle Cell News for March 2019– To join or leave the listserv visit http://scinfo.org/newsletter/

Dr. Doris Wethers, 91, on Front Lines Against Sickle Cell, Dies

https://www.nytimes.com/2019/02/07/obituaries/dr-doris-wethers-on-front-lines-against-sickle-cell-dies-at-91.html Dr. Doris L. Wethers, who broke racial barriers in the medical world before gaining renown for research and advocacy that helped lead to mandatory testing of all newborns for sickle cell anemia, died on Jan. 28 in Yonkers. She was 91.

The cause was complications of a stroke, her daughter-in-law Lisa Booker said.

In 1965, Dr. Wethers became the first black chief of a medical department at a New York City voluntary, or private nonprofit, hospital when she was named director of pediatrics at Knickerbocker Hospital in West Harlem.

She was later director of pediatrics from 1969 to 1974 at Sydenham Hospital (which was shuttered in 1980) and then, until 1979, at St. Luke’s Hospital Center (now Mount Sinai St. Luke’s). She became St. Luke’s first black attending physician in 1958. Dr. Wethers opened sickle cell anemia programs at all three hospitals, conducted research and helped draft landmark legislation in New York to require screening of infants for the disorder. Over the course of her career at the hospitals, the average life expectancy of children born with sickle cell rose from about 18 to 50.

The increase was attributed largely to early detection, infection prevention through the use of penicillin and other breakthroughs that helped mitigate pain and prolong life.

Funding Source

Feb. 19, 2019 – Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced the launch of the Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL) to provide grant funding to support novel projects aimed at improving access to high-quality healthcare for sickle cell patients in the United States.

GBT will fund as many as three proposals up to $50,000 each to accelerate the development of promising programs with the potential over time to deliver high-quality healthcare to people living with sickle cell disease (SCD).

“Studies show that healthcare delivery to people living with SCD is typically suboptimal. For example, in the United States, fewer than 10 percent of Medicaid and Medicare patients living with SCD see a hematologist at least once per year and approximately 20 percent of SCD patients receive most of their care in the emergency room,” said Jung Choi, who oversees patient advocacy and government affairs at GBT. “We are excited to launch ACCEL to encourage the development of innovative solutions to provide underserved SCD patients with better access to high-quality care and support.”

ACCEL builds on discussions from the SCD Access to Care Summit sponsored by GBT and held in September 2018, during which healthcare providers and members of the sickle cell community discussed programs that are successfully working to address the significant gaps in healthcare delivery for both adults and children living with SCD. During the Summit, participants created draft roadmaps of these models to help disseminate best practices and to encourage the initiation of new SCD access-to-care programs.

Proposals will be reviewed by a panel of GBT personnel and external stakeholders with expertise in the issues affecting people with SCD. The panel will select as many as three proposals based on strength, degree of innovation and highest potential impact to patient care.

For more information about ACCEL, visit https://www.gbt.com/patients/accel-grant-program/ or email patientadvocacy@gbt.com.

Web Resource

At the National Heart, Lung and Blood Institute (NHLBI), we’re dedicated to finding cures for sickle cell disease. Support the fight — and keep up with our progress.

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Singer Jordin Sparks is telling us about her personal connection to Sickle Cell Disease and why she’s raising awareness about it. https://www.wfsb.com/better/jordin-sparks-on-her-personal-connection-to-sickle-cell-disease/video_89b68ad5-59cc-5971-a221-acc7a91d0345.html

She’s joined by Dr. Venee Tubman. Visit joingens.com to learn more

The Warrior University Online Series

Sickle Cell Consortium and REdHHoTT presents: Blood Transfusions – What You Need to Know and Do https://ghpc.wufoo.com/forms/the-warrior-university-online-series/


Join us for an online presentation followed by a conversation with leading experts, Drs. James Eckman and Raymona Lawrence.

All sessions will begin at 6:30 p.m. 

March 4
Keeping it Safe: Iron Overload

March 6
African-American Blood Donations: Why are they important?

March 11
Let’s Talk: Patient-Provider Communication

Articles in the medical literature

1. Obes Surg. 2019 Feb 25. doi: 10.1007/s11695-019-03780-0. [Epub ahead of print] Impact of Bariatric Surgery on Outcomes of Patients with Sickle Cell Disease: a Nationwide Inpatient Sample Analysis, 2004-2014. Sharma P1,2McCarty TR1Yadav S3Ngu JN4Njei B5. Abstract BACKGROUND: With advances in disease-specific treatments and improved overall survival, obesity rates are rising among patients with sickle cell disease (SCD). The primary aim of this study was to evaluate the role of bariatric surgery on clinical outcomes among hospitalized obese patients with SCD. METHODS: The United States Nationwide Inpatient Sample database was queried between 2004 and 2014 for discharges with co-diagnoses of morbid obesity and SCD. The primary outcome was in-hospital mortality. Secondary outcomes included vaso-occlusive crisis, acute chest syndrome, biliary-pancreatic complications, renal failure, urinary tract infection, malnutrition, sepsis, pneumonia, respiratory failure, thromboembolic events, strictures, wound infection, length of stay, and hospitalization costs. Using Poisson regression, adjusted incidence risk ratios (IRR) were derived for clinical outcomes in patients with prior-bariatric surgery compared to those without bariatric surgery. RESULTS: Among 2549 patients with a discharge diagnosis of SCD and morbid obesity, only 42 patients (1.7%) had bariatric surgery. On multivariable analysis, bariatric surgery did not influence mortality (P = 0.98). Bariatric surgery was not associated with increased risk for acute chest syndrome, sepsis, multi-organ failure, biliary-pancreatic, or surgery-related complications (all P > 0.05). Interestingly, bariatric surgery decreased risk of vaso-occlusive crises (IRR 0.21; 95% CI, 0.07-0.69; P = 0.01) in these patients and was associated with a shorter length of stay (P < 0.001) but higher hospitalization costs (P < 0.001). CONCLUSIONS: Bariatric surgery may lower rates of vaso-occlusive crises in morbidly obese sickle cell patients without significantly affecting mortality and other adverse outcomes. In spite of this, these weight loss surgeries are underutilized in this select population.
PMID: 30805858
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2. Am J Kidney Dis. 2019 Feb 20. pii: S0272-6386(19)30007-1. doi: 10.1053/j.ajkd.2018.12.027. [Epub ahead of print] Progressive Decline in Estimated GFR in Patients With Sickle Cell Disease: An Observational Cohort Study. Derebail VK1Ciccone EJ2Zhou Q3Kilgore RR4Cai J5Ataga KI6. Abstract RATIONALE & OBJECTIVE: Progression of chronic kidney disease (CKD) in sickle cell disease (SCD) and its risk factors remain poorly defined. We identified characteristics associated with CKD as well as decline in estimated glomerular filtration rate (eGFR) and presence of proteinuria over time in adults with SCD. STUDY DESIGN: Retrospective observational study. SETTING & PARTICIPANTS: Patients with SCD 18 years or older in a single center from 2004 to 2013. PREDICTORS: Baseline clinical and laboratory measures, comorbid conditions, SCD-related complications, relevant treatments, and severity of genotypes defined as severe (homozygous SCD [HbSS]/sickle-β0-thalassemia [HbSβ0]) or mild (hemoglobin SC disease [HbSC]/sickle-β+-thalassemia [HbSβ+]-thalassemia). OUTCOMES: Presence at baseline of CKD, defined here as eGFR<90mL/min/1.73m2 or proteinuria (≥1+) on urinalysis or current kidney transplant or dialysis therapy; change in eGFR; and presence of proteinuria over time. ANALYTICAL APPROACH: Logistic regression for baseline CKD. Linear mixed-effects model for eGFR decline and generalized linear mixed-effects model for proteinuria during the study period evaluating for interaction with time. Stratified by genotype severity. RESULTS: Among 427 patients, 331 had 2 or more measurements of creatinine. During a median follow-up of 4.01 (interquartile range, 1.66-7.19) years, annual eGFR decline was 2.05mL/min/1.73m2 for severe genotypes (P<0.001) and 1.16mL/min/1.73m2 (P=0.02) for mild genotypes. At baseline, 21.4% of patients with severe genotypes had CKD versus 17.2% of those with mild genotypes. For severe genotypes, angiotensin-converting enzyme-inhibitor/angiotensin receptor blocker use (OR, 6.10; 95% CI, 2.03-18.29; P=0.001) and avascular necrosis (OR, 0.40; 95% CI, 0.16-0.97; P=0.04) were associated with baseline CKD. Among those with mild genotypes, higher hemoglobin level was associated with lower probability of CKD (OR per 1-g/dL greater hemoglobin level, 0.63; 95% CI, 0.43-0.93; P=0.02). Rate of eGFR decline was inversely related to hemoglobin level (β = 0.46 [SE, 0.23]; P=0.04) within the severe genotype subgroup. No factors were identified to be associated with proteinuria over time. LIMITATIONS: Retrospective observational study, limited direct measures of albuminuria. CONCLUSIONS: Patients with SCD exhibit rapid decline in eGFR over time. Decline in eGFR is associated with markers of disease severity and associated comorbid conditions. Copyright © 2019 National Kidney Foundation, Inc. Published by Elsevier Inc. All rights reserved.
PMID: 30797615
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3. Paediatr Child Health. 2019 Feb;24(1):e45-e50. doi: 10.1093/pch/pxy074. Epub 2018 May 31. Oral morphine protocol evaluation for the treatment of vaso-occlusive crisis in paediatric sickle cell patients. Paquin H1D Trottier E1Robitaille N2Pastore Y2Dore Bergeron MJ3Bailey B1. Abstract Background: Vaso-occlusive crisis (VOC) is one of the most frequent causes of emergency visit and admission in children with sickle cell disease (SCD). Objectives: This study aimed to evaluate whether the implementation of a protocol promoting the use of oral morphine as a primary intervention has led to improved care of SCD. Methods: We performed a retrospective chart review of patients with SCD who presented to the emergency department (ED) and hematology outpatient clinic (HOC) with VOC, in the year pre and postimplementation of the protocol. The primary outcome was the hospitalization rate. Results: The protocol resulted in a significant 43% reduction of hospitalization rate (95% confidence interval [CI] -53.0, 26.5). Results also showed a 35% increase in the use of oral morphine as first-line opiate treatment (95% CI 17.9, 45.2), a 28% increase in the use of pain scales (95% CI 17.3, 43.2) and a 30% net increase in patients eventually not requiring intravenous (IV) line placement (95% CI 16.0, 39.9). While we did observe an overall decrease in length of stay in ED of -55 min (95% CI -100.6, -12.0), there was a nonsignificant decrease of 7 minutes (95% CI -26, 3) in the opiate administration time. Conclusions: This study validates the use of our oral morphine protocol for the treatment of VOC by significantly reducing the admission rate and decreasing the number of IVs. PMCID: PMC6376293 [Available on 2020-02-01]
PMID: 30792609
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4. J Pediatr Hematol Oncol. 2019 Feb 15. doi: 10.1097/MPH.0000000000001433. [Epub ahead of print] Parent and Guardian Knowledge of Hematopoietic Cell Transplantation as a Treatment Option for Sickle Cell Disease. Stallings AM1Majhail NS2Nowacki AS1,3Onimoe GI4Hanna R4Wen J1Umana J5Quinn CT6Hsu LL7Piccone CM1,8. Abstract Beginning early in childhood, patients with sickle cell disease (SCD) are at risk of life-threatening and debilitating health events. Despite the high morbidity and mortality of this disease, hematopoietic cell transplantation (HCT), a curative treatment for SCD, remains underutilized. In the literature there is a paucity of data concerning medical decision maker (MDM) awareness of HCT as a treatment option for SCD. The objective of this study was to estimate the proportion of parents/guardians of children with SCD who are aware of HCT as a treatment option, and to identify the demographic factors associated with knowledge of this therapy’s curative potential. Between November 2015 and December 2016, 327 parents/guardians were surveyed across 4 clinical sites in 3 Midwestern US cities. Although 82% of parents/guardians had heard of HCT in the past and 78% were aware of the therapy’s curative potential, nearly half indicated that they did not know whether HCT could specifically cure their child of the disease. Respondents who had discussed HCT with their child’s physician had 5 times higher odds of being aware of HCT’s curative potential than those who had not. These findings suggest that additional efforts to enhance MDM knowledge of HCT as well as shared decision making in the use of this therapy, is warranted.
PMID: 30789459
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5. Child Neuropsychol. 2019 Feb 21:1-17. doi: 10.1080/09297049.2019.1577371. [Epub ahead of print] Social-environmental factors and cognitive and behavioral functioning in pediatric sickle cell disease. Bills SE1Schatz J1Hardy SJ2,3Reinman L1. Abstract Sickle cell disease (SCD), an inherited blood disorder that primarily affects individuals of African descent, is associated with serious medical complications as well as numerous social-environmental risk factors. These social-environmental factors are linked to long-standing social inequities, such as financial hardship and racial discrimination, both of which impact cognitive and behavioral functioning in youth. Previous research on the relationship between social-environmental risk and psychological functioning has primarily relied on non-modifiable, unidimensional measures of socioeconomic status (SES), such as income and parental education, as a proxy for social-environmental risk. The current study aimed to address the limitations associated with typical SES-type measures by comparing the unique and shared association of SES and more targeted and modifiable social-environmental factors (e.g., parent and family functioning) with specific areas of cognitive and behavioral adjustment in pediatric SCD. Seventy children ages 4-8 years old and their parents completed measures of social-environmental risk and psychological adjustment. Exploratory factor analysis indicated parent and family functioning measures were largely independent of SES. Parent and family functioning predicted phonological processing and ADHD symptoms above and beyond SES alone. In addition, the predictive ability of social-environmental risk factors appears to vary by genotype severity for measures of social functioning and math problem-solving ability. Future studies are needed to explore more specific and well-supported models of modifiable social-environmental risk and the relative impact of social-environmental risk on cognitive and behavioral functioning.
PMID: 30789071
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6. Pediatr Transplant. 2019 Feb 20:e13376. doi: 10.1111/petr.13376. [Epub ahead of print] Hematopoietic stem cell transplantation in children with sickle cell anemia: The parents’ experience. Cavadini R1,2Drain E2Bernaudin F3D’Autume C2Giannica D2Giraud F2Baubet T2Taïeb O2.   Abstract Genoidentical HSCT is currently the only curative treatment for SCA, preventing further vascular complications in high-risk children. Studies on the psychological implications of HSCT for recipient, sibling donor, and the rest of the family have been limited in SCA. This study enrolled ten families and used semi-structured interviews to explore the parents’ experience at three time points: first before transplantation, then 3 months later, and 1 year later. Three themes emerged from the results: (a) the presence of anxiety, experienced throughout the process, and alleviated by coping strategies (positive thinking, family support, praying); (b) the ability to remain parents to recipient and other family members, despite apprehension and feelings of helplessness, reinforced by the mobilization of important resources at the individual/family levels; (c) the ability to acknowledge the opportunity for their child to be cured of the disease, despite feelings of guilt toward families without a donor, or their own families back home. Overall, the parental experience with HSCT is complex, involving intra-psychic, familial, cultural, religious, and existential factors. Thus, it is important for medical teams to be cognizant of these issues in order to provide the best support to families during the HSCT process. 2019 Wiley Periodicals, Inc.
PMID: 30786109
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7. Am J Hematol. 2019 Feb 19. doi: 10.1002/ajh.25443. [Epub ahead of print] Rapid and reproducible characterization of sickling during automated deoxygenation in sickle cell disease patients. Rab MAE1,2van Oirschot BA1Bos J1Merkx TH1van Wesel ACW1Abdulmalik O3Safo MK4Versluijs AB5Houwing ME6Cnossen MH6Riedl J7Schutgens REG2Pasterkamp G1Bartels M5van Beers EJ2van Wijk R1. Abstract In sickle cell disease (SCD), sickle hemoglobin (HbS) polymerizes upon deoxygenation, resulting in sickling of red blood cells (RBCs). These sickled RBCs have strongly reduced deformability, leading to vaso-occlusive crises and chronic hemolytic anemia. To date, there are no reliable laboratory parameters or assays capable of predicting disease severity or monitoring treatment effects. We here report on the oxygenscan: a newly developed method to measure RBC deformability (expressed as Elongation Index – EI) as a function of pO2 . Upon a standardized, 22 minute, automated cycle of deoxygenation (pO2 median 16mmHg± 0.17) and reoxygenation, a number of clinically relevant parameters are produced in a highly reproducible manner (coefficients of variation <5%). In particular, physiological modulators of oxygen affinity, i.e. pH and 2,3-diphosphoglycerate showed a significant correlation (respectively R=-0.993 and R=0.980) with Point of Sickling (PoS5% ), which is defined as the pO2 where a 5% decrease in EI is observed during deoxygenation. Furthermore, in vitro treatment with anti-sickling agents, including GBT440, which alter the oxygen affinity of hemoglobin, caused a reproducible left-shift of the PoS, indicating improved deformability at lower oxygen tensions. When RBCs from 21 SCD patients were analyzed, we observed a significantly higher PoS in untreated homozygous SCD patients compared to treated patients and other genotypes. We conclude that the oxygenscan is a state-of-the-art technique that allows for rapid analysis of sickling behavior in SCD patients. The method is promising for personalized treatment, development of new treatment strategies and could have potential in prediction of complications. This article is protected by copyright. All rights reserved. This article is protected by copyright. All rights reserved.
PMID: 30784099
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8. Ann Hematol. 2019 Feb 20. doi: 10.1007/s00277-019-03635-9. [Epub ahead of print] Inhaled steroids associated with decreased macrophage markers in nonasthmatic individuals with sickle cell disease in a randomized trial. Langer AL1Leader A2Kim-Schulze S2Ginzburg Y1Merad M2Glassberg J3. Abstract Inhaled mometasone was shown to improve pain scores and decrease soluble vascular cell adhesion molecule (sVCAM) concentration in a randomized controlled trial of nonasthmatic patients with sickle cell disease. We sought to explore potential changes in systemic inflammation as a mechanism underlying this effect. Serum samples from 41 trial participants (15 placebo- and 26 mometasone-treated) were analyzed using a 92 inflammatory marker panel at baseline and after 8 weeks of mometasone therapy. Individual marker analysis and correlation analysis were conducted. Adjusted for age, the mometasone-treated group decreased the concentration of CXCL9, CXCL11, CD40, IL-10, and IL-18 relative to placebo-treated participants. Hierarchical clustering and correlation analysis identified additional evidence for a decrease in cytokines linking to macrophage signaling and migration. There was no statistically significant change in markers of asthma and allergy, indicating that the improvement was unlikely mediated by modulation of occult reactive airway disease. This analysis of inflammatory markers suggests that decrease in macrophage activity may be involved in the mediation of the clinical benefit seen with use of inhaled mometasone in nonasthmatic patients with sickle cell disease.Trial registration: clinicaltrials.gov identifier:NCT02061202.
PMID: 30783732
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10. Blood Cells Mol Dis. 2019 Feb 8. pii: S1079-9796(19)30019-1. doi: 10.1016/j.bcmd.2019.01.007. [Epub ahead of print] HemoTypeSC, a low-cost point-of-care testing device for sickle cell disease: Promises and challenges. Nnodu O1Isa H2Nwegbu M2Ohiaeri C3Adegoke S4Chianumba R2Ugwu N5Brown B6Olaniyi J6,Okocha E7Lawson J8Hassan AA9Diaku-Akinwumi I10Madu A11Ezenwosu O11Tanko Y2Kangiwa U12Girei A13Israel-Aina Y14Ladu A15Egbuzu P2Abjah U15Okolo A16Akbulut-Jeradi N17,Fernandez M17Piel FB18Adekile A19. Abstract BACKGROUND: Sickle cell disease (SCD) is a neglected burden of growing importance. >312,000 births are affected annually by sickle cell anaemia (SCA). Early interventions such as newborn screening, penicillin prophylaxis and hydroxyurea can substantially reduce the mortality and morbidity associated with SCD. Nevertheless, their implementation in African countries has been mostly limited to pilot projects. Recent development of low-cost point-of-care testing (POCT) devices for sickle haemoglobin (HbS) could greatly facilitate the diagnosis of those affected. METHODS: We conducted the first multi-centre, real-world assessment of a low-cost POCT device, HemoTypeSC, in a low-income country. Between September and November 2017, we screened 1121 babies using both HemoTypeSC and HPLC and confirmed discordant samples by molecular diagnosis. FINDINGS: We found that, in optimal field conditions, the sensitivity and specificity of the test for SCA were 93.4% and 99.9%, respectively. All 14 carriers of haemoglobin C were successfully identified. Our study reveals an overall accuracy of 99.1%, but also highlights the importance of rigorous data collection, staff training and accurate confirmatory testing. It suggests that HPLC results might not be as reliable in a resource-poor setting as usually considered. INTERPRETATION: The use of such a POCT device can be scaled up and routinely used across multiple healthcare centres in sub-Saharan Africa, which would offer great potential for the identification and management of vast numbers of individuals affected by SCD who are currently undiagnosed.
PMID: 30773433
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11. Expert Rev Hematol. 2019 Feb 16. doi: 10.1080/17474086.2019.1583554. [Epub ahead of print] Overcoming challenges of venous thromboembolism in sickle cell disease treatment. Ogunsile FJ1Naik R1Lanzkron S1. Abstract Venous thromboembolism (VTE) is a common comorbid condition found in sickle cell disease (SCD) and is associated with increased mortality for adults with SCD. The pathophysiology that leads to the thrombophilic state in SCD has been previously reviewed; however, evidence-based guidelines to aid in diagnosis, prevention and management of VTE are lacking. Areas covered: This review article will cover the pathophysiology underlying the hypercoagulable state, the epidemiology of VTE, and management strategies of VTE in SCD. Expert opinion: Providers should have a high suspicion for diagnosing VTE to help reduce morbidity and mortality in the SCD population. Unlike other thrombophilias, the risk of life-threatening anemia while being treated with anticoagulation is compounded with the potential complications surrounding red blood cell transfusions in this population (i.e. alloimunization, hyperhemolysis) and this provides another complexity to managing VTE in this population. Clinical trials evaluating the risk and benefit of treatment and treatment duration are needed.
PMID: 30773073
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12. Biol Blood Marrow Transplant. 2019 Feb 14. pii: S1083-8791(19)30136-3. doi: 10.1016/j.bbmt.2019.02.011. [Epub ahead of print] Non-myeloablative Matched Sibling Donor Hematopoietic Cell Transplantion in Children and Adolescents with Sickle Cell Disease. Guilcher GMT1Monagel DA2Nettel-Aguirre A2Truong TH3Desai SJ4Bruce A4Shah RM3Leaker MT2Lewis VA3. Abstract Sickle cell disease (SCD) is a potentially debilitating hemoglobinopathy associated with early mortality. The only established curative therapy is hematopoietic cell transplantation (HCT)with a matched sibling donor (MSD). The National Institutes of Health (NIH) nonmyeloablative regimen of alemtuzumab/300 cGy total body irradiation and prolonged sirolimus exposure for graft-versus-host disease (GVHD) prophylaxis was administered to 16 children and adolescents. Infused products were unmanipulated G-CSF mobilized peripheral blood stem cells. All patients achieved mixed donor-recipient engraftment with no cases of secondary graft failure to date. Two patients have donor myeloid chimerism in the range of 30-40%. No sickling crises post-HCT have been observed. Event-free and overall survival rates are 100% with median follow-up of 19.5 months. No cases of GVHD have been observed. Sirolimus weaning was possible in all but one eligible patient to date. Ongoing follow-up and a larger prospective clinical trial are required to determine the long-term safety and efficacy of this regimen in children. Copyright © 2019. Published by Elsevier Inc.
PMID: 30772511
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13. Transfus Med. 2019 Feb 10. doi: 10.1111/tme.12580. [Epub ahead of print] Transfusion service knowledge and immunohaematological practices related to sickle cell disease and thalassemia. Fasano RM1Branscomb J2Lane PA3Josephson CD1,3Snyder AB2Eckman JR4. Abstract OBJECTIVES: To assess current knowledge of National Heart, Lung and Blood Institutes (NHLBI) and Thalassemia International Federation (TIF) recommendations, blood banking practices and perceived challenges among transfusion services in the management of patients with haemoglobinopathies. BACKGROUND: Previous reports have demonstrated variations in transfusion practices for sickle cell disease (SCD) and thalassemia patients. Recently, NHLBI/TIF have provided transfusion recommendations for patients with haemoglobinopathies. METHODS: A cross-sectional survey was conducted of transfusion services from the state of Georgia previously identified as having SCD/thalassemia populations. The survey assessed transfusion service practices in pre-transfusion testing and blood product selection; awareness/implementation of NHLBI/TIF transfusion-based recommendations and perceived challenges in transfusing haemoglobinopathy patients. RESULTS: Responses were received from 35 of 49 (71%) institutions. Only institutions indicating transfusing SCD or thalassemia patients (32) were included in analysis. Seventy-one percent of non-sickle cell treatment centres (SCTCs) and 20% of non-thalassemia treatment centres follow NHLBI and TIF recommendations to perform a red blood cell phenotype beyond ABO/Rh(D) and provide Rh and Kell prophylactically matched units for SCD and thalassemia patients, respectively. Forty percent of institutions (33% of non-SCTCs) employ RBC genotyping to evaluate the red cell phenotype for SCD patients. Over 77% of institutions do not utilise a reliable method to identify SCD patients prior to transfusion, such as a required question/answer field on type/screen or crossmatch orders. CONCLUSION: Many healthcare systems’ transfusion practices for haemoglobinopathy patients are discordant with NHLBI/TIF recommendations. Efforts are needed to increase awareness and implementation of current recommendations among all transfusion services seeing these patients. © 2019 The Authors. Transfusion Medicine published by John Wiley & Sons Ltd on behalf of British Blood Transfusion Society.
PMID: 30740798
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14. Pediatr Blood Cancer. 2019 Feb 7:e27650. doi: 10.1002/pbc.27650. [Epub ahead of print] Hydroxyurea use in young infants with sickle cell disease. Schuchard SB1,2Lissick JR2Nickel A2Watson D2Moquist KL2Blaylark RM2Nelson SC2. Abstract BACKGROUND: Hydroxyurea (HU) reduces complications and improves quality and duration of life in sickle cell disease. Evidence supports the use of HU starting after nine months of age. PROCEDURES: We performed a retrospective study of patients starting HU at less than five years of age between January 1, 2008, and December 31, 2016. We evaluated clinical events, laboratory data, and toxicity between three different age groups: cohort 1 (0-1 year), cohort 2 (1-2 years), and cohort 3 (2-5 years). RESULTS: Sixty-five patients were included in the analysis. The mean age was 7.2 months (n = 35), 19.5 months (n = 13), and 35.5 months (n = 17) for cohorts 1, 2, and 3, respectively. Cohort 1 had higher hemoglobin (P = 0.0003) and MCV (P = 0.0199) and lower absolute reticulocyte count (P = 0.0304) at 24 months of age compared with cohort 3. The absolute neutrophil count (ANC) was lower compared with both older cohorts (P = 0.0364, 0.0025). The mean baseline hemoglobin F in cohort 1 was 31.5% compared with 19.7% and 16.5% in cohorts 2 and 3, respectively (P = 0.002, P < 0.0001). The mean duration of therapy was 31.3 months, 57.6 months (P = 0.018), and 29.1 months (P = 0.401), respectively. Mean Hb F levels remained higher in cohort 1 (29.9%) compared with cohorts 2 and 3 (20.4%, P = 0.007; 20.6%, P = 0.003). Cohort 1 experienced fewer hospitalizations (P = 0.0025), pain crises (P = 0.0618), and transfusions (P = 0.0426). There was no difference in toxicity between groups. CONCLUSION: HU is safe and effective in patients 5 to 12 months of age and generated a more robust response compared with initiation in older patients. © 2019 Wiley Periodicals, Inc.
PMID: 30729675
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15. Br J Haematol. 2019 Feb 3. doi: 10.1111/bjh.15773. [Epub ahead of print] Similar burden of type 2 diabetes among adult patients with sickle cell disease relative to African Americans in the U.S. population: a six-year population-based cohort analysis. Zhou J1Han J1,2,3Nutescu EA1Galanter WL2,4Walton SM1Gordeuk VR3Saraf SL3Calip GS1,5. Abstract Conflicting evidence exists on the epidemiology of type 2 diabetes mellitus (T2DM) among patients with sickle cell disease (SCD). This study measured the prevalence, incidence and clinical outcomes associated with T2DM in a large US population of commercially-insured adults aged ≥20 years with SCD between 2009 and 2014. Among 7070 patients with SCD, the mean age (median) was 39 (37) years and 60·8% were female. The standardized prevalence of T2DM among patients with SCD showed a modest increase, from 15·7% to 16·5% (P trend = 0·026), and was comparable to African-American respondents to the National Health and Nutrition Examination Survey (18·2%). Over 17 024 person-years, the crude incidence rate for T2DM was 25·4 per 1000 person-years. Incident T2DM was associated with comorbid hypertension (hazard ratio [HR] = 1·45, 95% confidence interval [CI] 1·14-1·83), and dyslipidaemia (HR = 1·43, 95%CI 1·04-1·96). Compared to SCD patients without T2DM, more SCD patients with T2DM had diagnoses of nephropathy (28·0% vs. 9·5%; P < 0·001), neuropathy (17·7% vs. 5·2%; P < 0·001) and stroke (24·1% vs. 9·2%; P < 0·001). Prevalence of T2DM in SCD patients is similar to the general African American population with an increasing trend in recent years. These trends support routine screening for T2DM in aging patients with SCD, especially those with comorbid hypertension and/or dyslipidaemia. © 2019 British Society for Haematology and John Wiley & Sons Ltd.
PMID: 30714090
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Sickle Cell Conferences and Events

Toronto, Canada: Sickle Cell Awareness Group of Ontario (SCAGO) hosts Sickle Cell Reception at the Legislative Assembly of Ontario from 11:30AM-1:30PM on March 27th 2019. This is part of the SCAGO’s awareness initiative to ensure policy makers have a better understanding of the disease.

If you are in the Greater Toronto Area (GTA), and would like to attend, Kindly RSVP by emailing your name, phone # and mailing address to: secretary@sicklecellanemia.ca. You may also register by completing the form in the link: www.sicklecellanemia.ca/sickle-cell-reception-queens-park/

Hemoglobinopathy Counselor Training Course will be held on April 17-18, 2019.  The two-day course, presented by the Cincinnati Comprehensive Sickle Cell Center, will be held at Cincinnati Children’s Hospital Medical Center.  The  course registration fee is $250. The deadline to register is April 1, 2019 and registration space is limited.  For more information, including a course brochure, please email: SCDEvents@cchmc.org  Registration is also available online at  

www.regonline.com/2019SCDCounselorCourse

4th Global Congress on SCD will be taking place on March 30 to April 1, 2020, in Paris, France. website link

The Foundation for Sickle Cell Disease Research will be hosting its 13th Annual Symposium June 7-9, 2019in Fort Lauderdale, FL. Registration is now open.  

Sickle Cell Disease Association of America’s 47th National Convention will be held October 9-12, 2019 in Baltimore, MD. https://www.sicklecelldisease.org/2019/01/18/47th-annual-national-convention-call-for-abstracts/

 5th Annual Sickle Cell Symposium: Saturday November 9th, 2019  At

The Speedway Club, 5555 Concord Parkway South Concord, NC 28027