Launch of the New SCInfo.org Website
The Sickle Cell Information Center website, www.scinfo.org began in 1997, shortly after internet communication took off. Founded by James R. Eckman, MD, Professor of Hematology and Medical Oncology at the Winship Cancer Institute and Professor of Medicine and Adjunct Professor of Pediatrics in Medical Genetics at Emory University School of Medicine, and Allan Platt, PA-C, MMSc, DFAAPA, Academic Coordinator and Director of Admissions for the Emory University Physician Assistant Program, https://scinfo.org has been the leading web-based resource for information about sickle cell disease for the past twenty years.
The website is widely used, both throughout the United States and around the globe, regularly averaging 13,500 hits per month, and over 150,000 per year. When Dr. Eckman retired in 2013, after three remarkable decades of work developing the Comprehensive Sickle Cell Program at Grady Health and caring for thousands of patients in the Atlanta area, Allan Platt continued to maintain www.scinfo.org.
Now, we are pleased to announce that the site has been completely reorganized with a new format, fresh images, and revised text. A collaborative interdisciplinary team of scholars drawn from the Emory Center for Digital Scholarship, the Rollins School of Public Health, and the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta (CHOA) completed this work. Both Dr. Eckman and Allan Platt have been involved in the re-design and we have taken care to leave many familiar aspects of the original website in place so that patients, families, educators, employers, and healthcare providers can access what they need with ease. Generous support for revising the website came from the University Research Committee (URC) of Emory University, from the Emory Center for Digital Scholarship (ECDS) Andrew W. Mellon Foundation Award, and from the Aflac Cancer and Blood Disorders Center. We are grateful for the opportunity made possible by this funding and proud to build on the excellent work started by Eckman and Platt so many years ago.
As always, www.scinfo.org is committed to providing sickle cell patients and their families and health care providers with professional education, news, research updates and access to up-to-date sickle cell resources worldwide. New features of the site include daily postings of new PubMed articles, links to recent articles from news outlets across the web, additional multilingual resources for patients, families and health care providers, listings of state, national and international sickle cell organizations, and an expanding list of global resources on sickle cell disease. Clinical guidelines are being up updated and will soon be available. The site maintains the privacy of all individuals and no information is gathered. The site is not supported by and does not accept advertising requests. Content and responses are reviewed by the Center Advisory Board. And finally, Information provided on this site is designed to support, not replace, the relationship that exists between a patient/site visitor and his/her physician.
In addition to thanking the institutions that supported this effort, it is important to recognize the work of Mary E. Frederickson, PhD, Clinton H. Joiner, MD, PhD, Peter A. Lane, MD, James R. Eckman, MD, Allan Platt, Allen Tullos, PhD, Wayne H. Morse, Jr., Erin Hecht, PhD, Mekdes Tsegas, Lauren McNaughton, Chase Lovellette, Adrya Stembridge. We also thank the following individuals and institutions for giving us permission to use images and materials from their collections: Oregon State University, Hertz Nazaire, Ellen Weinstein, and the Estate of Twins Seven Seven (Prince Taiwo Olaniyi Oyewale-Toyeje Oyelale Osuntoki).
We hope you enjoy the changes that have been made and the continuity that the site provides. We welcome comments and suggestions for more revisions, so let us know what you think. http://SCInfo.org
News from the website news feed
- Multiple myeloma drug could revolutionize treatment for sickle cell …
- Cure for sickle cell in adults validated — ScienceDaily
- Sickle Cell Anemia News — ScienceDaily
- Sickle Cell Disease – 5 Things to Know – NYTimes.com
- Pediatric sickle cell study stopped early due to positive results …
- Our healthcare system abandons adult sickle cell patients
Sickle What? Written by Lisa Rose
– Sickle What? arms patients and parents of newly diagnosed children with the knowledge they need to understand and manage sickle cell disease (SCD). Learn about SCD and how a person gets it in easy-to-understand terms to help parents better understand and be able to explain SCD to a child. Sickle What? shows readers how to read and understand a lab report, what action steps to take when pain occurs and the importance of asking questions and communicating openly with doctors.
To order please contact Daniel at email@example.com, or 219-922-4868.
Sickle Cell Journal
-Our Sickle Cell Journal is vital for any sickle cell patient. It allows the patient to journal about their experiences with Sickle Cell, allows the patient to journal about their medications and the different side effects. This is a vital tool for any physician or hospital since they will be able to see the history of the patient’s experience with the disease. To order please contact Daniel at firstname.lastname@example.org, or 219-922-4868.
Sickle Cell in the Medical Literature
- Exp Biol Med (Maywood). 2016 Mar 29. pii: 1535370216642048. [Epub ahead of print]
Prognostic factors and the response to hydroxurea treatment in sickle cell disease.
This review describes current considerations in the use of hydroxyurea for the management of sickle cell disease in the context of clinical severity. Randomized trials of hydroxyurea have generally enrolled subjects with increased severity based on frequent vaso-occlusive events. An exception was the BABY HUG study in infants which documented substantial benefit even for asymptomatic subjects. Increasing data indicate that hydroxyurea has a substantial effect on reducing mortality in both adults and children-perhaps the most compelling reason for advocating the drug’s widespread use. Although the efficacy of hydroxyurea is mediated primarily through increased erythrocyte fetal hemoglobin and much has been learned about the genomic influences on fetal hemoglobin levels in sickle cell disease, our ability to predict the fetal hemoglobin response to hydroxyurea remains limited; much more work in this area is indicated. The review is concluded with the recommendations of the 2014 NIH Evidence-Based Management of Sickle Cell Disease Expert Panel Report.
© 2016 by the Society for Experimental Biology and Medicine.
PMID: 27026724 [PubMed – as supplied by publisher]
- Patient. 2016 Mar 30. [Epub ahead of print]
Cognitive Testing of an Electronic Version of the Faces Pain Scale-Revised with Pediatric and Adolescent Sickle Cell Patients.
Gupta N1, Naegeli AN2, Turner-Bowker DM3, Flood EM4, Heath LE2, Mays SM5, Dampier C6.
Patient diaries and pain scales can capture the course and complications of pain managed at home in children. The Faces Pain Scale-Revised (FPS-R) is a validated scale showing reliability in children, but it has not been validated in children with sickle cell disease (SCD).
The purpose of this study was to evaluate comprehension and usability of an electronic modified version of the FPS-R among pediatric patients with SCD.
This was a cross-sectional, qualitative study involving in-person interviews with children/adolescents from the USA and their parents/legal guardians. Interviews involved cognitive debriefing and usability testing of the FPS-R.
In total, 22 children with SCD aged 4-17 years participated. Children aged 4-6 were generally unable to demonstrate clear understanding of the FPS-R and its response scale. Overall, children aged ≥7 years understood the instrument and could complete it on the electronic device, although children aged 7-8 often needed assistance from the parent. Children aged 9-17 years were able to read and complete the instrument independently. Most participants considered the electronic device easy to use.
The FPS-R was shown to be a comprehensible and usable pain measure for children aged 7-17 with SCD and to be beneficial for future clinical studies.
PMID: 27026180 [PubMed – as supplied by publisher
- Exp Biol Med (Maywood). 2016 Mar 27. pii: 1535370216642047. [Epub ahead of print]
A case-control genome-wide association study identifies genetic modifiers of fetal hemoglobin in sickle cell disease.
Liu L1, Pertsemlidis A2, Ding LH3, Story MD3, Steinberg MH4, Sebastiani P5, Hoppe C6, Ballas SK7, Pace BS8.
Sickle cell disease (SCD) is a group of inherited blood disorders that have in common a mutation in the sixth codon of the β-globin (HBB) gene on chromosome 11. However, people with the same genetic mutation display a wide range of clinical phenotypes. Fetal hemoglobin (HbF) expression is an important genetic modifier of SCD complications leading to milder symptoms and improved long-term survival. Therefore, we performed a genome-wide association study (GWAS) using a case-control experimental design in 244 African Americans with SCD to discover genetic factors associated with HbF expression. The case group consisted of subjects with HbF≥8.6% (133 samples) and control group subjects with HbF≤£3.1% (111 samples). Our GWAS results replicated SNPs previously identified in an erythroid-specific enhancer region located in the second intron of theBCL11Agene associated with HbF expression. In addition, we identified SNPs in theSPARC,GJC1,EFTUD2andJAZF1genes as novel candidates associated with HbF levels. To gain insights into mechanisms of globin gene regulation in theHBBlocus, linkage disequilibrium (LD) and haplotype analyses were conducted. We observed strong LD in the low HbF group in contrast to a loss of LD and greater number of haplotypes in the high HbF group. A search of knownHBBlocus regulatory elements identified SNPs 5′ of δ-globin located in an HbF silencing region. In particular, SNP rs4910736 created a binding site for a known transcription repressor GFi1 which is a candidate protein for further investigation. Another HbF-associated SNP, rs2855122 in the cAMP response element upstream of Gγ-globin, was analyzed for functional relevance. Studies performed with siRNA-mediated CREB binding protein (CBP) knockdown in primary erythroid cells demonstrated γ-globin activation and HbF induction, supporting a repressor role for CBP. This study identifies possible molecular determinants of HbF production.
© 2016 by the Society for Experimental Biology and Medicine.
PMID: 27022141 [PubMed – as supplied by publisher]
- Transfusion. 2016 Mar 28. doi: 10.1111/trf.13548. [Epub ahead of print]
Comparison of automated erythrocytapheresis versus manual exchange transfusion to treat cerebral macrovasculopathy in sickle cell anemia.
Koehl B1,2, Sommet J2,3,4, Holvoet L5,6, Abdoul H3, Boizeau P3, Ithier G5,6, Missud F5,6, Couque N7, Verlhac S8, Voultoury P9, Sellami F9, Baruchel A2,5, Benkerrou M4,5,6.
Chronic exchange transfusion is effective for primary and secondary prevention of stroke in children with sickle cell anemia (SCA). Erythrocytapheresis is recognized to be the most efficient approach; however, it is not widely implemented and is not suitable for all patients. The aim of our study was to compare automated exchange transfusion (AET) with our manual method of exchange transfusion and, in particular, to evaluate the efficacy, safety, and cost of our manual method.
STUDY DESIGN AND METHODS:
Thirty-nine SCA children with stroke and/or abnormal findings on transcranial Doppler were included in the study. We retrospectively analyzed 1353 exchange sessions, including 333 sessions of AET and 1020 sessions of manual exchange transfusion (MET).
Both methods were well tolerated. The median decrease in hemoglobin (Hb)S per session was 21.5% with AET and 18.8% with our manual method (p < 0.0001) with no major increase in red blood cell consumption. Iron overload was well controlled, even with the manual method, with a median (interquartile range) ferritin level of 312 (152-994) µg/L after 24 months of transfusions. The main differences in annual cost relate to equipment costs, which were 74 times higher with the automated method.
Our study shows that continuous MET has comparable efficacy to the automated method in terms of stroke prevention, decrease in HbS, and iron overload prevention. It is feasible in all hospital settings and is often combined with AET successively over time.
© 2016 AABB.
PMID: 27021622 [PubMed – as supplied by publisher]
- Hum Gene Ther. 2016 Mar 28. [Epub ahead of print]
Cell and gene therapy for the beta-thalassemias: advances and prospects.
Mansilla-Soto J1, Riviere I2, Boulad F3, Sadelain M4.
The beta-thalassemias are inherited anemias caused by mutations that severely reduce or abolish expression of the beta-globin gene. Like sickle cell disease, a related beta-globin gene disorder, they are ideal candidates for performing a genetic correction in patient hematopoietic stem cells (HSCs). The most advanced approach utilizes complex lentiviral vectors encoding the human β-globin gene, as first reported by May et al. in 2000. Considerable progress towards the clinical implementation of this approach has been made in the past five years, based on effective CD34+ cell mobilization and improved lentiviral vector manufacturing. Four trials have been initiated in the US and Europe. Of 16 evaluable subjects, 6 have achieved transfusion independence. One of them developed a durable clonal expansion, which regressed after several years without transformation. While globin lentiviral vectors have so far proven to be safe, this occurrence suggests that powerful insulators with robust enhancer-blocking activity will further enhance this approach. The combined discovery of Bcl11a-mediated γ-globin gene silencing and advances in gene editing are the foundations for another gene therapy approach, which aims to reactivate fetal hemoglobin (HbF) production. Its clinical translation will hinge on the safety and efficiency of gene targeting in true HSCs and the induction of sufficient levels of HbF to achieve transfusion independence. Altogether, the progress achieved over the past 15 years bodes well for finding a genetic cure for severe globin disorders in the next decade.
PMID: 27021486 [PubMed – as supplied by publisher]
- Br J Haematol. 2016 Mar 27. doi: 10.1111/bjh.13967. [Epub ahead of print]
Predictors of renal function progression in adults with homozygous sickle cell disease.
Asnani M1, Serjeant G2, Royal-Thomas T3, Reid M3.
Longitudinal studies of renal function may improve understanding of the pathophysiological mechanisms underlying sickle cell disease (SCD) nephropathy and may identify possible biological and clinical markers of renal function determined over time. Data from the Jamaica Sickle Cell Cohort Study (JSCCS) were extracted and the glomerular filtration rate (GFR) was estimated using the Chronic Kidney Disease Epidemiological and the SCD specific JSCCS-GFR equations from all adulthood serum creatinine measurements in homozygous SS patients. The other dataset consisted of measured GFR at two times about 13 years apart. Linear mixed model (LMM) regression analyses were conducted to determine predictors of GFR and serum creatinine over time. 191 individuals with SS disease had 867 GFR estimates available. Serum creatinine significantly increased from baseline whereas estimated GFR showed a significant decline. Serum creatinine showed positive association with increasing age, male gender, body mass index and sodium levels. Haemoglobin was a significant negative predictor of estimated GFR in age- and gender-adjusted models. A total of 24 females and 17 males had repeat measurements of their GFR. The mean annual decline in GFR was -3·2 ± 2·83 ml/min/1·73 m2 . Haemoglobin was a significant positive predictor whereas serum creatinine, systolic blood pressure and urinary albumin: creatinine ratio were negative predictors of GFR.
© 2016 John Wiley & Sons Ltd.
PMID: 27018388 [PubMed – as supplied by publisher]
- Rheumatology (Oxford). 2016 Mar 27. pii: kew042. [Epub ahead of print]
Haemoglobinopathies and the rheumatologist.
Hughes M1, Akram Q2, Rees DC3, Jones AK4.
The haemoglobinopathies are a relatively common, heterogeneous group of inherited conditions that are the result of either a quantitative abnormality (e.g. thalassaemia) or structural [e.g. sickle cell anaemia (SCA)] of the globin part of the haemoglobin molecule. Musculoskeletal (MSK) complications are common in patients with haemoglobinopathies and may affect the whole of the MSK system, in addition to bone, which is the primary site of the disease. Typical MSK complications include painful vaso-occlusive disease and osteomyelitis in SCA and reduced BMD in thalassaemia. Patients may also develop a number of related (e.g. gout) or unrelated rheumatic diseases (e.g. inflammatory arthritis and autoimmune CTDs). Treatment of MSK conditions in patients with haemoglobinopathies may be challenging (e.g. bone marrow suppression from disease-modifying agents) and in particular in SCA, steroid therapy (by any route) may precipitate potentially severe vaso-occlusive complications. Rheumatologists need to be aware of the range of MSK complications, treatment challenges and the need for such patients to be managed as part of a dedicated multidisciplinary team alongside haematology.
© The Author 2016. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For Permissions, please email: email@example.com.
PMID: 27018056 [PubMed – as supplied by publisher]
- Biol Blood Marrow Transplant. 2016 Mar 22. pii: S1083-8791(16)00152-X. doi: 10.1016/j.bbmt.2016.03.003. [Epub ahead of print]
Allogeneic stem cell transplantation in congenital hemoglobinopathies using tailored busulphan-based conditioning regimen: single center experience.
Zaidman I1, Rowe JM2, Khalil A1, Ben-Arush M3, Elhasid R4.
Hematopoietic stem cell transplantation (HSCT) is the only proven curative option for patients with hemoglobinopathies, both thalassemia and sickle cell anemia (SCA). Busulphan-based myeloablative conditioning regimen is the standard of care for HSCT in these patients, although increased treatment-related morbidity, including veno-occlusive disease (VOD), has been demonstrated.
PATIENTS AND METHODS:
A total of 38 pediatric patients, median age was 8 years (range, 6 months-22 years), suffering from hemoglobinopathy were treated at Rambam Medical Center in Haifa, Israel, between 1998 and 2011. Thirty four patients had thalassemia major and four had SCA. The 38 patients underwent 40 HSCT, 34 of which were first transplants and six second transplants. Most of the transplants (32/40) were from matched sibling donors. Sources of stem cells were peripheral blood in 30 transplants, bone marrow in seven transplants, and cord blood in three transplants. All received different customized busulphan-based conditioning regimens tailored by pharmacokinetic analysis of busulphan levels.
Primary engraftment occurred in 37/40 transplants. Neutrophil engraftment (>0.5×109/L) occurred at a median of 15.3 days post-transplantation (range, 10-45 days). Platelet transfusion independence (>20×109/L) occurred at a median of 22.3 days (range, 11-60 days). Five-year overall survival for thalassemia patients after first transplantation was 90.5±5.3%. Five-year thalassemia free survival was 81.7±6.8%. Cumulative incidence of acute GVHD was 17.6%. Rate of grade 3-4 GVHD was 8.8%. Cumulative incidence of chronic GVHD was 23.5%, with 11.8% incidence of extensive chronic GVHD. One patient developed VOD. Full donor chimerism occurred in 36.4% of patients with class I+II thalassemia, compared to 78.6% in class III thalassemia (p=0.049).
Overall survival above 90% in patients undergoing their first transplant was demonstrated using busulphan-based conditioning regimens. The low incidence of VOD was probably due to busulphan area under the curve measurements and dose adjustment.
Copyright © 2016 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
PMID: 27016193 [PubMed – as supplied by publisher]
- Exp Biol Med (Maywood). 2016 Mar 23. pii: 1535370216640385. [Epub ahead of print]
Clinical severity in sickle cell disease: the challenges of definition and prognostication.
Sickle cell disease (SCD) is a monogenic, yet highly phenotypically variable disease with multisystem pathology. This manuscript provides an overview of many of the known determinants, modifiers, and correlates of disease severity in SCD. Despite this wealth of data, modeling the variable and multisystem pathology of SCD continues to be difficult. The current status of prediction of specific adverse outcomes and global disease severity in SCD is also reviewed, highlighting recent successes and ongoing challenges.
© 2016 by the Society for Experimental Biology and Medicine.
PMID: 27013545 [PubMed – as supplied by publisher]
- Semin Hematol. 2016 Apr;53(2):120-8. doi: 10.1053/j.seminhematol.2016.01.001. Epub 2016 Jan 15.
Alternative donor allogeneic hematopoietic cell transplantation for hemoglobinopathies.
Alfraih F1, Aljurf M2, Fitzhugh CD3, Kassim AA3.
Allogeneic hematopoietic stem cell transplantation (HSCT) offers a curative therapy for patients with hemoglobinopathies, mainly severe sickle cell disease (SCD) and thalassemia (TM). However, the applicability of HSCT has been limited mainly by donor availability, with a less than 25%-30% of eligible patients having human leukocyte antigen (HLA)-matched sibling donors. Previous outcomes using alternate donor options have been markedly inferior due to increased regimen-related toxicity, transplant-related mortality, graft failure, and graft-versus-host disease (GVHD). Advances in transplant technology, including high-resolution HLA typing, improved GVHD prophylactic approaches with tolerance induction, and better supportive care over the last decade, are addressing these historical challenges, resulting in increasing donor options. Herein, we review alternate donor HSCT approaches for severe SCD and TM using unrelated donors, umbilical cord blood units, or related haploidentical donors. Though this is an emerging field, early results are promising and in selected patients, this may be the preferred option to mitigate against the age-related morbidity and early mortality associated with these disorders.
Copyright © 2016 Elsevier Inc. All rights reserved.
PMID: 27000737 [PubMed – in process]
- Clin Trials. 2016 Mar 21. pii: 1740774516636573. [Epub ahead of print]
Addressing challenges of clinical trials in acute pain: The Pain Management of Vaso-occlusive Crisis in Children and Young Adults with Sickle Cell Disease Study.
Nottage KA1, Hankins JS2, Faughnan LG3, James DM2, Richardson J4, Christensen R4, Kang G5, Smeltzer M5, Cancio MI2, Wang WC2, Anghelescu DL6.
Neuropathic pain is a known component of vaso-occlusive pain in sickle cell disease; however, drugs targeting neuropathic pain have not been studied in this population. Trials of acute pain are complicated by the need to obtain consent, to randomize participants expeditiously while optimally treating pain. We describe the challenges in designing and implementing the Pain Management of Vaso-occlusive Crisis in Children and Young Adults with Sickle Cell Disease Study (NCT01954927), a phase II, randomized, double-blind, placebo-controlled trial to determine the effect of gabapentin for vaso-occlusive crisis.
In the Pain Management of Vaso-occlusive Crisis in Children and Young Adults with Sickle Cell Disease Study, we aim to assess the analgesic effect of gabapentin during vaso-occlusive crisis. Difficulties we identified included avoiding delay of notification of study staff of potential participants which we resolved by automated notification. Concern for rapid randomization and drug dispensation was addressed through careful planning with an investigational pharmacy and a single liquid formulation. We considered obtaining consent during well-visits to avoid the time constraints with acute presentations, but the large number of patients and limited duration that consent is valid made this impractical.
In all, 79% of caregivers/children approached have agreed to participate. The trial is currently active, and enrollment is at 45.8% of that targeted (76 of 166) and expected to continue for two more years. Maintaining staff availability after-hours remains problematic, with 8% of screened patients missed for lack of available staff.
Lessons learned in designing a trial to expedite procedures in the acute pain setting include (1) building study evaluations upon a standard-of-care backbone; (2) implementing a simple study design to facilitate consent and data capture; (3) assuring ample, well-trained study staff; and (4) utilizing technology to automate procedures whenever possible.
This study design has circumvented many of the logistical barriers usually associated with acute pain trials and may serve as a prototype for future studies.
© The Author(s) 2016.
PMID: 27000103 [PubMed – as supplied by publisher]
- Am J Public Health. 2016 Mar 21:e1-e3. [Epub ahead of print]
CAREST-Multilingual Regional Integration for Health Promotion and Research on Sickle Cell Disease and Thalassemia.
Knight-Madden J1, Romana M1, Villaescusa R1, Reid M1, Etienne-Julan M1, Boutin L1, Elana G1, Elenga N1, Wheeler G1, Lee K1, Nieves R1, Jones Lecointe A1, Lalanne-Mistrih ML1, Loko G1, Keclard-Christophe L1, Hardy-Dessources MD1.
Sickle cell disease (SCD) is a significant problem in the Caribbean, where many individuals have African and Asian forebears. However, reliable prevalence data and specific health care programs for SCD are often missing in this region. Closer collaboration between Caribbean territories initiated in 2006 to set up strategies to promote better equity in the health care system for SCD patients led to the formation of CAREST: the Caribbean Network of Researchers on Sickle Cell Disease and Thalassemia. We present the effectiveness of collaborations established by CAREST to promote SCD newborn screening programs and early childhood care, to facilitate health worker training and approaches for prevention and treatment of SCD complications, and to carry out inter-Caribbean research studies. (Am J Public Health. Published online ahead of print March 17, 2016; e1-e3. doi:10.2105/AJPH.2016.303078).
PMID: 26999505 [PubMed – as supplied by publisher]
- Transfusion. 2016 Mar 20. doi: 10.1111/trf.13558. [Epub ahead of print]
Effectiveness of red blood cell exchange, partial manual exchange, and simple transfusion concurrently with iron chelation therapy in reducing iron overload in chronically transfused sickle cell anemia patients.
Fasano RM1,2, Leong T3, Kaushal M4, Sagiv E4, Luban NL4,5, Meier ER4,5.
Chronic transfusion therapy (CTT) is indicated for stroke prevention in children with sickle cell anemia (SCA) and is complicated by iron overload and alloimmunization. CTT is performed by simple transfusion (ST), partial manual exchange (PME), or erythrocytapheresis (RCE). Although small case series have demonstrated RCE in combination with iron chelation therapy stabilizes and/or decreases ferritin, there are no reports comparing the effect of ST, PME, and RCE on liver iron concentration (LIC). CTT modality effect on serum ferritin and LIC were compared in SCA patients on iron chelation, with hemoglobin (Hb)S goal of 30%.
STUDY DESIGN AND METHODS:
Medical records of SCA patients on CTT and deferasirox (≥25 mg/kg/day) were retrospectively reviewed. Mean HbS%, change in ferritin and LIC, and alloimmunization rate were determined for each CTT group.
Twenty-eight patients were included; six crossed over (one from ST to PME, one from ST to PME then to RCE, three from ST to RCE, and one from PME to RCE) to include 36 transfusion modality intervals. Median pretransfusion HbS% levels were 32.7% (ST), 36.2% (PME), and 34.7% (RCE; p = 0.732). Median ferritin changes were +15 (-17 to +45), +38 (+24 to +105), and -91 (-141 to -48) ng/mL/month (p = 0.003), and median LIC changes (available in 22 patient transfusion modality intervals) were +1.3 (-1.6 to +4.3), +2.3 (-6.5 to +8.9), and -5.7 (-10.7 to -0.5) mg/g/year (p = 0.024) in ST, PME, and RCE, respectively. There was no significant difference in alloimmunization rate between ST/PME and RCE groups.
We recommend RCE plus chelation as an effective method for reducing iron overload, while maintaining HbS at 30% to 35%.
© 2016 AABB.
PMID: 26997031 [PubMed – as supplied by publisher]
- Indian J Med Res. 2016 Jan;143(1):21-4. doi: 10.4103/0971-5916.178582.
Sickle cell disease in India: A perspective.
Serjeant GR1, Ghosh K, Patel J.
PMID: 26997009 [PubMed – in process]
- Exp Biol Med (Maywood). 2016 Mar 17. pii: 1535370216639737. [Epub ahead of print]
Use of hydroxyurea and phlebotomy in pediatric patients with hemoglobin sickle cell disease.
- 1Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Hydroxyurea is an excellent therapeutic agent for the pharmacological induction of HbF in patients with sickle cell disease (SCD). However, all completed clinical trials of hydroxyurea have excluded patients with hemoglobin SC (HbSC) disease. HbSC differs significantly in pathophysiology from HbSS, as HbC does not sickle, but instead causes cellular dehydration which potentiates sickling of HbS. Many severely affected HbSC patients have been placed on hydroxyurea on a case by case basis, but there are no large scale prospective data on safety or efficacy of hydroxyurea in this subset of patients with SCD. Here, we report a case series of 14 pediatric patients with HbSC treated to maximum tolerated dose (MTD) with hydroxyurea. Those who failed to show clinical improvement after at least six months at MTD were offered phlebotomy in addition to hydroxyurea. Five out of 11 patients with HbSC who achieved MTD failed to demonstrate clinical improvement on hydroxyurea. Of the four placed on dual hydroxyurea and phlebotomy therapy, all showed at least partial clinical improvement. Percent dense red blood cells (%DRBC) were measured via an ADVIA hematology analyzer. A marked rise in percent dense cells preceded clinical complications in three patients. Dual therapy with hydroxyurea and phlebotomy may be an effective approach to patients with HbSC that do not experience improvement with hydroxyurea alone. Monitoring of %DRBC may predict adverse events and aid in assessing hydroxyurea compliance. Large scale clinical trials are needed to evaluate the safety and efficacy of hydroxyurea and hydroxyurea with phlebotomy in patients with HbSC disease.
© 2016 by the Society for Experimental Biology and Medicine.
PMID: 26993671 [PubMed – as supplied by publisher]
- Br J Haematol. 2016 Mar 15. doi: 10.1111/bjh.14023. [Epub ahead of print]
A systematic review of the association between depression and health care utilization in children and adults with sickle cell disease.
Jonassaint CR1, Jones VL2, Leong S2, Frierson GM3.
Patients with sickle cell disease (SCD) experience a disproportionately high use of health care resources. Several studies have examined depression and other negative mood states as risk factors for increased health care utilization; however, there have been no systematic reviews examining and summarizing this evidence in SCD. The aim of this systematic review, therefore, was to determine whether depression or depressive symptoms are associated with health care utilization among children and adults with SCD. We followed a quantitative systematic review protocol based on the Preferred Reporting Items for Systematic Reviews and Meta- Analyses guidelines and performed a literature search of records from January 1980 to April 2014 using six databases. Empirical studies were eligible if the sample was primarily composed of patients with SCD and included data on depression, mood disorder diagnosis or depressive symptoms and health care utilization. We included 12 studies involving 54 036 unique participants. The prevalence estimates for depression ranged from 2-57%. Seven studies found a significant, or marginally significant, association between depression and utilization while five did not. Patients reporting depression had an estimated 2·8 times greater relative risk of being a high utilizer, and 2·9 versus 1·8 hospitalizations per year on average compared to patients without depression. Overall, depressive symptoms are common in SCD and may increase risk for poor outcomes including health care utilization. The available studies on depression in SCD, however, are limited by small sample sizes, retrospective designs or short follow-up. This systematic review found a modest association between depression and health care utilization in SCD.
© 2016 John Wiley & Sons Ltd.
PMID: 26991317 [PubMed – as supplied by publisher]
Sickle Cell Conferences and Events
Hemoglobinopathy Counselor Training Course will be held on April 7-8, 2016. 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 $150. The deadline to register is March 24, 2016 and registration is limited. For more information, including a course brochure, please email: SCDEvents@cchmc.org Registration is also available online at www.regonline.com/2016scdcounselorcourse
Conference Name: Integrating Care in Sickle Cell Disease from Pediatrics to Adulthood
Conference Date: Saturday, May 21, 2016
Time: 7:30 am – 4:30pm
Location: St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38104
Register at: https://www.stjude.org/education-training/advanced-training/seminars-symposia/sickle-cell-disease-conference.html
Target Audience: This conference is designed for any practitioner providing treatment to people with sickle cell disease: hematologists, internal medicine physicians, nurses, nurse practitioners, physician assistants, residents, fellows, emergency room physicians, psychologists, and social workers. Adults with sickle cell disease and their significant others are also invited to the conference. CME and Nursing credits will be offered.
Event: Sickle Cell in Focus (SCiF) 2016, National Institutes of Health, Bethesda, Maryland, USA
Date: Thursday 2nd – Friday 3rd June 2016
Venue: Natcher Conference Centre, National Institutes of Health, Bethesda, MD 20894 USA
The 10th Sickle Cell in Focus conference returns to the USA in June 2016. Sickle Cell in Focus is an internationally renowned educational update for sickle cell disease. It attracts a wide audience of clinicians, academics and other healthcare professionals involved in the disease from around the world.
To book: Free registration will be opening soob. If you would like to be kept up-to-date, please join the STSTN mailing list by sending an email to: firstname.lastname@example.org
Contact details: email@example.com / @STSTNetwork / www.ststn.co.uk
June 18, 2016 Sickle & Flow – Atlanta GA http://www.sickleandflow.org/about
Sickle & Flow is a partnership with members of the Atlanta community, including local hip hop producers, DJs, patient advocates, health care workers, and scientists. We’re hosting a sickle cell awareness event as part of World Blood Day, on Saturday June 18, 2016. We want to engage the ATL community to promote patient advocacy and biomedical research. As a part of the evening, we will host a Be the Match event, collecting cheek swabs from potential donors, to partner with us in our efforts to more effectively treat and cure sickle cell disease.
6th International African Symposium on Sickle Cell Disease July 11th – 15th, 2016 Accra, Ghana
Call for Abstracts
Abstract submissions will be accepted for the following categories:
- Laboratory Diagnosis
- Clinical Management
- Public Health and Health Education
- Psychosocial Interventions
- Blood Transfusion Services
- Newborn Screening and Follow Up
- New Therapies
More information regarding submission guidelines and deadlines will be available shortly. For more information, please email SickleCellSymposium@email.chop.edu
The Comprehensive Sickle Cell Center at The Children’s Hospital of Philadelphia, the Sickle Cell Foundation of Ghana, and other international partners have been organizing a series of international symposia aimed at improving knowledge and practices in the management of SCD in Africa.
The target audience for this symposium are healthcare workers, public health officials, laboratory technologists, community-based organizations, and support groups for patients and families.
Sickle Cell Partners of the Carolinas presents, Sickle Cell Disease, “Let’s talk about it” day conference www.sicklecellpartnersofthecarolinas.org
Saturday September 10, 2016 9 am to 2 pm
Friendship Missionary Baptist Church Conference Center
3400 Beatties Ford Road, Charlotte NC 28216
Calling All Abstracts! Submit your abstracts for the 44th SCDAA annual convention Baltimore, MD on September 27-October 1st, 2016
Interested in highlighting your work as a community based member organization, physician, nurse, social worker, or other team working on behalf of people with sickle cell disease and their families? Submit an abstract on the work that you or your team has completed or is in the process of completing for an opportunity to share at the 44th Annual Sickle Cell Convention. This year’s convention will be held in the great city of Baltimore, MD on September 27-October 1st, 2016 at the Hyatt Regency hotel. For more information on abstracts or to submit your application clicking HERE. http://www.sicklecelldisease.org/index.cfm?page=convention-abstract-submission
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