2,612 results match your criteria Pediatrics Sickle Cell Disease


Oral morphine protocol evaluation for the treatment of vaso-occlusive crisis in paediatric sickle cell patients.

Paediatr Child Health 2019 Feb 31;24(1):e45-e50. Epub 2018 May 31.

Division of Pediatric Emergency Medicine, CHU Ste-Justine, Montréal, Quebec.

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. Read More

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http://dx.doi.org/10.1093/pch/pxy074DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6376293PMC
February 2019

Hematopoietic stem cell transplantation in children with sickle cell anemia: The parents' experience.

Pediatr Transplant 2019 Feb 20:e13376. Epub 2019 Feb 20.

Department of Psychopathology, Avicenne Hospital, Paris XIII University & Assistance Publique-Hôpitaux de Paris, Bobigny, France.

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. Read More

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http://dx.doi.org/10.1111/petr.13376DOI Listing
February 2019

"A complex interface: Exploring sickle cell disease from a parent's perspective, after moving from Sub-Saharan Africa to North America".

Pediatr Hematol Oncol 2019 Feb 20:1-12. Epub 2019 Feb 20.

b Stollery Children's Hospital , Alberta Health Services , Edmonton , AB , Canada.

Introduction: Sickle cell disease (SCD) is an inherited, multi-system, chronic disease with the highest prevalence affecting people of Sub-Saharan African descent. While major advances in SCD care have occurred over the last few decades in many African countries these advances are not readily available. Prior literature from Ghana and Kenya describe stigma, despair, and economic burden as well as hope when a child has SCD. Read More

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http://dx.doi.org/10.1080/08880018.2018.1541949DOI Listing
February 2019

HemoTypeSC, a low-cost point-of-care testing device for sickle cell disease: Promises and challenges.

Blood Cells Mol Dis 2019 Feb 8. Epub 2019 Feb 8.

Department of Paediatrics, Kuwait University, Kuwait.

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. Read More

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http://dx.doi.org/10.1016/j.bcmd.2019.01.007DOI Listing
February 2019

Non-myeloablative Matched Sibling Donor Hematopoietic Cell Transplantion in Children and Adolescents with Sickle Cell Disease.

Biol Blood Marrow Transplant 2019 Feb 14. Epub 2019 Feb 14.

Section of Oncology/BMT, Departments of Oncology and Pediatrics, Alberta Children's Hospital, University of Calgary, Calgary, Canada.

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. Read More

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http://dx.doi.org/10.1016/j.bbmt.2019.02.011DOI Listing
February 2019

Concurrent homozygous sickle-cell disease and severe haemophilia A: Thromboelastography profiles.

Haemophilia 2019 Feb 14. Epub 2019 Feb 14.

Carman and Ann Adams Department of Pediatrics, Children's Hospital of Michigan, Wayne State University School of Medicine, Detroit, Michigan.

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http://dx.doi.org/10.1111/hae.13692DOI Listing
February 2019

Red blood cell alloimmunization and minor red blood cell antigen phenotypes in transfused Ghanaian patients with sickle cell disease.

Transfusion 2019 Feb 13. Epub 2019 Feb 13.

Department of Experimental Immunohematology, Sanquin, Amsterdam, Netherlands.

Background: The routine pretransfusion investigations in Southern Ghana involve only ABO-D blood group typing and ABO compatibility testing without screening for irregular red blood cell (RBC) antibodies. The prevalence and specificities of RBC antibodies and frequencies of most minor blood group antigens in transfused patients with sickle cell disease (SCD) in Ghana are not known and are the objectives of this study.

Study Design And Methods: This was a cross-sectional study that investigated transfused patients with SCD for the presence of irregular RBC antibodies and Rhesus, Kell, Duffy, Kidd, and Ss antigens. Read More

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http://dx.doi.org/10.1111/trf.15197DOI Listing
February 2019
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The use of bevacizumab in pediatric retinal and choroidal disease: A review.

Eur J Ophthalmol 2019 Feb 13:1120672119827773. Epub 2019 Feb 13.

3 Department of Ophthalmology, Icahn School of Medicine, Mount Sinai Medical Center and New York City Health and Hospitals, New York, NY, USA.

The use of intravitreal bevacizumab in pediatric retinal and uveitic disease has become more widespread over the past decade. This article serves to outline the rationale underlying the use of intravitreal bevacizumab, and which disease entities it should be appropriately thought of as a primary or solo therapy, as opposed to an adjuvant one. Also presented is the relevant literature regarding each of these retinopathies. Read More

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http://dx.doi.org/10.1177/1120672119827773DOI Listing
February 2019

Endocrine and metabolic complications in children and adolescents with Sickle Cell Disease: an Italian cohort study.

BMC Pediatr 2019 Feb 11;19(1):56. Epub 2019 Feb 11.

Post Graduate School of Pediatrics, Department of Medical and Surgical Sciences for Mothers, Children and Adults, University of Modena and Reggio Emilia, Via del Pozzo 71, 41124, Modena, Italy.

Background: Children with Sickle Cell Disease (SCD) show endocrine complications and metabolic alterations. The physiopathology of these conditions is not completely understood: iron overload due to chronic transfusions, ischemic damage, and inflammatory state related to vaso-occlusive crises may be involved. Aims of this study were to evaluate the growth pattern, endocrine complications, and metabolic alterations and to detect the relationship between these conditions and the SCD severity in affected children and adolescents. Read More

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http://dx.doi.org/10.1186/s12887-019-1423-9DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6371531PMC
February 2019
1 Read

Circulating Microparticles in Children With Sickle Cell Anemia in a Tertiary Center in Upper Egypt.

Clin Appl Thromb Hemost 2019 Jan-Dec;25:1076029619828839

6 Department of Community Health Nursing, Faculty of Nursing, Assiut University, Assiut, Egypt.

Sickle cell disease (SCD) is a genetically inherited hemolytic anemia increasingly appreciated as a chronic inflammatory condition and hypercoagulable state with high thrombotic risk. It is associated with disturbed immune phenotype and function and circulating microparticles (MPs) derived from multiple cell sources. This study was carried out to determine MPs profiles in patients with sickle cell anemia (either on hydroxyurea (HU) therapy or those with no disease-modifying therapy) and to compare these profiles with healthy children. Read More

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http://dx.doi.org/10.1177/1076029619828839DOI Listing
February 2019
1 Read
1.575 Impact Factor

Low-risk factors for severe bacterial infection and acute chest syndrome in children with sickle cell disease.

Pediatr Blood Cancer 2019 Feb 10:e27667. Epub 2019 Feb 10.

Pediatric Hematology and Oncology Unit, Department of Pediatrics, Hospital General Universitario Gregorio Marañón, Madrid, Spain.

Introduction: The rate of bacterial infections in children with sickle cell disease (SCD) has decreased in recent years, mainly due to penicillin prophylaxis and vaccination.

Objectives: To determine the rate of severe bacterial infection (SBI) in a cohort of children with SCD and to describe low-risk factors for confirmed SBI (CSBI) and acute chest syndrome (ACS).

Methods: This 11-year retrospective cohort study included children with febrile SCD admitted to a reference hospital in Spain. Read More

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http://dx.doi.org/10.1002/pbc.27667DOI Listing
February 2019
1 Read

Transfusion service knowledge and immunohaematological practices related to sickle cell disease and thalassemia.

Transfus Med 2019 Feb 10. Epub 2019 Feb 10.

Department of Hematology & Oncology, Emory University School of Medicine, Atlanta, Georgia, USA.

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. Read More

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http://dx.doi.org/10.1111/tme.12580DOI Listing
February 2019
1 Read

Sonographic Diagnosis and Clinical Correlates of Gallbladder Stones in Patients with Sickle Cell Disease in Calabar, Nigeria.

Open Access Maced J Med Sci 2019 Jan 12;7(1):68-72. Epub 2019 Jan 12.

Department of Paediatrics, University of Calabar and University of Calabar Teaching Hospital, Calabar, Nigeria.

Background: Sickle Cell Disease (SCD) causes chronic haemolysis which is a risk factor for cholelithiasis.

Aim: To determine the prevalence and clinical correlates of cholelithiasis in SCD patients in steady state treated at the University of Calabar Teaching Hospital.

Methods: This was a prospective study that took place at the Haematology and sickle cell disease clinics, University of Calabar Teaching Hospital, Calabar, Nigeria between January and June 2018. Read More

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http://dx.doi.org/10.3889/oamjms.2019.015DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6352481PMC
January 2019
1 Read

BMI percentile is an independent predictor of increase in lung function in children with sickle cell anemia.

Am J Hematol 2019 Feb 7. Epub 2019 Feb 7.

Department of Pediatrics, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, Nashville, Tennessee.

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http://dx.doi.org/10.1002/ajh.25433DOI Listing
February 2019
1 Read

Results of a multicenter universal newborn screening program for sickle cell disease in Italy: A call to action.

Pediatr Blood Cancer 2019 Feb 5:e27657. Epub 2019 Feb 5.

Clinic of Pediatric Hematology Oncology, Department of Woman's and Child Health, Azienda Ospedaliera-Università di Padova, Padova, Italy.

Background: Sickle cell disease (SCD) is a chronic multisystem disorder requiring comprehensive care that includes newborn screening (NBS) as the first step of care. Italy still lacks a national SCD NBS program and policy on blood disorders. Pilot single-center screening programs and a regional targeted screening have been implemented so far, but more evidence is needed in order to impact health policies. Read More

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http://dx.doi.org/10.1002/pbc.27657DOI Listing
February 2019
2 Reads
2.562 Impact Factor

Incidence of invasive Haemophilus influenzae infections in children with sickle cell disease.

Pediatr Blood Cancer 2019 Feb 5:e27642. Epub 2019 Feb 5.

Department of Pediatrics, Division of Infectious Diseases, Emory University, Atlanta, Georgia.

Background: Children with sickle cell disease (SCD) are at increased risk for invasive infection with encapsulated bacteria. Antibiotic prophylaxis and immunizations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) have decreased the overall incidence of invasive infections and have shifted distribution of serotypes causing disease toward those not covered by immunizations. We sought to determine the current incidence of invasive H. Read More

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http://dx.doi.org/10.1002/pbc.27642DOI Listing
February 2019

Emotion-Focused Avoidance Coping Mediates the Association Between Pain and Health-Related Quality of Life in Children With Sickle Cell Disease.

J Pediatr Hematol Oncol 2019 Feb 1. Epub 2019 Feb 1.

Department of Psychology, Georgia State University, Atlanta, GA.

Sickle cell disease (SCD) is associated with pain and decreased health-related quality of life (HRQOL). Coping strategies influence pain but have not been evaluated as mediating the relation between pain and HRQOL in pediatric SCD. The current study examined whether pain-related coping mediates the association between pain and HRQOL in children and adolescents with SCD. Read More

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http://dx.doi.org/10.1097/MPH.0000000000001429DOI Listing
February 2019

TALEN-Mediated Gene Editing of in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction.

Mol Ther Methods Clin Dev 2019 Mar 31;12:175-183. Epub 2018 Dec 31.

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Elements within the γ-hemoglobin promoters ( and ) function to bind transcription complexes that mediate repression of fetal hemoglobin expression. Sickle cell disease (SCD) subjects with a 13-bp deletion in the promoter exhibit a clinically favorable hereditary persistence of fetal hemoglobin (HPFH) phenotype. We developed TALENs targeting the homologous promoters to de-repress fetal hemoglobin. Read More

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http://dx.doi.org/10.1016/j.omtm.2018.12.008DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6348980PMC
March 2019
1 Read

Patients with sickle-cell disease exhibit greater functional connectivity and centrality in the locus coeruleus compared to anemic controls.

Neuroimage Clin 2019 Jan 22;21:101686. Epub 2019 Jan 22.

Center for Neurobiology of Stress and Resilience, Department of Medicine, Vatche and Tamar Division of Digestive Diseases, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.

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http://dx.doi.org/10.1016/j.nicl.2019.101686DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6356008PMC
January 2019
2 Reads

Use of the new pediatric PROMIS measures of pain and physical experiences for children with sickle cell disease.

Pediatr Blood Cancer 2019 Jan 28:e27633. Epub 2019 Jan 28.

Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin.

Background: There are new pediatric domains to measure patients' pain and physical experiences in the Patient-Reported Outcomes Measurement Information System (PROMIS). The objective of this study was to establish the psychometric properties of these domains for children with sickle cell disease (SCD).

Procedure: We conducted a cross-sectional analysis of PROMIS assessments of children with SCD recruited from a pediatric tertiary care clinic. Read More

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http://dx.doi.org/10.1002/pbc.27633DOI Listing
January 2019

Mechanisms of NRF2 activation to mediate fetal hemoglobin induction and protection against oxidative stress in sickle cell disease.

Exp Biol Med (Maywood) 2019 Jan 23:1535370219825859. Epub 2019 Jan 23.

1 Department of Pediatrics, Augusta University, Augusta, GA 30912, USA.

Impact Statement: Sickle cell disease (SCD) is a group of inherited blood disorders caused by mutations in the human β-globin gene, leading to the synthesis of abnormal hemoglobin S, chronic hemolysis, and oxidative stress. Inhibition of hemoglobin S polymerization by fetal hemoglobin holds the greatest promise for treating SCD. The transcription factor NRF2, is the master regulator of the cellular oxidative stress response and activator of fetal hemoglobin expression. Read More

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http://dx.doi.org/10.1177/1535370219825859DOI Listing
January 2019
1 Read

Surfactant protein D as a marker for pulmonary complications in pediatric patients with sickle cell disease: Relation to lung function tests.

Pediatr Pulmonol 2019 Jan 22. Epub 2019 Jan 22.

Faculty of Medicine, Pediatrics Department, Ain Shams University, Cairo, Egypt.

Background: Surfactant protein D (SP-D) is considered a candidate biomarker for lung integrity and for disease progression.

Aim: We determined the level of SP-D in children and adolescents with SCD and assessed its possible relation to pulmonary complications and lung function.

Methods: Serum SP-D levels were assessed in 50 SCD patients compared with 30 healthy controls. Read More

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http://dx.doi.org/10.1002/ppul.24257DOI Listing
January 2019
4 Reads

Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia.

JAMA 2019 01;321(3):266-276

Department of Statistics, Saint-Louis Hospital, ECSTRA Team, UMR1153, INSERM, University Paris-Diderot, Paris, France.

Importance: In children with sickle cell anemia (SCA), high transcranial Doppler (TCD) velocities are associated with stroke risk, which is reduced by chronic transfusion. Whether matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) can reduce velocities in patients with SCA is unknown.

Objective: To determine the association of MSD-HSCT with TCD velocities as a surrogate for the occurrence of ischemic stroke in children with SCA. Read More

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http://dx.doi.org/10.1001/jama.2018.20059DOI Listing
January 2019
13 Reads

Matched Sibling Donor Hematopoietic Stem Cell Transplantation to Prevent Stroke in Children With Sickle Cell Anemia.

JAMA 2019 01;321(3):251-252

Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

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http://dx.doi.org/10.1001/jama.2018.20390DOI Listing
January 2019
1 Read

Serum zinc level during and after acute painful episodes in children with sickle cell anemia at the aminu kano teaching hospital, Kano, Northern Nigeria.

Niger J Clin Pract 2019 Jan;22(1):16-23

Department of Pediatrics, Bayero University/Aminu Kano Teaching Hospital, Kano, Nigeria.

Background: Acute painful crisis due to vaso-occlusive event is the leading cause of hospitalization in patients with sickle cell anemia (SCA). Zinc deficiency in children with SCA is associated with increased frequency and severity of acute painful events. We determined serum zinc level in children with SCA during acute painful crisis and compared the same with children with SCA who are in steady state and healthy non-sickle cell disease children. Read More

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http://dx.doi.org/10.4103/njcp.njcp_169_18DOI Listing
January 2019
2 Reads

"Maximum tolerated dose" vs "fixed low-dose" hydroxyurea for treatment of adults with sickle cell anemia.

Am J Hematol 2019 Jan 21. Epub 2019 Jan 21.

Division of Hematology & Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, Illinois.

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http://dx.doi.org/10.1002/ajh.25412DOI Listing
January 2019
4 Reads

Advances in Understanding Ischemic Stroke Physiology and the Impact of Vasculopathy in Children With Sickle Cell Disease.

Stroke 2019 Feb;50(2):266-273

Department of Pediatrics and Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas (M.M.D.).

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http://dx.doi.org/10.1161/STROKEAHA.118.020482DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6385587PMC
February 2019
2 Reads

Risk factors for recurrent severe anemia among previously transfused children in Uganda: an age-matched case-control study.

BMC Pediatr 2019 Jan 18;19(1):27. Epub 2019 Jan 18.

Department of Global Child Health, Emma Children's Hospital, Academic Medical Centre, University of Amsterdam, Amsterdam, The Netherlands.

Background: In resource-poor settings, transfused children often experience recurrence of severe anemia (SA) following discharge from hospital. This study determined the factors associated with recurrent severe anemia (RSA) among previously transfused Ugandan children aged less than 5 years.

Methods: A case-control study was conducted in five hospitals in Uganda from March 2017 to September 2018. Read More

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https://bmcpediatr.biomedcentral.com/articles/10.1186/s12887
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http://dx.doi.org/10.1186/s12887-019-1398-6DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6337863PMC
January 2019
9 Reads

Invasive Nontyphoidal Disease in Africa.

EcoSal Plus 2019 01;8(2)

Jenner Institute, Nuffield Department of Medicine, University of Oxford, Oxford, UK.

Nontyphoidal salmonellae (NTS) are a major cause of invasive (iNTS) disease in sub-Saharan Africa, manifesting as bacteremia and meningitis. Available epidemiological data indicate that iNTS disease is endemic in much of the region. Antimicrobial resistance is common and case fatality rates are high. Read More

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http://www.asmscience.org/content/journal/ecosalplus/10.1128
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http://dx.doi.org/10.1128/ecosalplus.ESP-0007-2018DOI Listing
January 2019
7 Reads

Investigating the Sleep-Pain Relationship in Youth with Sickle Cell Utilizing mHealth Technology.

J Pediatr Psychol 2019 Jan 10. Epub 2019 Jan 10.

Department of Pediatrics, East Carolina University.

Objectives: The current study utilized mHealth technologies that were objective (e.g., sleep actigraphy and pulse oximetry) and time-sensitive (e. Read More

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http://dx.doi.org/10.1093/jpepsy/jsy105DOI Listing
January 2019
1 Read

Acute Vaso-Occlusive Pain is Temporally Associated with the Onset of Menstruation in Women with Sickle Cell Disease.

J Womens Health (Larchmt) 2019 Feb 16;28(2):162-169. Epub 2019 Jan 16.

1 Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, Nashville, Tennessee.

Background: Acute vaso-occlusive pain episodes in sickle cell disease (SCD) are associated with increased rates of hospitalization and early mortality. Despite the observation that women have higher rates of acute vaso-occlusive pain episodes than men, sex-specific risk factors for acute vaso-occlusive pain have not been identified. We tested the hypothesis that acute vaso-occlusive pain is temporally associated with the onset of menstruation in women with SCD. Read More

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https://www.liebertpub.com/doi/10.1089/jwh.2018.7147
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http://dx.doi.org/10.1089/jwh.2018.7147DOI Listing
February 2019
4 Reads

Functional and Radiologic Assessment of the Brain after Reduced-Intensity Unrelated Donor Transplantation for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network Study 0601.

Biol Blood Marrow Transplant 2019 Jan 11. Epub 2019 Jan 11.

Dept. of Pediatrics, Division of Hematology and Oncology, Washington University School of Medicine, St. Louis Children's Hospital, St Louis, Missouri.

Stroke and cognitive decline are hallmarks of sickle cell disease (SCD). The natural history of SCD predicts progressive loss of 1 IQ point per year attributable to disease-related pathology. Hematopoietic cell transplantation (HCT) is curative by reverting to donor-derived erythropoiesis, but evidence that HCT can positively influence disease-induced cognitive decline is lacking. Read More

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http://dx.doi.org/10.1016/j.bbmt.2019.01.008DOI Listing
January 2019
2 Reads

Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.

Am J Hematol 2019 Jan 13. Epub 2019 Jan 13.

Division of Hematology/Oncology, Department of Pediatrics, UCSF Benioff Children's Hospital of Oakland, Oakland, California.

We conducted a multicenter pilot investigation of the safety and feasibility of bone marrow transplantation (BMT) in adults with severe sickle cell disease (SCD) (NCT 01565616) using a reduced toxicity preparative regimen of busulfan (13.2 mg/kg), fludarabine (175 mg/m ) and thymoglobulin (6 mg/kg) and cyclosporine or tacrolimus and methotrexate for graft-vs-host disease (GVHD) prophylaxis. Twenty-two patients (median age 22 years; range 17-36) were enrolled at eight centers. Read More

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http://doi.wiley.com/10.1002/ajh.25401
Publisher Site
http://dx.doi.org/10.1002/ajh.25401DOI Listing
January 2019
10 Reads

Healthcare utilization and hydroxyurea adherence in youth with sickle cell disease.

Pediatr Hematol Oncol 2019 Jan 12:1-12. Epub 2019 Jan 12.

a Department of Pediatrics , Feinberg School of Medicine at Northwestern University , Chicago , IL, USA.

Background: Sickle cell disease (SCD) complications lead to poor health-related quality of life (HRQOL) and increased healthcare utilization in this population, which could be mitigated with hydroxyurea therapy; however, adherence is suboptimal. We assessed the relationship of healthcare utilization to hydroxyurea adherence and HRQOL amongst youth with SCD.

Methods: Thirty-four patients with SCD (median age 14 years, IQR 12-18) on hydroxyurea participated in this cross-sectional study and completed Morisky Adherence Scale 8-items and Patient Reported Outcomes Measurement Information System (PROMIS®) HRQOL measures. Read More

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http://dx.doi.org/10.1080/08880018.2018.1505988DOI Listing
January 2019

Novel Sickle Cell Disease Therapies: Targeting Pathways Downstream of Sickling.

Semin Hematol 2018 04 20;55(2):68-75. Epub 2018 Apr 20.

Department of Pediatrics, Division of Pediatric Hematology/Oncology, Albert Einstein College of Medicine, Children's Hospital at Montefiore, Bronx, NY. Electronic address:

Sickle cell disease is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. Although the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso-occlusion results from heterotypic interactions between the altered, adhesive sickle cell RBCs, neutrophils, endothelium, and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S00371963173020
Publisher Site
http://dx.doi.org/10.1053/j.seminhematol.2018.04.007DOI Listing
April 2018
16 Reads

Sickle cell disease: Translating clinical care to low-resource countries through international research collaborations.

Semin Hematol 2018 04 26;55(2):102-112. Epub 2018 Apr 26.

Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Global Health Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; University of Cincinnati College of Medicine, Cincinnati, OH. Electronic address:

The vast majority of the world's population of children and adults with sickle cell disease (SCD) are born in low-resource settings, particularly in sub-Saharan Africa, the Caribbean, the Middle East, and India. As a result numerous well-established, cost-effective, and evidence-based strategies for managing SCD such as newborn screening, early education, vaccinations, screening for stroke prevention, and treatments with safe transfusions and hydroxyurea are often unavailable, leading to substantial morbidity and increased mortality. Collaborations between high-income countries and these low-resource settings (North-South partnerships) have been advocated, with the goal of improving clinical care. Read More

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http://dx.doi.org/10.1053/j.seminhematol.2018.04.010DOI Listing
April 2018
3 Reads

Community perceptions of paediatric severe anaemia in Uganda.

PLoS One 2019 3;14(1):e0209476. Epub 2019 Jan 3.

Department of Global Child Health, Emma Children's Hospital, Academic Medical Centre, University of Amsterdam, Amsterdam, the Netherlands.

Background: Severe anaemia remains a major cause of morbidity and mortality among children in sub-Saharan Africa. There is limited research on the beliefs and knowledge for paediatric severe anaemia in the region. The effect of these local beliefs and knowledge on the healthcare seeking of paediatric severe anaemia remains unknown. Read More

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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0209476PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6317806PMC
January 2019
7 Reads

Gene therapy for blood diseases.

Authors:
Donald B Kohn

Curr Opin Biotechnol 2018 Dec 29;60:39-45. Epub 2018 Dec 29.

Department of Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, David Geffen School of Medicine, USA; Department of Pediatrics, University of California, Los Angeles, David Geffen School of Medicine, USA; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, David Geffen School of Medicine, USA. Electronic address:

Genetic diseases affecting proteins and cells composing the blood may be treated by gene therapy using gene addition or gene editing methods. Protein deficiencies (e.g. Read More

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http://dx.doi.org/10.1016/j.copbio.2018.11.016DOI Listing
December 2018
2 Reads

Sensorineural hearing loss in children with sickle cell disease.

Int J Pediatr Otorhinolaryngol 2019 Mar 5;118:110-114. Epub 2018 Dec 5.

Emory University School of Medicine, 1648 Pierce Drive NE, Atlanta, GA, 30307, USA; Children's Healthcare of Atlanta, Department of Otolaryngology, Division of Pediatric Otolaryngology, 2015 Uppergate Drive, Atlanta, GA, 30322, USA.

Introduction: Sensorineural hearing loss (SNHL) has been reported to occur at increased frequency in the pediatric sickle cell disease (SCD) population, likely secondary to ototoxic medication regimens and repeat sickling events that lead to end organ damage. Risk and protective factors of SNHL in this population are not fully characterized. The objective of this study was to describe audiology results in children with SCD and the prevalence and sequelae of SNHL. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S01655876183060
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http://dx.doi.org/10.1016/j.ijporl.2018.12.002DOI Listing
March 2019
10 Reads

Vaso-occlusive crisis in sickle cell disease: current paradigm on pain management.

J Pain Res 2018 11;11:3141-3150. Epub 2018 Dec 11.

Department of Pediatrics, College of Medicine, University of Nigeria, Enugu, Nigeria,

This narrative review aims to highlight the current paradigm on pain management in sickle cell vaso-occlusive crisis. It specifically examines the pathophysiologic mechanisms of sickle cell pain as well as the pharmacologic and nonpharmacologic methods of pain management. Recurrent painful episodes constitute the major morbidity in sickle cell disease (SCD). Read More

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http://dx.doi.org/10.2147/JPR.S185582DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6294061PMC
December 2018
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Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities, and Networks-American Pain Society-American Academy of Pain Medicine Pain Taxonomy Diagnostic Criteria for Acute Sickle Cell Disease Pain.

J Pain 2018 Dec 19. Epub 2018 Dec 19.

WTZ Department of Pediatrics, Connecticut Children's Medical Center, Hartford, Connecticut.

Acute pain episodes are the most common complication in patients with sickle cell disease (SCD). Classically attributed to vaso-occlusion, recent insights suggest that chronic pain may also contribute to the pathogenesis of acute pain episodes, which adds complexity to their diagnosis and management. A taxonomy, or classification system, for acute pain in patients with SCD would aid research efforts and enhance clinical care. Read More

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https://linkinghub.elsevier.com/retrieve/pii/S15265900183101
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http://dx.doi.org/10.1016/j.jpain.2018.12.003DOI Listing
December 2018
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Assessing Disease Knowledge and Self-Management in Youth With Sickle Cell Disease Prior to Transition.

J Pediatr Oncol Nurs 2018 Dec 19:1043454218819447. Epub 2018 Dec 19.

1 Children's National Health System, Washington, DC, USA.

Introduction: Transition of medical care from pediatrics to adult can be challenging and difficult. Until the 1970s, only half of patients diagnosed with sickle cell disease (SCD) reached adulthood. As a result of patients living longer, there is a growing need to understand factors that influence readiness to transition. Read More

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http://dx.doi.org/10.1177/1043454218819447DOI Listing
December 2018
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Psychological Predictors of Pain in Children and Adolescents With Sickle Cell Disease: A Scoping Review.

J Pediatr Oncol Nurs 2018 Dec 19:1043454218819457. Epub 2018 Dec 19.

1 University of Michigan School of Nursing, Ann Arbor, MI, USA.

Objective: Sickle cell disease (SCD) is a common red blood cell disorder that disrupts the lives of many African Americans and those of Middle Eastern heritage within the United States due to frequent pain. There is limited research quantifying biopsychosocial factors, specifically psychological characteristics, that influence pain in children and adolescents with SCD. The aim of this literature review was to identify psychological characteristics that are predictive or associated with pain in children and adolescents with SCD. Read More

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http://journals.sagepub.com/doi/10.1177/1043454218819457
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http://dx.doi.org/10.1177/1043454218819457DOI Listing
December 2018
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Sickle Cell Disease: Advances in Treatment.

Authors:
Renée V Gardner

Ochsner J 2018 ;18(4):377-389

Department of Pediatrics, Louisiana State University Health Sciences Center, Children's Hospital of New Orleans, New Orleans, LA.

Background: Sickle cell disease causes significant morbidity and mortality and affects the economic and healthcare status of many countries. Yet historically, the disease has not had commensurate outlays of funds that have been aimed at research and development of drugs and treatment procedures for other diseases.

Methods: This review examines several treatment modalities and new drugs developed since the late 1990s that have been used to improve outcomes for patients with sickle cell disease. Read More

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http://dx.doi.org/10.31486/toj.18.0076DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6292457PMC
January 2018
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Summer Camps for Children with Sickle Cell Disease.

Ochsner J 2018 ;18(4):358-363

Department of Pediatrics, College of Medicine, University of Illinois at Chicago, Chicago, IL.

Background: Children with sickle cell disease (SCD) face unique problems that attendance at a camp with their peers is well suited to address. However, because the staff members at ordinary summer camps are not accustomed to accommodating children with chronic diseases, the potential for significant health consequences exists.

Methods: We searched the literature in PubMed and CINAHL using the keywords summer camp, camp, sickle, and anemia to identify the unique characteristics of camps for children with SCD and the recommendations for care and/or lessons learned. Read More

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http://dx.doi.org/10.31486/toj.18.0045DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6292478PMC
January 2018
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Clinical Observations, Plasma Retinol Concentrations, and In Vitro Lymphocyte Functions in Children With Sickle Cell Disease.

Ochsner J 2018 ;18(4):308-317

Department of Pediatrics, Louisiana State University Health Sciences Center, New Orleans, LA.

Background: Children with sickle cell disease (SCD) often have infections, growth deficits, and impaired immunity, problems that also are observed in individuals with a vitamin A deficiency (plasma retinol concentration <20 μg/dL). The goal of this study was to investigate the association between vitamin A, health status, and the in vitro immune function of children with SCD.

Methods: Fifty-nine children (40 SS, 11 SC, and 8 Sβ [Sβ] hemoglobin genotypes) 9 months to 18 years old were investigated for plasma levels of retinol, retinol binding protein, C-reactive protein, alpha-1-acid glycoprotein, lymphocyte proliferation, and interleukin (IL)-2 activity in supernatant of phytohemagglutinin-treated lymphocytes. Read More

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http://dx.doi.org/10.31486/toj.17.0044DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6292479PMC
January 2018
2 Reads

Lung Function in Infants with Sickle Cell Anemia.

J Pediatr 2018 Dec 14. Epub 2018 Dec 14.

Department of Pediatrics, Federal University of São Paulo, São Paulo, Brazil. Electronic address:

Lung volumes and forced expiratory flows were evaluated in 22 infants with sickle cell anemia and compared with a control group. Forced expiratory flows showed significantly lower values in the sickle cell group. The majority of infants had normal lung function, and obstruction was the most common pattern of abnormality. Read More

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http://dx.doi.org/10.1016/j.jpeds.2018.11.036DOI Listing
December 2018
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Hidden hearing loss in children and adolescents with sickle cell anemia.

Int J Pediatr Otorhinolaryngol 2019 Jan 2;116:186-191. Epub 2018 Nov 2.

Bahiana School of Medicine and Public Health, Salvador, Bahia, Brazil.

Objective: To evaluate the auditory system for hidden hearing loss (HHL) and its association with clinical variables and endothelial dysfunction (ED) in children and adolescents with sickle cell anemia (SCA).

Methods: Participants included 37 patients with stable SCA and 44 healthy controls (HC group) (aged 6-18 years) with hearing thresholds ≤ 20 dB (dB) were evaluated for pure tone audiometry, tympanometry, acoustic reflex, otoacoustic emission, and auditory evoked potentials. Laboratory analysis of the lipid profile, and C-reactive protein levels and endothelial function using ultrasonographic imaging of the brachial artery to assess flow-mediated dilation were performed. Read More

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http://dx.doi.org/10.1016/j.ijporl.2018.10.042DOI Listing
January 2019
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Impact of erythrocytapheresis on natural anticoagulant levels in children with sickle cell disease: A pilot study.

Pediatr Blood Cancer 2019 Apr 13;66(4):e27588. Epub 2018 Dec 13.

Department of Pediatrics, The Ohio State University, Division of Hematology/Oncology, Nationwide Children's Hospital, Columbus, Ohio.

Venous thromboembolism (VTE) is being increasingly recognized in children with sickle cell disease (SCD). In a retrospective cohort study, we identified bilateral central venous catheter (CVC) placement as an independent risk factor for VTE. At our institution, the only indication for bilateral CVC placement in children with SCD is erythrocytapheresis. Read More

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http://dx.doi.org/10.1002/pbc.27588DOI Listing
April 2019
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2.562 Impact Factor

Subclinical Cardiac Dysfunction Is Associated With Extracardiac Organ Damages.

Front Med (Lausanne) 2018 20;5:323. Epub 2018 Nov 20.

Cardiology Department, Hôpital Européen Georges Pompidou, Assistance Publique des Hôpitaux de Paris and UMR_S970, Université Paris Descartes, Inserm, Paris, France.

Several studies conducted in America or Europe have described major cardiac remodeling and diastolic dysfunction in patients with sickle cell disease (SCD). We aimed at assessing cardiac involvement in SCD in sub-Saharan Africa where SCD is the most prevalent. In Cameroon, Mali and Senegal, SCD patients and healthy controls of the CADRE study underwent transthoracic echocardiography if aged ≥10 years. Read More

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http://dx.doi.org/10.3389/fmed.2018.00323DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6262341PMC
November 2018
1 Read