Developmental delay in infants and toddlers with sickle cell disease: a systematic review

2021 ◽  
Author(s):  
Catherine R Hoyt ◽  
Taniya E Varughese ◽  
Jeni Erickson ◽  
Natalie Haffner ◽  
Lingzi Luo ◽  
...  
Keyword(s):  
Systematic Review ◽  
Sickle Cell Disease ◽  
Developmental Delay ◽  
Sickle Cell ◽  
Infants And Toddlers ◽  
Cell Disease

Neurdevelopmental Deficits Among 80 Infants and Toddlers with Sickle Cell Disease

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 174-174
Author(s):  
Tara Brennan ◽  
Penny Glass ◽  
Christen Meisel ◽  
Brenda Martin ◽  
Lori Luchtman-Jones ◽  
...  
Keyword(s):  
Primary Care ◽  
Sickle Cell Disease ◽  
Developmental Delay ◽  
Sickle Cell ◽  
Maternal Education ◽  
College Graduates ◽  
Cell Phenotype ◽  
Infants And Toddlers ◽  
Education Attainment ◽  
Cell Disease

Abstract Abstract 174FN2 Introduction: Neurocognitive deficits by school age in children with sickle cell disease (SCD), with and without a history of stroke, has been well established. However, little is understood with respect to the onset and course of these deficits in early childhood and how these may relate to SES and disease status. We are undertaking a 4-year mixed cross-sectional longitudinal study of early neurodevelopmental status in children younger than 4 years of age with SCD. The primary aims are to: (1) characterize the prevalence and nature of the developmental deficits in infants and toddlers while controlling for socio-economic status (SES); (2) examine the moderating effects of sickle cell phenotype, hematologic severity, and parent characteristics on developmental outcome. The current presentation is based on the first 80 baseline neurodevelopmental evaluations. Patients and Methods: Children 3 ½ years of age or younger with SCD of any phenotype were eligible to participate. Comprehensive neurodevelopmental evaluations using the Bayley Scales of Infant Development were conducted when the child reached specific age levels (9, 15, 21, 30, and 40 months), with an ultimate planned sample size of a minimum of 45 children with SCD at each of the five age levels. Demographics and data regarding phenotype, hematologic variables, and occurrence of pain crises were obtained separately. Results: To date, 199 children have been enrolled in the study. We have conducted 205 evaluations and completed data analysis of the baseline data for 80 patients. Of those 80, phenotype distribution was representative of the sickle cell population (HbSS=56%, HbSC=31%). Gender was balanced (51% female). As expected, race/ethnicity of most children was African/African American (95%), about 5% Hispanic/Biracial. Maternal education attainment was high, as almost half of the mothers (48%) were college graduates. Mean Mental Index was 84.2 (sd=13.5) and Motor Index was 89.1 (sd=14.4) (compared to published norm means of 100, sd=15). Fourteen children (17.5%) had a significant delay (Index score > 2 sd below the mean) on either the Mental or Motor Index, with 7 children significantly delayed on both. Lower SES, based on maternal education attainment, was not sufficient to explain developmental delay. Poor performance was present in 10% of children whose mothers were college graduates. Male children were at significantly greater risk for delays, controlling for SES, pain crisis, pneumonia/ACS, and %hemoglobin. Odds ratio of significant developmental delay was >9 times more likely among those who had vaso-occlusive pain episode, controlling for SES, gender, pneumonia/ACS, and hemoglobin concentration. Of note, none of the children had been identified as delayed by their primary care physician or sickle cell provider. Conclusions: Early cognitive and motor delays are present in SCD and not sufficiently explained by lower SES. Increased vulnerability of male gender is consistent with other at-risk populations, but has not been previously addressed in SCD research. Significant developmental delay in children with chronic illness may go unrecognized by primary care practices, medical specialty clinics, or parents. The importance of routine neurodevelopmental assessment for children with chronic medical disease, and SCD in particular, is clear. This study was funded by the NHLBI as a Patient Services Project within the Washington Area Comprehensive Sickle Cell Center. Disclosures: No relevant conflicts of interest to declare.

Re: Manuscript titled, “Liver transplantation for sickle cell disease: a systematic review”

HPB ◽  
2021 ◽  
Author(s):  
Juliet Emamaullee ◽  
Linda Sher ◽  
Kambiz Etesami ◽  
Jeff Kahn ◽  
Jim Kim ◽  
...  
Keyword(s):  
Systematic Review ◽  
Liver Transplantation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Cell Disease

scholarly journals The effect of iron chelation therapy on overall survival in sickle cell disease and β-thalassemia: A systematic review

2018 ◽  
Vol 93 (7) ◽  
pp. 943-952 ◽  
Author(s):  
Samir K. Ballas ◽  
Amer M. Zeidan ◽  
Vu H. Duong ◽  
Michelle DeVeaux ◽  
Matthew M. Heeney
Keyword(s):  
Systematic Review ◽  
Overall Survival ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Cell Disease

scholarly journals Efficacy of Allogeneic Hematopoietic Cell Transplantation (HCT) in Sickle Cell Disease: Results of a Systematic Review/Meta-Analysis

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2106-2106
Author(s):  
Madiha Iqbal ◽  
Tea Reljic ◽  
Ernesto Ayala ◽  
Hemant S. Murthy ◽  
Ambuj Kumar ◽  
...  
Keyword(s):  
Systematic Review ◽  
Sickle Cell Disease ◽  
Clinical Outcomes ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Medical Literature ◽  
Meta Analysis ◽  
Adult Patients ◽  
Cell Disease ◽  
Allogeneic Hct

Background: Sickle cell disease (SCD) is an inherited hemoglobinopathy which affects over 300,000 children born each year worldwide. In spite of improvement in supportive care in recent years, there is still a lack of effective treatment options. SCD leads to debilitating and cyclic episodes of erythrocyte sickling with progressive organ injury, contributing to lifetime morbidity and shortened life expectancy. Allogeneic HCT (allo-HCT) is a potentially curative therapy for SCD because engraftment is associated with resolution of the clinical phenotype of the disease and abrogation of its complications. Medical literature on allo-HCT for SCD is largely limited to children. Recent studies have evaluated the efficacy of allo-HCT in the adult population. Here, we conduct a systematic review/meta-analysis to assess the totality of evidence pertaining to the efficacy (or lack thereof) of allo-HCT in children and adults. Materials and methods: We performed a comprehensive search of the medical literature using PubMed/Medline, EMBASE and Cochrane library on July 3rd, 2019. We extracted data on clinical outcomes related to benefits (overall [OS] and disease free/event free survival [EFS/DFS]) and harms (non-relapse mortality [NRM] and graft failure [GF]), independently by two authors. Our search strategy identified 1001 references but only 30 studies (n= 1995 patients) were included in this systematic review/meta-analysis. We also performed a sub analysis on clinical outcomes for studies that included only pediatric patients (defined as <18 years) and those in patients ≥18 years of age. Results: Median age for patients enrolled in all the studies was at 10 years. Recurrent veno-occlusive crises represented the most common indication for allo-HCT followed by acute chest syndrome and stroke; nevertheless, most patients had more than one indication. Matched related donors (MRD) were the most common donor source (93%). Bone marrow was the most common source of hematopoietic stem cells (77%). Majority of patients underwent conditioning with myeloablative regimens (77%). Pooled OS rates (n=29 studies, 1681 patients) after allogeneic HCT was 95% (95%CI=93-96%) with low heterogeneity (I2=6.4%) among included studies (Figure 1). Pooled EFS/DFS rates (n=29 studies, 1894 patients) post-allografting was 90% (95%CI=87-93%) with moderate heterogeneity (I2=54%). Pooled NRM rates from 30 studies (1995 patients) was 4% (95%CI=2-6%) with low heterogeneity (I2=29.4%). Pooled GF rates from 28 studies (1851 patients) was 4% (95%CI=2-6%) with moderate heterogeneity (I2=55%). A subset analysis specifically for pediatric patients (n= 11 studies, 1009 patients, median age at 9.7 years) showed a pooled OS rate of 96% (95%CI=94-97%) with low heterogeneity (I2=0%); and for adult patients (n=3 studies, 51 patients, median age at 33.4 years) the pooled OS was 94% (95%CI=80-100%) with moderate heterogeneity (I2=52%). Pooled EFS/DFS for pediatric patients (n= 11 studies, 1009 patients) was at 89 %( 95%CI=84-93%) with moderate heterogeneity (I2=55.1%); and for adult patients (n=2 studies, 30 patients) was at 95% (95%CI=83-100%) with high heterogeneity (I2=96.5%). Pooled NRM from 10 studies with pediatric patients (281 patients) was at 6 % (95%CI=3-10%) with low heterogeneity (I2=0%); and from 3 studies with adult patients (51 patients) was at 1% (95%CI=0-7%) with low heterogeneity (I2=15.1%). Pooled GF from 10 studies with pediatric patients (281 patients) was at 3 % (95%CI=1-7%) with moderate heterogeneity (I2=40%); and from 2 studies with adult patients (30 patients) was at 5% (95%CI=0-17%) with high heterogeneity (I2=95.4%). Conclusions: The results of our systematic review/meta-analysis show excellent OS, EFS/DFS in children and adults undergoing allo-HCT with pooled OS rates exceeding 90%. The main limitation to offering an allo-HCT in SCD remains the availability of a suitable donor as 85% of patients meeting criteria do not have a MRD. We anticipate that with emergence of haploidentical transplantation the number of allo-HCT will increase in the future. GF remains a significant concern in this population and future studies should focus on novel immune suppression strategies to help reduce GF. Disclosures Kharfan-Dabaja: Pharmacyclics: Consultancy; Daiichi Sankyo: Consultancy.

scholarly journals Sickle cell disease, sickle trait and the risk for venous thromboembolism: a systematic review and meta-analysis

2018 ◽  
Vol 16 (1) ◽  
Author(s):  
Jean Jacques Noubiap ◽  
Mazou N. Temgoua ◽  
Ronni Tankeu ◽  
Joel Noutakdie Tochie ◽  
Ambroise Wonkam ◽  
...  
Keyword(s):  
Systematic Review ◽  
Venous Thromboembolism ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Meta Analysis ◽  
Cell Disease

scholarly journals Iron stores in pregnant women with sickle cell disease: a protocol for a systematic review and meta-analysis

BMJ Open ◽  
2019 ◽  
Vol 9 (9) ◽  
pp. e026497 ◽  
Author(s):  
Desmond Aroke ◽  
Benjamin Momo Kadia ◽  
Tsi Njim
Keyword(s):  
Systematic Review ◽  
Sickle Cell Disease ◽  
Iron Deficiency ◽  
Pregnant Women ◽  
Sickle Cell ◽  
Iron Deficiency Anaemia ◽  
Meta Analysis ◽  
Cochrane Library ◽  
Cell Disease ◽  
Deficiency Anaemia

IntroductionSickle cell disease (SCD) is the most common inherited disease worldwide. The greatest disease burden is seen in sub-Saharan Africa. Early diagnosis and improved care of people living with SCD have led to an increase in the number of women with SCD reaching the reproductive age. Iron deficiency anaemia remains the most common cause of anaemia in pregnancy, affecting 51%–63% of pregnancies in Africa. However, the unavailability of guidelines on supplementation of iron in this pregnant subpopulation often leaves clinicians in a fix. We propose to conduct the first systematic review and possibly a meta-analysis on the prevalence, associated factors and maternal/fetal outcomes of iron deficiency anaemia among pregnant women with SCD.Methods and analysisWe will search the following electronic databases for studies on the iron status of pregnant women with SCD: PubMed, MEDLINE, EMBASE, Google Scholar, African Journals Online, African Index Medicus, Popline and the Cochrane Library. After the selection of eligible studies from the search output, review of full text, data extraction and data synthesis will be performed. Studies obtained from the review shall be evaluated for quality, risk of bias and heterogeneity. Appropriate statistical methods shall be used to pool prevalence estimates for matching studies globally and in subpopulations. This protocol has been reported as per the 2015 guidelines of the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols.Ethics and disseminationThere is no requirement for ethical approval as the proposed study will use published data. The findings of this study will be published in a peer-reviewed journal and will be presented at conferences.Trial registration numberCRD42018109803.

scholarly journals PF741 PERIOPERATIVE CARE OF CHILDREN WITH SICKLE CELL DISEASE: A SYSTEMATIC REVIEW AND CLINICAL RECOMMENDATIONS

HemaSphere ◽  
2019 ◽  
Vol 3 (S1) ◽  
pp. 324-325
Author(s):  
F. Schyrr ◽  
G. Canellini ◽  
H. Andreu-Ullrich ◽  
J.-M. Joseph ◽  
M. Dolci ◽  
...  
Keyword(s):  
Systematic Review ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Perioperative Care ◽  
Cell Disease ◽  
Clinical Recommendations

scholarly journals Transcranial Doppler screening for stroke risk in children with sickle cell disease: a systematic review

2017 ◽  
Vol 12 (6) ◽  
pp. 580-588 ◽  
Author(s):  
Sara Mazzucco ◽  
Marina Diomedi ◽  
Amrana Qureshi ◽  
Laura Sainati ◽  
Soundrie T Padayachee
Keyword(s):  
Systematic Review ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Transcranial Doppler ◽  
Stroke Risk ◽  
Screening Methods ◽  
Cell Disease ◽  
High Quality ◽  
Screening Practices

Background Sickle cell disease (SCD) is one of the most common causes of stroke in children worldwide. Based on the results of the Stroke Prevention Trial in Sickle Cell Anemia (STOP), annual transcranial Doppler ultrasound (TCD) screening for affected children is standard practice. However, the need for TCD surveillance programs could override the accuracy of the screening, affecting the correct stratification of stroke risk and subsequent clinical management of the target population. Aims To shed light on this issue, a systematic review of the literature on TCD screening for children and adolescents with SCD was carried out (CRD42016050549), according to a list of clinically relevant questions, with a particular focus on screening practices in European countries. Quality of the evidence was rated using the grading of recommendations assessment, development and evaluation. Summary of review Thirty-three studies published in English or French were included (5 randomized controlled trials, 8 experimental non-randomized, and 20 observational studies). The quality of the retrieved evidence ranged between low and high, but was rated as moderate or high most of the times. TCD is effective as a screening tool for the primary prevention of stroke in SCD children. There is no high-quality evidence on the effectiveness of alternative screening methods, such as imaging-TCD with or without angle correction or magnetic resonance angiography. No evidence was found on effectiveness of the screening on children on hydroxyurea and with genotypes other than HbSS and HbS/β0. No European data were found on screening rates or adherence of screening practices to the STOP protocol. Conclusions High-quality studies on alternative screening methods that are currently used in real-world practice, and on screening applicability to specific subgroups of patients are urgently needed. Considering the low awareness of the disease in European countries and the lack of data on screening practices and adherence, clinicians need up-to-date guidelines for more uniform and evidence-based surveillance of children with SCD.

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