{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T23:06:47Z","timestamp":1791932807050,"version":"4.3.4"},"reference-count":145,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2022,6,21]],"date-time":"2022-06-21T00:00:00Z","timestamp":1655769600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Neurol."],"abstract":"<jats:p>Pediatric patients with sickle cell disease (SCD) experience a range of medical complications that result in significant morbidity and mortality. Recent advances in prophylactic and curative treatment approaches have highlighted the need for sensitive and clinically-meaningful trial endpoints. The detrimental effects of cognitive and psychological difficulties on social and economic mobility are well described. Although numerous reviews have assessed cognitive outcomes in other rare genetic disorders, SCD has not received the same focus. This review describes the cognitive (i.e., executive function and processing speed) and psychological domains (i.e., depression and anxiety) that are consistently associated with SCD pathology and, therefore, may be of particular interest as clinical trial endpoints. We then discuss corresponding well-validated and reliable cognitive tests and patient-reported outcomes (PROs) that may be appropriate for clinical trials given their robust psychometric properties, ease of administration, and previous use in the SCD population. Further, we provide a discussion of potential pitfalls and considerations to guide endpoint selection. In line with the move toward patient-centered medicine, we identify specific tests (e.g., NIH Toolbox Cognition Module, Wechsler Cancellation Test) and psychological PROs (e.g., PROMIS depression and anxiety scales) that are sensitive to SCD morbidity and have the potential to capture changes that are clinically meaningful in the context of patients' day to day lives. In particularly vulnerable cognitive domains, such as executive function, we highlight the advantages of composite over single-test scores within the context of trials. We also identify general (i.e., practice effects, disease heterogeneity) and SCD-specific considerations (i.e., genotype, treatment course, and disease course, including degree of neurologic, pain, and sleep morbidity) for trial measures. Executive function composites hold particular promise as trial endpoints that are clinically meaningful, amenable to change, relatively easy to collect, and can be incorporated into the routine care of patients with SCD in various settings and countries.<\/jats:p>","DOI":"10.3389\/fneur.2022.835823","type":"journal-article","created":{"date-parts":[[2022,6,21]],"date-time":"2022-06-21T04:39:12Z","timestamp":1655786352000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":11,"title":["Considerations for Selecting Cognitive Endpoints and Psychological Patient-Reported Outcomes for Clinical Trials in Pediatric Patients With Sickle Cell Disease"],"prefix":"10.3389","volume":"13","author":[{"given":"Anna M.","family":"Hood","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lori E.","family":"Crosby","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanne","family":"Stotesbury","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie","family":"K\u00f6lbel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fenella J.","family":"Kirkham","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2022,6,21]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"2018","DOI":"10.1016\/S0140-6736(10)61029-X","article-title":"Sickle-cell disease","volume":"376","author":"Rees","year":"2010","journal-title":"Lancet."},{"key":"B2","doi-asserted-by":"publisher","first-page":"13","DOI":"10.4081\/thal.2021.9803","article-title":"Hemoglobin disorders in Europe: a systematic effort of identifying and addressing unmet needs and challenges by the Thalassemia International Federation","volume":"11","author":"Angastiniotis","year":"2021","journal-title":"Thalass Rep."},{"key":"B3","doi-asserted-by":"publisher","first-page":"480","DOI":"10.2471\/BLT.06.036673","article-title":"Global epidemiology of haemoglobin disorders and derived service indicators","volume":"86","author":"Modell","year":"2008","journal-title":"Bull World Health Organ."},{"key":"B4","doi-asserted-by":"publisher","first-page":"S512","DOI":"10.1016\/j.amepre.2009.12.022","article-title":"Population estimates of sickle cell disease in the U","volume":"38","author":"Hassell","year":"2010","journal-title":"Am J Prev Med."},{"key":"B5","doi-asserted-by":"publisher","first-page":"932","DOI":"10.7326\/0003-4819-148-12-200806170-00220","article-title":"National Institutes of Health Consensus Development Conference statement: hydroxyurea treatment for sickle cell disease","volume":"148","author":"Brawley","year":"2008","journal-title":"Ann Intern Med."},{"key":"B6","doi-asserted-by":"publisher","first-page":"1033","DOI":"10.1001\/jama.2014.10517","article-title":"Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members","volume":"312","author":"Yawn","year":"2014","journal-title":"JAMA."},{"key":"B7","doi-asserted-by":"publisher","first-page":"460","DOI":"10.1111\/bjh.15235","article-title":"Guidelines for the use of hydroxycarbamide in children and adults with sickle cell disease: a British Society for Haematology Guideline","volume":"181","author":"Qureshi","year":"2018","journal-title":"Br J Haematol."},{"key":"B8","doi-asserted-by":"publisher","first-page":"3925","DOI":"10.1182\/blood-2011-11-392340","article-title":"Stroke with transfusions changing to hydroxyurea (SWiTCH)","volume":"119","author":"Ware","year":"2012","journal-title":"Blood."},{"key":"B9","doi-asserted-by":"publisher","first-page":"1663","DOI":"10.1016\/S0140-6736(11)60355-3","article-title":"A multicenter randomised controlled trial of hydroxyurea (hydroxycarbamide) in very young children with sickle cell anaemia","volume":"377","author":"Wang","year":"2011","journal-title":"Lancet."},{"key":"B10","doi-asserted-by":"publisher","first-page":"2749","DOI":"10.1182\/blood-2007-03-079665","article-title":"Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease","volume":"110","author":"Bernaudin","year":"2007","journal-title":"Blood."},{"key":"B11","doi-asserted-by":"publisher","first-page":"552","DOI":"10.1016\/j.bbmt.2017.01.009","article-title":"Current results and future research priorities in late effects after hematopoietic stem cell transplantation (HCT) for children with sickle cell disease and thalassemia: a consensus statement from the second pediatric blood and marrow transplant consortium","volume":"23","author":"Shenoy","year":"2017","journal-title":"Biol Blood Marrow Transplant."},{"key":"B12","doi-asserted-by":"publisher","first-page":"226","DOI":"10.1056\/NEJMoa1715971","article-title":"A phase 3 trial of l-glutamine in sickle cell disease","volume":"379","author":"Niihara","year":"2018","journal-title":"N Engl J Med."},{"key":"B13","doi-asserted-by":"publisher","first-page":"535","DOI":"10.1002\/phar.2409","article-title":"Systematic review of crizanlizumab: a new parenteral option to reduce vaso-occlusive pain crises in patients with sickle cell disease","volume":"40","author":"Han","year":"2020","journal-title":"Pharmacother J Hum Pharmacol Drug Ther."},{"key":"B14","doi-asserted-by":"publisher","first-page":"509","DOI":"10.1056\/NEJMoa1903212","article-title":"A phase 3 randomized trial of voxelotor in sickle cell disease","volume":"381","author":"Vichinsky","year":"2019","journal-title":"N Engl J Med."},{"key":"B15","doi-asserted-by":"publisher","first-page":"257","DOI":"10.1146\/annurev-med-041817-125507","article-title":"Emerging genetic therapy for sickle cell disease","volume":"70","author":"Orkin","year":"2019","journal-title":"Annu Rev Med."},{"key":"B16","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13063-021-05626-6","article-title":"Study of montelukast in children with sickle cell disease (SMILES): a study protocol for a randomised controlled trial","volume":"22","author":"Hood","year":"2021","journal-title":"Trials."},{"key":"B17","doi-asserted-by":"publisher","first-page":"948","DOI":"10.1093\/jpepsy\/jsz031","article-title":"Cognitive function in sickle cell disease across domains, cerebral infarct status, and the lifespan: a meta-analysis","volume":"44","author":"Prussien K","year":"2019","journal-title":"J Pediatr Psychol."},{"key":"B18","doi-asserted-by":"publisher","first-page":"120","DOI":"10.1080\/09297040600800956","article-title":"Neurocognitive sequelae of pediatric sickle cell disease: a review of the literature","volume":"13","author":"Berkelhammer","year":"2007","journal-title":"Child Neuropsychol."},{"key":"B19","doi-asserted-by":"publisher","first-page":"672","DOI":"10.1111\/dmcn.13113","article-title":"Intelligence quotient in paediatric sickle cell disease: a systematic review and meta-analysis","volume":"58","author":"Kawadler","year":"2016","journal-title":"Dev Med Child Neurol."},{"key":"B20","doi-asserted-by":"publisher","first-page":"114","DOI":"10.1007\/s11920-007-0080-0","article-title":"Anxiety and depression in children and adolescents with sickle cell disease","volume":"9","author":"Benton","year":"2007","journal-title":"Curr Psychiatry Rep."},{"key":"B21","doi-asserted-by":"publisher","first-page":"27132","DOI":"10.4088\/PCC.10m01063","article-title":"Prevalence and treatment of depression in children and adolescents with sickle cell disease: a retrospective cohort study","volume":"13","author":"Jerrell","year":"2011","journal-title":"Prim Care Companion CNS Disord."},{"key":"B22","doi-asserted-by":"publisher","first-page":"1052","DOI":"10.1001\/jamaneurol.2015.0443","article-title":"Treatment of cognitive deficits in genetic disorders: a systematic review of clinical trials of diet and drug treatments","volume":"72","author":"van der Vaart","year":"2015","journal-title":"JAMA Neurol."},{"key":"B23","doi-asserted-by":"publisher","first-page":"3982","DOI":"10.1182\/bloodadvances.2019000882","article-title":"End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain","volume":"3","author":"Farrell","year":"2019","journal-title":"Blood Adv."},{"key":"B24","doi-asserted-by":"publisher","first-page":"871","DOI":"10.3389\/fneur.2019.00871","article-title":"Vascular instability and neurological morbidity in sickle cell disease; an integrative framework","volume":"10","author":"Stotesbury","year":"2019","journal-title":"Front Neurol."},{"key":"B25","doi-asserted-by":"publisher","first-page":"830","DOI":"10.1182\/blood-2014-06-582403","article-title":"Understanding sickle cell brain drain","volume":"124","author":"Hulbert","year":"2014","journal-title":"Blood, J Am Soc Hematol."},{"key":"B26","first-page":"113","article-title":"Depression, anxiety, and quality of life in children and adolescents with sickle cell disease","volume":"42","author":"Graves","year":"2016","journal-title":"Pediatr Nurs."},{"key":"B27","doi-asserted-by":"publisher","first-page":"527","DOI":"10.1097\/AJP.0000000000000289","article-title":"Psychosocial and functional outcomes in youth with chronic sickle cell pain","volume":"32","author":"Sil","year":"2016","journal-title":"Clin J Pain."},{"key":"B28","doi-asserted-by":"publisher","first-page":"814","DOI":"10.1353\/hpu.2018.0060","article-title":"Social and behavioral factors in sickle cell disease: employment predicts decreased health care utilization","volume":"29","author":"Williams","year":"2018","journal-title":"J Health Care Poor Underserved."},{"key":"B29","doi-asserted-by":"publisher","first-page":"e1269","DOI":"10.1542\/peds.2011-3565","article-title":"Status of the pediatric clinical trials enterprise: an analysis of the US ClinicalTrials. gov registry","volume":"130","author":"Pasquali","year":"2012","journal-title":"Pediatrics."},{"key":"B30","unstructured":"RitchieH RoserM Causes of Death. Our World Data2018"},{"key":"B31","doi-asserted-by":"publisher","first-page":"e1002520","DOI":"10.1371\/journal.pmed.1002520","article-title":"Delays in completion and results reporting of clinical trials under the Paediatric Regulation in the European Union: a cohort study","volume":"15","author":"Hwang","year":"2018","journal-title":"PLoS Med."},{"key":"B32","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s12887-020-02270-y","article-title":"The risk of acute events among patients with sickle cell disease in relation to early or late initiation of care at a specialist center: evidence from a retrospective cohort study","volume":"20","author":"Rankine-Mullings","year":"2020","journal-title":"BMC Pediatr."},{"key":"B33","doi-asserted-by":"publisher","first-page":"77","DOI":"10.1002\/ajh.21570","article-title":"The number of people with sickle-cell disease in the United States: national and state estimates","volume":"85","author":"Brousseau","year":"2010","journal-title":"Am J Hematol."},{"key":"B34","doi-asserted-by":"publisher","first-page":"739","DOI":"10.1093\/jpepsy\/27.8.739","article-title":"Cognitive functioning in children with sickle cell disease: a meta-analysis","volume":"27","author":"Schatz","year":"2002","journal-title":"J Pediatr Psychol."},{"key":"B35","unstructured":"CheckD Choosing Specifying Endpoints and Outcomes2019"},{"key":"B36","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s12874-021-01413-8","article-title":"coreSCD: multi-stakeholder consensus on core outcomes for sickle cell disease clinical trials","volume":"21","author":"Tambor","year":"2021","journal-title":"BMC Med Res Methodol."},{"key":"B37","doi-asserted-by":"publisher","first-page":"e27899","DOI":"10.1002\/pbc.27899","article-title":"Higher executive abilities following a blood transfusion in children and young adults with sickle cell disease","volume":"66","author":"Hood","year":"2019","journal-title":"Pediatr Blood Cancer."},{"key":"B38","doi-asserted-by":"publisher","first-page":"1578","DOI":"10.1002\/pbc.26019","article-title":"Feasibility of home-based computerized working memory training with children and adolescents with sickle cell disease","volume":"63","author":"Hardy","year":"2016","journal-title":"Pediatr Blood Cancer."},{"key":"B39","doi-asserted-by":"publisher","first-page":"1001","DOI":"10.1093\/jpepsy\/jsab030","article-title":"A randomized controlled trial of working memory training in pediatric sickle cell disease","volume":"46","author":"Hardy","year":"2021","journal-title":"J Pediatr Psychol."},{"key":"B40","doi-asserted-by":"publisher","first-page":"e2042","DOI":"10.1212\/WNL.0000000000005644","article-title":"White matter integrity and processing speed in sickle cell anemia","volume":"90","author":"Stotesbury","year":"2018","journal-title":"Neurology."},{"key":"B41","doi-asserted-by":"publisher","first-page":"244","DOI":"10.1097\/DBP.0b013e31824ba1b5","article-title":"Ameliorating attention problems in children with sickle cell disease: a pilot study of methylphenidate","volume":"33","author":"Daly","year":"2012","journal-title":"J Dev Behav Pediatr."},{"key":"B42","doi-asserted-by":"publisher","first-page":"651","DOI":"10.1007\/s10880-019-09654-y","article-title":"Brief screening measures identify risk for psychological difficulties among children with sickle cell disease","volume":"27","author":"Hood","year":"2020","journal-title":"J Clin Psychol Med Settings."},{"key":"B43","doi-asserted-by":"publisher","first-page":"25","DOI":"10.1016\/S0160-2896(97)90012-X","article-title":"Psychometrics, intelligence, and public perception","volume":"24","author":"Carroll","year":"1997","journal-title":"Intelligence."},{"key":"B44","volume-title":"The Measurement of Adult Intelligence.","author":"Wechsler","year":"1944"},{"key":"B45","doi-asserted-by":"publisher","first-page":"361","DOI":"10.1007\/s11060-017-2582-x","article-title":"Do we still need IQ-scores? Misleading interpretations of neurocognitive outcome in pediatric patients with medulloblastoma: a retrospective study","volume":"135","author":"Wegenschimmel","year":"2017","journal-title":"J Neurooncol."},{"key":"B46","doi-asserted-by":"publisher","first-page":"46","DOI":"10.3758\/s13423-010-0034-0","article-title":"Does working memory training work? The promise and challenges of enhancing cognition by training working memory","volume":"18","author":"Morrison","year":"2011","journal-title":"Psychon Bull Rev."},{"key":"B47","volume-title":"WASI-II: Wechsler Abbreviated Scale of Intelligence.","author":"Wechsler","year":"2011"},{"key":"B48","doi-asserted-by":"publisher","first-page":"202","DOI":"10.1016\/j.intell.2015.10.006","article-title":"The environment in raising early intelligence: a meta-analysis of the fadeout effect","volume":"53","author":"Protzko","year":"2015","journal-title":"Intelligence."},{"key":"B49","doi-asserted-by":"publisher","first-page":"34","DOI":"10.1016\/j.dcn.2015.11.005","article-title":"Conclusions about interventions, programs, and approaches for improving executive functions that appear justified and those that, despite much hype, do not","volume":"18","author":"Diamond","year":"2016","journal-title":"Dev Cogn Neurosci."},{"key":"B50","doi-asserted-by":"publisher","first-page":"686","DOI":"10.1111\/desc.12246","article-title":"Socioeconomic status and executive function: developmental trajectories and mediation","volume":"18","author":"Hackman","year":"2015","journal-title":"Dev Sci."},{"key":"B51","doi-asserted-by":"publisher","first-page":"6829","DOI":"10.1073\/pnas.0801268105","article-title":"Improving fluid intelligence with training on working memory","volume":"105","author":"Jaeggi","year":"2008","journal-title":"Proc Natl Acad Sci."},{"key":"B52","doi-asserted-by":"publisher","first-page":"49","DOI":"10.1006\/cogp.1999.0734","article-title":"The unity and diversity of executive functions and their contributions to complex \u201cfrontal lobe\u201d tasks: a latent variable analysis","volume":"41","author":"Miyake","year":"2000","journal-title":"Cogn Psychol."},{"key":"B53","doi-asserted-by":"publisher","first-page":"135","DOI":"10.1146\/annurev-psych-113011-143750","article-title":"Executive functions","volume":"64","author":"Diamond","year":"2013","journal-title":"Annu Rev Psychol."},{"key":"B54","doi-asserted-by":"publisher","first-page":"415","DOI":"10.1016\/S1364-6613(03)00197-9","article-title":"Persistent activity in the prefrontal cortex during working memory","volume":"7","author":"Curtis","year":"2003","journal-title":"Trends Cogn Sci."},{"key":"B55","doi-asserted-by":"publisher","first-page":"101686","DOI":"10.1016\/j.nicl.2019.101686","article-title":"Patients with sickle-cell disease exhibit greater functional connectivity and centrality in the locus coeruleus compared to anemic controls","volume":"21","author":"Bhatt","year":"2019","journal-title":"NeuroImage Clin."},{"key":"B56","doi-asserted-by":"publisher","first-page":"712","DOI":"10.1016\/S0022-3476(05)80844-3","article-title":"Neuropsychologic effects of stroke in children with sickle cell anemia","volume":"123","author":"Craft","year":"1993","journal-title":"J Pediatr."},{"key":"B57","doi-asserted-by":"publisher","first-page":"2177","DOI":"10.1093\/brain\/awl160","article-title":"Impact of frontal white matter lesions on performance monitoring: ERP evidence for cortical disconnection","volume":"129","author":"Hogan","year":"2006","journal-title":"Brain."},{"key":"B58","doi-asserted-by":"publisher","first-page":"17186","DOI":"10.1073\/pnas.1121251109","article-title":"Experience and the developing prefrontal cortex","volume":"109","author":"Kolb","year":"2012","journal-title":"Proc Natl Acad Sci."},{"key":"B59","doi-asserted-by":"publisher","first-page":"1974","DOI":"10.3389\/fpsyg.2020.01974","article-title":"Training inhibition and social cognition in the classrooms","volume":"11","author":"Honor\u00e9","year":"2020","journal-title":"Front Psychol."},{"key":"B60","doi-asserted-by":"publisher","first-page":"20","DOI":"10.1016\/j.jecp.2009.11.003","article-title":"Investigating the predictive roles of working memory and IQ in academic attainment","volume":"106","author":"Alloway","year":"2010","journal-title":"J Exp Child Psychol."},{"key":"B61","doi-asserted-by":"publisher","first-page":"2693","DOI":"10.1073\/pnas.1010076108","article-title":"A gradient of childhood self-control predicts health, wealth, and public safety","volume":"108","author":"Moffitt","year":"2011","journal-title":"Proc Natl Acad Sci."},{"key":"B62","doi-asserted-by":"publisher","first-page":"380","DOI":"10.1080\/02699931.2010.491647","article-title":"Flexible control in processing affective and non-affective material predicts individual differences in trait resilience","volume":"25","author":"Genet","year":"2011","journal-title":"Cogn Emot."},{"key":"B63","doi-asserted-by":"publisher","first-page":"168","DOI":"10.1017\/S1355617711001469","article-title":"Nocturnal oxygen desaturation and disordered sleep as a potential factor in executive dysfunction in sickle cell anemia","volume":"18","author":"Hollocks","year":"2012","journal-title":"J Int Neuropsychol Soc."},{"key":"B64","doi-asserted-by":"publisher","first-page":"e27823","DOI":"10.1002\/pbc.27823","article-title":"Neurocognitive and psychological effects of persistent pain in pediatric sickle cell disease","volume":"66","author":"Connolly","year":"2019","journal-title":"Pediatr Blood Cancer."},{"key":"B65","doi-asserted-by":"publisher","first-page":"584","DOI":"10.1097\/MPH.0000000000000431","article-title":"Fatigue in children with sickle cell disease: association with neurocognitive and social-emotional functioning and quality of life","volume":"37","author":"Anderson","year":"2015","journal-title":"J Pediatr Hematol Oncol."},{"key":"B66","doi-asserted-by":"publisher","first-page":"185","DOI":"10.1093\/jpepsy\/jsh020","article-title":"Transcranial Doppler ultrasonography and executive dysfunction in children with sickle cell disease","volume":"29","author":"Kral","year":"2004","journal-title":"J Pediatr Psychol."},{"key":"B67","doi-asserted-by":"publisher","first-page":"1830","DOI":"10.1161\/STROKEAHA.120.032741","article-title":"Cerebral hemodynamics and executive function in sickle cell anemia","volume":"52","author":"Prussien K","year":"2021","journal-title":"Stroke."},{"key":"B68","doi-asserted-by":"publisher","first-page":"1160","DOI":"10.1093\/jpepsy\/jsy057","article-title":"Executive functioning mediates the relationship between pain coping and quality of life in youth with sickle cell disease","volume":"43","author":"Ludwig","year":"2018","journal-title":"J Pediatr Psychol."},{"key":"B69","doi-asserted-by":"publisher","first-page":"889","DOI":"10.1080\/09297049.2016.1205011","article-title":"Executive functioning and health-related quality of life in pediatric sickle cell disease","volume":"23","author":"Allen","year":"2017","journal-title":"Child Neuropsychol."},{"key":"B70","doi-asserted-by":"publisher","first-page":"681137","DOI":"10.3389\/fpsyg.2021.681137","article-title":"Biopsychosocial Predictors of Quality of Life in Paediatric Patients With Sickle Cell Disease","volume":"12","author":"Hood","year":"2021","journal-title":"Front Psychol."},{"key":"B71","doi-asserted-by":"publisher","first-page":"35","DOI":"10.1016\/S0301-0511(00)00052-1","article-title":"Aging and measures of processing speed","volume":"54","author":"Salthouse","year":"2000","journal-title":"Biol Psychol."},{"key":"B72","doi-asserted-by":"crossref","DOI":"10.1016\/B978-0-12-822963-7.00047-5","volume-title":"Neurocognitive Testing.","author":"Silva","year":"2021"},{"key":"B73","doi-asserted-by":"publisher","first-page":"51","DOI":"10.1007\/s11065-019-09423-6","article-title":"The factor structure of cognitive functioning in cognitively healthy participants: a meta-analysis and meta-analysis of individual participant data","volume":"30","author":"Agelink van Rentergem","year":"2020","journal-title":"Neuropsychol Rev."},{"key":"B74","doi-asserted-by":"publisher","first-page":"36","DOI":"10.1186\/1824-7288-39-36","article-title":"Intellectual function evaluation of first generation immigrant children with sickle cell disease: the role of language and sociodemographic factors","volume":"39","author":"Montanaro","year":"2013","journal-title":"Ital J Pediatr."},{"key":"B75","doi-asserted-by":"publisher","first-page":"432","DOI":"10.1080\/09297049.2011.613813","article-title":"Executive function performance on the children's kitchen task assessment with children with sickle cell disease and matched controls","volume":"18","author":"Berg","year":"2012","journal-title":"Child Neuropsychol."},{"key":"B76","doi-asserted-by":"publisher","first-page":"892","DOI":"10.1093\/jpepsy\/jsp124","article-title":"Relationships between somatic growth and cognitive functioning in young children with sickle cell disease","volume":"35","author":"Puffer","year":"2010","journal-title":"J Pediatr Psychol."},{"key":"B77","doi-asserted-by":"publisher","first-page":"37","DOI":"10.1177\/08830738060210010701","article-title":"Radiographic predictors of neurocognitive functioning in pediatric sickle cell disease","volume":"21","author":"Kral","year":"2006","journal-title":"J Child Neurol."},{"key":"B78","doi-asserted-by":"publisher","first-page":"1006","DOI":"10.3324\/haematol.2008.005215","article-title":"Auto-adjusting positive airway pressure in children with sickle cell anemia: results of a phase I randomized controlled trial","volume":"94","author":"Marshall","year":"2009","journal-title":"Haematologica."},{"key":"B79","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13063-019-3461-x","article-title":"Prevention of morbidity in sickle cell disease (POMS2a)\u2014overnight auto-adjusting continuous positive airway pressure compared with nocturnal oxygen therapy: a randomised crossover pilot study examining patient preference and safety in adults and children","volume":"20","author":"Howard","year":"2019","journal-title":"Trials."},{"key":"B80","doi-asserted-by":"publisher","first-page":"e181471","DOI":"10.1001\/jamanetworkopen.2018.1471","article-title":"Twenty-year trends in diagnosed attention-deficit\/hyperactivity disorder among US children and adolescents, 1997-2016","volume":"1","author":"Xu","year":"2018","journal-title":"JAMA Netw Open."},{"key":"B81","doi-asserted-by":"publisher","first-page":"1087","DOI":"10.1177\/0009922815594356","article-title":"Risk factors for attention and behavioral issues in pediatric sickle cell disease","volume":"54","author":"Lance","year":"2015","journal-title":"Clin Pediatr."},{"key":"B82","doi-asserted-by":"publisher","first-page":"350","DOI":"10.1097\/MPH.0000000000000847","article-title":"Attention deficit hyperactivity disorder in children with sickle cell disease referred for an evaluation","volume":"39","author":"Acquazzino","year":"2017","journal-title":"J Pediatr Hematol Oncol."},{"key":"B83","doi-asserted-by":"publisher","first-page":"111","DOI":"10.1007\/s11920-011-0177-3","article-title":"Psychiatric diagnosis in adolescents with sickle cell disease: a preliminary report","volume":"13","author":"Benton","year":"2011","journal-title":"Curr Psychiatry Rep."},{"key":"B84","doi-asserted-by":"publisher","first-page":"343","DOI":"10.1080\/19371918.2019.1606754","article-title":"An integrative review of the prevalence of depression among pediatric patients with sickle cell disease","volume":"34","author":"Moody","year":"2019","journal-title":"Soc Work Public Health."},{"key":"B85","doi-asserted-by":"publisher","first-page":"e20161878","DOI":"10.1542\/peds.2016-1878","article-title":"National trends in the prevalence and treatment of depression in adolescents and young adults","volume":"138","author":"Mojtabai","year":"2016","journal-title":"Pediatrics."},{"key":"B86","doi-asserted-by":"publisher","first-page":"595","DOI":"10.1016\/j.jaac.2019.10.010","article-title":"Systematic review: anxiety in children and adolescents with chronic medical conditions","volume":"59","author":"Cobham","year":"2019","journal-title":"J Am Acad Child Adolesc Psychiatry."},{"key":"B87","doi-asserted-by":"publisher","first-page":"53","DOI":"10.1186\/s40359-016-0162-z","article-title":"Prevalence of psychological symptoms among adults with sickle cell disease in Korle-Bu Teaching Hospital, Ghana","volume":"4","author":"Anim","year":"2016","journal-title":"BMC Psychol."},{"key":"B88","doi-asserted-by":"publisher","first-page":"192","DOI":"10.1097\/PSY.0b013e31815ff5c5","article-title":"Depression and anxiety in adults with sickle cell disease: the PiSCES project","volume":"70","author":"Levenson","year":"2008","journal-title":"Psychosom Med."},{"key":"B89","doi-asserted-by":"publisher","first-page":"723","DOI":"10.1111\/j.1365-2141.2005.05500.x","article-title":"Psychological complications in sickle cell disease","volume":"129","author":"Anie","year":"2005","journal-title":"Br J Haematol."},{"key":"B90","doi-asserted-by":"publisher","first-page":"364","DOI":"10.1207\/s15327558ijbm0404_7","article-title":"Cognitive coping skills training in children with sickle cell disease pain","volume":"4","author":"Gil","year":"1997","journal-title":"Int J Behav Med."},{"key":"B91","doi-asserted-by":"publisher","first-page":"109","DOI":"10.1023\/A:1014940009788","article-title":"Longitudinal assessment of pain, coping, and daily functioning in children with sickle cell disease receiving pain management skills training","volume":"9","author":"Powers","year":"2002","journal-title":"J Clin Psychol Med Settings."},{"key":"B92","doi-asserted-by":"publisher","first-page":"209","DOI":"10.1348\/135910799168588","article-title":"Cognitive-behaviour therapy for the management of sickle cell disease pain: an evaluation of a community-based intervention","volume":"4","author":"Thomas","year":"1999","journal-title":"Br J Health Psychol."},{"key":"B93","doi-asserted-by":"publisher","first-page":"6","DOI":"10.1111\/j.1744-6155.2001.tb00240.x","article-title":"An intervention to increase coping and reduce health care utilization for school-age children and adolescents with sickle cell disease","volume":"12","author":"Broome","year":"2001","journal-title":"J Natl Black Nurses Assoc JNBNA."},{"key":"B94","doi-asserted-by":"publisher","first-page":"536","DOI":"10.1097\/AJP.0000000000000183","article-title":"Changes in coping, pain and activity following cognitive-behavioral training: a randomized clinical trial for pediatric sickle cell disease using smartphones","volume":"31","author":"Schatz","year":"2015","journal-title":"Clin J Pain."},{"key":"B95","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1007\/s40167-017-0050-2","article-title":"Bias in cross-cultural neuropsychological testing: problems and possible solutions","volume":"6","author":"Fern\u00e1ndez","year":"2018","journal-title":"Cult Brain."},{"key":"B96","doi-asserted-by":"publisher","first-page":"1194","DOI":"10.1097\/00005650-200412000-00006","article-title":"Monitoring depression treatment outcomes with the patient health questionnaire-9","volume":"42","author":"L\u00f6we","year":"2004","journal-title":"Med Care."},{"key":"B97","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1159\/000518939","article-title":"Minimal clinically important difference of executive function performance in older adults who fall: a secondary analysis of a randomized controlled trial","volume":"67","author":"Jehu","year":"2021","journal-title":"Gerontology."},{"key":"B98","volume-title":"Child and Parent Reported Outcome Measures: A Scoping Report Focusing on Feasibility for Routine Use in the NHS.","author":"Morris","year":"2009"},{"key":"B99","volume-title":"Neuropsychological Assessment.","author":"Lezak","year":"2012"},{"key":"B100","volume-title":"Psychological Testing in the Service of Disability Determination","author":"Committee","year":"2015"},{"key":"B101","doi-asserted-by":"publisher","first-page":"148","DOI":"10.1037\/tep0000155","article-title":"Graduate training implications of the Q-interactive platform for administering Wechsler intelligence tests","volume":"11","author":"Clark","year":"2017","journal-title":"Train Educ Prof Psychol."},{"key":"B102","doi-asserted-by":"publisher","first-page":"261","DOI":"10.18865\/ed.28.S1.261","article-title":"Anti-racism methods for big data research: lessons learned from the HIV testing, linkage, & retention in care (HIV TLR) study","volume":"28","author":"Ford","year":"2018","journal-title":"Ethn Dis."},{"key":"B103","doi-asserted-by":"publisher","first-page":"438","DOI":"10.1080\/13854046.2018.1560501","article-title":"Modern neuropsychological tests for a diversity of cultural contexts","volume":"33","author":"Fernandez","year":"2019","journal-title":"Clin Neuropsychol."},{"key":"B104","doi-asserted-by":"publisher","first-page":"363","DOI":"10.1089\/neu.2016.4443","article-title":"Developing a cognition endpoint for traumatic brain injury clinical trials","volume":"34","author":"Silverberg","year":"2017","journal-title":"J Neurotrauma."},{"key":"B105","doi-asserted-by":"publisher","first-page":"407","DOI":"10.1016\/0197-2456(89)90005-6","article-title":"Measurement of health status: ascertaining the minimal clinically important difference","volume":"10","author":"Jaeschke","year":"1989","journal-title":"Control Clin Trials."},{"key":"B106","doi-asserted-by":"publisher","first-page":"1342","DOI":"10.1001\/jama.2014.13128","article-title":"Minimal clinically important difference: defining what really matters to patients","volume":"312","author":"McGlothlin","year":"2014","journal-title":"Jama."},{"key":"B107","doi-asserted-by":"publisher","first-page":"1096","DOI":"10.1016\/j.jpain.2017.05.001","article-title":"Responsiveness and minimally important differences for 4 patient-reported outcomes measurement information system short forms: physical function, pain interference, depression, and anxiety in knee osteoarthritis","volume":"18","author":"Lee","year":"2017","journal-title":"J Pain."},{"key":"B108","doi-asserted-by":"publisher","first-page":"417","DOI":"10.1200\/OP.20.00275","article-title":"Early impact of COVID-19 on the conduct of oncology clinical trials and long-term opportunities for transformation: findings from an American Society of Clinical Oncology Survey","volume":"16","author":"Waterhouse","year":"2020","journal-title":"JCO Oncol Pract."},{"key":"B109","doi-asserted-by":"publisher","first-page":"e27650","DOI":"10.2196\/27650","article-title":"Engaging caregivers and providers of children with sickle cell anemia in shared decision making for hydroxyurea: protocol for a multicenter randomized controlled trial","volume":"10","author":"Hood","year":"2021","journal-title":"JMIR Res Protoc."},{"key":"B110","doi-asserted-by":"crossref","first-page":"1988","DOI":"10.1007\/978-0-387-79948-3_1139","article-title":"Practice effects","volume-title":"Encyclopedia of Clinical Neuropsychology.","author":"McCabe","year":"2011"},{"key":"B111","doi-asserted-by":"publisher","first-page":"212","DOI":"10.1111\/j.1748-6653.2011.02023.x","article-title":"Test\u2013retest reliability and practice effects of the Wechsler Memory Scale-III","volume":"6","author":"Lo","year":"2012","journal-title":"J Neuropsychol."},{"key":"B112","doi-asserted-by":"publisher","first-page":"103","DOI":"10.1016\/j.dadm.2014.11.003","article-title":"Practice effects due to serial cognitive assessment: implications for preclinical Alzheimer's disease randomized controlled trials","volume":"1","author":"Goldberg","year":"2015","journal-title":"Alzheimers Dement."},{"key":"B113","doi-asserted-by":"publisher","first-page":"419","DOI":"10.1017\/S1355617703930074","article-title":"The effects of practice on the cognitive test performance of neurologically normal individuals assessed at brief test-retest intervals","volume":"9","author":"Collie","year":"2003","journal-title":"J Int Neuropsychol Soc."},{"key":"B114","doi-asserted-by":"publisher","first-page":"955","DOI":"10.1016\/j.trf.2018.06.042","article-title":"Comparing the effectiveness of different methods to reduce the effect size of the practice effect in traffic psychological assessment","volume":"58","author":"Sommer","year":"2018","journal-title":"Transp Res Part F Traffic Psychol Behav."},{"key":"B115","doi-asserted-by":"publisher","first-page":"71","DOI":"10.1076\/chin.8.2.71.8724","article-title":"Assessment and development of executive function (EF) during childhood","volume":"8","author":"Anderson","year":"2002","journal-title":"Child Neuropsychol."},{"key":"B116","doi-asserted-by":"publisher","first-page":"248","DOI":"10.1093\/arclin\/acr120","article-title":"Evidence-based indicators of neuropsychological change in the individual patient: relevant concepts and methods","volume":"27","author":"Duff","year":"2012","journal-title":"Arch Clin Neuropsychol."},{"key":"B117","doi-asserted-by":"publisher","first-page":"1551","DOI":"10.1046\/j.1528-1157.2002.23602.x","article-title":"Determining reliable cognitive change after epilepsy surgery: development of reliable change indices and standardized regression-based change norms for the WMS-III and WAIS-III","volume":"43","author":"Martin","year":"2002","journal-title":"Epilepsia."},{"key":"B118","doi-asserted-by":"publisher","first-page":"142","DOI":"10.1080\/09297040600584626","article-title":"The association of oral hydroxyurea therapy with improved cognitive functioning in sickle cell disease","volume":"13","author":"Puffer","year":"2007","journal-title":"Child Neuropsychol."},{"key":"B119","doi-asserted-by":"publisher","first-page":"274","DOI":"10.1111\/bjh.13819","article-title":"Risk factor analysis of cerebral white matter hyperintensities in children with sickle cell disease","volume":"172","author":"Land","year":"2016","journal-title":"Br J Haematol."},{"key":"B120","doi-asserted-by":"publisher","first-page":"373","DOI":"10.1111\/bjh.12496","article-title":"Subcortical and cerebellar volumetric deficits in paediatric sickle cell anaemia","volume":"163","author":"Kawadler","year":"2013","journal-title":"Br J Haematol."},{"key":"B121","doi-asserted-by":"publisher","first-page":"1793","DOI":"10.1161\/STROKEAHA.115.008721","article-title":"White matter damage relates to oxygen saturation in children with sickle cell anemia without silent cerebral infarcts","volume":"46","author":"Kawadler","year":"2015","journal-title":"Stroke."},{"key":"B122","doi-asserted-by":"publisher","first-page":"864","DOI":"10.1542\/peds.97.6.864","article-title":"Cognitive functioning and brain magnetic resonance imaging in children with sickle cell disease","volume":"97","author":"Armstrong","year":"1996","journal-title":"Pediatrics."},{"key":"B123","doi-asserted-by":"publisher","first-page":"1055","DOI":"10.1002\/ajh.25570","article-title":"Anemia predicts lower white matter volume and cognitive performance in sickle and non-sickle cell anemia syndrome","volume":"94","author":"Choi","year":"2019","journal-title":"Am J Hematol."},{"key":"B124","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1080\/13854046.2020.1847325","article-title":"Executive function in children with sickle cell anemia on transfusion: NIH toolbox utility in the clinical context","volume":"62","author":"Downes","year":"2020","journal-title":"Clin Neuropsychol."},{"key":"B125","doi-asserted-by":"publisher","first-page":"151","DOI":"10.1016\/j.advms.2016.09.002","article-title":"Brain morphometric analysis predicts decline of intelligence quotient in children with sickle cell disease: a preliminary study","volume":"62","author":"Chen","year":"2017","journal-title":"Adv Med Sci."},{"key":"B126","doi-asserted-by":"crossref","DOI":"10.3389\/fneur.2022.867329","article-title":"Quantification of silent cerebral infarction on high-resolution FLAIR and cognition in sickle cell anemia","volume-title":"Front Neurol.","author":"Stotesbury","year":""},{"key":"B127","doi-asserted-by":"publisher","first-page":"490","DOI":"10.1016\/j.jpain.2016.12.016","article-title":"AAPT diagnostic criteria for chronic sickle cell disease pain","volume":"18","author":"Dampier","year":"2017","journal-title":"J Pain."},{"key":"B128","doi-asserted-by":"publisher","first-page":"360","DOI":"10.1111\/bjh.15841","article-title":"Index of pain experience in sickle cell anaemia (IPESCA): development from daily pain diaries and initial findings from use with children and adults with sickle cell anaemia","volume":"186","author":"Kawadler","year":"2019","journal-title":"Br J Haematol."},{"key":"B129","doi-asserted-by":"publisher","first-page":"1179","DOI":"10.1016\/j.jclinepi.2010.04.011","article-title":"The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005\u20132008","volume":"63","author":"Cella","year":"2010","journal-title":"J Clin Epidemiol."},{"key":"B130","doi-asserted-by":"publisher","first-page":"374","DOI":"10.5664\/jcsm.27880","article-title":"Polysomnographic characteristics of a referred sample of children with sickle cell disease","volume":"6","author":"Rogers","year":"2010","journal-title":"J Clin sleep Med JCSM."},{"key":"B131","doi-asserted-by":"publisher","first-page":"273","DOI":"10.1542\/peds.2013-4223","article-title":"Obstructive sleep apnea and sickle cell anemia","volume":"134","author":"Rosen","year":"2014","journal-title":"Pediatrics."},{"key":"B132","doi-asserted-by":"publisher","first-page":"244","DOI":"10.3109\/03630269.2014.919941","article-title":"Assessment of sleep-related disorders in children with sickle cell disease","volume":"38","author":"Hankins","year":"2014","journal-title":"Hemoglobin."},{"key":"B133","doi-asserted-by":"publisher","first-page":"207","DOI":"10.1186\/1471-244X-14-207","article-title":"Sleep disturbance, depression and pain in adults with sickle cell disease","volume":"14","author":"Wallen","year":"2014","journal-title":"BMC Psychiatry."},{"key":"B134","doi-asserted-by":"publisher","first-page":"1572","DOI":"10.1016\/j.juro.2012.02.021","article-title":"Enuresis associated with sleep disordered breathing in children with sickle cell anemia","volume":"188","author":"Lehmann","year":"2012","journal-title":"J Urol."},{"key":"B135","doi-asserted-by":"publisher","first-page":"154","DOI":"10.1111\/bjh.14811","article-title":"Periodic limb movement in sleep and sickle cell disease: a neglected association?","volume":"179","author":"Caba\u00f1as-Pedro","year":"2017","journal-title":"Br J Haematol."},{"key":"B136","doi-asserted-by":"publisher","first-page":"3607","DOI":"10.1002\/ppul.25632","article-title":"The burden of sleep disordered breathing in children with sickle cell disease","volume":"56","author":"Liguoro","year":"2021","journal-title":"Pediatr Pulmonol."},{"key":"B137","doi-asserted-by":"publisher","first-page":"981","DOI":"10.3390\/brainsci10120981","article-title":"Developmental profile of sleep and its potential impact on daytime functioning from childhood to adulthood in sickle cell anaemia","volume":"10","author":"K\u00f6lbel","year":"2020","journal-title":"Brain Sci."},{"key":"B138","doi-asserted-by":"publisher","first-page":"755","DOI":"10.1080\/02643290701754158","article-title":"A time to think: circadian rhythms in human cognition","volume":"24","author":"Schmidt","year":"2007","journal-title":"Cogn Neuropsychol."},{"key":"B139","doi-asserted-by":"publisher","first-page":"835","DOI":"10.4103\/2230-8210.117225","article-title":"Importance of achieving the composite endpoints in diabetes","volume":"17","author":"Unnikrishnan","year":"2013","journal-title":"Indian J Endocrinol Metab."},{"key":"B140","doi-asserted-by":"crossref","DOI":"10.1016\/B978-0-12-804217-5.00002-3","volume-title":"Clinical Trials: Study Design, Endpoints and Biomarkers, Drug Safety, and FDA and ICH Guidelines.","author":"Brody","year":"2016"},{"key":"B141","doi-asserted-by":"publisher","first-page":"312","DOI":"10.1093\/arclin\/acz070","article-title":"Developing an executive functioning composite score for research and clinical trials","volume":"35","author":"Iverson","year":"2020","journal-title":"Arch Clin Neuropsychol."},{"key":"B142","doi-asserted-by":"publisher","first-page":"517","DOI":"10.1007\/s11682-012-9176-1","article-title":"A composite score for executive functioning, validated in Alzheimer's Disease Neuroimaging Initiative (ADNI) participants with baseline mild cognitive impairment","volume":"6","author":"Gibbons","year":"2012","journal-title":"Brain Imaging Behav."},{"key":"B143","doi-asserted-by":"publisher","first-page":"59","DOI":"10.1348\/026151003321164627","article-title":"Dimensions of executive functioning: evidence from children","volume":"21","author":"Lehto","year":"2003","journal-title":"Br J Dev Psychol."},{"key":"B144","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1017\/S1355617713001239","article-title":"EXAMINER executive function battery and neurologic morbidity in pediatric sickle cell disease","volume":"20","author":"Schatz","year":"2014","journal-title":"J Int Neuropsychol Soc."},{"key":"B145","doi-asserted-by":"publisher","first-page":"473","DOI":"10.1080\/17474086.2021.1893687","article-title":"MRI detection of brain abnormality in sickle cell disease","volume":"14","author":"Stotesbury","year":"2021","journal-title":"Expert Rev Hematol."}],"container-title":["Frontiers in Neurology"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fneur.2022.835823\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,2,8]],"date-time":"2023-02-08T21:52:12Z","timestamp":1675893132000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fneur.2022.835823\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,6,21]]},"references-count":145,"alternative-id":["10.3389\/fneur.2022.835823"],"URL":"https:\/\/doi.org\/10.3389\/fneur.2022.835823","relation":{},"ISSN":["1664-2295"],"issn-type":[{"value":"1664-2295","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,6,21]]},"article-number":"835823"}}