A Phase 1b, Open-label, Multiple-Dose Study Evaluating Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Pociredir in Patients With Sickle Cell Disease (SCD): Trial Design
To assess safety, pharmacokinetics (PK), pharmacodynamics (PD), and biologic activity of pociredir in patients with SCD, including effects on fetal hemoglobin (HbF) and hemolysis.
Breast cancer screening in women with SCD: a single-center exploratory study
To evaluate breast cancer screening utilization and mammographic characteristics among women living with sickle cell disease (SCD).
Preliminary observations on cerebral blood flow and pain sensitivity in adults with SCD
To explore the relationship between cerebral blood flow (CBF) and pain sensitivity in adults with sickle cell disease (SCD).
An international multicenter cohort shows sickle cell disease is no longer uniformly associated with low body weight, with persistent undernutrition alongside increasing overweight and obesity across age groups, genotypes, and countries.
To characterize body mass index (BMI) distribution and associated clinical factors in individuals with sickle cell disease across diverse global settings.
Elevated ambient nitrogen dioxide (NO₂) levels were associated with greater odds of abnormal pulmonary function tests in children and young adults with sickle cell disease (SCD), highlighting an environmental factor that may influence respiratory health in this population.
To evaluate whether ambient air pollution exposures, particularly criteria pollutants, are associated with abnormal pulmonary function tests (PFTs) among young people living with SCD.
A 5-year-old girl with sickle cell disease (Hb SS) and poor hydroxyurea adherence developed concurrent warm autoimmune hemolytic anemia (wAIHA), splenic sequestration, and hyperhemolysis/delayed hemolytic transfusion reaction after transfusion, illustrating the complexity of immune-mediated hemolysis and the role of multidisciplinary, phenotype-matched intervention.
To present a clinical case illustrating the diagnostic and therapeutic challenges when multiple severe hemolytic complications of SCD converge in a single pediatric patient.
In chronically transfused adults with sickle cell disease (SCD), a retrospective analysis found that a substantial subgroup experienced sleep-associated hemoglobin desaturation (SpO₂ ≤ 88% for ≥5 minutes), which was associated with markers of hemolysis and inflammation, underscoring the need to consider sleep-related hypoxemia in comprehensive SCD care.
To characterize the prevalence and clinical correlates of nocturnal hemoglobin desaturation (NHD) in adults with SCD managed with chronic red blood cell exchange transfusion.
A pooled analysis of Phase 2 and Phase 3 clinical trials provides a detailed safety profile of crizanlizumab 5.0 mg/kg in sickle cell disease (SCD), showing predominantly mild to moderate adverse events alongside infrequent but notable serious infections and bleeding events.
To evaluate the safety of crizanlizumab 5.0 mg/kg by pooling adverse-event data across multiple clinical trials in individuals with SCD.
A retrospective analysis of Medicaid-insured children with sickle cell disease (SCD) found that those with longer continuous coverage had significantly better adherence to oral Hydroxyurea (HU), underscoring the role of stable insurance and continuity in optimizing SCD therapy.
To investigate whether the length of continuous Medicaid enrollment (12- vs. 24-month coverage) affects adherence to hydroxyurea among children with SCD.
Advanced neuroimaging in adults with sickle cell disease (SCD) revealed elevated cerebral blood flow (CBF), reduced cortical amplitude of low-frequency fluctuation (ALFF), increased white-matter ALFF, and altered functional connectivity — patterns associated with slower cognitive processing and higher pain sensitivity.
To characterize CBF, ALFF, and resting-state functional connectivity in adults with SCD compared with matched healthy controls, and relate these measures to cognition and pain sensitivity.
This study describes 46 children and adolescents with SCD who met criteria for high-impact chronic pain (HICP) at the time of hematopoietic cell transplant (HCT) consultation, highlighting significant pain burden, healthcare utilization, and clinical complexity within this referred subgroup.
To characterize the clinical phenotype of HICP among children and adolescents with SCD referred for HCT consultation, including pain frequency, healthcare utilization (HCU), analgesic exposure, and comorbidities such as AVN.
A preclinical study in humanized sickle-cell mice found that older animals exhibited heightened evoked and non-evoked pain-related behaviors along with age-associated gene-expression changes data that may help inform future research on aging and pain in SCD.
To examine how increasing age influences pain-related behaviors and associated gene-expression patterns in a humanized sickle cell disease mouse model (HbSS-BERK).
Adults with sickle cell shared visual and narrative reflections in a virtual photovoice study, highlighting how pain and self-care shape their daily lives
To capture and interpret adult perspectives on living with sickle cell (SCD) through a virtual photovoice methodology, with the aim of informing patient-centred clinical practice and care design.
A feasibility study in adults with sickle cell disease (SCD) found high acceptability and successful collection of salivary stress biomarkers, paving the way for research linking psychosocial stress and biological responses.
To assess the acceptability and practical feasibility of collecting salivary specimens for stress biomarkers (cortisol, alpha-amylase) in people living with SCD.
Interviews with 19 older adults with sickle cell disease (SCD) reveals aging challenges and wisdom for management of sickle cell and healthy aging.
To describe the aging experiences and priorities of adults with SCD aged ≥50 so clinicians can deliver better care for this growing population.
Study finds improvement in emergency medicine (EM) residents’ knowledge after a 40-minute lecture on sickle cell disease (SCD)
To evaluate whether a brief, structured educational intervention could improve EM residents’ knowledge and perceptions about SCD.
National Alliance of Sickle Cell Centers (NASCC) practice recommendations establish standardized criteria for sickle cell disease (SCD) care delivery
NASCC used a systematic, modified RAND/UCLA Delphi method to develop consensus-driven clinical practice recommendations for SCD. This process incorporated expert judgment alongside data from the Globin Research Network for Data...
Survey of U.S. Clinicians’ attitudes toward palliative care (PC) to enhance the quality of life for patients with sickle cell disease (SCD)
To assess clinician attitudes, referral practices, and barriers affecting the integration of PC in SCD management.
Studies Indicate That Dalcetrapib Shows in Vitro Antisickling Effects and Strong RBC Partitioning for β-chain Cys93 Allosteric Regulation of Hemoglobin
Review preclinical and translational evidence for targeting β-chain Cysteine-93 (βCys93) as an allosteric regulator of hemoglobin affinity and as a potential antisickling strategy distinct from α-chain modulators (e.g., voxelotor).
Study Finds Rare Atypical Bacteria in Pediatric SCD ACS, Underscoring Limited Utility of Empiric Macrolides
To determine the prevalence of atypical bacterial pathogens in children with sickle cell disease (SCD) presenting with acute chest syndrome (ACS) or pneumonia.
Study Suggests Psychological Stress Destabilizes Blood Pressure Control in Sickle Cell Disease, Potentially Contributing to Vaso-Occlusive Crises
To investigate whether hydroxyurea (HU) modulates expression of transcriptional repressors that silence fetal γ-globin genes, thereby clarifying the molecular mechanism of HbF induction.
Hydroxyurea Decreases BCL11A and ZBTB7A/LRF Expression in Erythroid Cells, Including Human Progenitors, Providing Support in Raising Fetal Hemoglobin Levels in Sickle Cell Disease
To investigate whether hydroxyurea (HU) modulates expression of transcriptional repressors that silence fetal γ-globin genes, thereby clarifying the molecular mechanism of HbF induction.
Neutrophil Phagocytic Activity Found Impaired in A Study of Sickle Cell Patients With Transfusion History
To compare the ability of neutrophils from individuals with sickle cell anemia (SCA) versus controls to perform phagocytosis (uptake of foreign particles), and to assess how transfusion exposure affects neutrophil...
Study Offers Preliminary Validity for Functional Status-Based Pain Assessment (FSPA) in Adults Hospitalized With Sickle Cell Disease Vaso-Occlusive Crises.
Evaluate preliminary validity of a daily functional status pain assessment (FSPA) tool for adults with sickle cell disease (SCD) during vaso-occlusive crises (VOCs), with the aim of enhancing discharge readiness...
Study Finds Specific Biomarkers to Predict Sickle Cell Anemia Disease Severity
To identify hematological, biochemical, and metabolic biomarkers associated with sickle cell anemia (SCA) severity and to assess machine learning models' ability to predict disease severity accurately.
Study Indicates Potential Link Between Chronic Transfusion and Higher Nocturnal Hemoglobin Desaturation (NHD) Rates in Adults With SCD
Assess prevalence of nocturnal hemoglobin desaturation (NHD) among adults with severe sickle cell disease (SCD) who receive chronic red blood cell exchange (RCE) transfusions. NHD related complications can significantly affect...
Study Indicates Adverse Childhood Experiences (ACEs) are Associated With a Higher Prevalence of Asthma Among Children and Adolescents With Sickle Cell Disease.
To examine the relationship between Adverse Childhood Experiences (ACEs) and asthma among children and adolescents with Sickle Cell Disease (SCD).
Children with sickle cell disease may experience nocturnal hypoxemia even without sleep apnea.
Retrospective study reviewed 81 pediatric SCD patients referred for polysomnography to explore factors associated with nocturnal hypoxemia (NH), independent of obstructive sleep apnea (OSA).
Recent report suggests buprenorphine may offer a viable option for chronic pain management in sickle cell disease during vaso-occlusive crises.
A recent study explored real-world experiences from a single-center case series of adults with sickle cell disease transitioned from full opioid agonists to buprenorphine for chronic pain management.
Pain Management and a Prepared Workforce
Optimal sickle cell disease treatment requires a multidisciplinary team delivering comprehensive care.