Alexis A. Thompson

Advancing Gene Editing for Children with Hemoglobinopathies

Alexis A. Thompson

us (code pays fourni par la source)

How Parents Navigate Hydroxyurea Adherence for Their Young Children with Sickle Cell Disease

Abigail Robbertz, Aimee K. Hildenbrand, Rogelle TA Hackworth, Charles T. Quinn et autres

Abstract Background Despite the benefits, hydroxyurea adherence in pediatric patients with sickle cell disease (SCD) remains low (Creary et al., 2019, Reeves et al., 2023), which may indicate adherence barriers at the patient, family, and healthcare systems levels (Treadwell et al., 2022). …

us (code pays fourni par la source)

Secretory phospholipase A2 (sPLA2) during vaso-occlusive pain episodes (VOE) and acute chest syndrome (ACS) in sickle cell disease (SCD): results from a prospective multi-center randomized controlled trial (RCT)

Noor Alzraikat, Rawan Korman, Fahd Ahmad, Gladstone Airewele et autres

Abstract Background Acute chest syndrome (ACS) is a leading cause of morbidity and mortality in children with sickle cell disease (SCD), often developing during vaso-occlusive episodes (VOE) with minimal early signs. Most children present with a normal lung exam, making early detection …

us (code pays fourni par la source)

Using Electronic-Medical Records to Facilitate Transition From Pediatric to Adult Hematology for Sickle Cell Disease

Venée N. Tubman, Abdullah Kutlar, Albert C Hergenroeder, Alexis A. Thompson et autres

Abstract Background Transition of care from pediatric to adult hematology for adolescents and young adults (AYA) with sickle cell disease (SCD) is a complex process and occurs during a critical time in both disease progression and life. In SCD, the death rates …

us (code pays fourni par la source)

ASH's commitment to SCD: building on 10 years of progress

Alexander A. Boucher, Payal Desai, Chancellor E. Donald, Titilope Fasipe et autres

us (code pays fourni par la source)

Hospital Variations in Time‐To‐Crisis‐Resolution Among Children and Adolescents With Sickle Cell Disease

Chris A. Rees, Dunia Hatabah, Rawan Korman, Fahd A. Ahmad et autres

Analysis of the placebo cohort in the STArT trial shows that patient characteristics and hospital site strongly influence time-to-crisis-resolution and total opioid use in children and young adults with sickle cell disease, highlighting the need to account for these factors in the …

us (code pays fourni par la source)

End-of-study results from the ICON3 pines trial, a phase 3, randomized trial of eltrombopag vs. standard first-line treatment for newly diagnosed immune thrombocytopenia in children

Kristin A. Shimano, Amanda B. Grimes, Melissa J. Rose, Shipra Kaicker et autres

Abstract Background: The Pediatric ITP Newly diagnosed patients Eltrombopag vs Standard therapy (PINES) trial, NCT03939637, was an investigator-initiated, prospective, open label, randomized, multi-center trial sponsored by the ITP Consortium of North America (ICON) and funded by Novartis. Patients (pts) ages 1-<18 with …

us, ca (code pays fourni par la source)

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study

Ashish Gupta, Akshay Sharma, Haydar Frangoul, Julie Kanter et autres

Abstract Introduction Elevated levels of fetal hemoglobin (HbF) ameliorate sickle cell disease (SCD) manifestations. BEAM-101 is an investigational cell therapy comprising ex vivo base-edited autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that directly target the HBG1/2 promoters to disrupt BCL11A binding, …

us, es (code pays fourni par la source)

Revisiting acute chest syndrome (ACS) associated with sickle cell disease (SCD) vaso-occlusive pain episodes (VOE): Insight from a prospective, multicenter Phase-3 randomized controlled trial

Dunia Hatabah, Fahd A. Ahmad, Gladstone Airewele, Bolanle Akinsola et autres

Abstract Background: ACS occurs in up to 20% of hospitalized patients with SCD-VOE, often prolonging and complicating hospital stay. Despite its clinical significance, variations exist in the management and outcomes of ACS across institutions. Objective: Determine prevalence of ACS in children and …

us (code pays fourni par la source)

The psychometric properties of the stoplight pain scale in children with sickle cell disease

Amy L. Drendel, Daniel S. Tsze, Samina Ali, Fahd A. Ahmad et autres

Abstract Background: Effective pain management begins with accurate pain assessment for children with sickle cell disease (SCD) experiencing a vaso-occlusive episodes (VOE). While most scales measure pain, these traditional tools do not guide therapeutic intervention directly and rely on a healthcare provider …

us, ca, gb, es (code pays fourni par la source)

The ASH consa consortium on newborn screening in Africa for sickle cell disease: Interim updates of progress in screening, follow-up and supporting initiatives

Obiageli Nnodu, Bernard Awuonda, Lulu Chirande, Charles Kiyaga et autres

Abstract Introduction: Children born with sickle cell disease (SCD) in sub-Saharan Africa (SSA) are at >50% risk for under age 5 mortality (U5M). To demonstrate whether newborn SCD screening in SSA and early entry into standard care will lower rates of U5M, …

Nigéria, Kenya, Tanzanie, Ouganda, Ghana, Zambie, us, mx, ca, Afrique du Sud (code pays fourni par la source)

Sickle cell disease treatment with arginine therapy (STArT) – results of a phase-3 randomized controlled trial

Claudia R. Morris, Fahd A. Ahmad, Gladstone Airewele, Bolanle Akinsola et autres

Abstract Background: Vaso-occlusive pain episodes (VOE) are the leading cause of emergency department (ED) visits and hospitalizations for patients with sickle cell disease (SCD). Yet, FDA-approved drugs for SCD-VOE are lacking. During SCD-VOE, patients develop an acute arginine (Arg) deficiency that is …

us (code pays fourni par la source)