Adding hydroxyurea to chronic transfusion therapy for sickle cell anemia reduces transfusion burden.

Background: Chronic red blood cell (RBC) transfusion is an established therapy to prevent stroke in patients with sickle cell anemia (SCA). It is unclear if adding daily hydroxyurea treatment to chronic transfusion is beneficial.

Study Design And Methods: We conducted a phase 2 clinical trial (NCT03644953) investigating the addition of dose-escalated hydroxyurea to chronic transfusion for patients with SCA receiving simple chronic transfusion for stroke prevention.

Disparity in HLA typing rates among hospitalized pediatric patients with sickle cell disease.

Hematopoietic stem cell transplantation (HSCT) using an HLA-identical sibling donor is a well-established cure for sickle cell disease (SCD). The proportion of patients with SCD who have completed HLA typing, a key first step in considering HSCT, is unknown. We sought to determine the prevalence of HLA typing among patients with SCD hospitalized at our institution, identify characteristics associated with having had typing, and describe the acceptability of this testing.

Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease.

Disease-modifying therapies are standard of care (SOC) for sickle cell disease (SCD), but hematopoietic cell transplantation (HCT) has curative potential. We compared outcomes prospectively through 2-years after biologic assignment to a Donor or No Donor (SOC) Arm based on the availability of an HLA-matched sibling or unrelated donor (BMTCTN 1503; NCT02766465). A donor search was commenced after eligibility confirmation.

Optimising the screening for haemoglobinopathies in pregnancy planning.

Haemoglobinopathies are among the most common inherited disorders around the world. In the United States the diagnosis of haemoglobinopathy or a carrier state is made by universal newborn screening. However, many individuals of childbearing age do not know they are a haemoglobinopathy carrier.

Clinical outcomes of children and adolescents with sickle cell disease and COVID-19 infection: A year in review at a metropolitan tertiary pediatric hospital.

Background: COVID-19 was declared a global pandemic in March 2020. Early reports were primarily in adults, and sickle cell disease (SCD) was classified as a risk factor for severe COVID-19 disease. However, there are a limited number of primarily multi-center studies reporting on the clinical course of pediatric patients with SCD and COVID-19.

Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance.

Hematopoietic cell transplantation (HCT) is an established cure for sickle cell disease (SCD) supported by long-term survival, but long-term organ function data are lacking. We sought to describe organ function and assess predictors for dysfunction in a retrospective cohort (n = 247) through the Sickle cell Transplant Advocacy and Research alliance. Patients with <1-year follow-up or graft rejection/second HCT were excluded.

Reduction in vaso-occlusive events following stem cell transplantation in patients with sickle cell disease.

Hematopoietic stem cell transplantation (HSCT) is potentially curative for patients with sickle cell disease (SCD). Patients with stable donor engraftment after allogeneic HSCT generally do not experience SCD-related complications; however, there are no published data specifically reporting the change in vaso-occlusive events (VOE) after HSCT. Data regarding the number of VOEs requiring medical attention in the 2 years before allogeneic HSCT were compared with the number of VOEs in the 2 years (0-12 months and 12-24 months) after allogeneic HSCT in patients with SCD.

Amino acid signature during sickle cell pain crisis shows significant alterations related to nitric oxide and energy metabolism.

Nitric oxide depletion secondary to arginase induced arginine deficiency has been shown to be important in the pathophysiology of vaso-occlusion in sickle cell pain crisis. Our objective of this study was to perform a comprehensive amino acid evaluation during sickle cell pain crisis. In a total of 58 subjects (29 in steady-state sickle cell disease and 29 with sickle cell pain crisis), the amino acids related to nitric oxide pathway was significantly decreased during sickle cell pain crisis compared to steady-state sickle cell disease: arginine (p = 0.

Fertility after Curative Therapy for Sickle Cell Disease: A Comprehensive Review to Guide Care.

Curative therapy for sickle cell disease (SCD) currently requires gonadotoxic conditioning that can impair future fertility. Fertility outcomes after curative therapy are likely affected by pre-transplant ovarian reserve or semen analysis parameters that may already be abnormal from SCD-related damage or hydroxyurea treatment. Outcomes are also likely affected by the conditioning regimen.

Preventing antibody positive delayed hemolytic transfusion reactions in sickle cell disease: Lessons learned from a case.

Introduction: Red blood cell (RBC) transfusions are important in the management of patients with sickle cell disease (SCD). However, a potentially catastrophic complication of transfusion in this population is the delayed hemolytic transfusion reaction (DHTR). The pathophysiology of all DHTRs is not understood, but some are known to be caused by an anamnestic resurgence of RBC alloantibodies.

Impact of universal irradiation on chronic transfusion for sickle cell disease.

Background: Irradiation of blood products prevents transfusion-associated graft-versus-host disease, but most patients do not require this modification which could have an adverse impact on transfusion outcomes. We hypothesized that irradiation may increase transfusion requirements for patients with sickle cell disease (SCD) receiving chronic transfusion.

Study Design And Methods: Our pediatric hospital implemented a new policy of universal blood product irradiation in May 2018.

Screening for new red blood cell alloantibodies after transfusion in patients with sickle cell disease.

Background: Patients with sickle cell disease (SCD) are frequent recipients of red blood cell (RBC) transfusions and are at risk for RBC alloimmunization. RBC alloimmunization is diagnosed by identifying RBC alloantibodies as part of pre-transfusion testing, but this testing fails to detect alloantibodies that have evanesced. It may be beneficial to screen for new RBC alloantibody development after transfusion before possible antibody evanescence.

Transcranial Doppler Screening in a Current Cohort of Children With Sickle Cell Anemia: Results From the DISPLACE Study.

Stroke prevention guidelines for sickle cell anemia (SCA) recommend transcranial Doppler (TCD) screening to identify children at stroke risk; however, TCD screening implementation remains poor. This report describes results from Part 1 of the 28-site DISPLACE (Dissemination and Implementation of Stroke Prevention Looking at the Care Environment) study, a baseline assessment of TCD implementation rates. This report describes TCD implementation by consortium site characteristics; characteristics of TCDs completed; and TCD results based on age.

The impact of pre-existing HLA and red blood cell antibodies on transfusion support and engraftment in sickle cell disease after nonmyeloablative hematopoietic stem cell transplantation from HLA-matched sibling donors: A prospective, single-center, observational study.

Background: Hematopoietic stem cell transplantation (HSCT) is curative for patients with sickle cell disease (SCD). Prior to HSCT, patients with SCD commonly receive RBC transfusions with some becoming RBC or HLA alloimmunized. This alloimmunization may impact post-HSCT transfusion requirements and donor engraftment.

Parents of Children with Sickle Cell Disease Are Interested in Preimplantation Genetic Testing.

Objective: To evaluate awareness of and attitudes toward preimplantation genetic testing (PGT) for sickle cell disease (SCD) among parents of children with SCD.

Study Design: Parents of children with SCD were given an educational handbook on PGT before a routine SCD clinic visit. After their clinic visit, parents were asked to complete an anonymous survey.

Parvovirus B19 infection in sickle cell disease: An analysis from the Centers for Disease Control haemoglobinopathy blood surveillance project.

Objective: In the multicentre Haemoglobinopathy Blood Surveillance Project, to evaluate the seroprevalence of parvovirus B19 and DNA viral load in sickle cell disease (SCD).

Background: Although the epidemiology of parvovirus B19 seropositivity in SCD has been well documented, there are few studies that have assessed possible persistent parvovirus DNAemia and associated risk factors including blood transfusion.

Methods: A qualitative analysis of parvovirus B19 serology using ELISA and quantitative parvovirus B19 DNA by RT-PCR was performed in patients with SCD.

Characteristics and outcomes of osteomyelitis in children with sickle cell disease: A 10-year single-center experience.

Background: Patients with sickle cell disease (SCD) are at increased risk for osteomyelitis (OM). Diagnosis of OM in SCD is challenging as the clinical presentation is similar to a vasoocclusive crisis (VOC) with no diagnostic gold standard. We report characteristics and outcomes of OM in SCD patients treated at our center over 10-year period.

Transcranial Doppler Screening Adherence among Children with Sickle Cell Anemia Seen in the Emergency Department.

Objective: To evaluate adherence to annual transcranial Doppler ultrasound (TCD) screening to prevent stroke among patients with sickle cell anemia (SCA) seen in the emergency department (ED).

Study Design: This retrospective chart review included patients with SCA seen at a large pediatric ED over 64 weeks. Patients who did not need a TCD (age <2 or ≥16 years, on chronic transfusions, history of an inadequate TCD) or were not followed at the study institution were excluded.

Inpatient Ordering of Home Hydroxyurea by Residents for Hospitalized Patients With Sickle Cell Disease.

Background: Hydroxyurea is a well-established disease-modifying medication for sickle cell disease (SCD). At some institutions, hydroxyurea can only be ordered by "chemotherapy-certified" providers which may not include pediatric resident physicians.

Methods: We conducted a survey of 39 American pediatric hospitals regarding their policy on resident hydroxyurea ordering for SCD.

Human leukocyte antigen (HLA) class I antibodies and transfusion support in paediatric HLA-matched haematopoietic cell transplant for sickle cell disease.

The relevance of donor-specific human leukocyte antigen (HLA) antibodies in HLA-mismatched haematopoietic cell transplant (HCT) is known, but the importance of HLA antibodies in HLA-matched HCT is unclear. We hypothesized that HLA antibodies detected before HCT would cause platelet transfusion refractoriness during HCT and investigated this in a multi-centre study. Pre-HCT samples from 45 paediatric patients with sickle cell disease (SCD) undergoing HLA-matched HCT were tested for HLA class I antibodies.