Early engraftment and immune kinetics following allogeneic transplant using a novel reduced-toxicity transplant strategy in children/adolescents with high-risk transfusion-dependent thalassemia: Early results of the ThalFAbS trial.

Background: Allogeneic transplant for patients with transfusion-dependent thalassemia is challenging once there has been iron overload and chronic transfusion support.

Objective(s): A transplant strategy that reduced intensity of the preparative regimen and tailored immunosuppression to both support donor engraftment and prevent GVHD was developed for this population. The combination of a pretransplant immunosuppression phase with reduced dosing of fludarabine/prednisone, treosulfan-based preparative regimen with reduced cyclophosphamide dosing, and introduction of a calcineurin/methotrexate-free GVHD prophylaxis/engraftment supporting regimen with abatacept/sirolimus/ATG was tested.

Impact of transcranial Doppler screening on stroke prevention in children and adolescents with sickle cell disease: A systematic review and meta-analysis.

Background: Children with sickle cell disease (SCD) have increased stroke risk, identifiable by elevated velocities on transcranial Doppler (TCD). This review assessed the impact of TCD screening on stroke, mortality, quality of life and morbidity in children with SCD.

Methods: A systematic search of MEDLINE, PubMed, Cochrane libraries, and trial registries was conducted from inception to 28th February 2023.

Transfusions, disease-modifying treatments, and curative therapies for sickle cell anemia in Africa: where are we now?

The mortality burden of sickle cell anemia (SCA) is centered in sub-Saharan Africa. In addition to a lack of systematic programs for early diagnosis, access to disease-modifying treatments is limited to only a few urban centers. Providing a safe and adequate blood supply is a major challenge, heightening mortality from SCA-associated complications that require urgent blood transfusion and making the delivery of regular transfusion therapy for stroke prevention nonfeasible.

Point-of-care diagnostic test accuracy in children and adolescents with sickle cell disease: A systematic review and meta-analysis.

Background: Detection of sickle cell disease (SCD) could be improved with greater use of point-of-care testing (POCT). This review assessed the accuracy of POCTs for SCD in children and adolescents.

Methods: We systematically searched EMBASE, PubMed, Cochrane libraries, registries and conference proceedings from inception to 28th February 2023.