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.

Computer-assisted evaluation of retinal vessel tortuosity in children with sickle cell disease without retinopathy.

Objective: We assessed the predictive efficacy of automatically quantified retinal vascular tortuosity from the fundus pictures of patients with sickle cell disease (SCD) without evident retinopathy.

Methods: Retinal images were obtained from 31 healthy and 31 SCD participants using fundus imaging and analyzed using a novel computational automated metric assessment. The local and global vessel tortuosity and their relationship with systemic disease parameters were analyzed based on the images.

Optical Coherence Tomography Angiography of Macular Microangiopathy in Children With Sickle Cell Disease.

In this study, we identified the presence of sickle cell maculopathy and determined correlations between hemolysis indicators and systemic and ocular manifestations in children with sickle cell disease (SCD). Thirty-three patients with SCD 5 to 18 years of age underwent optical coherence tomography angiography (OCTA) as part of a thorough eye examination. The hematological indices and clinical data (hemoglobin, fetal hemoglobin, mean corpuscular volume, and frequency of sickle cell-associated complications and therapy) were collected.

Abatacept-based Graft-Versus-Host Disease Prophylaxis in Haplo-identical Hematopoietic Cell Transplant in a High-risk Cohort.

Background: There is insufficient guidance in using posttransplant cyclophosphamide in patients with organ dysfunctions. Abatacept (Aba), a T cell costimulation blockade, has recently been shown to prevent severe acute graft-versus-host disease (GVHD).

Observation: We report adding Aba as GVHD prophylaxis in 4 pediatrics patients who received haplo-hematopoietic cell transplantation.

Outcome of Haploidentical Peripheral Blood Allografts Using Post-Transplantation Cyclophosphamide Compared to Matched Sibling and Unrelated Donor Bone Marrow Allografts in Pediatric Patients with Hematologic Malignancies: A Single-Center Analysis.

The introduction of post-transplantation cyclophosphamide (PTCy) as graft-versus-host disease (GVHD) prophylaxis has made haploidentical (haplo) hematopoietic stem cell transplantation (HSCT) a common approach in adults, but pediatric experience is limited. Based on the encouraging adult data and with the aim of decreasing the risk of graft failure, our center is increasingly using peripheral blood stem cells (PBSCs) from haplo donors with PTCy. Here we compare outcomes of bone marrow (BM) transplantation with traditional donor choices, including matched sibling donors (MSDs) and 10/10 HLA matched unrelated donors (MUDs), with those of haplo PBSC grafts in pediatric patients with hematologic malignancies.