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.

[Acute blindness in a patient with metformin-associated lactic acidosis].

Lactic acidosis is a rare metabolic complication that can occur in patients with diabetes mellitus type 2 who use metformin. We discuss a 79-year old woman with metformin-associated lactic acidosis (MALA) and acute kidney injury based on gastroenteritis. Patient reported acute blindness which in literature is described as a rare presentation of a metabolic acidosis (regardless of its underlying cause).

User-Centered Design and Usability of Voxe as a Pediatric Electronic Patient-Reported Outcome Measure Platform: Mixed Methods Evaluation Study.

Background: Electronic patient-reported outcome measures (ePROMs) are standardized digital instruments integrated into clinical care to collect subjective data regarding patients' health-related quality of life, functional status, and symptoms. In documenting patient-reported progress, ePROMs can guide treatment decisions and encourage measurement-based care practices. Voxe is a pediatric and user-centered ePROM platform for patients with chronic health conditions.