Late effects following hematopoietic cell transplantation for severe combined immunodeficiency: critical factors and therapeutic options.

Abstract excerpt

INTRODUCTION: Severe combined immunodeficiency (SCID) is an inborn error of immunity that is fatal without hematopoietic cell transplantation (HCT) or gene therapy (GT). Survival outcomes have improved, largely due to implementation of SCID newborn screening. A better understanding of the long-term outcomes and late effects to address critical aspects of monitoring immune and general health life-long is needed....

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