Long term outcomes of severe combined immunodeficiency: therapy implications.

ABSTRACT:

Introduction

Newborn screening has led to a better understanding of the prevalence of Severe Combined Immunodeficiency (SCID) overall and in terms of specific genotypes. Survival has improved following hematopoietic stem cell transplantation (HCT) with the best outcomes seen following use of a matched sibling donor. However, questions remain regarding the optimal alternative donor source, appropriate use of conditioning and the impact of these decisions on immune reconstitution and other late morbidities. Areas covered: The currently available literature reporting late effects after HCT for SCID and use of alternative therapies including enzyme replacement, alternative donors and gene therapy are reviewed. A literature search was performed on Pubmed and ClinicalTrials.gov using key

SUBMITTER: Heimall J 

PROVIDER: S-EPMC6019104 | biostudies-literature | 2017 Nov

REPOSITORIES: biostudies-literature