Treatment strategy for severe combined immunodeficiency in children

Abstract

Background. Severe Combined Immunodeficiency (SCID) is a rare but severe pediatric disease that is marked by a severe deficiency of T and B lymphocytes, which results in a high susceptibility to infections. Objective of the study. The purpose of this research is to assess current developments in management of SCID in children by analyzing key trends, clinical findings, and difficulties from academic publications published between 2019 and 2024. Material and methods. A literature review with a primary focus on clinical trials, meta-analyses, and peer-reviewed articles from 2019 to 2024 was conducted. The search terms “Severe Combined Immunodeficiency”, “SCID”, “pediatric immunodeficiency,” “gene therapy,” and “hematopoietic stem cell transplantation” were combined to search PubMed, MEDLINE, and the Cochrane Library. Results. Increased use of newborn screening allows for early diagnosis in these patients. Protective isolation, precautions related to transfusion of blood products, avoidance of live vaccines, and continuous prophylactic anti-infectious treatment should be included in initial management before a definitive diagnosis is made. The gold standard for curative care is still hematopoietic stem cell transplantation (HSCT), with better results now possible because of developments in donor matching and conditioning regimens. Gene therapy as an alternative has emerged, offering the potential for curative therapy for specific genetic variants of SCID. Studies indicate that gene therapy has a high success rate in correcting immune deficiencies. Conclusion. Newborn screening, HSCT advancements, and gene therapy have made significant advances in understanding and treating SCID in the previous five years. However, chronic illnesses need continuing research and international cooperation owing to treatment availability and long-term management difficulties.