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Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers
Morna J Dorsey1, Nicola A M Wright2, Natalia S Chaimowitz3
1Division of Pediatric Allergy, Immunology, & Bone Marrow Transplant, Benioff Children's Hospital, University of California San Francisco, San Francisco, CA, USA.
Insights
Infants with severe combined immunodeficiency (SCID) diagnosed by newborn screening (NBS) face more infections before hematopoietic stem cell transplant (HSCT). Evidence-based guidelines are needed for pre-HSCT management following NBS.
Area of Science:
- Immunology
- Pediatric Hematology
- Transplantation Medicine
Background:
- Severe combined immunodeficiency (SCID) is a group of rare genetic disorders that affect the immune system.
- Newborn screening (NBS) for SCID aims to enable early diagnosis and treatment, typically hematopoietic stem cell transplant (HSCT).
- Infections remain a significant concern for infants with SCID prior to HSCT, regardless of diagnosis method.
Purpose of the Study:
- To determine the incidence and types of infections in SCID patients before HSCT, comparing those diagnosed via NBS versus family history (FH).
- To assess the variability in pre-HSCT management strategies employed by the Primary Immune Deficiency Treatment Consortium (PIDTC) centers.
- To identify areas for improvement in infection prevention protocols for SCID infants.
Main Methods:
- Retrospective analysis of infection data and pre-transplant management in SCID patients treated with HSCT between 2010-2014.
- Survey of PIDTC centers in 2018 to gather information on their pre-HSCT management practices and protocols.
- Comparison of infection rates based on diagnosis method (NBS vs. FH) and management setting (outpatient vs. inpatient).
Main Results:
- Patients diagnosed with SCID via NBS experienced higher rates of infection (55%) before HSCT compared to those diagnosed via FH (19%).
- The setting of care (outpatient vs. inpatient) did not significantly impact infection rates prior to HSCT.
- While immunoglobulin replacement and antimicrobial prophylaxis were consistently used, other practices like isolation varied widely among centers.
Conclusions:
- SCID diagnosis through NBS is associated with a higher risk of pre-HSCT infections compared to FH diagnosis.
- Significant variability exists in pre-HSCT management protocols across different treatment centers.
- There is a critical need for standardized, evidence-based guidelines for managing infants with SCID after NBS to optimize HSCT outcomes.
Purpose:
The Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children with severe combined immunodeficiency (SCID) in a prospective natural history study of hematopoietic stem cell transplant (HSCT) outcomes over the last decade. Despite newborn screening (NBS) for SCID, infections occurred prior to HSCT. This study's objectives were to define the types and timing of infection prior to HSCT in patients diagnosed via NBS or by family history (FH) and to understand the breadth of strategies employed at PIDTC centers for infection prevention.
Methods:
We analyzed retrospective data on infections and pre-transplant management in patients with SCID diagnosed by NBS and/or FH and treated with HSCT between 2010 and 2014. PIDTC centers were surveyed in 2018 to understand their practices and protocols for pre-HSCT management.
Results:
Infections were more common in patients diagnosed via NBS (55%) versus those diagnosed via FH (19%) (p = 0.012). Outpatient versus inpatient management did not impact infections (47% vs 35%, respectively; p = 0.423). There was no consensus among PIDTC survey respondents as to the best setting (inpatient vs outpatient) for pre-HSCT management. While isolation practices varied, immunoglobulin replacement and antimicrobial prophylaxis were more uniformly implemented.
Conclusion:
Infants with SCID diagnosed due to FH had lower rates of infection and proceeded to HSCT more quickly than did those diagnosed via NBS. Pre-HSCT management practices were highly variable between centers, although uses of prophylaxis and immunoglobulin support were more consistent. This study demonstrates a critical need for development of evidence-based guidelines for the pre-HSCT management of infants with SCID following an abnormal NBS.
Trial Registration:
NCT01186913.
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