Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers

Authors

Morna Dorsey, UCSF Benioff Children‘s Hospital
Nicola A.M. Wright, University of Calgary
Natalia S. Chaimowitz, Baylor College of Medicine
Blachy J. Dávila Saldaña, Childrens National Health System
Holly Miller, Phoenix Children’s Hospital
Michael D. Keller, The George Washington University
Monica S. Thakar, University of Washington
Ami J. Shah, Stanford University School of Medicine
Rolla Abu-Arja, Nationwide Children’s Hospital
Jeffrey Andolina, Golisano Children‘s Hospital at Strong
Victor Aquino, The University of Texas at Dallas
J. L. Barnum, University of Pittsburgh Medical Center, Children's Hospital of Pittsburgh
Jeffrey J. Bednarski, Washington University School of Medicine in St. Louis
Monica Bhatia, Columbia University Irving Medical Center
Francisco A. Bonilla, Northeast Allergy, Asthma, & Immunology
Manish J. Butte, University of California, Los Angeles
Nancy J. Bunin, The Children's Hospital of Philadelphia
Sharat Chandra, Cincinnati Children's Hospital Medical Center
Sonali Chaudhury, Northwestern University Feinberg School of Medicine
Karin Chen, University of Utah School of Medicine

Document Type

Article

Publication Date

10-2-2020

Publication Title

Journal of Clinical Immunology

Abstract

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.

First Page

38

Last Page

50

PubMed ID

33006109

Volume

41

Issue

1

Publisher

Springer

Recommended Citation

Dorsey, Morna; Wright, Nicola A.M.; Chaimowitz, Natalia S.; Dávila Saldaña, Blachy J.; Miller, Holly; Keller, Michael D.; Thakar, Monica S.; Shah, Ami J.; Abu-Arja, Rolla; Andolina, Jeffrey; Aquino, Victor; Barnum, J. L.; Bednarski, Jeffrey J.; Bhatia, Monica; Bonilla, Francisco A.; Butte, Manish J.; Bunin, Nancy J.; Chandra, Sharat; Chaudhury, Sonali; and Chen, Karin, "Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers" (2020). School of Medicine Faculty Publications. 293.
https://digitalscholar.lsuhsc.edu/som_facpubs/293
10.1007/s10875-020-00865-9