Journal
JOURNAL OF CLINICAL IMMUNOLOGY
Volume 41, Issue 1, Pages 38-50Publisher
SPRINGER/PLENUM PUBLISHERS
DOI: 10.1007/s10875-020-00865-9
Keywords
Infections; hematopoietic stem cell transplant; newborn screening; primary immunodeficiency; prophylaxis; severe combined immunodeficiency
Categories
Funding
- Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD
- Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Bethesda, MD
- Public Health Service grant [U54-AI082973, U54-NS064808, U01-TR001263, R13-AI094943]
- Division of Intramural Research, NIAID, NIH
- PBMTC Operations Center (NHLBI/NCI) [U54-AI082973, U54-NS064808, U01-TR001263, R13-AI094943, U10HL069254]
- Johnny Cristopher Children's Charitable Foundation St. Baldrick's Consortium Grant
- National Cancer Institute (NCI), NIH [U24-CA76518]
- NHLBI, NIH [U24-CA76518]
- NIAID, NIH [U24-CA76518]
- NHLBI [U01HL069294]
- NCI [U01HL069294]
- Health Resources and Services Administration (HRSA/DHHS) [HHSH250201200016C, HHSH234200637015C]
- Office of Naval Research [N00014-13-1-0039, N00014-14-1-0028]
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The study found that patients diagnosed via NBS had higher infection rates compared to those diagnosed via FH, with no significant difference in infection rates between outpatient and inpatient management. PIDTC centers had varied pre-HSCT management practices, but there was more consistency in the use of immunoglobulin replacement and antimicrobial prophylaxis.
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.
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