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

Morna J. Dorsey; Nicola A.M. Wright; Natalia S. Chaimowitz; Blachy J. Dávila Saldaña; Holly Miller; Michael D. Keller; Monica S. Thakar; Ami J. Shah; Rolla Abu-Arja; Jeffrey Andolina; Victor Aquino; J. L. Barnum; Jeffrey J. Bednarski; Monica Bhatia; Francisco A. Bonilla; Manish J. Butte; Nancy J. Bunin; Sharat Chandra; Sonali Chaudhury; Karin Chen; Hey Chong; Geoffrey D.E. Cuvelier; Jignesh Dalal; Magee L. DeFelice; Kenneth B. DeSantes; Lisa R. Forbes; Alfred Gillio; Fred Goldman; Avni Y. Joshi; Neena Kapoor; Alan P. Knutsen; Lisa Kobrynski; Jay A. Lieberman; Jennifer W. Leiding; Benjamin Oshrine; Kiran P. Patel; Susan Prockop; Troy C. Quigg; Ralph Quinones; Kirk R. Schultz; Christine Seroogy; David Shyr; Subhadra Siegel; Angela R. Smith; Troy R. Torgerson; Mark T. Vander Lugt; Lolie C. Yu; Morton J. Cowan; Rebecca H. Buckley; Christopher C. Dvorak; Linda M. Griffith; Elie Haddad; Donald B. Kohn; Brent Logan; Luigi D. Notarangelo; Sung Yun Pai; Jennifer Puck; Michael A. Pulsipher; Jennifer Heimall

doi:10.1007/s10875-020-00865-9

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

Morna J. Dorsey, Nicola A.M. Wright, Natalia S. Chaimowitz, Blachy J. Dávila Saldaña, Holly Miller, Michael D. Keller, Monica S. Thakar, Ami J. Shah, Rolla Abu-Arja, Jeffrey Andolina, Victor Aquino, J. L. Barnum, Jeffrey J. Bednarski, Monica Bhatia, Francisco A. Bonilla, Manish J. Butte, Nancy J. Bunin, Sharat Chandra, Sonali Chaudhury, Karin ChenHey Chong, Geoffrey D.E. Cuvelier, Jignesh Dalal, Magee L. DeFelice, Kenneth B. DeSantes, Lisa R. Forbes, Alfred Gillio, Fred Goldman, Avni Y. Joshi, Neena Kapoor, Alan P. Knutsen, Lisa Kobrynski, Jay A. Lieberman, Jennifer W. Leiding, Benjamin Oshrine, Kiran P. Patel, Susan Prockop, Troy C. Quigg, Ralph Quinones, Kirk R. Schultz, Christine Seroogy, David Shyr, Subhadra Siegel, Angela R. Smith, Troy R. Torgerson, Mark T. Vander Lugt, Lolie C. Yu, Morton J. Cowan, Rebecca H. Buckley, Christopher C. Dvorak, Linda M. Griffith, Elie Haddad, Donald B. Kohn, Brent Logan, Luigi D. Notarangelo, Sung Yun Pai, Jennifer Puck, Michael A. Pulsipher, Jennifer Heimall

Research output: Contribution to journal › Article › peer-review

34 Scopus citations

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.

Original language	English (US)
Pages (from-to)	38-50
Number of pages	13
Journal	Journal of Clinical Immunology
Volume	41
Issue number	1
DOIs	https://doi.org/10.1007/s10875-020-00865-9
State	Published - Jan 2021

Keywords

Infections
hematopoietic stem cell transplant
newborn screening
primary immunodeficiency
prophylaxis
severe combined immunodeficiency

ASJC Scopus subject areas

Immunology and Allergy
Immunology

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10.1007/s10875-020-00865-9

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Dorsey, M. J., Wright, N. A. M., Chaimowitz, N. S., Dávila Saldaña, B. J., Miller, H., Keller, M. D., Thakar, M. S., Shah, A. J., Abu-Arja, R., Andolina, J., Aquino, V., Barnum, J. L., Bednarski, J. J., Bhatia, M., Bonilla, F. A., Butte, M. J., Bunin, N. J., Chandra, S., Chaudhury, S., ... Heimall, J. (2021). Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers. Journal of Clinical Immunology, 41(1), 38-50. https://doi.org/10.1007/s10875-020-00865-9

Dorsey, MJ, Wright, NAM, Chaimowitz, NS, Dávila Saldaña, BJ, Miller, H, Keller, MD, Thakar, MS, Shah, AJ, Abu-Arja, R, Andolina, J, Aquino, V, Barnum, JL, Bednarski, JJ, Bhatia, M, Bonilla, FA, Butte, MJ, Bunin, NJ, Chandra, S, Chaudhury, S, Chen, K, Chong, H, Cuvelier, GDE, Dalal, J, DeFelice, ML, DeSantes, KB, Forbes, LR, Gillio, A, Goldman, F, Joshi, AY, Kapoor, N, Knutsen, AP, Kobrynski, L, Lieberman, JA, Leiding, JW, Oshrine, B, Patel, KP, Prockop, S, Quigg, TC, Quinones, R, Schultz, KR, Seroogy, C, Shyr, D, Siegel, S, Smith, AR, Torgerson, TR, Vander Lugt, MT, Yu, LC, Cowan, MJ, Buckley, RH, Dvorak, CC, Griffith, LM, Haddad, E, Kohn, DB, Logan, B, Notarangelo, LD, Pai, SY, Puck, J, Pulsipher, MA & Heimall, J 2021, 'Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers', Journal of Clinical Immunology, vol. 41, no. 1, pp. 38-50. https://doi.org/10.1007/s10875-020-00865-9

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

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{\textquoteright}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.",

keywords = "Infections, hematopoietic stem cell transplant, newborn screening, primary immunodeficiency, prophylaxis, severe combined immunodeficiency",

author = "Dorsey, {Morna J.} and Wright, {Nicola A.M.} and Chaimowitz, {Natalia S.} and {D{\'a}vila Salda{\~n}a}, {Blachy J.} and Holly Miller and Keller, {Michael D.} and Thakar, {Monica S.} and Shah, {Ami J.} and Rolla Abu-Arja and Jeffrey Andolina and Victor Aquino and Barnum, {J. L.} and Bednarski, {Jeffrey J.} and Monica Bhatia and Bonilla, {Francisco A.} and Butte, {Manish J.} and Bunin, {Nancy J.} and Sharat Chandra and Sonali Chaudhury and Karin Chen and Hey Chong and Cuvelier, {Geoffrey D.E.} and Jignesh Dalal and DeFelice, {Magee L.} and DeSantes, {Kenneth B.} and Forbes, {Lisa R.} and Alfred Gillio and Fred Goldman and Joshi, {Avni Y.} and Neena Kapoor and Knutsen, {Alan P.} and Lisa Kobrynski and Lieberman, {Jay A.} and Leiding, {Jennifer W.} and Benjamin Oshrine and Patel, {Kiran P.} and Susan Prockop and Quigg, {Troy C.} and Ralph Quinones and Schultz, {Kirk R.} and Christine Seroogy and David Shyr and Subhadra Siegel and Smith, {Angela R.} and Torgerson, {Troy R.} and {Vander Lugt}, {Mark T.} and Yu, {Lolie C.} and Cowan, {Morton J.} and Buckley, {Rebecca H.} and Dvorak, {Christopher C.} and Griffith, {Linda M.} and Elie Haddad and Kohn, {Donald B.} and Brent Logan and Notarangelo, {Luigi D.} and Pai, {Sung Yun} and Jennifer Puck and Pulsipher, {Michael A.} and Jennifer Heimall",

note = "Publisher Copyright: {\textcopyright} 2020, Springer Science+Business Media, LLC, part of Springer Nature.",

year = "2021",

month = jan,

doi = "10.1007/s10875-020-00865-9",

language = "English (US)",

volume = "41",

pages = "38--50",

journal = "Journal of Clinical Immunology",

issn = "0271-9142",

publisher = "Springer New York",

number = "1",

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TY - JOUR

T1 - Infections in Infants with SCID

T2 - Isolation, Infection Screening, and Prophylaxis in PIDTC Centers

AU - Dorsey, Morna J.

AU - Wright, Nicola A.M.

AU - Chaimowitz, Natalia S.

AU - Dávila Saldaña, Blachy J.

AU - Miller, Holly

AU - Keller, Michael D.

AU - Thakar, Monica S.

AU - Shah, Ami J.

AU - Abu-Arja, Rolla

AU - Andolina, Jeffrey

AU - Aquino, Victor

AU - Barnum, J. L.

AU - Bednarski, Jeffrey J.

AU - Bhatia, Monica

AU - Bonilla, Francisco A.

AU - Butte, Manish J.

AU - Bunin, Nancy J.

AU - Chandra, Sharat

AU - Chaudhury, Sonali

AU - Chen, Karin

AU - Chong, Hey

AU - Cuvelier, Geoffrey D.E.

AU - Dalal, Jignesh

AU - DeFelice, Magee L.

AU - DeSantes, Kenneth B.

AU - Forbes, Lisa R.

AU - Gillio, Alfred

AU - Goldman, Fred

AU - Joshi, Avni Y.

AU - Kapoor, Neena

AU - Knutsen, Alan P.

AU - Kobrynski, Lisa

AU - Lieberman, Jay A.

AU - Leiding, Jennifer W.

AU - Oshrine, Benjamin

AU - Patel, Kiran P.

AU - Prockop, Susan

AU - Quigg, Troy C.

AU - Quinones, Ralph

AU - Schultz, Kirk R.

AU - Seroogy, Christine

AU - Shyr, David

AU - Siegel, Subhadra

AU - Smith, Angela R.

AU - Torgerson, Troy R.

AU - Vander Lugt, Mark T.

AU - Yu, Lolie C.

AU - Cowan, Morton J.

AU - Buckley, Rebecca H.

AU - Dvorak, Christopher C.

AU - Griffith, Linda M.

AU - Haddad, Elie

AU - Kohn, Donald B.

AU - Logan, Brent

AU - Notarangelo, Luigi D.

AU - Pai, Sung Yun

AU - Puck, Jennifer

AU - Pulsipher, Michael A.

AU - Heimall, Jennifer

PY - 2021/1

Y1 - 2021/1

N2 - 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.

AB - 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.

KW - Infections

KW - hematopoietic stem cell transplant

KW - newborn screening

KW - primary immunodeficiency

KW - prophylaxis

KW - severe combined immunodeficiency

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ER -

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

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