Early outcomes for low-risk pediatric heart transplant recipients and steroid avoidance: A multicenter cohort study (Clinical Trials in Organ Transplantation in Children - CTOTC-04)

for the CTOTC-04 investigators

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Abstract

BACKGROUND: Immunosuppression strategies have changed over time in pediatric heart transplantation. Thus, comorbidity profiles may have evolved. Clinical Trials in Organ Transplantation in Children-04 is a multicenter, prospective, cohort study assessing the impact of pre-transplant sensitization on outcomes after pediatric heart transplantation. This sub-study reports 1-year outcomes among recipients without pre-transplant donor-specific antibodies (DSAs). METHODS: We recruited consecutive candidates (<21 years) at 8 centers. Sensitization status was determined by a core laboratory. Immunosuppression was standardized as follows: Thymoglobulin induction with tacrolimus and/or mycophenolate mofetil maintenance. Steroids were not used beyond 1 week. Rejection surveillance was by serial biopsy. RESULTS: There were 240 transplants. Subjects for this sub-study (n = 186) were non-sensitized (n = 108) or had no DSAs (n = 78). Median age was 6 years, 48.4% were male, and 38.2% had congenital heart disease. Patient survival was 94.5% (95% confidence interval, 90.1–97.0%). Freedom from any type of rejection was 67.5%. Risk factors for rejection were older age at transplant and presence of non-DSAs pre-transplant. Freedom from infection requiring hospitalization/intravenous anti-microbials was 75.4%. Freedom from rehospitalization was 40.3%. New-onset diabetes mellitus and post-transplant lymphoproliferative disorder (PTLD) occurred in 1.6% and 1.1% of subjects, respectively. There was no decline in renal function over the first year. Corticosteroids were used in 14.5% at 1 year. CONCLUSIONS: Pediatric heart transplantation recipients without DSAs at transplant and managed with a steroid avoidance regimen have excellent short-term survival and a low risk of first-year diabetes mellitus and PTLD. Rehospitalization remains common. These contemporary observations allow for improved caregiver and/or patient counseling and provide the necessary outcomes data to help design future randomized controlled trials.

Original languageEnglish (US)
JournalJournal of Heart and Lung Transplantation
DOIs
StatePublished - Jan 1 2019

Fingerprint

Organ Transplantation
Multicenter Studies
Cohort Studies
Steroids
Clinical Trials
Pediatrics
Transplants
Heart Transplantation
Lymphoproliferative Disorders
Antibodies
Tissue Donors
Immunosuppression
Diabetes Mellitus
Mycophenolic Acid
Survival
Tacrolimus
Transplant Recipients
Caregivers
Comorbidity
Counseling

Keywords

  • immunosuppression
  • outcomes
  • pediatric heart transplant
  • rejection
  • steroid avoidance

ASJC Scopus subject areas

  • Surgery
  • Pulmonary and Respiratory Medicine
  • Cardiology and Cardiovascular Medicine
  • Transplantation

Cite this

@article{ca3f01aae9d24d06908b9ff615fffd6d,
title = "Early outcomes for low-risk pediatric heart transplant recipients and steroid avoidance: A multicenter cohort study (Clinical Trials in Organ Transplantation in Children - CTOTC-04)",
abstract = "BACKGROUND: Immunosuppression strategies have changed over time in pediatric heart transplantation. Thus, comorbidity profiles may have evolved. Clinical Trials in Organ Transplantation in Children-04 is a multicenter, prospective, cohort study assessing the impact of pre-transplant sensitization on outcomes after pediatric heart transplantation. This sub-study reports 1-year outcomes among recipients without pre-transplant donor-specific antibodies (DSAs). METHODS: We recruited consecutive candidates (<21 years) at 8 centers. Sensitization status was determined by a core laboratory. Immunosuppression was standardized as follows: Thymoglobulin induction with tacrolimus and/or mycophenolate mofetil maintenance. Steroids were not used beyond 1 week. Rejection surveillance was by serial biopsy. RESULTS: There were 240 transplants. Subjects for this sub-study (n = 186) were non-sensitized (n = 108) or had no DSAs (n = 78). Median age was 6 years, 48.4{\%} were male, and 38.2{\%} had congenital heart disease. Patient survival was 94.5{\%} (95{\%} confidence interval, 90.1–97.0{\%}). Freedom from any type of rejection was 67.5{\%}. Risk factors for rejection were older age at transplant and presence of non-DSAs pre-transplant. Freedom from infection requiring hospitalization/intravenous anti-microbials was 75.4{\%}. Freedom from rehospitalization was 40.3{\%}. New-onset diabetes mellitus and post-transplant lymphoproliferative disorder (PTLD) occurred in 1.6{\%} and 1.1{\%} of subjects, respectively. There was no decline in renal function over the first year. Corticosteroids were used in 14.5{\%} at 1 year. CONCLUSIONS: Pediatric heart transplantation recipients without DSAs at transplant and managed with a steroid avoidance regimen have excellent short-term survival and a low risk of first-year diabetes mellitus and PTLD. Rehospitalization remains common. These contemporary observations allow for improved caregiver and/or patient counseling and provide the necessary outcomes data to help design future randomized controlled trials.",
keywords = "immunosuppression, outcomes, pediatric heart transplant, rejection, steroid avoidance",
author = "{for the CTOTC-04 investigators} and Lamour, {Jacqueline M.} and Mason, {Kristen L.} and Hsu, {Daphne T.} and Brian Feingold and Blume, {Elizabeth D.} and Canter, {Charles E.} and Dipchand, {Anne I.} and Shaddy, {Robert E.} and Mahle, {William T.} and Zuckerman, {Warren A.} and Carol Bentlejewski and Armstrong, {Brian D.} and Yvonne Morrison and Helena Diop and Ikl{\'e}, {David N.} and Jonah Odim and Adriana Zeevi and Webber, {Steven A.}",
year = "2019",
month = "1",
day = "1",
doi = "10.1016/j.healun.2019.06.006",
language = "English (US)",
journal = "Journal of Heart and Lung Transplantation",
issn = "1053-2498",
publisher = "Elsevier USA",

}

TY - JOUR

T1 - Early outcomes for low-risk pediatric heart transplant recipients and steroid avoidance

T2 - A multicenter cohort study (Clinical Trials in Organ Transplantation in Children - CTOTC-04)

AU - for the CTOTC-04 investigators

AU - Lamour, Jacqueline M.

AU - Mason, Kristen L.

AU - Hsu, Daphne T.

AU - Feingold, Brian

AU - Blume, Elizabeth D.

AU - Canter, Charles E.

AU - Dipchand, Anne I.

AU - Shaddy, Robert E.

AU - Mahle, William T.

AU - Zuckerman, Warren A.

AU - Bentlejewski, Carol

AU - Armstrong, Brian D.

AU - Morrison, Yvonne

AU - Diop, Helena

AU - Iklé, David N.

AU - Odim, Jonah

AU - Zeevi, Adriana

AU - Webber, Steven A.

PY - 2019/1/1

Y1 - 2019/1/1

N2 - BACKGROUND: Immunosuppression strategies have changed over time in pediatric heart transplantation. Thus, comorbidity profiles may have evolved. Clinical Trials in Organ Transplantation in Children-04 is a multicenter, prospective, cohort study assessing the impact of pre-transplant sensitization on outcomes after pediatric heart transplantation. This sub-study reports 1-year outcomes among recipients without pre-transplant donor-specific antibodies (DSAs). METHODS: We recruited consecutive candidates (<21 years) at 8 centers. Sensitization status was determined by a core laboratory. Immunosuppression was standardized as follows: Thymoglobulin induction with tacrolimus and/or mycophenolate mofetil maintenance. Steroids were not used beyond 1 week. Rejection surveillance was by serial biopsy. RESULTS: There were 240 transplants. Subjects for this sub-study (n = 186) were non-sensitized (n = 108) or had no DSAs (n = 78). Median age was 6 years, 48.4% were male, and 38.2% had congenital heart disease. Patient survival was 94.5% (95% confidence interval, 90.1–97.0%). Freedom from any type of rejection was 67.5%. Risk factors for rejection were older age at transplant and presence of non-DSAs pre-transplant. Freedom from infection requiring hospitalization/intravenous anti-microbials was 75.4%. Freedom from rehospitalization was 40.3%. New-onset diabetes mellitus and post-transplant lymphoproliferative disorder (PTLD) occurred in 1.6% and 1.1% of subjects, respectively. There was no decline in renal function over the first year. Corticosteroids were used in 14.5% at 1 year. CONCLUSIONS: Pediatric heart transplantation recipients without DSAs at transplant and managed with a steroid avoidance regimen have excellent short-term survival and a low risk of first-year diabetes mellitus and PTLD. Rehospitalization remains common. These contemporary observations allow for improved caregiver and/or patient counseling and provide the necessary outcomes data to help design future randomized controlled trials.

AB - BACKGROUND: Immunosuppression strategies have changed over time in pediatric heart transplantation. Thus, comorbidity profiles may have evolved. Clinical Trials in Organ Transplantation in Children-04 is a multicenter, prospective, cohort study assessing the impact of pre-transplant sensitization on outcomes after pediatric heart transplantation. This sub-study reports 1-year outcomes among recipients without pre-transplant donor-specific antibodies (DSAs). METHODS: We recruited consecutive candidates (<21 years) at 8 centers. Sensitization status was determined by a core laboratory. Immunosuppression was standardized as follows: Thymoglobulin induction with tacrolimus and/or mycophenolate mofetil maintenance. Steroids were not used beyond 1 week. Rejection surveillance was by serial biopsy. RESULTS: There were 240 transplants. Subjects for this sub-study (n = 186) were non-sensitized (n = 108) or had no DSAs (n = 78). Median age was 6 years, 48.4% were male, and 38.2% had congenital heart disease. Patient survival was 94.5% (95% confidence interval, 90.1–97.0%). Freedom from any type of rejection was 67.5%. Risk factors for rejection were older age at transplant and presence of non-DSAs pre-transplant. Freedom from infection requiring hospitalization/intravenous anti-microbials was 75.4%. Freedom from rehospitalization was 40.3%. New-onset diabetes mellitus and post-transplant lymphoproliferative disorder (PTLD) occurred in 1.6% and 1.1% of subjects, respectively. There was no decline in renal function over the first year. Corticosteroids were used in 14.5% at 1 year. CONCLUSIONS: Pediatric heart transplantation recipients without DSAs at transplant and managed with a steroid avoidance regimen have excellent short-term survival and a low risk of first-year diabetes mellitus and PTLD. Rehospitalization remains common. These contemporary observations allow for improved caregiver and/or patient counseling and provide the necessary outcomes data to help design future randomized controlled trials.

KW - immunosuppression

KW - outcomes

KW - pediatric heart transplant

KW - rejection

KW - steroid avoidance

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UR - http://www.scopus.com/inward/citedby.url?scp=85068969829&partnerID=8YFLogxK

U2 - 10.1016/j.healun.2019.06.006

DO - 10.1016/j.healun.2019.06.006

M3 - Article

C2 - 31324444

AN - SCOPUS:85068969829

JO - Journal of Heart and Lung Transplantation

JF - Journal of Heart and Lung Transplantation

SN - 1053-2498

ER -