Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT

submitted on behalf of the Primary Immune Deficiency Treatment Consortium

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

Introduction: Inflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD on transplant outcomes is lacking. Methods: We collected data retrospectively from 145 patients with CGD who had received allogeneic HCT at 26 Primary Immune Deficiency Treatment Consortium (PIDTC) centers between January 1, 2005 and June 30, 2016. Results: Forty-nine CGD patients with IBD and 96 patients without IBD underwent allogeneic HCT. Eighty-nine percent of patients with IBD and 93% of patients without IBD engrafted (p = 0.476). Upper gastrointestinal acute GVHD occurred in 8.5% of patients with IBD and 3.5% of patients without IBD (p = 0.246). Lower gastrointestinal acute GVHD occurred in 10.6% of patients with IBD and 11.8% of patients without IBD (p = 0.845). The cumulative incidence of acute GVHD grades II–IV was 30% (CI 17–43%) in patients with IBD and 20% (CI 12–29%) in patients without IBD (p = 0.09). Five-year overall survival was equivalent for patients with and without IBD: 80% [CI 66–89%] and 83% [CI 72–90%], respectively (p = 0.689). All 33 surviving evaluable patients with a history of IBD experienced resolution of IBD by 2 years following allogeneic HCT. Conclusions: In this cohort, allogeneic HCT was curative for CGD-associated IBD. IBD should not contraindicate HCT, as it does not lead to an increased risk of mortality. This study is registered at clinicaltrials.gov NCT02082353.

Original languageEnglish (US)
Pages (from-to)653-667
Number of pages15
JournalJournal of Clinical Immunology
Volume39
Issue number7
DOIs
StatePublished - Oct 1 2019
Externally publishedYes

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Chronic Granulomatous Disease
Cell Transplantation
Inflammatory Bowel Diseases
Survival

Keywords

  • allogeneic bone marrow transplantation
  • Allogeneic hematopoietic cell transplantation
  • allogeneic hematopoietic stem cell transplantation
  • chronic granulomatous disease
  • inflammatory bowel disease
  • primary immunodeficiency

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology

Cite this

Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. / submitted on behalf of the Primary Immune Deficiency Treatment Consortium.

In: Journal of Clinical Immunology, Vol. 39, No. 7, 01.10.2019, p. 653-667.

Research output: Contribution to journalArticle

submitted on behalf of the Primary Immune Deficiency Treatment Consortium. / Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. In: Journal of Clinical Immunology. 2019 ; Vol. 39, No. 7. pp. 653-667.
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title = "Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT",
abstract = "Introduction: Inflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD on transplant outcomes is lacking. Methods: We collected data retrospectively from 145 patients with CGD who had received allogeneic HCT at 26 Primary Immune Deficiency Treatment Consortium (PIDTC) centers between January 1, 2005 and June 30, 2016. Results: Forty-nine CGD patients with IBD and 96 patients without IBD underwent allogeneic HCT. Eighty-nine percent of patients with IBD and 93{\%} of patients without IBD engrafted (p = 0.476). Upper gastrointestinal acute GVHD occurred in 8.5{\%} of patients with IBD and 3.5{\%} of patients without IBD (p = 0.246). Lower gastrointestinal acute GVHD occurred in 10.6{\%} of patients with IBD and 11.8{\%} of patients without IBD (p = 0.845). The cumulative incidence of acute GVHD grades II–IV was 30{\%} (CI 17–43{\%}) in patients with IBD and 20{\%} (CI 12–29{\%}) in patients without IBD (p = 0.09). Five-year overall survival was equivalent for patients with and without IBD: 80{\%} [CI 66–89{\%}] and 83{\%} [CI 72–90{\%}], respectively (p = 0.689). All 33 surviving evaluable patients with a history of IBD experienced resolution of IBD by 2 years following allogeneic HCT. Conclusions: In this cohort, allogeneic HCT was curative for CGD-associated IBD. IBD should not contraindicate HCT, as it does not lead to an increased risk of mortality. This study is registered at clinicaltrials.gov NCT02082353.",
keywords = "allogeneic bone marrow transplantation, Allogeneic hematopoietic cell transplantation, allogeneic hematopoietic stem cell transplantation, chronic granulomatous disease, inflammatory bowel disease, primary immunodeficiency",
author = "{submitted on behalf of the Primary Immune Deficiency Treatment Consortium} and Marsh, {Rebecca A.} and Leiding, {Jennifer W.} and Logan, {Brent R.} and Griffith, {Linda M.} and Arnold, {Danielle E.} and Elie Haddad and Falcone, {E. Liana} and Ziyan Yin and Kadam Patel and Erin Arbuckle and Bleesing, {Jack J.} and Sullivan, {Kathleen E.} and Jennifer Heimall and Burroughs, {Lauri M.} and Suzanne Skoda-Smith and Shanmuganathan Chandrakasan and Yu, {Lolie C.} and Oshrine, {Benjamin R.} and Cuvelier, {Geoffrey D.E.} and Thakar, {Monica S.} and Karin Chen and Pierre Teira and Shalini Shenoy and Rachel Phelan and Forbes, {Lisa R.} and Deepak Chellapandian and {D{\'a}vila Salda{\~n}a}, {Blachy J.} and Shah, {Ami J.} and Weinacht, {Katja G.} and Avni Joshi and Farid Boulad and Quigg, {Troy C.} and Dvorak, {Christopher C.} and Debi Grossman and Troy Torgerson and Pamela Graham and Vinod Prasad and Alan Knutsen and Hey Chong and Holly Miller and {de la Morena}, {M. Teresa} and Kenneth DeSantes and Cowan, {Morton J.} and Notarangelo, {Luigi D.} and Kohn, {Donald B.} and Elizabeth Stenger and Pai, {Sung Yun} and Routes, {John M.} and Puck, {Jennifer M.} and Neena Kapoor",
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TY - JOUR

T1 - Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT

AU - submitted on behalf of the Primary Immune Deficiency Treatment Consortium

AU - Marsh, Rebecca A.

AU - Leiding, Jennifer W.

AU - Logan, Brent R.

AU - Griffith, Linda M.

AU - Arnold, Danielle E.

AU - Haddad, Elie

AU - Falcone, E. Liana

AU - Yin, Ziyan

AU - Patel, Kadam

AU - Arbuckle, Erin

AU - Bleesing, Jack J.

AU - Sullivan, Kathleen E.

AU - Heimall, Jennifer

AU - Burroughs, Lauri M.

AU - Skoda-Smith, Suzanne

AU - Chandrakasan, Shanmuganathan

AU - Yu, Lolie C.

AU - Oshrine, Benjamin R.

AU - Cuvelier, Geoffrey D.E.

AU - Thakar, Monica S.

AU - Chen, Karin

AU - Teira, Pierre

AU - Shenoy, Shalini

AU - Phelan, Rachel

AU - Forbes, Lisa R.

AU - Chellapandian, Deepak

AU - Dávila Saldaña, Blachy J.

AU - Shah, Ami J.

AU - Weinacht, Katja G.

AU - Joshi, Avni

AU - Boulad, Farid

AU - Quigg, Troy C.

AU - Dvorak, Christopher C.

AU - Grossman, Debi

AU - Torgerson, Troy

AU - Graham, Pamela

AU - Prasad, Vinod

AU - Knutsen, Alan

AU - Chong, Hey

AU - Miller, Holly

AU - de la Morena, M. Teresa

AU - DeSantes, Kenneth

AU - Cowan, Morton J.

AU - Notarangelo, Luigi D.

AU - Kohn, Donald B.

AU - Stenger, Elizabeth

AU - Pai, Sung Yun

AU - Routes, John M.

AU - Puck, Jennifer M.

AU - Kapoor, Neena

PY - 2019/10/1

Y1 - 2019/10/1

N2 - Introduction: Inflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD on transplant outcomes is lacking. Methods: We collected data retrospectively from 145 patients with CGD who had received allogeneic HCT at 26 Primary Immune Deficiency Treatment Consortium (PIDTC) centers between January 1, 2005 and June 30, 2016. Results: Forty-nine CGD patients with IBD and 96 patients without IBD underwent allogeneic HCT. Eighty-nine percent of patients with IBD and 93% of patients without IBD engrafted (p = 0.476). Upper gastrointestinal acute GVHD occurred in 8.5% of patients with IBD and 3.5% of patients without IBD (p = 0.246). Lower gastrointestinal acute GVHD occurred in 10.6% of patients with IBD and 11.8% of patients without IBD (p = 0.845). The cumulative incidence of acute GVHD grades II–IV was 30% (CI 17–43%) in patients with IBD and 20% (CI 12–29%) in patients without IBD (p = 0.09). Five-year overall survival was equivalent for patients with and without IBD: 80% [CI 66–89%] and 83% [CI 72–90%], respectively (p = 0.689). All 33 surviving evaluable patients with a history of IBD experienced resolution of IBD by 2 years following allogeneic HCT. Conclusions: In this cohort, allogeneic HCT was curative for CGD-associated IBD. IBD should not contraindicate HCT, as it does not lead to an increased risk of mortality. This study is registered at clinicaltrials.gov NCT02082353.

AB - Introduction: Inflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD on transplant outcomes is lacking. Methods: We collected data retrospectively from 145 patients with CGD who had received allogeneic HCT at 26 Primary Immune Deficiency Treatment Consortium (PIDTC) centers between January 1, 2005 and June 30, 2016. Results: Forty-nine CGD patients with IBD and 96 patients without IBD underwent allogeneic HCT. Eighty-nine percent of patients with IBD and 93% of patients without IBD engrafted (p = 0.476). Upper gastrointestinal acute GVHD occurred in 8.5% of patients with IBD and 3.5% of patients without IBD (p = 0.246). Lower gastrointestinal acute GVHD occurred in 10.6% of patients with IBD and 11.8% of patients without IBD (p = 0.845). The cumulative incidence of acute GVHD grades II–IV was 30% (CI 17–43%) in patients with IBD and 20% (CI 12–29%) in patients without IBD (p = 0.09). Five-year overall survival was equivalent for patients with and without IBD: 80% [CI 66–89%] and 83% [CI 72–90%], respectively (p = 0.689). All 33 surviving evaluable patients with a history of IBD experienced resolution of IBD by 2 years following allogeneic HCT. Conclusions: In this cohort, allogeneic HCT was curative for CGD-associated IBD. IBD should not contraindicate HCT, as it does not lead to an increased risk of mortality. This study is registered at clinicaltrials.gov NCT02082353.

KW - allogeneic bone marrow transplantation

KW - Allogeneic hematopoietic cell transplantation

KW - allogeneic hematopoietic stem cell transplantation

KW - chronic granulomatous disease

KW - inflammatory bowel disease

KW - primary immunodeficiency

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U2 - 10.1007/s10875-019-00659-8

DO - 10.1007/s10875-019-00659-8

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C2 - 31376032

AN - SCOPUS:85072546464

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JO - Journal of Clinical Immunology

JF - Journal of Clinical Immunology

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