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

Journal Article (Journal Article)

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 NCT02082353.

Full Text

Duke Authors

Cited Authors

  • Marsh, RA; Leiding, JW; Logan, BR; Griffith, LM; Arnold, DE; Haddad, E; Falcone, EL; Yin, Z; Patel, K; Arbuckle, E; Bleesing, JJ; Sullivan, KE; Heimall, J; Burroughs, LM; Skoda-Smith, S; Chandrakasan, S; Yu, LC; Oshrine, BR; Cuvelier, GDE; Thakar, MS; Chen, K; Teira, P; Shenoy, S; Phelan, R; Forbes, LR; Chellapandian, D; Dávila Saldaña, BJ; Shah, AJ; Weinacht, KG; Joshi, A; Boulad, F; Quigg, TC; Dvorak, CC; Grossman, D; Torgerson, T; Graham, P; Prasad, V; Knutsen, A; Chong, H; Miller, H; de la Morena, MT; DeSantes, K; Cowan, MJ; Notarangelo, LD; Kohn, DB; Stenger, E; Pai, S-Y; Routes, JM; Puck, JM; Kapoor, N; Pulsipher, MA; Malech, HL; Parikh, S; Kang, EM; submitted on behalf of the Primary Immune Deficiency Treatment Consortium,

Published Date

  • October 2019

Published In

Volume / Issue

  • 39 / 7

Start / End Page

  • 653 - 667

PubMed ID

  • 31376032

Pubmed Central ID

  • PMC6754755

Electronic International Standard Serial Number (EISSN)

  • 1573-2592

Digital Object Identifier (DOI)

  • 10.1007/s10875-019-00659-8


  • eng

Conference Location

  • Netherlands