Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Jennifer W. Leiding, Johns Hopkins University, Baltimore, MD
Danielle E. Arnold, National Institutes of Health, Bethesda, MD
Suhag Parikh, Emory University and Children's Healthcare of Atlanta, Atlanta, GA
Brent Logan, Medical College of Wisconsin, Milwaukee, WI
Rebecca A. Marsh, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH
Linda M. Griffith, National Institutes of Health, Bethesda, MD
Ruizhe Wu, Medical College of Wisconsin, Milwaukee, WI
Sharon Kidd, UCSF Benioff Children's Hospital, San Francisco, CA
Kanwaldeep Mallhi, University of Washington, and Seattle Children's Hospital, Seattle, WA
Deepak Chellapandian, Johns Hopkins All Children's Hospital, St Petersburg, FL
Stephanie J. Si Lim, University of Hawai'i at Mānoa, Honolulu, HI
Eyal Grunebaum, The Hospital for Sick Children, Toronto, ON, Canada
E Liana Falcone, Montreal Clinical Research Institute, Montreal, QC, Canada
Luis Murguia-Favela, University of Calgary, Calgary, AB, Canada
Debbi Grossman, National Institutes of Health, Bethesda, MD
Vinod K. Prasad, Duke University Medical Center, Durham, NC
Jennifer R. Heimall, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA
Fabien Touzot, University of Montreal, Montreal, QC, Canada
Lauri M. Burroughs, University of Washington, and Seattle Children's Hospital, Seattle, WA
Jack Bleesing, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH
Neena Kapoor, Children's Hospital, Los Angeles, CA
Jasmeen Dara, UCSF Benioff Children's Hospital, San Francisco, CA
Olatundun Williams, Northwestern University, Chicago, IL
Malika Kapadia, Dana-Farber Cancer Institute, Boston, MA
Benjamin R. Oshrine, Johns Hopkins All Children's Hospital, St Petersburg, FL
Jeffrey J. Bednarski, Washington University, St. Louis Children's Hospital, St. Louis, MO
Ahmad Rayes, University of Utah Spencer Fox Eccles School of Medicine, Salt Lake City, UT
Hey Chong, UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA
Lolie C. Yu, LSU Health Sciences Center - New OrleansFollow
et al

Document Type

Article

Publication Date

12-14-2023

Publication Title

Blood

Abstract

Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by life-threatening infections and inflammatory conditions. Hematopoietic cell transplantation (HCT) is the definitive treatment for CGD, but questions remain regarding patient selection and impact of active disease on transplant outcomes. We performed a multi-institutional retrospective and prospective study of 391 patients with CGD treated either conventionally (non-HCT) enrolled from 2004 to 2018 or with HCT from 1996 to 2018. Median follow-up after HCT was 3.7 years with a 3-year overall survival of 82% and event-free survival of 69%. In a multivariate analysis, a Lansky/Karnofsky score Age, genotype, and oxidase status did not affect outcomes. Before HCT, patients had higher infection density, higher frequency of noninfectious lung and liver diseases, and more steroid use than conventionally treated patients; however, these issues did not adversely affect HCT survival. Presence of pre-HCT inflammatory conditions was associated with chronic graft-versus-host disease. Graft failure or receipt of a second HCT occurred in 17.6% of the patients and was associated with melphalan-based conditioning and/or early mixed chimerism. At 3 to 5 years after HCT, patients had improved growth and nutrition, resolved infections and inflammatory disease, and lower rates of antimicrobial prophylaxis or corticosteroid use compared with both their baseline and those of conventionally treated patients. HCT leads to durable resolution of CGD symptoms and lowers the burden of the disease. Patients with active infection or inflammation are candidates for transplants; HCT should be considered before the development of comorbidities that could affect performance status. This trial was registered at www.clinicaltrials.gov as #NCT02082353.

First Page

2105

Last Page

2118

PubMed ID

37562003

Volume

142

Issue

24

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