Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A  Primary Immune Deficiency Treatment Consortium study.

Autor: Grunebaum E; Division of Immunology and Allergy, Hospital for Sick Children, Toronto, Ontario, Canada. Electronic address: eyal.grunebaum@sickkids.ca., Arnold DE; Immune Deficiency-Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, Bethesda, Md., Logan B; Division of Biostatistics, Institute for Health and Equity, Medical College of Wisconsin, Milwaukee, Wis; Center for International Blood and Marrow Transplant Research, Milwaukee, Wis., Parikh S; Department of Pediatrics, Emory University School of Medicine, Atlanta, Ga; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Ga., Marsh RA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Pharming Healthcare Inc, Warren, NJ., Griffith LM; Division of Allergy, Immunology, and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Mallhi K; Seattle Children's Hospital, The University of Washington School of Medicine, Fred Hutchinson Cancer Research Center, Seattle, Wash., Chellapandian D; Cancer and Blood Disorders Institute, Johns Hopkins All Children's Hospital, St Petersburg, Fla., Lim SS; Division of Pediatric Haematology and Oncology, Kapi'olani Medical Center for Women and Children, Honolulu, Hawaii., Deal CL; Division of Allergy and Immunology, University of Pittsburgh Medical Center, Children's Hospital of Pittsburgh, Pittsburgh, Pa., Kapoor N; Transplant and Cell Therapy Program and Laboratory, Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, Calif; Hematology, Oncology, and Transplant and Cell Therapy, Children's Hospital Los Angeles, Los Angeles, Calif., Murguía-Favela L; Section of Hematology/Immunology, Department of Pediatrics, Alberta Children's Hospital Calgary, Calgary, Canada., Falcone EL; Center for Immunity, Inflammation and Infectious Diseases, Montreal Clinical Research Institute, Montréal, Quebec, Canada; Department of Medicine, Université de Montréal, Montréal, Quebec, Canada., Prasad VK; Division of Pediatric Transplant and Cellular Therapy, Duke University Medical Center, Durham, NC., Touzot F; Immunology and Rheumatology Division, Department of Pediatrics, CHU Ste-justine, Universite de Montreal, Montreal, Quebec, Canada., Bleesing JJ; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio., Chandrakasan S; Department of Pediatrics, Emory University School of Medicine, Atlanta, Ga; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Ga., Heimall JR; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pa; Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, Pa., Bednarski JJ; Department of Pediatrics, Washington University School of Medicine, St Louis, Mo., Broglie LA; Center for International Blood and Marrow Transplant Research, Milwaukee, Wis; Department of Pediatrics, Division of Pediatric Hematology, Oncology, Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee., Chong HJ; Division of Allergy and Immunology, University of Pittsburgh Medical Center, Children's Hospital of Pittsburgh, Pittsburgh, Pa., Kapadia M; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Mass; Department of Pediatrics, Harvard Medical School, Boston, Mass., Prockop S; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Mass; Department of Pediatrics, Harvard Medical School, Boston, Mass., Dávila Saldaña BJ; Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, DC; Division of Blood and Marrow Transplantation and Center for Cancer and Immunology Research, Children's National Hospital, Washington, DC., Schaefer E; Division of Pediatric Hematology, Oncology and Stem Cell Transplantation, New York Medical College, Valhalla, NY., Bauchat AL; Division of Pediatric Transplant and Cellular Therapy, Duke University Medical Center, Durham, NC., Teira P; Department of Pediatrics, Immunology and Infectious Diseases, University of Montreal, Montréal, Quebec, Canada; Department of Microbiology, Immunology and Infectious Diseases, Department of Pediatrics, University of Montreal, Montréal, Quebec, Canada; Centre Hospitalier Universitaire Sainte-Justine, University of Montreal, Montréal, Quebec, Canada., Chandra S; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio., Parta M; Division of Blood and Marrow Transplantation and Center for Cancer and Immunology Research, Children's National Hospital, Washington, DC., Cowan MJ; Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif., Dvorak CC; Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif., Haddad E; Department of Pediatrics, Immunology and Infectious Diseases, University of Montreal, Montréal, Quebec, Canada; Department of Microbiology, Immunology and Infectious Diseases, Department of Pediatrics, University of Montreal, Montréal, Quebec, Canada., Kohn DB; Department of Microbiology, Immunology, and Molecular Genetics; Division of Pediatric Hematology/Oncology in the Department of Pediatrics, University of California Los Angeles, Los Angeles, Calif., Notarangelo LD; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Pai SY; Immune Deficiency-Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, Bethesda, Md., Puck JM; Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif., Pulsipher MA; Pediatric Immunology and Blood and Marrow Transplant Program, University of Utah, Salt Lake City, Utah; Intermountain Primary Children's Hospital, Salt Lake City, Utah., Torgerson TR; Experimental Immunology, Allen Institute for Immunology, Seattle, Wash., Malech HL; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Kang EM; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md., Leiding JW; Division of Allergy and Immunology, Department of Pediatrics, Johns Hopkins University, Baltimore, Md.
Jazyk: angličtina
Zdroj: The Journal of allergy and clinical immunology [J Allergy Clin Immunol] 2024 May; Vol. 153 (5), pp. 1423-1431.e2. Date of Electronic Publication: 2024 Jan 28.
DOI: 10.1016/j.jaci.2024.01.013
Abstrakt: Background: P47phox (neutrophil cytosolic factor-1) deficiency is the most common cause of autosomal recessive chronic granulomatous disease (CGD) and is considered to be associated with a milder clinical phenotype. Allogeneic hematopoietic cell transplantation (HCT) for p47phox CGD is not well-described.
Objectives: We sought to study HCT for p47phox CGD in North America.
Methods: Thirty patients with p47phox CGD who received allogeneic HCT at Primary Immune Deficiency Treatment Consortium centers since 1995 were included.
Results: Residual oxidative activity was present in 66.7% of patients. In the year before HCT, there were 0.38 CGD-related infections per person-years. Inflammatory diseases, predominantly of the lungs and bowel, occurred in 36.7% of the patients. The median age at HCT was 9.1 years (range 1.5-23.6 years). Most HCTs (90%) were performed after using reduced intensity/toxicity conditioning. HCT sources were HLA-matched (40%) and -mismatched (10%) related donors or HLA-matched (36.7%) and -mismatched (13.3%) unrelated donors. CGD-related infections after HCT decreased significantly to 0.06 per person-years (P = .038). The frequency of inflammatory bowel disease and the use of steroids also decreased. The cumulative incidence of graft failure and second HCT was 17.9%. The 2-year overall and event-free survival were 92.3% and 82.1%, respectively, while at 5 years they were 85.7% and 77.0%, respectively. In the surviving patients evaluated, ≥95% donor myeloid chimerism at 1 and 2 years after HCT was 93.8% and 87.5%, respectively.
Conclusions: Patients with p47phox CGD suffer from a significant disease burden that can be effectively alleviated by HCT. Similar to other forms of CGD, HCT should be considered for patients with p47phox CGD.
(Copyright © 2024 American Academy of Allergy, Asthma & Immunology. All rights reserved.)
Databáze: MEDLINE
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