ArticleThe Journal of allergy and clinical immunology2012
Excellent survival after sibling or unrelated donor stem cell transplantation for chronic granulomatous disease.
Article in The Journal of allergy and clinical immunology, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03055247 (A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD), which is not on this map. Cited by 30 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD
Who cites it
30 citing papers in PubMed, 72 citations in OpenAlex.
- Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.Cell reports. Medicine · 2023Trial
- Reduced-intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies.Blood · 2018Trial
- Hematopoietic stem cell transplantation following invasive mold infection in chronic granulomatous disease: Insights from a case series and literature review.Journal of human immunity · 2026Article
- Chronic Granulomatous Disease: A Multicentre Study of DHR Flow Cytometry Results and Clinical Correlations in Malaysia.The Malaysian journal of medical sciences : MJMS · 2025Article
- Allogeneic Hematopoietic cell Transplantation Using Alemtuzumab in Asian Patients with Inborn Errors of Immunity.Journal of clinical immunology · 2024Article
- Disease Presentation, Treatment Options, and Outcomes for Myeloid Immunodeficiencies.Current allergy and asthma reports · 2021Review
- Competitive sgRNA Screen Identifies p38 MAPK as a Druggable Target to Improve HSPC Engraftment.Cells · 2020Article
- Health-Related Quality of Life and Emotional Difficulties in Chronic Granulomatous Disease: Data on Adult and Pediatric Patients from Italian Network for Primary Immunodeficiency (IPINet).Journal of clinical immunology · 2020Article
- Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan.Frontiers in immunology · 2020Article
- Variable Presentation of the CYBB Mutation in One Family, Approach to Management, and a Review of the Literature.Case reports in medicine · 2020Article
- Cellular Therapies in Chronic Granulomatous Disease.Frontiers in pediatrics · 2020Review
- Geographic Variability and Pathogen-Specific Considerations in the Diagnosis and Management of Chronic Granulomatous Disease.Pediatric health, medicine and therapeutics · 2020Review
- Considerations for hematopoietic stem cell transplantation in primary immunodeficiency disorders.World journal of transplantation · 2019Review
- Clinical and genetic characteristics of Chinese pediatric patients with chronic granulomatous disease.Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology · 2019Article
- Role of Allogeneic Hematopoietic Stem Cell Transplant for Chronic Granulomatous Disease (CGD): a Report of the United States Immunodeficiency Network.Journal of clinical immunology · 2019Article
- Allogeneic Hematopoietic Cell Transplantation for Chronic Granulomatous Disease: Controversies and State of the Art.Journal of the Pediatric Infectious Diseases Society · 2018Review
- A Cohort of 169 Chronic Granulomatous Disease Patients Exposed to BCG Vaccination: a Retrospective Study from a Single Center in Shanghai, China (2004-2017).Journal of clinical immunology · 2018Article
- A Review of Chronic Granulomatous Disease.Advances in therapy · 2017Review
- Allogeneic Reduced-Intensity Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease: a Single-Center Prospective Trial.Journal of clinical immunology · 2017Article
- NADPH Oxidase Deficiency: A Multisystem Approach.Oxidative medicine and cellular longevity · 2017Review
Corrections and comments
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Authors and funding
14 authors at 5 institutions in 1 country.
Funding
Abstract
backgroundMatched related donor (MRD) hematopoietic stem cell transplantation (HSCT) is a successful treatment for chronic granulomatous disease (CGD), but the safety and efficacy of HSCT from unrelated donors is less certain.
objectiveWe evaluated the outcomes and overall survival in patients with CGD after HSCT.
methodsWe report the outcomes for 11 children undergoing HSCT from an MRD (n = 4) or an HLA-matched unrelated donor (MUD) (n = 7); 9 children were boys, and the median age was 3.8 years (range, 1-13 years). We treated both X-linked (n = 9) and autosomal recessive (n = 2) disease. Nine children had serious clinical infections before transplantation. The conditioning regimens contained busulfan, cyclophosphamide, cytarabine, or fludarabine according to the donor used. All patients received alemtuzumab (anti-CD52 antibody). Additional graft-versus-host disease (GvHD) prophylaxis included cyclosporine and methotrexate for MUD recipients and cyclosporine and prednisone for MRD recipients.
resultsNeutrophil recovery took a median of 16 days (range, 12-40 days) and 18 days (range, 13-24 days) for MRD and MUD recipients, respectively. Full donor neutrophil engraftment occurred in 9 patients, and 2 had stable mixed chimerism; all patients had sustained correction of neutrophil oxidative burst defect. Four patients had grade I skin acute GVHD responding to topical treatment. No patient had grade II to IV acute GvHD or chronic GvHD. All patients are alive between 1 and 8 years after HSCT.
conclusionFor CGD, equivalent outcomes can be obtained with MRD or MUD stem cells, and HSCT should be considered an early treatment option.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.