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Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000

Seger, Reinhard A
Gungor, Tayfun
Belohradsky, Bernd H
Blanche, Stephane
Bordigoni, Pierre
Di Bartolomeo, Paolo
Flood, Terence
Landais, Paul
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Published in Blood. 2002, vol. 100, no. 13, p. 4344-50
Abstract Treatment of chronic granulomatous disease (CGD) with myeloablative bone marrow transplantation is considered risky. This study investigated complications and survival according to different risk factors present at transplantation. The outcomes of 27 transplantations for CGD, from 1985 to 2000, reported to the European Bone Marrow Transplant Registry for primary immunodeficiencies were assessed. Most transplant recipients were children (n = 25), received a myeloablative busulphan-based regimen (n = 23), and had unmodified marrow allografts (n = 23) from human leukocyte antigen (HLA)-identical sibling donors (n = 25). After myeloablative conditioning, all patients fully engrafted with donor cells; after myelosuppressive regimens, 2 of 4 patients fully engrafted. Severe (grade 3 or 4) graft-versus-host disease (GVHD) disease developed in 4 patients: 3 of 9 with pre-existing overt infection, 1 of 2 with acute inflammatory disease. Exacerbation of infection during aplasia was observed in 3 patients; inflammatory flare at the infection site during neutrophil engraftment in 2: all 5 patients belonged to the subgroup of 9 with pre-existing infection. Overall survival was 23 of 27, with 22 of 23 cured of CGD (median follow-up, 2 years). Survival was especially good in patients without infection at the moment of transplantation (18 of 18). Pre-existing infections and inflammatory lesions have cleared in all survivors (except in one with autologous reconstitution). Myeloablative conditioning followed by transplantation of unmodified hemopoietic stem cells, if performed at the first signs of a severe course of the disease, is a valid therapeutic option for children with CGD having an HLA-identical donor.
Keywords AdolescentAdultAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedAntibodies, NeoplasmAntilymphocyte SerumBone Marrow/drug effects/radiation effectsBusulfanCause of DeathChildChild, PreschoolCyclophosphamideEuropeFemaleGenetic HeterogeneityGraft vs Host Disease/epidemiology/etiologyGranulomatous Disease, Chronic/genetics/therapyHematopoietic Stem Cell Transplantation/adverse effects/statistics & numerical dataHumansInfection/complicationsInfection ControlInflammationMaleMelphalanNeutrophils/physiologySurvival AnalysisTransplantation Conditioning/adverse effects/statistics & numerical dataTransplantation, Homologous/adverse effectsTreatment OutcomeVidarabine/analogs & derivatives
PMID: 12393596
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SEGER, Reinhard A et al. Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000. In: Blood, 2002, vol. 100, n° 13, p. 4344-50. doi: 10.1182/blood-2002-02-0583 https://archive-ouverte.unige.ch/unige:55904

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Deposited on : 2015-04-29

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