Reconstitution of normal neutrophil function in chronic granulomatous disease by bone marrow transplantation

Allogeneic bone marrow transplantation was carried out on an 11-year-old boy with chronic granulomatous disease and severe chronic pulmonary insufficiency of restrictive type. After preparative regimen with busulfan (13 mg/kg) and cyclophosphamide (200 mg/kg), the patient received marrow cells from...

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Bibliographic Details
Published inBone marrow transplantation (Basingstoke) Vol. 4; no. 6; pp. 695 - 700
Main Authors DI BARTOLOMEO, P, DI GIROLAMO, G, ANGRILLI, F, SCHETTINI, F, DE MATTIA, D, MANZIONNA, M. M, DRAGANI, A, IACONE, A, TORLONTANO, G
Format Journal Article
LanguageEnglish
Published Basingstoke Nature Publishing Group 01.11.1989
Subjects
Adolescent
Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy
Biological and medical sciences
Bone Marrow Transplantation
Bone marrow, stem cells transplantation. Graft versus host reaction
Granulomatous Disease, Chronic - genetics
Granulomatous Disease, Chronic - physiopathology
Granulomatous Disease, Chronic - surgery
Humans
Lung - physiopathology
Male
Medical sciences
Neutrophils - physiology
Pedigree
Transfusions. Complications. Transfusion reactions. Cell and gene therapy
Human
Cell function
Respiratory disease
Family study
Granulomatosis
Homograft
Hemopathy
Hereditary
Recovery
Case study
Chronic
Treatment
Respiratory failure
Neutrophile
Bone marrow
Graft
Child
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Summary:Allogeneic bone marrow transplantation was carried out on an 11-year-old boy with chronic granulomatous disease and severe chronic pulmonary insufficiency of restrictive type. After preparative regimen with busulfan (13 mg/kg) and cyclophosphamide (200 mg/kg), the patient received marrow cells from his HLA-identical and MLC-nonreactive sister. Durable sustained engraftment of donor hematopoietic and lymphoid populations occurred, as documented by analysis of genetic markers and complete reversal of the neutrophil function defect. No episode of infection occurred in the post-transplant course and, currently, 40 months after transplantation the patient is in excellent health and growing normally and showing an increasing improvement of his respiratory capacity. The successful outcome in this patient demonstrates that marrow transplantation is at present the only curative approach for this congenital disorder of neutrophil function.
Bibliography:ObjectType-Case Study-2
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ISSN:0268-3369
1476-5365