Allogeneic bone marrow transplantation for fucosidosis

Bone marrow transplantation was performed on an 8-month-old boy who was diagnosed as having fucosidosis following the diagnosis of the disease in his older brother. Although he was asymptomatic and his development was normal, abnomalities were found on an MRI scan prior to transplant. In the absence...

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Published inBone marrow transplantation (Basingstoke) Vol. 15; no. 1; pp. 153 - 158
Main Authors VELLODI, A, CRAGG, H, WINCHESTER, B, YOUNG, E, YOUNG, J, DOWNIE, C. J. C, HOARE, R. D, STOCKS, R, BANERJEE, G. K
Format Journal Article
LanguageEnglish
Published Basingstoke Nature Publishing Group 1995
Subjects
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
Fucosidosis - diagnosis
Fucosidosis - therapy
Humans
Infant
Male
Medical sciences
Transfusions. Complications. Transfusion reactions. Cell and gene therapy
Transplantation, Homologous
Human
Fucosidosis
Transfusion
Unrelated donor
Homograft
Metabolic diseases
Infant
Enzymopathy
Genetic disease
Case study
Treatment
Bone marrow
Graft
Carbohydrate
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Summary:Bone marrow transplantation was performed on an 8-month-old boy who was diagnosed as having fucosidosis following the diagnosis of the disease in his older brother. Although he was asymptomatic and his development was normal, abnomalities were found on an MRI scan prior to transplant. In the absence of a suitable related donor, an unrelated volunteer donor was used. Conditioning for the transplant consisted of busulphan and cyclophosphamide. Graft-versus-host disease prophylaxis consisted of in vitro T cell-depletion of the bone marrow and in vivo administration of cyclosporin. The post-transplant period was complicated by moderately severe graft-versus-host disease. Engraftment was documented by the presence of donor levels of alpha-fucosidase, donor blood group and tissue type (difference in the DQ antigen), and chromosomal polymorphism pattern of donor origin. Eighteen months after transplant, there is evidence of mild neurodevelopmental delay. By contrast, his elder sibling showed far greater developmental delay at the same age. The patient's MRI scan shows improvement. We believe this to be the first case of human fucosidosis treated by bone marrow transplantation.
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ISSN:0268-3369
1476-5365