Bone marrow transplantation for aspartylglucosaminuria: follow-up study of transplanted and non-transplanted patients.
We describe the state of health, intellectual skills, and dysmorphic features of 19 young patients with aspartylglucosaminuria. Of them, 5 had undergone a successful bone marrow transplantation between 1991 and 1997. The first 2 patients who received transplants were, after 7 and 5 years' follow-up, more severely mentally retarded than the non-transplanted patients. The general health of the later patients was quite good, whereas the 5 patients who underwent bone marrow transplantation had post-transplant complications. Their dysmorphic status remained unchanged. We cannot encourage bone marrow transplantation for the treatment of patients with aspartylglucosaminuria after infancy.
Arvio
M
M
Pääjärvi Communal Union, Lammi, Finland.
Sauna-Aho
O
O
Peippo
M
M
D054880:Aspartylglucosaminuria; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D016896:Treatment Outcome