Bone marrow transplantation in Lesch-Nyhan disease

Abstract

The patient has had no change in neurologic status or behavior. Vomiting had been a major problem prior to transplantation and was initially markedly reduced. Soon after transplantation he was able to have restraints removed to feed himself and to sleep at night, which was not possible previously. Approximately one month following transplantation, he required instrumentation for a right-sided renal stone that intermittently obstructed his ureter and led to a candidal infection. Ultimately, the calculus was removed surgically, and then the infection was successfully treated. During this period, he began vomiting again, and had to be restrained. At discharge, approximately two months after transplantation, his behavior did not appear appreciably different from that prior to transplantation.