Stabilization of juvenile metachromatic leukodystrophy after bone marrow transplantation: a 13-year follow-up.

TY - JOUR

T1 - Stabilization of juvenile metachromatic leukodystrophy after bone marrow transplantation: a 13-year follow-up.

AU - Görg, Maria

AU - Wilck, Wanja

AU - Granitzny, Barbara

AU - Suerken, Anne

AU - Lukacs, Zoltan

AU - Ding, Xiaoqi

AU - Schulte-Markwort, Michael

AU - Kohlschütter, Alfried

PY - 2007

Y1 - 2007

N2 - A 29-year-old female patient with juvenile metachromatic leukodystrophy diagnosed at age 14 years received a bone marrow transplant at age 16 years. A report was published 6 years after bone marrow transplantation concluding that the disease had slowly progressed in the 2 years following bone marrow transplantation. We now report on a further 7-year follow-up, typified by a steady state of spastic paraplegia and mild dementia. Neurophysiological, neuroradiological, and psychological status also remained stable. In the patient's leukocytes, the activity of arylsulfatase A, the enzyme deficient in untreated metachromatic leukodystrophy, was within the normal range whereas urinary sulfatides remained elevated. Data on the natural course of juvenile metachromatic leukodystrophy are rare, so in the present case it is difficult to establish whether the rather favorable course can be attributed with certainty to bone marrow transplantation. The long-term stabilization in this patient, however, suggested that bone marrow transplantation may halt the progression of juvenile metachromatic leukodystrophy.

AB - A 29-year-old female patient with juvenile metachromatic leukodystrophy diagnosed at age 14 years received a bone marrow transplant at age 16 years. A report was published 6 years after bone marrow transplantation concluding that the disease had slowly progressed in the 2 years following bone marrow transplantation. We now report on a further 7-year follow-up, typified by a steady state of spastic paraplegia and mild dementia. Neurophysiological, neuroradiological, and psychological status also remained stable. In the patient's leukocytes, the activity of arylsulfatase A, the enzyme deficient in untreated metachromatic leukodystrophy, was within the normal range whereas urinary sulfatides remained elevated. Data on the natural course of juvenile metachromatic leukodystrophy are rare, so in the present case it is difficult to establish whether the rather favorable course can be attributed with certainty to bone marrow transplantation. The long-term stabilization in this patient, however, suggested that bone marrow transplantation may halt the progression of juvenile metachromatic leukodystrophy.

M3 - SCORING: Zeitschriftenaufsatz

VL - 22

SP - 1139

EP - 1142

JO - J CHILD NEUROL

JF - J CHILD NEUROL

SN - 0883-0738

IS - 9

M1 - 9

ER -