TY - JOUR
T1 - Hurler syndrome with severe complication in post-bone marrow transplantation course
T2 - Life threatening interstitial pneumonitis and hypertension
AU - Ohashi, T.
AU - Tahara, T.
AU - Fujisawa, K.
AU - Hoshi, Y.
AU - Ito, F.
AU - Suzuki, N.
AU - Eto, Y.
AU - Maekawa, K.
PY - 1995
Y1 - 1995
N2 - Bone marrow transplantation (BMT) was performed in a 3 year old patient with Hurler syndrome. The post-BMT course was complicated by interstitial pneumonitis and severe hypertension, which were life threatening. The patient responded well to therapy and recovered. BMT in this patient resulted in significant clinical improvement in the signs and symptoms of Hurler syndrome. Biochemical improvement, including elevated α-L-iduronidase activity in white blood cells and decreased urinary glycosaminoglycan excretion was significant. However, skeletal and neurological impairment were not improved. We conclude that BMT for Hurler syndrome should be performed at an earlier stage, before severe neuronal damage has occurred. Moreover, BMT is a high risk procedure and there will always be a possibility that life threatening complications will occur, as in our case.
AB - Bone marrow transplantation (BMT) was performed in a 3 year old patient with Hurler syndrome. The post-BMT course was complicated by interstitial pneumonitis and severe hypertension, which were life threatening. The patient responded well to therapy and recovered. BMT in this patient resulted in significant clinical improvement in the signs and symptoms of Hurler syndrome. Biochemical improvement, including elevated α-L-iduronidase activity in white blood cells and decreased urinary glycosaminoglycan excretion was significant. However, skeletal and neurological impairment were not improved. We conclude that BMT for Hurler syndrome should be performed at an earlier stage, before severe neuronal damage has occurred. Moreover, BMT is a high risk procedure and there will always be a possibility that life threatening complications will occur, as in our case.
KW - bone marrow transplantation
KW - high blood pressure
KW - Hurler syndrome
KW - interstitial pneumonitis
KW - mucopolysaccharidosis
KW - transplantation
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M3 - Article
C2 - 8775554
AN - SCOPUS:0029592217
VL - 37
SP - 697
EP - 700
JO - Pediatrics International
JF - Pediatrics International
SN - 1328-8067
IS - 6
ER -