TY - JOUR
T1 - Enzyme replacement therapy for infantile hypophosphatasia attempted by intravenous infusions of alkaline phosphatase-rich Paget plasma
T2 - Results in three additional patients
AU - Whyte, Michael P.
AU - McAlister, William H.
AU - Patton, Lance S.
AU - Magill, H. Lynn
AU - Fallon, Michael D.
AU - Lorentz, William B.
AU - Herrod, Henry G.
N1 - Funding Information:
From the Departments of Medicine and Pediatrics, and Pediatric Radiology, Washington University School of Medicine; the Departments of Pediatrics and Radiology, University of Tennessee Center for the Health Sciences; the Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine; and the Department of Pediatrics, Bowman Gray School of Medicine of Wake Forest University. Supported in part by Grant RR-OOO36f rom the General Clinical Research Center Branch, Division of Research Facilities and Resources, National Institutes of Health; and by a Grant-in-Aid from the Shriners Hospitals for Crippled Children. Presented in part at the Eighth International Conference on Calcium Regulating Hormones, Kobe, Japan, October 16 to 24, 1983. Submitted for publication April 5, 1984; accepted May 18, 1984. Reprint requests: Michael P. Whyte, M.D., Division of Bone and Mineral Metabolism, The Jewish Hospital of St. Louis, 216 S. Kingshighway, St. Louis, MO 63110.
PY - 1984/12
Y1 - 1984/12
N2 - After biochemical and radiographic studies, enzyme replacement therapy in three patients with the infantile form of hypophosphatasia was attempted by weekly intravenous infusions of bone alkaline phosphatase-rich (BAP) plasma from patients with Paget bone disease. Subsequently, circulating BAP activity was substantially increased in each patient, and in one was maintained in the normal range for nearly 2 months. Despite partial or complete correction of the deficiency of circulating BAP activity, we observed no radiographic evidence for arrest of progressive osteopenia or improvement in rachitic defects in any of the patients. Failure of infants with hypophosphatasia to show significant healing of rickets on correction of circulating BAP activity supports the hypothesis that this isoenzyme functions in situ during normal skeletal mineralization.
AB - After biochemical and radiographic studies, enzyme replacement therapy in three patients with the infantile form of hypophosphatasia was attempted by weekly intravenous infusions of bone alkaline phosphatase-rich (BAP) plasma from patients with Paget bone disease. Subsequently, circulating BAP activity was substantially increased in each patient, and in one was maintained in the normal range for nearly 2 months. Despite partial or complete correction of the deficiency of circulating BAP activity, we observed no radiographic evidence for arrest of progressive osteopenia or improvement in rachitic defects in any of the patients. Failure of infants with hypophosphatasia to show significant healing of rickets on correction of circulating BAP activity supports the hypothesis that this isoenzyme functions in situ during normal skeletal mineralization.
UR - http://www.scopus.com/inward/record.url?scp=0021719343&partnerID=8YFLogxK
U2 - 10.1016/S0022-3476(84)80079-7
DO - 10.1016/S0022-3476(84)80079-7
M3 - Article
C2 - 6502342
AN - SCOPUS:0021719343
SN - 0022-3476
VL - 105
SP - 926
EP - 933
JO - The Journal of Pediatrics
JF - The Journal of Pediatrics
IS - 6
ER -