Haploidentical bone marrow transplantation for osteopetrosis.
HLA-matched bone marrow transplantation is an effective form of treatment for some patients with malignant osteopetrosis, a defect of osteoclast function. Following transplant, normal osteoclasts differentiate from donor-derived marrow stem cells and can function normally in some of these patients. For patients without an HLA-matched marrow donor, pharmacologic treatments have not yet proved effective. This article demonstrates that normal osteoclast function can be obtained following the transplantation of HLA-nonidentical marrow that has been purged of T lymphocytes in vitro.
Orchard
P J
PJ
Department of Pediatrics, University of Wisconsin, Madison 53792.
Dickerman
J D
JD
Mathews
C H
CH
Frierdich
S
S
Hong
R
R
Trigg
M E
ME
Shahidi
N T
NT
Finlay
J L
JL
Sondel
P M
PM
D000961:Antilymphocyte Serum / Q000627:therapeutic use; D001853:Bone Marrow / Q000473:pathology; D016026:Bone Marrow Transplantation; D005260:Female; D006680:HLA Antigens; D006239:Haplotypes; D006801:Humans; D007223:Infant; D008212:Lymphocyte Depletion; D010010:Osteoclasts / Q000473:pathology; D010022:Osteopetrosis / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D013601:T-Lymphocytes / Q000276:immunology
D000961:Antilymphocyte Serum; D006680:HLA Antigens