Receptor-mediated uptake of acid alpha-glucosidase corrects lysosomal glycogen storage in cultured skeletal muscle.

Attempts at treatment of glycogenosis type II and other lysosomal storage disorders by enzyme replacement have been reported. Parenteral enzyme administration has been ineffectual. Treatment by bone marrow transplantation is currently under investigation. We have used cultured skeletal muscle cells from a patient with infantile glycogenosis type II to study fundamental aspects of enzyme replacement therapy. Efficient uptake of acid alpha-glucosidase was achieved by using the mannose-6-phosphate receptor on the cell surface as a target for an enzyme precursor with phosphorylated high-mannose types carbohydrate chains purified from human urine. We found that the enzyme was channeled to the lysosomes and converted to mature acid alpha-glucosidase. Glycogen storage was reversed. The results are discussed in relation to treatment of glycogenosis type II.

Blunt bovine and equine trauma.

During the past 6 years 134 patients were admitted as the result of bovine (cow) and equine (horse) trauma. The mechanism of injury was fall from horse in 45 patients, animal assault in 42, animal kick in 39, and animal-drawn vehicle accident in eight. Injury Severity Score (ISS) ranged from 1 to 41 and was greater than or equal to 25 in 11 patients. One hundred seventeen operative procedures were performed by ten groups of surgical subspecialists. Mortality was nil. Ideal management of these injuries includes treatment in a regional trauma center and an educational program of preventive measures.

Farm accidents in children.

During a 6 1/2 year period, 105 children were admitted to the hospital as the result of trauma that occurred on farms. The mechanism of injury was animal related in 42 (40%), tractor or wagon accident in 28 (26%), farm machinery in 21 (20%), fall from farm building in six (6%), and miscellaneous in eight (8%). Injury Severity Score was calculated for each patient. An Injury Severity Score of greater than or equal to 25 was determined for 11 children (11%). Life-threatening injuries, therefore, are frequently the result of childhood activities that take place in agricultural environments. The most common injuries were orthopedic, neurologic, thoracoabdominal, and maxillofacial. There was one death in the series, and only one survivor sustained major long-term disability. Such injuries are managed with optimal outcome in a regional trauma center. Educational programs with an emphasis on prevention and safety measures may reduce the incidence of farm accidents.

The spectrum of agricultural trauma.

During the past 6 years, 375 patients were hospitalized with injuries resulting from farm accidents. The mechanism of injury was farm animal in 135 patients (36%), tractor in 89 (24%), corn picker or auger in 57 (15%), power take-off in 29 (8%), other farm machinery in 50 (13%), and miscellaneous in 15 (4%). Injury severity score (ISS) of 25 or greater was calculated for 29 individuals (8%). Eleven groups of surgical subspecialists performed 539 procedures. Eight patients (2.1%) died as a result of their injuries. All eight deaths occurred after tractor accidents secondary to pelvic fractures, head and spinal cord injury, or blunt chest trauma. Thirty-nine patients (10%) were left with serious permanent disability. Unnecessary morbidity and mortality in many cases were attributed to excessive prehospital care times within a largely rural area. Better prevention by farmer education and the initiation of mandatory safety devices on agricultural equipment may lower the incidence of farm accidents. Major agricultural trauma is frequent and diverse and is optimally managed in a regional trauma center.