Cyclic administration
of pamidronate in children with severe osteogenesis
imperfecta
Montreal, Canada — A study conducted at the
Montreal Shriners for Hospital for Children promises new
hope for children born with a serious, sometimes fatal,
bone disease. The results of the study, headed up by
Francis H. Glorieux, M.D., Ph.D., were published in this
week's issue of the New England Journal of Medicine.
Osteogenesis Imperfecta, or brittle bone disease,
affects about one in nearly 20,000 children born each
year. This crippling disorder, similar to osteoporosis,
causes frequent bone fractures, progressive deformities
of the arms and legs, and chronic bone pain. Children
born with OI are often confined to wheelchairs and
sometimes don't survive childhood. Up until now, various
treatments aimed at increasing bone mass and reducing
fractures had been tried in children suffering from the
disease but without significant success.
The study involved the periodic administration of a
drug called pamidronate intravenously to 30 children with
severe osteogenesis imperfecta for a period of one to
four years. It was was initially targeted at reducing
bone destruction. Results showed that after the initial
treatment cycle, these children experienced not only a
reduction of bone loss caused by the disease, but also
significant increases in bone mineral content.
Furthermore, the number of fractures fell dramatically,
indicating that the increase in bone mass was
substantial. X-rays suggested new bone formation in 25 of
the children.
The chronic pain experienced by these children was
also reduced, and most patients also became more mobile.
Some children progressed from being wheelchair-bound to
walking independently, and others who previously used
crutches, walkers or canes were able to walk
independently.
Osteogenesis imperfecta is often misdiagnosed, and
there could be as many as 20,000 children currently
suffering from OI in North America alone.
The results of this research program, headed by Dr
Francis H. Glorieux, M.D., Ph.D., and funded by Shriners
Hospitals for Children, offer tremendous promise for
children with this disease. It also provides new
perspectives for treating other forms of bone disease in
children.
The study at the Montreal Shriners Hospital
involved the administration of pamidronate intravenously
to 30 children with severe osteogenesis imperfecta for a
period of one to four years. It was initially targeted at
reducing bone destruction by the disease and increasing
bone mass.
Results showed that after the initial treatment cycle,
bone resorption fell sharply with annual increases in
bone mineral content and density of 81 percent and 66
percent respectively. Furthermore, the number of
fractures fell dramatically, indicating that the increase
in bone mass was substantial. Lateral spine X-rays
suggested new bone formation in 25 of the children.
The relief of chronic pain was an unexpected but
welcome side effect of the treatment. An improvement in
mobility in most patients also accompanied the apparent
reduction of pain. This was particularly significant for
some children who progressed from being wheelchair-bound
to walking independently and others who progressed from
walking with aids such as crutches, walkers or canes to
walking independently.The study was headed by Francis H.
Glorieux, M.D., Ph.D., director of research at the
Shriners Hospital for Children in Montreal. He was
assisted by Dr. Horacio Plotkin.
