Boys with Duchenne muscular dystrophy (DMD) and their parents appear to be ambivalent toward the use of bisphosphonates to prevent osteoporosis in DMD, according to a recently published study in PLOS One.
Corticosteroids represent the current standard of treatment for patients with DMD. Although the clinical benefits associated with corticosteroid therapy in DMD are evident, long-term corticosteroid use can result in osteoporosis.
Osteoporosis can lead to long bone and vertebral fractures after minimal trauma, and there is a need for therapeutic alternatives to help prevent this complication.
Bisphosphonates are a type of osteoporosis medication widely used to treat postmenopausal women, but they are also used in DMD: the International Care Considerations for DMD (2018) recommend the initiation of intravenous (IV) bisphosphonate treatment after vertebral fractures or low trauma long bone fractures have occurred. However, the use of bisphosphonates as a preventive therapy before the onset of pathological fractures is controversial. The treatment requires visiting a hospital once every four months for an IV infusion, and is associated with feeling unwell on the day of administration.
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The study’s authors aimed to determine the perspectives of patients with DMD, their parents and health professionals on the preventive use of bisphosphonates.
The study included semi-structured interviews and focus groups involving four patients with DMD, 20 parents and 27 health professionals involved in the management of patients with DMD.
The authors identified a range of opinions, from those who supported its use as it “bought them time,” to those who were open to the idea but required more supporting evidence, and those who were against it and believed that bone health should involve more than bisphosphonate use.
Most patients weighed the negatives and positives of the treatment and believed that fracture prevention was worth the adverse effects associated with bisphosphonate administration.
“It’d be worth it in the long run [having preventative IV bisphosphonate treatment] ‘cause then if you think about it, just a day every four months [for treatment] instead of it being four weeks out of everything and not being involved in school and stuff [for fractures]… I’d say it would be an improvement to that,” said a 16-year-old patient quoted in the study.
For many parents, the decision would depend on the recommendation from health professionals or other families of children with DMD. Many remarked that they would not proceed without solid evidence supporting the treatment.
Other groups of parents and health professionals expressed concern that children with DMD already endured enough and were hesitant to introduce new drugs with potential side effects.
“Future research is warranted to investigate the risks, benefits, and effectiveness of using preventative bisphosphonate therapy in this population to inform clinical practice, as recommended by health professionals,” the study’s authors concluded.
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