Developmental Endocrinology Research Group, Royal Hospital for Children, University of Glasgow, Glasgow, UK.
Paediatric Neurosciences Research Group, Royal Hospital for Children, Glasgow, UK.
Clin Trials. 2021 Feb;18(1):39-50. doi: 10.1177/1740774520958395. Epub 2020 Oct 4.
BACKGROUND/AIMS: Given the extent of osteoporosis in people with Duchenne muscular dystrophy treated with glucocorticoids and the limited evidence of bone-protective therapies, clinical trials are needed. We conducted surveys to obtain the opinion of young people with Duchenne muscular dystrophy, parents/guardians and neuromuscular clinicians on the feasibility of osteoporosis clinical trials in this population.
Online surveys were sent to three groups: (a) people with a confirmed diagnosis of Duchenne muscular dystrophy (≥14 years), (b) parents and guardians and (c) neuromuscular clinicians in the UK NorthStar Clinical Network. Surveys (a) and (b) were distributed via the UK Duchenne muscular dystrophy Registry.
Survey respondents included 52 people with Duchenne muscular dystrophy with a median age of 17 years (range: 14, 40) and 183 parents/guardians. Fourteen out of 23 (61%) NorthStar centres responded. Of the 52 people with Duchenne muscular dystrophy, 13 (25%) were very concerned about their bone health and 21 (40%) were slightly concerned. Of the 183 parents/guardians, 75 (41%) were very concerned about their son's bone health and 90 (49%) were slightly concerned. Fractures and quality of life were the top two main outcome measures identified by people with Duchenne muscular dystrophy. Fractures and bone density were the top two main outcome measures identified by parents/guardians and neuromuscular clinicians. Thirty percent of people with Duchenne muscular dystrophy and 40% of parents/guardians would not take part if an osteoporosis trial involved a placebo that was administered parenterally. Only 2 of the 14 NorthStar centres (14%) would enrol people with Duchenne muscular dystrophy if a parenteral placebo was used in an osteoporosis trial in Duchenne muscular dystrophy.
There is great awareness of bone health and the need for bone-protective trials among people with Duchenne muscular dystrophy and their carers. However, a proportion of people with Duchenne muscular dystrophy and parents are reluctant to participate in a placebo-controlled osteoporosis trial that included a parenteral therapy. A larger proportion of health care experts are unwilling to enrol their patients in such a trial. Our finding is relevant for the design of bone-protective studies in Duchenne muscular dystrophy.
背景/目的：鉴于接受糖皮质激素治疗的杜氏肌营养不良症患者骨质疏松症的严重程度，以及骨保护疗法的有限证据，需要进行临床试验。我们进行了调查，以了解患有杜氏肌营养不良症的年轻人、父母/监护人以及神经肌肉临床医生对该人群中进行骨质疏松症临床试验的可行性的看法。
在线调查分发给三组人员：（a）确诊患有杜氏肌营养不良症（≥ 14 岁）的人；（b）父母/监护人；（c）英国 NorthStar 临床网络中的神经肌肉临床医生。（a）和（b）调查通过英国杜氏肌营养不良症登记处进行分发。
调查回复者包括 52 名患有杜氏肌营养不良症的患者，中位年龄为 17 岁（范围：14-40 岁），以及 183 名父母/监护人。23 个 NorthStar 中心中的 14 个（61%）做出了回复。在 52 名患有杜氏肌营养不良症的患者中，有 13 名（25%）非常关注他们的骨骼健康，有 21 名（40%）略有担忧。在 183 名父母/监护人中，有 75 名（41%）非常担心他们儿子的骨骼健康，有 90 名（49%）略有担忧。骨折和生活质量是患有杜氏肌营养不良症的患者确定的前两个主要结局指标。骨折和骨密度是父母/监护人以及神经肌肉临床医生确定的前两个主要结局指标。30%的患有杜氏肌营养不良症的患者和 40%的父母/监护人表示，如果骨质疏松症试验涉及到皮下给予安慰剂，则不会参与。如果在杜氏肌营养不良症的骨质疏松症试验中使用皮下安慰剂，只有 14 个 NorthStar 中心中的 2 个（14%）会招募患有杜氏肌营养不良症的患者。
患有杜氏肌营养不良症的患者及其护理人员对骨骼健康和骨保护试验有很高的认识。然而，一部分患有杜氏肌营养不良症的患者和父母不愿意参加包括皮下治疗的安慰剂对照骨质疏松症试验。更大比例的医疗保健专家不愿意在这样的试验中招募他们的患者。我们的发现与杜氏肌营养不良症中骨保护研究的设计有关。
