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The impact of testosterone therapy on quality of life in adolescents with Duchenne muscular dystrophy
Neuromuscular Disorders ( IF 2.8 ) Pub Date : 2021-09-25 , DOI: 10.1016/j.nmd.2021.09.007
C L Wood 1 , J Page 2 , J Foggin 3 , M Guglieri 4 , V Straub 4 , T D Cheetham 1
Affiliation  

Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy in childhood. It is associated with progressive muscle function decline and premature death. Long-term oral glucocorticoid use slows muscle weakness but is associated with several side effects including delayed puberty. This study assessed the impact of a 2-year incremental intramuscular testosterone regimen on quality of life (QoL) in a cohort of 15 adolescents with DMD. The Pediatric Quality of Life Inventory (PedsQL) Neuromuscular module was used to assess QoL and was completed by parent-child dyads. Semi-structured interviews were carried out to understand patient views on testosterone therapy. QoL scores increased in 10 of the 15 participants during treatment, with a mean total PedsQL score of 74.6 pre-treatment v 80.2 post treatment (p = 0.04). This was supported by comments in the semi-structured interviews. Parent-reported PedsQL scores were lower than their child's post treatment (p = 0.007). Testosterone therapy for pubertal induction was associated with an improvement in QoL and the observed physical changes during puberty played an important role. Low self-esteem was also a prevailing theme. This data supports the inclusion of testosterone therapy for pubertal induction as a Standard of Care.



中文翻译:

睾酮治疗对杜氏肌营养不良症青少年生活质量的影响

杜氏肌营养不良症 (DMD) 是儿童时期最常见的肌营养不良症。它与进行性肌肉功能下降和过早死亡有关。长期口服糖皮质激素可减缓肌肉无力,但会产生一些副作用,包括青春期延迟。本研究评估了 2 年增量肌肉注射睾酮方案对 15 名 DMD 青少年队列的生活质量 (QoL) 的影响。儿科生活质量量表 (PedsQL) 神经肌肉模块用于评估生活质量,并由亲子二人组完成。进行半结构化访谈以了解患者对睾酮治疗的看法。治疗期间 15 名参与者中有 10 人的 QoL 评分增加,平均总 PedsQL 评分为治疗前 74.6 分与治疗后 80.2 分(p = 0.04)。这得到了半结构化访谈中的评论的支持。家长报告的 PedsQL 分数低于他们孩子的治疗后 ( p  = 0.007)。用于青春期诱导的睾酮治疗与生活质量的改善有关,并且观察到的青春期身体变化发挥了重要作用。低自尊也是一个普遍的主题。该数据支持将用于青春期诱导的睾酮治疗纳入护理标准。

更新日期:2021-09-25
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