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Objective <p>Duchenne muscular dystrophy (DMD) is a rare, progressive, severely disabling and ultimately fatal genetic neuromuscular disease requiring lifelong multidisciplinary clinical care and support. The objective of this study was to conduct a systematic review and synthesis of estimates of costs of illness of DMD.</p> Methods <p>In this systematic review (International Prospective Register of Systematic Reviews [PROSPERO] identifier: CRD420251153340), we searched PubMed, MEDLINE, Embase, the Health Technology Assessment Database, and the National Health Service Economic Evaluation Database for studies reporting costs of DMD. Risk of bias was assessed using the Newcastle–Ottawa scale.</p> Results <p>We identified 19 publications involving 6993 children and adults with DMD from 14 countries (Australia, Brazil, Bulgaria, Denmark, Egypt, France, Germany, Hungary, Italy, Portugal, Spain, Sweden, the UK, and the USA). Across studies and strata, the mean per-patient annual direct medical cost of illness (in 2025 international US dollars) was estimated at between $2620 and $209,980, the mean per-patient annual direct nonmedical cost between $5670 and $103,800, and the mean per-patient annual indirect (productivity) cost between $400 and $48,390. Most studies exhibited some risk of bias.</p> Conclusions <p>DMD is associated with a substantial economic burden related to the formal disease management, as well as extensive informal care and indirect (productivity) costs shouldered by parents and other nonprofessional caregivers. Although relatively well-captured for a few countries, comprehensive cost data are lacking for many geographical settings. Our portfolio of costs of DMD will help inform assessments of burden of illness, health technology evaluations of new therapies, and data gap analyses for future research.</p>

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The Economic Burden of Duchenne Muscular Dystrophy: A Systematic Review

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月28日

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  • Erik Landfeldt,
  • Maria Åberg,
  • Luca Bello,
  • Valérie Deroo,
  • Alfred Peter Born,
  • Silene Giusti,
  • Anne-Berit Ekström

摘要

Objective

Duchenne muscular dystrophy (DMD) is a rare, progressive, severely disabling and ultimately fatal genetic neuromuscular disease requiring lifelong multidisciplinary clinical care and support. The objective of this study was to conduct a systematic review and synthesis of estimates of costs of illness of DMD.

Methods

In this systematic review (International Prospective Register of Systematic Reviews [PROSPERO] identifier: CRD420251153340), we searched PubMed, MEDLINE, Embase, the Health Technology Assessment Database, and the National Health Service Economic Evaluation Database for studies reporting costs of DMD. Risk of bias was assessed using the Newcastle–Ottawa scale.

Results

We identified 19 publications involving 6993 children and adults with DMD from 14 countries (Australia, Brazil, Bulgaria, Denmark, Egypt, France, Germany, Hungary, Italy, Portugal, Spain, Sweden, the UK, and the USA). Across studies and strata, the mean per-patient annual direct medical cost of illness (in 2025 international US dollars) was estimated at between $2620 and $209,980, the mean per-patient annual direct nonmedical cost between $5670 and $103,800, and the mean per-patient annual indirect (productivity) cost between $400 and $48,390. Most studies exhibited some risk of bias.

Conclusions

DMD is associated with a substantial economic burden related to the formal disease management, as well as extensive informal care and indirect (productivity) costs shouldered by parents and other nonprofessional caregivers. Although relatively well-captured for a few countries, comprehensive cost data are lacking for many geographical settings. Our portfolio of costs of DMD will help inform assessments of burden of illness, health technology evaluations of new therapies, and data gap analyses for future research.