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Objective <p>This exploratory survey study aimed to evaluate the epidemiology, treatment patterns (including pharmacotherapy, rehabilitation, and supportive care), economic burden, drug preferences, and unmet needs of Chinese SMA patients.</p> Methods <p>We conducted an anonymous online survey using a self-developed questionnaire from March 4 to March 31, 2024. The questionnaire collected comprehensive data on epidemiology, treatment patterns, rehabilitative interventions, nursing management, economic burden, and preferences. Data analysis was performed using descriptive statistics and chi-square tests.</p> Results <p>Analysis of 82 valid questionnaires revealed that 91.5% of SMA patients had type I or II disease, with 69.5% diagnosed by genetic testing within 12 months of birth. Awareness of prenatal screening was low. Nusinersen and Risdiplam showed comparable utilization rates and efficacy, though Risdiplam was associated with significantly higher rates of adverse events. Over 75% of caregivers reported financial constraints, with more than 42% expressing long-term affordability concerns. Rehabilitation participation reached 86.6%, predominantly involving motor function training, stretching therapy, and the use of assistive devices. Critical gaps included the finding that 89.0% of caregivers lacked formal training, 40.8% of patients lacking respiratory interventions, and a high demand for emergency skills training (61.0%). Treatment preferences prioritized oral administration, liquid formulations, and fruit flavors, with efficacy, affordability, and ease of use (80.5%) being the primary decision-driving factors.</p> Conclusion <p>The management of SMA in China faces three primary challenges: inadequate prevention awareness, critical caregiving deficiencies, and prohibitively high treatment costs. Urgent actions include scaling up prenatal/newborn screening, establishing multidisciplinary care models to address respiratory/nutritional gaps, and implementing policy reforms to improve affordable drug access.</p>

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Real-world treatment patterns and unmet needs in spinal muscular atrophy: a caregiver-centric survey study from China

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年2月27日

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  • Wenxiang Fan,
  • Wan-Er Zheng,
  • Ji Li,
  • Rui Wang,
  • Shaoqing Ni,
  • Pingping Xue,
  • Chi Xu

摘要

Objective

This exploratory survey study aimed to evaluate the epidemiology, treatment patterns (including pharmacotherapy, rehabilitation, and supportive care), economic burden, drug preferences, and unmet needs of Chinese SMA patients.

Methods

We conducted an anonymous online survey using a self-developed questionnaire from March 4 to March 31, 2024. The questionnaire collected comprehensive data on epidemiology, treatment patterns, rehabilitative interventions, nursing management, economic burden, and preferences. Data analysis was performed using descriptive statistics and chi-square tests.

Results

Analysis of 82 valid questionnaires revealed that 91.5% of SMA patients had type I or II disease, with 69.5% diagnosed by genetic testing within 12 months of birth. Awareness of prenatal screening was low. Nusinersen and Risdiplam showed comparable utilization rates and efficacy, though Risdiplam was associated with significantly higher rates of adverse events. Over 75% of caregivers reported financial constraints, with more than 42% expressing long-term affordability concerns. Rehabilitation participation reached 86.6%, predominantly involving motor function training, stretching therapy, and the use of assistive devices. Critical gaps included the finding that 89.0% of caregivers lacked formal training, 40.8% of patients lacking respiratory interventions, and a high demand for emergency skills training (61.0%). Treatment preferences prioritized oral administration, liquid formulations, and fruit flavors, with efficacy, affordability, and ease of use (80.5%) being the primary decision-driving factors.

Conclusion

The management of SMA in China faces three primary challenges: inadequate prevention awareness, critical caregiving deficiencies, and prohibitively high treatment costs. Urgent actions include scaling up prenatal/newborn screening, establishing multidisciplinary care models to address respiratory/nutritional gaps, and implementing policy reforms to improve affordable drug access.