Assessment of health-related quality of life in patients with spina muscular atrophy in China

Duan, Chengaxin; Ai, Dandan; Xu, Qian; Sui, Binyan; Zhao, Kun

doi:10.5582/irdr.2022.01094

Cited by 4 publications

(1 citation statement)

References 18 publications

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“…PANDA adds to this limited body of evidence. The data are consistent with studies showing efficacy and overall safety of nusinersen in children with type I and II SMA [ 24 , 25 ] and in improving health-related quality of life [ 26 ].…”

Section: Discussionsupporting

confidence: 89%

A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results

Jiang,

Wang,

Xiong

et al. 2024

Adv Ther

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Introduction: Spinal muscular atrophy (SMA) is a rare, autosomal recessive, neuromuscular disease that leads to progressive muscular weakness and atrophy. Nusinersen, an antisense oligonucleotide, was approved for SMA in China in February 2019. We report interim results from a post-marketing surveillance phase 4 study, PANDA (NCT04419233), that collects data on the safety, efficacy, and pharmacokinetics of nusinersen in children with SMA in routine clinical practice in China. Methods: Participants enrolled in PANDA will be observed for 2 years following nusinersen treatment initiation. The primary endpoint is

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Section: Discussionsupporting

confidence: 89%

A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results

Jiang,

Wang,

Xiong

et al. 2024

Adv Ther

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Cytokines in cerebrospinal fluid as a prognostic predictor after treatment of nusinersen in SMA patients

Cheng,

Li,

Fan

et al. 2024

Clinical Neurology and Neurosurgery

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Health service utilization, economic burden and quality of life of patients with mucopolysaccharidosis in China

Kang,

Fang,

Yang

et al. 2024

Orphanet J Rare Dis

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BackgroundPatients with mucopolysaccharidosis (MPS) often face delayed diagnoses, limited treatment options and high healthcare costs, that may significantly affect patients' quality of life. The objective of this study was to understand medical service utilization related to diagnosis and treatment, economic burden during diagnosis period, and health-related quality of life among MPS patients in China.MethodsA series of patients diagnosed with MPS registered in the national patient organization were recruited for a cross-sectional survey from May to July 2019. Information were collected from patients or their parents via phone interview, including demographic data, utilization of services related to diagnosis and treatment, total cost during the period of MPS diagnosis and health-related quality of life (HRQoL). HRQoL was assessed by PedsQL 4.0 Generic Core Scale (PedsQL) and 36-item short-form health survey (SF-36) depending on the age of patients with MPS and compared with the general Chinese population.ResultsA total of 180 MPS patients (50, 67, 15, 46, 1 and 1 for type I, II, III, IV, VI and VII), with a mean age of 9.54 years and 137 (76.11%) males, were included in analysis. The mean age at first visit to a medical doctor for MPS related symptoms was 3.65 ± 2.58 years old, while only 12 patients (6.67%) were diagnosed on their first visit. The mean diagnostic delay, which is defined as the time between the first visit to a medical doctor for MPS related symptoms and the final diagnosis, was 9.42 months, with no significant difference between types. The average number of misdiagnosis was 4.56. Before the confirmed diagnosis, the patients made an average of 6.31 visits and visited 4.3 hospitals. During diagnosis period, the mean of ¥81,086.72 direct medical costs accounted for 63.75% of the total cost. Only 32.78% of the patients had ever received specific treatments. The mean scores of PedsQL and SF-36 of patients were significantly lower than the Chinese norms. Household annual income per person, specific treatment use and MPS subtype were significantly associated HRQoL of patients.ConclusionThe results highlight challenges faced by MPS patients in terms of diagnosis, access to specific treatments, economic burden and low HRQoL. There is an urgent need to improve early detection and diagnosis, create fair and consistent mechanisms to increase access to specialized treatment and reduce the economic burden of MPS patients in China.

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Assessment of health-related quality of life in patients with spina muscular atrophy in China

Cited by 4 publications

References 18 publications

A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results

A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results

Cytokines in cerebrospinal fluid as a prognostic predictor after treatment of nusinersen in SMA patients

Health service utilization, economic burden and quality of life of patients with mucopolysaccharidosis in China

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