Survival analysis of spinal muscular atrophy type I
The life expectancy of patients with spinal muscular atrophy (SMA) type I is generally considered to be less than 2 years. Recently, with the introduction of proactive treatments, a longer survival and an improved survival rate have been reported. In this study, we analyzed the natural courses and s...
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Published in | Clinical and experimental pediatrics Vol. 53; no. 11; pp. 965 - 970 |
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Main Authors | , , , , , , |
Format | Journal Article |
Language | English |
Published |
Korea (South)
Clinical and Experimental Pediatics / Korean Pediatric Society
01.11.2010
The Korean Pediatric Society Korean Pediatric Society 대한소아청소년과학회 |
Subjects | |
Online Access | Get full text |
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Summary: | The life expectancy of patients with spinal muscular atrophy (SMA) type I is generally considered to be less than 2 years. Recently, with the introduction of proactive treatments, a longer survival and an improved survival rate have been reported. In this study, we analyzed the natural courses and survival statistics of SMA type I patients and compared the clinical characteristics of the patients based on their survival periods.
We reviewed the medical records of 14 pediatric patients diagnosed with SMA type I during a 9-year period. We examined the demographic and clinical characteristics of these patients, calculated their survival probabilities, and plotted survival curves as on the censoring date, January 1, 2010. We also compared the characteristics of the patients who died before the age of 24 months (early-death, ED group) and those who survived for 24 months or longer (long-survival, LS group).
The mean survival time was 22.8±2.0 months. The survival probabilities at 6 months, 12 months, 18 months, 24 months, and 30 months were 92.9%, 92.9%, 76.0%, 76.0%, and 65.1%, respectively. Birth weight was the only factor that showed a statistically significant difference between the ED and LS groups (P=0.048).
In this study, the survival probabilities at 2 years were far greater than expected. Because of the limited number of patients and information in this study, the contribution of improved supportive care on longer survival could not be clarified; this may be elucidated in larger cohort studies. |
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Bibliography: | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 G704-000560.2010.53.11.006 |
ISSN: | 1738-1061 2092-7258 2713-4148 |
DOI: | 10.3345/kjp.2010.53.11.965 |