Effect of comprehensive health management on the prognosis of children with type I spinal muscular atrophy

: Objective: To analyze the effect of comprehensive health management on the prognosis of children with type Ⅰ spinal muscular atrophy (SMA). Methods: Eighty patients with type Ⅰ SMA (39 males and 41 females) visited-Capital Institute of Pediatrics from January 2003 to December 2017, were enrolled in this case-control study retrospectively. They were divided into the health management group and the natural history group. Main statistical parameter, including demographic indicators, survival time, 2-year survival rate and incidence of complications were compared and analyzed. Patients were evaluated every 3-6 months. All data were processed by SPSS 19.0. Differences between the two groups were compared using rank sum test or chi square test. Survival analysis was performed by using Kaplan-Meier method, and survival difference test was performed by log-rank method. Results: Among 80 SMA patients, 14 cases (7 males and 7 females) were in the health management group and 66 cases (32 males and 34 females) in the natural history group. There was no statistically significant difference in gender ratio between the two groups (χ(2)=0.01,P=0.918) . The ages of onset and death in the two groups were 2 (0-8) and 1 (0-14) month, 11 (5-17) and 6 (1-60) months, without statistically significant difference (Z=0.91, 1.19; P=0.386, 0.116) . As of the follow-up date (June 2019) , 10 patients died and 4 survived in the health management group, while 62 (93.9%) died and 4 (6.1%) survived in the natural history group (χ(2)=6.50,P=0.011) . The median survival time in the health management group was 12 months, and the 1, 2 and 3-year survival rates were 77.9%, 54.5% and 34.1%, respectively. The median survival time of the natural history group was 6 months, and the 1, 2 and 3-year survival rates were 48.5%, 15.2% and 7.6%, respectively. For the two groups, the difference in survival rates was statistically significant (χ(2)=9.11 P=0.003). The incidence rate of pneumonia combined with respiratory failure in the health management group was lower than that in the natural history group. Conclusion: Active health management can improve the survival rate of type Ⅰ SMA patients, reduce the incidence of complications, and also improve the prognosis of patients.