The natural history of infant spinal muscular atrophy in China: a study of 237 patients.

The authors retrospectively studied the natural history of 237 patients with infantile spinal muscular atrophy in China. The onset ages (mean ± SD) for types I to III were 3.1 ± 2.7, 8.7 ± 3.8, and 21.1 ± 11.7 months, respectively. The survival probabilities for type I patients at 1, 2, and 5 years were 44.9%, 38.1%, and 29.3%, respectively, and for type II patients, the probabilities were 100%, 100%, and 97%, respectively. All type III patients were alive. Type I patients with onset age after 2 months had significantly increased survival than those with onset before 2 months (P < .05). It should be noticed that survival probability at 2 years in type I patients in our study was close to that in other Asian samples of spinal muscular atrophy, but slightly better than that among whites. Patients accepted minimal proactive interventions other than antibiotics for pulmonary infection, so our study provides reliable baseline data of natural history of spinal muscular atrophy in China.