The changing natural history of spinal muscular atrophy type 1.
BACKGROUND: Noninvasive ventilation has become increasingly available to spinal muscular atrophy (SMA) patients since the early 1990 s. This is expected to have improved survival for SMA type 1 patients. OBJECTIVE: To assess whether there has been a change in survival in patients with SMA type 1 bet...
| Main Authors: | , , , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2007
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| _version_ | 1797099975804452864 |
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| author | Oskoui, M Levy, G Garland, C Gray, J O'Hagen, J De Vivo, D Kaufmann, P |
| author_facet | Oskoui, M Levy, G Garland, C Gray, J O'Hagen, J De Vivo, D Kaufmann, P |
| author_sort | Oskoui, M |
| collection | OXFORD |
| description | BACKGROUND: Noninvasive ventilation has become increasingly available to spinal muscular atrophy (SMA) patients since the early 1990 s. This is expected to have improved survival for SMA type 1 patients. OBJECTIVE: To assess whether there has been a change in survival in patients with SMA type 1 between 1980 and 2006. METHODS: We used deidentified, family-reported data from participants in the International Spinal Muscular Atrophy Patient Registry and obtained additional clinical information through a mail-in questionnaire. One hundred forty-three patients with SMA type 1 were included in the analysis. Survival of patients born in 1995-2006 (n = 78) was compared with that of patients born in 1980-1994 (n = 65), using the Kaplan-Meier method and Cox proportional hazards models with age at death as the outcome. RESULTS: Patients born in 1995 though 2006 had significantly increased survival compared with those born in 1980-1994 (log-rank test, p < 0.001). In a Cox model, patients born in 1995-2006 had a 70% reduction in the risk of death compared with those born in 1980-1994 (hazard ratio [HR] 0.3, 95% CI 0.2-0.5, p < 0.001) over a mean follow-up of 49.9 months (SD 61.1, median 22.0). However, when controlling for demographic and clinical care variables, year of birth was no longer significantly associated with age at death (HR 1.0, 95% CI 0.6-1.8, p = 0.9), whereas ventilation for more than 16 h/d, use of a mechanical insufflation-exsufflation device, and gastrostomy tube feeding showed a significant effect in reducing the risk of death. CONCLUSION: Survival in spinal muscular atrophy type 1 patients has increased in recent years, in relation to the growing trend toward more proactive clinical care. |
| first_indexed | 2024-03-07T05:31:10Z |
| format | Journal article |
| id | oxford-uuid:e251b93e-37a9-4d63-b0b9-e3ecb991975d |
| institution | University of Oxford |
| language | English |
| last_indexed | 2024-03-07T05:31:10Z |
| publishDate | 2007 |
| record_format | dspace |
| spelling | oxford-uuid:e251b93e-37a9-4d63-b0b9-e3ecb991975d2022-03-27T10:00:17ZThe changing natural history of spinal muscular atrophy type 1.Journal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:e251b93e-37a9-4d63-b0b9-e3ecb991975dEnglishSymplectic Elements at Oxford2007Oskoui, MLevy, GGarland, CGray, JO'Hagen, JDe Vivo, DKaufmann, PBACKGROUND: Noninvasive ventilation has become increasingly available to spinal muscular atrophy (SMA) patients since the early 1990 s. This is expected to have improved survival for SMA type 1 patients. OBJECTIVE: To assess whether there has been a change in survival in patients with SMA type 1 between 1980 and 2006. METHODS: We used deidentified, family-reported data from participants in the International Spinal Muscular Atrophy Patient Registry and obtained additional clinical information through a mail-in questionnaire. One hundred forty-three patients with SMA type 1 were included in the analysis. Survival of patients born in 1995-2006 (n = 78) was compared with that of patients born in 1980-1994 (n = 65), using the Kaplan-Meier method and Cox proportional hazards models with age at death as the outcome. RESULTS: Patients born in 1995 though 2006 had significantly increased survival compared with those born in 1980-1994 (log-rank test, p < 0.001). In a Cox model, patients born in 1995-2006 had a 70% reduction in the risk of death compared with those born in 1980-1994 (hazard ratio [HR] 0.3, 95% CI 0.2-0.5, p < 0.001) over a mean follow-up of 49.9 months (SD 61.1, median 22.0). However, when controlling for demographic and clinical care variables, year of birth was no longer significantly associated with age at death (HR 1.0, 95% CI 0.6-1.8, p = 0.9), whereas ventilation for more than 16 h/d, use of a mechanical insufflation-exsufflation device, and gastrostomy tube feeding showed a significant effect in reducing the risk of death. CONCLUSION: Survival in spinal muscular atrophy type 1 patients has increased in recent years, in relation to the growing trend toward more proactive clinical care. |
| spellingShingle | Oskoui, M Levy, G Garland, C Gray, J O'Hagen, J De Vivo, D Kaufmann, P The changing natural history of spinal muscular atrophy type 1. |
| title | The changing natural history of spinal muscular atrophy type 1. |
| title_full | The changing natural history of spinal muscular atrophy type 1. |
| title_fullStr | The changing natural history of spinal muscular atrophy type 1. |
| title_full_unstemmed | The changing natural history of spinal muscular atrophy type 1. |
| title_short | The changing natural history of spinal muscular atrophy type 1. |
| title_sort | changing natural history of spinal muscular atrophy type 1 |
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