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An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Spinal Muscular Atrophy (SMA) Cohort | Pediatric patients with SMA type I, SMA type II, or presymptomatic SMA. |
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| Measure | Description | Time Frame |
|---|---|---|
| Cost per Year of Use of Resources for Medical Visits | Medical visits included:
| Up to 12 months |
| Costs per Year of Use of Resources for Medical Tests | Medical tests included:
| Up to 12 months |
| Cost per Year of Use of Resources for Treatment | Treatments included:
| Up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients per Sociodemographic Category | Sociodemographics included:
|
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Inclusion criteria:
Exclusion criteria:
• Patients who participated in an experimental design study except for those in the long-term follow-up period, during the observation period.
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This was a retrospective, noninterventional cohort study.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Pharmaceuticals | East Hanover | New Jersey | 07936 | United States |
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| ID | Term |
|---|---|
| D014897 | Spinal Muscular Atrophies of Childhood |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| Baseline |
| Sociodemographic: Age |
| Baseline |
| Sociodemographic: Weight | Baseline |
| Sociodemographic: Height | Baseline |
| Sociodemographic: Body mass index (BMI) | Baseline |
| Number of Patients per Clinical Characteristic Category | Clinical characteristics included:
| Baseline |
| Clinical Characteristic: Age From Which Information Was Available | Baseline |
| Clinical Characteristic: Time From Onset of Symptoms to Diagnosis | Time intervals included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by International Classification of Diseases (ICD) Code | ICD codes included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Type of Diagnosis of Muscle Atrophy | Diagnosis types included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Type of Mutation | Mutations included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Survival of Motor Neuron 2 (SMN2) Gene Copy Number | SMN2 copy numbers: 0, 1, 2, 3, 4, and greater than 4. | Baseline |
| Clinical Characteristic: Number of Patients Categorized by SMA-associated Comorbidities | SMA-associated comorbidities included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Family History of SMA | Family history categories included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Type of Ventilatory Support | Types of ventilatory support included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Reason of Ventilatory Support Regimen | Reasons of ventilatory support included:
| Baseline |
| Clinical Characteristic: Duration of Ventilatory Support | Baseline |
| Clinical Characteristic: Number of Patients With Nutritional Supplementation | Baseline |
| Clinical Characteristic: Number of Patients Categorized by Type of Administration of Nutritional Supplementation | Types of administration included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Type of Nutritional Supplementation Received by Mouth | Types of nutritional supplementation included liquid thickeners (corn starch and Nutilis Clear thickener, neutral taste, Nutricia) and nutrition supplements (Resource shakes, High protein shakes, Ensure Junior drink, Infatrini, Pediasure Complete, RESOURCE C, Vitamin D, Fortini high protein shakes, protein shakes, Pediasure, Resource with fiber, Resource protein). | Baseline |
| Clinical Characteristic: Number of Patients Who Received Liquid Thickener and Nutritional Supplements by Mouth | Baseline |
| Clinical Characteristic: Number of Patients Who Received Liquid Thickener and Nutritional Supplements Through a Feeding Tube | Baseline |
| Clinical Characteristic: Duration of Nutritional Supplementation Received by Mouth and Through a Feeding Tube or Gastrostomy | Baseline |
| Clinical Characteristic: Number of Surgeries for Patients With Scoliosis | Baseline |
| Clinical Characteristic: Number of Patients Categorized by Type of Surgery Received for Scoliosis | Types of surgeries included:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Type of Joint Contracture | Types of joint contractures included proximal and distal. | Baseline |
| Clinical Characteristic: Number of Patients Who Reached Each Motor Development Milestone | Developmental Milestones:
| Baseline |
| Clinical Characteristic: Number of Patients Categorized by Relevant Surgical History in Addition to Scoliosis Surgery Related to SMA Categorized by Type of Surgery | Surgeries included:
| Baseline |
| Number of Scheduled and Unscheduled Healthcare Visits per Patient per Year | Healthcare visits included:
| Up to 12 months |
| Total Number of Scheduled and Unscheduled Healthcare Visits per Patient per Year | Up to 12 months |
| Number of Scheduled and Unscheduled SMA-related Medical Visits | SMA-related medical visits included:
| Up to 12 months |
| Number of SMA-related Emergency Room Visits | SMA-related emergency room visits included outpatient and hospital visits. | Up to 12 months |
| Number of SMA-related Hospitalizations | SMA-related hospitalizations included visits to day hospital, admissions to ward, intensive care unit admissions. | Up to 12 months |
| Number of Patients Categorized by Reason for Hospitalization | Hospitalization included admission to ward. Reason for hospitalization included related to treatment and not related to treatment. | Up to 12 months |
| Number of Patients Categorized by Reason for Admission to Intensive Care Unit (ICU) | Reason for admission to ICU included related to treatment and not related to treatment. | Up to 12 months |
| Number of Patients With a Multidisciplinary Follow-up | Up to 12 months |
| Number of Patients Categorized by Medical Areas Involved in Follow-up | Medical areas included:
| Up to 12 months |
| Number of Patients Categorized by Type of Additional Tests Needed | Additional tests included:
| Up to 12 months |
| Number of Times Each Additional Test Was Performed | Additional tests included:
| Up to 12 months |
| Total Additional Tests | Up to 12 months |
| Number of Patients Categorized by Type of Treatment for SMA | Treatments included:
| Up to 6 years and 4 months |
| Number of Nusinersen Doses | Nusinersen doses included loading and maintenance doses. | Up to 6 years and 4 months |
| Risdiplam Dose | Risdiplam dose included starting dose and dose changes. | Up to 6 years and 4 months |
| Palivizumab Dose | Up to 6 years and 4 months |
| Number of High Protein Shakes per Day | Up to 6 years and 4 months |
| Number of Months of Taking High Protein Shakes | Up to 6 years and 4 months |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D016472 | Motor Neuron Disease |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |