Therapeutic Management and Current Situation Related to the Use of Resources and Costs of Spinal Muscular Atrophy (SMA) in Spain: A Retrospective, Multicenter Analysis Based on Medical Record Review. TREaSURE Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Novartis
- 入组人数
- 69
- 试验地点
- 1
- 主要终点
- Cost per Year of Use of Resources for Medical Visits
研究概览
简要总结
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- — 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Cost per Year of Use of Resources for Medical Visits
时间窗: Up to 12 months
Medical visits included: * Primary care * Pediatric neurology * Rehabilitation physician * Traumatologist * Nutritionist * Pulmonologist * Cardiologist * Physical therapist * Nursing staff * Phoniatrist * Speech therapist * Rehabilitation sessions * Other specialists * Total medical visits * Hospital emergency room visits * Outpatient emergency room visit * Hospitalizations/Admissions to ward * Hospitalizations due to admissions to the intensive care unit (ICU) * Total hospitalizations (ward and ICU) * Therapy session separately from medical visits
Costs per Year of Use of Resources for Medical Tests
时间窗: Up to 12 months
Medical tests included: * Blood count * Vitamin D test * Blood creatine kinase test * Electromyogram * Brain magnetic resonance imaging * Muscle magnetic resonance imaging * Bone densitometry * Nocturnal polysomnography * Muscle biopsy * Nerve conduction velocity study * Blood amino acid test * DNA test to confirm diagnosis * Total additional tests
Cost per Year of Use of Resources for Treatment
时间窗: Up to 12 months
Treatments included: * Nusinersen (loading and maintenance dose) * Total Nusinersen * Risdiplam * Onasemnogene abeparvovec * Total treatments for SMA * Palivizumab * Influenza vaccine * Meningococcal vaccine * High protein shakes * Total adjuvant treatments * Total treatments
次要结局
- Number of Patients per Sociodemographic Category(Baseline)
- Sociodemographic: Age(Baseline)
- Sociodemographic: Weight(Baseline)
- Sociodemographic: Height(Baseline)
- Sociodemographic: Body mass index (BMI)(Baseline)
- Number of Patients per Clinical Characteristic Category(Baseline)
- Clinical Characteristic: Age From Which Information Was Available(Baseline)
- Clinical Characteristic: Time From Onset of Symptoms to Diagnosis(Baseline)
- Clinical Characteristic: Number of Patients Categorized by International Classification of Diseases (ICD) Code(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Type of Diagnosis of Muscle Atrophy(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Type of Mutation(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Survival of Motor Neuron 2 (SMN2) Gene Copy Number(Baseline)
- Clinical Characteristic: Number of Patients Categorized by SMA-associated Comorbidities(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Family History of SMA(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Type of Ventilatory Support(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Reason of Ventilatory Support Regimen(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(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Type of Nutritional Supplementation Received by Mouth(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(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Type of Joint Contracture(Baseline)
- Clinical Characteristic: Number of Patients Who Reached Each Motor Development Milestone(Baseline)
- Clinical Characteristic: Number of Patients Categorized by Relevant Surgical History in Addition to Scoliosis Surgery Related to SMA Categorized by Type of Surgery(Baseline)
- Number of Scheduled and Unscheduled Healthcare Visits per Patient per Year(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(Up to 12 months)
- Number of SMA-related Emergency Room Visits(Up to 12 months)
- Number of SMA-related Hospitalizations(Up to 12 months)
- Number of Patients Categorized by Reason for Hospitalization(Up to 12 months)
- Number of Patients Categorized by Reason for Admission to Intensive Care Unit (ICU)(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(Up to 12 months)
- Number of Patients Categorized by Type of Additional Tests Needed(Up to 12 months)
- Number of Times Each Additional Test Was Performed(Up to 12 months)
- Total Additional Tests(Up to 12 months)
- Number of Patients Categorized by Type of Treatment for SMA(Up to 6 years and 4 months)
- Number of Nusinersen Doses(Up to 6 years and 4 months)
- Risdiplam Dose(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)