Clinical trial NCT06632730: Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Spinal Muscular Atrophy Type I
Spinal Muscular Atrophy Type II
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-10-09
Enrollment: 69
Sex: ALL
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This was a retrospective, noninterventional cohort study.

Brief summary: An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).


Eligibility criteria: Inclusion criteria:

* Patients diagnosed with SMA type I or SMA type II.
* Patients with medical records available in the hospital.
* Patients diagnosed with SMA type I or SMA type II after 2017 for whom a minimum of 6 months of follow-up was available.

Exclusion criteria:

• Patients who participated in an experimental design study except for those in the long-term follow-up period, during the observation period.

Primary outcomes:
measure: Cost per Year of Use of Resources for Medical Visits
timeFrame: Up to 12 months
measure: Costs per Year of Use of Resources for Medical Tests
timeFrame: Up to 12 months
measure: Cost per Year of Use of Resources for Treatment
timeFrame: Up to 12 months
Secondary outcomes:
measure: Number of Patients per Sociodemographic Category
timeFrame: Baseline
measure: Sociodemographic: Age
timeFrame: Baseline
measure: Sociodemographic: Weight
timeFrame: Baseline
measure: Sociodemographic: Height
timeFrame: Baseline
measure: Sociodemographic: Body mass index (BMI)
timeFrame: Baseline
measure: Number of Patients per Clinical Characteristic Category
timeFrame: Baseline
measure: Clinical Characteristic: Age From Which Information Was Available
timeFrame: Baseline
measure: Clinical Characteristic: Time From Onset of Symptoms to Diagnosis
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by International Classification of Diseases (ICD) Code
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Type of Diagnosis of Muscle Atrophy
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Type of Mutation
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Survival of Motor Neuron 2 (SMN2) Gene Copy Number
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by SMA-associated Comorbidities
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Family History of SMA
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Type of Ventilatory Support
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Reason of Ventilatory Support Regimen
timeFrame: Baseline
measure: Clinical Characteristic: Duration of Ventilatory Support
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients With Nutritional Supplementation
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Type of Administration of Nutritional Supplementation
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Type of Nutritional Supplementation Received by Mouth
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Who Received Liquid Thickener and Nutritional Supplements by Mouth
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Who Received Liquid Thickener and Nutritional Supplements Through a Feeding Tube
timeFrame: Baseline
measure: Clinical Characteristic: Duration of Nutritional Supplementation Received by Mouth and Through a Feeding Tube or Gastrostomy
timeFrame: Baseline
measure: Clinical Characteristic: Number of Surgeries for Patients With Scoliosis
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Type of Surgery Received for Scoliosis
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Type of Joint Contracture
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Who Reached Each Motor Development Milestone
timeFrame: Baseline
measure: Clinical Characteristic: Number of Patients Categorized by Relevant Surgical History in Addition to Scoliosis Surgery Related to SMA Categorized by Type of Surgery
timeFrame: Baseline
measure: Number of Scheduled and Unscheduled Healthcare Visits per Patient per Year
timeFrame: Up to 12 months
measure: Total Number of Scheduled and Unscheduled Healthcare Visits per Patient per Year
timeFrame: Up to 12 months
measure: Number of Scheduled and Unscheduled SMA-related Medical Visits
timeFrame: Up to 12 months
measure: Number of SMA-related Emergency Room Visits
timeFrame: Up to 12 months
measure: Number of SMA-related Hospitalizations
timeFrame: Up to 12 months
measure: Number of Patients Categorized by Reason for Hospitalization
timeFrame: Up to 12 months
measure: Number of Patients Categorized by Reason for Admission to Intensive Care Unit (ICU)
timeFrame: Up to 12 months
measure: Number of Patients With a Multidisciplinary Follow-up
timeFrame: Up to 12 months
measure: Number of Patients Categorized by Medical Areas Involved in Follow-up
timeFrame: Up to 12 months
measure: Number of Patients Categorized by Type of Additional Tests Needed
timeFrame: Up to 12 months
measure: Number of Times Each Additional Test Was Performed
timeFrame: Up to 12 months
measure: Total Additional Tests
timeFrame: Up to 12 months
measure: Number of Patients Categorized by Type of Treatment for SMA
timeFrame: Up to 6 years and 4 months
measure: Number of Nusinersen Doses
timeFrame: Up to 6 years and 4 months
measure: Risdiplam Dose
timeFrame: Up to 6 years and 4 months
measure: Palivizumab Dose
timeFrame: Up to 6 years and 4 months
measure: Number of High Protein Shakes per Day
timeFrame: Up to 6 years and 4 months
measure: Number of Months of Taking High Protein Shakes
timeFrame: Up to 6 years and 4 months
Other outcomes:
Locations:
city: East Hanover
country: United States
facility: Novartis Pharmaceuticals
state: New Jersey
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: