The investigators conduct a prospective cohort study to explore the treatment effectiveness of continuous infusion of intrathecal baclofen (ITB) for hereditary spastic paraplegia (HSP) in China, delve into the optimal timing for starting treatment, and investigate the response differences among different subtypes. The ultimate goal is to provide clinical evidence and guidance for the application of ITB in treating HSP in China, as well as improve the life expectancy and quality of life for HSP patients. The main questions it aims to answer are: 1. Changes in gait and motor function, as well as spasticity levels, compared to pre-surgery and control group after ITB surgery. 2. Changes in quality of life, pain, psychological and emotional status, and cognition compared to pre-surgery and control group after ITB surgery. 3. Complications following ITB surgery. 4. Impact of ITB surgery on the occurrence and progression of skeletal deformities. 5. Subgroup analysis: comparing surgical outcomes between different genotypes and between simple versus complex types. 6. Determine the optimal timing for ITB intervention.
Hereditary spastic paraplegias (HSPs) are a heterogeneous group of neurodegenerative disorders characterized by progressive spasticity and weakness of the lower limbs due to retrograde axonal degeneration of the corticospinal tract. Baclofen is a selective GABA-B receptor agonist and is commonly used for the treatment of spasticity. Baclofen can be administered orally or intrathecally by the surgical implantation of a specialized pump. Intrathecal baclofen (ITB) is significantly more potent for the treatment of spasticity than the oral form. The goal of this clinical trial is to explore the efficacy and safety of ITB in the treatment of HSP patients. This study is prospective, open-label, single center, and this trial will last for 3 years. A total of 50 patients will participate. Twenty-five patients who will receive ITB and the other 25 control patients will receive oral baclofen. Patients with HSP aged 14 to 70 years, with a Modified Ashworth Scale score of ≥3 in ≥2 joints of lower limb, will be recruited. Patients who do not agree to undergo ITB and/or those who have an inadequate response during intrathecal baclofen test will receive oral baclofen therapy and undergo natural history observation. Professional clinical evaluations are conducted regularly for both groups.
Study Type
OBSERVATIONAL
Enrollment
50
Shanghai Sixth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
RECRUITINGSix-Minute Walk Test(6-MWT)
The 6-MWT measures the distance a person can walk in six minutes. A shorter distance indicates lower exercise capacity.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Step length
Step length is measured by gait analysis device. Step length refers to the distance from the heel strike of one foot to the heel strike of the opposite foot. Longer step length indicates that the patient has improved lower limb muscle strength and coordination, and that spastic symptoms have been alleviated.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
walking speed
Walking speed is measured by gait analysis device. Walking speed refers to the distance covered by a person within a unit of time while walking, expressed in meters per minute (m/min). An increase in walking speed indicates an improvement in spasticity and motor function.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
knee flexion angle
Knee flexion angle is measured by gait analysis device.An increase in the knee flexion angle indicates improved motor function and reduced spasticity.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Modified Ashworth Scale(MAS)
range: 0-4, higher scores mean more severe spasticity.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Range of Motion (ROM) of Joints
ROM refers to the distance and direction a joint can move to its full potential. Flexion, extension, abduction, adduction of joints are measured.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Patient Global Impression of Change (PGIC) scale
range (1-7), lower scores mean a better outcome
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Oswestry Disability Index (ODI)
The ODI consists of 10 sections (items). Each section is scored on a scale of 0 to 5, with higher scores indicating greater levels of disability.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Numeric Rating Scale (NRS)
rang 0 to 10, higher scores mean a worse outcome
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
MMSE score
Mini-Mental State Examination (MMSE) : range: 0-30, higher scores mean a better outcome
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
MoCA score
Montreal Cognitive Assessment (MoCA) score: range: 0-30, higher scores mean a better outcome.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SDS
Self-Rating Depression Scale: Index score \< 50 Normal range; 50-59 Mild depression; 60-69 Moderate depression; ≥70 Severe depression
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SAS
Self-Rating Anxiety Scale (SAS): 20-44 Normal range; 45-59 Mild to moderate anxiety levels; 60-74 Marked to severe anxiety levels; 75 and above Extreme anxiety levels
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Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SPRS score
The change in the Spastic Paraplegia Rating Scale (SPRS) score : range: 0-52, higher scores mean a worse outcome
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Complications
Catheter breakage, blockage, infection, etc.
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
GMFM-88
Gross Motor Function Measure-88 (GMFM-88) score: range: 0-264, higher scores mean a better outcome
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
GAS
Goal Attainment Scale (GAS) - Family Goal Selection Version: range: -2-+2, higher scores mean a better outcome
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Foot deformity molding
Utilizing CT digital 3D reconstruction technology
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Scoliosis assessment
Measurement of the Cobb angle using spinal X-ray imaging
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively