Immediate and Sustained Effects of Intensive Equine-Assisted Physiotherapy in Children With Spinal Muscular Atrophy
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 21
- 试验地点
- 2
- 主要终点
- The ability of the thorax to change shape
研究概览
简要总结
This prospective interventional study evaluates the immediate and sustained effects of intensive equine-assisted physiotherapy in children with spinal muscular atrophy (SMA). The study compares an intensive equine-assisted physiotherapy program (NEUROEQUIP-SMA) with an individualized physiotherapy program based on SMA standard of care and neuroproprioceptive facilitation and inhibition principles (SMA-SOC-N). Both interventions are delivered as intensive 6-day rehabilitation programs. Outcomes include motor function, postural control, respiratory function, movement quality assessed by three-dimensional motion analysis, neuromuscular activity, quality of life, and selected molecular biomarkers. The study also evaluates the safety and tolerability of intensive rehabilitation in children with SMA.
详细描述
Spinal muscular atrophy is rare neuromuscular disease, it is caused by gene mutation survival motor neuron 1 (SMN1). This gene encodes survival motor neuron (SMN) protein. This protein is most important to survive alfa-motoric neurons in spinal cord. When this protein doesn´t work, no signals go from brain to skeletal muscles and fazic muscles of body. In the end weakening breathing muscles and internal organs muscles.
Disease-modifying therapies have substantially changed the natural history of SMA. In the Czech Republic, currently available pharmacological treatment includes nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma) and risdiplam (Evrysdi). As survival and motor abilities of children with SMA improve, rehabilitation has become an increasingly important part of comprehensive care. Physiotherapy is one of the recommended rehabilitation types.
The use of rehabilitation procedures is common in clinical practice. In a 2016 study, 105 adults with SMA were asked how many of them used physiotherapy. A total of 86% of respondents received these services, yet the authors of the article conclude that further research is needed to understand the impact of physiotherapy in SMA.
Nevertheless, there is a lot of discussion if physiotherapy is really good for people and children with SMA. Some research shows us, that physiotherapy doesn´t worsen this disease. Researchers showed that strength training 3 times a week does not show side effects in children under 10 years of age, in some cases trends in improving muscle strength and motor functions have been observed.
Because in physiotherapy therapists work primarily with muscles, and a lot of activity can cause fatigue of skeletal muscles and worsen their function. But from clinical work scientists know that the effect of physiotherapy is extensive. There is a research on mice which shows the effect of physiotherapy on the strength activities and the aerobic activities.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Single (Outcomes Assessor)
盲法说明
All participants of the study will undergo a clinical examination including a questionnaire survey, led by a blinded independent examiner, who will not have access to intervention documentation. The examiner will see each patient twice, before the first intervention and 6 days later in the end last therapy. The examiner will be prohibited to talk about the therapy attended. The parents of patients will be informed about the character of the examination ant the importance of the most objectivity possible. All therapists can apply both interventions, therefore masking is more efficient.
入排标准
- 年龄范围
- 1 Year 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •clinic diagnosis SMA (I, II, and III types)
- •no changing medicine 6 months at least
- •without another grave diseases
排除标准
- •hip luxation
- •allergies to horses and the environment of horse stables
- •insurmountable fear of the horse
研究组 & 干预措施
Therapeutic grooming
In order to influence the psychomotor development in a comprehensive way, the psychosocial activity of therapeutic grooming will also be included in the study. Its goal is to support children's communication, their interaction with the environment, the ability to establish contact with the horse and the overall emotional support of children with SMA.
干预措施: Therapeutic grooming (Procedure)
Individual physiotherapy (SMA-SOC-N)
Individual physiotherapy will be performed according to the SMA-SOC-N protocol, based on the principles of the international Standard of Care (SMA-SOC) recommendations and adapted to the individual needs of children with spinal muscular atrophy.
干预措施: Individual physiotherapy (SMA-SOC-N) (Procedure)
Equine-Assisted Physiotherapy Based on Neuroproprioceptive Facilitation and Inhibition
Equine-assisted physiotherapy based on neuroproprioceptive facilitation and inhibition is an accredited form of hippotherapy. Its methodology was originally developed based on the clinical presentation of children with cerebral palsy; however, its therapeutic principles are applicable to a broader group of children with neurological disabilities, including children with spinal muscular atrophy.
干预措施: Equine-Assisted Physiotherapy Based on Neuroproprioceptive Facilitation and Inhibition (Procedure)
结局指标
主要结局
The ability of the thorax to change shape
时间窗: 15 minutes
The examination will take place lying on your back. 3 position markers will be placed on each side of the thorax, in the area of the distal end of the sternum axes, at the level of the 3rd rib and at the lowest arch of the 10th rib. Their position during exhalation and inspiration will be measured, and the change in thorax circumference will be evaluated. The aim of this test is to objectify the change in respiratory stereotype. After therapy, investigators expect a greater ability of the thorax to change direction.
Work with the center of gravity
时间窗: 10 minutes
Observation will be performed by sitting on therapeutic cylinder. 3 position markers will be used on each side of the body, at the root of the palm, in the area of the SI joint and at the level of the temporomandibular joint. The change of markers will be measured from the starting position until the child is stretched to the side (torso tilt with lug) and forward (torso flexion with forearm). The monitoring child will be motivated to reach for the toy. The goal is to evaluate the stability of children. Investigators assume, that after therapy investigators can see better work with the center of gravity.
Muscle fatigue
时间窗: 10 minutes
Investigators will measure muscle fatigue after physical activity using surface EMG. The child will lie on his back, therapist will provide a toy in the area of his opposite knee. The child will stretch for a toy. This act will be 30 s. Investigators will evaluate activity m.obliquus externus abdominis at the beginning and end of this specific activity. Goal is to evaluate muscle fatigue. Investigators assume, that after intensive therapeutic program will be muscle fatigue lower.
Sitting symmetry
时间窗: 5 minutes
Observations will be made by sitting on a therapy cylinder. There will be special markers on the body (on clothes) of children: lower angles of the scapulae axes, ridge of the iliac axes, sacroiliac joint (SI) on both sides and in the area of the L4 vertebra and navel. Investigators will measure the symmetry of these marks in each other's position and the movement in which the child will walk back and forth with his hands. Markers will be analyzed using Qualisys Motion Capture Systems 2020.3. Acceleration and change of position will be evaluated in the second time.
Deep neck flexor endurance test
时间窗: 5 minutes
The examination will follow a standardized procedure. Longer holding in the position means a better result, the exact holding time is not standardized for this test.
Neck extensor endurance test
时间窗: 5 minutes
The examination will follow a standardized procedure. Longer holding in the position means a better result, the exact holding time is not standardized for this test.
Hammersmith function scale for children with spinal muscular atrophy
时间窗: 40 minutes
The examination will follow a standardized procedure. The maximum score of the standardized test is 64 points on each side, the minimum is 0 points. A higher score means an improvement in the child's movement possibilities.
Spirometric measure
时间窗: 2 minutes
The spirometer will be used to determine the forced expiratory volume in 1 second forced expiratory volume at one second (FEV1) value, i.e the volume of air exhaled after the maximum inspiration with the greatest effort within 1 second. The change in value at the beginning and end of the rehabilitation stay will be evaluated.
Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
时间窗: Baseline and Day 7
The CHOP INTEND is a validated clinical scale designed to assess motor function in infants and young children with spinal muscular atrophy. It consists of 16 items. Total scores range from 0 to 64, with higher scores indicating better motor function.
Deep neck flexor endurance test
时间窗: Baseline and Day 7
The examination will follow a standardized procedure. Longer holding in the position means a better result, the exact holding time is not standardized for this test.
Abdominal Breathing Ability Test
时间窗: Baseline and end Day 7
The child performs deep breathing while sitting astride a therapy cylinder. Reflective markers attached to predefined anatomical landmarks are recorded using the Qualisys Motion Capture System during a 30-second measurement. The test evaluates thoracic and abdominal movement during breathing. Improved function is reflected by greater abdominal contribution to the breathing pattern.
Test of Trunk and Cervical Spine Coordination Ability
时间窗: Baseline and Day 7
The child performs repeated back-and-forth upper limb movements while sitting on a therapy cylinder (typically 3-6 repetitions). The test evaluates trunk and cervical spine coordination by analyzing the synchronization of markers placed on the dorsum of the hand, C7 vertebra, and temporomandibular joint. Improved function is reflected by better coordination of trunk, upper limb, and cervical spine movements with reduced compensatory cervical spine extension.
Ability to Work With the Center of Gravity
时间窗: Baseline and day 7
While sitting on a therapy cylinder, the child performs repeated reaching movements with one upper limb (3-6 repetitions during a 30-second recording). The test evaluates postural stability and control of the center of gravity by analyzing displacement of body markers during the task. Improved function is reflected by greater controlled displacement of the center of gravity while maintaining postural stability.
Muscle fatigue
时间窗: Baseline and Day 7
Investigators will measure muscle fatigue after physical activity using surface EMG. The child will lie on his back, therapist will provide a toy in the area of his opposite knee. The child will reach for a toy. This act will be 30 s. Investigators will evaluate activity m.obliquus externus abdominis at the beginning and end of this specific activity. Goal is to evaluate muscle fatigue. Investigators assume, that after intensive therapeutic program will be muscle fatigue lower.
Hammersmith function scale for children with spinal muscular atrophy
时间窗: Baseline and Day 7
The examination will follow a standardized procedure. The maximum score of the standardized test is 64 points on each side, the minimum is 0 points. A higher score means an improvement in the child's movement possibilities.
Neck extensor endurance test
时间窗: Baseline and Day 7
The examination will follow a standardized procedure. Longer holding in the position means a better result, the exact holding time is not standardized for this test.
Spirometric measure
时间窗: Baseline and Day 7
The spirometer will be used to determine the forced expiratory volume in one second (FEV1) value, i.e. the volume of air exhaled after maximal inspiration with the greatest effort within the first second and peak expiratory flow (PEF). The change in both parameters at the beginning and end of the rehabilitation stay will be evaluated.
次要结局
- Monitoring of molecular biological indicators of rehabilitation(10 minutes)
- Quality of life assessment(120 minutes)
- ICF-based assessment(Baseline; 28 days after completion of rehabilitation)
- Monitoring of molecular biological indicators of rehabilitation(Baseline and Day 7)
- Home video recording(Baseline; 28 days after completion of rehabilitation)
研究者
Kamila Řasová
assoc. prof. PhDr. Kamila Řasová, Ph.D.
Charles University, Czech Republic