Outcomes of Autologous Bone Marrow Mononuclear Cell Administration in the Treatment of Neurologic Sequelea in Children With Spina Bifida

Study Overview
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Brief Summary
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The aim of this study was to evaluate the safety and efficacy of autologous bone marrow mononuclear cell infusion in the management of neurological sequelae in children with spina bifida

Detailed Description
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The aim of this study was to evaluate the safety and effectiveness of autologous bone marrow mononuclear cells in 11 patients with spina bifida at Vinmec Research Institute of Stem Cell and Gene Technology in Hanoi, Vietnam from 2016 to 2020

Official Title
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Autologous Bone Marrow Mononuclear Cell Administration in the Treatment of Neurologic Sequela in Children With Spina Bifida

Conditions
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Stem Cell Infusion

Intervention / Treatment
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* Combination Product: Autologous bone marrow mononuclear cell transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The patient who was diagnosed with lumbar spina bifida underwent spinal cord close-up surgery. Both genders. Aged between 6 months and 15 years old. Exhibited bowel disorders (constipation, fecal incontinence) and urinary dysfunction (urinary retention or leakage). Exclusion Criteria: Vertebrae clefts in the chest, neck, and other spinal locations. Coagulopathy. Acute and chronic infection. Kidney function disorder, liver failure Patients with complex cardiovascular diseases (including valvular heart disease, cardiomyopathy, arrhythmia, congenital heart disease, hypertrophy syndrome). Distress

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 15 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Autologous BMMNC transplantation<br>- Autologous bone marrow mononuclear cell transplantation 2 intrathecal administrations of autologous bone marrow mononuclear cells at baseline and 6 months afterward | Combination Product: Autologous bone marrow mononuclear cell transplantation<br>* Transplantation of Autologous Bone Marrow Mononuclear cells<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events and serious adverse events | Incidence of the adverse events or serious adverse events after infusion | up to the 12-month period following treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Bristol stool scale | The Bristol stool scale comes in 7 types: Type 1-2 indicate constipation; type 3-4 are ideal stools as they are easier to pass; type 5-7 may show diarrhea and urgency. | up to the 12-month period following treatment | 
| Rectoanal inhibitory reflex | The rectoanal inhibitory reflex (RAIR) is a reflex characterized by transient involuntary relaxation of the internal anal sphincter in response to distention of the rectum with the normal value <= 14.7 ml | up to the 12-month period following treatment | 
| Bladder sensation | The cystometry was used to assess bladder sensation | up to the 12-month period following treatment | 
| Urinary retention | urinary retention is assessed via cytometry | up to the 12-month period following treatment | 
| Urinary incontinence | Urinary incontinence is assessed via cytometry | up to the 12-month period following treatment | 
| Lower limb motor functions | Lower limb motor function was assessed via manual muscle testing (MMT) | up to the 12-month period following treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Spina bifida, mononuclear cell, neurologic sequelea