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ONH-SOD Female, 10 years old, March 2019

Summary

Diagnosis Sex Age Nationality
Optic Nerve Hypoplasia (ONH) / Septooptic Dysplasia (SOD) Female 10 years Brazilian
Injections Cell type Admission date Discharge date
8 UCBSC 2019-03-11 2019-03-28

Condition On Admission

The patient had light perception only. She had hormone deficiency (GH, thyroid, and cortisol) due to an atrophy in the pituitary gland.

Treatment Schedule

The patient received 8 umbilical cord blood-derived stem cell (UCBSC) packets by intravenous (IV), intrathecal and retrobulbar injections, as per the schedule below:

Number Date Cell Type Delivery Method Side Effects
1 2019-03-12 UCBSC Intravenous Injection none reported
2 2019-03-15 UCBSC Intrathecal Injection &
Intravenous Injection
none reported
3 2019-03-18 UCBSC &
Intrathecal Injection
none reported
4 2019-03-22 UCBSC &
Intrathecal Injection
none reported
5 2019-03-26 UCBSC Intravenous Injection none reported

Condition at discharge

At discharge the patient already started to show some improvements; She could move her eyes following directions better than before. She could slowly move her eyes up and down and left and right. She could name the colors yellow, red, blue and purple by looking at colored lights (after she learned these colors). In addition, she could compare the size of shiny objects, finding out which ones were smaller or bigger and she could also identify their shapes.

Condition 12 months after treatment

At the 12 month follow up point, the patient’s parents reported that the patient’s quality of life had moderately improved and that improvements were continually being made. They also mentioned: “Before the treatment he only had light perception, today he is able to count on fingers.” Please see an excerpt from the patient’s parents 12 month assessment below:

Symptom Parents’ Assessment of Improvement
Ability to focus eyes quickly Small improvement
Ability to keep eyes focused for a long time Moderate improvement
Ability to see hand movement Moderate improvement
Ability to see things at a close distance Moderate improvement
Ability to see things at a far distance Significant improvement
Ability to see things clearly Small improvement
Able to count fingers Moderate improvement
Blindness Moderate improvement
Colour vision Significant improvement
Light perception Moderate improvement
Night vision Significant improvement
Nystagmus Moderate improvement
Strabismus Small improvement
Vision in left eye Moderate improvement
Vision in right eye Moderate improvement
Visual field Small improvement