English


ONH-SOD Female, 10 years old, March 2019

Summary

DiagnosisSexAgeNationality
Optic Nerve Hypoplasia (ONH) / Septooptic Dysplasia (SOD)Female 10 yearsBrazilian
InjectionsCell typeAdmission dateDischarge date
8UCBSC2019-03-112019-03-28

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:

NumberDateCell TypeDelivery MethodSide Effects
12019-03-12UCBSCIntravenous Injectionnone reported
22019-03-15UCBSCIntrathecal Injection &
Intravenous Injection
none reported
32019-03-18UCBSC &
Intrathecal Injection
none reported
42019-03-22UCBSC &
Intrathecal Injection
none reported
52019-03-26UCBSCIntravenous Injectionnone 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:

SymptomParents’ Assessment of Improvement
Ability to focus eyes quicklySmall improvement
Ability to keep eyes focused for a long timeModerate improvement
Ability to see hand movementModerate improvement
Ability to see things at a close distanceModerate improvement
Ability to see things at a far distanceSignificant improvement
Ability to see things clearlySmall improvement
Able to count fingersModerate improvement
BlindnessModerate improvement
Colour visionSignificant improvement
Light perceptionModerate improvement
Night visionSignificant improvement
NystagmusModerate improvement
StrabismusSmall improvement
Vision in left eyeModerate improvement
Vision in right eyeModerate improvement
Visual fieldSmall improvement