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Non-Malignant Disorders Treated With Cord Blood |
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| Disorder |
Number Treated |
Outcome |
Ref. |
|
|
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| Hurler's syndrome |
20 |
17 of the 20 children were alive a median of 905 days after transplantation, with complete donor chimerism and normal peripheral-blood alpha-L-iduronidase activity |
[100] |
| Duchenne muscular dystrophy |
1 |
On 42nd day, physical examination revealed obviously improvement in walking, turning the body over, and standing up |
[101] |
| Malignant infantile osteopetrosis |
1 |
Normalization of spine bone mineral density. |
[102] |
| Rothmund-Thomson syndrome |
1 |
Complete immune reconstitution |
[55] |
| Buerger's disease |
4 |
Ischemic rest pain suddenly disappeared. Digital capillaries were increased in number and size. |
[84] |
| Spinal Cord Injury |
1 |
Improved sensory perception and movement in the SPI patient's hips and thighs within 41 days of cell transplantation. Regeneration of the spinal cord at the injured site |
[85] |
| Krabbe's disease |
25 |
Progressive central myelination and continued gains in developmental skills, and most had age-appropriate cognitive function and receptive language skills in patient subset |
[14] |
| Omenn syndrome |
1 |
T cell reconstitution |
[103] |
| Non-healing wounds |
2 |
Accelerated healing |
[86] |
| Refractory anemia |
3 |
All patients are alive and free of disease at between 17 and 39 months after cord blood administration |
[104] |
| Diamond-Blackfan anemia |
1 |
Successful seroconversion to vaccines (diphtheria, pertussis, tetanus, rubella, measles, and BCG) administered 22–34 months post-transplant. |
[105] |
| Severe chronic active Epstein-Barr virus |
1 |
Complete remission without circulating EBV-DNA has continued for 15 months transplant. |
[106] |
| Behcet's disease |
1 |
Twenty-three months after CBT, the patient is doing well and has no signs or symptoms of Behcet's disease |
[9] |
| Mucopolysaccharidosis type IIB (Hunter syndrome) |
1 |
Two years after transplant approximately 55% normal plasma iduronate sulfatase. activity has been restored and abnormal urinary excretion of glycosaminoglycans has nearly completely resolved. |
[107] |
Riordan et al. Journal of Translational Medicine 2007 5:8 doi:10.1186/1479-5876-5-8 |
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