Abstract | AIM: METHODS: RESULTS: All patients achieved successful engraftment. Urine glycosaminoglycans excretion was reduced or normalized, and the activity of leukocyte iduronate-2-sulphatase enzyme, absent before hematopoietic stem cell transplantation, remained constant, in all patients. Dysostosis multiplex progressed over time, according to the natural evolution of the disease. Joint stiffness improved in all affected districts. Hepatosplenomegaly decreased until it disappeared. The cardiovascular involvement stayed unchanged, as well as hearing loss. Skin became hyperelastical; face features seemed less coarse if compared to the natural evolution of the disease. Cerebral white matter alterations were constant in time. On the contrary, the hematopoietic stem cell transplantation did not prove to have long-term effectiveness on neurological symptoms of Hunter syndrome. CONCLUSION:
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Authors | R Annibali, L Caponi, A Morganti, M Manna, O Gabrielli, A Ficcadenti |
Journal | Minerva pediatrica
(Minerva Pediatr)
Vol. 65
Issue 5
Pg. 487-96
(Oct 2013)
ISSN: 0026-4946 [Print] Italy |
PMID | 24056375
(Publication Type: Case Reports, Journal Article)
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Topics |
- Child, Preschool
- Follow-Up Studies
- Hematopoietic Stem Cell Transplantation
- Humans
- Male
- Mucopolysaccharidosis II
(genetics, surgery)
- Phenotype
- Severity of Illness Index
- Time Factors
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