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Otologic and laryngologic manifestations of mucopolysaccharidoses after bone marrow transplantation
B C Papsin1, A Vellodi, C M Bailey
1Department of Otolaryngology, Great Ormond Street Hospital for Children, Toronto, Ontario, Canada.
Insights
Bone marrow transplantation may lessen hearing loss and voice issues in children with mucopolysaccharidosis. This treatment appears to reduce the severity and progression of these common symptoms in affected children.
Area of Science:
- Pediatric medicine
- Genetics
- Otolaryngology
Background:
- Mucopolysaccharidoses (MPS) cause widespread functional impairment due to glycosaminoglycan deposition.
- Otolaryngologic manifestations in MPS include hearing loss and voice abnormalities.
- These symptoms significantly impact quality of life in affected children.
Purpose of the Study:
- To evaluate the impact of bone marrow transplantation (BMT) on otolaryngologic symptoms in children with MPS.
- To assess changes in hearing and voice function post-BMT.
Main Methods:
- Hearing and voice assessments were conducted on eleven children post-BMT.
- Non-transplanted patients' data were used for comparison.
- Study focused on functional outcomes in the otolaryngologic system.
Main Results:
- Children who underwent BMT showed variable, but on average less severe, hearing loss compared to non-transplanted peers.
- The incidence of otitis media with effusion was reduced in the BMT group.
- Approximately 50% of transplanted children exhibited normal voice function post-treatment.
Conclusions:
- Bone marrow transplantation likely diminishes the progression of hearing loss in MPS patients.
- BMT may also reduce the severity of voice abnormalities in children with mucopolysaccharidosis.
Introduction:
Children with mucopolysaccharidosis have significant functional impairment of a number of systems because of deposition of glycosaminoglycans. The otolaryngologic system is affected, resulting in conductive and sensorineural hearing loss and voice abnormality.
Method:
Eleven children who had undergone bone marrow transplantation had their hearing and voices assessed. Unmatched data from patients who had not undergone transplantation were collected for nonstatistical comparison.
Results:
The hearing loss in children who had undergone bone marrow transplantation was variable but on average was less severe than in the younger children who had not undergone transplantation. The incidence of otitis media with effusion was diminished in the transplanted group. Approximately half had normal voices.
Discussion:
Diminution of the progression of hearing loss and voice abnormality probably occurs in patients after treatment by bone marrow transplantation.