Hearing improvement in patients with Fabry disease treated with agalsidase alfa
| Autor: | D Hajioff, S Goodwin, Atul Mehta, KD MacDermot, R. E. Quiney, J Zuckerman |
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| Rok vydání: | 2007 |
| Předmět: |
Adult
Male medicine.medical_specialty Pediatrics Adolescent Hearing loss Hearing Loss Sensorineural Otoacoustic emission Placebo Double-Blind Method otorhinolaryngologic diseases medicine Humans Alpha-galactosidase biology medicine.diagnostic_test Otitis Media with Effusion business.industry General Medicine Enzyme replacement therapy Middle Aged medicine.disease Fabry disease Recombinant Proteins Surgery Isoenzymes alpha-Galactosidase Pediatrics Perinatology and Child Health biology.protein Fabry Disease Sensorineural hearing loss medicine.symptom Audiometry business |
| Zdroj: | Acta Paediatrica. 92:28-30 |
| ISSN: | 0803-5253 |
| DOI: | 10.1111/j.1651-2227.2003.tb00217.x |
| Popis: | Aim: To describe the nature and prevalence of hearing loss in Fabry disease, and its response to enzyme replacement therapy (ERT) with agalsidase alfa. Methods: Fifteen male patients with Fabry disease were enrolled in a randomized, double-blind study and received placebo (n = 8) or ERT (n = 7) with agalsidase alfa for 6 months. This was followed by an open-label extension of 36 months thus far. Alongside this trial, an additional eight men and two women have so far received open-label ERT for between 6 and 30 months. Pure-tone audiometry, impedance audiometry and otoacoustic emission testing were performed at 0 (baseline), 6, 18, 30 and 42 months. Results: Nine patients (36%) had bilateral and ten (40%) had unilateral high-frequency sensorineural hearing loss (SNHL). Three (12%) had unilateral middle ear effusions with conductive losses persisting beyond 6 months. Only five patients (20%) had normal hearing. The highfrequency SNHL deteriorated over the first 6 months in both placebo and active treatment groups by a median 6.3 dB (p < 0.0001, Wilcoxon matched-pairs). This hearing loss subsequently improved above baseline by 1.5 dB at 18 months (p = 0.07), by 5.0 dB at 30 months (p = 0.006) and by 4.0 dB at 42 months (p = 0.01). Conclusion: Significant hearing loss, usually high-frequency SNHL, is a common manifestation of Fabry disease in adults. α-Galactosidase A replacement therapy with agalsidase alfa appears to reverse the hearing deterioration in these patients. This improvement, however, is gradual, suggesting the need for long-term ERT. |
| Databáze: | OpenAIRE |
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