Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation.
Pediatr Res
; 87(1): 104-111, 2020 01.
Article
em En
| MEDLINE
| ID: mdl-31434105
ABSTRACT
BACKGROUND:
Mucopolysaccharidosis I (MPS IH) is a lysosomal storage disease treated with hematopoietic cell transplantation (HCT) because it stabilizes cognitive deterioration, but is insufficient to alleviate all somatic manifestations. Intravenous laronidase improves somatic burden in attenuated MPS I. It is unknown whether laronidase can improve somatic disease following HCT in MPS IH. The objective of this study was to evaluate the effects of laronidase on somatic outcomes of patients with MPS IH previously treated with HCT.METHODS:
This 2-year open-label pilot study of laronidase included ten patients (age 5-13 years) who were at least 2 years post-HCT and donor engrafted. Outcomes were assessed semi-annually and compared to historic controls.RESULTS:
The two youngest participants had a statistically significant improvement in growth compared to controls. Development of persistent high-titer anti-drug antibodies (ADA) was associated with poorer 6-min walk test (6MWT) performance; when patients with high ADA titers were excluded, there was a significant improvement in the 6MWT in the remaining seven patients.CONCLUSIONS:
Laronidase seemed to improve growth in participants <8 years old, and 6MWT performance in participants without ADA. Given the small number of patients treated in this pilot study, additional study is needed before definitive conclusions can be made.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Mucopolissacaridose I
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Transplante de Células-Tronco Hematopoéticas
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Terapia de Reposição de Enzimas
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Iduronidase
Tipo de estudo:
Clinical_trials
/
Diagnostic_studies
Limite:
Adolescent
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Child
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Child, preschool
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Female
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Humans
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Male
Idioma:
En
Revista:
Pediatr Res
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
Estados Unidos