CASE SERIES OF PATIENTS UNDER BIWEEKLY TREATMENT WITH LARONIDASE: A REPORT OF A SINGLE CENTER EXPERIENCE
Autor: | Helena B. Nader, Marion Coting Braga, Vânia D'Almeida, Sandra Obikawa Kyosen, João Bosco Pesquero, Ana Maria Martins, Leny Toma, Vanessa Gonçalves Pereira, Sueli Canossa |
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Rok vydání: | 2019 |
Předmět: |
Male
Every Two Weeks Adolescent Mucopolysaccharidosis I Urinary system Terapia de reposição de enzimas Single Center Iduronidase Young Adult 03 medical and health sciences Mucopolysaccharidosis type I 0302 clinical medicine Erros inatos do metabolismo Humans Medicine Child Glicosaminoglicanos Adverse effect Retrospective Studies Glycosaminoglycans 030304 developmental biology 0303 health sciences business.industry lcsh:RJ1-570 lcsh:Pediatrics Retrospective cohort study Original Articles Enzyme replacement therapy Mucopolissacaridose I Regimen Treatment Outcome Metabolism inborn errors Anesthesia Pediatrics Perinatology and Child Health Female business 030217 neurology & neurosurgery |
Zdroj: | Revista Paulista de Pediatria, Iss 0 (2019) Revista Paulista de Pediatria v.37 n.3 2019 Revista Paulista de Pediatria (Ed. Português. Online) Sociedade de Pediatria de São Paulo (SPSP) instacron:SPSP Revista Paulista de Pediatria |
ISSN: | 1984-0462 0103-0582 |
Popis: | Objective: To report the stabilization of urinary glycosaminoglicans (GAG) excretion and clinical improvements in patients with mucopolysaccharidosis type I (MPS I) under an alternative dose regimen of laronidase of 1.2 mg/kg every other week. Methods: We participated in a dose-optimization trial for laronidase in MPS-I patients using four alternative regimens: 0.58 mg/kg every week, 1.2 mg/kg every two weeks, 1.2 mg/kg every week and 1.8 mg/kg every other week (EOW). After the trial ended, the patients resumed the recommended dose and regimen of 0.58 mg/kg every week. Under this regimen, some patients presented difficulties in venous access and were unable to commute weekly to the treatment center. Therefore, we used an alternative regimen that consisted of 1.2 mg/kg EOW in eight patients. A retrospective study of medical records of MPS-I patients who underwent both enzyme replacement therapy (ERT) regimens, of 0.58 mg/kg every week and 1.2 mg/kg EOW, was done. Results: Patients remained clinically stable under the alternative regimen, did not present elevation of urinary GAG nor any adverse event. Conclusions: The switch of dose regimen to 1.2 mg/kg EOW of laronidase was safe, and did not cause any clinical worsening in patients who had been previously under standard dose ERT. |
Databáze: | OpenAIRE |
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