10.6084/m9.figshare.9985997.v1
Sandra Obikawa Kyosen
Sandra Obikawa
Kyosen
Leny Toma
Leny
Toma
Helena Bonciani Nader
Helena Bonciani
Nader
Marion Coting Braga
Marion Coting
Braga
Vanessa Gonçalves Pereira
Vanessa Gonçalves
Pereira
Sueli Canossa
Sueli
Canossa
João Bosco Pesquero
João Bosco
Pesquero
Vânia D’Almeida
Vânia
D’Almeida
Ana Maria Martins
Ana Maria
Martins
CASE SERIES OF PATIENTS UNDER BIWEEKLY TREATMENT WITH LARONIDASE: A REPORT OF A SINGLE CENTER EXPERIENCE
SciELO journals
2019
Enzyme replacement therapy
Metabolism, inborn errors
Glycosaminoglycans
Mucopolysaccharidosis I
2019-10-16 02:52:08
Dataset
https://scielo.figshare.com/articles/dataset/CASE_SERIES_OF_PATIENTS_UNDER_BIWEEKLY_TREATMENT_WITH_LARONIDASE_A_REPORT_OF_A_SINGLE_CENTER_EXPERIENCE/9985997
<div><p>ABSTRACT 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.</p></div>