CASE SERIES OF PATIENTS UNDER BIWEEKLY TREATMENT WITH LARONIDASE: A REPORT OF A SINGLE CENTER EXPERIENCE

• Main Authors: Sandra Obikawa Kyosen, Leny Toma, Helena Bonciani Nader, Marion Coting Braga, Vanessa Gonçalves Pereira, Sueli Canossa, João Bosco Pesquero, Vânia D’Almeida, Ana Maria Martins
• Format: Article
• Language: English
• Published: Sociedade de Pediatria de São Paulo 2019-05-01
• Series: Revista Paulista de Pediatria
• Subjects: Terapia de reposição de enzimas; Erros inatos do metabolismo; Glicosaminoglicanos; Mucopolissacaridose I
• Online Access: http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0103-05822019005008106&lng=en&tlng=en

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.