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Real-World Eculizumab Dosing Patterns Among Patients with Paroxysmal Nocturnal Hemoglobinuria in a US Population.
Cheng, Wendy Y; Sarda, Sujata P; Mody-Patel, Nikita; Krishnan, Sangeeta; Yenikomshian, Mihran; Kunzweiler, Colin; Vu, Jensen Duy; Cheung, Hoi Ching; Duh, Mei Sheng.
Afiliação
  • Cheng WY; Analysis Group, Inc., Boston, MA, USA.
  • Sarda SP; Apellis Pharmaceuticals, Inc., Waltham, MA, USA.
  • Mody-Patel N; Apellis Pharmaceuticals, Inc., Waltham, MA, USA.
  • Krishnan S; Apellis Pharmaceuticals, Inc., Waltham, MA, USA.
  • Yenikomshian M; Analysis Group, Inc., Boston, MA, USA.
  • Kunzweiler C; Analysis Group, Inc., Boston, MA, USA.
  • Vu JD; Analysis Group, Inc., Boston, MA, USA.
  • Cheung HC; Analysis Group, Inc., Boston, MA, USA.
  • Duh MS; Analysis Group, Inc., Boston, MA, USA.
Clinicoecon Outcomes Res ; 14: 357-369, 2022.
Article em En | MEDLINE | ID: mdl-35535299
Purpose: Current pharmacologic management of paroxysmal nocturnal hemoglobinuria (PNH) consists of C5 inhibitors, eculizumab and ravulizumab; however, because patients experience incomplete symptom control, off-label doses may be utilized. We conducted a retrospective, longitudinal cohort study of provider-based claims data to assess the real-world eculizumab dosing patterns in PNH patients. Patients and Methods: Patients were ≥12 years, received ≥2 eculizumab infusions between January 1, 2015 and September 30, 2019, and had ≥3 months of continuous clinical activity prior to index. The index date was the first claim for eculizumab. Patients with ≥1 diagnosis of another indication for eculizumab were excluded. Treatment patterns including the proportion with high, label-recommended, and low dosages during induction (first 28 days) and maintenance (beginning day 29) phases were described. The proportion and time-to-first dose escalation, defined as an increase in dose or frequency of infusion, were assessed among a subset of patients (ie, escalation analysis cohort). Results: A total of 707 patients were examined. Mean (standard deviation [SD]) starting dose was 862mg (412mg) and was higher than label-recommended 600mg for 64% of the patients. Mean (SD) dose per infusion was 859mg (391mg) during the induction phase; average dose was higher than label-recommended 600mg for 68%. Mean (SD) dose per infusion during the maintenance phase was 1005mg (335mg); average dose was higher than label-recommended 900mg for 43%. Dose escalation occurred in 40/121 escalation analysis cohort patients. Median time-to-first dose escalation was ~12 months. Conclusion: Results suggest that deviations from label-recommended dosing patterns were common. Future budget impact assessments of eculizumab should account for real-world dosing patterns to comprehensively assess costs and benefits.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Observational_studies Idioma: En Revista: Clinicoecon Outcomes Res Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Observational_studies Idioma: En Revista: Clinicoecon Outcomes Res Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos