PD71 survival outcomes and adherence to defined daily doses of imiglucerase: a 16-year Brazilian cohort study in Gaucher disease

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dc.creatorMarcus Carvalho Borin
dc.creatorFrancisco de Assis Acurcio
dc.creatorJuliana Alvares Teodoro
dc.creatorAugusto Afonso Guerra Júnior
dc.date.accessioned2026-01-15T18:24:05Z
dc.date.issued2025-01-07
dc.descriptionPôster apresentado no HTAi 2024, encontro que ocorreu em Sevilha, na Espanha.
dc.identifier.doi10.1017/S0266462324003283
dc.identifier.issn1471-6348
dc.identifier.urihttps://hdl.handle.net/1843/1431
dc.languageeng
dc.publisherUniversidade Federal de Minas Gerais
dc.relation.ispartofInternational Journal of Technology Assessment in Health Care
dc.rightsAcesso aberto
dc.subjectDoença de Gaucher
dc.subject.otherDoença de Gaucher
dc.subject.otherEstudo de coorte
dc.subject.otherImiglucerase
dc.subject.otherPacientes
dc.titlePD71 survival outcomes and adherence to defined daily doses of imiglucerase: a 16-year Brazilian cohort study in Gaucher disease
dc.typeArtigo de periódico
local.citation.issue1
local.description.resumoIntroduction Gaucher disease is characterized by a deficiency of the enzyme glucocerebrosidase and requires lifelong enzyme replacement therapy. Imiglucerase is the standard treatment, which improves patient survival and quality of life. While defined daily doses (DDD) offer a standardized metric, the relative efficacy of adhering strictly to these guidelines, compared with tailored lower doses, has not been fully explored. Methods A retrospective cohort study was conducted on 1,234 patients to investigate the survival outcomes associated with various levels of adherence to DDDs of imiglucerase, factoring in demographic diversity and comorbidity profiles, and to evaluate the feasibility of a more personalized dosing approach in the management of Gaucher disease. DDD adherence was categorized as equal to DDD, higher than DDD, or lower than DDD. Kaplan-Meier survival analysis, log-rank tests, and Cox proportional hazards models were used to assess survival probabilities over 16 years. Data on age, sex, comorbidities, and other demographic factors were collected to adjust for potential confounders. Results Over the 16-year period, our Kaplan-Meier survival analysis revealed distinct survival probabilities across the three groups based on their adherence to DDD of imiglucerase. Patients who received doses lower than DDD (n=880) had a survival probability of 91.8 percent. In contrast, those receiving doses equal to the DDD (n=15) had a 100 percent survival probability, since no events were observed in this group. The greater than DDD group (n=339) exhibited a survival probability of 81 percent. A log-rank test indicated a borderline statistical significance (p=0.058) in the survival distributions among the various DDD adherence levels, with a favorable trend in the lower dose group. Conclusions Our research indicates that lower than usual doses of imiglucerase may improve survival rates in patients with Gaucher disease. This finding suggests that reduced dosages could lead to better clinical outcomes with fewer side effects, highlighting the potential benefits of personalized dosing strategies. Further studies are needed to confirm these preliminary results and optimize dosing protocols.
local.publisher.countryBrasil
local.publisher.departmentFAR - DEPARTAMENTO DE FARMÁCIA SOCIAL
local.publisher.initialsUFMG
local.subject.cnpqCIENCIAS DA SAUDE
local.url.externahttps://www.cambridge.org/core/journals/international-journal-of-technology-assessment-in-health-care/article/pd71-survival-outcomes-and-adherence-to-defined-daily-doses-of-imiglucerase-a-16year-brazilian-cohort-study-in-gaucher-disease/B4821D204EC5F505E0998A1B4D998160

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