Agalsidase beta treatment slows estimated glomerular filtration rate loss in classic Fabry disease patients: results from an individual patient data meta-analysis
| Autor: | Alaa Hamed, Steve Kanters, Rachel Goldgrub, Manish Maski, Pronabesh DasMahapatra, Dieter Ayers, Jeroen P. Jansen, Eugene Poggio, Alberto Ortiz, Elvira Ponce, Robert J. Desnick, Mario Aguiar |
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| Rok vydání: | 2020 |
| Předmět: |
medicine.medical_specialty
030232 urology & nephrology Urology Renal function 03 medical and health sciences 0302 clinical medicine Linear regression medicine 030212 general & internal medicine individual patient data meta-analysis AcademicSubjects/MED00340 Fabry disease glomerular filtration rate Transplantation Proteinuria chronic kidney disease outcomes business.industry Original Articles medicine.disease Confidence interval AGALSIDASE BETA Nephrology Meta-analysis agalsidase beta medicine.symptom business classic phenotype Kidney disease |
| Zdroj: | Clinical Kidney Journal |
| ISSN: | 2048-8513 |
| DOI: | 10.1093/ckj/sfaa065 |
| Popis: | Background Fabry disease is a rare, X-linked genetic disorder that, if untreated in patients with the Classic phenotype, often progresses to end-stage kidney disease. This meta-analysis determined the effect of agalsidase beta on loss of estimated glomerular filtration rate (eGFR) in the Classic phenotype using an expansive evidence base of individual patient-level data. Methods The evidence base included four Sanofi-Genzyme studies and six studies from a systematic literature review. These were restricted to Classic Fabry patients meeting the eligibility criteria from Phases III and IV agalsidase beta trials, including 315 patients (161 treated). Linear regression was first used to model annual change in eGFR for each patient and the resulting annualized eGFR slopes were modelled with treatment and covariates using quantile regression. These results were then used to estimate median annualized eGFR change in agalsidase beta treated versus untreated groups. Results Imbalances across treatment groups were found in baseline age, sex and proteinuria, but not in the use of renin–angiotensin system blockers. The adjusted model suggests that treated (agalsidase beta) patients experienced a slower median eGFR decrease [2.46 mL/min/1.73 m2/year slower; 95% confidence interval (CI) 0.63–4.29; P = 0.0087] than comparable untreated patients. The median eGFR decrease was 2.64 mL/min/1.73 m2/year slower (95% CI 0.53–4.78; P = 0.0141) in treated Classic males. Conclusions Using an expansive evidence base and robust modelling approach, these data indicate that agalsidase beta-treated patients with the Classic phenotype conserve their renal function better than untreated patients. |
| Databáze: | OpenAIRE |
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