Back to Search
Start Over
Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults
- Source :
- Scientia, Kidney international reports, Vol. 6, no.6, p. 1603-1613 (2021), Kidney International Reports, Kidney International Reports, Elsevier, 2021, 6 (6), pp.1603-1613. ⟨10.1016/j.ekir.2021.03.884⟩, Kidney International Reports, Vol 6, Iss 6, Pp 1603-1613 (2021)
- Publication Year :
- 2021
-
Abstract
- Hemolytic uremic syndrome; Kidney failure; Ravulizumab Síndrome hemolítico urémico; Insuficiencia renal; Ravulizumab Síndrome hemolític urèmic; Insuficiència renal; Ravulizumab Introduction Atypical hemolytic uremic syndrome (aHUS) is a rare, complex, multisystem disease of dysregulated complement activity, characterized by progressive thrombotic microangiopathy (TMA), acute kidney injury, and multiorgan dysfunction, which often progresses to chronic kidney disease. Results from the prospective clinical trial of ravulizumab (NCT02949128) reveal rapid resolution of TMA in patients with aHUS, with sustained efficacy and safety in a 26-week initial evaluation period. Methods The aim of this analysis was to characterize the long-term efficacy and the safety profile of ravulizumab in adults with aHUS who had completed the initial evaluation period of the trial. Complete TMA response, hematologic and kidney functions, and safety were evaluated for all patients available for follow-up in the extension period (median follow-up: 76.7 weeks; range: 0.6–118.3). This trial included a total of 58 patients, 49 of whom entered the extension period. Results A total of 4 additional patients achieved complete TMA response during the follow-up period. Normalization of platelet count, serum lactate dehydrogenase (LDH), and hemoglobin observed in the 26-week initial evaluation period was sustained until the last available follow-up, as were the improvements in the estimated glomerular filtration rate (eGFR) and patient quality of life. All efficacy endpoints were correlated with the sustained inhibition of complement C5. Most adverse events (AEs) occurred early during the initial evaluation period and decreased substantially during the extension period. No patient developed a meningococcal infection or died during the extension period. Conclusion This analysis reveals that ravulizumab administered every 8 weeks is efficacious with an acceptable safety profile for the long-term treatment of adults with aHUS and provides additional clinical benefit beyond 6 months of treatment.
- Subjects :
- medicine.medical_specialty
Thrombotic microangiopathy
[SDV]Life Sciences [q-bio]
Hemic and Lymphatic Diseases::Hematologic Diseases::Anemia::Anemia, Hemolytic::Hemolytic-Uremic Syndrome::Hemic and Lymphatic Diseases::Hematologic Diseases::Atypical Hemolytic Uremic Syndrome [DISEASES]
030232 urology & nephrology
Renal function
Otros calificadores::Otros calificadores::/farmacoterapia [Otros calificadores]
030204 cardiovascular system & hematology
Other subheadings::Other subheadings::/drug therapy [Other subheadings]
03 medical and health sciences
0302 clinical medicine
afecciones patológicas, signos y síntomas::procesos patológicos::atributos de la enfermedad::enfermedades raras [ENFERMEDADES]
Clinical Research
Internal medicine
Atypical hemolytic uremic syndrome
Medicine
complement
Adverse effect
Complement component 5
business.industry
Pathological Conditions, Signs and Symptoms::Pathologic Processes::Disease Attributes::Rare Diseases [DISEASES]
atypical hemolytic uremic syndrome
Acute kidney injury
diagnóstico::pronóstico::resultado del tratamiento [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS]
medicine.disease
Diagnosis::Prognosis::Treatment Outcome [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT]
ravulizumab
Diseases of the genitourinary system. Urology
3. Good health
enfermedades hematológicas y linfáticas::enfermedades hematológicas::anemia::anemia hemolítica::síndrome hemolítico-urémico::enfermedades hematológicas y linfáticas::enfermedades hematológicas::síndrome hemolítico urémico atípico [ENFERMEDADES]
kidney failure
thrombotic microangiopathy
Clinical trial
Nephrology
Avaluació de resultats (Assistència sanitària)
hemolytic uremic syndrome
RC870-923
Síndrome hemolíticourèmica - Tractament
Malalties rares
business
Kidney disease
Subjects
Details
- Language :
- English
- ISSN :
- 24680249
- Database :
- OpenAIRE
- Journal :
- Scientia, Kidney international reports, Vol. 6, no.6, p. 1603-1613 (2021), Kidney International Reports, Kidney International Reports, Elsevier, 2021, 6 (6), pp.1603-1613. ⟨10.1016/j.ekir.2021.03.884⟩, Kidney International Reports, Vol 6, Iss 6, Pp 1603-1613 (2021)
- Accession number :
- edsair.doi.dedup.....e93611cd369b5d9e8ce50d9bc608f0db