[Eculizumab in the management of paroxysmal nocturnal hemoglobinuria and atypical hemolytic-uremic syndrome]

Mengarelli C, Pichon-Riviere A, Augustovski F, Alcaraz A, García Martí S, Bardach A, Ciapponi A.
Record ID 32018001592
Original Title: Eculizumab en hemoglobinuria paroxística nocturna y síndrome urémico hemolítico atípico
Authors' recommendations: Moderate-quality evidence suggests that the use of Eculizumab in patients with paroxysmal nocturnal hemoglobinuria decreases the need for transfusions, reduces thrombotic events and improves survival in this group of patients. Moderate-quality evidence from patients with atypical hemolytic-uremic syndrome suggests that treatment with Eculizumab might prevent disease relapse and improve kidney function recovery in adults and children at long term. In the cohorts of patients with kidney transplantation, the use of Eculizumab pre- and post-transplantation might reduce relapsing microangiopathy associated to this syndrome and especially in patients who show gene mutation associated to the membrane cofactor protein. Many consensuses of experts and recommendations from health systems such as those from the U.S., Australia, UK, France, Brazil, Colombia and Mexico recognize its benefits; however as the treatment is extremely expensive, they limit its coverage to patients who meet specific criteria and usually under special agreement on prices only, in order to reduce its budget impact. Other countries such as Canada and Scotland do not cover Eculizumab for any indication due to its poor cost-effectiveness and its high budget impact. In Argentina, its use is covered under the Exceptional Regime for Access to Non-registered Drugs (Régimen de Acceso de Excepción de Medicamentos No Registrados).
Project Status: Completed
Year Published: 2020
URL for published report: https://www.iecs.org.ar/home-ets/
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Argentina
MeSH Terms
  • Atypical Hemolytic Uremic Syndrome
  • Hemoglobinuria, Paroxysmal
  • Antibodies, Monoclonal, Humanized
  • Complement Inactivating Agents
  • Complement System Proteins
Organisation Name: Institute for Clinical Effectiveness and Health Policy
Contact Address: Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA
Contact Name: info@iecs.org.ar
Contact Email: info@iecs.org.ar
Copyright: Institute for Clinical Effectiveness and Health Policy (IECS)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.