Eculizumab in nocturnal paroxysmal hemoglobinuria and atypical hemolytic-uremic syndrome
Mengarelli C, Rey-Ares L,Soto N, Alcaraz A,Pichon-Riviere A, Augustovski F, García Martí S, Bardach A, Ciapponi A, López A
Record ID 32016000900
Spanish
Authors' recommendations:
There is evidence of moderate quality suggesting that eculizumab reduces the need for transfusions in patients with nocturnal paroxysmal hemoglobinuria. When used to treat the atypical hemolytic-uremic syndrome it returns platelet levels to normal and improves renal function by reducing dialysis requirements. No evidence of good quality showing benefit in survival was found. The evidence on its effectiveness in preventing recurrences in patients with hemolytic-uremic syndrome who will undergo kidney transplantation is scarce, consequently, no conclusion can be drawn as regards this indication.
The consulted consensus statements recommend it for all the indications. US health sponsors and the Australia, British, Canadian and French healthcare systems cover it for the treatment of atypical hemolytic uremic syndrome and nocturnal paroxysmal hemoglobinuria when the patients meet the stated criteria. These sponsors do not mention the use of eculizumab in preventing recurrences.
Latin American health sponsors do not cover it, being its high cost the main restriction to indicate it. Should it be covered, it is to note that there are no criteria in terms of length of treatment.
Details
Project Status:
Completed
Year Published:
2016
URL for published report:
www.iecs.org.ar/home-ets/
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Argentina
MeSH Terms
- Humans
- Hemoglobinuria, Paroxysmal
- Antibodies, Monoclonal, Humanized
- Atypical Hemolytic Uremic Syndrome
Contact
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.