Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study.
Adult
Antibodies, Monoclonal, Humanized
/ therapeutic use
Antineoplastic Combined Chemotherapy Protocols
/ therapeutic use
Complement Inactivating Agents
/ therapeutic use
Drug Resistance, Neoplasm
/ drug effects
Female
Follow-Up Studies
Hemoglobinuria, Paroxysmal
/ drug therapy
Hemolysis
/ drug effects
Humans
Male
Middle Aged
Prognosis
Salvage Therapy
Journal
Blood
ISSN: 1528-0020
Titre abrégé: Blood
Pays: United States
ID NLM: 7603509
Informations de publication
Date de publication:
07 02 2019
07 02 2019
Historique:
received:
26
09
2018
accepted:
18
11
2018
pubmed:
5
12
2018
medline:
19
10
2019
entrez:
5
12
2018
Statut:
ppublish
Résumé
Ravulizumab (ALXN1210), a new complement C5 inhibitor, provides immediate, complete, and sustained C5 inhibition. This phase 3, open-label study assessed the noninferiority of ravulizumab to eculizumab in complement inhibitor-naive adults with paroxysmal nocturnal hemoglobinuria (PNH). Patients with lactate dehydrogenase (LDH) ≥1.5 times the upper limit of normal and at least 1 PNH symptom were randomized 1:1 to receive ravulizumab or eculizumab for 183 days (N = 246). Coprimary efficacy end points were proportion of patients remaining transfusion-free and LDH normalization. Secondary end points were percent change from baseline in LDH, change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score, proportion of patients with breakthrough hemolysis, stabilized hemoglobin, and change in serum free C5. Ravulizumab was noninferior to eculizumab for both coprimary and all key secondary end points (
Identifiants
pubmed: 30510080
pii: S0006-4971(20)42810-1
doi: 10.1182/blood-2018-09-876136
pmc: PMC6367644
doi:
Substances chimiques
Antibodies, Monoclonal, Humanized
0
Complement Inactivating Agents
0
eculizumab
A3ULP0F556
ravulizumab
C3VX249T6L
Banques de données
ClinicalTrials.gov
['NCT02946463']
Types de publication
Clinical Trial, Phase III
Comparative Study
Journal Article
Multicenter Study
Randomized Controlled Trial
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
530-539Commentaires et corrections
Type : CommentIn
Informations de copyright
© 2019 by The American Society of Hematology.
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