Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study.


Journal

Blood
ISSN: 1528-0020
Titre abrégé: Blood
Pays: United States
ID NLM: 7603509

Informations de publication

Date de publication:
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, a new complement component C5 inhibitor administered every 8 weeks, was noninferior to eculizumab administered every 2 weeks in complement-inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria (PNH). This study assessed noninferiority of ravulizumab to eculizumab in clinically stable PNH patients during previous eculizumab therapy. In this phase 3, open-label, multicenter study, 195 PNH patients on labeled-dose (900 mg every 2 weeks) eculizumab for >6 months were randomly assigned 1:1 to switch to ravulizumab (n = 97) or continue eculizumab (n = 98). Primary efficacy end point was percentage change in lactate dehydrogenase (LDH) from baseline to day 183. Key secondary end points included proportion of patients with breakthrough hemolysis, change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score, transfusion avoidance, and stabilized hemoglobin. In 191 patients completing 183 days of treatment, ravulizumab was noninferior to eculizumab (

Identifiants

pubmed: 30510079
pii: S0006-4971(20)42811-3
doi: 10.1182/blood-2018-09-876805
pmc: PMC6368201
doi:

Substances chimiques

Antibodies, Monoclonal, Humanized 0
Complement C5 0
Complement Inactivating Agents 0
eculizumab A3ULP0F556
ravulizumab C3VX249T6L

Banques de données

ClinicalTrials.gov
['NCT03056040']

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

540-549

Commentaires et corrections

Type : CommentIn

Informations de copyright

© 2019 by The American Society of Hematology.

Références

Ann Hematol. 2017 Feb;96(2):171-181
pubmed: 27837250
Biol Pharm Bull. 2016;39(2):285-8
pubmed: 26830487
Br J Haematol. 2013 Jul;162(1):62-73
pubmed: 23617322
Lancet Neurol. 2017 Dec;16(12):976-986
pubmed: 29066163
Blood. 2019 Feb 7;133(6):530-539
pubmed: 30510080
F1000Res. 2016 Jun 27;5:
pubmed: 27408701
Hematol Rep. 2017 Jun 01;9(2):7053
pubmed: 28626544
PLoS One. 2018 Apr 12;13(4):e0195909
pubmed: 29649283
N Engl J Med. 2006 Sep 21;355(12):1233-43
pubmed: 16990386
Blood. 2015 Jan 29;125(5):775-83
pubmed: 25477495
Am J Hematol. 2016 Jun;91(4):366-70
pubmed: 26689746
Blood. 2014 Oct 30;124(18):2804-11
pubmed: 25237200
Blood. 2008 Feb 15;111(4):1840-7
pubmed: 18055865
Am J Kidney Dis. 2016 Jul;68(1):84-93
pubmed: 27012908
MMWR Morb Mortal Wkly Rep. 2017 Jul 14;66(27):734-737
pubmed: 28704351
N Engl J Med. 2013 Jun 6;368(23):2169-81
pubmed: 23738544
Blood. 2011 Jun 23;117(25):6786-92
pubmed: 21460245
Blood. 2007 Dec 1;110(12):4123-8
pubmed: 17702897
Blood. 2013 Jun 20;121(25):4985-96; quiz 5105
pubmed: 23610373
Kidney Int. 2015 May;87(5):1061-73
pubmed: 25651368
Kidney Int. 2016 Mar;89(3):701-11
pubmed: 26880462
Nat Rev Dis Primers. 2017 May 18;3:17028
pubmed: 28516949
Neurol Clin. 2018 May;36(2):311-337
pubmed: 29655452
Biologics. 2008 Jun;2(2):205-22
pubmed: 19707355

Auteurs

Austin G Kulasekararaj (AG)

Department of Haematological Medicine, King's College Hospital, National Institute of Health Research/Wellcome King's Clinical Research Facility, London, United Kingdom.

Anita Hill (A)

Department of Haematology, Leeds Teaching Hospitals, Leeds, United Kingdom.

Scott T Rottinghaus (ST)

Alexion Pharmaceuticals, Inc., Boston, MA.

Saskia Langemeijer (S)

Radboudumc, Nijmegen, The Netherlands.

Richard Wells (R)

Sunnybrook Health Sciences Centre, Toronto, ON, Canada.

F Ataulfo Gonzalez-Fernandez (FA)

Hospital Clinico Universitario San Carlos, Department of Hematology, The Clínic Institute of Haematological and Oncological Diseases, Madrid, Spain.

Anna Gaya (A)

Hospital Clínic de Barcelona, Barcelona, Spain.

Jong Wook Lee (JW)

Department of Hematology, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea.

Emilio Ojeda Gutierrez (EO)

Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain.

Caroline I Piatek (CI)

Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles, CA.

Jeff Szer (J)

Clinical Haematology, Royal Melbourne Hospital, Melbourne, VIC, Australia.

Antonio Risitano (A)

Hematology, Department of Clinical Medicine and Surgery, Federico II University, Naples, Italy.

Shinji Nakao (S)

Haematology/Respiratory Medicine, Faculty of Medicine, Institute of Medical Pharmaceutical and Health Sciences, Kanazawa University, Kanazawa, Ishikawa, Japan.

Eric Bachman (E)

Alexion Pharmaceuticals, Inc., Boston, MA.

Lori Shafner (L)

Alexion Pharmaceuticals, Inc., Boston, MA.

Andrew I Damokosh (AI)

Alexion Pharmaceuticals, Inc., Boston, MA.

Stephan Ortiz (S)

Alexion Pharmaceuticals, Inc., Boston, MA.

Alexander Röth (A)

Department of Hematology, West German Cancer Center, University Hospital Essen, Essen, Germany.

Regis Peffault de Latour (R)

Bone Marrow Transplantation Unit, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint Louis Hospital and University Paris Diderot, Paris, France; and.
Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation, Leiden, The Netherlands.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH