Beneficial effects of eculizumab regardless of prior transfusions or bone marrow disease: Results of the International Paroxysmal Nocturnal Hemoglobinuria Registry.
Antibodies, Monoclonal, Humanized
/ administration & dosage
Blood Transfusion
Bone Marrow Diseases
Combined Modality Therapy
Comorbidity
Complement Inactivating Agents
/ administration & dosage
Health Care Surveys
Hemoglobinuria, Paroxysmal
/ epidemiology
Humans
Prognosis
Registries
Treatment Outcome
aplastic anemia
blood transfusion
bone marrow diseases
eculizumab
paroxysmal hemoglobinuria
registries
thrombosis
Journal
European journal of haematology
ISSN: 1600-0609
Titre abrégé: Eur J Haematol
Pays: England
ID NLM: 8703985
Informations de publication
Date de publication:
Nov 2020
Nov 2020
Historique:
received:
10
03
2020
revised:
29
06
2020
accepted:
30
06
2020
pubmed:
9
7
2020
medline:
30
7
2021
entrez:
9
7
2020
Statut:
ppublish
Résumé
To evaluate the effects of eculizumab on transfusions and thrombotic events (TEs) in patients with and without prior history of transfusion in the International Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry. Registry patients enrolled on or before January 1, 2018, initiated on eculizumab no more than 12 months prior to enrollment, having known transfusion status for the 12 months before eculizumab initiation, and ≥12 months of Registry follow-up after eculizumab initiation, were included. Eculizumab treatment was associated with a 50% reduction in transfusions in patients with a transfusion history (10.6 units/patient-year before eculizumab vs 5.4 after; P < .0001), with greater reduction observed in those with no history of bone marrow disease vs those with bone marrow disease. Mean lactate dehydrogenase levels decreased from a mean of 6.7 to 1.4 times the upper limit of normal (ULN) in patients with transfusion history and from 5.1 to 1.2 times ULN in those with no transfusion history. TE and major adverse vascular event rates also decreased by 70% in patients with and without history of transfusion. The benefit of eculizumab therapy does not appear to be limited to any group defined by transfusion history or bone marrow disease history.
Substances chimiques
Antibodies, Monoclonal, Humanized
0
Complement Inactivating Agents
0
eculizumab
A3ULP0F556
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
561-570Subventions
Organisme : Alexion Pharmaceuticals, Inc.
Informations de copyright
© 2020 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
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