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Use of letermovir in off-label indications: Infectious Diseases Working Party of European Society of Blood and Marrow Transplantation retrospective study
J. Styczyński, G. Tridello, A. Xhaard, M. Medinger, S. Mielke, M. Taskinen, N. Blijlevens, MAB. Rodriguez, C. Solano, E. Nikolousis, A. Biffi, AH. Groll, C. Junghanss, P. Tsirigotis, B. Lioure, J. Šrámek, E. Holler, F. Galaverna, F. Fagioli, N....
Language English Country Great Britain
Document type Journal Article
NLK
Free Medical Journals
from 1997 to 1 year ago
Freely Accessible Science Journals
from 1997 to 1 year ago
ProQuest Central
from 2000-01-01 to 1 year ago
Open Access Digital Library
from 1997-01-01
Health & Medicine (ProQuest)
from 2000-01-01 to 1 year ago
- MeSH
- Acetates MeSH
- Antiviral Agents therapeutic use MeSH
- Quinazolines MeSH
- Cytomegalovirus MeSH
- Child MeSH
- Adult MeSH
- Communicable Diseases * MeSH
- Bone Marrow MeSH
- Humans MeSH
- Off-Label Use MeSH
- Retrospective Studies MeSH
- Hematopoietic Stem Cell Transplantation * MeSH
- Check Tag
- Child MeSH
- Adult MeSH
- Humans MeSH
- Publication type
- Journal Article MeSH
Letermovir (LMV) is licensed for prophylaxis of CMV infection in allogeneic hematopoietic cell transplant adult CMV-seropositive patients. Due to its favorable safety profile, LMV brings potential for use in other clinical situations, outside the approved indication. The objective of the study was to analyze the efficacy and safety of the use of LMV in off-label indications in EBMT centers. A total of 49 patients were reported including 44 adults and 5 children. LMV was administered for: secondary prophylaxis (37 adults, 3 children), primary prophylaxis (2 children), pre-emptive treatment (5 adults), and therapy of CMV disease (2 adults; pneumonia, colitis). Cyclosporine was concomitantly used in 26 patients. Overall, LMV was used for a median 112 days (range: 10-473). Cumulative incidence of breakthrough infections during secondary prophylaxis was 10.1% (95% CI = 3.1-21.9). Prophylactic treatment with LMV resulted in 94.9% (95% CI = 81.0-98.7), and 81.9% (95% CI = 65.7-90.9) probability of, respectively, 60 and 120-day survival without CMV infection in patients receiving secondary prophylaxis. During therapy of CMV infection/disease, probability of 60 and 120-day overall survival was 100% and 71.4% (95% CI = 25.8-92.0), respectively. No breakthrough infection occurred in children on LMV prophylaxis. Adverse events were reported in 15/49 (30.4%) patients: the most common being nausea/vomiting (22.4%). In conclusion, the efficacy of the use of LMV as secondary prophylaxis was high, and the preliminary experience with the use of LMV for the treatment of patients with refractory CMV infection/disease was positive. Our data showed that higher dose or prolonged therapy did not result in increased rate of adverse events.
Attikon University General Hospital Athens Greece
Birmingham Heartlands Hospital Birmingham UK
Department of Hematology and Hematopoietic Cell Transplantation Medical University Poznan Poland
Department of Hematology and Oncology University Hospital Pilsen Pilsen Czech Republic
Department of Hematology Radboud University Center Nijmegen Nijmegen The Netherlands
Department of Histology and Embryology Faculty of Medicine Pilsen Czech Republic
Division of Hematology Department of Medicine Huddinge Karolinska Institutet Stockholm Sweden
EBMT Data Office Dept of Medical Statistics and Bioinformatics Leiden Netherlands
Hematology ICANS Strasbourg France
Hospital Clínico Universitario INCLIVA University of Valencia Valencia Spain
Hospital de la Princesa Madrid Spain
Hospital U Marqués de Valdecilla Santander Spain
Onco Ematologia Pediatrica Torino Italy
Pediatric Hematology Oncology Verona Italy
Service d'hématologie greffe Hôpital Saint Louis Université Paris Diderot 75010 Paris France
References provided by Crossref.org
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