Letermovir for CMV Prophylaxis in Very High-Risk Pediatric Hematopoietic Stem Cell Transplantation Recipients for Inborn Errors of Immunity.

Autor: César T; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France., Le MP; Pharmacology Department, APHP, Bichat Hospital, Paris, France.; INSERM UMR_S 1144, Paris, France., Klifa R; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France.; Université Paris Cité, Paris, France., Castelle M; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France., Fournier B; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France.; Université Paris Cité, Paris, France.; Laboratory of Lymphocyte Activation and Susceptibility to EBV Infection, INSERM UMR1163, Institut Imagine, Paris, France., Lévy R; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France.; Université Paris Cité, Paris, France.; Laboratory of Human Genetics of Infectious Diseases, Inserm UMR 1163, Imagine Institute, Paris, France., Chbihi M; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France.; Université Paris Cité, Paris, France., Courteille V; French reference center for primary immune deficiencies (CEREDIH), Necker University Hospital, APHP, Paris, France., Moshous D; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France.; Université Paris Cité, Paris, France.; French reference center for primary immune deficiencies (CEREDIH), Necker University Hospital, APHP, Paris, France.; Laboratory of Genome Dynamics in the Immune System, Imagine Institute, INSERM UMR 1163, Paris, France., Blanche S; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France.; Université Paris Cité, Paris, France., Alligon M; French reference center for primary immune deficiencies (CEREDIH), Necker University Hospital, APHP, Paris, France., Leruez-Ville M; Laboratory of Clinical Microbiology, APHP, Necker University Hospital, & Université Paris Cité, Paris, 7328 FETUS, URP, France., Peytavin G; Pharmacology Department, APHP, Bichat Hospital, Paris, France.; IAME, INSERM UMR 1137, Paris, France., Frange P; Laboratory of Clinical Microbiology, APHP, Necker University Hospital, & Université Paris Cité, Paris, 7328 FETUS, URP, France., Neven B; Pediatric Hematology-Immunology and Rheumatology Department, Assistance Publique Hôpitaux de Paris (APHP), Necker University Hospital, Paris, France. benedicte.neven@aphp.fr.; Université Paris Cité, Paris, France. benedicte.neven@aphp.fr.; Laboratory of Immunogenetics of Pediatric Autoimmune Diseases, Imagine Institute, INSERM UMR- S_1163, Paris, France. benedicte.neven@aphp.fr.
Jazyk: angličtina
Zdroj: Journal of clinical immunology [J Clin Immunol] 2023 Dec 20; Vol. 44 (1), pp. 6. Date of Electronic Publication: 2023 Dec 20.
DOI: 10.1007/s10875-023-01617-1
Abstrakt: The burden of CMV infection and disease is important in pediatric hematopoietic stem cell transplantation (HSCT), notably in the subgroup of patients with inborn errors of immunity (IEIs). Letermovir (LMV) is now a standard of care for CMV prophylaxis in adult sero-positive (R+) recipients, but is not yet labeled for children. Published pediatric studies are still scarce. We report a monocentric real-life use of LMV in 36 HSCT pediatric recipients with IEIs considered at high-risk of CMV infection including 14 patients between 2 and 12 months of age. A homogenous dosage proportional to the body surface area was used. Pharmacokinetic (PK) was performed in 8 patients with a median of 6 years of age (range 0,6;15). The cumulative incidence of clinically significant CMV infections (CS-CMVi) and the overall survival of patients under LMV were compared to a very similar historical cohort under (val)aciclovir prophylaxis. LMV tolerance was good. As compared to the historical cohort, the incidence of CS-CMVi was significantly lower in LMV group (5 out of 36 transplants (13.9%) versus 28 of the 62 HSCT (45.2%)) (p = 0.002). Plasma LMV exposures did not significantly differ with those reported in adult patients. In this high-risk pediatric HSCT cohort transplanted for IEIs, CMV prophylaxis with LMV at a homogenous dosage was well tolerated and effective in preventing CS-CMVi compared with a historical cohort.
(© 2023. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)
Databáze: MEDLINE
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