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

被引：12

作者：

Cesar, Thibaut ^{[1
]}

Le, Minh P. ^{[2
,3
]}

Klifa, Roman ^{[1
,4
]}

Castelle, Martin ^{[1
]}

Fournier, Benjamin ^{[1
,4
,5
]}

Levy, Romain ^{[1
,4
,6
]}

Chbihi, Marwa ^{[1
,4
]}

Courteille, Virginie ^{[7
]}

Moshous, Despina ^{[1
,4
,7
,8
]}

Blanche, Stephane ^{[1
,4
]}

Alligon, Mickael ^{[7
]}

Leruez-Ville, Marianne ^{[9
,10
]}

Peytavin, Gilles ^{[2
,11
]}

Frange, Pierre ^{[9
,10
]}

Neven, Benedicte ^{[1
,4
,12
]}

机构：

[1] Necker Univ Hosp, Assistance Publ Hop Paris APHP, Pediat Hematol Immunol & Rheumatol Dept, Paris, France

[2] Hop Xavier Bichat, APHP, Pharmacol Dept, Paris, France

[3] INSERM, UMR S 1144, Paris, France

[4] Univ Paris Cite, Paris, France

[5] INSERM, Inst Imagine, Lab Lymphocyte Activat & Susceptibil EBV Infect, UMR1163, Paris, France

[6] Inserm, Imagine Inst, Lab Human Genet Infect Dis, UMR 1163, Paris, France

[7] Necker Univ Hosp, APHP, French Reference Ctr Primary Immune Deficiencies C, Paris, France

[8] INSERM, Imagine Inst, Lab Genome Dynam Immune Syst, UMR 1163, Paris, France

[9] Necker Univ Hosp, APHP, Lab Clin Microbiol, Paris 7328 FETUS,URP, Paris, France

[10] Univ Paris Cite, Paris 7328 FETUS,URP, Paris, France

[11] INSERM, IAME, UMR 1137, Paris, France

[12] INSERM, Inst Imagine, UMR S 1163, Lab Immunogenet Pediat Autoimmune Dis, Paris, France

来源：

JOURNAL OF CLINICAL IMMUNOLOGY | 2024年 / 44卷 / 01期

关键词：

Letermovir; CMV Prophylaxis; Hematopoietic stem cell Transplant; Pediatrics; Inborn Errors of Immunity; Pharmacokinetics; CYTOMEGALOVIRUS-INFECTION; REACTIVATION; CHILDREN; THERAPY; DISEASE; BLOOD;

D O I：

10.1007/s10875-023-01617-1

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

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.

引用

页数：9

共 32 条

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