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Updated: Jul 19, 2025
Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
Published on: May 27, 2011
Letermovir for Cytomegalovirus prophylaxis in pediatric hematopoietic stem cell transplantation
Teaghan T Chen1, Alexandria P David1, Erin K Barthelmess1
1Department of Pharmacy, Children's Hospital Colorado, Aurora, Colorado, USA.
Insights
Letermovir (LTV) prophylaxis shows promise in reducing Cytomegalovirus (CMV) DNAemia in pediatric hematopoietic cell transplant (HCT) patients. This study suggests LTV is well-tolerated and effective for CMV prevention in this vulnerable population.
Area of Science:
- Hematology
- Infectious Diseases
- Pharmacology
Background:
- Letermovir (LTV) is approved for Cytomegalovirus (CMV) prophylaxis in adult hematopoietic cell transplant (HCT) recipients.
- Its use in pediatric HCT patients is off-label with limited supporting literature.
Purpose of the Study:
- To evaluate the efficacy and safety of LTV prophylaxis for CMV infection in pediatric HCT recipients.
- To compare rates of clinically significant CMV DNAemia between LTV and non-LTV prophylaxis groups.
Main Methods:
- A single-center, retrospective, matched (1:1) cohort study of pediatric allogeneic HCT recipients (2015-2022).
- Primary endpoint: clinically significant CMV DNAemia (>1000 copies/mL or requiring preemptive treatment) within 6 months post-transplant.
- Secondary outcomes: time to DNAemia, adverse effects, and interactions with cyclosporine and voriconazole.
Main Results:
- 41 patients received LTV prophylaxis, compared to 41 controls.
- Clinically significant CMV DNAemia occurred less frequently in the LTV group (9.8% vs. 17.0%, p=0.33) through Day 180.
- LTV was generally well-tolerated (87.8% no drug-related adverse effects) and associated with decreased voriconazole trough levels.
Conclusions:
- LTV prophylaxis in pediatric HCT patients was associated with reduced clinically significant CMV DNAemia.
- LTV appears to be a safe and potentially effective option for CMV prevention in this population.
Background:
Letermovir (LTV), an antiviral with exclusive activity against Cytomegalovirus (CMV), is approved for prophylaxis of CMV infection and disease in adult hematopoietic cell transplant (HCT) patients. The use of LTV in the pediatric HCT population is off-label, and has limited literature to support its use.
Procedure:
This was a single-center, retrospective, matched (1:1 LTV:non-LTV) cohort study of allogeneic HCT recipients transplanted at Children's Hospital Colorado from 2015 to 2022. The primary endpoint was clinically significant CMV DNAemia (defined as a CMV viral load >1000 copies/mL or any CMV DNAemia leading to preemptive treatment) through 6 months post transplant. Secondary outcomes included time to clinically significant CMV DNAemia, drug adverse effects, and dose adjustments of concomitant cyclosporine and voriconazole (known drug interactions).
Results:
We compared 41 patients who received LTV prophylaxis to 41 patients who received no CMV prophylaxis. There was less clinically significant CMV DNAemia through D+180 in the LTV group (9.8% vs. 17.0%, p = .33). Overall, LTV was well tolerated, and 87.8% of patients experienced no adverse effects related to the drug. There was no observed pattern in LTV effect on cyclosporine serum concentrations, but LTV was associated with decreased voriconazole trough levels.
Conclusions:
In this retrospective study, the use of LTV prophylaxis in pediatric stem cell patients was associated with reduced clinically significant CMV DNAemia through D+180.
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