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This study investigates the impact of letermovir prophylaxis on viral infections (including CMV, EBV, BKV, HHV-6/7, RSV, ADV, HSV, etc.) following allogeneic hematopoietic stem cell transplantation in pediatric patients with EBV-associated T/NK-cell lymphoproliferative diseases and refractory/relapsed EBV-related hemophagocytic lymphohistiocytosis. Additionally, we examine its effects on other transplantation complications, including engraftment failure, graft-versus-host disease (GvHD), disease relapse, thrombotic microangiopathy (TMA), overall survival (OS), post-transplant lymphoproliferative disorder (PTLD) incidence, and immune reconstitution.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Children with Letermovir for Cytomegalovirus prophylaxis after HSCT | Experimental |
| |
| Children with preemptive therapy, without Letermovir for Cytomegalovirus prophylaxis after HSCT | No Intervention |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Letermovir | Drug | Arm 1 (Letermovir Prophylaxis): Pediatric patients receive oral letermovir once daily from day 0 to day 100 post-transplant. Prophylaxis may be extended to day 200 if high-risk factors persist (steroid use, poor immune reconstitution). Dosing: 480mg (≥30kg), 240mg (15-30kg), 120mg (7.5-15kg), 80mg (6-7.5kg); halved if co-administered with cyclosporine. Arm 2 (Control): Historical control cohort (2018-2023) receiving no routine CMV prophylaxis; preemptive therapy with ganciclovir/foscarnet initiated only when plasma PCR exceeds threshold. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Clinically Significant CMV Infection (cs-CMVi) and EBV Infection (cs-EBVi) | To evaluate the incidence of clinically significant CMV and EBV infections in pediatric patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT) with or without letermovir prophylaxis. | Up to 180 days and 360 days post-transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Other Viral Infections and Transplant-Related Complications | To assess the incidence of other viral infections (e.g., BKV, HHV-6/7, RSV, ADV, HSV), graft-versus-host disease (GvHD), post-transplant lymphoproliferative disorder (PTLD), thrombotic microangiopathy (TMA), graft failure, relapse, overall survival (OS), and immune reconstitution (T/B/NK cell counts and function). | Up to 100, 180, 270, and 360 days post-transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability of Letermovir in Pediatric allo-HSCT Recipients | To monitor adverse events (AEs) and serious adverse events (SAEs) related to letermovir prophylaxis, including gastrointestinal symptoms, liver function abnormalities, cardiac events, and other treatment-emergent AEs. | From initiation of letermovir (Day 0-28 post-transplant) until 30 days after discontinuation (up to approximately 360 days post-transplant) |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jun Yang, Doctor | Contact | +86 13699293825 | yangjundabby@outlook.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Beijing Children's Hospital, Capital Medical University | Recruiting | Beijing | Beijing Municipality | 100032 | China |
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| ID | Term |
|---|---|
| C000588473 | letermovir |
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