Burden of Cytomegalovirus Reactivation in Pediatric Patients After Allogeneic Hematopoietic Stem Cell Transplantation (CMV PED)

CMV PED Study - A Retrospective Observational Study To Understand The Clinical And Economic Burden Associated With Cytomegalovirus Reactivation and Related Health Outcomes In Pediatric Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation In Italy

This observational retrospective analysis will provide useful information for clinicians and payers, and local guidelines committee members, to improve the understanding of Cytomegalovirus clinical and economic burden and clinical management of pediatric patients undergoing allogeneic Hematopoietic Stem Cells Transplantation in Italy.

Study Overview

• Status: Recruiting
• Conditions: Cytomegalovirus Infection; Hematopoietic Stem Cells Transplantation

Detailed Description

This is an observational retrospective analysis from the main pediatric centers in Italy (approximately 5 sites). The selected sites will be the most representative of Italy because they perform about 2/3 of all allogeneic HSCT per year.

Index date: date of allogeneic HSCT; retrospective data will be captured in consecutive patients undergoing allogeneic HSCT from January 2018 to June 2020 with maximum 12 months of follow-up for each patient. The data of the patients undergoing allogeneic HSCT after June 2020 will not be captured in order to avoid any possible bias due to off-label access to letermovir.

Medical Records (MR) will be used to describe the risk factors, patient characteristics, treatment patterns, and healthcare resource utilization of subjects who had a CMV infection.

Electronic or paper hospital charts (inpatient), clinical charts (inpatient and outpatient), and outpatient records will all be considered as MR for study purposes.

This is a secondary data collection study from electronic or paper medical chart review, no data from registry will be collected.

Patients (or their legally acceptable representatives) must have signed and dated the Informed Consent & Privacy Form (ICF), if applicable.

• Study Type: Observational
• Enrollment (Estimated): 230

Contacts and Locations

• Study Contact: Name: Antonia Maffucci; Phone Number: +39 03626331; Email: info.studiclinici@opisresearch.com

Study Locations

• Italy
  • Genova, Italy
    • Recruiting
    • IRCCS Istituto Giannina Gaslini, Trapianto di Cellule Staminali Emopoietiche, Dipartimento di Onco-Ematologia Pediatrica
  • Monza, Italy, 20900
    • Recruiting
    • Ospedale San Gerardo, Clinica Pediatrica
  • Padova, Italy, 35128
    • Not yet recruiting
    • Azienda Ospedale-Università di Padova, Clinica di Oncoematologia Pediatrica
  • Roma, Italy, 00165
    • Recruiting
    • IRCCS Ospedale Pediatrico Bambino Gesù, Dipartimento di Pediatria Ematologia e Oncologia
  • Torino, Italy, 10126
    • Recruiting
    • Ospedale Infantile Regina Margherita, Dipartimento Patologia e Cura del Bambino

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

About 230 patients from birth to less than 18 years of age allogeneic HSCT recipients at risk of developing CMV infection and/or disease who underwent allogeneic HSCT between January 2018 and June 2020 in the participating hospitals will be enrolled in the study. The selected hospitals will be the main ones in Italy based on the number of transplants performed every year and also considering the geographical distribution.

Description

Inclusion Criteria:

• Patients from birth to less than 18 years of age (at the moment of the allogeneic HSCT);
• Patients who received allogeneic HSCT between January 2018 and June 2020;
• Patients (or their legally acceptable representatives) must have signed and dated the Informed Consent & Privacy Form (ICF), if applicable.

Exclusion Criteria:

• Letermovir use at any time

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Summary of demographic and baseline characteristics	Description through frequencies and percentages (for categorical variables) and mean and median values, SD, quartiles, interquartile ranges and extreme values (for continuous variables).	at baseline (day of transplant)
CMV Seroprevalence in the under 18 population undergoing allogeneic HSCT	Proportion of patients CMV R+ at transplantation. CMV-seroprevalence and serostatus will be described according to the recipient or donor positivity. Comparisons will be conducted using the Chi-square or Fisher's exact test (for categorical variables), or the t-test or Wilcoxon rank sum test (for continuous variables).	at baseline (day of transplant)
CMV serostatus	R+/D-, R+/D+ and R-/D+ combination frequencies. CMV-seroprevalence and serostatus will be described according to the recipient or donor positivity. Comparisons will be conducted using the Chi-square or Fisher's exact test (for categorical variables), or the t-test or Wilcoxon rank sum test (for continuous variables).	at baseline (day of transplant)
Donor type	Allogeneic HSCT characteristics	at baseline (day of transplant)
Stem cell source	Allogeneic Hematopoietic Stem Cells Transplantation characteristics	at baseline (day of transplant)
Conditioning intensity	Allogeneic Hematopoietic Stem Cells Transplantation characteristics	at baseline (day of transplant)
Underlying condition	Allogeneic Hematopoietic Stem Cells Transplantation characteristics	at baseline (day of transplant)
Usage of immunosuppression	Allogeneic Hematopoietic Stem Cells Transplantation characteristics	at baseline (day of transplant)
Current standard of care in CMV management in terms of PET approach	It will be described quantitatively by tabulating frequencies and percentages.	during the first-year post-transplant
Current standard of care in CMV management in terms of GCV prophylaxis	It will be described quantitatively by tabulating frequencies and percentages.	during the first-year post-transplant
Current standard of care in CMV management in terms of ACV prophylaxis	It will be described quantitatively by tabulating frequencies and percentages.	during the first-year post-transplant
Current standard of care in CMV management in terms of other	It will be described quantitatively by tabulating frequencies and percentages.	during the first-year post-transplant

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of clinically significant CMV infection	Incidence rates will be computed by dividing the number of events by the total number of person-years and then multiplying per 100. A Kaplan-Meier curve will describe the timing of first CS-CMVi and mortality.	at day +100, day +200 and during the first year post allogeneic HCT
Median time to first CS-CMVi	A Kaplan-Meier curve will describe the timing of first CS-CMVi and mortality.	during the first year after transplantation
Rate of viral infections other than CMV	Incidence rates will be computed by dividing the number of events by the total number of person-years and then multiplying per 100. A Kaplan-Meier curve will describe the timing of first CS-CMVi and mortality.	at day +100, day +200 and during the first year post allogeneic HCT
Rate of bacterial infections	Incidence rates will be computed by dividing the number of events by the total number of person-years and then multiplying per 100. A Kaplan-Meier curve will describe the timing of first CS-CMVi and mortality.	at day +100, day +200 and during the first year post allogeneic HCT
Rate of fungal infections	Incidence rates will be computed by dividing the number of events by the total number of person-years and then multiplying per 100. A Kaplan-Meier curve will describe the timing of first CS-CMVi and mortality.	at day +100, day +200 and during the first year post allogeneic HCT
Allogeneic HSCT risk factors	Underlying condition, donor type, stem cell source, conditioning intensity, immunosuppressive therapies. The relation between complications and risk factors will be analyzed through Cox regression models.	at baseline (day of transplant)
CMV-related complications	Proportion of patients with at least one CMV disease, "overall" and by type of disease, and number of diseases per patient. The relation between complications and risk factors will be analyzed through Cox regression models.	during the first-year post-transplant
CMV-related complications	Rate of acute and chronic Graft vs Host Disease Incidence rates will be computed by dividing the number of events by the total number of person-years and then multiplying per 100. A Kaplan-Meier curve will describe the timing of first CS-CMVi and mortality.	at day +100, day +200 and during the first year post allogeneic HCT
CMV-related complications	Rate of opportunistic infection declared by the investigator "CMV related-complication"	during the first-year post-transplant
Number of hospitalizations and length of stay (LOS) after allogeneic HSCT	Utilization of several healthcare resources will be described to quantify the economic burden. Data will include frequency of re-hospitalization, exams, diagnostic tests, visits, drugs use and the duration of re-hospitalization. Statistical tests (Chi-square, Fisher's exact, Student's T or Wilcoxon rank-sum) will be applied based on the variable type.	at day +100, day +200 and during the first year post allogeneic HCT
All-cause mortality rate	Kaplan-Meier curves will describe the timing of events.	at day +100, day +200 and during the first year post allogeneic HCT

Collaborators and Investigators

• Sponsor: MSD Italia S.r.l.

Study record dates

Study Major Dates

• Study Start (Actual): May 9, 2025
• Primary Completion (Estimated): April 30, 2026
• Study Completion (Estimated): July 15, 2026

Study Registration Dates

• First Submitted: March 31, 2025
• First Submitted That Met QC Criteria: March 31, 2025
• First Posted (Actual): April 8, 2025

Study Record Updates

• Last Update Posted (Actual): April 6, 2026
• Last Update Submitted That Met QC Criteria: March 31, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Infections; Virus Diseases; DNA Virus Infections; Herpesviridae Infections; Cytomegalovirus Infections
• Other Study ID Numbers: NIS103043

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No