Study of MSC-Exo on the Therapy for Intensively Ill Children

Study of Exosomes Derived From Mesenchymal Stem Cells on the Therapy for Children With Severe Infection

At present, a number of projects related to MSCs have been approved for graft-versus-host disease, myocardial infarction, Crohn's disease and other diseases, indicating a strong therapeutic potential of MSCs. However, the efficacy of MSC-Exo for severely infected children is not fully evaluated. In our study, patients who met the inclusion criteria will be divided into trial group and control group. Clinical and demographic data, as well as treatment outcome will be collected from the electronic health record. This study will evaluate the application and therapeutic effect of MSC-Exo in severely infected children, and determine the Optimal dosage and infusion.

Study Overview

• Status: Withdrawn
• Conditions: Sepsis; Critical Illness

Detailed Description

Mesenchymal stem cells (MSCs) are pluripotent stem cells with high self-renewal ability and multidirectional differentiation potential. studies revealed that local or systemic administration of mesenchymal stem cell-derivrd exosomes (MSC-Exo) efficiently suppressed detrimental immune response in inflamed tissues and promoted survival and regeneration of injured parenchymal cells. European Union countries have written MSCs into treatment guidelines for refractory graft-versus-host disease.

In China, the technology for isolation, in vitro culture and expansion of MSCs is already mature. With further research on the therapeutic efficacy of MSC-Exo and the development of clinical trials, it is expected to become a treatment for severe illness.

A predictable infection plan is going to be widely used in clinics to provide individualized treatment for patients, improve the prognosis of patients and improve the quality of life.

The investigators intend to enroll all children who were hospitalized in pediatric intensive care unit PICU of Children's Hospital of Fudan University from January 2022 to December 2023. Children with a PICU length of day less than 48h will be excluded.

• Study Type: Observational

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China
      • Children's Hospital of Fudan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 weeks to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

All children hospitalized in PICU of Children's Hospital of Fudan University from January 2022 to December 2023

Description

Inclusion Criteria:

• children hospitalized in PICU of Children's Hospital of Fudan University

Exclusion Criteria:

• discharge within 48 hours
• patients without informed consent
• incomplete clinical and demographic data

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
control group; conventional treatment
trial group; Mesenchymal Stem Cell-Derived Exosomes

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The death rate of children	The death rate of children in 28 days after their discharged from PICU	within 28 days after they discharged from PICU
Marshall Multiple Organ Dysfunction Score	The Marshall Multiple Organ Dysfunction Score (MODS) assesses the same six organ systems using slightly different values for four grades of organ dysfunction. The total score, ranging from 0 to 24, arises from the sum of all single organ scores using the first measured value of the day. Intervals for the most abnormal value of each variable were constructed on a scale from 0 to 4 so that a value of 0 represented essentially normal function and was associated with an ICU mortality rate of < 5%, whereas a value of 4 represented marked functional derangement and an ICU mortality rate of > or = 50%.	Within 3 to 15 days after the start of treatment
the APPS score	The APPS is a 9-point score that is calculated by measuring the age, PaO2/FIO2 ratio, and plateau pressure at 24 h after the patient is diagnosed with moderate to severe ARDS and counting each one to 1-3. Since many variables are not needed for calculations, clinicians can easily predict the in-hospital mortality of mechanically ventilated patients with moderate to severe ARDS patients at bedside.	Within 3 to 15 days after the start of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
length of stay in PICU	Time from PICU admission to discharge	up to 28 days

Collaborators and Investigators

• Sponsor: Children's Hospital of Fudan University

Study record dates

Study Major Dates

• Study Start (Anticipated): September 1, 2022
• Primary Completion (Anticipated): December 31, 2023
• Study Completion (Anticipated): December 31, 2024

Study Registration Dates

• First Submitted: March 26, 2021
• First Submitted That Met QC Criteria: April 19, 2021
• First Posted (Actual): April 20, 2021

Study Record Updates

• Last Update Posted (Actual): June 15, 2022
• Last Update Submitted That Met QC Criteria: June 13, 2022
• Last Verified: June 1, 2022

More Information

Terms related to this study

• Keywords: Sepsis; Critical Illness; Mesenchymal Stem Cell Derived Exosomes
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Critical Illness
• Other Study ID Numbers: fdpicu-13

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No