Clinical Study of UC-MSC Combined With Immunosuppressive Agents in the Treatment of Pediatric HAAA

NCT07850362 | Completed

• 1 other identifier: IIT2020027
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to evaluate the effectiveness of umbilical cord mesenchymal stem cells combined with standard immunosuppressive therapy in children with HAAA, and to provide a new therapeutic idea for children with HAAA. Whether UC-MSC combined with immunosuppressants can improve outcomes in children with HAAA. Whether UC-MSC is safe in the treatment of children with HAAA. Participants will: 1 Age \< 18 years old, gender is not limited; 2 Diagnosis of hepatitis related aplastic anemia according to the criteria for hepatitis related aplastic anemia;

1. 1.The patient has a history of acute prehepatitis, and the course of hepatitis is generally less than 6 months. The main clinical manifestations are fever, jaundice, anemia, bleeding, generally no liver enlargement.
2. 2.Routine blood test: pancytopenia, HGB \< 100 g/L; Absolute neutrophil count \< 1.5×109/L; PLT \< 100×109/L (PLT reduction must be included in case of two-series reduction). Serum aminotransferase levels were elevated at least 200 U/L and hepatitis autoantibodies were negative.
3. 3.Bone marrow aspiration and biopsy confirmed aplastic anemia.
4. 4.Jaundice caused by other reasons and other pancytopenia diseases (such as congenital AA, clonal diseases and autoimmune diseases, etc.) are excluded.

Conditions

Hepatitis-associated Aplastic Anemia; Severe Aplastic Anemia(SAA); Umbilical Cord Blood; Mesenchymal Stem Cells; Pediatric

Outcome Measures

Primary Outcomes (1)

• Complete response rate at six months

  Complete response rate at six months

  Up to 6 months

Study Arms (1)

UC-MSC + IST group

EXPERIMENTAL

UC-MSC Combined With Immunosuppressive Agents in the Treatment of Pediatric HAAA

Other: UC-MSC

Interventions

UC-MSC OTHER

UC-MSC Combined With Immunosuppressive Agents

UC-MSC + IST group

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age \< 18 years old, gender is not limited; 2 Diagnosis of hepatitis related aplastic anemia according to the criteria for hepatitis related aplastic anemia;
• The patient has a history of acute prehepatitis, and the course of hepatitis is generally less than 6 months. The main clinical manifestations are fever, jaundice, anemia, bleeding, generally no liver enlargement.
• Routine blood test: pancytopenia, HGB \< 100 g/L; Absolute neutrophil count \< 1.5×109/L; PLT \< 100×109/L (PLT reduction must be included in case of two-series reduction). Serum aminotransferase levels were elevated at least 200 U/L and hepatitis autoantibodies were negative.
• Bone marrow aspiration and biopsy confirmed aplastic anemia.
• Jaundice caused by other reasons and other pancytopenia diseases (such as congenital AA, clonal diseases and autoimmune diseases, etc.) are excluded.
• No hematopoietic stem cell transplantation (HSCT); 4 The guardian and the subject will participate voluntarily and sign the informed consent;

You may not qualify if:

• Patients will not be enrolled in this study if they meet any of the following conditions: 2. Patients with a history of hematopoietic stem cell transplantation; HIV infection or carrier in the past or at the time of screening; Positive for syphilis; 4. The subject had developed malignant solid tumors of any organ system within 5 years prior to screening; 5. Participated in other clinical trials within 3 months;

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

Location

Related Publications (1)

• Fang L, Meikuang L, Ye G, Xiaojuan C, Wenyu Y, Min R, Lixian C, Weiqiang W, Zhibo H, Zhongchao H, Xiaofan Z. Successful Treatment of a 19-Month-Old Boy with Hepatitis Associated Aplastic Anemia by Infusion of Umbilical Cord-Derived Mesenchymal Stromal Cells: A Case Report. Cell Transplant. 2021 Jan-Dec;30:963689720977144. doi: 10.1177/0963689720977144.

  PMID: 33525921 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• lixian chang, MD

  State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 13, 2025
• First Posted: September 30, 2026
• Study Start: September 1, 2021
• Primary Completion: August 30, 2025
• Study Completion: October 31, 2025
• Last Updated: September 30, 2026

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)