Modified Transplantation Regimen and aGVHD Prophylaxis for Severe Aplastic Anemia in the Setting of Allogeneic HSCT. (aGVHD)

An Observation of the Safety and Efficacy of a Modified Transplantation Conditioning and aGVHD Prophylaxis for Severe Aplastic Anemia--a Retrospective, Double-center, Single-arm Clinical Study.

The investigators retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in 4 transplantation centers.

Study Overview

• Status: Completed
• Conditions: Aplastic Anaemia; Transplantation Conditioning
• Intervention / Treatment: Other: Modified transplantation system

Detailed Description

Aplastic anemia (AA) is a group of myelo-hemopoietic failure syndromes caused by a variety of etiologies. If not intervened, the average expected survival time is less than half a year.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the possible cures for this disease. The success rate of treatment for this disease can be further improved under the previous regimen system. The survival rate reported in the literature is 60%-90%. From Jun. 2020 to Dec. 2023, 72 patients in 4 transplantation center received this modified transplantation system, The investigators designed this clinical trial to retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in the 4 clinical center.

• Study Type: Observational
• Enrollment (Actual): 72

Contacts and Locations

Study Locations

• China
  • Yunnan
    • Kunming, Yunnan, China, 650000
      • Department of Hematology，920th Hospital of Joint Logistics Support Force

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;

Description

Inclusion Criteria:

1. Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
2. Age 3-65 years old;
3. Weight 10Kg-100Kg;
4. Eastern Cooperative Oncology Group (ECOG) score ≤3;
5. No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
6. No severe infection;
7. Subjects voluntarily participated in this clinical trial and signed the informed consent.

Exclusion Criteria:

1. Patients with other hematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;
2. Patients with an expected survival of less than 1 month;
3. Patients with previous autologous or allogeneic hematopoietic stem cell transplantation;
4. pregnant patients;
5. Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
6. Other conditions that the investigator determines to be inappropriate for enrollment.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Primary graft falure rate	no apperance or complete loss of donor-derived neutrophils by +28 days	From the day of HSCs transfusion to +100 day after HSCT.
aGVHD incidence	proportion of patients who developed aGVHD within 100 days after HSCT	From the day of HSCT transfusion to 100 days after HSCT

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
2-year Overall survival rate	proportioin of patients who still be alive within 24 months after HSCT	24 months
Disease-free survival	proportion of patients still be alive without GVHD or other diseases.	24 months
Poor graft function rate	the presence of at least two cytopenic counts beyond +28 days with a transfusion requirement associated with hypoplastic-aplastic bone marrow	From the day of HSCs transfusion to 24 months after HSCT
cGVHD incidence	the proportion of patients who developed chronic GVHD within the observation duration	From +100 days after HSCT to the follow-up date
adverse events	the incidence of adverse events during the transplantation preconditioning periods.	From the first day of preconditioning to +100 days after hematopoietic stem cell Transfusion

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Fungal infections rate	Proportion of patients who developed fungal infection after HSCT	2 years
EB virus reactivation rate	Proportion of patient who experience EB virus reactivation	within 100 days after HSCT
cytomegalovirus reactivatioin	proportion of patient who experience cytomegalovirus reactivatioin	within 100 days after HSCT

Collaborators and Investigators

• Sponsor: Hematology department of the 920th hospital

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2020
• Primary Completion (Actual): June 30, 2023
• Study Completion (Actual): August 1, 2025

Study Registration Dates

• First Submitted: February 16, 2025
• First Submitted That Met QC Criteria: February 16, 2025
• First Posted (Actual): February 20, 2025

Study Record Updates

• Last Update Posted (Actual): September 22, 2025
• Last Update Submitted That Met QC Criteria: September 16, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: aplastic anemia; aGVHD prophylaxis; modified transplantation conditioning
• Additional Relevant MeSH Terms: Bone Marrow Failure Disorders; Hematologic Diseases; Bone Marrow Diseases; Anemia; Hemic and Lymphatic Diseases; Anemia, Aplastic
• Other Study ID Numbers: KM-08; 202301AY070001-226 (Other Grant/Funding Number: the Joint and Special Project of Kunming medical university)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

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