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

NCT06837987 | Completed DMC

• 2 other identifiers: KM-08202301AY070001-226
• Study Type: observational
• Target: 72
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Aplastic Anaemia; Transplantation Conditioning

Keywords

modified transplantation conditioning; aplastic anemia; aGVHD prophylaxis

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• Poor graft function rate

  From the day of HSCs transfusion to 24 months after HSCT

• cGVHD incidence

  From +100 days after HSCT to the follow-up date

• adverse events

  From the first day of preconditioning to +100 days after hematopoietic stem cell Transfusion

• 2-year Overall survival rate

  24 months

• Disease-free survival

  24 months

Interventions

Modified transplantation system OTHER

Modified transplantation conditioning include: Fludarabine 30mg/m2\*6 day, Melphalan 100mg/m2\*1 day, cyclophosphamide 50mg/kg\*2 day.

Eligibility Criteria

• Age: 3 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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;

You may qualify if:

• Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
• Age 3-65 years old;
• Weight 10Kg-100Kg;
• Eastern Cooperative Oncology Group (ECOG) score ≤3;
• 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);
• No severe infection;
• Subjects voluntarily participated in this clinical trial and signed the informed consent.

You may not qualify if:

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

Sponsors & Collaborators

• Hematology department of the 920th hospital: lead

Study Sites (1)

Department of Hematology，920th Hospital of Joint Logistics Support Force

Kunming, Yunnan, 650000, China

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 16, 2025
• First Posted: February 20, 2025
• Study Start: June 1, 2020
• Primary Completion: June 30, 2023
• Study Completion: August 1, 2025
• Last Updated: September 22, 2025

Record last verified: 2025-09

Locations

CN (1)