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

NCT ID: NCT06837987

Last Updated: 2025-09-22

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 72 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2020-06-01
• Study Completion Date: 2025-08-01

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.

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.

Conditions

Aplastic Anaemia; Transplantation Conditioning

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: RETROSPECTIVE

Interventions

Modified transplantation system

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

Intervention Type: OTHER

Eligibility Criteria

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.

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hematology department of the 920th hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

Kunming, Yunnan, China

Countries

China

Other Identifiers

202301AY070001-226

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

KM-08

Identifier Type: -

Identifier Source: org_study_id