The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA

Phase 4

Recruiting

• Conditions: Severe Aplastic Anemia
• Interventions: Drug: Flu/Cy/ATG or Cy/ATG; Drug: Busulfan

• Registration Number: NCT06069180
• Lead Sponsor: Peking University People's Hospital

Brief Summary

Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. The occurrence of mixed chimerism after transplantation is associated with secondary graft failure and poor failure-free survival. Previo...

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2023-10-01
• Study First Submitted QC: 2023-10-01
• Study First Posted: 2023-10-05
• Status Verified: 2023-11
• Study Start: 2023-11-15
• Last Update Submitted: 2023-11-15
• Last Update Posted: 2023-11-18
• Primary Completion: 2024-12-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 160

Inclusion Criteria

1. Diagnosed as SAA/vSAA

2. Indication for hematopoietic stem cell transplantation

3. Available HLA matched sibling or unrelated donor

4. No active infection

5. No serious organ damage: liver and kidney function (ALT and AST < 2.5 times normal value, normal renal function, no cardiac insufficiency)

6. Signed informed consent

7. High risk factors of mixed chimerism, at least one of the following

   1. Age < 18 years old
   2. Ferritin level ≥2500ng/ml before transplantation

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Exclusion Criteria

1. Age > 50 years old
2. ECOG≥3
3. Active infections that were difficult to control
4. Severe liver and kidney dysfunction
5. Mental illness
6. Not signing the informed consent
7. pregnant or lactating women
8. Any condition considered by the investigators to be unsuitable for enrollment

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Control group	Flu/Cy/ATG or Cy/ATG	The conditioning regimens were Flu/Cy/ATG or Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.
Busulfan included group	Busulfan	The conditioning regimens were Bu/Flu/Cy/ATG or Bu/Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.

Primary Outcome Measures

Name	Time	Method
Failure free survival	1 year post HSCT	Failure free survival was defined as survival with a response to therapy.

Secondary Outcome Measures

Name	Time	Method
The incidence of mixed chimerism	1 year post HSCT	The mixed chimerism was defined as the presence of 5%-95% donor haematopoietic cells.
Regimen related toxicity	100 days post HSCT	The regimen related toxicity (RTT) was measured according to the Seattle Toxicity Criteria (Bearman et al, 1988).
Myeloid and platelet engraftment	100 days post HSCT	Myeloid and platelet engraftment were defined as international criteria.
The incidence of graft versus host disease	100 days post HSCT for aGvHD and 1 year post HSCT for cGvHD	The severity of acute and chronic GVHD was evaluated according to standard criteria.
The incidence of CMV and EBV reactivation	100 days post HSCT	The incidence of CMV and EBV reactivation was defined as CMV and EBV viremia.
The incidence of Transplantation related mortality	1 year post HSCT	Transplantation related mortality was defined as death without disease progression.
The probability of Overall survival	1 year post HSCT	Overall survival was defined as the time from transplantation to death from any cause or to the last follow-up

Trial Locations

Locations (1)

Deparment of Hematology, Peking University People's Hospital; 🇨🇳 Beijing, Beijing, China