A Novel Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplant in Patients Aged ≥ 40 Years Old With Severe Aplastic Anemia

A Novel Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplant in Patients Aged ≥ 40 Years Old With Severe Aplastic Anemia: a Multicenter, Single-arm, Observational Clinical Trial

The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT.

Study Overview

• Status: Not yet recruiting
• Conditions: Busulfan 0.8 mg/kg/q6h (Days -8 to -7) Fludarabine 30mg/m2/Day (Days -6 to -2) Cyclophosphamide 25 mg/kg/Day and r-ATG 2.5 mg/kg/Day (Days -5 to -2)
• Intervention / Treatment: Other: BFC conditiong regimen

• Study Type: Observational
• Enrollment (Estimated): 64

Contacts and Locations

• Study Contact: Name: Tingting Han; Phone Number: 8601088326666; Email: htt1984.love@163.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100044
      • Peking Universtiy Peoples' Hospital
      • Contact: Tingting Han, Doctor; Phone Number: 86-01088424953; Email: htt1984.love@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients aged ≥40 years old and <60 years old, diagnosed with severe aplastic anemia by NCCN guidelines and will receive haploidentical hematopoietic stem cell transplantation in the centers will be recruited.

Description

Inclusion Criteria:

Severe aplastic anemia; Age 40-60 years old; Weight 45Kg-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.

Exclusion Criteria:

With other hematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation; Expected survival of less than 1 month; 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.

Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection A history of symptomatic herpes zoster infection within 12 weeks prior to screening Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB Exposure to a live vaccine within 12 weeks prior to enrollment or expected to receive a live vaccine during the study Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data

Any of the following specific abnormalities on screening laboratory tests:

1. ALT or AST >2 x ULN, or total bilirubin ≥1.5 x ULN
2. hemoglobin <9 g/dL, or total white blood cell (WBC) count <2,500/µL, or neutropenia (absolute neutrophil count <1,200/µL), or lymphopenia (lymphocyte count <750/µL)
3. eGFR <50 mL/min/1.73 m2

Study Plan

How is the study designed?

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
The First Affiliated Hospital of Soochow University	Other: BFC conditiong regimen; Conditioning regimens include 0.8 mg/kg/6h busulfan (days -8 to -7), 30mg/m2/day fludarabine (days -6 to -2), 25 mg/kg/day cyclophosphamide (days -5 to -2) and 2.5 mg/kg/day r-ATG (days -5 to -2).
Peking unviversity Peoples' Hospital	Other: BFC conditiong regimen; Conditioning regimens include 0.8 mg/kg/6h busulfan (days -8 to -7), 30mg/m2/day fludarabine (days -6 to -2), 25 mg/kg/day cyclophosphamide (days -5 to -2) and 2.5 mg/kg/day r-ATG (days -5 to -2).
Guangzhou First People's Hospital,	Other: BFC conditiong regimen; Conditioning regimens include 0.8 mg/kg/6h busulfan (days -8 to -7), 30mg/m2/day fludarabine (days -6 to -2), 25 mg/kg/day cyclophosphamide (days -5 to -2) and 2.5 mg/kg/day r-ATG (days -5 to -2).
The First Affiliated Hospital of USTC	Other: BFC conditiong regimen; Conditioning regimens include 0.8 mg/kg/6h busulfan (days -8 to -7), 30mg/m2/day fludarabine (days -6 to -2), 25 mg/kg/day cyclophosphamide (days -5 to -2) and 2.5 mg/kg/day r-ATG (days -5 to -2).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Faliure free survival (FFS)	survival with a response to therapy after HSCT. Death, GF and relapse were considered as treatment failure.	From enrollment to 2 years post-HSCT

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The incidence of mixed chimerism	The mixed chimerism was defined as the presence of 5%-95% donor haematopoietic cells.	1 year post HSCT
Regimen related toxicity	The regimen related toxicity (RTT) was measured according to the Seattle Toxicity Criteria (Bearman et al, 1988).	100 days post HSCT
Myeloid and platelet engraftment	Myeloid and platelet engraftment were defined as international criteria.	100 days post HSCT
The probability of Overall survival	Overall survival was defined as the time from transplantation to death from any cause or to the last follow-up	From enrollment to 2 years post-HSCT
The incidence of graft versus host disease	The severity of acute and chronic GVHD was evaluated according to standard criteria.	100 days post HSCT for aGvHD and 2 years post HSCT for cGvHD
The incidence of CMV and EBV reactivation	The incidence of CMV and EBV reactivation was defined as CMV and EBV viremia.	180 days post HSCT
The incidence of Transplantation related mortality	Transplantation related mortality was defined as death without disease progression.	2 years post HSCT

Collaborators and Investigators

• Sponsor: Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 30, 2028

Study Registration Dates

• First Submitted: May 8, 2026
• First Submitted That Met QC Criteria: May 8, 2026
• First Posted (Actual): May 14, 2026

Study Record Updates

• Last Update Posted (Actual): May 14, 2026
• Last Update Submitted That Met QC Criteria: May 8, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Other Study ID Numbers: PUPH20260508

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No