Thiotepa-Based Conditioning for Haploidentical Hematopoietic Stem Cell Transplantation in Older Patients With Severe Aplastic Anemia

NCT07819812 | Not Yet Recruiting

• 1 other identifier: PUPH20260907-01
• Study Type: observational
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief Summary

This prospective, single-center observational cohort study will follow participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen as part of routine clinical care. Treatment decisions, including conditioning, prevention of graft-versus-host disease, and supportive care, are made by the clinical care team and are not assigned or changed by this study. The study will collect routine clinical and follow-up information for 1 year after transplantation to describe transplant-related mortality, survival, engraftment, graft failure, graft-versus-host disease, infections, and serious adverse events.

Conditions

Severe Aplastic Anemia; Hematopoietic Cell Transplant

Outcome Measures

Primary Outcomes (1)

• 1-year transplant-related mortality (TRM)

  The proportion of participants who die from transplant-related causes, including regimen-related toxicity, infection, and graft-versus-host disease, as defined in the study protocol.

  From Day 0 through 1 year after transplantation

Secondary Outcomes (6)

• Neutrophil Engraftment

  From Day 0 through Day 28

• Platelet Engraftment

  From Day 0 through Day 100

• Primary Graft Failure

  From Day 0 through Day 100

• Overall Survival

  From Day 0 through 1 year after transplantation

• Grade II-IV Acute graft-versus-host disease（ GVHD）

  From Day 0 through Day 100

Study Arms (1)

Thiotepa-Based Conditioning Cohort

Participants aged 50 years or older with severe aplastic anemia or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen. The conditioning regimen is selected by the treating clinical team before study enrollment. The study does not assign or alter treatment.

Drug: Thiotepa

Interventions

Thiotepa DRUG

The exposure of interest is a conditioning regimen containing thiotepa. Thiotepa and all accompanying treatment are selected by treating clinicians as part of routine care, independently of study participation. The study does not mandate drug dose, schedule, graft-versus-host disease prophylaxis, or supportive care.

Thiotepa-Based Conditioning Cohort

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will consist of consecutively identified participants aged 50 years or older with severe or very severe aplastic anemia who are scheduled to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital. The treating clinical team will independently select a thiotepa-based conditioning regimen as part of routine clinical care. The study will prospectively observe participants with available baseline, transplantation, and routine follow-up information for 1 year after transplantation. Up to 10 participants are planned for enrollment.

You may qualify if:

• Age 50 years or older at transplantation.
• Diagnosis of severe aplastic anemia or very severe aplastic anemia.
• Planned to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital.
• The treating clinical team has independently selected a thiotepa-based conditioning regimen as part of routine clinical care before study enrollment.
• Required baseline and transplantation information is available in the medical record.

You may not qualify if:

• Diagnosis or transplantation type does not meet the study eligibility criteria.
• The conditioning regimen does not include thiotepa.
• Essential baseline information is unavailable, such that study eligibility cannot be determined.
• For a participant with more than one transplantation episode, only the first episode meeting all eligibility criteria will be included in the analysis.

Sponsors & Collaborators

• Peking University People's Hospital: lead

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Thiotepa

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Phosphoramides; Organophosphorus Compounds; Organic Chemicals; Triethylenephosphoramide; Aziridines; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Central Study Contacts

Xu zheng li

CONTACT

88324002; xuzhengli0202@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: September 9, 2026
• First Posted: September 15, 2026
• Study Start: September 20, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2028
• Last Updated: September 21, 2026

Record last verified: 2026-09