Haploidentical Transplantation in Severe Aplastic Anemia

Wu Xiaoxiong

Status: Unknown

Conditions: Severe Aplastic Anemia

Treatments: Other: HFD-HSCT; Other: MSD-HSCT

Study type: Interventional

Funder types: Other

Identifiers

NCT03246178

2016QX-KS008

Details and patient eligibility

About

This is a prospective case-control study on SAA patients treated with HSCT, order to further discuss and assess the safety, feasibility and effectiveness of HFD-HSCT which performed with reduced-intensity fludarabine-based conditioning regimen.Our findings would indicate that SAA patients who lack MSD benefited most if HFD-HSCT was performed with reduced-intensity fludarabine-based conditioning regimen, and our improved outcomes with HFD-HSCT may lead to a salvaged therapy and an expanded direct role for SAA in the future.

Enrollment: 60 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

(i) Diagnosis of SAA, very SAA or SAA and paroxysmal nocturnal hemoglobinuria (PNH) according to the International Aplastic Anemia Study Group; (ii) SAA patients no response to previous IST; (iii) adequate performance status [Eastern Cooperative Oncology Group (ECOG) score 0-2].

Exclusion criteria

(i) Congenital forms of aplastic anemia; (ii)Patients with any severe pulmonary, cardiac, liver, or renal diseases or active infection.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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60 participants in 2 patient groups

MSD-HSCT

Other group

Description:

This group received treatment of matched sibling donor - hematopoietic stem cell transplantation (MSD-HSCT).

Treatment:

Other: MSD-HSCT

HFD-HSCT

Experimental group

Description:

This group received treatment of haploid family donor - hematopoietic stem cell transplantation (HFD-HSCT).

Treatment:

Other: HFD-HSCT