Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT

Launched by ANHUI PROVINCIAL HOSPITAL · Sep 9, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment approach for patients with severe aplastic anemia (SAA), a condition where the bone marrow doesn’t produce enough blood cells. The researchers want to find out if using a low dose of ATG (a medication that helps the body accept a transplant) along with a transplant from a single umbilical cord blood is safe and effective for treating SAA. The trial is currently looking for participants aged 1 to 50 years who have not responded to previous treatments and do not have a suitable donor from family or friends.

To be eligible for the study, participants must meet certain criteria, such as having a confirmed diagnosis of severe aplastic anemia and being in relatively good health without severe organ failure. They also need to agree to participate in the study with their family’s support. If you or someone you know is interested in this treatment option, it’s important to discuss it with a healthcare provider who can provide more information about the trial and what to expect if you join.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Patients diagnosed with severe aplastic anemia who do not respond to initial diagnosis or immunosuppressive therapy;
• • Patients meet diagnostic criteria for severe aplastic anemia (Camitta criteria);
• • Age from 1 to 50 years old, male or female;
• • Patients without HLA-matched sibling or unrelated donors avaliable;
• • Patients who have undergone unrelated cord blood transplantation need to have HLA high-resolution matching、TNC (before cord blood freezing)≥3.0×107/kg、CD34+(before cord blood freezing)≥1.5×107/kg、tubule recovery rate≥80%;
• • patients and their family members or guardians agreed to the study and signed informed consent;
• • No severe organ failure;
• • ECOG score≤2 and HCT-CI≤2;
• • serum ferritin (SF)\<1000ug/L;
• • women of reproductive agemust have a negative pregnancy test and agree to use effective contraception during treatment and for one year thereafter.
• Exclusion Criteria:
• • patients with inherited bone marrow failure syndromes;
• • Positive for HLA-A, B, Cw, DRB1, DQB1, DPB1 site-specific HLA antibodies for donor cord blood with solid-phase fluorescence assay;
• • patients who have previously received allogeneic hematopoietic stem cell transplantation or solid organ transplantation;
• • Uncontrolled bacterial, viral, or fungal infections;
• • HIV infection or active viral hepatitis B or C infection;
• • Pregnant or breastfeeding women;
• • patients with a history of primary malignancy within 3 years prior to transplant treatment;
• • patients receiving ATG treatment within 2 weeks before transplantation;
• • patients with drug dependent or uncontrolled mental illness or cognitive impairment;
• • Participants in other similar clinical studies within 3 months;
• • Patients allergic to ATG;
• • patients considered unsuitable for inclusion by the researchers.