NCT07319793

Pre-DLI or Pro-DLI in Relapsed/Refractory Myeloid Neoplasms After HSCT

Clinical Study on Optimized Treatment for Allogeneic Hematopoietic Stem Cell Transplantation in Relapsed/Refractory Myeloid Neoplasms

• Status: Not Yet Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: Peking University People's Hospital · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Donor lymphocyte infusion (DLI) based on minimal residual disease (MRD) has been widely adopted worldwide to enhance the graft-versus-leukemia effect following allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, there is a lack of studies comparing the efficacy and safety of prophylactic versus preemptive DLI in patients with refractory/relapsed (R/R) acute myeloid leukemia (AML), and its effectiveness in other myeloid neoplasms (MN, such as myelodysplastic syndromes with excess blasts, MDS-IB) remains unknown, particularly with a scarcity of data from randomized controlled trials. This multicenter, randomized controlled study aims to prospectively compare the efficacy and safety of prophylactic versus preemptive DLI in patients with R/R myeloid neoplasms undergoing allo-HSCT. The study will enroll patients with MN (including AML and MDS-IB, excluding Ph+ cases) undergoing allo-HSCT, who are in R/R status at the time of transplant and achieve MRD-negative remission at 1 month post-transplant. Eligible patients must have no evidence of graft-versus-host disease (GVHD) or controlled GVHD, no severe infections, and no organ failure within 30-60 days post-transplant. One hundred patients will be enrolled in both the experimental and control groups. The primary endpoint is the relapse rate at 1 year post-randomization. Secondary endpoints include: 1-year leukemia-free survival and overall survival, and the incidence of bone marrow suppression, pancytopenia, GVHD, and infections following DLI. This study aims to explore strategies to reduce relapse rates and improve survival in patients with R/R MN following allo-HSCT.

Conditions

• Acute Myeloid Leukemia
• Myelodysplastic Syndromes (MDS)

Interventions

Type	Name	Description
OTHER	Prophylactic DLI	DLI is performed between day +45 and day +60 post-transplantation. Peripheral blood hematopoietic stem cells are infused, with short-course immunosuppressive therapy administered after DLI. The infused mononuclear cell and CD3⁺ cell doses are 1.0×10⁸/kg and 3.0×10⁷/kg, respectively. Following DLI, graft-versus-host disease (GVHD) prophylaxis is administered using cyclosporine A or methotrexate. For patients undergoing fully matched transplants, prophylaxis lasts 4-6 weeks; for those receiving haploidentical transplants, it lasts 6-8 weeks.
OTHER	Preemptive DLI	DLI is initiated if the patient meets the criteria for MRD positivity. The regimen includes chemotherapy followed by DLI. Chemotherapy is administered 48-72 hours prior to peripheral blood hematopoietic stem cell infusion, using one of the following regimens: HAA (homoharringtonine + aclarubicin + cytarabine), AA (aclarubicin + cytarabine), HA (homoharringtonine + cytarabine), or VA (venetoclax + azacitidine). The principles of cell infusion and subsequent short-course immunosuppressive therapy are the same as in the prophylactic DLI group.

Timeline

• Start date: 2026-01-01
• Primary completion: 2027-01-01
• Completion: 2028-06-30
• First posted: 2026-01-06
• Last updated: 2026-01-06