Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI

Phase 2Not Yet RecruitingNCT07796620

Peking University People's Hospital40 enrolled

Overview

Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Ruxolitinib and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.

Study Type

INTERVENTIONAL

Allocation

Purpose

PREVENTION

Masking

NONE

Enrollment

Conditions

Acute Leukemia Myelodysplastic Syndromes Severe Aplastic Anemia (SAA)

Interventions

Eligibility

Sex: ALLMin age: 14 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Primary disease: hematological malignancies (including acute leukemia, myelodysplastic syndromes), nonmalignant disorders (including severe aplastic anaemia) * Renal injury or inability to tolerate the side effects of CNI: such as CNI renal toxicity (creatinine levels above the upper limit of normal), uncontrolled hypertension, and neurotoxicity rrom the time of hematopoietic stem cell infusion until +90 days after transplantation * Receiving haplo-HSCT for the first time Exclusion Criteria: * Allergy or intolerance to study drugs * Active infection * Active GVHD * Transplantation-associated thrombotic microangiopathy * Key organ dysfunction: liver injury (total bilirubin more than 2 upper limit of normal) or heart injury (symptomatic heart failure or ejection fraction\<50%) * Eastern Cooperative Oncology Group (ECOG) score \>2 * Expected survival time \<30 days * Patients could not cooperate

Outcomes

Primary Outcomes

Incidence of Grade 2-4 aGVHD within 100 days post transplantation

Time frame: Participants will be followed for an expected average of 100 days post transplantation