This phase 1 trial will investigate the safety and effectiveness of Thiotepa, Busulfan, and Fludarabine (TBF) conditioning regimen with post-transplant cyclophosphamide (PTCy) in HLA-matched related or unrelated donor allogeneic stem cell transplantation (alloSCT).
Allogeneic stem cell transplantation (alloSCT) offers potential cures for patients with high-risk hematologic malignancies. Establishing appropriate immune tolerance between the donor and recipient is crucial to prevent graft-versus-host disease (GVHD) and graft rejection. Over the last decade, the introduction of post-graft cyclophosphamide (PTCy)1,2 as a strategy for inducing immune tolerance has significantly transformed the landscape of alloSCT. This trial will optimize the PTCy regimen through two main strategies: 1) de-escalating the PTCy dose to reduce toxicities, and 2) incorporating thiotepa to enhance the anti-leukemia effect. We hypothesize that this optimization will improve transplant outcomes, specifically with respect to GVHD and relapse-free survival (GRFS), for recipients of HLA-matched donor alloSCT who have high-risk hematologic malignancies. Additionally, in our exploratory aim, we will investigate potential pre-transplant biomarkers that can help stratify toxicity and relapse risks, allowing us to personalize the optimal regimen intensity for individual recipients.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
48
Thiotepa is an alkylating agent used in combination with other chemotherapy agents to treat cancer.
Fludarabine is a chemotherapy drug used in the treatment of chronic lymphocytic leukemia. It acts at DNA polymerase alpha, ribonucleotide reductase and DNA primase, results in the inhibition of DNA synthesis, and destroys the cancer cells.
Busulfan is a chemotherapy drug used in preparation for a stem cell transplant.
Peripheral Blood Stem Cell (PBSC) infusion is a medical procedure used to replace diseased or damaged stem cells in patients, particularly after cancer treatments.
UMPC Hillman Cancer Center
Pittsburgh, Pennsylvania, United States
GVHD-free, relapse-free survival (GRFS)
Graft Versus Host Disease (GVHD)-free, relapse-free survival (GRFS) is defined by survival without a qualifying event including Death, Relapse of primary disease, Grade III-IV acute graft-versus-host disease (GVHD), graded via MAGIC criteria, Chronic moderate or severe GVHD requiring systemic immunosuppression, graded via NIH consensus criteria.
Time frame: At 1 year
Median time to neutrophil engraftment
Median time to neutrophil engraftment will be defined as the median number of days from transplant at which the cumulative engraftment rate reaches 50%.
Time frame: Up to 30 days
Median time to platelet engraftment
Median number of days from transplant at which the cumulative engraftment rate of platelet recovery to 20,000/mm3 and 50,000/mm3 reaches 50% respectively.
Time frame: Up to 30 days
Frequency of severe mucositis
Percentage of patients experiencing severe mucositis, defined as grade 3-4 by WHO criteria.
Time frame: Up to 30 days post-transplant
Frequency of total parental nutrition
Percentage of patients who require TPN at any time from the start of conditioning through day +30 post-transplant.
Time frame: Up to 30 days post-transplant
Frequency of severe pulmonary complications requiring ICU-level support
Percentage of patients with first occurrence of any respiratory failure, severe infectious lower respiratory tract infection, or noninfectious acute lung injury, requiring ICU-level support between Day 0 and Day +30 after stem cell infusion. ICU-level support is defined by either 1) ICU admission due to respiratory failure, 2) need for new non-invasive ventilation (BiPAP or CPAP), or 3) requirement of high-flow nasal cannula \>30L/min at FiO2\>40%.
Time frame: Up to 30 days
Cumulative incidence of infectious disease complications
Cumulative incidence of all Grade II and higher infections will be reported according to Blood and Marrow Transplant Clinical Trials Network (BMT-CTN) Infection Grading Criteria. The BMT CTN grading system provides a standardized approach for capturing and monitoring infectious complications in clinical trials.
Time frame: Up to 1-year post-transplant
Cumulative incidence of thrombotic microangiopathy
1. . Microangiopathic hemolytic anemia, defined by: a. Evidence of schistocytes on peripheral blood smear, and b. Elevated lactate dehydrogenase (LDH) above the upper limit of normal, and c. Decreased haptoglobin or other laboratory evidence of hemolysis (e.g., indirect hyperbilirubinemia), and d. Negative Coombs (direct antiglobulin) test. 2. Thrombocytopenia: a. New or progressive thrombocytopenia not explained by disease relapse, infection, drug effect, or disseminated intravascular coagulation (DIC). 3. Renal and/or neurologic dysfunction temporally associated with hemolysis, not fully explained by other causes: a. Rising serum creatinine or new/worsening hypertension, and/or b. New-onset or worsening neurologic signs/symptoms (e.g., confusion, seizures, focal deficits). 4. No alternative explanation: a. ADAMTS13 activity not suggestive of TTP (if available) and clinical evaluation not consistent with DIC, severe sepsis, or malignant hypertension as the primary cause.
Time frame: Up to 180 days post-transplant
Grade III-IV acute GVHD-free survival
Time from date of stem cell infusion to the first occurrence of grade III or IV acute Graft Versus Host Disease (GVHD), with follow-up through 100 days post-transplant or death.
Time frame: Up to 100 days post-transplant
Moderate to severe chronic GVHD-free survival
Time from date of stem cell infusion to the first occurrence of moderate-to-severe chronic Graft Versus Host Disease (GVHD) with follow up through 1-year post-transplant or death.
Time frame: At 1 year1 year post-transplant
Primary graft failure
Failure to achieve an absolute neutrophil count (ANC) \> 0.5 x 109/L by day +42 after stem cell infusion.
Time frame: Up to Day 42
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