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Pharmacological-mediated purging with mafosfamide in acute and chronic myeloid leukemias. The Italian Study Group
Abstract:
The success of autologous bone marrow transplantation (ABMT) in acute leukemia (AL) in complete remission (CR) is limited by the high relapse rate. It is generally accepted that minimal residual disease (MRD) plays a major role in determining the relapse of disease. In our study we investigated in adult acute leukemia and in chronic myelogenous leukemia (CML), the efficacy of ex vivo marrow purging with mafosfamide (an in vitro derivative of cyclophosphamide). We also describe an improved purging approach ("programmed method") based on the evaluation of sensitivity to the drug measured in each individual patient prior to ABMT. The analysis of clinical data in terms of disease-free survival (DFS) shows that the "programmed method" gives significantly better results than those obtained using the standard dose of mafosfamide (80% DFS in 18 AL patients vs. 44% in 33 ANLL patients and 33% in 56 ALL patients). The evaluation of the CR to purging interval in ALL and ANLL shows that a period of greater than 6 months is necessary to obtain longer DFS. Considering pre-transplant regimens, busulfan-cyclophosphamide is more effective in ANLL, and cyclophosphamide-fractionated total body irradiation is the best treatment for ALL. The studies using mafosfamide marrow purging in CML demonstrate that the drug is able to achieve a decrease of the Ph1+ marker. Three patients who showed conversion of this cytogenetic marker have been autografted with interesting clinical results.
Insights
A programmed method for ex vivo marrow purging with mafosfamide significantly improves disease-free survival in adult acute leukemia patients undergoing autologous bone marrow transplantation (ABMT), reducing relapse rates. This approach optimizes drug sensitivity for better patient outcomes.
Area of Science:
- Hematology
- Oncology
- Transplantation Immunology
Background:
- High relapse rates limit autologous bone marrow transplantation (ABMT) success in acute leukemia (AL).
- Minimal residual disease (MRD) is a key factor in post-transplant relapse.
- Ex vivo marrow purging aims to eliminate residual leukemia cells before ABMT.
Purpose of the Study:
- To evaluate the efficacy of ex vivo marrow purging with mafosfamide in adult acute leukemia and chronic myelogenous leukemia (CML).
- To introduce and assess an improved "programmed method" of mafosfamide purging based on individual patient drug sensitivity.
- To compare the "programmed method" with standard mafosfamide dosing for improving disease-free survival (DFS).
Main Methods:
- Adult patients with acute leukemia (AL) and chronic myelogenous leukemia (CML) underwent ex vivo marrow purging with mafosfamide.
- A "programmed method" was developed, involving pre-ABMT assessment of individual mafosfamide sensitivity.
- Clinical data, including DFS, were analyzed to compare treatment outcomes.
- Pre-transplant regimens and the optimal interval between complete remission (CR) and purging were evaluated.
Main Results:
- The "programmed method" yielded significantly better DFS (80%) compared to standard mafosfamide dosing (44% in ANLL, 33% in ALL).
- A CR-to-purging interval exceeding 6 months correlated with longer DFS in ALL and ANLL.
- Busulfan-cyclophosphamide was more effective for ANLL, while cyclophosphamide-fractionated total body irradiation was optimal for ALL.
- Mafosfamide purging in CML reduced the Ph1+ marker, with three patients showing cytogenetic conversion and positive clinical outcomes post-autograft.
Conclusions:
- The "programmed method" of mafosfamide marrow purging represents a significant advancement in reducing relapse after ABMT for acute leukemia.
- Optimizing the purging strategy and pre-transplant regimens is crucial for improving outcomes in leukemia patients.
- Mafosfamide purging shows promise in managing CML by reducing the Philadelphia chromosome marker.