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Autologous transplantation for chronic myelogenous leukemia with mafosfamide-treated marrow
V Rizzoli1, L Mangoni, C Almici
1Department of Hematology, Bone Marrow Transplantation Unit, University of Parma, Italy.
Insights
Autologous bone marrow transplantation using mafosfamide-treated marrow in chronic myelogenous leukemia (CML) patients showed promising Ph-negative hematopoiesis. Pre-transplant screening of marrow progenitors correlated well with engraftment success.
Area of Science:
- Hematology
- Oncology
- Stem Cell Transplantation
Background:
- Chronic myelogenous leukemia (CML) is a myeloproliferative neoplasm.
- Autologous bone marrow transplantation (ABMT) is a treatment option for CML.
- Mafosfamide is used for ex vivo marrow treatment.
Purpose of the Study:
- To evaluate the efficacy of ABMT with mafosfamide-treated marrow in Ph-positive CML patients.
- To assess the correlation between in vitro progenitor cell screening and in vivo engraftment.
- To determine the duration of Ph-negative hematopoiesis post-transplant.
Main Methods:
- Ten adult Ph-positive CML patients received ABMT with ex vivo mafosfamide-treated marrow.
- Patients were selected based on > or = 50% Ph-negative stroma-adherent progenitor cells.
- Engraftment and hematopoiesis were monitored post-transplant.
Main Results:
- Six out of nine patients achieved Ph-negative engraftment.
- Median duration of Ph-negative hematopoiesis was 6.5 months.
- A good correlation was observed between in vitro screening and in vivo results.
Conclusions:
- ABMT with mafosfamide-treated marrow can lead to durable Ph-negative hematopoiesis in CML.
- In vitro assessment of Ph-negative progenitor cells is a valuable predictor of engraftment.
- Further research is needed to optimize outcomes in accelerated phase CML.
Abstract:
Ten adult patients with Ph-positive chronic myelogenous leukemia (CML) received autologous bone marrow transplantation (ABMT) using marrow treated ex vivo with mafosfamide. At the time of ABMT, six patients were in chronic phase and four in accelerated phase. Seven of ten patients reported herein were selected on the basis of a previous laboratory assessment of the numbers of normal and leukemic stroma-adherent progenitor cells within mafosfamide-treated marrow. Only patients showing > or = 50% Ph-negative stroma-adherent progenitor cells within mafosfamide-treated marrow were considered eligible for autografting. In nine out of ten evaluable patients, the median time to achieve 500 neutrophils/microliters was 32 days (range: 25-72). A platelet count of 2 x 10(4)/microliters was achieved at a median of 40 days (range: 27-97). Six out of nine analyzable patients engrafted Ph-negative. The median duration of the Ph-negative hematopoiesis, confirmed also by Southern blot analysis, was 6.5 months (range: 4-30). A good correlation was evident between the results of the in vitro preharvest screening test and the in vivo occurrence of normal hematopoiesis post-transplant. Two patients who showed 75% and 89% Ph-negative stroma-adherent progenitors engrafted Ph-positive, whereas four out of five evaluable patients who had 100% Ph-negative stroma-adherent progenitors engrafted Ph-negative. After a median follow-up of 16 months (range: 3-31), five patients evolved into blast crisis, three are alive in hematologic and cytogenetic relapse, and one died without evolving into blast crisis.(ABSTRACT TRUNCATED AT 250 WORDS)