Hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients with acute myeloid leukemia (AML). AML of the FAB M0 subtype is rare, often associated with a complex karyotype and a poor prognosis. Results of HSCT for this AML subtype have never been reported separately from other subtypes. We did a survey of the results of 274 HSCT in adults with M0 AML in first complete remission (CR1), performed in EBMT centres since January 1990 until 2002. One hundred fifty patients were transplanted with an HLA identical donor (HLA-id), 30 with an HLA-matched unrelated donor (MUD) and 94 received an autologous transplant (auto). The median age was 45 years (16–71), the median interval from diagnosis to HSCT was 4 months for HLA-id, 6 months for MUD and 5 months for auto HSCT. The median follow-up time (range) was 20 months (1–109), 12 (2–53) and 10 months (1–96) for HLA-id, MUD and auto-HSCT respectively. The source of stem cells was peripheral blood stem cells for 67% of cases, and bone marrow for the remaining. The majority of grafts were non-T-cell depleted. Acute GVHD (grade I–IV) occurred in 56% of HLA-id and in 64% of MUD cases. The table shows the outcomes at two years according to the type of transplant. In conclusion, outcomes after HLA identical HSCT and MUD in adult patients with AML FAB subtype M0 in CR1 are encouraging. In comparison to allogeneic transplant cases, LFS is decreased in patients receiving an autologous transplant due to a high relapse incidence, reflecting the probable role of a graft-versus-leukemia effect in this FAB subtype.

Results of HSCT in AML M0 CR1 patients

OutcomesHLA-id n=150MUD n= 30Auto n=94
LFS: leukemia free survival; OS: overall survival; RI: relapse incidence; TRM: treatment-related mortality 
2y LFS 50% 45% 33% 
2y OS 58% 50% 49% 
2y RI 25% 40% 57% 
2y TRM 24% 14% 9% 
OutcomesHLA-id n=150MUD n= 30Auto n=94
LFS: leukemia free survival; OS: overall survival; RI: relapse incidence; TRM: treatment-related mortality 
2y LFS 50% 45% 33% 
2y OS 58% 50% 49% 
2y RI 25% 40% 57% 
2y TRM 24% 14% 9% 

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