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Bone marrow transplantation for myelodysplastic syndromes
R Bélanger1, M Gyger, C Perreault
1Department of Haematology, Maisonneuve-Rosemont Hospital, Montreal, Quebec, Canada.
Abstract:
Eight patients with myelodysplastic syndromes (MDS) were treated with bone marrow transplantation (BMT). Median age was 34.5 years and ranged between 3 and 45. FAB diagnosis was refractory anaemia (RA) in three, RA with excess of blasts (RAEB) in four and RAEB in transformation (RAEB-t) in one case. Four patients were prepared with cyclophosphamide and total body irradiation whilst the other four received busulphan and cyclophosphamide. Engraftment was documented in seven of eight patients. Two patients died from complications related to the procedure. One had early veno-occlusive disease of the liver whilst the other died 46 months after BMT from pulmonary fibrosis. One patient died from recurrent disease 11 months after BMT. Five patients are alive and in complete remission 9-35 months post-transplantation. Four of these patients have a Karnofsky score greater than or equal to 90%. These results suggest that BMT can induce prolonged disease-free survival in patients under 50 years of age. If a compatible donor is available, marrow transplantation should be seriously considered in the treatment of MDS.
Insights
Bone marrow transplantation (BMT) offers prolonged disease-free survival for myelodysplastic syndromes (MDS) patients under 50. Consider BMT when a compatible donor is available for MDS treatment.
Area of Science:
- Hematology
- Oncology
- Transplantation Medicine
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- Treatment options for MDS can be limited, especially for younger patients.
Purpose of the Study:
- To evaluate the efficacy and outcomes of bone marrow transplantation (BMT) in patients with myelodysplastic syndromes (MDS).
Main Methods:
- Eight patients with MDS (ages 3-45) underwent BMT.
- Conditioning regimens included cyclophosphamide/total body irradiation or busulphan/cyclophosphamide.
- Engraftment, survival, and functional status (Karnofsky score) were assessed.
Main Results:
- Engraftment occurred in seven of eight patients.
- Two treatment-related deaths occurred (veno-occlusive disease, pulmonary fibrosis).
- One patient died from disease recurrence; five patients achieved long-term remission (9-35 months), with four having Karnofsky scores >= 90%.
Conclusions:
- BMT can achieve prolonged disease-free survival in MDS patients under 50.
- Marrow transplantation is a viable treatment option for MDS if a compatible donor is available.