Optimising pre and post transplant management and Q&A
. Platzbecker U. 05/21/11; 7613
Disclosure(s): I have received honoraria from Celgene.
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Risks of allogeneic HSCT
Importance of patient selection
Ongoing efforts to improve outcome
Importance of patient selection
Ongoing efforts to improve outcome
Because myelodysplastic syndromes (MDS) are a disease of the elderly population, there are potential risks from intensive treatment approaches. Whereas in low-risk MDS supportive care only can be considered in many patients, in high-risk MDS the considerable rate of AML transformation as well as mortality from infectious complications make therapeutic approaches desirable, which can alter the disease history. In fact, over the past few years several therapeutic modalities for MDS have been developed. Treatment with DNA-methyltransferase inhibitors (MTI) such as 5-azacytidine, has changed the natural course of the disease and prolonged survival compared to supportive care only. However, with the rare exception of patients who achieve long lasting remissions with chemotherapy, allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only modality with proven curative potential. Although improvements in donor selection, post-grafting immunosuppression and supportive therapy have been achieved, allogeneic HSCT after standard conditioning remains restricted to a small minority of younger patients. The development of reduced intensity conditioning regimens may allow patients with higher age or comorbidities to undergo this procedure. Published reports suggest that allogeneic HSCT can be successful in elderly patients and thus possibly provide a survival advantage compared to non-transplant approaches. However, a reduction in the intensity of the preparative regimen is associated with a higher risk of relapse after HSCT. Identification of clinical markers, which might help in selecting elderly patients who are likely to benefit from HSCT and in determining the optimum time point for HSCT would be helpful. Current approaches focus on the relevance of pre-transplant therapy including iron chelation with additional efforts to integrate preemptive or maintenance strategies post transplantation in order to improve outcome of patients undergoing allogeneic HSCT.
Because myelodysplastic syndromes (MDS) are a disease of the elderly population, there are potential risks from intensive treatment approaches. Whereas in low-risk MDS supportive care only can be considered in many patients, in high-risk MDS the considerable rate of AML transformation as well as mortality from infectious complications make therapeutic approaches desirable, which can alter the disease history. In fact, over the past few years several therapeutic modalities for MDS have been developed. Treatment with DNA-methyltransferase inhibitors (MTI) such as 5-azacytidine, has changed the natural course of the disease and prolonged survival compared to supportive care only. However, with the rare exception of patients who achieve long lasting remissions with chemotherapy, allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only modality with proven curative potential. Although improvements in donor selection, post-grafting immunosuppression and supportive therapy have been achieved, allogeneic HSCT after standard conditioning remains restricted to a small minority of younger patients. The development of reduced intensity conditioning regimens may allow patients with higher age or comorbidities to undergo this procedure. Published reports suggest that allogeneic HSCT can be successful in elderly patients and thus possibly provide a survival advantage compared to non-transplant approaches. However, a reduction in the intensity of the preparative regimen is associated with a higher risk of relapse after HSCT. Identification of clinical markers, which might help in selecting elderly patients who are likely to benefit from HSCT and in determining the optimum time point for HSCT would be helpful. Current approaches focus on the relevance of pre-transplant therapy including iron chelation with additional efforts to integrate preemptive or maintenance strategies post transplantation in order to improve outcome of patients undergoing allogeneic HSCT.
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