Decision to treat

Whether to treat a patient with HSC transplantation is a complex decision. For numerous diseases, HSC transplantation represents the only therapy with a potential 100% curative effect. However, the relatively high risk of transplant related mortality/morbidity is a contraindication and while evaluating this risk, the physician will also consider more classical therapies. HSC transplantation is indicated only when the expected result of an alternative therapy would be significantly less favorable than the potential result of transplantation.

This is not a simple task because the risk varies per patient according to numerous parameters such as the patient's age (increased risk of severe GVHD and other complications with increasing age), the disease stage and the availability of a compatible (HLA matched) donor. These parameters should be estimated before transplant.

A patient with CML in chronic phase represents an example of how complex this decision may be. It is beyond doubt that an allogeneic HSCT is the only treatment that can cure CML completely. When an HLA-identical donor is available, the patient has an excellent chance of a long disease-free survival and HSCT is the therapy of choice. Until a few years ago, HSCT was also the therapy of choice when the chance of a long disease free survival was less because the alternative therapies (hydroxyurea or interferon-alpha or a combination thereof) had a wide range of side effects and only induced complete cytogenetic responses in fewer than half of the patients. With the advent of imatinib mesylate (Glivec) that induces a much better overall and event-free survival than therapy with interferon-alpha, the decision as to whether to transplant or to try Glivec first is not obvious. The problem is that resistance to Glivec may develop and that when this occurs, the chance of a successful transplantation may have decreased in the meantime.

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© Faculté de médecine de Genève | Contact | Updated : 21 January 2015