Hematopoietic Cell Transplantation (HCT)

Abstract

The availability of drugs such as azacitidine, decitabine, and lenalidomide offers treatment options for patients with MDS that can alter the disease course. Randomized trials have shown that azacitidine, for example, extends the life expectancy by about 9 months, but eventually the disease will progress. Aside from rare patients with MDS who may have been cured by intensive chemotherapy, the only currently available treatment that offers the potential of cure is hematopoietic cell transplantation (HCT). However, HCT is associated with certain risks, related primarily to the “conditioning” regimen administered in preparation for HCT, and the immunologic reaction of donor cells against the patient, known as graft-versus-host disease (GVHD). Furthermore, as the average age of patients with MDS is in the 70s, comorbid conditions are common and may increase the risk of complications of HCT. Finally, HCT does not guarantee eradication of the disease; the probability of relapse correlates with the disease stage at the time of HCT and the disease risk reflected in the patient’s karyotype. Thus, consultations with patients regarding HCT involve a comprehensive discussion of timing of HCT, alternative therapy before or instead of HCT, donor availability, and quality of life (QOL) issues, among others.