Management of patients with higher risk myelodysplastic syndromes.
Higher risk myelodysplastic syndromes (MDS) include patients in the Intermediate-2 and high-risk categories of the International Prognostic Scoring System, as well as patients with MDS secondary to radiation or chemical exposure. Ideally, the goal of therapy is to alter the natural history of disease in these patients to achieve cure or durable remission. High-intensity chemotherapy can achieve moderate rates of complete remission, however, durability of remission and overall survival tend to be short. Hematopoietic stem cell transplantation (HSCT) offers the possibility of cure, with long-term disease-free survival inversely related to age. Patients who are elderly or have poor functional status are candidates for reduced intensity HSCT, although this is still an experimental modality. Azacitidine is a hypomethylating agent that is a reasonable option for many patients ineligible for high-intensity therapies. Other therapies, such as immunomodulatory agents, arsenic trioxide, and farnesyl transferase inhibitors have thus far shown limited usefulness in higher risk MDS. This paper reviews the various therapeutic options for higher risk MDS, providing rationale for specific management approaches for these patients.
Fukumoto
Jon S
JS
Division of Hematology, Stanford University Medical Center, 875 Blake Wilbur Drive, Stanford, CA 94305-5821, USA. jon.fukumoto@stanford.edu
Greenberg
Peter L
PL
D000367:Age Factors; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use; D000077237:Arsenic Trioxide; D001152:Arsenicals / Q000627:therapeutic use; D001374:Azacitidine / Q000627:therapeutic use; D003131:Combined Modality Therapy / Q000379:methods; D018572:Disease-Free Survival; D051231:Farnesyltranstransferase / Q000037:antagonists & inhibitors; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D009190:Myelodysplastic Syndromes / Q000209:etiology / Q000401:mortality / Q000628:therapy*; D010087:Oxides / Q000627:therapeutic use; D011379:Prognosis; D012307:Risk Factors; D016896:Treatment Outcome
D000964:Antimetabolites, Antineoplastic; D001152:Arsenicals; D010087:Oxides; D051231:Farnesyltranstransferase; D001374:Azacitidine; D000077237:Arsenic Trioxide