The myelodysplastic syndromes: current approaches to therapy.
OBJECTIVE
To review the current therapeutic options for patients with the myelodysplastic syndromes.


METHODS
Studies reported between 1968 and September 1989 were identified through computer searches using MEDLINE and through extensive searching of bibliographies of identified articles.


METHODS
All articles, abstracts, and reviews were evaluated, and those that contained therapeutic data were analyzed for response rates, survival, and toxicity.


RESULTS
Therapies for the myelodysplastic syndromes have included hormones, chemotherapy, bone marrow transplantation, and differentiating agents, including hematopoietic growth factors. With the exception of bone marrow transplantation, none is curative or increases survival. Hematopoietic growth factors are of interest for clinical trials, because they increase the number of neutrophils and, occasionally, the number of platelets in patients with the myelodysplastic syndromes. Nonetheless, hematopoietic growth factors are not without toxicity; most notably, they are associated with a risk for acceleration to acute myeloid leukemia, and their effect on survival remains unknown.


CONCLUSIONS
Standard therapy for the myelodysplastic syndromes is supportive care. When the disease progresses, patients with aggressive histologic or other poor-risk features should be considered for aggressive chemotherapy, with or without growth factors, or bone marrow transplantation. Patients with good prognostic features are candidates for therapy with growth factors or other potential differentiating agents. Carefully designed and conducted clinical trials that incorporate correlative laboratory science are essential to developing a more rational approach to therapy.
National Cancer Institute, Bethesda, Maryland.
B D
BD
Cheson
D000970:Antineoplastic Agents / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D016026:Bone Marrow Transplantation; D002454:Cell Differentiation / Q000187:drug effects; D006133:Growth Substances / Q000627:therapeutic use; D006728:Hormones / Q000627:therapeutic use; D006801:Humans; D007951:Leukemia, Myeloid / Q000188:drug therapy; D009190:Myelodysplastic Syndromes / Q000188:drug therapy / Q000628:therapy*
D000970:Antineoplastic Agents; D006133:Growth Substances; D006728:Hormones