Blood Res.  2013 Jun;48(2):87-98. 10.5045/br.2013.48.2.87.

Use of azacitidine for myelodysplastic syndromes: controversial issues and practical recommendations

Affiliations
  • 1Department of Hematology, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
  • 2Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
  • 3Department of Internal Medcine, Chonbuk National University Medical School & Hospital, Jeonju, Korea.
  • 4Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
  • 5Deparment of Hematology-Oncology, Chonnam National University Hwasun Hospital, Hwasun, Korea. hjoonk@chonnam.ac.kr

Abstract

Azacitidine is recommended for patients with higher-risk myelodysplastic syndromes (MDS) who are not eligible for intensive therapy or for patients with lower-risk MDS who have thrombocytopenia or neutropenia or have anemia that is unresponsive to other therapies. However, standard treatment with azacitidine has not been optimized and many issues about the use of azacitidine remain unresolved. The use of azacitidine is expanding rapidly, but limited comparative clinical trial data are available to (i) define the optimal use of azacitidine in patients with higher-risk MDS or around the time of allogeneic hematopoietic stem cell transplantation, (ii) identify those patients with lower-risk MDS who may benefit from treatment, and (iii) guide physicians on alternative therapies after treatment failure. Increasing evidence suggests that the clinical features, prognostic factors, and cytogenetic profiles of patients with MDS in Asia differ significantly from those of patients in Western countries, so the aim of this review is to summarize the evidence and provide practical recommendations on the use of azacitidine in patients with MDS in the Republic of Korea. Evidence considered in this review is based on published clinical data and on the clinical experience of an expert panel from the acute myeloid leukemia/MDS Working Party of the Korean Society of Hematology.

Keyword

Azacitidine; Hypomethylating agents; Myelodysplastic syndromes; Practice guidelines as topic

MeSH Terms

Anemia
Asia
Azacitidine
Complementary Therapies
Cytogenetics
Hematology
Hematopoietic Stem Cell Transplantation
Humans
Myelodysplastic Syndromes
Neutropenia
Practice Guidelines as Topic
Republic of Korea
Thrombocytopenia
Treatment Failure
Azacitidine

Figure

  • Fig. 1 Recommended dose adaptations for patients with MDS who experience hematologic toxicity after treatment with azacitidine and who have (A) no reduction or (B) a reduction in blood counts before treatment [7]. Nadir is the lowest count reached in a given cycle. Recovery is defined as a blood count greater than the nadir count+(0.5×baseline count-nadir count). a)Improvement in differentiation includes any cell line, not only the cell line with decreased counts before treatment (e.g., percent counts are higher without transfusion than baseline counts). Abbreviations: ANC, absolute neutrophil count; BM, bone marrow; WBC, white blood cell.


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