Journal of the National Cancer Institute Advance Access originally published online on October 28, 2008
JNCI Journal of the National Cancer Institute 2008 100(21):1542-1551; doi:10.1093/jnci/djn349
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© The Author 2008. Published by Oxford University Press.
ARTICLES |
Characteristics of US Patients with Myelodysplastic Syndromes: Results of Six Cross-sectional Physician Surveys
Affiliations of authors: Department of Hematologic Oncology and Blood Disorders, Cleveland Clinic Taussig Cancer Institute, Cleveland, OH (MAS, JPM); Amgen Ltd, London, UK (WMS); The University of Texas M. D. Anderson Cancer Center, Houston, TX (HK); H. Lee Moffitt Cancer Center & Research Institute, Tampa, FL (AL); Amgen Inc., Thousand Oaks, CA (JF); University of California Los Angeles, Los Angeles, CA (RP)
Correspondence to: Mikkael A. Sekeres, MD, MS, Department of Hematologic Oncology and Blood Disorders, Cleveland Clinic Taussig Cancer Institute, Desk R35, 9500 Euclid Ave, Cleveland, OH 44195 (e-mail: sekerem{at}ccf.org).
Background: Myelodysplastic syndromes (MDS) comprise a group of pathologically and cytogenetically distinct bone marrow disorders. Little is known about the characteristics of MDS patients, including their pathological and prognostic classifications, cytopenias, transfusion and supportive care needs, and treatment regimens. We describe these characteristics in a large group of recently diagnosed and existing (ie, established) MDS patients.
Methods: We conducted six consecutive cross-sectional surveys among US hematology and medical oncology specialists (identified from an American Medical Association [AMA] database of physicians who administer chemotherapy) between June 2005 and January 2007. A questionnaire collected data on the characteristics and treatment patterns of the 4–10 most recently seen MDS patients for each physician, including demographic data, transfusion needs, treatment approaches, and consideration for clinical trials or bone marrow transplantation.
Results: A panel of 101 physicians who were geographically representative of physicians registered with the AMA characterized 614–827 patients per survey, for a total of 4514 responses. Among recently diagnosed patients, 55% were male (95% confidence interval [CI] = 52% to 59%), the median age at diagnosis was 71 years (range = 65–80 years), and 10% (95% CI = 8% to 12%) had MDS secondary to chemotherapy, radiation therapy, or environmental exposure. The median duration of MDS in established patients ranged from 13 to 16 months over the six surveys. Among recently diagnosed MDS patients, fewer patients with lower-risk disease than with higher-risk disease were dependent on either red blood cell transfusions (22% vs 68%) or platelet transfusions (6% vs 33%). More than 50% of all newly diagnosed and established patients used erythropoiesis-stimulating agents. A small percentage of all patients either had had or were being considered for bone marrow transplantation (recently diagnosed: 4%; established: 4% or less) or were being treated on clinical trials (recently diagnosed: 1%; established: 4% or less).
Conclusions: MDS patients in the United States have substantial transfusion needs, and use of erythropoiesis-stimulating agents and are seldom considered for bone marrow transplantation or clinical trials. These data may be useful in characterizing the health care resource use and pharmacoeconomic impact of MDS in the United States.
| CONTEXT AND CAVEATS Prior knowledge Myelodysplastic syndromes (MDS) comprise a group of pathologically and cytogenetically distinct bone marrow disorders. Recent changes in the therapeutic options for MDS support the need to characterize MDS patients, including their pathological and prognostic classifications, transfusion and supportive care needs, and treatment regimens. Study design Six consecutive cross-sectional surveys of 101 hematology and medical oncology specialists in the United States were conducted between June 2005 and January 2007 via questionnaires to ascertain the characteristics and treatment patterns of the 4–10 most recently seen MDS patients for each physician. Contribution The physicians characterized 614–827 patients per survey, for a total of 4514 responses. A high proportion of MDS patients were dependent on red blood cell or platelet transfusions. Among recently diagnosed MDS patients, fewer patients with lower-risk disease than with higher-risk disease were dependent on transfusions. More than half of MDS patients were treated with erythropoiesis-stimulating agents. Only a small percentage of MDS patients either had had or were being considered for bone marrow transplantation or were being treated on clinical trials. Implications These data may be useful in characterizing the health care resource use and pharmacoeconomic impact of MDS in the United States. Limitations The data were collected retrospectively by asking physicians to report on their 4–10 most recently seen MDS patients. Because of the lack of unique identifiers for patients and their physicians, it was not possible to identify a cohort of MDS patients who could be followed over time. From the Editors
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Manuscript received June 9, 2008; revised August 12, 2008; accepted August 28, 2008.
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