Characteristics of US patients with myelodysplastic syndromes: results of six cross-sectional physician surveys

Abstract

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.

Figure 1

Flow diagram of the administration of six cross-sectional surveys completed by physicians treating patients with myelodysplastic syndrome (MDS) in the United States. *Includes medical oncologists, hematologists/oncologists, hematologists, internal medicine physicians with a secondary specialty in oncology, gynecological oncologists, and urologists. †Sample of 426 physicians was compared with internal data from more than 50 pharmaceutical and biotechnology companies and was found to be representative in terms of tumor types seen and treatment patterns in the United States. ‡Considered to be representative of the larger group of 426 physicians. AMA = American Medical Association.

Figure 2

Transfusion dependency among recently diagnosed patients. IPSS = International Prognostic Scoring System; Low/Int-1 = low and intermediate-1 risk classification; Int-2/High = intermediate-2 and high risk classification.

Figure 3

Mean proportion of recently diagnosed patients (N = 670) and range of percentages of established patients across six surveys (N = 3844) taking specific types of therapies at the time of the survey. ESA = erythropoiesis-stimulating agent; G-CSF = granulocyte colony-stimulating agent; GM-CSF = granulocyte–macrophage colony-stimulating agent.