SUMMARY
The research of Ayalew Tefferi, M.D., focuses on the pathophysiology, natural history and treatment of chronic myeloproliferative neoplasms (MPNs). Examples of MPNs include polycythemia vera, essential thrombocythemia and primary myelofibrosis. Dr. Tefferi's research efforts involve database and laboratory-based studies, as well as the development of new drugs for therapy.
Focus areas
- Study of MPN natural history and patient outcomes. Dr. Tefferi analyzes and identifies clinical and laboratory risk factors for MPN, especially those that are predictive of patient outcomes of interest. These include fibrotic transformation, leukemic transformation and overall survival. Dr. Tefferi uses disease-specific clinical databases with long patient follow-up. He has maintained data over several decades for this purpose.
- Molecular pathophysiology. Dr. Tefferi has directed research efforts for comprehensive genetic and mutational profiling of MPNs. This work has led to the development of genetic and clinical-molecular prognostic models that are in contemporary clinical use for patient care.
- Novel therapeutics. Dr. Tefferi has conducted numerous clinical trials that have led to licensing of several drugs in the U.S. for MPN indications. These therapeutic agents include ruxolitinib and momelotinib for the treatment of myelofibrosis.
- Study of rare diseases. Dr. Tefferi has led many studies of rare MPNs. These included systemic mastocytosis, eosinophilic conditions, histiocytic disorders and hemophagocytic lymphohistiocytosis.
Significance to patient care
Chronic myeloproliferative neoplasms (MPNs) make up a group of relatively uncommon diseases with overlapping clinical and biological features that are poorly understood. The only curative treatment is stem cell transplantation.
Dr. Tefferi's work in this area has provided a better understanding of the mechanisms of these diseases. This research has helped identify patient characteristics that predict disease-related complications. His studies have played a part in the development of new treatments for these diseases.
Dr. Tefferi's contributions have had a major impact on patient care. His research has been widely adopted by MPN experts and used to develop international guidelines. In addition, Dr. Tefferi's work on rare MPNs, such as systemic mastocytosis and eosinophilic disorders, has advanced the understanding of disease biology and assisted in the development of rational treatment strategies for these diseases.
Dr. Tefferi also promotes patient care by helping other healthcare professionals hone their expertise. He has served as a key faculty member of many national board review courses, including those sponsored by Harvard Medical School, The University of Texas MD Anderson Cancer Center and George Washington University.
Professional highlights
- Physician author, MPN content, UpToDate, 2022.
- Barbara Woodward Lips Professor II, Mayo Clinic, 2020.
- Henry M. Stratton Medal for Clinical/Translational Science, American Society of Hematology, 2016.