Clinical Research Center for Myeloproliferative Neoplasia
The Department of Leukemia has established a Clinical Research Center for Myeloproliferative Neoplasia (MPN) as a distinct operational unit within the Leukemia Department and Leukemia Outpatient Center. The goal of this clinical research center is to develop a comprehensive approach to understanding the biology of myeloproliferative neoplasms through translational research, leading to new and effective therapies for myeloproliferative disorders.
Under the guidance of Dr. Srdan Verstovsek, Chief of the Section of Myeloproliferative Neoplasms, MD Anderson has become the largest center in the world for MPN patient referral and research. We see annually more than 250 new patients with MPN, far more than any other clinical center in the world, and are engaged continuously in clinical research (conduct of clinical studies) to try to find effective therapies. Nothing of similar proportion exists anywhere in the world.
Components of the Clinical Research Center for Myeloproliferative Neoplasia include:
- Clinical trials with novel agents developed specifically for patients with MPN
- Centralized tissue bank and database for collection and annotation of patient samples
- Education about MPN and dissemination of information on new treatment and research strategies to patients, families and healthcare providers
Myeloproliferative neoplasias (also known as myeloproliferative diseases) have been traditionally divided into two groups: classic and atypical.
Classic MPNs include:
- Essential thrombocythemia (ET)
- Polycythemia vera (PV)
- Primary myelofibrosis (PMF)
Atypical MPNs include:
- Juvenile myelomonocytic leukemia (JMML)
- Systemic mastocytosis (SM)
- Chronic Eosinophilic Leukemia/Hypereosinophilic Syndrome (HES)
Classic MPNs are characterized by overproduction of cells in the bone marrow, leading to too many cells in the bloodstream. The incidence of these diseases varies from ~1.5 (for PMF) to ~2.3-2.5 (for ET and PV) per 100,000 people/year; this means that every year about 20,000 new patients are diagnosed with classic MPN. The total number of patients with MPN in the United States is estimated to be about 250,000. The average age of patients at diagnosis is 60-65 years, but the diseases spare no age group. Life expectancy varies from decades for patients with ET and PV to only 5-7 years for those with PMF.
Patients with PMF, the most aggressive of the classic MPNs, typically experience worsening bone marrow fibrosis (scarring) with progressive lowering of blood cell counts requiring transfusions, massive enlargement of the spleen and liver, profound fatigue, weight loss, night sweats, and low-grade fever, leading to very bad quality of life. Terrible body wasting and multi-organ failure eventually causes death. Therapy for MPNs, especially PMF, is unsatisfactory. While new therapies are improving outcomes, no therapy has been shown to eradicate the disease. However, Dr. Verstovsek is the Principal Investigator on several clinical studies testing new treatments for these diseases.
MD Anderson Resources
MPN Focus is a periodic newsletter published by the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasia to provide members of the MPN community with information on current research and treatments.
- Read the latest issue here.