Chart Review of Reduced Intensity Conditioning (RIC) Allogeneic Transplants in Elderly Patients With Myelofibrosis.
Primary myelofibrosis (PMF) is among the Philadelphia Chromosome Negative Myeloproliferative
Disorders. This diagnosis can be present at a patient's initial diagnosis or it can arise
out of preceding Polycythemia Vera or Essential Thrombocythemia. While the clinical course
is variable, it is defined by varying degrees of splenomegaly, anemia, fatigue and other
constitutional symptoms. Patients with PMF are at increased risk of acute leukemia, bone
marrow failure and thrombosis. Currently, the only curative treatment for PMF is
allogeneic stem cell transplant. However, as the median age at diagnosis is in the mid to
late 60s, most patients are no longer candidates for transplant due to their age and/or
other comorbid illnesses.
Unfortunately, all other treatments for PMF are palliative in nature and often of limited
efficacy. Over the last several years, many advances have occurred that have increased the
safety and improved the outcomes of allogeneic transplants. Perhaps most important has been
the ongoing refinement of reduced intensity conditioning (RIC) regimens prior to transplant.
Over the last few years, many groups have published data suggesting that these RIC
transplants can be very effective in the treatment of PMF and it is felt to be a potentially
curative procedure. However, the vast majority of these data are reported in persons
younger than 65 years old. The current protocol for RIC transplant for PMF available at the
University of Utah excludes patients older than the age of 65.
We would like to see if there is sufficient successful experience with transplant in persons
older than 60 years old (including many older than 65 years of age) to justify the creation
of a clinical trial using RIC regimens in this older age group. We will be reviewing the
medical records of approximately 30 patients at four different institutions:
- University of Utah/Huntsman Cancer Hospital
- Fred Hutchinson Cancer Research Center
- Baylor College of Medicine
- M.D. Anderson Cancer Centers
We will evaluate: engraftment rate, relapse rate, disease free survival, overall survival,
causes of death and other pertinent statistics. We will compare the outcomes to appropriate
historical controls. We hypothesize that RIC regimens may be justifiably safe in older
patients with PMF and hope that our data will allow the development of a corollary clinical
Observational Model: Cohort, Time Perspective: Retrospective
Evaluation of engraftment rate, relapse rate, disease free survival, overall survival, causes of death and other pertinent statistics. Data will be compared to appropriate historical controls.
Josef T Prchal, MD
University of Utah
United States: Institutional Review Board
|Baylor College of Medicine||Houston, Texas 77030|
|Fred Hutchinson Cancer Research Center||Seattle, Washington 98109|
|University of Utah||Salt Lake City, Utah|
|M.D. Anderson Cancer Centers||Houston, Texas 77030|