Clinical trial NCT00723333: Bone Marrow Transplant Chart Review for RIC
Official title: Chart Review of Reduced Intensity Conditioning (RIC) Allogeneic Transplants in Elderly Patients With Myelofibrosis.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myelofibrosis
Keywords: Bone Marrow Transplant
Myelofibrosis
Stem Cell Transplant
Allogenic Transplant
Lead sponsor: University of Utah
Sponsor class: OTHER
Collaborators: name: Fred Hutchinson Cancer Center
name: Baylor College of Medicine
name: M.D. Anderson Cancer Center
First posted: 2008-07-28
Enrollment: 30
Sex: ALL
Minimum age (years): 60
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients \> 60 years of age with Primary Myelofibrosis that have undergone an allogeneic transplant

Brief summary: We will evaluate the records of 30 patients that have undergone allogeneic transplant, specifically looking at 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.

Detailed description: 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 trial.

Eligibility criteria: Inclusion Criteria:

* \> 60 years of age
* Diagnosed with Primary Myelofibrosis
* Undergone Allogeneic Transplant

Exclusion Criteria:

* Any subjects not meeting the criteria above

Primary outcomes:
measure: 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.
timeFrame: 30 days
Secondary outcomes:
Other outcomes:
Locations:
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: M.D. Anderson Cancer Centers
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center
state: Washington
Study officials:
affiliation: University of Utah
name: Josef T Prchal, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Hoffman R, Rondelli D. Biology and treatment of primary myelofibrosis. Hematology Am Soc Hematol Educ Program. 2007:346-54. doi: 10.1182/asheducation-2007.1.346.
citation: Mesa RA, Silverstein MN, Jacobsen SJ, Wollan PC, Tefferi A. Population-based incidence and survival figures in essential thrombocythemia and agnogenic myeloid metaplasia: an Olmsted County Study, 1976-1995. Am J Hematol. 1999 May;61(1):10-5. doi: 10.1002/(sici)1096-8652(199905)61:13.0.co;2-i.
citation: Rondelli D, Barosi G, Bacigalupo A, Prchal JT, Popat U, Alessandrino EP, Spivak JL, Smith BD, Klingemann HG, Fruchtman S, Hoffman R; Myeloproliferative Diseases-Research Consortium. Allogeneic hematopoietic stem-cell transplantation with reduced-intensity conditioning in intermediate- or high-risk patients with myelofibrosis with myeloid metaplasia. Blood. 2005 May 15;105(10):4115-9. doi: 10.1182/blood-2004-11-4299. Epub 2005 Jan 25.