Bone Marrow Transplant Chart Review for RIC

NCT ID: NCT00723333

Last Updated: 2009-10-12

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 30 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2008-05-31
• Study Completion Date: 2009-07-31

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.

Conditions

Myelofibrosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

Affected Group

Patients \> 60 years of age with Primary Myelofibrosis that have undergone an allogeneic transplant

Reduced Intensity Conditioning (RIC) Allogenic Transplant

Intervention Type: PROCEDURE

Reduced Intensity Conditioning (RIC) Regimen Allogenic Stem Cell Transplant

Interventions

Reduced Intensity Conditioning (RIC) Allogenic Transplant

Reduced Intensity Conditioning (RIC) Regimen Allogenic Stem Cell Transplant

Intervention Type: PROCEDURE

Other Intervention Names

Stem Cell Transplants; Bone Marrow Transplants; Mini Transplants

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

• Any subjects not meeting the criteria above

• Minimum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fred Hutchinson Cancer Center

OTHER

Sponsor Role: collaborator

Baylor College of Medicine

OTHER

Sponsor Role: collaborator

M.D. Anderson Cancer Center

OTHER

Sponsor Role: collaborator

University of Utah

OTHER

Sponsor Role: lead

Responsible Party

University of Utah

Principal Investigators

Josef T Prchal, MD

Role: PRINCIPAL_INVESTIGATOR

University of Utah

Locations

Baylor College of Medicine

Houston, Texas, United States

M.D. Anderson Cancer Centers

Houston, Texas, United States

University of Utah

Salt Lake City, Utah, United States

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Countries

United States

References

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.

Reference Type: BACKGROUND

PMID: 18024650 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 10331505 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 15671439 (View on PubMed)

Other Identifiers

29021

Identifier Type: -

Identifier Source: org_study_id