Protocol Number: 00-CC-0165

Title:

Filgrastim-Mobilized Peripheral Blood Stem Cells for Primary Allogeneic Transplantation Using Unrelated Donors Participating in the National Marrow Donor Program (NMDP)

Number:

00-CC-0165

Summary:

The National Marrow Donor Program (NMDP) was established 15 years ago to provide unrelated bone marrow donors for persons in need of a stem cell transplant, but lacking suitable family donors. As of August 2006, the Program has facilitated greater than 25,000 marrow transplants via its national and international network of Donor Centers, Transplant Centers, and Collection Centers, and is currently operating at about 250 transplants facilitated per month. The program has had phenomenon success in recruiting potential donors, and the Registry size is now 6.2 million. Transplant outcomes have been more problematic, with acute transplant-related mortality and more severe grades of acute and chronic graft-versus-host disease occurring with nearly twice the frequency seen in related, HLA-matched sibling donor transplants.

Peripheral blood stem cell (PBSC) grafts, collected by apheresis of filgrastim-mobilized donors, are being increasingly used in the setting of related-donor, HLA-identical transplants. The advantages of PBSC over marrow in allotransplant settings include more rapid engraftment, decreased acute transplant-related mortality, more rapid immunologic reconstitution, and larger stem cell doses. From the donor's perspective, PBSC harvests do not require anesthesia and pelvic surgery, and may result in less discomfort and long-term sequelae than marrow harvests. These potential advantages to both the donor and the recipient have led to the creation of the current protocol, which describes a system for the collection and evaluation of PBSC as an alternative to marrow in unrelated donor stem cell transplantation. The protocol describes processes for donor evaluation and education, establishes procedures for administration and monitoring of filgrastim and indefinite donor follow-up, and includes policies for the collection of PBSC components by leukapheresis. This protocol will apply to all PBSC collections performed by NMDP Donor and Apheresis Centers and will interface with IRB-approved transplantation protocols maintained separately by NMDP Transplant Centers.

Since filgrastim is not licensed for use in healthy subjects as a mobilizing agent to facilitate collection of PBSC's by apheresis, this protocol is being performed under an FDA IND sponsored by the NMDP. It is intended for use with minimal changes by participating institutions, per IND application.

The NIH Marrow Donor Center, operated by the CC Department of Transfusion Medicine (DTM), currently has a registry size of 55,457 local participants and has provided 395 marrow or blood stem cell donors for NMDP-facilitated transplants to date. DTM staff consider the mission of the NMDP to be of critical national importance and have been prime movers in the creation of this protocol. Drs. Stroncek and Leitman were members of the Writing Group for this study.

Sponsoring Institute:

National Institutes of Health Clinical Center (CC)

Recruitment Detail

Type: Participants currently recruited/enrolled

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): None

Eligibility Criteria:

INCLUSION CRITERIA:

PBSC donors must meet the same criteria as NMDP marrow donors. These criteria are set forth in NMDP Standards and the Donor center manual of Operations.

EXCLUSION CRITERIA:

Failure to qualify as an NMDP marrow donor.

Pregnancy or uninterruptible breast-feeding. Pregnancy is an absolute contraindication under this protocol. Women who are breast-feeding must be willing and able to interrupt breast-feeding during the administration of Filgrastim and for two days following the final dose.

Sensitivity to Filgrastim or to E. coli-derived recombinant protein products.

History of autoimmune disorders, including rheumatic diseases and thyroid disorders. Exception: As with bone marrow donations, donors with a history of autoimmune thyroid disease who have undergone medical or surgical thyroid ablation may be eligible. Approval must be obtained from the NMDP.

History of deep vein thrombosis or pulmonary embolism. History of iritis or episcleritis. Thrombocytopenia less than 150 x 10(9)/L (less than 150,000/microliter) at baseline evaluation.

Current treatment with Lithium. Drug interactions between Filgrastim and Lithium which may potentiate the release of neutrophils, have not been fully evaluated.

History of coronary artery disease. History of chest pain consistent with angina shall be evaluated by cardiology consultation, and subjects with symptomatic coronary heart disease shall be excluded.

Special Instructions:

Currently Not Provided

Keywords:

Unrelated Donor

Peripheral Blood Stem Cells

Hematopoietic Stem Cell Transplantation

Filgrastim

National Marrow Donor Program (NMDP)

Recruitment Keyword(s):

Bone Marrow Donation

Condition(s):

Hematopoietic Stem Cell Mobilization

Investigational Drug(s):

Filgrastim

Investigational Device(s):

None

Intervention(s):

Drug: Filgrastim

Supporting Site:

Warren G. Magnuson Clinical Center

Contact(s):

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citation(s):

Syngeneic transplantation with peripheral blood mononuclear cells collected after the administration of recombinant human granulocyte colony-stimulating factor

Allogeneic blood stem cell transplantation: peripheralization and yield of donor-derived primitive hematopoietic progenitor cells (CD34+ Thy-1dim) and lymphoid subsets, and possible predictors of engraftment and fraft-versus-h

Allogeneic blood stem cell transplantation for refractory leukemia and lymphoma: potential advantage of blood over marrow allografts

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National Institutes of Health Clinical Center Bethesda, Maryland 20892. Last update: 01/30/2009