Among the primary objectives of the American Society for Blood are Marrow Transplantation are to:
▪Define commonly accepted medical practice
▪Develop standards of medical care for autologous and allogeneic transplants
▪Provide recommendations and guidelines for the role of transplantation as a therapeutic approach for reimbursement by third-party payers.
To this end, in 1999 the Society began the development of evidence-based reviews of the scientific and medical literature to document when blood and marrow transplantation is indicated in the treatment of selected diseases.
Goals
The goals of the evidence-based reviews are to:
▪Determine which disease will be the subject of each review, establish the focus for each review and develop a list of questions to be addressed
▪Assemble and critically evaluate all the valid, peer-reviewed evidence regarding the role of cytotoxic therapy with hematopoietic stem cell transplantation related to the disease
▪Make treatment recommendations based on the available evidence
▪Identify discrepancies in study design or methodology among published studies that may impact on the quality of the evidence
▪Identify needed areas of additional research
Guidelines
The following guidelines are offered for the role of stem cell transplantation (SCT) as therapy for acute myeloid leukemia (AML) in adults, and are based on consensus reached by an expert panel1 following an evidence-based review of the literature2:
Transplantation versus Chemotherapy
1.There is no significant advantage of autologous SCT over chemotherapy. Most of the data reflect outmoded treatment strategies, however, and studies using modern technologies may affect outcomes.
2.There is a survival advantage for allogeneic SCT vs. chemotherapy for patients under age 55 with high risk cytogenetics.
3.There is insufficient evidence to routinely recommend allogeneic SCT for patients with intermediate risk cytogenetics, although this is a reasonable strategy.
4.There is no survival advantage for allogeneic SCT in patients under age 55 with low risk cytogenetics.
5.There are insufficient data to make a recommendation for the use of myeloablative regimens for patients over age 55.
6.There are insufficient data to make a recommendation for reduced intensity conditioning (RIC) allogeneic SCT vs. chemotherapy.
7.For patients in second complete remission, allogeneic SCT is recommended if there is an available donor. Otherwise an autologous SCT is recommended.
Transplantation Techniques
1.An HLA-matched related donor allogeneic SCT is recommended over autologous SCT, if a matched related donor is available. For matched unrelated donor allogeneic SCT, there are insufficient data to make a recommendation over autologous SCT. Available studies, however, do not reflect modern techniques in supportive care, stem cell source, or the use of molecular HLA typing.
2.Autologous peripheral blood stem cell transplant (PBSCT) is recommended over autologous bone marrow transplant (BMT) due to improvements in safety and early mortality. Long-term outcomes have not been studied prospectively, however, and the impact of autologous PBSCT on overall survival is not known.
3.There is no evidence of a survival advantage with purged BMT and insufficient data to make a recommendation for purging of PBSCT for autologous SCT.
4.There are insufficient data to make a recommendation for tandem vs. single autologous SCT.
5.Allogeneic SCT with a matched related donor is recommended if available. A matched unrelated donor allogeneic SCT using reduced intensity conditioning may provide equivalent outcomes.
6.There is no evidence of a survival advantage with T-cell depleted grafts from allogeneic donors.
7.For high risk disease, allogeneic PBSCT is recommended over BMT. For low risk disease, allogeneic PBSCT and BMT have equivalent outcomes.
8.There are insufficient data to make a recommendation for PBSCT vs. BMT in matched unrelated donor SCT.
Therapy Regimens
1.There is no evidence of a survival advantage with any one high dose therapy regimen in autologous SCT.
2.There is no significant survival advantage with any one myeloablative conditioning regimen in allogeneic SCT. Studies of late effects may change this recommendation.
3.Fractionated rather than a single dose total body irradiation (TBI) conditioning regimen is recommended in allogeneic SCT.
4.There are insufficient data to make a recommendation for RIC for allogeneic SCT. The use of RIC is dependent on patient characteristics.
∗A separate evidence-based review3 is available for the role of SCT in pediatric AML patients.
—Adopted by the ASBMT Executive Committee
On November 16, 2007
1 Expert panel members and authors of the review are: Denise M. Oliansky, Roswell Park Cancer Institute (RPCI), Buffalo, NY; Frederick Appelbaum, Fred Hutchinson Cancer Institute, Seattle, WA; Peter A. Cassileth, University of Miami Sylvester Cancer Center, Miami, FL; Armand Keating, University of Toronto, Toronto, Ontario, Canada; Jamie Kerr, Excellus Blue Cross/Blue Shield, Rochester, NY; Yago Nieto, MD Anderson Cancer Center, Houston, TX; Susan Stewart, BMTInfonet, Chicago, IL; Richard M. Stone, Dana Farber Cancer Institute, Boston, MA; Martin Tallman, Northwestern University Feinberg School of Medicine, Robert H. Lurie Comprehensive Cancer Center, Chicago, IL; Philip L. McCarthy, Jr., RPCI, Theresa Hahn, RPCI.
2 Oliansky D, Appelbaum F, Cassileth P, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute myeloid leukemia in adults: an evidence-based review. Biol Blood Marrow Transplant. 2007;14:135-136.
3 Oliansky D, Rizzo JD, Aplan PT, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute myeloid leukemia in children: an evidence-based review. Biol Blood Marrow Transplant. 2007;13(1):1-25.
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