The survival graphs below illustrate outcomes of unrelated donor hematopoietic cell transplants (bone marrow, PBSC, or cord blood transplants — BMT) coordinated by the National Marrow Donor Program® (NMDP) for both adult and pediatric patients.
NMDP outcomes for adult AML patients
Five-year survival for adult patients (≥18 years of age) transplanted for acute myelogenous leukemia (AML) using marrow (Figure 1) or peripheral blood stem cells (PBSC) (Figure 2) is significantly increased in patients transplanted in first or second complete remission compared to those patients transplanted with advanced disease (log-rank p-value <0.001). Unrelated transplant for AML in first complete remission is indicated if the patient has poor-risk cytogenetics at diagnosis or induction failure.
In a 2007 study of 261 AML patients undergoing matched unrelated donor transplantation, cytogenetic risk factors had no effect on five-year overall survival and DFS for patients in first complete remission. In second complete remission, there was a non-significant trend toward better survival for patients with favorable cytogenetics. The researchers concluded that matched unrelated donor transplantation should be considered for patients with unfavorable cytogenetics lacking a suitable HLA-matched sibling donor. [1]
Both PBSC and cord blood are being studied under FDA investigational new drug (IND) protocols. Researchers continue to investigate rates of graft-versus-host disease (GVHD) and other characteristics of both stem cell sources.
In one such study, the NMDP and the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) conducted a large-scale, multi-center phase III clinical trial to determine if graft source (PBSC or marrow) affects transplant outcomes in unrelated donor transplantation for AML and other hematologic malignancies. [2]
The study found that PBSC and bone marrow transplant recipients had comparable two-year survival: 51% vs. 46%, respectively (p=0.29). However, chronic GVHD developed more often and was more severe in patients who received PBSC compared with those receiving marrow. [3]
Figure 1.
Acute myelogenous leukemia: Survival of adult marrow myeloablative transplant patients by disease stage 2000-2009. (NMDP data)
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Figure 2.
Acute myelogenous leukemia: Survival of adult PBSC myeloablative transplant patients by disease stage 2000-2009. (NMDP data)
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NMDP outcomes for pediatric AML patients
Five-year survival for pediatric patients (<18 years of age) transplanted for AML is significantly increased for those patients transplanted in second complete remission compared to those transplanted in first complete remission or with advanced disease. However, a greater percent of patients transplanted in first complete remission had poor-risk cytogenetics compared to those patients who were transplanted in second complete remission (log-rank p-value < 0.0004). Unrelated transplant for AML in first complete remission is indicated if the patient has poor-risk cytogenetics at diagnosis or induction failure.
Figure 3.
Acute myelogenous leukemia: Survival of pediatric myeloablative marrow transplant patients by disease stage 2000-2009. (NMDP data)
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NMDP outcomes for adult AML patients ≥ age 55
Due to their lowered toxicity, non-myeloablative or reduced-intensity transplants have expanded the number of patients eligible for hematopoietic cell transplantation. For more information, see Advances in Conditioning Regimens. Figure 4 shows five-year survival for adult patients 55 years of age or older undergoing transplantation for AML.
Figure 4.
Acute myelogenous leukemia: Survival of marrow and PBSC transplant patients ≥55 years old by disease stage 2000-2009. (NMDP data)
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References
- Tallman MS, Dewald GW, Gandham S, et al. Impact of cytogenetics on outcome of matched unrelated donor hematopoietic stem cell transplantation for acute myeloid leukemia in first or second complete remission. Blood. 2007; 110(1):409-417.
http://bloodjournal.hematologylibrary.org/cgi/content/abstract
/110/1/409
- BMT CTN Protocol 0201 - A Phase III randomized multicenter trial comparing G-CSF mobilized peripheral blood stem cell with marrow transplantation from HLA compatible unrelated donors.
https://web.emmes.com/study/bmt2/protocol/0201__protocol/
0201_protocol.html
- Anasetti C, Logan BR, Lee SJ, et al. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med. 2012; 367(16): 1487-1496.
http://www.nejm.org/doi/full/10.1056/NEJMoa1203517