0

The full content of Annals is available to subscribers

Subscribe/Learn More  >
Articles |

Primary Treatment of Acquired Aplastic Anemia: Outcomes with Bone Marrow Transplantation and Immunosuppressive Therapy

Kristine Doney, MD; Wendy Leisenring, ScD; Rainer Storb, MD; and Frederick R. Appelbaum, MD
[+] Article and Author Information

For the Seattle Bone Marrow Transplant Team From the Fred Hutchinson Cancer Research Center, the University of Washington School of Medicine, and the Seattle Veterans Administration Medical Center, Seattle, Washington Acknowledgments: The authors thank the physicians, nurses, and support staff who comprise the Seattle Bone Marrow Transplant Team for their excellent patient care. They also thank the many physicians who referred their patients to the Fred Hutchinson Cancer Research Center. Grant Support: By Public Health Service grant HL36444 (awarded by the National Heart, Lung, and Blood Institute) and grants CA 18221, CA 15704, and CA 18029 (awarded by the National Cancer Institute). Requests for Reprints: Kristine Doney, MD, Fred Hutchinson Cancer Research Center, M-120, 1124 Columbia Street, Seattle, WA 98104. Current Author Addresses: Drs. Doney, Leisenring, Storb, and Appelbaum: Fred Hutchinson Cancer Research Center, Clinical Research Division, 1124 Columbia Street, Seattle, WA 98104.


Copyright ©2004 by the American College of Physicians


Ann Intern Med. 1997;126(2):107-115. doi:10.7326/0003-4819-126-2-199701150-00003
Text Size: A A A

Background: Both immunosuppressive therapy and bone marrow transplantation are accepted treatments for patients with aplastic anemia. Choosing one of these therapies for a given patient depends not only on donor availability but also on such factors as patient age.

Objective: To compare survival rates and long-term complications after bone marrow transplantation or immuno-suppressive therapy in patients with acquired aplastic anemia and to identify prognostic factors associated with improved survival.

Design: Center-based, retrospective analysis.

Setting: Referral center for patients with aplastic anemia.

Patients: 395 patients with acquired aplastic anemia.

Intervention: Bone marrow transplant from an HLA-identical, related donor or immunosuppressive therapy.

Measurements: Kaplan-Meier survival curves, results of log-rank tests, and cumulative incidence curves.

Results: Of 168 bone marrow transplant recipients, 89% had sustained engraftment. Forty-six patients developed grade II to IV acute graft-versus-host disease, and 68 developed chronic graft-versus-host disease that required therapy. Of 227 patients who received immunosuppressive therapy, 44% achieved a complete, partial, or minimal response. Fifty-four percent died or had no response to therapy. Actuarial survival at 15 years was 69% for bone marrow transplant recipients and 38% for patients receiving immunosuppressive therapy (P < 0.001). Improved survival was associated with having bone marrow transplantation as primary therapy, being younger, having no transfusion before transplantation, and having a higher absolute neutrophil count. Disease duration, year of therapy, sex, refractoriness to platelet transfusions, and previous treatment with androgens or corticosteroids did not significantly affect survival.

Conclusions: Data from this center suggest that bone marrow transplantation may be preferred for younger patients with acquired aplastic anemia who have matched, related donors. Long-term survival is excellent for patients who respond to either form of therapy.

Figures

Grahic Jump Location
Figure 1.
Cumulative incidence of long-term complications. Top.Bottom.

Cumulative incidence of rejection or evolution of a secondary malignant condition in patients having bone marrow transplantation. Cumulative incidence of evolution of a myelodysplastic syndrome or acute leukemia, paroxysmal nocturnal hemoglobinuria (PNH) or recurrent aplasia after immunosuppressive therapy.

Grahic Jump Location
Grahic Jump Location
Figure 2.
Actuarial survival after therapy for acquired aplastic anemia. Top.Bottom.P

Survival of 168 patients who had bone marrow transplantation. Survival of 227 patients who received immunosuppressive therapy. Tick marks denote censoring times of surviving patients. The difference in actuarial survival between patients who had transplantation and those who received immunosuppressive therapy was statistically significant ( < 0.001; log-rank test).

Grahic Jump Location
Grahic Jump Location
Figure 3.
Effect of patient age on actuarial survival by treatment group.PPPP

Tick marks denote censoring times of surviving patients. A. Patient age, younger than 6 years; 12 patients had transplantation and 25 patients received immunosuppressive therapy ( = 0.006). B. Patient age, 6 to 19 years; 63 patients had transplantation and 62 patients received immunosuppressive therapy ( = 0.001). C. Patient age, 20 to 39 years; 82 patients had transplantation and 73 patients received immunosuppressive therapy ( = 0.04). D. Patient age, 40 years or older; 11 patients had transplantation and 67 patients received immunosuppressive therapy ( > 0.2).

Grahic Jump Location
Grahic Jump Location
Figure 4.
Effect of transfusion status on actuarial survival by treatment group. Top.PBottom.P

Survival of 53 transplant recipients who did not receive transfusion and 115 transplant recipients who received transfusion ( = 0.04; log-rank test). Survival of 41 patients who received immunosuppressive therapy and did not receive transfusion and 174 patients who received immunosuppressive therapy and transfusion ( = 0.11; log-rank test). Tick marks denote censoring times of surviving patients.

Grahic Jump Location
Grahic Jump Location
Figure 5.
Effect of the absolute neutrophil count (ANC) before therapy on actuarial survival by treatment group.PP9999

Tick marks denote censoring times of surviving patients. > 0.2 for the bone marrow transplantation group and = 0.002 for the immunosuppressive therapy group. In the bone marrow transplantation group, 98 patients had an absolute neutrophil count greater than 2.0 × 10 /L and 70 had a count less than 2.0 × 10 /L. In the immunosuppressive therapy group, 133 patients had an absolute neutrophil count greater than 2.0 × 10 /L and 94 had a count less than 2.0 × 10 /L.

Grahic Jump Location

Tables

References

Letters

NOTE:
Citing articles are presented as examples only. In non-demo SCM6 implementation, integration with CrossRef’s "Cited By" API will populate this tab (http://www.crossref.org/citedby.html).

Comments

Submit a Comment
Submit a Comment

Summary for Patients

Clinical Slide Sets

Terms of Use

The In the Clinic® slide sets are owned and copyrighted by the American College of Physicians (ACP). All text, graphics, trademarks, and other intellectual property incorporated into the slide sets remain the sole and exclusive property of the ACP. The slide sets may be used only by the person who downloads or purchases them and only for the purpose of presenting them during not-for-profit educational activities. Users may incorporate the entire slide set or selected individual slides into their own teaching presentations but may not alter the content of the slides in any way or remove the ACP copyright notice. Users may make print copies for use as hand-outs for the audience the user is personally addressing but may not otherwise reproduce or distribute the slides by any means or media, including but not limited to sending them as e-mail attachments, posting them on Internet or Intranet sites, publishing them in meeting proceedings, or making them available for sale or distribution in any unauthorized form, without the express written permission of the ACP. Unauthorized use of the In the Clinic slide sets will constitute copyright infringement.

Toolkit

Buy Now

to gain full access to the content and tools.

Want to Subscribe?

Learn more about subscription options

Advertisement
Related Articles
Related Point of Care
Topic Collections
PubMed Articles

Buy Now

to gain full access to the content and tools.

Want to Subscribe?

Learn more about subscription options

Forgot your password?
Enter your username and email address. We'll send you a reminder to the email address on record.
(Required)
(Required)