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Durable Treatment-Free Remission after High-Dose Cyclophosphamide Therapy for Previously Untreated Severe Aplastic Anemia

Robert A. Brodsky, MD; Lyle L. Sensenbrenner, MD; B. Douglas Smith, MD; Donna Dorr, RN; Phillip J. Seaman, PA-C; Shing M. Lee, ScM; Judith E. Karp, MD; Isadore Brodsky, MD; and Richard J. Jones, MD
[+] Article and Author Information

From the Johns Hopkins Oncology Center and the Greenbaum Cancer Center, University of Maryland, Baltimore, Maryland; and MCP Hahnemann University, Philadelphia, Pennsylvania.


Acknowledgment: The authors thank the physicians, nurses, and support staff of the Johns Hopkins University Bone Marrow Transplant Team, MCP Hahnemann University, and the Greenbaum Cancer Center for their excellent patient care.

Grant Support: In part by grant CA70970 from the National Institutes of Health. Dr. R. Brodsky is a Leukemia and Lymphoma Society Clinical Research Scholar and The Thomas E. Sandefur Jr. Fellow in Stem Cell Research.

Request for Single Reprints: Robert A. Brodsky, MD, Johns Hopkins Oncology Center, Bunting-Blaustein Cancer Research Building, Room 242, 1650 Orleans Street, Baltimore, MD 21231; e-mail, rbrodsky@jhmi.edu.

Current Author Addresses: Dr. R. Brodsky, Dr. Smith, Ms. Dorr, Mr. Seaman, and Dr. Jones: Johns Hopkins Oncology Center, Bunting-Blaustein Cancer Research Building, Room 242, 1650 Orleans Street, Baltimore MD 21231.

Dr. Sensenbrenner: 1613 Camden Avenue, Salisbury, MD 21801-7010.

Ms. Lee: Johns Hopkins Oncology Center, Oncology Biostatistics, 550 North Broadway, Suite 1103, Baltimore, MD 21205.

Dr. I. Brodsky: MCP Hahnemann University, Broad and Vine Streets, Mailstop 412, Philadelphia, PA 19102.

Dr. Karp: Greenbaum Cancer Center, University of Maryland School of Medicine, 22 South Greene Street, Room S9D15, Baltimore, MD 21201-1544.

Author Contributions: Conception and design: R.A. Brodsky, L.L. Sensenbrenner, R.J. Jones.

Analysis and interpretation of the data: R.A. Brodsky, B.D. Smith, J.E. Karp, I. Brodsky, R.J. Jones.

Drafting of the article: R.A. Brodsky.

Critical revision of the article for important intellectual content: L.L. Sensenbrenner, B.D. Smith, R.J. Jones.

Final approval of the article: R.A. Brodsky, R.J. Jones.

Provision of study materials or patients: R.A. Brodsky, J.E. Karp, I. Brodsky.

Statistical expertise: S.M. Lee.

Obtaining of funding: R.A. Brodsky, R.J. Jones.

Administrative, technical, or logistic support: B.D. Smith, D. Dorr, P.J. Seaman.

Collection and assembly of data: R.A. Brodsky, B.D. Smith, D. Dorr, P.J. Seaman.


Ann Intern Med. 2001;135(7):477-483. doi:10.7326/0003-4819-135-7-200110020-00006
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Acquired severe aplastic anemia is a life-threatening bone marrow failure disorder characterized by hypocellular bone marrow and peripheral blood pancytopenia (1). Although most cases of severe aplastic anemia are idiopathic, bone marrow failure usually results from an immune-mediated attack on hematopoietic stem cells regardless of the inciting event (23). In patients treated with antibiotics and blood transfusions alone, the mortality rate is 80% at 2 years (4).

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Figure 1.
Kaplan–Meier probability of survival in patients with severe aplastic anemia given high-dose cyclophosphamide as first-line therapy.
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Figure 2.
Kaplan-Meier probability of treatment-free remission (solid line) and complete remission (dotted line) in patients with severe aplastic anemia given high-dose cyclophosphamide as first-line therapy.

Three patients died and were included in the analyses.

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Summary for Patients

High-Dose Cyclophosphamide To Treat Severe Aplastic Anemia

The summary below is from the full report titled “Durable Treatment-Free Remission after High-Dose Cyclophosphamide Therapy for Previously Untreated Severe Aplastic Anemia.” It is in the 2 October 2001 issue of Annals of Internal Medicine (volume 135, pages 477-483). The authors are RA Brodsky, LL Sensenbrenner, BD Smith, D Dorr, PJ Seaman, SM Lee, JE Karp, I Brodsky, and RJ Jones.

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