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High-Dose Melphalan and Autologous Stem-Cell Transplantation in Patients with AL Amyloidosis: An 8-Year Study

Martha Skinner, MD; Vaishali Sanchorawala, MD; David C. Seldin, MD, PhD; Laura M. Dember, MD; Rodney H. Falk, MD; John L. Berk, MD; Jennifer J. Anderson, PhD; Carl O'Hara, MD; Kathleen T. Finn, RN, NP; Caryn A. Libbey, MD; Janice Wiesman, MD; Karen Quillen, MD; Niall Swan, MD; and Daniel G. Wright, MD
[+] Article and Author Information

From Boston University School of Medicine, Boston, Massachusetts.


Acknowledgments: The authors appreciate the contributions of former members of the Amyloid Treatment and Research Program and the Stem Cell Transplant Program who participated in the initial years of this work. They gratefully acknowledge the excellent data management of Arquimedes Areche and Akira Murakami and the patient coordination of Salli Fennessey, Karen Donovan, Serena Baqui, Ceit McCaleb, Lisa Marie Paul, and Julie Cosio. They also thank the many nurses, particularly Rick Kunz, stem-cell transplant nurse coordinator, who participated in the care of these patients.

Grant Support: By grants from the National Institutes of Health (HL 68705), U.S. Food and Drug Administration (FD-R-001346), the Gerry Foundation, the Young Family Amyloid Research Fund, the Sue Sellors Finley Cardiac Amyloid Research Fund, and the Amyloid Research Fund at Boston University. Dr. Seldin is a Scholar of the Leukemia and Lymphoma Society of America.

Potential Financial Conflicts of Interest: None disclosed.

Requests for Single Reprints: Martha Skinner, MD, Amyloid Treatment and Research Program, K503, Boston University School of Medicine, 715 Albany Street, Boston, MA 02118; e-mail, mskinner@bu.edu.

Current Author Addresses: Drs. Skinner, Sanchorawala, Seldin, Dember, Falk, Berk, Anderson, O'Hara, Finn, Libbey, Wiesman, Quillen, Swan, and Wright: Amyloid Treatment and Research Program, K503, Boston University School of Medicine, 715 Albany Street, Boston, MA 02118.

Author Contributions: Conception and design: M. Skinner, D.C. Seldin, L.M. Dember, J.L. Berk, C. O'Hara, D.G. Wright,

Analysis and interpretation of the data: M. Skinner, V. Sanchorawala, D.C. Seldin, L.M. Dember, R.H. Falk, J.J. Anderson, D.G. Wright.

Drafting of the article: M. Skinner, V. Sanchorawala, D.C. Seldin, R.H. Falk, D.G. Wright.

Critical revision of the article for important intellectual content: M. Skinner, V. Sanchorawala, D.C. Seldin, L.M. Dember, J.L. Berk, C. O'Hara, J. Wiesman, K. Quillen, D.G. Wright.

Final approval of the article: M. Skinner, V. Sanchorawala, D.C. Seldin, L.M. Dember, R.H. Falk, J.L. Berk, J.J. Anderson, C. O'Hara, K.T. Finn, C.A. Libbey, J. Wiesman, K. Quillen, N. Swan, D.G. Wright.

Provision of study materials or patients: M. Skinner, V. Sanchorawala, D.C. Seldin, R.H. Falk, J.L. Berk, K. Quillen, N. Swan, D.G. Wright.

Statistical expertise: J.J. Anderson.

Obtaining of funding: M. Skinner.

Administrative, technical, or logistic support: M. Skinner, K.T. Finn, K. Quillen, N. Swan, D.G. Wright.

Collection and assembly of data: M. Skinner, C.A. Libbey, J. Wiesman, K. Quillen, N. Swan, D.G. Wright.


Ann Intern Med. 2004;140(2):85-93. doi:10.7326/0003-4819-140-2-200401200-00008
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This study examined 701 consecutive patients with AL amyloidosis who were referred to a specialty amyloid clinic during an 8-year period. Of necessity, patients treated with high-dose melphalan and stem-cell transplantation represent a selected sample, yet we found that more than half of the referral sample (394 of 701) were eligible for treatment. Reasons patients did not meet relatively liberal eligibility criteria were most often related to severely impaired major organ function, a finding that was significantly less frequent among patients referred early after diagnosis. The survival, hematologic response, and organ system improvements of the patients who completed treatment with high-dose melphalan and stem-cell transplantation were substantially greater than those reported for any other therapy (811).

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Figures

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Figure 1.
Algorithm for patient selection and treatment with high-dose melphalan and stem-cell transplantation.
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Grahic Jump Location
Figure 2.
Survival curve for all patients (top) and for patients with cardiac versus noncardiac involvement (bottom).P

The top panel shows survival for all 312 patients who initiated treatment with high-dose melphalan and stem-cell transplantation. Median survival is 4.6 years, and vertical lines indicate patients who are still alive. The bottom panel shows survival for 137 of 312 patients treated with high-dose melphalan and stem-cell transplantation who had cardiac involvement and for 175 of 312 patients treated with high-dose melphalan and stem-cell transplantation who did not have cardiac involvement. The median survival for patients with cardiac involvement was 1.6 years, and the median survival for patients who did not have cardiac involvement was 6.4 years ( < 0.001).

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Grahic Jump Location
Figure 3.
Survival curves according to hematologic response at 1 year from treatment.

One hundred eighty-one patients were evaluable for hematologic response at 1 year after treatment. Survival is shown for 73 of 181 patients with a complete hematologic response and for 108 of 181 patients with a noncomplete response. The median survival cannot be determined for the complete response group because 85% of those patients are still alive; the median survival for the noncomplete response group is 5.2 years (p = 0.001).

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Summary for Patients

Improved Treatment of AL Amyloidosis

The summary below is from the full report titled “High-Dose Melphalan and Autologous Stem-Cell Transplantation in Patients with AL Amyloidosis: An 8-Year Study.” It is in the 20 January 2004 issue of Annals of Internal Medicine (volume 140, pages 85-93). The authors are M. Skinner, V. Sanchorawala, D.C. Seldin, L.M. Dember, R.H. Falk, J.L. Berk, J.J. Anderson, C. O'Hara, K.T. Finn, C.A. Libbey, J. Wiesman, K. Quillen, N. Swan, and D.G. Wright.

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