0
Sign In  | Subscribe
20 January 2004, Vol 140, No. 2>
Articles | 20 January 2004

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
Text Size: A A A

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 (8 - 11).

Buy This Article

to gain full access to the content and tools.

Want to Subscribe?

Learn more about subscription options

Register Now for a free account

First Page Preview

View Large
First page PDF preview

Figures

Grahic Jump Location
Figure 1.
Algorithm for patient selection and treatment with high-dose melphalan and stem-cell transplantation.
Grahic Jump Location
+Image not available.
View Large  |  Save Figure  |  Download Slide (.ppt)  |  View in Article Context
Image not available.
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).

Grahic Jump Location
+Image not available.
View Large  |  Save Figure  |  Download Slide (.ppt)  |  View in Article Context
Image not available.
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).

Grahic Jump Location
+Image not available.
View Large  |  Save Figure  |  Download Slide (.ppt)  |  View in Article Context
Image not available.

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).
Compression-Only CPR: Pushing the Science Forward
Cone
AAM 2010;304:1493-1495.
All you need to read in the other general jounals
BMJ 2010;341:c5893-c1494.

Comments

Submit a Comment
Submit a Comment

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.

Read More...

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 This Article

to gain full access to the content and tools.

Want to Subscribe?

Learn more about subscription options

Register Now for a free account
Advertisement

Related Content

Customize your page view by dragging & repositioning the boxes below.

Topic Collections
PubMed Articles
[Heart transplantation and the subsequent treatment of AL amyloidosis].
Vnitr?ní lékar?ství Issue date: 2013 Feb
Multiple myeloma: an update.
Oman medical journal Issue date: 2013 Jan

Annals of Internal Medicine

Other Resources

Copyright © 2013 The American College of Physicians. All Rights Reserved.
Print ISSN: 0003-4819 | Online ISSN: 1539-3704

Buy This Article

to gain full access to the content and tools.

Want to Subscribe?

Learn more about subscription options

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