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Blood, 15 June 2002, Vol. 99, No. 12, pp. 4276-4282
REVIEW ARTICLE
Autologous stem cell transplantation for primary systemic
amyloidosis
Raymond L. Comenzo and
Morie A. Gertz
From the Hematology Service, Department of Medicine,
Memorial Sloan-Kettering Cancer Center, New York, NY; and the Division
of Hematology, Mayo Clinic, Rochester, MN.
High-dose melphalan with autologous blood stem cell transplantation
(SCT) can reverse the disease process in selected patients with primary
systemic amyloidosis (AL); however, SCT for AL remains controversial
because of the treatment-related mortality in patients with cardiac and
multisystem organ involvement. In this review, we briefly discuss
recent advances in AL, such as the free light-chain assay and the role
of immunoglobulin light-chain variable region germline genes in the
disease, and then we discuss the current status of SCT for AL with
emphases on patient selection, approaches to stem cell
mobilization, and peri-SCT management. It is clear that patients with
AL who have advanced amyloid cardiomyopathy or more than 2 major
viscera involved with disease are poor candidates for SCT. Therefore,
the importance of patient selection cannot be overemphasized, and
patients with 1 or 2 involved organs or with early cardiac involvement
are usually appropriate candidates for SCT. Because the toxicity of
melphalan is dose-related and survival with AL may be
age-related, patient age and the extent of organ involvement can
provide a basis for patient stratification. We discuss such a
risk-adapted approach to melphalan dosing in detail and conclude with a
brief overview of current research using SCT to treat patients with AL.
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