Autologous stem cell transplantation for primary systemic amyloidosis

DOI PDF 被引用文献25件
  • Raymond L. Comenzo
    From the Hematology Service, Department of Medicine, Memorial Sloan-Kettering Cancer Center, New York, NY; and the Division of Hematology, Mayo Clinic, Rochester, MN.
  • 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.

抄録

<jats:p>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.</jats:p>

収録刊行物

  • Blood

    Blood 99 (12), 4276-4282, 2002-06-15

    American Society of Hematology

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