Tandem intermediate dose melphalan supported by autologous progenitor cells in the management of primary amyloidosis

Rafat Abonour, Merrill D. Benson, Michael J. Robertson, Hromas A. Robert, Cornetta Kenneth

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1 Scopus citations


Primary amyloidosis, an uncommon plasma cell disorder that leads to organ dysfunction and short survival, poses a major therapeutic challenge. Oral melphalan and prednisone has a response rate of only 18%. High dose melphalan (200 mg/m2) supported by G-CSF mobilized peripheral blood progenitor cells (G-PBPC) has been used for the treatment of primary amyloidosis with encouraging results. However, treatment-related mortality and morbidity were substantial. In order to minimize toxicity and improve efficacy, we explored tandem intermediate dose melphalan (140 mg/m2) administered 2 month apart and supported by G-PBPC (10 H/kg/d). Four patients were enrolled on this study between December 1996 and March 1997. All had nephrotic syndrome, 1 had definite and 1 had probable cardiac involvement. Leukaphersis was completed in single (2 subjects) and 2 (2 subjects) sessions. CD34 AGC PRBC PLT CD34 1 AGC PRBC PLT 13.7 8 4 2 14.7 8 0 1 2.9 10 0 2 3.4 10 0 0 2.8 10 ND ND NA NA NA NA 4 9 2 1 4 10 2 0 (CD34+ cell dose for first transplant (x 106/kg). AGC= days to AGC>1000. 1 data for the second transplant. Number of red blood cells and platelet units infused =RBC and PLT infused. NA=not available, ND=not determined. Data in each row correspond to subject 1 through 4) All 4 patients developed neutropenic fever. Grade l-II mucositis was seen during 5 cycles and grade III in one cycle. Subject 2 (with definite cardiac involvement) developed sudden cardiac death on day 10 after the second cycle of high-dose chemotherapy. Subject 3 developed sepsis and gastrointestinal bleeding following the first cycle, leading to renal failure requiring hemodialysis. He died 22 months later from complications related to peritoneal dialysis. Subject 1 continues to show resolution of his nephrotic range proteinuria 44 months following transplantation and enjoys good performance status. Subject 4 has stable proteinuria with 41 months of follow up with good health. Tandem intermediate dose melphalan supported by autologous PBPC warrants further investigation in primary amyloidosis.

Original languageEnglish (US)
Pages (from-to)367b
Issue number11 PART II
StatePublished - Dec 1 2000
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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