BACKGROUND: AL amyloidosis is a fatal disease resulting from tissue deposition of amyloid fibrils derived from monoclonal immunoglobulin light chains. Treatment with oral chemotherapy is minimally effective. OBJECTIVE: To test survival and organ response in a large sample of patients treated with high-dose intravenous melphalan (100 to 200 mg/m2) and autologous blood stem-cell transplantation. DESIGN: 8-year longitudinal analysis of clinical effectiveness. SETTING: University-affiliated specialty referral clinic. PATIENTS: 701 consecutive new patients with AL amyloidosis. INTERVENTION: High-dose chemotherapy and autologous stem-cell transplantation for patients who met eligibility requirements based on organ involvement and clinical status. MEASUREMENTS: Survival analysis of all patients evaluated and a detailed analysis of treatment outcome in the subgroup that received high-dose melphalan and stem-cell transplantation. RESULTS: Among 701 patients with AL amyloidosis, 394 (56%) were eligible for high-dose melphalan and stem-cell transplantation; 82 did not proceed with treatment because of patient choice or disease progression. Median survival of the 312 patients who initiated treatment was 4.6 years. A complete hematologic response, defined as no evidence of an underlying plasma cell dyscrasia 1 year after treatment, was achieved in 40% of patients and was associated with prolonged survival. Statistically significant improvements occurred in end-organ disease and were greater in patients with a complete hematologic response. Mortality rate within 100 days of treatment with high-dose melphalan and stem-cell transplantation was 13%; patients with cardiomyopathy had the highest mortality rates. CONCLUSIONS: Treatment of selected patients with AL amyloidosis by using high-dose melphalan and stem-cell transplantation resulted in hematologic remission, improved 5-year survival, and reversal of amyloid-related disease in a substantial proportion.
BACKGROUND:AL amyloidosis is a fatal disease resulting from tissue deposition of amyloid fibrils derived from monoclonal immunoglobulin light chains. Treatment with oral chemotherapy is minimally effective. OBJECTIVE: To test survival and organ response in a large sample of patients treated with high-dose intravenous melphalan (100 to 200 mg/m2) and autologous blood stem-cell transplantation. DESIGN: 8-year longitudinal analysis of clinical effectiveness. SETTING: University-affiliated specialty referral clinic. PATIENTS: 701 consecutive new patients with AL amyloidosis. INTERVENTION: High-dose chemotherapy and autologous stem-cell transplantation for patients who met eligibility requirements based on organ involvement and clinical status. MEASUREMENTS: Survival analysis of all patients evaluated and a detailed analysis of treatment outcome in the subgroup that received high-dose melphalan and stem-cell transplantation. RESULTS: Among 701 patients with AL amyloidosis, 394 (56%) were eligible for high-dose melphalan and stem-cell transplantation; 82 did not proceed with treatment because of patient choice or disease progression. Median survival of the 312 patients who initiated treatment was 4.6 years. A complete hematologic response, defined as no evidence of an underlying plasma cell dyscrasia 1 year after treatment, was achieved in 40% of patients and was associated with prolonged survival. Statistically significant improvements occurred in end-organ disease and were greater in patients with a complete hematologic response. Mortality rate within 100 days of treatment with high-dose melphalan and stem-cell transplantation was 13%; patients with cardiomyopathy had the highest mortality rates. CONCLUSIONS: Treatment of selected patients with AL amyloidosis by using high-dose melphalan and stem-cell transplantation resulted in hematologic remission, improved 5-year survival, and reversal of amyloid-related disease in a substantial proportion.
Authors: Sumit Madan; Shaji K Kumar; Angela Dispenzieri; Martha Q Lacy; Suzanne R Hayman; Francis K Buadi; David Dingli; S Vincent Rajkumar; William J Hogan; Nelson Leung; Martha Grogan; Morie A Gertz Journal: Blood Date: 2011-12-06 Impact factor: 22.113
Authors: Martin Tam; David C Seldin; Benjamin M Forbes; Lawreen H Connors; Martha Skinner; Betul Oran; Karen Quillen; Vaishali Sanchorawala Journal: Amyloid Date: 2009 Impact factor: 7.141
Authors: D C Seldin; N Andrea; I Berenbaum; J L Berk; L Connors; L M Dember; G Doros; S Fennessey; K Finn; S Girnius; A Lerner; C Libbey; H K Meier-Ewert; R O'Connell; C O'Hara; K Quillen; F L Ruberg; F Sam; A Segal; A Shelton; M Skinner; J M Sloan; J F Wiesman; V Sanchorawala Journal: Amyloid Date: 2011-06 Impact factor: 7.141
Authors: Abdullah S Al Saleh; M Hasib Sidiqi; Eli Muchtar; Angela Dispenzieri; Francis K Buadi; David Dingli; Martha Q Lacy; Rahma M Warsame; Wilson I Gonsalves; Taxiarchis V Kourelis; William J Hogan; Suzanne R Hayman; Prashant Kapoor; Shaji K Kumar; Morie A Gertz Journal: Biol Blood Marrow Transplant Date: 2019-05-02 Impact factor: 5.742