Table 6.

Outcome of high (200 mg/m2)/modified (100–140 mg/m2) dose melphalan followed by peripheral blood stem cell transplantation (PBSCT).*

PatientsTreatedHDM200 mg/m2TRM#ClonalResponse(partial +complete)CompleteResponseOrganResponseOrgan Resp./Clonal Resp.Center,Year, Reference
*Studies reporting 20 or more patients published in year 2000 or later are listed 
#Treatment-related mortality within 100 days from melphalan administration 
§ Organ response was listed according to organs and not to patients (renal 46%, cardiac 25%, liver 50%, neurologic 0%) 
‡ Study performed at the Pavia Amyloidosis Centre and at the National Cancer Institute, Milan, data unpublished. 
†After new selection criteria and prophylactic measures were introduced in January 1999, TRM decreased from 50% (5/10) to 20% (2/10) 
¥Includes consecutive patients from 6 separate trials over 8 years, please note that 122 patients received an intermediate dose of melphalan (100–140 mg/m2
** Hematologic response was evaluated at 1 year: in 39 patients 1 year had not passed since treatment 
Abbreviations: HDM, high-dose melphalan; TRM, treatment-related mortality; NR, not reported 
66 38 9/66 (14%) 33/66 (50%) NR 32/66 (48%) 23/33 (70%) Single center, 20024  
22 14 3/22 (14%) 13/22 (59%) 8/22 (36%) 10/22 (45%) 10/13 (77%) Two-centers, 2004‡ 
20 7/20 (35%)† 56% 28% § NR Single center, 200420  
277¥ 155 36/277 (13%) NR 73/238 (31%)** 80/238 (34%) 48/73 (66%) Single center, 20043  
PatientsTreatedHDM200 mg/m2TRM#ClonalResponse(partial +complete)CompleteResponseOrganResponseOrgan Resp./Clonal Resp.Center,Year, Reference
*Studies reporting 20 or more patients published in year 2000 or later are listed 
#Treatment-related mortality within 100 days from melphalan administration 
§ Organ response was listed according to organs and not to patients (renal 46%, cardiac 25%, liver 50%, neurologic 0%) 
‡ Study performed at the Pavia Amyloidosis Centre and at the National Cancer Institute, Milan, data unpublished. 
†After new selection criteria and prophylactic measures were introduced in January 1999, TRM decreased from 50% (5/10) to 20% (2/10) 
¥Includes consecutive patients from 6 separate trials over 8 years, please note that 122 patients received an intermediate dose of melphalan (100–140 mg/m2
** Hematologic response was evaluated at 1 year: in 39 patients 1 year had not passed since treatment 
Abbreviations: HDM, high-dose melphalan; TRM, treatment-related mortality; NR, not reported 
66 38 9/66 (14%) 33/66 (50%) NR 32/66 (48%) 23/33 (70%) Single center, 20024  
22 14 3/22 (14%) 13/22 (59%) 8/22 (36%) 10/22 (45%) 10/13 (77%) Two-centers, 2004‡ 
20 7/20 (35%)† 56% 28% § NR Single center, 200420  
277¥ 155 36/277 (13%) NR 73/238 (31%)** 80/238 (34%) 48/73 (66%) Single center, 20043  

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