Table 3

Therapeutic studies in Waldenström macroglobulinemia

StudyRegimenNo. of patientsORR, %MS or MRD, mo
Facon et al78  Chlorambucil 128 31 MS, 60 
Dimopoulos et al79  Cladribine 26 85 MRD, 13 
Dhodapkar et al80  Fludarabine 118 38 OS 5 y, 62% 
Gertz et al81  Rituximab 34 35 MRD, 27 
Petrucci et al82  Melphalan, cyclophosphamide, chlorambucil, prednisolone 31 68 EFS, 66 
Case et al83  Carmustine, cyclophosphamide, vincristine, melphalan, prednisolone 33 82 MRD, 43 for CR and 39 for PR 
Dimopoulos and Alexanian84  Cyclophosphamide, vincristine, prednisolone 16 44 MRD, 36 
 CHOP 20 65 MRD, 88 
Leblond et al85  CAP (pretreated with an alkylating agent) 45 11 MRD, 3 and OS, 45 
Hunter et al86  CHOP/rituximab 13 77 90% remain in remission at 9 mo 
Dimopoulos et al87  Dexamethasone, cyclophosphamide, rituximab 34 78 90% progression free at 18 mo 
Annibali et al88  Melphalan, cyclophosphamide, prednisone 72 87 EFS, 47 
Weber et al89  Cladribine, cyclophosphamide, rituximab 27 94 MRD, 60 
 Cladribine, cyclophosphamide 37 84 MRD, 36 
Tam et al90  Fludarabine, cyclophosphamide, rituximab 80 MRD, 39 
 Fludarabine, cyclophosphamide 88 MRD, 38 
Tamburini et al91  Fludarabine, cyclophosphamide 49 78 MRD, 27 
Branagan et al92  Thalidomide, rituximab 23 68 No relapses at 10 mo median 
Hensel et al93  Pentostatin, cyclophosphamide, rituximab 17 90 No patient had relapse at publication time 
StudyRegimenNo. of patientsORR, %MS or MRD, mo
Facon et al78  Chlorambucil 128 31 MS, 60 
Dimopoulos et al79  Cladribine 26 85 MRD, 13 
Dhodapkar et al80  Fludarabine 118 38 OS 5 y, 62% 
Gertz et al81  Rituximab 34 35 MRD, 27 
Petrucci et al82  Melphalan, cyclophosphamide, chlorambucil, prednisolone 31 68 EFS, 66 
Case et al83  Carmustine, cyclophosphamide, vincristine, melphalan, prednisolone 33 82 MRD, 43 for CR and 39 for PR 
Dimopoulos and Alexanian84  Cyclophosphamide, vincristine, prednisolone 16 44 MRD, 36 
 CHOP 20 65 MRD, 88 
Leblond et al85  CAP (pretreated with an alkylating agent) 45 11 MRD, 3 and OS, 45 
Hunter et al86  CHOP/rituximab 13 77 90% remain in remission at 9 mo 
Dimopoulos et al87  Dexamethasone, cyclophosphamide, rituximab 34 78 90% progression free at 18 mo 
Annibali et al88  Melphalan, cyclophosphamide, prednisone 72 87 EFS, 47 
Weber et al89  Cladribine, cyclophosphamide, rituximab 27 94 MRD, 60 
 Cladribine, cyclophosphamide 37 84 MRD, 36 
Tam et al90  Fludarabine, cyclophosphamide, rituximab 80 MRD, 39 
 Fludarabine, cyclophosphamide 88 MRD, 38 
Tamburini et al91  Fludarabine, cyclophosphamide 49 78 MRD, 27 
Branagan et al92  Thalidomide, rituximab 23 68 No relapses at 10 mo median 
Hensel et al93  Pentostatin, cyclophosphamide, rituximab 17 90 No patient had relapse at publication time 

Data from Treon et al.76 

ORR indicates overall response rate; MS, median survival; MRD, median response duration; OS, overall survival; EFS, event-free survival; CR, complete response; PR, partial response; CHOP, cyclophosphamide, doxorubicin, vincristine, prednisone; and CAP, cyclophosphamide, doxorubicin, prednisone.

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