Clinical and hematological features of patients with ES enrolled before TPO-RA treatment
| Patients . | . |
|---|---|
| Study group, n | 29 |
| Median age, y (range) | 61 (24-88) |
| Male, n (%) | 19 (66) |
| Female, n (%) | 10 (34) |
| ES type, n (%) | |
| AIHA + ITP | 25 (86) |
| AIHA + ITP + AIN | 2 (7) |
| ITP + AIN | 2 (7) |
| Presenting cytopenia, n (%) | |
| ITP | 18 (62) |
| AIHA | 4 (14) |
| Concomitant | 7 (6 ITP+AIHA; 1 AIN+AIHA) |
| Anti-PLT autoantibodies, n (%) | 10/22 (45) |
| DAT positive for IgG, n (%) | 17/27 (63) |
| DAT positive for IgG+C low titer, n (%) | 9/27 (33) |
| DAT positive for C, n (%) | 1/27 (4) |
| Associated conditions, n (%) | 8 (28) |
| Lymphoproliferative syndromes | 2 (25) |
| Primary immunodeficiencies | 2 (25) |
| Antiphospholipid syndrome | 2 (25) |
| Connective tissue disease | 1 (12.5) |
| Hematopoietic stem cell transplant* | 1 (12.5) |
| BM evaluation before TPO-RA, n (%) | 25 (86) |
| Median cellularity, % (range) | 40 (18-85) |
| Hypocellular/normocellular to hypercellular, n (%) | 5 (31)/20 (69) |
| Increased MGKs, n (%) | 22 (88) |
| Dysmegakaryopoiesis, n (%) | 10 (34) |
| Reticulin fibrosis, n (%) | 6 (21) |
| Median lymphoid infiltrate (n = 17), % (range) | 9 (0-35) |
| B cell/T cell/mixed, n (%) | 2 (12)/9(53)/6(35) |
| Abnormal cytogenetics, n (%) | 3 (14) delY; t(X;20); t(1;3) |
| Previous therapy lines for ITP, median (range) | 6 (1-8) |
| Steroids with or without IVIG, n (%); ORR, n (%) | 28 (96); 17 (61) |
| IVIG, n (%); ORR, n (%) | 23 (79); 0 (0) |
| Rituximab, n (%); ORR, n (%) | 13 (45); 9 (69) |
| Cytotoxic immunosuppressors, n (%); ORR, n (%) | 9 (31); 5 (55) |
| Splenectomy, n (%); ORR, n (%) | 7 (24); 3 (43) |
| Danazol, n (%); ORR, n (%) | 3 (10); 2 (66) |
| Patients . | . |
|---|---|
| Study group, n | 29 |
| Median age, y (range) | 61 (24-88) |
| Male, n (%) | 19 (66) |
| Female, n (%) | 10 (34) |
| ES type, n (%) | |
| AIHA + ITP | 25 (86) |
| AIHA + ITP + AIN | 2 (7) |
| ITP + AIN | 2 (7) |
| Presenting cytopenia, n (%) | |
| ITP | 18 (62) |
| AIHA | 4 (14) |
| Concomitant | 7 (6 ITP+AIHA; 1 AIN+AIHA) |
| Anti-PLT autoantibodies, n (%) | 10/22 (45) |
| DAT positive for IgG, n (%) | 17/27 (63) |
| DAT positive for IgG+C low titer, n (%) | 9/27 (33) |
| DAT positive for C, n (%) | 1/27 (4) |
| Associated conditions, n (%) | 8 (28) |
| Lymphoproliferative syndromes | 2 (25) |
| Primary immunodeficiencies | 2 (25) |
| Antiphospholipid syndrome | 2 (25) |
| Connective tissue disease | 1 (12.5) |
| Hematopoietic stem cell transplant* | 1 (12.5) |
| BM evaluation before TPO-RA, n (%) | 25 (86) |
| Median cellularity, % (range) | 40 (18-85) |
| Hypocellular/normocellular to hypercellular, n (%) | 5 (31)/20 (69) |
| Increased MGKs, n (%) | 22 (88) |
| Dysmegakaryopoiesis, n (%) | 10 (34) |
| Reticulin fibrosis, n (%) | 6 (21) |
| Median lymphoid infiltrate (n = 17), % (range) | 9 (0-35) |
| B cell/T cell/mixed, n (%) | 2 (12)/9(53)/6(35) |
| Abnormal cytogenetics, n (%) | 3 (14) delY; t(X;20); t(1;3) |
| Previous therapy lines for ITP, median (range) | 6 (1-8) |
| Steroids with or without IVIG, n (%); ORR, n (%) | 28 (96); 17 (61) |
| IVIG, n (%); ORR, n (%) | 23 (79); 0 (0) |
| Rituximab, n (%); ORR, n (%) | 13 (45); 9 (69) |
| Cytotoxic immunosuppressors, n (%); ORR, n (%) | 9 (31); 5 (55) |
| Splenectomy, n (%); ORR, n (%) | 7 (24); 3 (43) |
| Danazol, n (%); ORR, n (%) | 3 (10); 2 (66) |
In one of the 2 patients with lymphoproliferative syndrome.
BM bone marrow; C, complement; DAT direct antiglobulin test; MGK megakaryocyte; ORR, overall response rate.