Baseline demographic and disease characteristics
| Characteristic . | Treatment-naïve (n = 5) . | Relapsed/refractory (n = 23) . | Overall (N = 28) . |
|---|---|---|---|
| Median age (min, max), y | 81 (71, 87) | 71 (39, 87) | 72 (39, 87) |
| >75 y, n (%) | 4 (80) | 8 (35) | 12 (43) |
| Male, n (%) | 3 (60) | 11 (48) | 14 (50) |
| ECOG PS, n (%) | |||
| 0/1 | 4 (80) | 20 (87) | 24 (86) |
| 2 | 1 (20) | 3 (13) | 4 (14) |
| Prognostic category at study entry, n (%)* | |||
| Low | 0 | 5 (22) | 5 (18) |
| Intermediate | 3 (60) | 8 (35) | 11 (39) |
| High | 2 (40) | 10 (44) | 12 (43) |
| Median time from initial diagnosis (min, max), y | 1.5 (0.1, 12.4) | 4.0 (0.5, 20.3) | 3.7 (0.1, 20.3) |
| Median prior lines of therapy, n (min, max) | 0 (0, 0) | 1 (1, 5) | 1 (0, 5) |
| 0, n (%) | 5 (100) | 0 | 5 (18) |
| 1-3, n (%) | 0 | 20 (87) | 20 (71) |
| >3, n (%) | 0 | 3 (13) | 3 (11) |
| Prior stem cell transplant, n (%) | 0 | 1 (4) | 1 (4) |
| Median IgM (min, max), g/L† | 36.1 (13.8, 73.4) | 28.5 (5.6, 69.9) | 28.5 (5.6, 73.4) |
| ≥40 g/L, n (%) | 2 (40) | 6 (26) | 8 (29) |
| <40 g/L, n (%) | 2 (40) | 17 (74) | 19 (68) |
| Missing, n (%) | 1 (20) | 0 | 1 (4) |
| Median β2-microglobulin, mg/L (min, max) | 3.7 (2.2, 8.1) | 3.8 (1.7, 13.7) | 3.8 (1.7, 13.7) |
| >3 mg/L, n (%) | 3 (60) | 17 (74) | 20 (71) |
| MYD88/CXCR4 genotype, n (%) | |||
| MYD88WT/ CXCR4WT | 5 (100) | 18 (78) | 23 (82) |
| MYD88WT/ CXCR4WHIM | 0 | 1 (4) | 1 (4) |
| MYD88WT/ CXCR4UNK | 0 | 2 (9) | 2 (7) |
| MYD88UNK/ CXCR4UNK | 0 | 2 (9) | 2 (7) |
| Bone marrow involvement, n (%) | 4 (80) | 22 (96) | 26 (93) |
| Median percent tumor cells (min, max) | 13 (0, 70) | 25 (0, 90) | 23 (0, 90) |
| Extramedullary disease, n (%)‡ | 4 (80) | 17 (74) | 21 (75) |
| Lymphadenopathy | 4 (80) | 16 (70) | 20 (71) |
| Splenomegaly | 1 (20) | 5 (22) | 6 (21) |
| Other | 0 | 0 | 0 |
| Peripheral blood cytopenias | |||
| Hemoglobin ≤110 g/L, n (%) | 3 (60) | 12 (52) | 15 (54) |
| Platelet count ≤100 × 109/L, n (%) | 0 | 3 (13) | 3 (11) |
| Absolute neutrophil count ≤1.5 × 109/L, n (%) | 1 (20) | 0 | 1 (4) |
| Characteristic . | Treatment-naïve (n = 5) . | Relapsed/refractory (n = 23) . | Overall (N = 28) . |
|---|---|---|---|
| Median age (min, max), y | 81 (71, 87) | 71 (39, 87) | 72 (39, 87) |
| >75 y, n (%) | 4 (80) | 8 (35) | 12 (43) |
| Male, n (%) | 3 (60) | 11 (48) | 14 (50) |
| ECOG PS, n (%) | |||
| 0/1 | 4 (80) | 20 (87) | 24 (86) |
| 2 | 1 (20) | 3 (13) | 4 (14) |
| Prognostic category at study entry, n (%)* | |||
| Low | 0 | 5 (22) | 5 (18) |
| Intermediate | 3 (60) | 8 (35) | 11 (39) |
| High | 2 (40) | 10 (44) | 12 (43) |
| Median time from initial diagnosis (min, max), y | 1.5 (0.1, 12.4) | 4.0 (0.5, 20.3) | 3.7 (0.1, 20.3) |
| Median prior lines of therapy, n (min, max) | 0 (0, 0) | 1 (1, 5) | 1 (0, 5) |
| 0, n (%) | 5 (100) | 0 | 5 (18) |
| 1-3, n (%) | 0 | 20 (87) | 20 (71) |
| >3, n (%) | 0 | 3 (13) | 3 (11) |
| Prior stem cell transplant, n (%) | 0 | 1 (4) | 1 (4) |
| Median IgM (min, max), g/L† | 36.1 (13.8, 73.4) | 28.5 (5.6, 69.9) | 28.5 (5.6, 73.4) |
| ≥40 g/L, n (%) | 2 (40) | 6 (26) | 8 (29) |
| <40 g/L, n (%) | 2 (40) | 17 (74) | 19 (68) |
| Missing, n (%) | 1 (20) | 0 | 1 (4) |
| Median β2-microglobulin, mg/L (min, max) | 3.7 (2.2, 8.1) | 3.8 (1.7, 13.7) | 3.8 (1.7, 13.7) |
| >3 mg/L, n (%) | 3 (60) | 17 (74) | 20 (71) |
| MYD88/CXCR4 genotype, n (%) | |||
| MYD88WT/ CXCR4WT | 5 (100) | 18 (78) | 23 (82) |
| MYD88WT/ CXCR4WHIM | 0 | 1 (4) | 1 (4) |
| MYD88WT/ CXCR4UNK | 0 | 2 (9) | 2 (7) |
| MYD88UNK/ CXCR4UNK | 0 | 2 (9) | 2 (7) |
| Bone marrow involvement, n (%) | 4 (80) | 22 (96) | 26 (93) |
| Median percent tumor cells (min, max) | 13 (0, 70) | 25 (0, 90) | 23 (0, 90) |
| Extramedullary disease, n (%)‡ | 4 (80) | 17 (74) | 21 (75) |
| Lymphadenopathy | 4 (80) | 16 (70) | 20 (71) |
| Splenomegaly | 1 (20) | 5 (22) | 6 (21) |
| Other | 0 | 0 | 0 |
| Peripheral blood cytopenias | |||
| Hemoglobin ≤110 g/L, n (%) | 3 (60) | 12 (52) | 15 (54) |
| Platelet count ≤100 × 109/L, n (%) | 0 | 3 (13) | 3 (11) |
| Absolute neutrophil count ≤1.5 × 109/L, n (%) | 1 (20) | 0 | 1 (4) |
Percentages may not total 100 due to rounding.
CXCR4, chemokine receptor 4; ECOG PS, Eastern Cooperative Oncology Group performance status; max, maximum; min, minimum; WHIM, warts, hypogammaglobulinemia, infections, and myelokathexis; WT, wild-type.
Per Morel et al,30 patients were assigned 1 point for each of the following baseline characteristics: age, >65 y; hemoglobin ≤11.5 g/dL; platelet count ≤100 × 109/L; β2 microglobulin level >3 mg/L; and M paraprotein levels >7.0 g/dL. Patients with a score of 0 or 1 (excepting age) were assigned to the low-risk category, those >65 y or with a score of 2 were assigned to the intermediate-risk category, and those with a score ≥3 were assigned to the high-risk category. M-paraprotein levels were quantitated by serum protein electrophoresis.
Central laboratory nephelometric assessments; n = 4 for the TN subset.
Based on imaging studies, as assessment by independent review. Lymphadenopathy was defined as the presence of lymph nodes with a long axis of more than 1.5 cm. Splenomegaly was defined as a spleen depth (cranial to caudal) of more than 13 cm.