Distribution of clinical features of WM diagnosis and treatment with adjusted ORs and 95% CIs for reporting specific symptoms or signs according to family type
| Characteristic . | Familial WM patients . | Nonfamilial WM patients, n = 28 . | OR† (95% CI) for nonfamilial vs all familial . | ||
|---|---|---|---|---|---|
| Multiple-case WM, n = 57* . | Mixed WM/BCD, n = 46 . | All familial WM, n = 103 . | |||
| Age at WM diagnosis, y | |||||
| Mean (95% CI) | 58.7 (55.3-62.1) | 58.3 (54.4-60.9) | 58.3 (55.9-60.6) | 60.4 (56.5-64.3) | — |
| Median (95% CI) | 58.7 (54.6-62.7) | 59.8 (57.0-62.5) | 59.0 (56.7-61.3) | 62.2 (57.9-66.5) | — |
| Range | 33.5-85.1 | 34.8-84.1 | 33.5-85.1 | 37.3-76.2 | — |
| Prior history of MGUS, no. (%) | 23 (40.4) | 12 (26.1) | 35 (34.0) | 10 (35.7) | 0.98 (0.42-2.30) |
| Occurrence of symptoms and/or signs of disease | |||||
| Ever had symptoms, no. (%) | 44 (77.2) | 43 (97.7) | 87 (86.1) | 25 (89.3) | 1.45 (0.46-4.55) |
| Had symptoms before diagnosis, no. (%) | 29 (51.8) | 29 (64.4) | 58 (57.4) | 18 (66.7) | — |
| Yes: time, symptoms to diagnosis; mean y (range)‡ | 2.2 (0.1-16.8) | 1.4 (0.7-16.0) | 1.6 (0.1-16.8) | 1.4 (0.2-20.2) | — |
| No: time, diagnosis to symptoms; mean y (range)§ | 0.8 (0.0-21.6) | 0.4 (0.0-6.3) | 0.8 (0.0-21.6) | 0.2 (0.0-0.4) | — |
| Ever had signs of WM, no. (%)¶ | 29 (50.9) | 32 (69.6) | 61 (59.2) | 19 (67.8) | 0.70 (0.29-1.67) |
| Reported symptoms, n (%) | |||||
| Fatigue | 29 (50.9) | 36 (80.0) | 65 (63.1) | 16 (57.1) | 0.81 (0.34-1.93) |
| Malaise | 25 (43.9) | 24 (53.3) | 49 (47.6) | 15 (53.6) | 1.27 (0.54-2.97) |
| Paresthesia | 12 (21.1) | 15 (33.3) | 27 (26.2) | 9 (32.1) | 1.42 (0.57-3.54) |
| Dyspnea | 8 (14.0) | 15 (33.3) | 23 (22.3) | 9 (32.1) | 1.61 (0.62-4.20) |
| Visual problems | 5 (8.8) | 4 (8.9) | 9 (8.7) | 6 (21.4) | 2.76 (0.94-8.06) |
| Bruising | 7 (12.3) | 9 (20.0) | 16 (15.5) | 5 (17.8) | 0.96 (0.32-2.91) |
| Joint pain | 5 (14.3) | 11 (24.4) | 16 (19.8) | 4 (14.3) | 0.49 (0.13-1.87) |
| Bleeding | 4 (7.1) | 10 (22.2) | 14 (13.6) | 3 (10.7) | 0.85 (0.22-3.39) |
| Other‖, no. (%) | 28 (49.1) | 23 (50.0) | 51 (49.5) | 11 (39.3) | — |
| Reported signs (%) | |||||
| Anemia or low blood count | 26 (45.6) | 27 (58.7) | 53 (51.5) | 12 (42.8) | 0.71 (0.29-1.71) |
| Enlarged lymph nodes | 7 (12.3) | 7 (15.2) | 14 (13.6) | 2 (7.1) | 0.58 (0.12-2.86) |
| Hepatosplenomegaly | 3 (5.3) | 3 (6.5) | 6 (5.8) | 4 (14.3) | 2.10 (0.37-11.99) |
| Treatment characteristics** | |||||
| Never treated, no. (%) | 16 (32.0) | 4 (8.7) | 20 (20.8) | 6 (21.4) | — |
| Time from diagnosis, mean y (range) | 2.4 (0.0-10.4) | 1.2 (0.6-5.9) | 2.0 (0.0-10.4) | 3.3 (2.2-13.9) | — |
| Treated, no. (%) | 34 (68.0) | 42 (91.3) | 76 (79.2) | 22 (78.6) | — |
| Time, diagnosis to first treatment; mean y (range) | 0.2 (0.0-22.0) | 0.2 (0.0-9.8) | 0.2 (0.0-22.0) | 0.2 (0.0-13.3) | — |
| Cytotoxic, no. (%) | |||||
| Chemotherapy and/or immunotherapy†† | 34 (68.0) | 42 (91.3) | 76 (79.2) | 22 (78.6) | — |
| Plasmapheresis | 7 (14.3) | 11 (23.9) | 18 (18.9) | 4 (14.3) | — |
| Supportive‡‡ | 10 (18.9) | 6 (14.0) | 16 (16.7) | 3 (11.5) | — |
| Characteristic . | Familial WM patients . | Nonfamilial WM patients, n = 28 . | OR† (95% CI) for nonfamilial vs all familial . | ||
|---|---|---|---|---|---|
| Multiple-case WM, n = 57* . | Mixed WM/BCD, n = 46 . | All familial WM, n = 103 . | |||
| Age at WM diagnosis, y | |||||
| Mean (95% CI) | 58.7 (55.3-62.1) | 58.3 (54.4-60.9) | 58.3 (55.9-60.6) | 60.4 (56.5-64.3) | — |
| Median (95% CI) | 58.7 (54.6-62.7) | 59.8 (57.0-62.5) | 59.0 (56.7-61.3) | 62.2 (57.9-66.5) | — |
| Range | 33.5-85.1 | 34.8-84.1 | 33.5-85.1 | 37.3-76.2 | — |
| Prior history of MGUS, no. (%) | 23 (40.4) | 12 (26.1) | 35 (34.0) | 10 (35.7) | 0.98 (0.42-2.30) |
| Occurrence of symptoms and/or signs of disease | |||||
| Ever had symptoms, no. (%) | 44 (77.2) | 43 (97.7) | 87 (86.1) | 25 (89.3) | 1.45 (0.46-4.55) |
| Had symptoms before diagnosis, no. (%) | 29 (51.8) | 29 (64.4) | 58 (57.4) | 18 (66.7) | — |
| Yes: time, symptoms to diagnosis; mean y (range)‡ | 2.2 (0.1-16.8) | 1.4 (0.7-16.0) | 1.6 (0.1-16.8) | 1.4 (0.2-20.2) | — |
| No: time, diagnosis to symptoms; mean y (range)§ | 0.8 (0.0-21.6) | 0.4 (0.0-6.3) | 0.8 (0.0-21.6) | 0.2 (0.0-0.4) | — |
| Ever had signs of WM, no. (%)¶ | 29 (50.9) | 32 (69.6) | 61 (59.2) | 19 (67.8) | 0.70 (0.29-1.67) |
| Reported symptoms, n (%) | |||||
| Fatigue | 29 (50.9) | 36 (80.0) | 65 (63.1) | 16 (57.1) | 0.81 (0.34-1.93) |
| Malaise | 25 (43.9) | 24 (53.3) | 49 (47.6) | 15 (53.6) | 1.27 (0.54-2.97) |
| Paresthesia | 12 (21.1) | 15 (33.3) | 27 (26.2) | 9 (32.1) | 1.42 (0.57-3.54) |
| Dyspnea | 8 (14.0) | 15 (33.3) | 23 (22.3) | 9 (32.1) | 1.61 (0.62-4.20) |
| Visual problems | 5 (8.8) | 4 (8.9) | 9 (8.7) | 6 (21.4) | 2.76 (0.94-8.06) |
| Bruising | 7 (12.3) | 9 (20.0) | 16 (15.5) | 5 (17.8) | 0.96 (0.32-2.91) |
| Joint pain | 5 (14.3) | 11 (24.4) | 16 (19.8) | 4 (14.3) | 0.49 (0.13-1.87) |
| Bleeding | 4 (7.1) | 10 (22.2) | 14 (13.6) | 3 (10.7) | 0.85 (0.22-3.39) |
| Other‖, no. (%) | 28 (49.1) | 23 (50.0) | 51 (49.5) | 11 (39.3) | — |
| Reported signs (%) | |||||
| Anemia or low blood count | 26 (45.6) | 27 (58.7) | 53 (51.5) | 12 (42.8) | 0.71 (0.29-1.71) |
| Enlarged lymph nodes | 7 (12.3) | 7 (15.2) | 14 (13.6) | 2 (7.1) | 0.58 (0.12-2.86) |
| Hepatosplenomegaly | 3 (5.3) | 3 (6.5) | 6 (5.8) | 4 (14.3) | 2.10 (0.37-11.99) |
| Treatment characteristics** | |||||
| Never treated, no. (%) | 16 (32.0) | 4 (8.7) | 20 (20.8) | 6 (21.4) | — |
| Time from diagnosis, mean y (range) | 2.4 (0.0-10.4) | 1.2 (0.6-5.9) | 2.0 (0.0-10.4) | 3.3 (2.2-13.9) | — |
| Treated, no. (%) | 34 (68.0) | 42 (91.3) | 76 (79.2) | 22 (78.6) | — |
| Time, diagnosis to first treatment; mean y (range) | 0.2 (0.0-22.0) | 0.2 (0.0-9.8) | 0.2 (0.0-22.0) | 0.2 (0.0-13.3) | — |
| Cytotoxic, no. (%) | |||||
| Chemotherapy and/or immunotherapy†† | 34 (68.0) | 42 (91.3) | 76 (79.2) | 22 (78.6) | — |
| Plasmapheresis | 7 (14.3) | 11 (23.9) | 18 (18.9) | 4 (14.3) | — |
| Supportive‡‡ | 10 (18.9) | 6 (14.0) | 16 (16.7) | 3 (11.5) | — |
WM indicates Waldenström macroglobulinemia; OR, odds ratio; CI, confidence interval; BCD, B-cell disorder; and —, no relevant data.
Numbers and percentages may not sum to total because of missing data and rounding, respectively. Percentages are based on nonmissing values.
Adjusted for age and sex.
Includes participants who reported experiencing symptoms before a WM diagnosis.
Includes participants who reported experiencing symptoms only after a WM diagnosis.
Signs include anemia or cytopenia, lymphadenopathy, or hepatosplenomegaly.
Other reported symptoms were defined as symptoms reported by fewer than 20% of participants in each category. Other symptoms included fever, night sweats, weight loss, headaches, Raynaud phenomenon, gastrointestinal symptoms, confusion, inability to concentrate, memory changes, myalgia, rash, lightheadedness/syncope, edema, and palpitations.
Treatment data for multiple-case WM patients exclude 7 patients who were diagnosed during the study after questionnaire completion.
Includes one patient from a multiple-case family who had a bone marrow transplantation and one patient from a mixed WM/BCD family who had a stem cell transplantation.
Supportive treatment includes red blood cell and/or platelet transfusion.