Characteristics and treatment of AYAs with chronic primary ITP
| Patient characteristics . | Initial . | 6-mo FU . | 12-mo FU . | 24-mo FU . | 36-mo FU . | 48-mo FU . |
|---|---|---|---|---|---|---|
| No. of patients at FU∗ | 427 | 427 | 425 | 375 | 327 | 250 |
| Female (%) | 273 (64) | 273 (64) | 272 (64) | 240 (64) | 207 (63) | 155 (62) |
| Mean age (SD) | 14 (2.7) | 15 (2.7) | 16 (2.6) | 17 (2.3) | 18 (2.4) | |
| Platelet count (×109/L) at FU, median (IQR) | 15 (7-35) | 45 (21-79) | 51 (26-81) | 68 (30-115) | 76 (35-143) | 87 (43-169) |
| No. of patients with platelet count (%) | ||||||
| <20 × 109/L | 237 (56) | 88 (21) | 79 (19) | 57 (15) | 36 (11) | 18 (7) |
| 20 × 109 to 49 × 109/L | 107 (25) | 118 (28) | 110 (26) | 75 (20) | 58 (18) | 33 (13) |
| 50 × 109 to 99 × 109/L | 62 (15) | 105 (25) | 138 (32) | 103 (27) | 71 (22) | 56 (22) |
| >100 × 109/L | - | 72 (17) | 65 (15) | 104 (28) | 111 (34) | 82 (33) |
| No data on platelet count | 21 (5) | 44 (10) | 33 (8) | 36 (10) | 51 (16) | 61 (24) |
| Bleeding in the last FU period, yes (%)† | 325 (76) | 254 (59) | 215 (51) | 185 (49) | 132 (40) | 74 (30) |
| No (%)† | 74 (17) | 135 (32) | 157 (37) | 148 (39) | 140 (43) | 117 (47) |
| No data (%)† | 28 (7) | 38 (9) | 53 (12) | 42 (11) | 55 (17) | 59 (24) |
| Bleeding location‡ (% of patients with bleeding) | ||||||
| Skin | 267 (82) | 207 (81) | 168 (78) | 142 (77) | 93 (70) | 52 (70) |
| Oral | 94 (29) | 76 (30) | 53 (25) | 45 (24) | 32 (24) | 22 (30) |
| Epistaxis | 99 (30) | 70 (28) | 57 (27) | 60 (32) | 36 (27) | 18 (24) |
| Gynecological (% of women with bleeding) | 73/209 (35) | 69/171 (40) | 50/144 (35) | 59/124 (48) | 43/90 (48) | 22/52 (42) |
| Gastrointestinal and/or hematuria | 10 (3) | 12 (5) | 10 (5) | 7 (4) | 5 (4) | 7 (10) |
| ICH | 3 (1) | 3 (1) | - | 2 (1) | - | - |
| Muscle and/or joint | 4 (1) | 4 (2) | 4 (2) | 2 (1) | - | - |
| Total wet bleeding§ | 193 (59) | 156 (61) | 124 (58) | 122 (66) | 87 (66) | 49 (66) |
| No data on bleeding site | 3 | 7 | 2 | 2 | 1 | - |
| Platelet count (×109/L) at FU, median (IQR) for patients with | ||||||
| Any bleeding | 11 (6-24) | 38 (16-70) | 38 (19-65) | 45 (22-95) | 42 (20-114) | 55 (23-140) |
| Only cutaneous bleeding | 16 (7-27) | 41 (21-75) | 40 (19-65) | 52 (28-98) | 55 (25-94) | 77 (41-193) |
| Wet or internal bleeding | 10 (5-21) | 32 (14-59) | 38 (19-66) | 41 (19-95) | 40 (18-149) | 43 (19-108) |
| No bleeding | 40 (20-65) | 58 (35-94) | 68 (45-97) | 77 (52-133) | 103 (60-172) | 114 (70-179) |
| Drug treatment in the last FU period, yes (%)† | 252 (59) | 254 (59) | 210 (49) | 177 (47) | 129 (39) | 85 (34) |
| No (watch and wait) (%)† | 170 (40) | 165 (39) | 206 (48) | 192 (51) | 191 (58) | 158 (63) |
| No data (%)† | 5 (1) | 8 (2) | 9 (2) | 6 (2) | 7 (2) | 7 (3) |
| Drug used (% of treated patients) | ||||||
| Corticosteroid | 129 (51%) | 188 (74%) | 127 (60%) | 84 (47%) | 40 (31%) | 31 (36%) |
| IV immunoglobulin and/or anti-D | 163 (65%) | 141 (56%) | 80 (38%) | 71 (40%) | 51 (40%) | 34 (40%) |
| Second line and/or third line | 10 (4%) | 59 (23%) | 76 (36%) | 95 (54%) | 79 (61%) | 55 (65%) |
| No data on drug used | 0 | 6 | 3 | 2 | 0 | 1 |
| Type of second-/third-line drugs | ||||||
| Hydroxychloroquine|| | 3 | 18 | 26 | 30 | 20 | 14 |
| TPO-RA | 2 | 17 | 17 | 20 | 22 | 9 |
| Azathioprine | - | 8 | 12 | 15 | 14 | 11 |
| Anti-CD20 (rituximab) | - | 5 | 9 | 17 | 12 | 8 |
| MMF | - | 4 | 6 | 4 | 7 | 6 |
| Vinka alkaloids | - | 5 | 4 | 3 | 1 | 1 |
| Tacrolimus/sirolimus | - | 3 | - | 1 | 1 | 1 |
| Other¶ | 6 | 14 | 14 | 20 | 12 | 10 |
| New splenectomy at each FU (not cumulative) | 0 | 3 | 4 | 16 | 11 | 13 |
| Platelet transfusion | 16 | 12 | 5 | 5 | 1 | 3 |
| Red blood cell transfusion total, (% represent proportion of female) | 16 (100%) | 5 (60%) | 4 (100%) | 5 (60%) | 2 (50%) | 2 (100%) |
| Patient characteristics . | Initial . | 6-mo FU . | 12-mo FU . | 24-mo FU . | 36-mo FU . | 48-mo FU . |
|---|---|---|---|---|---|---|
| No. of patients at FU∗ | 427 | 427 | 425 | 375 | 327 | 250 |
| Female (%) | 273 (64) | 273 (64) | 272 (64) | 240 (64) | 207 (63) | 155 (62) |
| Mean age (SD) | 14 (2.7) | 15 (2.7) | 16 (2.6) | 17 (2.3) | 18 (2.4) | |
| Platelet count (×109/L) at FU, median (IQR) | 15 (7-35) | 45 (21-79) | 51 (26-81) | 68 (30-115) | 76 (35-143) | 87 (43-169) |
| No. of patients with platelet count (%) | ||||||
| <20 × 109/L | 237 (56) | 88 (21) | 79 (19) | 57 (15) | 36 (11) | 18 (7) |
| 20 × 109 to 49 × 109/L | 107 (25) | 118 (28) | 110 (26) | 75 (20) | 58 (18) | 33 (13) |
| 50 × 109 to 99 × 109/L | 62 (15) | 105 (25) | 138 (32) | 103 (27) | 71 (22) | 56 (22) |
| >100 × 109/L | - | 72 (17) | 65 (15) | 104 (28) | 111 (34) | 82 (33) |
| No data on platelet count | 21 (5) | 44 (10) | 33 (8) | 36 (10) | 51 (16) | 61 (24) |
| Bleeding in the last FU period, yes (%)† | 325 (76) | 254 (59) | 215 (51) | 185 (49) | 132 (40) | 74 (30) |
| No (%)† | 74 (17) | 135 (32) | 157 (37) | 148 (39) | 140 (43) | 117 (47) |
| No data (%)† | 28 (7) | 38 (9) | 53 (12) | 42 (11) | 55 (17) | 59 (24) |
| Bleeding location‡ (% of patients with bleeding) | ||||||
| Skin | 267 (82) | 207 (81) | 168 (78) | 142 (77) | 93 (70) | 52 (70) |
| Oral | 94 (29) | 76 (30) | 53 (25) | 45 (24) | 32 (24) | 22 (30) |
| Epistaxis | 99 (30) | 70 (28) | 57 (27) | 60 (32) | 36 (27) | 18 (24) |
| Gynecological (% of women with bleeding) | 73/209 (35) | 69/171 (40) | 50/144 (35) | 59/124 (48) | 43/90 (48) | 22/52 (42) |
| Gastrointestinal and/or hematuria | 10 (3) | 12 (5) | 10 (5) | 7 (4) | 5 (4) | 7 (10) |
| ICH | 3 (1) | 3 (1) | - | 2 (1) | - | - |
| Muscle and/or joint | 4 (1) | 4 (2) | 4 (2) | 2 (1) | - | - |
| Total wet bleeding§ | 193 (59) | 156 (61) | 124 (58) | 122 (66) | 87 (66) | 49 (66) |
| No data on bleeding site | 3 | 7 | 2 | 2 | 1 | - |
| Platelet count (×109/L) at FU, median (IQR) for patients with | ||||||
| Any bleeding | 11 (6-24) | 38 (16-70) | 38 (19-65) | 45 (22-95) | 42 (20-114) | 55 (23-140) |
| Only cutaneous bleeding | 16 (7-27) | 41 (21-75) | 40 (19-65) | 52 (28-98) | 55 (25-94) | 77 (41-193) |
| Wet or internal bleeding | 10 (5-21) | 32 (14-59) | 38 (19-66) | 41 (19-95) | 40 (18-149) | 43 (19-108) |
| No bleeding | 40 (20-65) | 58 (35-94) | 68 (45-97) | 77 (52-133) | 103 (60-172) | 114 (70-179) |
| Drug treatment in the last FU period, yes (%)† | 252 (59) | 254 (59) | 210 (49) | 177 (47) | 129 (39) | 85 (34) |
| No (watch and wait) (%)† | 170 (40) | 165 (39) | 206 (48) | 192 (51) | 191 (58) | 158 (63) |
| No data (%)† | 5 (1) | 8 (2) | 9 (2) | 6 (2) | 7 (2) | 7 (3) |
| Drug used (% of treated patients) | ||||||
| Corticosteroid | 129 (51%) | 188 (74%) | 127 (60%) | 84 (47%) | 40 (31%) | 31 (36%) |
| IV immunoglobulin and/or anti-D | 163 (65%) | 141 (56%) | 80 (38%) | 71 (40%) | 51 (40%) | 34 (40%) |
| Second line and/or third line | 10 (4%) | 59 (23%) | 76 (36%) | 95 (54%) | 79 (61%) | 55 (65%) |
| No data on drug used | 0 | 6 | 3 | 2 | 0 | 1 |
| Type of second-/third-line drugs | ||||||
| Hydroxychloroquine|| | 3 | 18 | 26 | 30 | 20 | 14 |
| TPO-RA | 2 | 17 | 17 | 20 | 22 | 9 |
| Azathioprine | - | 8 | 12 | 15 | 14 | 11 |
| Anti-CD20 (rituximab) | - | 5 | 9 | 17 | 12 | 8 |
| MMF | - | 4 | 6 | 4 | 7 | 6 |
| Vinka alkaloids | - | 5 | 4 | 3 | 1 | 1 |
| Tacrolimus/sirolimus | - | 3 | - | 1 | 1 | 1 |
| Other¶ | 6 | 14 | 14 | 20 | 12 | 10 |
| New splenectomy at each FU (not cumulative) | 0 | 3 | 4 | 16 | 11 | 13 |
| Platelet transfusion | 16 | 12 | 5 | 5 | 1 | 3 |
| Red blood cell transfusion total, (% represent proportion of female) | 16 (100%) | 5 (60%) | 4 (100%) | 5 (60%) | 2 (50%) | 2 (100%) |
anti-D, anti-D immunoglobulin; FU, follow-up; IQR, interquartile range; MMF, mycophenolate mophetil; SD, standard deviation.
Patients with available FU (also including patients with incomplete information). Two patients had no information at 12 months but had chronic ITP at a later FU.
% of patients with available FU (including patients with incomplete information). Bleeding and treatment information is given for the time between 2 FUs.
Oral bleeding includes spontaneous bleeding or after dental care. We included some rare cases of conjunctival bleeding or otorrhagia in this category. Epistaxis includes also rare cases of hemoptysis. Gynecological bleeding was defined as menorrhagia and/or metrorrhagia and included also patients with ovarian cyst hemorrhage. Gastrointestinal bleeding involved upper or lower gastrointestinal bleeding. ICH included also a single case of isolated retinal bleed.
Wet bleeding included all mucosal bleeding (oral, epistaxis, urological, gynecological, and gastrointestinal). Patients with different types of wet bleeding were counted once.
Hydroxychloroquine is only specified and documented in the OBS’CEREVANCE cohort and the CARMEN-France registry. Patients of the PARC-ITP receiving hydroxychloroquine are documented as having “other drugs.” Therefore, the number of patients receiving the drug might be underestimated.
Dapsone, cyclosporine, interferon, colchicine, plasmapheresis or protein A immunoadsorption, anti-CD52, infliximab, belimumab, golimumab, vedolizumab, ustekinumab, and others.