Comparison of the 2 recently published case series of patients with hereditary TTP to a systematic review of published case reports
| Data . | UK registry3 . | International registry4 . | Case report patients . |
|---|---|---|---|
| Year of publication | 2019 | 2019 | 2019 |
| Patients | |||
| N | 73 | 120 | 155 |
| Female, n (%) | 51 (70) | 62 (52) | 89 (57) |
| Age, median (range) | |||
| Diagnosis | 24 y (newborn to 71 y) | 17 y (newborn to 70 y) | 15 y (newborn to 77 y) |
| Last follow-up | NA | NA | 24 y (2 d to 79 y) |
| First symptoms | |||
| Age, median (range) | 18 y (newborn to 67 y) | 5 y (newborn to 70 y) | 11 mo (newborn to 63 y) |
| At birth,* n (%) | NA | 30 (25) | 60 (39) |
| Exchange transfusion,† n (%) | NA | NA | 33 (21) |
| With pregnancy (% of women) | 31 (61) | NA | 20 (22) |
| TIA/stroke‡ | |||
| n (%) | 18 (25) | 37 (31) | 40 (26) |
| Age, median (range) | NA | NA | 19 y (1 d to 77 y) |
| Disability | NA | NA | 9 |
| Recurrence | NA | NA | 12 |
| MI | |||
| n (%) | 0 (0) | 5 (4) | 5 (3) |
| Age, median (range) | NA | NA | 20 y (22 mo to 50 y) |
| Death | |||
| n (%) | 5 (7) | NA | 13 (8) |
| Age, median (range) | NA | NA | 23 y (newborn to 79 y) |
| Data . | UK registry3 . | International registry4 . | Case report patients . |
|---|---|---|---|
| Year of publication | 2019 | 2019 | 2019 |
| Patients | |||
| N | 73 | 120 | 155 |
| Female, n (%) | 51 (70) | 62 (52) | 89 (57) |
| Age, median (range) | |||
| Diagnosis | 24 y (newborn to 71 y) | 17 y (newborn to 70 y) | 15 y (newborn to 77 y) |
| Last follow-up | NA | NA | 24 y (2 d to 79 y) |
| First symptoms | |||
| Age, median (range) | 18 y (newborn to 67 y) | 5 y (newborn to 70 y) | 11 mo (newborn to 63 y) |
| At birth,* n (%) | NA | 30 (25) | 60 (39) |
| Exchange transfusion,† n (%) | NA | NA | 33 (21) |
| With pregnancy (% of women) | 31 (61) | NA | 20 (22) |
| TIA/stroke‡ | |||
| n (%) | 18 (25) | 37 (31) | 40 (26) |
| Age, median (range) | NA | NA | 19 y (1 d to 77 y) |
| Disability | NA | NA | 9 |
| Recurrence | NA | NA | 12 |
| MI | |||
| n (%) | 0 (0) | 5 (4) | 5 (3) |
| Age, median (range) | NA | NA | 20 y (22 mo to 50 y) |
| Death | |||
| n (%) | 5 (7) | NA | 13 (8) |
| Age, median (range) | NA | NA | 23 y (newborn to 79 y) |
Data on 155 patients from 76 case reports are presented and compared with the data from the 2 recently published large case series from the United Kingdom Hereditary Thrombotic Thrombocytopenic Purpura Registry3 and the International Hereditary Thrombotic Thrombocytopenic Purpura Registry.4 The registries’ databases may include additional information about their patients’ individual data, but only the published data are included in this table. Among the case reports, not all clinical data described in this table were reported for each patient. For neonatal exchange transfusion, TIA/stroke, and MI, the frequency may be greater because we assumed that if these events were not reported, they did not occur. Ages are reported as median years, except where days or months are noted. We estimated that 68 of the patients (44%) we identified in the case reports were enrolled in the international registry (see appendix B in the international registry). Eleven of the 40 case report patients with TIA/stroke were included in the international registry report. None of the 5 case report patients with MI were included among the 5 patients with MI reported by the international registry. We believe that none of the patients we identified in the case reports were included in the UK registry report.
NA, data not included in these publications.
The international registry reported 30 patients with hyperbilirubinemia at birth. The case reports described hemolysis, thrombocytopenia, and neurologic abnormalities at birth in addition to hyperbilirubinemia.
Exchange transfusion indicates whole blood exchange at birth.
TIA and stroke are combined because imaging data were often not reported to provide a distinction.