Diversity of response to treatments and long-term outcomes
| Patient No. . | Age, y . | Race . | Sex . | Date . | Summary . | Patient’s presentation, clinical course, and outcome . |
|---|---|---|---|---|---|---|
| 5 | 19 | NA | F | 2001 | Rapid response to PEX, normal ADAMTS13 activity during remission, no relapse | Headache, chest pain. Platelets, 7 × 109/L; hematocrit, 24%; Cr, 0.9 mg/dL; LDH, 594 U/L. Five PEXs, no corticosteroids or other treatment. ADAMTS13, <5%, <5%; inh, 0. Remission ADAMTS13 (11 times from 2004 to 2016), 94%-100%. No relapse during 17 y. |
| 6 | 21 | H | F | 1995 | Refractory initial and relapse episodes, continuous severe ADAMTS13 deficiency during remission, no additional relapse | Seizures. Twenty-two PEXs, steroids, vincristine (ADAMTS13 not measured). Relapse, 1996: platelets, 16 × 109/L; hematocrit, 33%; Cr, 0.8 mg/dL; LDH, 612 U/L. ADAMTS13, <5%, <5%; inh, >2 BU. Recovery with 145 PEXs (221 d), steroids, vincristine, aspirin, persantin, and splenectomy. Remission ADAMTS13 (9 times from 2006 to 2016): <5%-15%; 5 times, inh, >2 BU; 4 times, inh, 0-0.7 BU. No additional relapse during 22 y. |
| 7 | 22 | W | F | 2000 | Refractory initial episode, severe ADAMTS13 deficiency during remission for 9 y, then normal ADAMTS13 activity for 7 y, no relapse | Nausea, vomiting, weakness. Platelets, 5 × 109/L; hematocrit, 21%; Cr, 0.9 mg/dL; LDH, 4296 U/L; ADAMTS13, <5%, <5%; inh, >2 BU. Seventy-four PEXs, corticosteroids, and splenectomy. Remission ADAMTS13 (4 times from 2004 to 2009): <5%-20%; (3 times from 2010 to 2016): 50%-68%. No relapse during 18 y. |
| 8 | 56 | W | F | 2000 | Refractory initial episode with cardiac arrest, ADAMTS13 activity during remission, 45%-100% for 13 y, then 6%; relapse 3 mo later. | Purpura. Platelets, 7 × 109/L; hematocrit, 23%; Cr, 0.7 mg/dL; LDH, 1135 U/L. ADAMTS13, 12%, <5%; inh, 1.2 BU. Cardiac arrest during twelfth PEX, complete recovery. Remission ADAMTS13 (8 times from 2007 to 2013), 45%-100%; 13 June 2014: ADAMTS13, 6%, 50%. 21 September 2014, relapse: platelets, 15 × 109/L; hematocrit, 21%; Cr, 0.7; LDH, 732. Hypotension during second PEX. Subsequent recovery without additional PEX. Healthy during next 3 y; ADAMTS13, 68%-89%. |
| Patient No. . | Age, y . | Race . | Sex . | Date . | Summary . | Patient’s presentation, clinical course, and outcome . |
|---|---|---|---|---|---|---|
| 5 | 19 | NA | F | 2001 | Rapid response to PEX, normal ADAMTS13 activity during remission, no relapse | Headache, chest pain. Platelets, 7 × 109/L; hematocrit, 24%; Cr, 0.9 mg/dL; LDH, 594 U/L. Five PEXs, no corticosteroids or other treatment. ADAMTS13, <5%, <5%; inh, 0. Remission ADAMTS13 (11 times from 2004 to 2016), 94%-100%. No relapse during 17 y. |
| 6 | 21 | H | F | 1995 | Refractory initial and relapse episodes, continuous severe ADAMTS13 deficiency during remission, no additional relapse | Seizures. Twenty-two PEXs, steroids, vincristine (ADAMTS13 not measured). Relapse, 1996: platelets, 16 × 109/L; hematocrit, 33%; Cr, 0.8 mg/dL; LDH, 612 U/L. ADAMTS13, <5%, <5%; inh, >2 BU. Recovery with 145 PEXs (221 d), steroids, vincristine, aspirin, persantin, and splenectomy. Remission ADAMTS13 (9 times from 2006 to 2016): <5%-15%; 5 times, inh, >2 BU; 4 times, inh, 0-0.7 BU. No additional relapse during 22 y. |
| 7 | 22 | W | F | 2000 | Refractory initial episode, severe ADAMTS13 deficiency during remission for 9 y, then normal ADAMTS13 activity for 7 y, no relapse | Nausea, vomiting, weakness. Platelets, 5 × 109/L; hematocrit, 21%; Cr, 0.9 mg/dL; LDH, 4296 U/L; ADAMTS13, <5%, <5%; inh, >2 BU. Seventy-four PEXs, corticosteroids, and splenectomy. Remission ADAMTS13 (4 times from 2004 to 2009): <5%-20%; (3 times from 2010 to 2016): 50%-68%. No relapse during 18 y. |
| 8 | 56 | W | F | 2000 | Refractory initial episode with cardiac arrest, ADAMTS13 activity during remission, 45%-100% for 13 y, then 6%; relapse 3 mo later. | Purpura. Platelets, 7 × 109/L; hematocrit, 23%; Cr, 0.7 mg/dL; LDH, 1135 U/L. ADAMTS13, 12%, <5%; inh, 1.2 BU. Cardiac arrest during twelfth PEX, complete recovery. Remission ADAMTS13 (8 times from 2007 to 2013), 45%-100%; 13 June 2014: ADAMTS13, 6%, 50%. 21 September 2014, relapse: platelets, 15 × 109/L; hematocrit, 21%; Cr, 0.7; LDH, 732. Hypotension during second PEX. Subsequent recovery without additional PEX. Healthy during next 3 y; ADAMTS13, 68%-89%. |
Date indicates the year of the patient's initial presentation with TTP. Summary represents key point for including the patient's story in this review.
H, Hispanic; NA, Native American.