Table 2.

Principal studies reporting patients treated for endothelial syndromes

ReferencesCharacteristicsPatients, nMedian age (range), yDiagnostic criteriaOnset, days from HSCT (range)Response rate or survival, n (%)
TA-TMA
 Rituximab
  Au et al93Retrospective rituximab (375 mg/m2 per wk × 4)548 (1-56)BMT-CTN42 (18-505)CR 4/5 (80)
  Jodele et al44Prospective biological study; rituximab (375 mg/m2 per wk for 2-10 courses)63.5 (2-17)O-TMA32 (9-111)CR 4/6 (67)
 Defibrotide
  Corti et al94Retrospective (40 mg/kg p.o. daily for 30-50 d)1226 (1-55)Hemolytic anemia, antiglobulin test (−), schystocytosis, haptoglobin ↓, LDH ↑, thrombocytopenia, CNS or renal involvement47 (9-100)CR 5/12 (42)/PR 3/12 (25)
  Yeates et al95Retrospective: median dose in ped: 30 mg/kg per d (range, 25-60); in ad: 400 mg/kg per d (range, 200-800)39 (22 ped/17 ad)8 (0-17)/40 (18-63)O-TMA51 (10-215)/33 (15-152)17/22 (77)/13/17 (77)
 Vincristine
  Silva et al96Retrospective: 1-2 mg IV weekly or 0.2 mg IV daily (associated with PE and CCS)624.5 (13-35)Hemolytic anemia, antiglobulin test (−), LDH ↑, thrombocytopenia, CNS or renal involvement49 (19-110)CR 1/6 (17), PR 2/6 (33)
  Mateos et al97Retrospective: 1 mg once daily IV on days 1, 4, and 8 ± day 11718 (6-48)Anemia, LDH ↑, thrombocytopenia, schistocytosis49 (25-276)CR 6/7 (86)
 Eculizumab
  Jodele et al49Prospective: induction dose: 600 (<40 kg) or 900 mg (>40 kg); maintenance dose: 900 or 1200 every 14 d65 (2-10)O-TMA49 (6-390)CR 4/6 (67)
  de Fontbrune et al48Retrospective: induction dose: 900 mg weekly for 4 wk, followed by maintenance (1200 mg every 14 d)12 (3 ped/9 ad)39 (1.2-66)+121 (37-455)OR 6/12 (50)
  Jodele et al78Prospective: induction dose: 600 mg (<40 kg) or 900 mg (>40 kg), subsequent doses based on CH50 monitoring184.6 (2-15.2)Refined TMA30 (18-55)CR 11/18 (61)
  Bohl et al98Retrospective: 900 mg once weekly until clinical response, followed by maintenance therapy (1200 mg every 14 d)2448 (23-66)O-TMA180 (15-984)OR 13/15 (93)
IPS
 Anti-TNF
  Yanik et al80Retrospective anti-TNF+CCS (2 mg/kg per d)1521 (1-60)14 (9-87)10/15 (66)
  Yanik et al81Prospective randomized ARM A: anti-TNF+CCS (2 mg/kg per d). ARM B: placebo + CCS (2 mg/kg per d).A: 16; B: 18A: 10/16 (62); B: 12/18 (66.7)
  Yanik et al92Prospective phase 2 anti-TNF+CCS (2 mg/kg per d)3911 (1-17)20 (6-119)20/39 (71)
  Thompson et al82Retrospective anti-TNF+CCS (2 mg/kg per d)2356 (35-72)233 (104-555)10/23 (43)
  • Note the lack of studies concerning endothelial syndromes other than TA-TMA and IPS.

  • ad, adults; BMT-CTN, Blood and Marrow Transplant Clinical Trials Network criteria; CCS, corticosteroid; CH50, complement inhibition 50; CR, complete response; LDH, lactate dehydrogenase; OR, overall response; O-TMA, overall TMA criteria; PE, plasma exchange; ped, pediatrics; p.o., by mouth; PR, partial response.