Defibrotide Dose-escalation for SOS Post-HSCT

Inclusion Criteria:

• HCT recipients (Auto or Allograft)
• SOS/VOD as defined by Cairo/Cooke Diagnostic criteria (1) (Table 3) with either renaland/or pulmonary dysfunction as defined by Cairo/Cooke Grading criteria (1) (AppendixI).
• Unresponsive to standard defibrotide therapy as defined by at least one of thefollowing:
• Patients with SOS/VOD failing to obtain a complete response (CR) defined by Grade I orless by Cairo/Cooke Grading criteria (1) (Appendix I). This would therefore includepatients with stable disease after at least 14 days of defibrotide or partial responseafter at least 21 days of defibrotide (25mg/kg/day).
• Progressive disease defined by progression of at least one grade or more fromdiagnostic grade as defined by Cairo/Cooke Grading criteria (1) (Appendix I) followingat least 7 days of defibrotide (25mg/kg/day).
• Age 1 month - 75 years

Exclusion Criteria:

• Patients who did not receive HCT.
• Concomitant systemic anticoagulation (excluding central venous line management,fibrinolytic instillation for central venous line occlusion, management ofintermittent dialysis or ultrafiltration of CVVH).
• Active bleeding and/or hemorrhage of at least grade 2 and above.
• History of development of Grade III/IV anaphylaxis probably or directly secondary todefibrotide.
• Female patients who are pregnant or breast feeding.