Abstract 815. Richardson P, Carreras E, Pagliuca A, Ryan R, Tappe W, and Mohty M
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1 A Pooled Analysis of Survival by Defibrotide Timing of Initiation in Adults with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Following Hematopoietic Stem Cell Transplant (HSCT) Abstract 815 Richardson P, Carreras E, Pagliuca A, Ryan R, Tappe W, and Mohty M
2 Hepatic VOD/SOS Potentially life-threatening complication of HSCT conditioning With MOD (renal and/or pulmonary dysfunction), may be associated with >80% mortality post-hsct if untreated 1 Defibrotide is approved to treat: Background Hepatic VOD/SOS with renal or pulmonary dysfunction post-hsct in the US 2 and Canada 3 Severe hepatic VOD/SOS post-hsct in patients aged >1 month in the EU 4 The EBMT encourages prompt diagnosis of VOD/SOS and early intervention with defibrotide when VOD/SOS may be most amenable to treatment 5 VOD, veno-occlusive disease; SOS, sinusoidal obstruction syndrome; HSCT, hematopoietic stem cell transplantation; MOD, multi-organ dysfunction; EBMT, European Group for Blood and Bone Marrow Transplantation 1. Coppell JA, et al. Biol Blood Marrow Transplant. 2010;16(2): Defibrotide [Prescribing Information]. Palo Alto, CA: Jazz Pharmaceuticals, Inc; Health Canada. Notice - Prescription Drug List (PDL): Multiple Additions. October 19, Accessed December 3, Defibrotide [Summary of Product Characteristics]. Accessed December 7, Mohty M, et al. Bone Marrow Transplant. 2016;51(7):
3 Endothelial Cells: The Primary Target in VOD/SOS Vascular endothelium is proposed to be the primary target in transplantation-related complications including VOD/SOS and GvHD 1,2 Gene expression markers of vascular endothelial damage suggest that vwf and other endothelial stress products may have utility as markers of endothelial cell injury 3,4 Images kindly provided by H Shulman and GB McDonald GVHD, graf-versus-host disease; vwf, von Willebrand factor 1. Eissner G, et al. Blood. 2002:100(1): Richardson P, et al. Acta Haematol. 2001;106(1): Rubbia-Brandt L, et al. Mol Cancer Ther. 2011;10(4): Mir E, et al. Bone Marrow Transpl. 2017:52(9):
4 Activation and Damage to the Sinusoidal Endothelium Hepatocytes Space of Disse SEC Red blood cell Toxic metabolites Adhesion molecule Cytokines TF Kupffer (eg ICAM-1, VCAM-1) cell Heparanase TNF-α, ICAM-1, VCAM-1, PAI-1, vwf, TF, heparanase; t-pa ICAM-1, intercellular adhesion molecule 1; VCAM-1, vascular adhesion molecule 1; PAI-1, plasminogen activator inhibitor 1; TF, tissue factor, t-pa, tissue-plasminogen activator Richardson PG, et al. Expert Opin Drug Saf. 2013;12(1):
5 Gap Formation, Fibrin Deposition, and Narrowing of the Sinusoids Hepatocytes Space of Disse SEC Red blood cell TF Toxic metabolites Kupffer cell Adhesion molecule (eg ICAM-1, VCAM-1) Heparanase Cytokines vwf PAI-1 Fibrin TNF-α, ICAM-1, VCAM-1, PAI-1, vwf, TF, heparanase; t-pa Richardson PG, et al. Expert Opin Drug Saf. 2013;12(1):
6 Proposed Mechanism of Action of Defibrotide in VOD/SOS In vitro, defibrotide has demonstrated endothelial cell protection 1 VOD/SOS pathophysiology Unstable endothelium; inflammation; increased sinusoidal vascular permeability 1,2 Proposed defibrotide effect May maintain endothelial integrity 1-4 Expression of heparanase 1 Expression of heparanase 1,5,6 1. Richardson PG, et al. Expert Opin Drug Saf. 2013;12(1): Pescador R, et al. Cardiovasc Drug Rev. 2000;18: Bracht F, et al. Biochem Biophys Res Commun. 1994;200(2): Coccheri S, et al. Eur J Clin Pharmacol. 1988;35(2): Echart C, et al. Bone Marrow Transpl. 2010;45:S Mitsiades CS, et al. Clin Cancer Res. 2009;15(4):
7 Proposed Mechanism of Action of Defibrotide in VOD/SOS In vitro, defibrotide has demonstrated endothelial cell protection 1 VOD/SOS Pathophysiology Pro-thrombotic changes 1 4 Proposed defibrotide effect May reduce activation of coagulation pathway 5 and facilitate fibrinolysis 6 tissue factor 1,2 PAI-1, t-pa, vwf 1,2,7 TF expression 5,8 PAI-1, 1,9 t-pa, 1 vwf PAI-1, plasminogen activator inhibitor 1; t-pa, tissue-plasminogen activator 1. Richardson PG, et al. Expert Opin Drug Saf. 2013;12(1): Pescador R, et al. Cardiovasc Drug Rev. 2000;18: Shulman HM, et al. Gastroenterology. 1980;79(6): Shulman HM, et al. Am J Pathol. 1987;127(3): Francischetti IMB, et al. Arterioscler Thromb Vasc Biol. 2012;32(3): Echart CL, et al. Blood Coagul Fibrinolysis. 2009;20(8): Palomo M, et al. Biol Blood Marrow Transpl. 2011;17(4): Falanga A. Leukemia. 2003;17(8): Benimetskaya L, et al. Blood. 2008; 112(10)
8 Estimated Day +100 Survival With Defibrotide: Patients With VOD/SOS and MOD MOD typically defined by Renal: Creatinine 2-3 x baseline, CrCl or GFR 40% to 50%, dialysis dependence Pulmonary: O 2 saturation 90%, supplemental oxygen, or ventilator dependence CrCl, creatinine clearance; GFR, glomerular filtration rate 1. Richardson PG, et al. Biol Blood Marrow Transpl. 2010;16(7): Locatelli F, et al. Blood. 2015;126(23): Martinez BL, et al. Eur J Hosp Pharm. 2016;23(suppl 1):A27-A Richardson PG, et al. Blood. 2016;127(13): Richardson PG, et al. Int J Hematol Oncol. 2017;6(3):75-93.
9 Improved Outcomes With Earlier Treatment Retrospective study of pediatric patients (N = 45) 1 ; average days to DF initiation shorter in patients with CR Phase 2 trial of adult and pediatric patients (N = 19) 2 ; Kaplan-Meier estimated Day +100 survival higher in patients initiating DF 2 days post-diagnosis of VOD/SOS Daily From VOD/SOS Diagnosis to Start of Defibrotide Treatment, Days Probability, % Time Since Stem Cell Transplant, Days CR, complete remission; DF, defibrotide 1. Corbacioglu S, et al. Bone Marrow Transpl. 2004;33(2): Kikuta A, et al. Poster presented at EBMT annual meeting; March 18-21, 2018; Lisbon, Portugal. Abstract A233.
10 Methods Post hoc pooled analysis of HSCT patients with VOD/SOS from: International compassionate-use program (CUP; ; N = 710) 1 Expanded-access protocol (T-IND; ; N = 1154) 2 Analysis pools adults aged 18 years given defibrotide 25 mg/kg/day (N = 537) CUP (n = 125): acute leukemias, 48.8%; allograft, 83.2% T-IND (n = 412): acute leukemias, 56.8%; allograft, 93.7% VOD/SOS diagnosed by Baltimore or modified Seattle criteria or biopsy CUP also allowed hemodynamic, ultrasound, or histologic evidence 1 Defibrotide dosing: CUP: Defibrotide median dose 25 mg/kg/day for a median of 15 days T-IND: Defibrotide dose of 25 mg/kg/day for a recommended 21 days 1. Corbacioglu S, et al. Biol Blood Marrow Transpl. 2016;22(10): Kernan NA, et al. Br J Haematol. 2018;181(6):
11 Objective and Analyses Objective: Investigate impact of time from VOD/SOS diagnosis to defibrotide initiation on day +100 survival Patient population Adults (aged 18 years) with and without MOD post-hsct who received defibrotide 25 mg/kg/day and had recorded time-to-dose data Two analyses were performed, examining patients who initiated defibrotide: Before/after days 1, 2, 3, 4, 7, and 14 postdiagnosis, using Fisher s exact test On a particular day (0, 1, 2, 3, 4, 5, 6, 7, 8-14, and 15 after diagnosis), using Cochran-Armitage test for trend across days Causes of delay were not assessed
12 Demographic and Clinical Characteristics Variable All VOD/SOS (n = 537 a ) VOD/SOS with MOD (n = 301) VOD/SOS without MOD (n = 236) Male, n (%) 302 (56.2) 162 (53.8) 140 (59.3) Mean age at HSCT (SD) (14.9) (13.8) (16.1) Most common ( 15%) primary diagnosis, n (%) Acute myelogenous leukemia Acute lymphocytic/lymphoblastic leukemia 187 (34.8) 107 (20.0) 117 (38.9) 57 (19.0) 70 (29.7) 50 (21.1) Most common ( 15%) GVHD prophylaxis, n (%) Tacrolimus Cyclosporine 316 (58.8) 132 (24.6) 179 (59.5) 78 (25.9) 137 (58.1) 54 (22.9) Median (range) days from HSCT to VOD/SOS diagnosis a 16 (0-493) 17 (0-493) 15 (1-318) Allograft transplant 490 (91.2) 280 (93.0) 210 (89.0) a 3 patients did not have days from transplant to dosing recorded (1 with MOD; 2 without MOD)
13 Analysis 1: Before/After Specific Days Day +100 Survival Rate, % All Adult Patients (n = 534 a ) Adult Patients with MOD (n = 300 b ) ** ** * * Day +100 Survival Rate, % > > > > > > Treatment Initiation Day n surviving > > > > > > Treatment Initiation Day n surviving Surviving Patients Treated by Cutoff Day Surviving Patients Treated After Cutoff Day *Nominal P.05; **Nominal P.01 by Fisher s exact test, calculated using known alive and dead patients only. a 26 subjects with negative time adjusted to 0 day. 3 subjects excluded due to missing time to dosing. b For VOD/SOS with MOD, the latter of VOD/SOS data and MOD date was used as the diagnosis date; 20 subjects with negative time adjusted to 0 day.
14 Analysis 2: On Specific Days All Adult Patients (n = 534 a ) Adult Patients With MOD (n = 300 b ) Day +100 Survival Rate, % P =.011 * 90 P =.048 * 80 Day +100 Survival Rate, % Treatment Initiation Day Day Treatment Initiation Day Day n = n = *Two-sided nominal P-values from Cochran-Armitage test for trend. Bars represent 95% confidence intervals. a 14 patients with negative time to dosing adjusted to have 0 day; 3 patients excluded due to missing time to dosing. b 13 patients with negative time to dosing adjusted to 0 day; 1 patient excluded due to missing time to dosing.
15 Safety: Summary of Adverse Events Safety information for the CUP and T-IND were not pooled Category of AE T-IND Analysis Population (n = 4 12) 1 n (%) CUP Analysis Population (n = 125) 2 n (%) Any TEAE ( 1) 322 (78.2) 73 (58.4) Serious TEAE 244 (59.2) 71 (56.8) TEAE leading to discontinuation 134 (32.5) 11 (8.8) TEAE leading to death 193 (46.8) 68 (54.4) Treatment-related TEAE a 84 (20.4) 12 (9.6) Hemorrhage b 124 (30.1) 15 (12.0) Treatment-related AEs in 2% of patients: T-IND: gastrointestinal hemorrhage, 3.6%; epistaxis, 3.4%; hematuria, pulmonary hemorrhage, and hypotension, 2.2%. 1 CUP: gastrointestinal hemorrhage, 2.4% 2 a Related TEAEs are events with a relationship to study medication of definitely, probably, or possibly related. b Includes all prespecified haemorrhage categories. 1. Study data on file, Jazz Pharmaceuticals. 2. Corbacioglu S, et al. Biol Blood Marrow Transpl. 2016;22(10):
16 Safety: Treatment-Emergent AEs ( 5%) AE, n (%) T-IND (n = 412) 1 CUP (n = 125) 2 MOF, new or worsening 70 (17.0) 30 (24.0) Hypotension 65 (15.8) NA Progression of VOD/SOS 57 (13.8) 17 (13.6) Diarrhea 45 (10.9) NA Respiratory failure 40 (9.7) 2 (1.6) Nausea 39 (9.5) NA Renal failure 36 (8.7) NA Vomiting 35 (8.5) NA Pyrexia 30 (7.3) 2 (1.6) Tachycardia 28 (6.8) NA AE, n (%) T-IND (n = 412) 1 CUP (n = 125) 2 Confusional state 27 (6.6) NA Sepsis 27 (6.6) 12 (9.6) Epistaxis 25 (6.1) NA Dyspnea 25 (6.1) NA Cough 23 (5.6) NA Constipation 23 (5.6) NA Abdominal pain 22 (5.3) NA Hypoxia 22 (5.3) NA Gastrointestinal hemorrhage 21 (5.1) 4 (3.2) Graft-vs-host disease 13 (3.2) 11 (8.8) 1. Study data on file, Jazz Pharmaceuticals. 2. Corbacioglu S, et al. Biol Blood Marrow Transpl. 2016;22(10):
17 Conclusions Consistent with studies showing improved outcomes with earlier defibrotide These results suggest outcomes may be improved with prompt defibrotide initiation following VOD/SOS diagnosis irrespective of MOD status Safety profile consistent with other defibrotide VOD/SOS studies in adults Results support further evaluation of defibrotide s role in the adult HSCT population Current studies of prevention of VOD/SOS and GVHD ongoing Future directions include thrombotic microangiopathy in transplant, other settings of endothelial cell injury in cellular therapy (eg, CAR T) and immuno-oncology CAR-T, chimeric antigen receptor T-cell therapy
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