Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure

Author:

Richardson Paul G.1,Riches Marcie L.2,Kernan Nancy A.3,Brochstein Joel A.4,Mineishi Shin5,Termuhlen Amanda M.6,Arai Sally7,Grupp Stephan A.8,Guinan Eva C.19,Martin Paul L.10,Steinbach Gideon11,Krishnan Amrita12,Nemecek Eneida R.13,Giralt Sergio14,Rodriguez Tulio15,Duerst Reggie16,Doyle John17,Antin Joseph H.1,Smith Angela18,Lehmann Leslie19,Champlin Richard19,Gillio Alfred20,Bajwa Rajinder21,D’Agostino Ralph B.22,Massaro Joseph22,Warren Diane1,Miloslavsky Maja23,Hume Robin L.24,Iacobelli Massimo25,Nejadnik Bijan26,Hannah Alison L.27,Soiffer Robert J.1

Affiliation:

1. Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA;

2. Division of Hematology/Oncology, University of North Carolina Hospitals, Bone Marrow and Stem Cell Transplant Clinic, University of North Carolina Cancer Hospital, Chapel Hill, NC;

3. Pediatric Bone Marrow Transplant Service, Memorial Sloan-Kettering Cancer Center, New York, NY;

4. Department of Pediatrics, Stem Cell Transplant Program, Cohen Children’s Medical Center, New Hyde Park, NY;

5. Division of Hematology and Oncology, University of Alabama at Birmingham, Birmingham, AL;

6. Division of Pediatric Hematology, Oncology, Blood and Marrow Transplantation, Children’s Hospital Los Angeles, Los Angeles, CA;

7. Blood and Marrow Transplantation, Stanford University Medical Center, Stanford, CA;

8. Pediatric Oncology, The Children’s Hospital of Philadelphia, Philadelphia, PA;

9. Department of Hematology/Oncology, Children’s Hospital, Boston, MA;

10. Division of Pediatric Blood and Marrow Transplant, Duke University Medical Center, Durham, NC;

11. Gastroenterology Division, University of Washington School of Medicine and Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;

12. Department of Hematology & Hematopoietic Cell Transplantation, City of Hope, Duarte, CA;

13. Pediatric Bone Marrow Transplant Program, Oregon Health and Science University, Portland, OR;

14. Adult Bone Marrow Transplant Service, Memorial Sloan-Kettering Cancer Center, New York, NY;

15. Hematology/Oncology, Loyola University Medical Center, Chicago, IL;

16. Stem Cell Transplant Program, Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL;

17. Pediatric Hematology/Oncology, CancerCare Manitoba and the University of Manitoba, Winnipeg, Manitoba, Canada;

18. Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN;

19. Department of Stem Cell Transplantation and Cellular Therapy, MD Anderson Cancer Center, The University of Texas, Houston, TX;

20. Department of Pediatrics, Hackensack University Medical Center, Hackensack, NJ;

21. Department of Hematology/Oncology/BMT, The Ohio State University/Nationwide Children’s Hospital, Columbus, OH;

22. Department of Mathematics and Statistics, Boston University, Boston, MA;

23. Biostatistics, Jazz Pharmaceuticals, Palo Alto, CA;

24. Regulatory Affairs, Jazz Pharmaceuticals, Palo Alto, CA;

25. Life Sciences, Techitra Srl, Milano, Italy;

26. Research and Clinical Development, Galena Biopharma, San Ramon, CA; and

27. Clinical Operations, Jazz Pharmaceuticals, Palo Alto, CA

Abstract

Key Points Defibrotide improves day +100 survival and CR in patients with VOD and MOF compared with a historical control. The historical control selection methodology offers a novel approach for investigation of a life-threatening orphan disease.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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