- David J. Araten1,*,
- Rosario Notaro2,
- Howard T. Thaler3,
- Nancy Kernan3,
- Farid Boulad3,
- Hugo Castro-Malaspina3,
- Trudy Small3,
- Andromachi Scaradavou3,
- Heather Magnan3,
- Susan Prockop3,
- Sara Chaffee4,
- Jason Gonsky5,
- Raymond Thertulien6,
- Roberto Tarquini7 and
- Lucio Luzzatto8
- 1 NYU School of Medicine, New York VA Medical Center, USA;
- 2 University of Firenze, Istituto Tuscano Tumori, Italy;
- 3 Memorial Sloan Kettering Cancer Center, USA;
- 4 Norris Cotton Cancer Center Dartmouth-Hitchcock Medical Center, USA;
- 5 SUNY Downstate Medical Center and Kings County Hospital Center;
- 6 Cancer Centers of North Carolina, USA;
- 7 University of Florence, Firenze, Italy;
- 8 University of Firenze, Istituto Toscano Tumori, Italy
- ↵* Corresponding author; email:
Background. Thrombosis is the major risk factor for death in patients with paroxysmal nocturnal hemoglobinuria. Previous case reports indicate that venous thrombosis in patients with paroxysmal nocturnal hemoglobinuria is amenable to thrombolysis.
Design and Methods. We have reviewed the outcome of thrombolytic therapy for patients with paroxysmal nocturnal hemoglobinuria who had thromboses refractory to anticoagulation at our institutions.
Results. In this study of 41 patients who had at least one thrombotic event, we confirmed a very high incidence of recurrence despite anticoagulation. Nine patients with thrombosis were regarded as eligible for administration of intravenous tissue plasminogen activator, which was effective in reversing thrombi in all of 15 occasions in which it was given. Serious hemorrhagic complications developed in 3 cases. At last follow-up visit, of the 9 patients treated, 3 have died, and 6 were in very good to excellent condition in terms of clinical outcome and radiologic findings. The only patient in whom thrombolysis may have contributed to a fatal outcome also had complications of heparin induced thrombocytopenia with thrombosis which we diagnosed in 3 additional patients. In our review of the literature, 9 out of 15 patients treated with thrombolysis have had a good outcome.
Conclusions. Although it is associated with a significant but manageable risk of bleeding, systemic thrombolysis is a highly effective treatment in order to reverse venous thromboses in patients with paroxysmal nocturnal hemoglobinuria.
- Received June 15, 2011.
- Accepted November 15, 2011.
- Copyright © 2011, Ferrata Storti Foundation