Vol 52, No 2 (2021)
Clinical vignette
Published online: 2021-04-29

open access

Page views 441
Article views/downloads 478
Get Citation

Connect on Social Media

Connect on Social Media

Defibrotide in preventing veno-occlusive disease after hematopoietic stem cell transplantation in children with osteopetrosis

Justyna Miśkiewicz-Bujna1, Izabella Miśkiewicz-Migoń1, Monika Rosa1, Karolina Liszka1, Krzysztof Kałwak1
Acta Haematol Pol 2021;52(2):140-141.

Abstract

Not available

Article available in PDF format

View PDF Download PDF file

References

  1. Corbacioglu S, Carreras E, Ansari M, et al. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: a new classification from the European society for blood and marrow transplantation. Bone Marrow Transplant. 2017; 53(2): 138–145.
  2. Penna S, Capo V, Palagano E, et al. One disease, many genes: implications for the treatment of osteopetroses. Front Endocrinol (Lausanne). 2019; 10: 85.
  3. Palagano E, Menale C, Sobacchi C, et al. Genetics of osteopetrosis. Curr Osteoporos Rep. 2018; 16(1): 13–25.
  4. Orchard PJ, Fasth AL, Le Rademacher J, et al. Hematopoietic stem cell transplantation for infantile osteopetrosis. Blood. 2015; 126(2): 270–276.
  5. Shadur B, Zaidman I, NaserEddin A, et al. Successful hematopoietic stem cell transplantation for osteopetrosis using reduced intensity conditioning. Pediatr Blood Cancer. 2018; 65(6): e27010.
  6. Driessen GJA, Gerritsen EJA, Fischer A, et al. Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report. Bone Marrow Transplant. 2003; 32(7): 657–663.
  7. Corbacioglu S, Hönig M, Lahr G, et al. Stem cell transplantation in children with infantile osteopetrosis is associated with a high incidence of VOD, which could be prevented with defibrotide. Bone Marrow Transplant. 2006; 38(8): 547–553.
  8. Qureshi A, Marshall L, Lancaster D. Defibrotide in the prevention and treatment of veno-occlusive disease in autologous and allogeneic stem cell transplantation in children. Pediatr Blood Cancer. 2008; 50(4): 831–832.
  9. Baker DE, Demaris K. Defibrotide. Hosp Pharm. 2016; 51(10): 847–854.
  10. Corbacioglu S, Schulz A, Sedlacek P, et al. Defibrotide for prophylaxis of hepatic veno-occlusive disease in pediatric hematopoietic stem cell transplantation: subanalysis data from an open-label, phase III, randomized trial. Blood. 2015; 126(23): 4310–4310.