Based on published reports and analyses of participant-level data, the expected survival rates 100 days after HSCT would be 21 to 31 percent for participants with severe hepatic VOD who received only supportive care or interventions other than defibrotide. Society and culture
Legal status
Defibrotide was approved in the European Union for use in treating veno-occlusive disease of the liver of people having had a bone marrow transplant in 2013; Gentium had developed it. At the end of that year, Jazz Pharmaceuticals acquired Gentium.In March 2016, the U.S. Food and Drug Administration (FDA) approved it for a similar use. Defibrotide is the first FDA-approved therapy for treatment of severe hepatic VOD, a rare and life-threatening liver condition. The FDA granted the application for defibrotide priority review status and orphan drug designation. The FDA granted approval of  Defitelio to Jazz Pharmaceuticals.Defibrotide was approved for medical use in Japan in June 2019.Defibrotide was approved for medical use in Australia in July 2020. References
Further reading
Richardson P, Aggarwal S, Topaloglu O, Villa KF, Corbacioglu S (December 2019). "Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS)". Bone Marrow Transplant. 54 (12): 1951–1962. doi:10.1038/s41409-019-0474-8. PMC 6957462. PMID 30804485. Richardson PG, Carreras E, Iacobelli M, Nejadnik B (June 2018). "The use of defibrotide in blood and marrow transplantation". Blood Adv. 2 (12): 1495–1509. doi:10.1182/bloodadvances.2017008375. PMC 6020812. PMID 29945939. External links
"Defibrotide". Drug Information Portal. U.S. National Library of Medicine.
However, dapsone can potentially cause severe side effects, meaning that sometimes steroids or other antibiotics should be used instead, although these alternative treatments are much less effective.An August 2015 review notes that dapsone is reported to be effective against generalized granuloma annulare.Dapsone has been used as a monomer in the design of dye adsorbent polymers. Contraindications and precautions
People with porphyria, anemia, cardiac disease, lung disease, HIV infection, G6PD deficiency, and liver impairment are at higher risks of adverse effects when using dapsone. Adverse effects
Hypersensitivity reactions occur in 1.4% of persons treated with dapsone, and can be fatal in medical settings with low resources. It is a form of severe cutaneous adverse reactions (SCARs) in which a SCARs disorder, primarily the DRESS syndrome or a DRESS syndrome-like reaction occurs. Blood
Hemolysis is the most prominent side-effect, occurring in about 20 % of patients treated with dapsone, although it is dose-related. It may lead to hemolytic anemia and methemoglobinemia. The side-effect is more common and severe in those with glucose-6-phosphate dehydrogenase deficiency, leading to the dapsone-containing antimalarial combination Lapdap being withdrawn from clinical use. A case of hemolysis in a neonate from dapsone in breast milk has been reported. Agranulocytosis occurs rarely when dapsone is used alone but more frequently in combination regimens for malaria prophylaxis. Abnormalities in white blood cell formation, including aplastic anemia, are rare, yet are the cause of the majority of deaths attributable to dapsone therapy.Methemoglobinemia occurs in about 15 % of patients treated with long-term dapsone at standard doses (100 mg/day).