Successful Treatment of Transplant Associated Thrombotic Microangiopathy (TA-TMA) with Low Dose Defibrotide
- 118 Downloads
Transplant associated microangiopathy (TA-TMA) is a potentially serious complication of stem cell transplantation. Though stopping calcineurin/mTOR inhibitor is the first step in managing TA-TMA, this is not always adequate. The pathophysiology of TA-TMA is different from microangiopathy seen in other settings. Many drugs have been used in TA-TMA with modest responses. Defibrotide has been explored in TA-TMA in the past with good results. However, its availability is erratic and cost of therapy very high. Hence its routine use in low middle income country (LMIC) is financially demanding. We report the use of low dose defibrotide safely and successfully in this case series. This is pertinent more to LMIC’s and warrants prospective evaluation.
KeywordsDefibrotide Allogeneic Transplant Microangiopathy
Compliance with Ethical Standards
Conflict of interest
The authors declare no conflict of interest.
All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.
Informed consent was obtained from all individual participants included in the study.
- 12.Hahn T, Alam AR, Lawrence D, Ford L, Baer MR, Bambach B et al (2004) Thrombotic microangiopathy after allogeneic blood and marrow transplantation is associated with dose-intensive myeloablative conditioning regimens, unrelated donor, and methylprednisolone T-cell depletion. Transplantation 78(10):1515–1522CrossRefPubMedGoogle Scholar
- 17.Richardson PG, Murakami C, Jin Z, Warren D, Momtaz P, Hoppensteadt D et al (2002) Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome. Blood 100(13):4337–4343CrossRefPubMedGoogle Scholar
- 19.Herper M (2010) The world’s most expensive drugs. Forbes. https://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rarediseases.html#4ef154795e10