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Pediatric Transplants

Stem cell transplantation in children with infantile osteopetrosis is associated with a high incidence of VOD, which could be prevented with defibrotide

Bone Marrow Transplantation volume 38, pages 547–553 (2006)Cite this article

Abstract

Malignant infantile osteopetrosis (MIOP) is a rare hereditary disorder of osteoclast function, which can be reversed by hematopoietic stem cell transplantation (SCT). We observed a high incidence of hepatic veno-occlusive disease (VOD) in transplanted patients and explored the prevention of this complication by using defibrotide (DF) as a prophylaxis. Twenty children with MIOP were consecutively transplanted in our center between 1996 and 2005. Eleven of these patients were transplanted between 1996 and 2001 and experienced an overall incidence of VOD of 63.6% (7/11). VOD was severe in three patients and one patient succumbed to VOD-related multi-organ failure. Owing to this very high incidence of VOD, DF prophylaxis was initiated in nine patients consecutively transplanted between 2001 and 2005. In this group, only one patient (11.1%) was diagnosed with moderate VOD. We report here a very high risk in patients with MIOP to develop VOD after transplantation. Prophylactic DF was implemented in our current transplant protocol and reduced the VOD rate significantly in this high-risk population.

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Authors and Affiliations

  1. Department of Pediatrics, University of Ulm, Ulm, Germany

    S Corbacioglu, M Hönig, G Lahr, S Stöhr, G Berry, W Friedrich & A S Schulz

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  1. S Corbacioglu
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  2. M Hönig
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  3. G Lahr
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  7. A S Schulz
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Correspondence to S Corbacioglu.

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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 38, 547–553 (2006). https://doi.org/10.1038/sj.bmt.1705485

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