Published online October 30, 2006
PEDIATRICS Vol. 118 No. 6 December 2006, pp. e1896-e1899 (doi:10.1542/peds.2006-0833)
This Article
Right arrow Full Text
Right arrow Full Text (PDF)
Right arrow Submit a response
Right arrow Alert me when this article is cited
Right arrow Alert me when eLetters are posted
Right arrow Alert me if a correction is posted
Services
Right arrow E-mail this article to a friend
Right arrow Similar articles in this journal
Right arrow Similar articles in PubMed
Right arrow Alert me to new issues of the journal
Right arrow Add to My File Cabinet
Right arrow Download to citation manager
Right arrowRequest Permissions
Citing Articles
Right arrow Citing Articles via CrossRef
Right arrow Citing Articles via Google Scholar
Google Scholar
Right arrow Articles by Rand, E. B.
Right arrow Articles by Bloomer, J. R.
Right arrow Search for Related Content
PubMed
Right arrow PubMed Citation
Right arrow Articles by Rand, E. B.
Right arrow Articles by Bloomer, J. R.
Related Collections
Right arrow Blood
Social Bookmarking
Add to CiteULike   Add to Connotea   Add to Del.icio.us   Add to Digg   Add to Facebook   Add to Reddit   Add to Technorati   Add to Twitter  
What's this?

EXPERIENCE & REASON

Sequential Liver and Bone Marrow Transplantation for Treatment of Erythropoietic Protoporphyria

Elizabeth B. Rand, MDa, Nancy Bunin, MDb, William Cochran, MDc, Eduardo Ruchelli, MDd, Kim M. Olthoff, MDe and Joseph R. Bloomer, MDf

a Divisions of Gastroenterology
b Oncology, Department of Pediatrics
e Division of Transplantation, Department of Surgery
d Department of Pathology, University of Pennsylvania, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania
c Division of Gastroenterology, Department of Pediatrics, Geisinger Medical Center, Danville, Pennsylvania
f Division of Gastroenterology, Department of Medicine, University of Alabama, Tuscaloosa, Alabama

ABSTRACT

Erythropoietic protoporphyria is a disorder of heme synthesis in which deficient ferrochelatase activity leads to excess production and biliary excretion of protoporphyrin. The main clinical features, photosensitivity and hepatobiliary disease that may progress to liver failure, are caused by the toxicity of protoporphyrin. Liver transplantation has been used to treat liver failure in erythropoietic protoporphyria, but excess production of protoporphyrin by the bone marrow continues causing recurrence of liver disease in the majority of patients. This is the first report of successful sequential liver and bone marrow transplantation in a patient with liver failure as a result of erythropoietic protoporphyria. This combination corrected the severe phenotype, resolving the severe photosensitivity and halting erythropoietic protoporphyria associated liver graft injury. Splenectomy seemed to facilitate the successful bone marrow transplant.

Key Words: liver transplantation • bone marrow transplantation • porphyria

Abbreviations: EPP, erythropoietic protoporphyria • FECH, ferrochelatase • OLT, orthotopic liver transplantation • ERCP, endoscopic retrograde cholangiopancreatography • HSC, hematopoietic stem cells

Accepted Jun 20, 2006.


Add to CiteULike CiteULike   Add to Connotea Connotea   Add to Del.icio.us Del.icio.us   Add to Digg Digg   Add to Facebook Facebook   Add to Reddit Reddit   Add to Technorati Technorati   Add to Twitter Twitter    What's this?