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Blood, 12 November 2009, Vol. 114, No. 20, pp. 4354-4360. Prepublished online as a Blood First Edition Paper on September 10, 2009; DOI 10.1182/blood-2009-05-224840. This Article Full Text Full Text (PDF) Supplemental Table All Versions of this Article: blood-2009-05-224840v1 114/20/4354    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via CrossRef Google Scholar Articles by Jacobsohn, D. A. Articles by Vogelsang, G. B. PubMed PubMed Citation Articles by Jacobsohn, D. A. Articles by Vogelsang, G. B. Related Collections Transplantation Free Research Articles Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  CLINICAL TRIALS AND OBSERVATIONS Evaluation of pentostatin in corticosteroid-refractory chronic graft-versus-host disease in children: a Pediatric Blood and Marrow Transplant Consortium study David A. Jacobsohn1, Andrew L. Gilman2, Alfred Rademaker1, Brittan Browning3, Michael Grimley4, Leslie Lehmann5, Eneida R. Nemecek6, Kimberly Thormann1, Kirk R. Schultz7, and Georgia B. Vogelsang8 1 Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL; 2 Levine Children's Hospital, Charlotte, NC; 3 University of Utah Health Sciences Center, Salt Lake City; 4 Methodist Children's Hospital, San Antonio, TX; 5 Dana-Farber Cancer Institute, Boston, MA; 6 Oregon Health & Science University, Portland; 7 University of British Columbia, Vancouver, BC; and 8 Sidney Kimmel Cancer Center of Johns Hopkins University, Baltimore, MD There is no standard therapy for steroid-refractory chronic graft-versus-host disease (GVHD). This problem is particularly daunting in children with chronic GVHD, whereby the effects of the disease and its treatment may impair normal growth and development. Children are also particularly vulnerable to failure and/or toxicity of therapy; for example, joint contractures or joint damage may result in life-long disability. The Pediatric Blood and Marrow Transplant Consortium performed a phase 2 trial of pentostatin for steroid-refractory chronic GVHD in 51 children (median age, 9.8 years) from 24 institutions. Overall response was 53% (95% confidence interval, 40%-64%), with a response of 59% (95% confidence interval, 42%-75%) in sclerosis. Thirteen subjects (25%) had toxicity requiring them to stop pentostatin. The drug had a significant steroid-sparing effect in those that responded. A trend was also observed toward increased survival at 3 years in responders versus nonresponders (69% vs 50%; P = .06). The intravenous administration of the drug ensures compliance in a patient group in which oral therapy is difficult to monitor. Pentostatin has activity in refractory chronic GVHD in children, and future studies, including treatment of children newly diagnosed with high-risk chronic GVHD, are warranted. The trial was registered at www.Clinicaltrials.gov as #NCT00144430 [ClinicalTrials.gov] . CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this?

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