Evaluation of pentostatin in corticosteroid-refractory chronic graft-versus-host disease in children: A Pediatric Blood and Marrow Transplant Consortium study

David A. Jacobsohn, Andrew L. Gilman, Alfred Rademaker, Brittan Browning, Michael Grimley, Leslie Lehmann, Eneida Nemecek, Kimberly Thormann, Kirk R. Schultz, Georgia B. Vogelsang

Research output: Contribution to journalArticle

28 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)4354-4360
Number of pages7
JournalBlood
Volume114
Issue number20
DOIs
StatePublished - Nov 12 2009

Fingerprint

Pentostatin
Transplants
Pediatrics
Graft vs Host Disease
Grafts
Refractory materials
Adrenal Cortex Hormones
Blood
Bone Marrow
Steroids
Joints
Toxicity
Confidence Intervals
Therapeutics
Sclerosis
Contracture
Growth and Development
Pharmaceutical Preparations
Intravenous Administration
Compliance

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Jacobsohn, D. A., Gilman, A. L., Rademaker, A., Browning, B., Grimley, M., Lehmann, L., ... Vogelsang, G. B. (2009). Evaluation of pentostatin in corticosteroid-refractory chronic graft-versus-host disease in children: A Pediatric Blood and Marrow Transplant Consortium study. Blood, 114(20), 4354-4360. https://doi.org/10.1182/blood-2009-05-224840

Evaluation of pentostatin in corticosteroid-refractory chronic graft-versus-host disease in children : A Pediatric Blood and Marrow Transplant Consortium study. / Jacobsohn, David A.; Gilman, Andrew L.; Rademaker, Alfred; Browning, Brittan; Grimley, Michael; Lehmann, Leslie; Nemecek, Eneida; Thormann, Kimberly; Schultz, Kirk R.; Vogelsang, Georgia B.

In: Blood, Vol. 114, No. 20, 12.11.2009, p. 4354-4360.

Research output: Contribution to journalArticle

Jacobsohn, DA, Gilman, AL, Rademaker, A, Browning, B, Grimley, M, Lehmann, L, Nemecek, E, Thormann, K, Schultz, KR & Vogelsang, GB 2009, 'Evaluation of pentostatin in corticosteroid-refractory chronic graft-versus-host disease in children: A Pediatric Blood and Marrow Transplant Consortium study', Blood, vol. 114, no. 20, pp. 4354-4360. https://doi.org/10.1182/blood-2009-05-224840
Jacobsohn, David A. ; Gilman, Andrew L. ; Rademaker, Alfred ; Browning, Brittan ; Grimley, Michael ; Lehmann, Leslie ; Nemecek, Eneida ; Thormann, Kimberly ; Schultz, Kirk R. ; Vogelsang, Georgia B. / Evaluation of pentostatin in corticosteroid-refractory chronic graft-versus-host disease in children : A Pediatric Blood and Marrow Transplant Consortium study. In: Blood. 2009 ; Vol. 114, No. 20. pp. 4354-4360.
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