Reduced-Intensity Allogeneic Stem Cell Transplantation in Children and Young Adults with Ultrahigh-Risk Pediatric Sarcomas

Baird, Kristin
Fry, Terry J.
Steinberg, Seth M.
Bishop, Michael R.
Fowler, Daniel H.
Delbrook, Cynthia P.
Humphrey, Jennifer L.
Rager, Alison
Richards, Kelly
Wayne, Alan S.
Mackall, Crystal L.

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Publication date

May 2012

DOI

10.1016/j.bbmt.2011.08.020

ISSN

1083-8791

Citation count (estimate)

Abstract

Some subsets of pediatric sarcoma patients have very poor survival rates. We sought to determine the feasibility and efficacy of allogeneic hematopoietic stem cell transplantation (alloHSCT) in pediatric sarcoma populations with <25% predicted overall survival (OS). Patients with ultrahigh-risk Ewing’s sarcoma family of tumors (ESFT), alveolar rhabdomyosarcoma, or desmoplastic small round cell tumors received EPOCH-fludarabine induction, a cyclophosphamide/fludarabine/melphalan preparative regimen, and HLA matched related peripheral blood stem cells. Thirty patients enrolled; 7 did not undergo alloHSCT because of progressive disease with diminishing performance status during induction. All 23 alloHSCT recipients experienced rapid full-donor...