Sole Upfront Therapy with Beclomethasone and Budesonide for Upper Gastrointestinal Acute Graft-versus-Host Disease

Chiara Frairia, Maura Nicolosi, Jamie Shapiro, Jongphil Kim, Brian C. Betts, Hugo F. Fernandez, Frederick L. Locke, Asmita Mishra, Taiga Nishihori, Jose Leonel Ochoa-Bayona, Lia Perez, Joseph Pidala, Claudio Anasetti

Research output: Contribution to journalArticlepeer-review


Systemic glucocorticoids remain the standard treatment for gastrointestinal (GI) acute graft-versus-host disease (aGVHD) despite their toxicity and incomplete efficacy. Controlled trials have tested poorly absorbable steroids as adjuncts with systemic glucocorticoids, but only small case series have reported treatment with poorly absorbed beclomethasone dipropionate (BDP) and budesonide (BUD) alone. Our team has adopted the practice of administering BDP or BDP+BUD without systemic glucocorticoids as first-line therapy for isolated upper GI (UGI) aGVHD. We report results in 76 patients treated with BDP alone and in 81 patients treated with BDP+BUD, with allocation by physician choice. Almost all patients received peripheral blood stem cells (92%) from a fully HLA-matched related or unrelated donor (80%) after myeloablative conditioning (76%) for acute leukemia (49%), myelodysplastic syndrome (17%), non-Hodgkin lymphoma (14%), or another hematopoietic disorders (20%). After 28 days of treatment with BDP, 46% of the patients had a complete response (CR) and 10% had a partial response (PR); after 200 days, 61 (80%) patients were alive, 34% maintained a CR, and 3% maintained a PR, whereas 53% required additional immunosuppression (IS). After 28 days of treatment with BDP+BUD, 67% had a CR and 10% a PR; after 200 days, 74 (91%) patients were alive, 46% maintained a CR, and 2% maintained a PR, whereas 43% required additional IS. Among the entire cohort of 157 patients, 66 (42%) were treated successfully without systemic glucocorticoids. This study reports the efficacy of poorly absorbable steroids alone for patients with isolated UGI aGVHD. Prospective trials should test for the potential advantages of BDP and BUD use over systemic glucocorticoids.

Original languageEnglish (US)
Pages (from-to)1303-1311
Number of pages9
JournalBiology of Blood and Marrow Transplantation
Issue number7
StatePublished - Jul 2020
Externally publishedYes

Bibliographical note

Funding Information:
Financial disclosure: This study was supported in part by grant P30 CA076292 from the National Cancer Institute, National Insitutes of Health, Bethesda, MD.

Publisher Copyright:
© 2020 American Society for Transplantation and Cellular Therapy


  • Beclomethasone
  • Budesonide
  • Hematopoietic stem cell transplantation
  • Upper gastrointestinal acute GVHD

PubMed: MeSH publication types

  • Journal Article
  • Research Support, N.I.H., Extramural


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